title: Pivotal, Open-label, Randomized Study of Radiosurgery With or Without Tumor Treating Fields (TTFields) for 1-10 Brain Metastases From Non-small Cell Lung Cancer (NSCLC).

summary: The study is a prospective, randomized controlled phase III trial, to test the efficacy, safety and neurocognitive outcomes of advanced NSCLC patients, following stereotactic radiosurgery (SRS) for 1 inoperable brain metastasis or 2-10 brain metastases, treated with NovoTTF-200M and supportive treatment compared to supportive treatment alone. The device is an experimental, portable, battery operated device for chronic administration of alternating electric fields (termed TTFields or TTF) to the region of the malignant tumor, by means of surface, insulated electrode arrays.

ReferenceType: background

ReferenceCitation: Kirson ED, Gurvich Z, Schneiderman R, Dekel E, Itzhaki A, Wasserman Y, Schatzberger R, Palti Y. Disruption of cancer cell replication by alternating electric fields. Cancer Res. 2004 May 1;64(9):3288-95.

ReferenceCitation: Kirson ED, Giladi M, Gurvich Z, Itzhaki A, Mordechovich D, Schneiderman RS, Wasserman Y, Ryffel B, Goldsher D, Palti Y. Alternating electric fields (TTFields) inhibit metastatic spread of solid tumors to the lungs. Clin Exp Metastasis. 2009;26(7):633-40. doi: 10.1007/s10585-009-9262-y. Epub 2009 Apr 23.

ReferenceCitation: Stupp R, Wong ET, Kanner AA, Steinberg D, Engelhard H, Heidecke V, Kirson ED, Taillibert S, Liebermann F, Dbalý V, Ram Z, Villano JL, Rainov N, Weinberg U, Schiff D, Kunschner L, Raizer J, Honnorat J, Sloan A, Malkin M, Landolfi JC, Payer F, Mehdorn M, Weil RJ, Pannullo SC, Westphal M, Smrcka M, Chin L, Kostron H, Hofer S, Bruce J, Cosgrove R, Paleologous N, Palti Y, Gutin PH. NovoTTF-100A versus physician's choice chemotherapy in recurrent glioblastoma: a randomised phase III trial of a novel treatment modality. Eur J Cancer. 2012 Sep;48(14):2192-202. doi: 10.1016/j.ejca.2012.04.011. Epub 2012 May 18.

ReferenceCitation: Giladi M, Schneiderman RS, Voloshin T, Porat Y, Munster M, Blat R, Sherbo S, Bomzon Z, Urman N, Itzhaki A, Cahal S, Shteingauz A, Chaudhry A, Kirson ED, Weinberg U, Palti Y. Mitotic Spindle Disruption by Alternating Electric Fields Leads to Improper Chromosome Segregation and Mitotic Catastrophe in Cancer Cells. Sci Rep. 2015 Dec 11;5:18046. doi: 10.1038/srep18046.

ReferenceCitation: Giladi M, Weinberg U, Schneiderman RS, Porat Y, Munster M, Voloshin T, Blatt R, Cahal S, Itzhaki A, Onn A, Kirson ED, Palti Y. Alternating electric fields (tumor-treating fields therapy) can improve chemotherapy treatment efficacy in non-small cell lung cancer both in vitro and in vivo. Semin Oncol. 2014 Oct;41 Suppl 6:S35-41. doi: 10.1053/j.seminoncol.2014.09.006. Epub 2014 Sep 8.

ReferenceCitation: Kirson ED, Dbalý V, Tovarys F, Vymazal J, Soustiel JF, Itzhaki A, Mordechovich D, Steinberg-Shapira S, Gurvich Z, Schneiderman R, Wasserman Y, Salzberg M, Ryffel B, Goldsher D, Dekel E, Palti Y. Alternating electric fields arrest cell proliferation in animal tumor models and human brain tumors. Proc Natl Acad Sci U S A. 2007 Jun 12;104(24):10152-7. Epub 2007 Jun 5.

ReferenceCitation: Stupp R, Taillibert S, Kanner AA, Kesari S, Steinberg DM, Toms SA, Taylor LP, Lieberman F, Silvani A, Fink KL, Barnett GH, Zhu JJ, Henson JW, Engelhard HH, Chen TC, Tran DD, Sroubek J, Tran ND, Hottinger AF, Landolfi J, Desai R, Caroli M, Kew Y, Honnorat J, Idbaih A, Kirson ED, Weinberg U, Palti Y, Hegi ME, Ram Z. Maintenance Therapy With Tumor-Treating Fields Plus Temozolomide vs Temozolomide Alone for Glioblastoma: A Randomized Clinical Trial. JAMA. 2015 Dec 15;314(23):2535-43. doi: 10.1001/jama.2015.16669.

ReferenceCitation: Pless M, Droege C, von Moos R, Salzberg M, Betticher D. A phase I/II trial of Tumor Treating Fields (TTFields) therapy in combination with pemetrexed for advanced non-small cell lung cancer. Lung Cancer. 2013 Sep;81(3):445-450. doi: 10.1016/j.lungcan.2013.06.025. Epub 2013 Jul 23.

ReferenceCitation: Lin NU, Lee EQ, Aoyama H, Barani IJ, Barboriak DP, Baumert BG, Bendszus M, Brown PD, Camidge DR, Chang SM, Dancey J, de Vries EG, Gaspar LE, Harris GJ, Hodi FS, Kalkanis SN, Linskey ME, Macdonald DR, Margolin K, Mehta MP, Schiff D, Soffietti R, Suh JH, van den Bent MJ, Vogelbaum MA, Wen PY; Response Assessment in Neuro-Oncology (RANO) group. Response assessment criteria for brain metastases: proposal from the RANO group. Lancet Oncol. 2015 Jun;16(6):e270-8. doi: 10.1016/S1470-2045(15)70057-4. Epub 2015 May 27. Review.

ReferenceCitation: Brozova H, Lucas A, Salmaggi A, Vymazal J. COMET: A phase II randomized study of TTFields versus supportive care in non-small cell lung cancer patients with 1-5 brain metastases - initial safety results. Neuro Oncol. 2015 Nov; 17 (suppl 5): v46. doi:10.1093/neuonc/nov208.6

lead_sponsor: NovoCure GmbH

location_country:
Croatia
Poland
Hong Kong
Serbia
Spain
Italy
Austria
Canada
Hungary
United States
France
China
Israel
Germany
Bulgaria

location_facility: Abbott Northwestern Hospital - Givens Brain Tumor Center

location_facility: Geza Hetenyi Hospital-Clinic of Jasz-Nagykun-Szolnok County

location_facility: Le CIUSSS de I'Est-de-L'ile de Montreal - Hôpital Maisonneuve Rosemont

location_facility: Center for Neuro-oncology, Neurosurgery Clinic, Clinical Center of Serbia

location_facility: Wake Forest University Baptist Medical Center (WFUBMC) - Comprehensive Cancer Center

location_facility: Azienda Socio Sanitaria Territoriale di Lecco

location_facility: Memorial Health University Medical Center

location_facility: The First Affiliated Hospital of Guangdong Pharmaceutical University

location_facility: Grandview Medical Center - Cancer Center

location_facility: University Hospital of Messina AOU Policlinico "G. Martino"

location_facility: Walter Reed National Military Medical Center

location_facility: Mischer Neuroscience Associates - Texas Medical Center

location_facility: A.O.S.G. Moscati Azienda Ospedaliera di Rilievo Nazionale e di Alta Specialità

location_facility: Medical University of South Carolina- Hollings Cancer Center

location_facility: University of Alabama at Birmingham Comprehensive Cancer Center

location_facility: The First Affiliated Hospital of Soochow University

location_facility: Marek Harat Private Practice, Neurosurgery and Radiation Oncology

location_facility: Universitätsklinikum Halle (Saale), Klinik für Innere Medizin IV, Hämatologie / Onkologie

location_facility: Centre Hospitalier de l'Universite de Montreal (CHUM)

location_facility: Tianjin Medical University Cancer Institute and Hospital

location_facility: A.O.U Città della Salute e della Scienza di Torino

location_facility: UNC - Lineberger Comprehensive Cancer Center

location_facility: Dr. Senckenbergisches Institut für Neuroonkologie, Zentrum der Neurologie und Neurochirurgie

location_facility: University of Louisville-James Graham Brown Cancer Center

location_facility: Klinik für Radioonkologie und Strahlentherapie der Charité Universitätsmedizin Berlin Campus Charité Virchow-Klinikum

location_facility: (CHUS) Centre Hospitalier Universitaire de Sherbrooke, Service de Neurochirurgie

location_facility: University Multiprofile Hospital for Active Treatment Sofiamed, Department of Medical Oncology

location_facility: National Koranyi Institute of Tb and Pulmonology

location_facility: University of Minnesota Medical Center (UMMC) - Fairview - Masonic Cancer Clinic

location_facility: The Second Affiliated Hospital Of Xingtai Medical College

location_facility: First Affiliated Hospital of Xi'an Jiaotong University

location_facility: Cancer Hospital Chinese Academy of Medical Sciences

location_facility: John Nasseff Neuroscience Institute ANW Brain Tumor Center

location_facility: University of Mississippi Medical Center

location_facility: UMHAT Sv. Ivan Rilski EAD, Department of Medical Oncology

location_facility: The First Affiliated Hospital of Xiamen University

location_facility: The First Hospital of China Medical University

location_facility: The Center for Cancer Prevention and Treatment at St. Joseph Hospital of Orange

location_facility: The University of Hong Kong-Shenzhen Hospital

location_facility: Szpital Kliniczny Przemienienia Pańskiego Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu

location_facility: Ellis Fischel Cancer Center, University of Missouri Healthcare

location_facility: The IRCCS Carlo Besta Neurological Institute Foundation

location_facility: Onkologiai Osztaly, Balassa Janos Korhaz

location_facility: Baylor Scott & White Medical Center - Temple


status: Completed

primary_purpose: Treatment

intervention_type: Drug

acronym: Gemini 2

title: A Phase III, Randomised, Double-blind, Multicentre, Parallel-group, Non-inferiority Study Evaluating the Efficacy, Safety, and Tolerability of Dolutegravir Plus Lamivudine Compared to Dolutegravir Plus Tenofovir/Emtricitabine in HIV-1-infected Treatment-naïve Adults

study_completion_date_type: Actual

lead_sponsor: ViiV Healthcare

type: EudraCT Number

source: euctr

design_allocation: Randomized

study_start_date_type: Actual

study_type: Interventional

ReferenceCitation: Cahn P, Madero JS, Arribas JR, Antinori A, Ortiz R, Clarke AE, Hung CC, Rockstroh JK, Girard PM, Sievers J, Man C, Currie A, Underwood M, Tenorio AR, Pappa K, Wynne B, Fettiplace A, Gartland M, Aboud M, Smith K; GEMINI Study Team. Dolutegravir plus lamivudine versus dolutegravir plus tenofovir disoproxil fumarate and emtricitabine in antiretroviral-naive adults with HIV-1 infection (GEMINI-1 and GEMINI-2): week 48 results from two multicentre, double-blind, randomised, non-inferiority, phase 3 trials. Lancet. 2019 Jan 12;393(10167):143-155. doi: 10.1016/S0140-6736(18)32462-0. Epub 2018 Nov 9. Erratum in: Lancet. 2018 Nov 28;:.

ReferenceType: derived

ReferenceCitation: Ait-Khaled M, Sierra Madero J, Estrada V, Gulminetti R, Hagins D, Tsai HC, Man C, Sievers J, Grove R, Zolopa A, Wynne B, van Wyk J. Impact of treatment adherence on efficacy of dolutegravir plus lamivudine and dolutegravir plus tenofovir disoproxil fumarate/emtricitabine: pooled analysis of the GEMINI-1 and GEMINI-2 clinical studies. HIV Res Clin Pract. 2021 Dec 9;23(1):9-14. Epub 2021 Dec 16.

enrollment_type: Actual

location_country:
Poland
Taiwan
Portugal
Romania
Spain
Italy
Russian Federation
Australia
Mexico
Argentina
Canada
Switzerland
United Kingdom
United States
France
Peru
Belgium
Germany

summary: This study will compare safety, efficacy, and tolerability of a two drug regimen of dolutegravir (DTG) plus (+) lamivudine (3TC) administered once daily with DTG plus two nucleoside reverse transcriptase inhibitors (tenofovir disoproxil fumarate [TDF]/emtricitabine [FTC] fixed dose combination [FDC]) administered once daily in human immunodeficiency virus (HIV) 1 infected adult subjects that have not previously received antiretroviral therapy. The study is designed to demonstrate the non inferior antiviral activity of DTG + 3TC regimen to that of DTG + TDF/FTC FDC and will characterise the long term antiviral activity, tolerability and safety of DTG plus 3TC through Week 148. Approximately, 700 subjects will be randomised 1:1 to receive DTG + 3TC or DTG + TDF/FTC FDC. Subjects will be stratified by screening HIV 1 ribonucleotide nucleic acid (RNA) levels and by screening CD4+ (cluster of differentiation 4) cell count.

lead_sponsor_type: industriel

status_simplified: Completed


title: An Open Label, Phase I Study to Assess the Effect of Itraconazole (CYP3A4 and P-gp Inhibitor) on the Pharmacokinetics of Anetumab Ravtansine and to Assess the ECG Effects, Safety and Immunogenicity of Anetumab Ravtansine Given as a Single Agent and Together With Itraconazole in Subjects With Mesothelin-expressing Advanced Solid Cancers

summary: Characterize the safety, tolerability, ECG effects, pharmacokinetics and immunogenicity of anetumab ravtansine given as single agent and after inhibition of CYP3A4 and P-gp by concomitant administration of itraconazole in subjects with mesothelin-expressing advanced solid cancers

ipd_sharing: Undecided

ipd_sharing_description: Availability of this study's data will be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.clinicalstudydatarequest.com to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the Study sponsors section of the portal.

lead_sponsor: Bayer

location_country:
Spain
Australia
United States
France
Belgium
Netherlands

location_facility: Universitair Medisch Centrum St. Radboud

location_facility: Washington University School of Medicine

location_facility: Ciutat Sanitària i Universitaria de la Vall d'Hebron

location_facility: University Hospitals Cleveland Medical Center

location_facility: Fundacion Jimenez Diaz (Clinica de la Concepcion)


acronym: CENTERA

title: A Multi-center, Single-arm, Interventional Phase 4 Study to Evaluate a Treat and Extend Regimen of Intravitreal Aflibercept for Treatment of Macular Edema Secondary to Central Retinal Vein Occlusion

ReferenceCitation: Korobelnik JF, Larsen M, Eter N, Bailey C, Wolf S, Schmelter T, Allmeier H, Chaudhary V. Efficacy and Safety of Intravitreal Aflibercept Treat-and-Extend for Macular Edema in Central Retinal Vein Occlusion: the CENTERA Study. Am J Ophthalmol. 2021 Jul;227:106-115. doi: 10.1016/j.ajo.2021.01.027. Epub 2021 Feb 6.

location_country:
Denmark
Spain
Italy
United Kingdom
Australia
Canada
France
Germany

summary:
Central retinal vein occlusion (CRVO) occurs when the main blood vessel that transports blood away from the retina (the very back portion of the eye) becomes blocked, causing the leakage of fluid into the retina and thereby causing a swelling of the macula (the portion of the retina responsible for fine vision). This swelling is called macular edema. When the macula swells with fluid, central vision becomes blurry. The study drug aflibercept has been shown to reduce the amount of fluid and blood leaked into the retina. It can help to stabilize, and in many cases, improve the vision loss related to CRVO. Aflibercept has been approved for the treatment of macular edema secondary to CRVO in the United States (US), European Union (EU), Japan, and other countries.

The study was considered research because, although the study drug was already on the market for macular edema secondary to CRVO, there were no studies available that addressed the questions of what were useful intervals for treating and assessing patients, how did they differ among patients, and how were criteria applied for retreatment. The purpose of this study was to evaluate the effectiveness, treatment interval, and safety of the treatment regimen (pattern for administering treatment) in subjects with macular edema secondary to CRVO. In addition, this study explored new imaging methods for assessing the affected eye.


ipd_sharing: Yes

acronym: HAVEN 2

title: A Multicenter, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Subcutaneous Administration of Emicizumab in Hemophilia A Pediatric Patients With Inhibitors

lead_sponsor: Hoffmann-La Roche

design_allocation: Non-Randomized

ReferenceCitation: Kruse-Jarres R, Peyvandi F, Oldenburg J, Chang T, Chebon S, Doral MY, Croteau SE, Lambert T, Kempton CL, Pipe SW, Ko RH, Trzaskoma B, Dhalluin C, Bienz NS, Niggli M, Lehle M, Paz-Priel I, Young G, Jiménez-Yuste V. Surgical outcomes in people with hemophilia A taking emicizumab prophylaxis: experience from the HAVEN 1-4 studies. Blood Adv. 2022 Aug 8. pii: bloodadvances.2022007458. doi: 10.1182/bloodadvances.2022007458. [Epub ahead of print]

ReferenceCitation: Young G, Liesner R, Chang T, Sidonio R, Oldenburg J, Jiménez-Yuste V, Mahlangu J, Kruse-Jarres R, Wang M, Uguen M, Doral MY, Wright LY, Schmitt C, Levy GG, Shima M, Mancuso ME. A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors. Blood. 2019 Dec 12;134(24):2127-2138. doi: 10.1182/blood.2019001869.

ipd_sharing_description:
Qualified researchers may request access to individual patient level data through the request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/).

For further details on Roche's Global Policy on Sharing of Clinical Study Information and how to request access to related clinical study documents, see here (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).

location_country:
Spain
Italy
United Kingdom
United States
Costa Rica
France
Turkey
South Africa
Japan
Germany

location_facility: Children's Hospital of Michigan; Pediatrics

location_facility: North Shore/Long Island Jewish PRIME; Pediatric Hematology/Oncology & Stem Cell Transplantation

location_facility: CH de Bicetre; Centre de Traitement d' Hemophilie

location_facility: Hospital of the University of Occupational and Environmental Health,Japan

location_facility: Great Ormond street Hospital for Children NHS Foundation Trust; Haemophilia Centre

location_facility: Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia

location_facility: Oregon Health & Science Uni ; Dept of Pediatrics

location_facility: Istanbul University, Cerrahpasa Medical Faculty; Pediatrics Department

location_facility: IRCCS Ca' Granda Ospedale Maggiore Policlinico; Centro Emofilia e Trombosi "Angelo Bianchi e Bonomi"

location_facility: Groupe Hospitalier Necker Enfants Malades

location_facility: University of Colorado Denver, Children's Hospital

location_facility: Universitätsklinikum Bonn; Institut für Experimentelle Hämatologie und Transfusionsmedizin

location_facility: Ege University, School of Medicine; Pediatrics Department

location_facility: Hopital Cardio-vasculaire Louis Pradel; Hemostase clinique

location_facility: Hospital Universitario la Paz; Servicio de Hematologia

location_facility: Adana Acibadem Hospital; Pediatric Hematology

location_facility: Hospital Universitario la Fe; Servicio de Hematologia

location_facility: Charlotte Maxeke Johannesburg Hospital; Haemophilia Comprehensive Care Center

location_facility: Hospital Universitario Virgen del Rocio; Servicio de Hematologia

summary: This non-randomized, multicenter, open-label, Phase III clinical study will evaluate the efficacy, safety, and pharmacokinetics of emicizumab administered subcutaneously initially once weekly (QW) in pediatric participants with hemophilia A with FVIII inhibitors. This study will open two additional non-randomized cohorts to investigate once every 2 weeks (Q2W) and once every 4 weeks (Q4W) regimens in pediatric participants.


title: A Phase I, Open-label, Dose Escalation Study to Investigate the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of GSK3326595 in Subjects With Solid Tumors and Non-Hodgkin's Lymphoma

acronym: Meteor 1

summary: This first time in human (FTIH) open-label, dose escalation study will assess the safety, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of GSK3326595 in participants with advanced or recurrent solid tumors, as well as clinical activity in participants with a subset of solid tumors and non-Hodgkin's lymphoma (NHL).

status: Active, not recruiting

study_completion_date_type: Anticipated

ipd_sharing_description: IPD for this study will be made available via the Clinical Study Data Request site.

lead_sponsor: GlaxoSmithKline

location_country:
Netherlands
United States
Canada
France

status_simplified: Ongoing


title: A Phase 1b Study of Abemaciclib in Combination With Pembrolizumab for Patients With Stage IV Non-Small Cell Lung Cancer or Hormone Receptor Positive, HER2 Negative Breast Cancer

lead_sponsor: Eli Lilly and Company

type: Other Identifier

ReferenceCitation: Pujol JL, Vansteenkiste J, Paz-Ares Rodríguez L, Gregorc V, Mazieres J, Awad M, Jänne PA, Chisamore M, Hossain AM, Chen Y, Beck JT. Abemaciclib in Combination With Pembrolizumab for Stage IV KRAS-Mutant or Squamous NSCLC: A Phase 1b Study. JTO Clin Res Rep. 2021 Sep 25;2(11):100234. doi: 10.1016/j.jtocrr.2021.100234. eCollection 2021 Nov.

enrollment_type: Anticipated

location_country:
Taiwan
Spain
Italy
United States
France
Turkey
Belgium

location_facility: Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg

location_facility: Istanbul University Cerrahpasa Medical Faculty

location_facility: Centre Hospitalier Universitaire Sart Tilman

location_facility: University of Colorado School of Medicine

location_facility: CHU de Montpellier-Hopital Arnaud de Villeneuve

location_facility: Istituto Scientifico Romagnolo - Studio e la Cura dei Tumori

location_facility: Taipei Medical University- Shuang Ho Hospital

summary: The main purpose of this study is to evaluate the safety and efficacy of abemaciclib in combination with pembrolizumab in participants with advanced non-small cell lung cancer (NSCLC) or hormone receptor positive (HR+), human epidermal growth factor receptor negative (HER2-) breast cancer.


title: A Study to Determine the Concordance of Key Actionable Genomic Alterations as Assessed in Tumor Tissue and Plasma From Patients With Non Small Cell Lung Carcinoma (NSCLC)

summary: A study to determine the concordance of key actionable genomic alterations as assessed in tumor tissue and plasma from patients with non small cell lung carcinoma (NSCLC)

status: Terminated

study_type: Observational

time_perspective: Prospective

lead_sponsor: Genomic Health®, Inc.

location_country:
Spain
Ireland
United Kingdom
United States
Chile
France
Japan

location_facility: Hospital Clinico Universitario Lozano Blesa

location_facility: Cancer Care Associates d/b/a Torrance Memorial Physician Network

location_facility: Clatterbridge Cancer Center NHS Foundation Trust

status_simplified: Unknown


title: A Phase 1, Multi-center, Open-label, Dose De-escalation Study to Evaluate the Safety and Efficacy of Talimogene Laherparepvec in Pediatric Subjects With Advanced Non Central Nervous System Tumors That Are Amenable to Direct Injection

lead_sponsor: Amgen

ipd_sharing_description: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request.

location_country:
Spain
Italy
Canada
Switzerland
United States
France
Belgium

location_facility: Cincinnati Childrens Hospital Medical Center

location_facility: Centre Hospitalier Universitaire Sainte Justine

location_facility: Ann and Robert H Lurie Childrens Hospital of Chicago

location_facility: Hospital Universitario Infantil Niño Jesus

location_facility: Institut Hematologie et Oncologie Pediatrique

location_facility: Hospital Universitari i Politecnic La Fe

location_facility: Centre Hospitalier Universitaire de Marseille - Hopital de la Timone

summary: This is a phase 1 study to evaluate the safety of intralesional talimogene laherparepvec administration in pediatric subjects with advanced non-CNS tumors that are amenable to direct injection


title: A Phase 2, Fast Real Time Assessment of Combination Therapies in Immuno-Oncology Study in Subjects With Advanced Non-Small Cell Lung Cancer (FRACTION-Lung)

acronym: FRACTION-Lung

summary: The purpose of this study is to determine whether Nivolumab, in combination with other therapies, is effective in patients with advanced Non-Small Cell lung cancer

lead_sponsor: Bristol-Myers Squibb

location_country:
Spain
Italy
Austria
Australia
Canada
Switzerland
United States
France

location_facility: University of Michigan Health System (UMHS) - University Hospital (University of Michigan Medical Ce

location_facility: University of Californa, Los Angeles (UCLA)

location_facility: Washington University, The Center for Advanced Medicine

location_facility: University Of Ottawa - The Ottawa Hospital Cancer centre

location_facility: University of Washington-Seattle Cancer Care Alliance

location_facility: Juravinski Cancer Centre, Hamilton Health Sciences-Mcmaster Univeristy's Faculty Of Health Sciences

location_facility: The University of Texas MD Anderson Cancer Center

location_facility: Sidney Kimmel Comprehensive Cancer Center

location_facility: The West Clinic, P.C. d/b/a West Cancer Center

location_facility: University of Maryland - Marlene and Stewart Greenebaum Cancer Center

intervention_type: Biological


title: An Open-label, Randomised, Non-comparative Phase 2 Study Evaluating S 95005 (TAS-102) Plus Bevacizumab and Capecitabine Plus Bevacizumab in Patients With Previously Untreated Metastatic COlorectal Cancer Who Are Non-eligible for Intensive Therapy (TASCO1 Study).

lead_sponsor: Institut de Recherches Internationales Servier

ReferenceCitation: Van Cutsem E, Danielewicz I, Saunders MP, Pfeiffer P, Argilés G, Borg C, Glynne-Jones R, Punt CJA, Van de Wouw AJ, Fedyanin M, Stroyakovskiy D, Kroening H, Garcia-Alfonso P, Wasan H, Falcone A, Fougeray R, Egorov A, Amellal N, Moiseyenko V. First-line trifluridine/tipiracil + bevacizumab in patients with unresectable metastatic colorectal cancer: final survival analysis in the TASCO1 study. Br J Cancer. 2022 Jun;126(11):1548-1554. doi: 10.1038/s41416-022-01737-2. Epub 2022 Apr 19.

ReferenceCitation: Van Cutsem E, Danielewicz I, Saunders MP, Pfeiffer P, Argilés G, Borg C, Glynne-Jones R, Punt CJA, Van de Wouw AJ, Fedyanin M, Stroyakovskiy D, Kroening H, Garcia-Alfonso P, Wasan H, Falcone A, Kanehisa A, Egorov A, Aubel P, Amellal N, Moiseenko V. Trifluridine/tipiracil plus bevacizumab in patients with untreated metastatic colorectal cancer ineligible for intensive therapy: the randomized TASCO1 study. Ann Oncol. 2020 Sep;31(9):1160-1168. doi: 10.1016/j.annonc.2020.05.024. Epub 2020 Jun 1.

ipd_sharing_description:
Qualified scientific and medical researchers can request access to anonymized patient-level and study-level clinical trial data.

Access can be requested for all interventional clinical studies:

used for Marketing Authorization (MA) of medicines and new indications approved after 1 January 2014 in the European Economic Area (EEA) or the United States (US).
where Servier is the Marketing Authorization Holder (MAH). The date of the first MA of the new medicine (or the new indication) in one of the EEA Member States will be considered for this scope.

In addition, access can be requested for all interventional clinical studies in patients:

sponsored by Servier
with a first patient enrolled as of 1 January 2004 onwards
for New Chemical Entity or New Biological Entity (new pharmaceutical form excluded) for which development has been terminated before any Marketing authorization (MA) approval.

location_country:
Denmark
Poland
Brazil
Spain
Italy
Russian Federation
Australia
Netherlands
United Kingdom
France
Belgium
Germany

location_facility: Sint Antonius Ziekenhuis Interne Geneeskunde/Oncologie

location_facility: Seconda Università degli Studi di Napoli, U.O.C. di Oncologia Medica ed Ematologia

location_facility: Instituto do Câncer do Estado de São Paulo - ICESP, Núcleo de Pesquisa

location_facility: The Beatson West of Scotland Cancer Centre GI cancers

location_facility: Centralny Szpital Kliniczny MSW Klinika Onkologii i Hematologii

location_facility: SP ZOZ Szpital Uniwersytecki w Krakowie Oddzial Kliniczny Onkologii

location_facility: Schwerpunktpraxis für Hämatologie und Onkologie

location_facility: AMC Academisch Medisch Centrum Medische Oncologie

location_facility: Scientific Centre for Specialized Medical Care (oncological)

location_facility: Odense Universitetshospital - Department of Oncology

location_facility: Imperial healthcare NHS Trust Charing Cross Hospital

location_facility: Hospital General Universitario, Oncología Médica

location_facility: Instituto Catalan De Oncología, Hospitalet de Llobregat

location_facility: H. Universitario La Paz Oncología Médica

location_facility: Hospital Universitario Reina Sofia, Deparatmento Oncología Médica

location_facility: Complejo Hospitalario de Navarra, Oncología Médica

location_facility: Städtisches Krankenhaus München Neuperlach, Klinik für Onkologie und Hämatologie

location_facility: Szpitale Pomorskie Sp. z o.o. Oddzial Onkologii i Radioterapii

location_facility: Hospital do Câncer de Barretos - Fundação Pio XII

location_facility: Centro de Pesquisa Hospital de Caridade de Ijuí

location_facility: Isala Klinieken, Medische oncologie, Dokter Van Heeweg 2

location_facility: Zuyderland Medisch Centrum Interne Geneeskunde

location_facility: Amphia Ziekenhuis, Interne Geneeskunde/Oncologie, Langendijk 75

location_facility: Centre René Gauducheau, Oncologie Médicale

location_facility: VieCurie Medisch Centrum, Interne Geneeskunde, Tegelseweg 210

location_facility: Tergooi Hilversum, Medische Oncologie, Van Riebeeckweg 212

location_facility: .O.U. Pisana-Ospedale Santa Chiara, U.O. di Oncologia Medica 2

location_facility: Catharina Ziekenhuis, Interne Geneeskunde/Oncologie

location_facility: Grand Hôpital de Charleroi Oncologie-Hématologie

location_facility: NZOZ MAGODENT Oddzial Onkologii Klinicznej / Chemioterapii

location_facility: Hospital Ramón y Cajal, Oncología Médica

location_facility: Mount Vernon Hospital Department of Oncology

location_facility: Hospital Valle de Hebrón, Servicio de Oncología

location_facility: Martini Ziekenhuizen, Interne Geneeskunde/Oncologie, Van Swietenplein 1

location_facility: Moscow City Oncology Hospital # 62, Chemotherapy

location_facility: Christie Hospital NHS Foundation Trust, GI & Endocrine

location_facility: Hôpital Saint Antoine, oncology department

location_facility: A.O. Ospedale Niguarda Ca' Granda-Milano, Department of Onco-Haematology- Onoclogia Falck

location_facility: Instituto Nacional do Câncer - INCA Unidade de Pesquisa ClínicaInstituto Nacional do Câncer - INCA Unidade de Pesquisa Clínica

location_facility: Russian Cancer Research Center n.a. NN Blokhin, Department of Research for New Antitumour Medicines

location_facility: Austin Hospital Olivia Newton-John Cancer & Wellness Centre

location_facility: A.O.U. SanMartino-IST, Unità Operativa Oncologia Medica 1

location_facility: Fondazione Poliambulanza Istituto Ospedaliero, Clinical Oncology

location_facility: The Queen Elizabeth Hospital Haematology and Oncology Unit

location_facility: CHU Jean Minjoz, Service d'oncologie médicale

location_facility: Hospital de Base, Centro Intergrado de Pesquisa

location_facility: Leicester Royal Infirmary, The HOPE Clinical Trials Unit

location_facility: UZ Leuven Campus Gasthuisberg Digestieve Oncologie

summary: The main purpose of this study is to evaluate the progression-free survival (PFS) in patients receiving S 95005 + bevacizumab (experimental arm) or capecitabine + bevacizumab (control arm) as first-line treatment for unresectable metastatic colorectal cancer in patients non-eligible for intensive therapy.


primary_purpose: Other

title: Long-term Follow-up Study of Patients Who Have Previously Been Exposed to UCART19 (Allogeneic Engineered T-cells Expressing a Lentiviral-based Anti-CD19 Chimeric Antigen Receptor)

location_country:
United Kingdom
Spain
United States
France
Japan

location_facility: University of Texas Southwestern Medical Center

location_facility: University of Texas MD Anderson Cancer Center

location_facility: King's College Hospital NHS Foundation Trust

summary: The purpose of this study is to evaluate the long-term safety and efficacy of UCART19 administration to patients with advanced lymphoid leukemia.


title: A Double-blind, Randomized, Placebo Controlled, Two Arm Multi-center Study to Assess the Efficacy and Safety of a Once Nightly Formulation of Sodium Oxybate for Extended-Release Oral Suspension (FT218) for the Treatment of Excessive Daytime Sleepiness and Cataplexy in Subjects With Narcolepsy

lead_sponsor: Avadel

ReferenceCitation: Roth T, Dauvilliers Y, Thorpy MJ, Kushida C, Corser BC, Bogan R, Rosenberg R, Dubow J, Seiden D. Effect of FT218, a Once-Nightly Sodium Oxybate Formulation, on Disrupted Nighttime Sleep in Patients with Narcolepsy: Results from the Randomized Phase III REST-ON Trial. CNS Drugs. 2022 Apr;36(4):377-387. doi: 10.1007/s40263-022-00904-6. Epub 2022 Apr 5.

ReferenceCitation: Kushida CA, Shapiro CM, Roth T, Thorpy MJ, Corser BC, Ajayi AO, Rosenberg R, Roy A, Seiden D, Dubow J, Dauvilliers Y. Once-nightly sodium oxybate (FT218) demonstrated improvement of symptoms in a phase 3 randomized clinical trial in patients with narcolepsy. Sleep. 2022 Jun 13;45(6). pii: zsab200. doi: 10.1093/sleep/zsab200.

location_country:
Australia
Canada
United States
France
Germany
Czechia

location_facility: Sleep Medicine Specialists of South Florida

location_facility: CIUSS de Nord-de-I'ile-de- Montreal- Hopital de Sacre-Coeur de Montreal

location_facility: Sleep Medicine Specialist of South Florida

location_facility: NeuroMedical Research Institute/Global Research Holdings, LLC

location_facility: Baptist Health Center for Clinical Research

location_facility: Florida Pulmonary Research Institute LLC

location_facility: Medical University of South Carolina - Institute of Psychiatry

location_facility: Sleep Management Institute Intrepid Research

location_facility: INSERM - Centre d'Investigation Clinque Hopital Robert Debre

location_facility: NHMRC CEntre for Translational Sleep and Circadian Neurobiology

location_facility: University Hospitals Case Medical Center

location_facility: Yale-New Haven Hospital's Sleep Medicine Center

location_facility: OSF Healthcare Saint Francis Medical Center

summary: The purpose of this study is to determine whether once-nightly FT218 is safe and effective for the treatment of excessive daytime sleepiness and cataplexy in subjects with narcolepsy.


intervention_type: Other

title: Interventional, Randomised, Double-blind, Placebo-controlled, Active Reference (Fluoxetine), Fixed-dose Study of Vortioxetine in Paediatric Patients Aged 12 to 17 Years, With Major Depressive Disorder (MDD)

lead_sponsor: H. Lundbeck A/S

ReferenceCitation: Findling RL, DelBello MP, Zuddas A, Emslie GJ, Ettrup A, Petersen ML, Schmidt SN, Rosen M. Vortioxetine for Major Depressive Disorder in Adolescents: 12-Week Randomized, Placebo-Controlled, Fluoxetine-Referenced, Fixed-Dose Study. J Am Acad Child Adolesc Psychiatry. 2022 Sep;61(9):1106-1118.e2. doi: 10.1016/j.jaac.2022.01.004. Epub 2022 Jan 13.

location_country:
Poland
Serbia
Spain
Italy
Latvia
Estonia
Mexico
Canada
Russian Federation
United Kingdom
Ukraine
Hungary
Korea, Republic of
United States
France
South Africa
Colombia
Germany
Bulgaria

location_facility: RS1006 Clinic of Neurology and Psychiatry for Children and Adolescents

location_facility: US1385 Ericksen Research And Development

summary: Evaluating the efficacy of vortioxetine 10 mg/day and 20 mg/day versus placebo on depressive symptoms in adolescents (age ≥12 and ≤17 years) with a DSM-5™ (Diagnostic and Statistical Manual of Mental Disorders, 5th edition) diagnosis of Major depressive disorder (MDD).


title: Interventional, Randomized, Double-blind, Placebo-controlled, Active-reference (Fluoxetine), Fixed-dose Study of Vortioxetine in Paediatric Patients Aged 7 to 11 Years, With Major Depressive Disorder (MDD)

location_country:
Poland
Serbia
Spain
Italy
Latvia
Estonia
Mexico
Canada
Russian Federation
Ukraine
Hungary
Korea, Republic of
United States
France
Israel
South Africa
Colombia
Germany
Bulgaria

location_facility: E.S.E. Hospital Mental de Antioquia HOMO

location_facility: Arkhangelsk Regional Clinical Mental Hospital

location_facility: Specjalistyczny Szpital im. dra A. Sokolowskiego w Walbrzychu

location_facility: Bekes Megyei Kozponti Korhaz Pandy Kalman Tagkorhaza

location_facility: State Budgetary Healthcare Institution of Sverdlovsk Region Â¿Sverdlovsk Regional Clinical Psychi...

location_facility: Centro para el Desarrollo de la Medicina y de Asistencia Medica Especializada S.C

location_facility: Ternopil Regional Clinical Municipal Psycho-Neurological Hospital, Ternopil State Medical Univers...

location_facility: Unidad de Salud Mental Infanto-Juvenil (USMI-J) Edificio de Consultas Externas. Hospital MarAtimo

location_facility: Medical Research Group of Central Florida

location_facility: State Budgetary Healthcare Institution (SBHI) Specialized Clinical Psychiatric Hospital 1 of the ...

location_facility: Alliance for Wellness dba Alliance for Research

location_facility: St. Charles Psychiatric Associates - Midwest Research Group

location_facility: Instituto Nacional de Pediatria (INP) (National Institute of Pediatrics)

location_facility: Hospital Universitario Fundacion Alcorcon

location_facility: Yaroslavl Regional Clinical Psychiatry Hospital

location_facility: Rheinhessen-Fachklinik Mainz, Kinder und Jugendpsychiatri

location_facility: University Hospital Cleveland Medical Center Division of Child and Adolescent Psychiatry

location_facility: Centro de Investigaciones y Proyectos en Neurociencias CIPNA LTDA IPS.

location_facility: Maltsev Poltava Regional Clinical Psychiatric Hospital, Higher State Educational Institution Of U...

location_facility: Kansas University School of Medicine-Wichita

location_facility: Daily Hospital for Children and Adolescents

location_facility: Filip Rybakowski Specjalistyczna Praktyka Lekarska

location_facility: CRI Centro Regiomontano de Investigacion SC

location_facility: Centro de investigaciones del Sistema Nervioso SAS Grupo CISNE SAS

location_facility: Stavropol Region Psychiatric Hospital No.2

location_facility: ICARO Investigaciones en Medicina S.A. de C.V.

location_facility: Children's National Medical Center Merge

location_facility: Child and Adolescent Neurology and Psychiatry Clinic

location_facility: Medicorehabilitation Research Center Phoenix

location_facility: Przychodnia Syntonia Poradnia Zdrowia Psychicznego

location_facility: City Psychiatric Hospital No.3 named after I.I. Skvortsov-Stepanov

location_facility: LLC City Neurological Center Sibneuromed

location_facility: Ukrainian Research Institute Of Social, Forensic Psychiatry And Drug Abuse, Kiev City Psychoneuro...

location_facility: Diagnostic Consultative Center Mladost-M Varna OOD

location_facility: Spectrum Centrum Psychiatrii Specjalistyczny Gabinet Psychiatryczny

location_facility: Rostov State Medical University of the Minzdravsotsrazvitiya of Russia

location_facility: Odessa Regional Medical Centre of Mental Health

location_facility: Centro Investigacion Medico Biologica Y Terapia Avanzada

location_facility: Scientific Institute Fondazione Stella Maris

location_facility: Guz Saratov Regional Psychiatric Hospital St. Sofii

location_facility: Centrum Badan Klinicznych PI-House Sp. z o.o.

location_facility: The Chaim Sheba Medical Center - The Edmond and Lily Safra Children's Hospital

location_facility: Clinical Center of Vojvodina - Clinic of Psychiatry

summary: Investigation of the efficacy and safety of a new potential treatment of Major depressive disorder (MDD) in paediatric participants (age 7 to 11 years).


title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Tolerability of Entospletinib, a Selective SYK Inhibitor, in Combination With Systemic Corticosteroids as First-Line Therapy in Subjects With Chronic Graft Versus Host Disease (cGVHD)

lead_sponsor: Gilead Sciences

location_country:
Spain
United Kingdom
Canada
Korea, Republic of
United States
France
Germany

location_facility: Severance Hospital, Yonsei University Health System

location_facility: Ohio State University, Wexner Medical Center

location_facility: Hospital Clinico Universitario de Valencia

location_facility: Hospital General Universitario Gregorio Marañon

location_facility: Hospital Universitario Marques de Valdecilla

summary: The primary objective of this study is to evaluate the effect of entospletinib (ENTO) on the best overall response rate in adults with chronic graft versus host disease (cGVHD) who are currently receiving systemic corticosteroids as part of first-line therapy for cGVHD.


title: Transcatheter Aortic Valve Replacement With the Medtronic Transcatheter Aortic Valve Replacement System In Patients at Low Risk for Surgical Aortic Valve Replacement

summary:
The study objective is to demonstrate that the safety and effectiveness of the Medtronic TAVR system as measured by rates of all-cause mortality or disabling stroke at two years is noninferior to SAVR in the treatment of severe aortic stenosis in subjects who have a low predicted risk of operative mortality for SAVR.

The purpose of the expanded use addendum to the Medtronic TAVR in Low Risk Patients Trial protocol is to conclude the randomized phase of the trial and initiate the single-arm, non-randomized, continued access phase of the trial.

ReferenceCitation: Ramlawi B, Deeb GM, Yakubov SJ, Markowitz AH, Hughes GC, Kiaii RB, Huang J, Kleiman NS, Reardon MJ. Mechanisms of Death in Low-Risk Patients After Transcatheter or Surgical Aortic Valve Replacement. Cardiovasc Revasc Med. 2022 Sep;42:1-5. doi: 10.1016/j.carrev.2022.03.027. Epub 2022 Mar 29.

ReferenceCitation: Forrest JK, Deeb GM, Yakubov SJ, Rovin JD, Mumtaz M, Gada H, O'Hair D, Bajwa T, Sorajja P, Heiser JC, Merhi W, Mangi A, Spriggs DJ, Kleiman NS, Chetcuti SJ, Teirstein PS, Zorn GL 3rd, Tadros P, Tchétché D, Resar JR, Walton A, Gleason TG, Ramlawi B, Iskander A, Caputo R, Oh JK, Huang J, Reardon MJ. 2-Year Outcomes After Transcatheter Versus Surgical Aortic Valve Replacement in Low-Risk Patients. J Am Coll Cardiol. 2022 Mar 8;79(9):882-896. doi: 10.1016/j.jacc.2021.11.062.

ReferenceCitation: Lanz J, Reardon MJ, Pilgrim T, Stortecky S, Deeb GM, Chetcuti S, Yakubov SJ, Gleason TG, Huang J, Windecker S. Incidence and Outcomes of Infective Endocarditis After Transcatheter or Surgical Aortic Valve Replacement. J Am Heart Assoc. 2021 Oct 5;10(19):e020368. doi: 10.1161/JAHA.120.020368. Epub 2021 Sep 28.

ReferenceCitation: Blanke P, Leipsic JA, Popma JJ, Yakubov SJ, Deeb GM, Gada H, Mumtaz M, Ramlawi B, Kleiman NS, Sorajja P, Askew J, Meduri CU, Kauten J, Melnitchouk S, Inglessis I, Huang J, Boulware M, Reardon MJ; Evolut Low Risk LTI Substudy Investigators. Bioprosthetic Aortic Valve Leaflet Thickening in the Evolut Low Risk Sub-Study. J Am Coll Cardiol. 2020 May 19;75(19):2430-2442. doi: 10.1016/j.jacc.2020.03.022. Epub 2020 Mar 28.

ReferenceCitation: Popma JJ, Deeb GM, Yakubov SJ, Mumtaz M, Gada H, O'Hair D, Bajwa T, Heiser JC, Merhi W, Kleiman NS, Askew J, Sorajja P, Rovin J, Chetcuti SJ, Adams DH, Teirstein PS, Zorn GL 3rd, Forrest JK, Tchétché D, Resar J, Walton A, Piazza N, Ramlawi B, Robinson N, Petrossian G, Gleason TG, Oh JK, Boulware MJ, Qiao H, Mugglin AS, Reardon MJ; Evolut Low Risk Trial Investigators. Transcatheter Aortic-Valve Replacement with a Self-Expanding Valve in Low-Risk Patients. N Engl J Med. 2019 May 2;380(18):1706-1715. doi: 10.1056/NEJMoa1816885. Epub 2019 Mar 16.

ReferenceCitation: Leipsic J, Bax JJ, Webb JG, Martin R, Blanke P. Trials Testing the Value of Imaging Use in Valve Disease and in Transcatheter Valvular Interventions. JACC Cardiovasc Imaging. 2017 Mar;10(3):286-295. doi: 10.1016/j.jcmg.2016.09.031. Review.

lead_sponsor: Medtronic Cardiovascular

location_country:
Australia
New Zealand
Canada
United States
France
Japan
Netherlands

location_facility: University Hospitals Case Medical Center

location_facility: Baylor Jack and Jane Hamilton Heart and Vascular Hospital

location_facility: University of Southern California University Hospital

location_facility: Centre Hospitalier Régional Universitaire de Lille

location_facility: University of Pittsburgh Medical Center UPMC Presbyterian

location_facility: The University of Vermont Medical Center

location_facility: National Cerebral and Cardiovasclular Center

intervention_type: Device


title: An Open-label, Randomised, Active-controlled, Parallel Group, Multicentre, Phase 3 Study to Investigate the Safety and Efficacy of PA21 (Velphoro®) and Calcium Acetate (Phoslyra®) in Paediatric and Adolescent CKD Patients With Hyperphosphataemia

lead_sponsor: Vifor Fresenius Medical Care Renal Pharma

location_country:
Poland
Russian Federation
Lithuania
United States
France
Romania
Germany

location_facility: Hospital Of Lithuanian University Of Health Sciences Kaunas Clinics

location_facility: Spitalul Clinic de Urgenţă pentru copii "Maria Sklodowska Curie"

location_facility: Washington University School of Medicine in St. Louis

location_facility: University of Iowa Hospitals and Clinics

location_facility: St. Petersburg GBUZ "Children's City Hospital No. 1"

location_facility: Texas Children's Hospital - Texas Children's Feigin Center

location_facility: The University of Texas Southwestern Medical Center

location_facility: Service de Néphrologie et Endocrinologie pédiatriques

location_facility: Oregon Health and Science University, Doernbecher Children's Hospital

location_facility: Uniwersytecki Szpital Dzieciecy w Krakowie - Prokocimiu

location_facility: Children's Hospital, Affiliate of Vilnius University Hospital Santariskiu Klinikos

location_facility: The University of Texas Medical School at Houston

location_facility: St. Vladimir Children's City Clinical Hospital

location_facility: University of Miami - Miller School of Medicine

location_facility: Instytut "Pomnik - Centrum Zdrowia Dziecka"

location_facility: Universitätsklinikum Gießen und Marburg GmbH

location_facility: University of Alabama at Birmingham School of Medicine

location_facility: Uniwersytecki Dziecięcy Szpital Kliniczny im. L. Zamenhofa w Białymstoku

summary: This is a Phase 3, Open-label, Randomised, Active-controlled, Parallel Group, Multicentre Study to Investigate the Safety and Efficacy of PA21 (Velphoro®) and Calcium Acetate (Phoslyra®) in Paediatric and Adolescent CKD Patients with Hyperphosphataemia. The aim of this Phase 3 clinical study is to demonstrate similar efficacy of PA21 (Velphoro) in paediatric and adolescent patients with CKD, and to provide safety and dosing information for this patient population. The Phoslyra (comparator) group provides information for a descriptive comparison of PA21 against a commonly used calcium-based phosphate binder (calcium acetate).


title: Multicenter, Open-label Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of LCZ696 Followed by a 52-week Randomized, Double-blind, Parallel Group, Active-controlled Study to Evaluate the Efficacy and Safety of LCZ696 Compared With Enalapril in Pediatric Patients From 1 Month to < 18 Years of Age With Heart Failure Due to Systemic Left Ventricle Systolic Dysfunction

lead_sponsor: Novartis Pharmaceuticals

ipd_sharing_description:
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

location_country:
Croatia
Saudi Arabia
Italy
Spain
United States
South Africa
Japan
Czechia
Poland
Portugal
Austria
Russian Federation
Argentina
India
Israel
Turkey
Singapore
Lebanon
Canada
Switzerland
Hungary
China
Germany
Bulgaria
Taiwan
Jordan
Finland
Thailand
Korea, Republic of
France

summary:
This study consist of two parts (Part 1 and Part 2). The purpose of Part 1 is to evaluate the way the body absorbs, distributes and removes the drug LCZ696. This will help determine the proper dose of LCZ696 for Part 2 of the study.

The purpose for Part 2 is to compare the effectiveness and safety of LCZ696 with enalapril in pediatric heart failure patients over 52 weeks of treatment.


title: An Open Label Multi-Center Extension Study to Evaluate the Long-term Safety of Zorblisa™ (SD-101-6.0) in Patients With Epidermolysis Bullosa

summary: The study aimed to assess the long-term safety of topical use of Zorblisa (SD-101-6.0) in participants with Epidermolysis Bullosa (EB).

lead_sponsor: Scioderm, Inc.

location_country:
Poland
Serbia
Spain
United Kingdom
Austria
Australia
Netherlands
Lithuania
United States
France
Israel
Germany


title: A Phase IB Multi-Arm Study With Venetoclax in Combination With Cobimetinib and Venetoclax in Combination With Idasanutlin in Patients With Relapsed or Refractory Acute Myeloid Leukemia Who Are Not Eligible for Cytotoxic Therapy

location_country:
United States
Italy
Canada
France

location_facility:
Dana Farber Cancer Institute
USC Norris Cancer Center
UC Davis; Comprehensive Cancer Center
Wake Forest Baptist Medical Center
Institut Paoli Calmettes
Presidio san salvatore muraglia
Princess Margaret Cancer Center
Hopital Avicenne, Paris
Universita di Roma
University of Alberta Hospital
Jewish General Hospital
University of Bologna
CHU de Bordeaux
University of Colorado
Univ of Calif, San Francisco
MD Anderson Cancer Center
Weill Cornell Medical College

summary: The primary objective for this study is to assess the safety and tolerability as well as preliminary efficacy of venetoclax in combination with cobimetinib, and venetoclax in combination with idasanutlin in patients with relapsed or refractory acute myeloid leukemia (R/R) AML who are not eligible for cytotoxic therapy.


acronym: PDUS

title: A 52-week, Multicenter Study to Assess the Time Course of Response to Secukinumab on Joint Inflammation Using Power Doppler Ultrasonography in Patients With Active Psoriatic Arthritis

ReferenceCitation: D'Agostino MA, Schett G, Lopez-Rdz A, Senolt L, Fazekas K, Burgos-Vargas R, Maldonado-Cocco J, Naredo E, Carron P, Duggan AM, Goyanka P, Boers M, Gaillez C. Response to secukinumab on synovitis using Power Doppler ultrasound in psoriatic arthritis: 12-week results from a phase III study, ULTIMATE. Rheumatology (Oxford). 2022 May 5;61(5):1867-1876. doi: 10.1093/rheumatology/keab628.

ipd_sharing_description:
Novartis is committed to sharing access to patient-level data and supporting clinical documents from eligible studies with qualified external researchers. Requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to protect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

location_country:
Spain
Colombia
Austria
Ireland
Italy
Argentina
Canada
Mexico
Netherlands
United Kingdom
Hungary
United States
France
Belgium
Norway
Germany
Czechia

summary: This study was designed to leverage the sensitivity of ultrasonography available in clinical practice setting to better describe the time course of response to secukinumab (150 mg and 300 mg) on joint synovitis and enthesitis in PsA patients with an inadequate response to non-biologic DMARDs. PDUS changes in joint synovitis will be assessed using the global Outcome Measures in Rheumatology (OMERACT)-European League against Rheumatism (EULAR) synovitis score (GLOESS) and changes in joint enthesitis were assessed using the OMERACT enthesitis score.


acronym: BERSON

title: A Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate Safety and Efficacy of Evolocumab (AMG 145) in Combination With Statin Therapy in Diabetic Subjects With Hyperlipidemia or Mixed Dyslipidemia (BERSON)

ReferenceCitation: Blom DJ, Chen J, Yuan Z, Borges JLC, Monsalvo ML, Wang N, Hamer AW, Ge J. Effects of evolocumab therapy and low LDL-C levels on vitamin E and steroid hormones in Chinese and global patients with type 2 diabetes. Endocrinol Diabetes Metab. 2020 Mar 6;3(2):e00123. doi: 10.1002/edm2.123. eCollection 2020 Apr.

ReferenceCitation: Chen Y, Yuan Z, Lu J, Eliaschewitz FG, Lorenzatti AJ, Monsalvo ML, Wang N, Hamer AW, Ge J. Randomized study of evolocumab in patients with type 2 diabetes and dyslipidaemia on background statin: Pre-specified analysis of the Chinese population from the BERSON clinical trial. Diabetes Obes Metab. 2019 Jun;21(6):1464-1473. doi: 10.1111/dom.13700. Epub 2019 Apr 14.

location_country:
Brazil
Russian Federation
Argentina
Canada
Korea, Republic of
United States
France
China
Turkey
Colombia

summary: The primary objective of this study was to evaluate the effect of 12 weeks of subcutaneous evolocumab (AMG 145) in combination with statin therapy (atorvastatin) on percent change from baseline in low-density lipoprotein cholesterol (LDL-C) in diabetic adults with hyperlipidemia or mixed dyslipidemia.


title: A Phase 1b (Open-Label)/Phase 2 (Randomized, Double-Blinded) Study Evaluating Gemcitabine and Docetaxel With or Without Olaratumab in the Treatment of Advanced Soft Tissue Sarcoma

acronym: ANNOUNCE 2

summary: The main purpose of this study is to evaluate the safety and efficacy of two anti-cancer drugs (gemcitabine and docetaxel) with and without the study drug known as olaratumab in participants with advanced soft tissue sarcoma (STS) or STS that has spread to another part(s) of the body.

ipd_sharing_description: Anonymized individual patient level data will be provided in a secure access environment upon approval of a research proposal and a signed data sharing agreement.

location_country:
Poland
Spain
Italy
United Kingdom
Australia
Hungary
United States
France
Israel
Germany

location_facility: Oklahoma Cancer Specialists & Research Institute, LLC

location_facility: Johns Hopkins University School of Medicine

location_facility: University of Texas MD Anderson Cancer Center

location_facility: Fondazione Piemonte l'Oncologia-Istituto Ricerca Cura Cancro

location_facility: University of Texas Southwestern Medical Center at Dallas

location_facility: Jasz-Nagykun-Szolnok Megyei Hetenyi Geza Korhaz

location_facility: Centrum Onkologii-Instytut im Marii Sklodowskiej-Curie


title: A Phase 1b/3 Multicenter, Randomized, Trial of Talimogene Laherparepvec in Combination With Pembrolizumab for the Treatment of Subjects With Recurrent or Metastatic Squamous Cell Carcinoma of the Head and Neck

ReferenceCitation: Harrington KJ, Kong A, Mach N, Chesney JA, Fernandez BC, Rischin D, Cohen EEW, Radcliffe HS, Gumuscu B, Cheng J, Snyder W, Siu LL. Talimogene Laherparepvec and Pembrolizumab in Recurrent or Metastatic Squamous Cell Carcinoma of the Head and Neck (MASTERKEY-232): A Multicenter, Phase 1b Study. Clin Cancer Res. 2020 Oct 1;26(19):5153-5161. doi: 10.1158/1078-0432.CCR-20-1170. Epub 2020 Jul 15.

ipd_sharing_description: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request

location_country:
Spain
Greece
Austria
Australia
Italy
Canada
United Kingdom
Switzerland
United States
France
Belgium

summary: The primary objective of this study was to evaluate the safety, as assessed by incidence of dose limiting toxicity (DLT), of talimogene laherparepvec in combination with pembrolizumab in adults with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN).


acronym: Avelumab in Third-Line Gastric Cancer

title: A Phase III Open-label, Multicenter Trial of Avelumab (MSB0010718C) as a Third-line Treatment of Unresectable, Recurrent, or Metastatic Gastric or Gastroesophageal Junction Adenocarcinoma

lead_sponsor: EMD Serono Research & Development Institute, Inc.

ReferenceType: result

ReferenceCitation: Bang YJ, Ruiz EY, Van Cutsem E, Lee KW, Wyrwicz L, Schenker M, Alsina M, Ryu MH, Chung HC, Evesque L, Al-Batran SE, Park SH, Lichinitser M, Boku N, Moehler MH, Hong J, Xiong H, Hallwachs R, Conti I, Taieb J. Phase III, randomised trial of avelumab versus physician's choice of chemotherapy as third-line treatment of patients with advanced gastric or gastro-oesophageal junction cancer: primary analysis of JAVELIN Gastric 300. Ann Oncol. 2018 Oct 1;29(10):2052-2060. doi: 10.1093/annonc/mdy264.

location_country:
Poland
Spain
Italy
Australia
Korea, Republic of
United States
France
Belgium
Germany
Czechia

location_facility: The Catholic University of Korea, Seoul St. Mary's Hospital

location_facility: Hospital Universitario la Paz - site 546

location_facility: Hematology and Oncology Associates of SC, LLC 900 West Faris Rd, 3rd Floor

location_facility: Oncology Consultants, P.A. 2130 W. Holcombe Blvd. 10th Floor

location_facility: Southern Nevada Cancer Research Foundation 601 S Rancho Drive

location_facility: Advanced Medical Specialties 8940 North Kendall Drive, Suite 300E

location_facility: Severance Hospital, Yonsei University Health System

location_facility: Texas Oncology, P.A. - McAllen 1901 South 2nd Street

location_facility: New York Oncology Hematology, P.C. 400 Patroon Creek Blvd, Suite 1

location_facility: Penn State University Milton S. Hershey Medical Center 500 University Drive

location_facility: Schwerpunktpraxis für Haematologie und OnkologieOnkologische Schwerpunktpraxis Eppendorf

location_facility: Oncology Specialists, S.C. 1700 Luther Ln, Ste 2200, Park Ridge, IL 60068 7900 Milwaukee Ave, Ste 16

location_facility: Carle Cancer Center 509 W. University Avenue

location_facility: Fondazione del Piemonte per l'Oncologia IRCC Candiolo

location_facility: Rocky Mountain Cancer Centers 1800 Williams Street, Suite 100

location_facility: Chonnam National University Hwasun Hospital

location_facility: Kyungpook National University Medical Center

location_facility: Ocala Oncology Center, P.L. 433 S.W. 10th Street

location_facility: Sanford Roger Maris Cancer Center - Fargo 801 Broadway North Route 1058

location_facility: A.O.U. Ospedali Riuniti Ancona- Clinica Oncologica

location_facility: Henry Ford Health System 2799 West Grand Boulevard

location_facility: Texas Oncology Bedford 1609 Hospital Parkway

location_facility: Texas Oncology, P.A. 3410 Worth Street, Suites 300 & 400

location_facility: Texas Oncology, P.A. - Tyler 910 E. Houston St, Suite 100

location_facility: Northwest Cancer Specialists, P.C. 265 N Broadway

location_facility: Nemocnice Rudolfa a Stefanie Benesov, a. s.

location_facility: Scott and White Memorial Hospital and Clinic 2401 South 31st Street

location_facility: Illinois Cancer Specialists 8915 W. Golf Rd.

location_facility: Ingalls Memorial Hospital One Ingalls Drive, W741

location_facility: Hospital General Universitario Gregorio Marañon

location_facility: Metairie Oncologist, LLC Office of Jayne Gurtler MD, Laura Brinz MD, Janet Burroff MD 3939 Houma Blvd, Suite 6

location_facility: Texas Oncology, P.A. - Denton 3720 South I-35 East

location_facility: Florida Cancer Specialists 560 Jackson Street, Suite 220

location_facility: Minnesota Oncology Hematology, P.A. 910 East 26th Street, Suites 100 and 200

location_facility: Rocky Mountain Cancer Centers, LLP 3676 Parker Blvd #350

location_facility: Cotton-O'Neil Clinical Research Center, Hematology and Oncology and Stormont Vail Cancer Center 1414 SW 8th St

location_facility: Centrum Onkologii-Instytut im. M. Sklodowskiej Curie

location_facility: Seoul National University Bundang Hospital

summary: The purpose of this study was to demonstrate superiority of treatment with avelumab plus best supportive care (BSC) versus physician's choice (chosen from a pre-specified list of therapeutic options) plus BSC.


acronym:
AIPAC

 AIPAC

title: AIPAC (Active Immunotherapy PAClitaxel): A Multicentre, Phase IIb, Randomised,Double Blind, Placebo-controlled Study in Hormone Receptor-positive Metastatic Breast Carcinoma Patients Receiving IMP321 (LAG-3Ig Fusion Protein) or Placebo as Adjunctive to a Standard Chemotherapy Treatment Regimen of Paclitaxel

lead_sponsor: Immutep S.A.S.

location_country:
Poland
United Kingdom
Netherlands
Hungary
France
Belgium
Germany

location_facility: Universitair Ziekenhuis Antwerpen Breast and Gynecological Oncology Unit

location_facility: UFKT - Universitäts-Frauenklinik Tübingen

location_facility: GZA Ziekenhuizen campus Sint-Augustinus Oncologische Research

location_facility: Onkologische Gemeinschaftspraxis am Bethanien-Krankenhaus Centrum für Hämatologie und Onkologie

location_facility: Nottingham University Hospitals NHS Trust

location_facility: Institut Claudius Regaud - IUC Toulouse - Oncopôle

location_facility: Kierownik Oddziału Onkologii i Radioterapii Szpital Morski im. PCK w Gdyni

location_facility: NCT - Nationales Centrum für Tumorerkrankungen

location_facility: UZ Leuven, campus Gasthuisberg Department of General Medical Oncology and Multidisciplinary Breast Centre

location_facility: KEM- Brustzentrum der Kliniken Essen-Mitte

location_facility: Cliniques universitaires Saint-Luc - Institut Roi Albert II - Cancérologie et Hématologie Oncologie clinique

location_facility: The Christie NHS Foundation Trust The Christie Clinic - Medical Oncology

location_facility: MH Egészségügyi Központ Onkológiai Osztály

summary: The proposed Phase IIb clinical study aims to investigate the safety and efficacy of the active immunotherapy IMP321 in combination (adjunctive) with paclitaxel chemotherapy in patients with hormone receptor-positive metastatic breast cancer.


title: A Phase 2, Randomized, Controlled, Open-Label, Clinical Study of the Efficacy and Safety of Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine in Patients With Higher-Risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia and Low-Blast Acute Myelogenous Leukemia

lead_sponsor: Millennium Pharmaceuticals, Inc.

type: Registry Identifier

ReferenceCitation: Sekeres MA, Watts J, Radinoff A, Sangerman MA, Cerrano M, Lopez PF, Zeidner JF, Campelo MD, Graux C, Liesveld J, Selleslag D, Tzvetkov N, Fram RJ, Zhao D, Bell J, Friedlander S, Faller DV, Adès L. Randomized phase 2 trial of pevonedistat plus azacitidine versus azacitidine for higher-risk MDS/CMML or low-blast AML. Leukemia. 2021 Jul;35(7):2119-2124. doi: 10.1038/s41375-021-01125-4. Epub 2021 Jan 22. Erratum in: Leukemia. 2021 Dec;35(12):3637.

ipd_sharing_description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

location_country:
Spain
Ireland
Italy
Netherlands
Canada
United States
France
Israel
Belgium
Germany
Bulgaria
Czechia

location_facility: ICO I'Hospitalet Hospital Duran i Reynals Instituto Catalan de Oncologia de Hospitalet (ICO)

location_facility: Azienda Ospedaliera Bianchi Melacrino Morelli

location_facility: University of North Carolina at Chapel Hill

location_facility: University of Miami Miller School of Medicine

location_facility: Azienda Ospedaliero Universitaria Di Bologna - Policlinico S Orsola Malpighi

location_facility: Hospital Universitari i Politecnic La Fe de Valencia

location_facility: University Multiprofile Hospital for Active Treatment Sv Ivan Rilski EAD

location_facility: Marien Hospital Akademisches Lehrkrankenhaus

location_facility: Hospital Universitario Germans Trias i Pujol

location_facility: H Lee Moffitt Cancer Center and Research Institute

location_facility: Hospital Universitario Quironsalud Madrid

location_facility: Azienda Ospedaliera Universitaria Careggi

location_facility: Compassionate Cancer Care Medical Group Incorporated

location_facility: Hospital Regional Universitario de Malaga - Hospital Universitario Virgen de la Victoria

location_facility: Specialized Hospital for Active Treatment of Haematological Diseases - Sofia

location_facility: Hospital General Universitario Gregorio Maranon

location_facility: Hospital Clinico Universitario de Valencia

location_facility: Smilow Cancer Center at Yale New Haven Hospital

location_facility: University Multi-Profile Hospital for Active Treatment Dr Georgi Stranski

location_facility: Cliniques Universitaires UCL de Mont-Godinne

location_facility: Azienda Ospedaliero Universitaria San Giovanni Battista Di Torino

summary: The purpose of this study is to evaluate the efficacy and safety of pevonedistat plus azacitidine versus single-agent azacitidine in participants with HR-MDS or CMML, or low-blast AML.


title: A Phase 2 Multicenter Study Evaluating the Efficacy of KTE-X19 in Subjects With Relapsed/Refractory Mantle Cell Lymphoma

lead_sponsor: Kite, A Gilead Company

ReferenceCitation: Cheson BD, Fisher RI, Barrington SF, Cavalli F, Schwartz LH, Zucca E, Lister TA; Alliance, Australasian Leukaemia and Lymphoma Group; Eastern Cooperative Oncology Group; European Mantle Cell Lymphoma Consortium; Italian Lymphoma Foundation; European Organisation for Research; Treatment of Cancer/Dutch Hemato-Oncology Group; Grupo Español de Médula Ósea; German High-Grade Lymphoma Study Group; German Hodgkin's Study Group; Japanese Lymphorra Study Group; Lymphoma Study Association; NCIC Clinical Trials Group; Nordic Lymphoma Study Group; Southwest Oncology Group; United Kingdom National Cancer Research Institute. Recommendations for initial evaluation, staging, and response assessment of Hodgkin and non-Hodgkin lymphoma: the Lugano classification. J Clin Oncol. 2014 Sep 20;32(27):3059-68.

ReferenceCitation: Locke F, Wang M, Siddiqi T, Castro J, Shah B, Lee H, et al. ZUMA-2: A Phase 2 Multicenter Study Evaluating the Efficacy of KTE-C19 (Anti-CD19 CAR T cells) in Subjects with Relapsed/Refractory Mantle Cell Lymphoma (r/r MCL). American Society Of Clinical Oncology (ASCO) Annual Meeting 2016.

ReferenceCitation: Wang M, Locke F, Siddiqi T, Castro J, Shah B, Lee H, et al. ZUMA-2: A Phase 2 Multicenter Study Evaluating the Efficacy of KTE-C19 (Anti-CD19 CAR T cells) in Subjects with Relapsed/Refractory Mantle Cell Lymphoma (r/r MCL). European Society for Medical Oncology (ESMO) Congress 2016;Abstract 3305

ReferenceCitation: Wang M, Locke FL, Munoz J, Goy A, Holmes HE, Siddiqi T, et al. ZUMA-2: Phase 2 Multicenter Study Evaluating Efficacy of Kte-C19 in Patients with Relapsed/Refractory Mantle Cell Lymphoma [Abstract]. J Clin Oncol 2018;36 (Suppl 15):TPS3102.

ReferenceCitation: Wang M, Munoz J, Goy A, Locke FL, Jacobson CA, Hill BT, Timmerman JM, Holmes H, Jaglowski S, Flinn IW, McSweeney PA, Miklos DB, Pagel JM, Kersten MJ, Milpied N, Fung H, Topp MS, Houot R, Beitinjaneh A, Peng W, Zheng L, Rossi JM, Jain RK, Rao AV, Reagan PM. KTE-X19 CAR T-Cell Therapy in Relapsed or Refractory Mantle-Cell Lymphoma. N Engl J Med. 2020 Apr 2;382(14):1331-1342. doi: 10.1056/NEJMoa1914347.

ReferenceCitation: Wang, Michael; Rossi, John M.; Munoz, Javier; Goy, Andre; Lundry Locke, Frederick; Michael Reagan, Patrick; Jacobson, Caron A.; Hill, Brian T; Holmes, Houston; Mary Jaglowski, Samantha; Peng, Weimin; Zheng, Lianqing; Fang, Xiang; Xue, Allen; Rao, Arati V.; Bot, Adrian

ReferenceCitation: Wang, Michael(1); Munoz, Javier(2); Goy, Andre(3); Locke, Frederick L.(4); Jacobson, Caron A.(5); Hill, Brian T.(6); Timmerman, John M.(7); Holmes, Houston(8); Jaglowski, Samantha(9); Flinn, Ian W.(10); McSweeney, Peter A.(11); Miklos, David B.(12); Pagel, John M.(13); José Kersten, Marie(14); Peng, Weimin(15); Zheng, Lianqing(15); Rossi, John M.(15); Jain, Rajul K.(15); Rao, Arati V.(15); Reagan, Patrick M.(16)

ReferenceCitation: Wang, M.(1); Munoz, J.(2); Goy, A.(3); Locke, F.(4); Jacobson, C.(5); Hill, B.(6); Timmerman, J.(7); Holmes, H.(8); Jaglowski, S.(9); Flinn, I.(10); McSweeney, P.(11); Miklos, D.(12); Pagel, J.(13); Kersten, M. J.(14)(15)

ReferenceCitation: Michael Wang,1 Javier Munoz,2 Andre Goy,3 Frederick L. Locke,4 Caron A. Jacobson,5 Brian T. Hill,6 John M. Timmerman,7 Houston Holmes,8 Samantha Jaglowski,9 Ian W. Flinn,10 Peter A. McSweeney,11 David B. Miklos,12 John M. Pagel,13 Marie José Kersten,14 Noel Milpied,15 Henry Fung,16 Max S. Topp,17 Roch Houot,18 Amer Beitinjaneh,19 Weimin Peng,20 Lianqing Zheng,20 John M. Rossi,20 Rajul K. Jain,20 Arati V. Rao,20 and Patrick M. Reagan21

ReferenceCitation: Wang M, Munoz J, Goy A, Locke FL, Jacobson CA, Hill BT, Timmerman JM, Holmes H, Jaglowski S, Flinn IW, McSweeney PA,Miklos DB, Pagel JM, Kersten MJ, Peng W,Zheng L,Rossi JM, Jain RK, Rao AV, Reagan PM

ReferenceCitation: Wang, Michael L., MD(1); Munoz, Javier, MD(2); Goy, Andre, MD(3); Locke, Frederick L., MD(4); Jacobson, Caron A., MD, MMSc(5); Hill, Brian T., MD(6); Timmerman, John M., MD(7); Holmes, Houston, MD(8); Jaglowski, Samantha, MD, MPH(9); Flinn, Ian W., MD, PhD(10); McSweeney, Peter A., MD(11); Miklos, David B., MD, PhD(12); Pagel, John M., MD, PhD, DSc(13); Jose Kersten, Marie, MD, PhD(14); Peng, Weimin, MS(15); Zheng, Lianqing, PhD(15); Rossi, John M., MS(15); Jain, Rajul K., MD(15); Rao, Arati V., MD(15); Reagan, Patrick M, MD(16)

ReferenceCitation: Wang, Michael; Lundry Locke, Frederick; Munoz, Javier; Goy, Andre; Eccleston Holmes, Houston; Siddiqi, Tanya; Flinn, Ian; McSweeney, Peter A; Michael Reagan, Patrick; Thomas Hill, Brian; Jacobson, Caron A.; Rizzieri, David A.; Heffner, Leonard T.; Mary Jaglowski, Samantha; Bernard Miklos, David; Shaughnessy, Paul; Unabia, Sherry; Rossi, John M.; Jiang, Yizhou; Jain, Rajul K.

ReferenceCitation: Wang M, Jain P, Chi TL, Chen SE, Heimberger A, Weathers SP, Zheng L, Rao AV, Rossi JM. Management of a patient with mantle cell lymphoma who developed severe neurotoxicity after chimeric antigen receptor T-cell therapy in ZUMA-2. J Immunother Cancer. 2020 Oct;8(2). pii: e001114. doi: 10.1136/jitc-2020-001114.

location_country:
Germany
United States
Netherlands
France

location_facility: University of Texas MD Anderson Cancer Center

location_facility: University California Los Angeles (UCLA)

location_facility: Winship Cancer Institute of Emory University

summary: The primary objective of the study is to evaluate the efficacy of brexucabtagene autoleucel (KTE-X19) in participants with relapsed/refractory mantle cell lymphoma (MCL)


title: A Phase 1/2a Dose Escalation and Cohort Expansion Study for Safety, Tolerability, and Efficacy of BMS-986156 Administered Alone and in Combination With Nivolumab (BMS-936558, Anti PD-1 Monoclonal Antibody) in Advanced Solid Tumors

ReferenceCitation: Heinhuis KM, Carlino M, Joerger M, Di Nicola M, Meniawy T, Rottey S, Moreno V, Gazzah A, Delord JP, Paz-Ares L, Britschgi C, Schilder RJ, O'Byrne K, Curigliano G, Romano E, Patah P, Wang R, Liu Y, Bajaj G, Siu LL. Safety, Tolerability, and Potential Clinical Activity of a Glucocorticoid-Induced TNF Receptor-Related Protein Agonist Alone or in Combination With Nivolumab for Patients With Advanced Solid Tumors: A Phase 1/2a Dose-Escalation and Cohort-Expansion Clinical Trial. JAMA Oncol. 2020 Jan 1;6(1):100-107. doi: 10.1001/jamaoncol.2019.3848.

location_country:
Spain
Italy
Australia
Netherlands
Canada
Switzerland
United States
France
Belgium
Germany

location_facility:
UCSD Moores Cancer Center
Universitair Ziekenhuis Gent
Institut Claudius Regaud
Providence Portland Medical Center
Istituto Europeo Di Oncologia
Cantonal Hospital St. Gallen
Liverpool Cancer Therapy Center
University Of Alabama At Birmingham
Princess Alexandra Hospital
IRCCS Istituto Nazionale Tumori Milano
Emory University
The West Clinic, P.C.
Thomas Jefferson University Hospital
Institut Gustave Roussy
Linear Clinical Research Ltd
The Ohio State University
Local Institution

summary: The purpose of this study is to evaluate the safety and tumor-shrinking ability of experimental medication BMS-986156, when given by itself or in combination with nivolumab in patients with solid cancers that are advanced or cancers that have spread.


title: An Exploratory Multicenter, Open-Label, Single Arm Study of the Safety and Tolerability of Pirfenidone (Esbriet®) in Combination With Nintedanib (Ofev®) in Patients With Idiopathic Pulmonary Fibrosis

summary: This clinical study will evaluate the safety and tolerability of combination treatment of nintedanib and pirfenidone in participants with IPF. Eligible participants must have received pirfenidone for at least 16 weeks on a stable dose. Nintedanib will be added on Day 1 of the study as a combination treatment for IPF for 24 weeks.

ReferenceCitation: Flaherty KR, Fell CD, Huggins JT, Nunes H, Sussman R, Valenzuela C, Petzinger U, Stauffer JL, Gilberg F, Bengus M, Wijsenbeek M. Safety of nintedanib added to pirfenidone treatment for idiopathic pulmonary fibrosis. Eur Respir J. 2018 Aug 2;52(2). pii: 1800230. doi: 10.1183/13993003.00230-2018. Print 2018 Aug. Erratum in: Eur Respir J. 2018 Oct 4;52(4):.

lead_sponsor: Hoffmann-La Roche

location_country:
Denmark
Spain
Italy
Netherlands
Canada
United States
France
Germany

location_facility: Hospital del Henares; Medicina Interna. Unidad de Neumología

location_facility: A.O. Universitaria San Luigi Gonzaga di Orbassano; Malattie Apparato Respiratorio (MAR2)

location_facility: Azienda Ospedaliero Universitaria Pisana; U.O. Pneumologia

location_facility: Klinikum Fulda gAG; Universitätsmedizin Marburg, Campus Fulda

location_facility: Inova Health Care Services; Advanced Lung Disease Transplant Program

location_facility: Ruhrlandklinik Lungenzentrum der UNI Essen Abt.Pneumologie-Allergologie

location_facility: Hospital Universitari de Bellvitge ; Servicio de Neumologia

location_facility: Hopital de Pontchaillou; Service de Pneumologie

location_facility: Complejo Asistencial Universitario de Leon; Pneumology

location_facility: John A. Butler, M.D. - Oregon Pulmonary Associates

location_facility: Hospital General Universitario De Valencia; Servicio de Neumologia

location_facility: Hospital Universitario Virgen del Rocio; Servicio de Neumologia

location_facility: Stanford University School of Medicine ; Pulmonary/Critical Care Medicine

location_facility: Medical University of South Carolina (MUSC); MUSC Pulmonary

location_facility: A.O. Univ. Senese Policlinico S. Maria alle Scotte; UOC Malattie Resepiratorie e Trapianto Polmonare

location_facility: Gentofte Hospital, Lungemedicinsk Afdeling

location_facility: Hospital Universitario La Princesa; Servicio de Neumologia

location_facility: Cardio-Pulmonary Associates of St. Luke's Hospital

location_facility: Antonius Ziekenhuis; Dept of Lung Diseases

location_facility: Fachkrankenhaus Coswig GmbH Zentrum f.Pneumologie Beatmungsmedizin Thorax-u.Gefäßchirurgie

location_facility: South Health Campus/Alberta Health Services/ University of Calgary

location_facility: David Geffen School of Medicine at UCLA;Division of Pulmonary & Critical Care/ Department of Medic

location_facility: Hospital Universitario de Canarias; Servicio de Neumologia


acronym: ASSENT

title: A Phase 1b/2, Multi-Center, Double-Blind (Principal Investigators and Study Subjects Blinded, Sponsor Unblinded), Placebo-Controlled, Randomized, Single-Ascending Dose Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of DS-1040b in Subjects With Acute Ischemic Stroke

lead_sponsor: Daiichi Sankyo, Inc.

ipd_sharing_description: De-identified individual participant data (IPD) and applicable supporting clinical trial documents may be available upon request at https://vivli.org/. In cases where clinical trial data and supporting documents are provided pursuant to our company policies and procedures, Daiichi Sankyo will continue to protect the privacy of our clinical trial participants. Details on data sharing criteria and the procedure for requesting access can be found at this web address: https://vivli.org/ourmember/daiichi-sankyo/

location_country:
Taiwan
Spain
Italy
United Kingdom
Australia
Korea, Republic of
United States
France
Germany
Slovakia
Czechia

location_facility: Chang Gung Memorial Hospital-Linkou Branch

location_facility: Oregon Health Sciences University Hospital

location_facility: Penn State Milton S. Hershey Medical Center

location_facility: Hospital Clínico Universitario de Santiago de Compostela

location_facility: Klinikum der Johann Wolfgang Goethe-Universitat

location_facility: University of Kentucky Chandler Medical Center

location_facility: Chattanooga Center for Neurologic Research

location_facility: University College Hospitals NHS Foundation Trust

location_facility: University of South Alabama USA Health System

location_facility: Ospedaliero di Albenga - Pietra Ligure Dept Neurology

location_facility: Svet zdravia a.s.,Vseobecna nemocnica Rimavska Sobota

location_facility: Universittsklinikum Essen AR Klinik fr Neurologie

location_facility: Hospital Clinico Universitario de Valladolid

location_facility: Hospital Clinico Universitario Lozano Blesa de Zaragoza

location_facility: Ospedale Guglielmo da Saliceto Via Taverna

location_facility: Seoul National University Bundang Hospital

summary: This is a Phase 1b/2, double-blind (study participants and Investigators), placebo-controlled, randomized, single-ascending dose, multi-center study to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of DS-1040b in participants with Acute Ischemic Stroke (AIS).


title: A Phase 3, Randomized, Vehicle-Controlled, Double-Blind, Multicenter Study to Evaluate the Safety and Efficacy of Once-Daily CLS001 Topical Gel Versus Vehicle Administered for 12 Weeks to Subjects With Papulopustular Rosacea With a 4 Week Follow-up Period

summary: This study evaluates the safety and efficacy of once-daily application of CLS001 topical gel compared to vehicle gel in subjects with severe papulopustular rosacea.

lead_sponsor: Maruho Co., Ltd.

location_country:
United Kingdom
Australia
Netherlands
Canada
New Zealand
Sweden
United States
France
Germany


title: Confirmatory Validation of Oral Macimorelin as a Growth Hormone (GH) Stimulation Test (ST) for the Diagnosis of Adult Growth Hormone Deficiency (AGHD) in Comparison With the Insulin Tolerance Test (ITT)

summary: The Macimorelin Growth Hormone Stimulation Test (GHST) will be compared with the Insulin Tolerance Test (ITT) in an open-label, randomized, 2-way crossover Trial. The trial will include subjects suspected to have adult growth hormone deficiency (AGHD) and a group of healthy control subjects.

primary_purpose: Diagnostic

ReferenceCitation: Garcia JM, Biller BMK, Korbonits M, Popovic V, Luger A, Strasburger CJ, Chanson P, Medic-Stojanoska M, Schopohl J, Zakrzewska A, Pekic S, Bolanowski M, Swerdloff R, Wang C, Blevins T, Marcelli M, Ammer N, Sachse R, Yuen KCJ. Macimorelin as a Diagnostic Test for Adult GH Deficiency. J Clin Endocrinol Metab. 2018 Aug 1;103(8):3083-3093. doi: 10.1210/jc.2018-00665.

lead_sponsor: AEterna Zentaris

location_country:
Poland
Serbia
Spain
Italy
Austria
United Kingdom
United States
France
Germany

location_facility: Klinik für Endokrinologie, Diabetes und Ernährungsmedizin der Charité

location_facility: Phase I - MTZ Clinical Research Sp. z o.o.

location_facility: Medical University & General Hospital of Vienna, AKH,

location_facility: Baylor College of Medicine-Endocrinology


title: An Early-Phase, Multicenter, Open-Label Study of the Safety and Pharmacokinetics of Atezolizumab (MPDL3280A) In Pediatric and Young Adult Patients With Previously Treated Solid Tumors

ReferenceCitation: Geoerger B, Zwaan CM, Marshall LV, Michon J, Bourdeaut F, Casanova M, Corradini N, Rossato G, Farid-Kapadia M, Shemesh CS, Hutchinson KE, Donaldson F, Liao M, Caron H, Trippett T. Atezolizumab for children and young adults with previously treated solid tumours, non-Hodgkin lymphoma, and Hodgkin lymphoma (iMATRIX): a multicentre phase 1-2 study. Lancet Oncol. 2020 Jan;21(1):134-144. doi: 10.1016/S1470-2045(19)30693-X. Epub 2019 Nov 25.

ReferenceCitation: Shemesh CS, Chanu P, Jamsen K, Wada R, Rossato G, Donaldson F, Garg A, Winter H, Ruppel J, Wang X, Bruno R, Jin J, Girish S. Population pharmacokinetics, exposure-safety, and immunogenicity of atezolizumab in pediatric and young adult patients with cancer. J Immunother Cancer. 2019 Nov 21;7(1):314. doi: 10.1186/s40425-019-0791-x.

location_country:
Denmark
Spain
Italy
United Kingdom
Netherlands
Canada
Switzerland
United States
France
Israel
Germany

location_facility: Ospedale Pediatrico Bambino Gesù - IRCCS; Dipartimento di Onco-Ematologia Pediatrica

location_facility: Stanford University/Lucile Packard Children's Hospital

location_facility: Fondazione IRCCS Istituto Nazionale dei Tumori; Struttura Complessa di Pediatria Oncologica

location_facility: Hospital Universitari i Politecnic La Fe de Valencia

location_facility: University of Texas Health Science Center at San Antonio

location_facility: Penn State Milton S. Hershey Medical Center

location_facility: Erasmus MC / location Sophia Kinderziekenhuis

location_facility: IRCCS Istituto Giannina Gaslini; Unità Operativa Oncologica Pediatrica

location_facility: Institut Gustave Roussy; Service Pediatrique

location_facility: Universitäts-Kinderspital; Abteilung für Onkologie

location_facility: Rigshospitalet; BØRNEUNGEKLINIKKEN, Ambulatoriet for kræft- og Blodsygdomme hos børn og unge

location_facility: Azienda Ospedaliera Universitaria Città della Salute e della Scienza di Torino

location_facility: Azienda Ospedaliera di Padova; Clinica di Onco-ematologia pediatrica

location_facility: Leeds General Infirmary; Paediatric Oncology & Haematology

location_facility: Centre Léon Bérard, Institut d'Hémato-Oncologie Pédiatrique

location_facility: The Royal Victoria Infirmary; Paediatric and Adolescent Oncology Unit

location_facility: Hospital Infantil Universitario Nino Jesus

summary: This early phase, multicenter, open-label, single-arm study evaluated the safety, tolerability, pharmacokinetics, immunogenicity, and preliminary efficacy of atezolizumab in pediatric and young adult participants with solid tumors for which prior treatment was proven to be ineffective.


title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study With Open-Label Extension to Assess the Efficacy and Safety of KRN23 in Adults With X-linked Hypophosphatemia (XLH)

lead_sponsor: Ultragenyx Pharmaceutical Inc

ReferenceCitation: Insogna KL, Briot K, Imel EA, Kamenický P, Ruppe MD, Portale AA, Weber T, Pitukcheewanont P, Cheong HI, Jan de Beur S, Imanishi Y, Ito N, Lachmann RH, Tanaka H, Perwad F, Zhang L, Chen CY, Theodore-Oklota C, Mealiffe M, San Martin J, Carpenter TO; AXLES 1 Investigators. A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial Evaluating the Efficacy of Burosumab, an Anti-FGF23 Antibody, in Adults With X-Linked Hypophosphatemia: Week 24 Primary Analysis. J Bone Miner Res. 2018 Aug;33(8):1383-1393. doi: 10.1002/jbmr.3475. Epub 2018 Jun 26.

ReferenceCitation: Portale AA, Carpenter TO, Brandi ML, Briot K, Cheong HI, Cohen-Solal M, Crowley R, Jan De Beur S, Eastell R, Imanishi Y, Imel EA, Ing S, Ito N, Javaid M, Kamenicky P, Keen R, Kubota T, Lachmann R, Perwad F, Pitukcheewanont P, Ralston SH, Takeuchi Y, Tanaka H, Weber TJ, Yoo HW, Zhang L, Theodore-Oklota C, Mealiffe M, San Martin J, Insogna K. Continued Beneficial Effects of Burosumab in Adults with X-Linked Hypophosphatemia: Results from a 24-Week Treatment Continuation Period After a 24-Week Double-Blind Placebo-Controlled Period. Calcif Tissue Int. 2019 Sep;105(3):271-284. doi: 10.1007/s00223-019-00568-3. Epub 2019 Jun 4.

ReferenceCitation: Brandi ML, Jan de Beur S, Briot K, Carpenter T, Cheong HI, Cohen-Solal M, Crowley RK, Eastell R, Imanishi Y, Imel EA, Ing SW, Insogna K, Ito N, Javaid K, Kamenicky P, Keen R, Kubota T, Lachmann RH, Perwad F, Pitukcheewanont P, Portale A, Ralston SH, Tanaka H, Weber TJ, Yoo HW, Sun W, Williams A, Nixon A, Takeuchi Y. Efficacy of Burosumab in Adults with X-linked Hypophosphatemia (XLH): A Post Hoc Subgroup Analysis of a Randomized Double-Blind Placebo-Controlled Phase 3 Study. Calcif Tissue Int. 2022 Oct;111(4):409-418. doi: 10.1007/s00223-022-01006-7. Epub 2022 Aug 4.

ReferenceCitation: Briot K, Portale AA, Brandi ML, Carpenter TO, Cheong HI, Cohen-Solal M, Crowley RK, Eastell R, Imanishi Y, Ing S, Insogna K, Ito N, Jan de Beur S, Javaid MK, Kamenicky P, Keen R, Kubota T, Lachmann RH, Perwad F, Pitukcheewanont P, Ralston SH, Takeuchi Y, Tanaka H, Weber TJ, Yoo HW, Nixon A, Nixon M, Sun W, Williams A, Imel EA. Burosumab treatment in adults with X-linked hypophosphataemia: 96-week patient-reported outcomes and ambulatory function from a randomised phase 3 trial and open-label extension. RMD Open. 2021 Sep;7(3):e001714. doi: 10.1136/rmdopen-2021-001714.

location_country:
United Kingdom
Ireland
Italy
Korea, Republic of
United States
France
Japan

location_facility: University of California-San Francisco Medical Center

location_facility: Azienda Ospedaliero Universitaria Careggi - Neurofarba

location_facility: Northern General Hospital, Metabolic Bone Centre (Sorby Wing)

location_facility: The Ohio State University Wexner Medical Center

location_facility: Indiana University Department of Medicine

location_facility: University of Edinburgh Edinburgh Clinical Trials Unit (ECTU) - Western General Hospital

location_facility: CHU de Bicêtre, Service d'Endocrinologie et des Maladies de la Reproduction

location_facility: Hôpital Lariboisière, Dept. of Rheumatology

summary: The primary efficacy objective of this study is to establish the effect of burosumab treatment compared with placebo on increasing serum phosphorus levels in adults with XLH.


title: An Open-label, Single Arm, Phase III Study to Assess the Self-administration of AOP2014 Using a Pre-filled Pen, Developed for the Treatment of Polycythemia Vera Patients

acronym: PEN-PV

summary:
Polycythemia Vera (PV) is a disease of bone marrow stem cells that manifests in a drastic increase of red blood cells and frequently also of white blood cells. The "thickening" of the blood in relation with a modified function of the cells has several consequences like increased blood pressure, pruritus of the skin, fatigue, disturbed blood circulation in the brain as well as fingers and toes and an increased risk of arterial and venous thrombosis (thrombosis is the formation of a blood clot in a vessel); like stroke, cardiac infarction, deep vein thrombosis in the legs. In case of a strong increase of platelets there is an additional risk of bleedings. As the disease progresses the size of spleen and liver increased in most cases and the bone marrow shows signs of fibrosis. In some cases of PV a progression at a later time point to a leukemia (increased formation of white blood cells) can occur.

The aim of this study is to assess the ease of AOP2014 self-administration using dedicated questionnaires.

To assess safety and tolerability: adverse events (AEs), laboratory parameters, electrocardiogram (ECG) throughout study.
To assess maintenance of the blood efficacy parameters Hct (Hematocrit), WBC (white blood cells) and PLTs (platelets) and spleen size (comparing values at Visit P7 vs. values at Visit P1).
To assess the feasibility of AOP2014 self-administration: defined as the ability of the patients to use the pen as a self-administration tool (ease of handling, safety, tolerability and efficacy).

lead_sponsor: AOP Orphan Pharmaceuticals AG

location_country:
Poland
Austria
Czech Republic
Ukraine
Hungary
France
Bulgaria
Slovakia

location_facility: Dnipropetrovsk City Multispecialty Clinical Hospital #4

location_facility: Multiprofile Hospital for Active Treatment - Hristo Botev, Vratsa, First Department of Internal Medicine

location_facility: National Research Center for Radiation Medicine, Institute of Clinical Radiology

location_facility: Cherkasy Regional Oncology Center, Regional Treatment and Diagnostics Hematology Center

location_facility: Bekes County Pandy Kalman Hospital, 1st Department of Medicine, Hematology

location_facility: Institute of Hematology and Blood Transfusion

location_facility: Andrzej Mielecki Independent Public Clinical Hospital of Medical University of Silesia in Katowice

location_facility: Nicolaus Copernicus Municipal Specialist Hospital

location_facility: Institute of Hematology and Transfusion Medicine

location_facility: Independent Public Teaching Hospital No.1 in Lublin

location_facility: Specialized Hospital for Active Treatment of Hematological Diseases

location_facility: Fryderyk Chopin Provincial Specialized Hospital

location_facility: O.F. Herbachevskyi Regional Clinical Hospital

location_facility: University of Szeged, Albert Szent-Gyorgyi Clinical Center, Koranyi fasor 6

location_facility: University Hospital with Outpatient Clinic F.D. Roosevelt

location_facility: Institute of Blood Pathology and Transfusion Medicine

location_facility: St Istvan and St Laszlo Hospital of Budapest

location_facility: Saint Cyril and Metod University Hospital Bratislava


title: A Multi-center, Double-blind, Randomized, Three-Arm, Parallel-Group, Placebo Controlled Study to Assess the Efficacy and Safety of NTRA-2112 on Intestinal Malabsorption in Preterm Infants

summary: The study will evaluate the effect of NTRA-2112 on intestinal malabsorption in preterm infants.

status: Unknown status

ReferenceCitation: Mank E, Sáenz de Pipaón M, Lapillonne A, Carnielli VP, Senterre T, Shamir R, van Toledo L, van Goudoever JB; FIT-04 Study Group. Efficacy and Safety of Enteral Recombinant Human Insulin in Preterm Infants: A Randomized Clinical Trial. JAMA Pediatr. 2022 May 1;176(5):452-460. doi: 10.1001/jamapediatrics.2022.0020.

ipd_sharing: No

lead_sponsor: Nutrinia

location_country:
Spain
Italy
United Kingdom
Netherlands
Hungary
United States
France
Israel
Belgium
Germany

location_facility: Kinderklinik, Evangelisches Waldkrankenhaus Spandau

location_facility: UMC Groningen (UMCG), Beatrix Kinderziekenhuis

location_facility: Presidio Ospedaliero G. Salesi - AOUI Ospedali Riuniti di Ancona

location_facility: Ospedale dei Bambini "V.Buzzi"- Azienda ICP

location_facility: CHUS - Complejo Hospitalario Universitario de Santiago

location_facility: Klinik fur Neugeborene, Kinder und Jugendliche, Universitatsklinik der Paracelsus Medizinischen Privatuniversitat

location_facility: Szegedi Tudomanyegyetem Szent Gyorgyi Albert Klinikai Kozpont Gyermekgyogyaszati klinika Koraszulott Intenzív Osztaly

location_facility: University Hospital of Leicester NHS Trust- Royal Infirmary

location_facility: Klinik und Poliklinik fur Kinder-und Jugendmedizin, Universitaetsklinikum Carl Gustav Carus Dresden

location_facility: Hospital General Universitario Gregorio Marañon

location_facility: University of Oklahoma Health Sciences Center

location_facility: Ospedale F. Del Ponte - Azienda Ospedaliera di Varese

location_facility: Hospital Universitario Virgen de la Arrixaca

location_facility: Klinik fuer Kinder und Jugendliche, Helios Klinikum Pforzheim

location_facility: Fejéktató Kórhz Újszülött, Csecsemő és Gyermekosztály - Megyei Szent György Egyetemi


title: A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy

acronym: ESSENCE

summary: The main objective of this study is to evaluate the efficacy of SRP-4045 (casimersen) and SRP-4053 (golodirsen) compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively.

status: Recruiting

ReferenceCitation: Wagner KR, Kuntz NL, Koenig E, East L, Upadhyay S, Han B, Shieh PB. Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double-blind, placebo-controlled, dose-titration trial. Muscle Nerve. 2021 Sep;64(3):285-292. doi: 10.1002/mus.27347. Epub 2021 Jun 29.

lead_sponsor: Sarepta Therapeutics, Inc.

location_country:
Serbia
Spain
Italy
United Kingdom
United States
Belgium
Czechia
Poland
Russian Federation
Australia
Argentina
India
Israel
Mexico
Sweden
Canada
Hungary
Germany
Bulgaria
Denmark
Greece
Ireland
Korea, Republic of
France

location_facility: Rigshospitalet Copenhagen University Hospital

location_facility: Federal state budget educational institution of higher education "Russian national research medical university n.a. N.I. Pirogov" of Ministry of healthcare of Russian Federation

location_facility: Hospital Universitari i Politecnic La Fe de Valencia

location_facility: Ann and Robert H. Lurie Children's Hospital of Chicago

location_facility: Rady Children's Hospital San Diego/ UCSD

location_facility: Children's Hospital of Pittsburgh of UPMC

location_facility: Semmelweis Egyetem Genomikai Medicina és Ritka Betegsegek Intezete

location_facility: Samodzielny Publiczny Centralny Szpital Kliniczny

location_facility: Clinic for Neurology and Psychiatry for Children and Youth

location_facility: Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna

location_facility: Center for Integrative Rare Disease Research (CIRDR)

location_facility: Ippokratio General Hospital of Thessaloniki

location_facility: University Multiprofile Hospital for Active Treatment Aleksandrovska EAD

location_facility: Az Ospedaliera Universitaria Policlinico G Martino

location_facility: State Autonomous Healthcare Institution of Sverdlovsk Region Children's Clinical Hospital No. 9 City of Ekaterinburg

location_facility: Children's Hospital of the King's Daughters

location_facility: Reference Centre for Neuromuscular Diseases

location_facility: Pusan National University Yangsan Hospital

location_facility: Drottning Silvias Barn Och Ungdomssjukhus

location_facility: Children's and Women's Health Centre of British Columbia

location_facility: Cincinnati Children's Hospital Medical Center (CCHMC)

location_facility: Instituto de Investigaciones Clínicas para la Salud A.C

location_facility: Stanford University School of Medicine/Medical Center

location_facility: University of Rochester Clinical Research Center

location_facility: Schneider Children's Medical Center of Israel

location_facility: Fondazione IRCCS Istituto Neurologico Carlo Besta


title: A Multi-centre, Randomised, Placebo Controlled, Double Blinded, Multiple Dose Trial Investigating Safety, Pharmacokinetics and Pharmacodynamics of Concizumab Administered Subcutaneously to Haemophilia A Subjects (Explorer™3)

summary: This trial is conducted globally. The aim of this trial is to investigate safety, pharmacokinetics (the exposure of the trial drug in the body) and pharmacodynamics (the effect of the investigated drug on the body) of concizumab administered subcutaneously to haemophilia A subjects.

lead_sponsor: Novo Nordisk A/S

location_country:
Croatia
Poland
Spain
United Kingdom
Austria
Australia
Netherlands
Thailand
Ukraine
United States
France
Israel
Malaysia
Turkey
Germany


title: A Multicenter, Double-Blind, Randomized, Placebo-Controlled, Parallel-Group Study to Investigate the Efficacy and Safety of Lacosamide as Adjunctive Therapy in Subjects With Epilepsy >=1 Month to <4 Years of Age With Partial-Onset Seizures

lead_sponsor: UCB BIOSCIENCES, Inc.

location_country:
Croatia
Serbia
Italy
Ukraine
United States
Romania
Philippines
Czechia
Poland
Portugal
Russian Federation
Argentina
Israel
Mexico
Slovakia
Brazil
Hungary
China
Bulgaria
Moldova, Republic of
Taiwan
Greece
Georgia
Thailand
Korea, Republic of
France

summary: The purpose of this trial is to assess the efficacy, safety and tolerability of lacosamide administered as add-on therapy with 1 to 3 anti-seizure medications. This trial is for children aged 1 month to less than 4 years with epilepsy who currently have uncontrolled partial-onset seizures.


title: A Randomized, Double-blind, Placebo- and Active Controlled Multicenter Trial to Demonstrate Efficacy of Subcutaneous Secukinumab Compared to Placebo and Etanercept (in a Single-blinded Arm) After Twelve Weeks of Treatment, and to Assess the Safety, Tolerability, and Long-term Efficacy in Subjects From 6 to Less Than 18 Years of Age With Severe Chronic Plaque Psoriasis

location_country:
Poland
Belgium
Romania
Spain
Italy
Latvia
Estonia
Russian Federation
United Kingdom
Switzerland
Guatemala
Hungary
United States
France
Israel
Colombia
Japan
Germany
Egypt

summary: This was a multicenter, randomized, double-blind, placebo- and active-controlled (etanercept in single blinded arm) study in pediatric subjects aged 6 years to less than 18 years with severe chronic plaque psoriasis. Approximately 160 subjects aged 6 years to <18 years were enrolled, of which at least 30 were 6 years to <12 years old. Subjects were enrolled at approximately 70 study sites worldwide.


title: A Phase 2, Double-blind, Randomized, Placebo-controlled Multicenter Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of JBT-101 in Cystic Fibrosis

summary: The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and efficacy of JBT-101 in adult subjects with cystic fibrosis (CF).

ReferenceCitation: Chmiel JF, Flume P, Downey DG, Dozor AJ, Colombo C, Mazurek H, Sapiejka E, Rachel M, Constantine S, Conley B, Dgetluck N, Dinh Q, White B, Elborn JS; Lenabasum JBT101-CF-001 Study Group. Safety and efficacy of lenabasum in a phase 2 randomized, placebo-controlled trial in adults with cystic fibrosis. J Cyst Fibros. 2021 Jan;20(1):78-85. doi: 10.1016/j.jcf.2020.09.008. Epub 2020 Oct 1.

lead_sponsor: Corbus Pharmaceuticals Inc.

location_country:
Poland
United Kingdom
Italy
United States
France
Belgium
Germany

location_facility: Podkarpacki Ośrodek Pulmunologii i Alergologii

location_facility: Instytut Gruźlicy i Chorób Płuc I Klinika Chorób Płuc

location_facility: Christiane Herzog CF-Zentrum Frankfurt am Main

location_facility: University Hospitals Cleveland Medical Center

location_facility: The University of Texas Southwestern Medical Center

location_facility: Rutgers Robert-Wood Johnson Medical School

location_facility: University of Arkansas for Medical Sciences

location_facility: AOUI di Verona - Ospedale Borgo Trento UOC Fibrosi Cistica

location_facility: Szpital Dziecięcy Polanki im. Macieja Płażyńskiego w Gdańsku Spółka z o.o. Poradnia Leczenia Mukowiscydozy

location_facility: Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

location_facility: Texas Children's Hospital Clinical Care Center

location_facility: Long Beach Memorial Medical Center/Miller Children's and Women's Hospital

location_facility: Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico