title: A Phase 3, Randomized, Double-Blind Study Comparing Upadacitinib (ABT-494) to Placebo in Subjects With Moderately to Severely Active Rheumatoid Arthritis Who Are on a Stable Dose of Conventional Synthetic Disease-Modifying Anti-Rheumatic Drugs (csDMARDs) and Have an Inadequate Response to csDMARDs

acronym: SELECT-NEXT

summary: The primary objectives of this study are to compare the efficacy, safety, and tolerability of upadacitinib 30 mg once daily (QD) and 15 mg QD versus placebo for the treatment of signs and symptoms of adults with moderately to severely active rheumatoid arthritis who were on a stable dose of csDMARDs and had an inadequate response to csDMARDs.

ReferenceCitation: Burmester GR, Kremer JM, Van den Bosch F, Kivitz A, Bessette L, Li Y, Zhou Y, Othman AA, Pangan AL, Camp HS. Safety and efficacy of upadacitinib in patients with rheumatoid arthritis and inadequate response to conventional synthetic disease-modifying anti-rheumatic drugs (SELECT-NEXT): a randomised, double-blind, placebo-controlled phase 3 trial. Lancet. 2018 Jun 23;391(10139):2503-2512. doi: 10.1016/S0140-6736(18)31115-2. Epub 2018 Jun 18.

ReferenceCitation: Bergman M, Buch MH, Tanaka Y, Citera G, Bahlas S, Wong E, Song Y, Zueger P, Ali M, Strand V. Routine Assessment of Patient Index Data 3 (RAPID3) in Patients with Rheumatoid Arthritis Treated with Long-Term Upadacitinib Therapy in Five Randomized Controlled Trials. Rheumatol Ther. 2022 Dec;9(6):1517-1529. doi: 10.1007/s40744-022-00483-4. Epub 2022 Sep 20.

ReferenceCitation: Bergman M, Tundia N, Yang M, Orvis E, Clewell J, Bensimon A. Economic Benefit from Improvements in Quality of Life with Upadacitinib: Comparisons with Tofacitinib and Methotrexate in Patients with Rheumatoid Arthritis. Adv Ther. 2021 Dec;38(12):5649-5661. doi: 10.1007/s12325-021-01930-4. Epub 2021 Oct 12.

ReferenceCitation: Yamaoka K, Tanaka Y, Kameda H, Khan N, Sasaki N, Harigai M, Song Y, Zhang Y, Takeuchi T. The Safety Profile of Upadacitinib in Patients with Rheumatoid Arthritis in Japan. Drug Saf. 2021 Jun;44(6):711-722. doi: 10.1007/s40264-021-01067-x. Epub 2021 May 27.

ReferenceCitation: Cohen SB, van Vollenhoven RF, Winthrop KL, Zerbini CAF, Tanaka Y, Bessette L, Zhang Y, Khan N, Hendrickson B, Enejosa JV, Burmester GR. Safety profile of upadacitinib in rheumatoid arthritis: integrated analysis from the SELECT phase III clinical programme. Ann Rheum Dis. 2021 Mar;80(3):304-311. doi: 10.1136/annrheumdis-2020-218510. Epub 2020 Oct 28. Erratum In: Ann Rheum Dis. 2021 May;80(5):e83.

publisher_dissemination: British Medical Journal

ReferenceCitation: Strand V, Pope J, Tundia N, Friedman A, Camp HS, Pangan A, Ganguli A, Fuldeore M, Goldschmidt D, Schiff M. Upadacitinib improves patient-reported outcomes in patients with rheumatoid arthritis and inadequate response to conventional synthetic disease-modifying antirheumatic drugs: results from SELECT-NEXT. Arthritis Res Ther. 2019 Dec 9;21(1):272. doi: 10.1186/s13075-019-2037-1. Erratum In: Arthritis Res Ther. 2020 Jun 9;22(1):137.

ReferenceCitation: Nader A, Mohamed MF, Winzenborg I, Doelger E, Noertersheuser P, Pangan AL, Othman AA. Exposure-Response Analyses of Upadacitinib Efficacy and Safety in Phase II and III Studies to Support Benefit-Risk Assessment in Rheumatoid Arthritis. Clin Pharmacol Ther. 2020 Apr;107(4):994-1003. doi: 10.1002/cpt.1671. Epub 2019 Nov 30.

ipd_sharing_description: AbbVie is committed to responsible data sharing regarding the clinical trials we sponsor. This includes access to anonymized, individual and trial-level data (analysis data sets), as well as other information (e.g., protocols, analyses plans, clinical study reports), as long as the trials are not part of an ongoing or planned regulatory submission. This includes requests for clinical trial data for unlicensed products and indications.

location_country:
Finland
Germany
Ireland
Poland
New Zealand
Argentina
Australia
Bosnia and Herzegovina
Turkey
Croatia
Czechia
Canada
France
Lithuania
Portugal
Slovakia
Hungary
Hong Kong
Switzerland
United States
Latvia
Ukraine
Romania
Belgium
Estonia
Puerto Rico
Italy
United Kingdom
Austria
Mexico
Korea, Republic of
Spain
Greece
South Africa
Taiwan
Bulgaria
Russian Federation
Kazakhstan

location_facility: Klinicki bolnicki centar Rijeka /ID# 138649

location_facility: Centro Peninsular de Investigación Clínica SCP /ID# 148160

location_facility: Arthritis & Osteoporosis Clinic /ID# 138703

location_facility: CHRU Tours - Hopital Trousseau /ID# 138969

location_facility: Center of Clinical and Basic Research /ID# 141116

location_facility: Arthritis Clinical Research Tr /ID# 138945

location_facility: Deerbrook Medical Associates /ID# 139694

location_facility: Unidad de Investigacion de las Enfermedades Reumatologicas SA de CV /ID# 138841

location_facility: Hosp Nuestra Senora Esperanza /ID# 138997

location_facility: Cath Univ Seoul St Mary's Hosp /ID# 138652

location_facility: Seoul National University Hospital /ID# 138659

location_facility: Centro Hospitalar Lisboa Ocidental, EPE /ID# 140594

location_facility: East Tallinn Central Hospital /ID# 140618

location_facility: Western Washington Arthritis C /ID# 138728

location_facility: AZ Arthritis and Rheum Researc /ID# 138500

location_facility: Mautalen Salud e Investigacion /ID# 141419

location_facility: Chungnam National University Hospital /ID# 138653

location_facility: Rheuma Zentrum Favoriten GmbH /ID# 138787

location_facility: AZ Arthritis and Rheum Researc /ID# 139286

location_facility: Four Rivers Clinical Research /ID# 141134

location_facility: AZ Arthritis & Rheuma Research /ID# 138598

location_facility: Cincinnati Rheumatic Disease Study Group, Inc. /ID# 138868

location_facility: University Clinical Centre of the Republic of Srpska /ID# 140372

location_facility: NZOZ Centrum Reumatologiczne /ID# 138353

location_facility: Republican Clin Hos n.a. Baran /ID# 139273

location_facility: Hospital Clin Univ San Carlos /ID# 138993

location_facility: Fejer Megyei Szent Gyorgy Korh /ID# 138554

location_facility: Ulyanovsk Regional Clin Hosp /ID# 139279

location_facility: Revita Reumatologiai Rendelo /ID# 140761

location_facility: Clinical Investigation Special /ID# 139696

location_facility: PMG Research of Wilmington LLC /ID# 140951

location_facility: MNCE "Lviv City Clinical Hospital #4" /ID# 139873

location_facility: Bluegrass Community Research /ID# 138295

location_facility: Trinity Universal Research Association /ID# 148649

location_facility: Inland Rheum Clin Trials Inc. /ID# 138853

location_facility: Charité Universitätsmedizin Campus Mitte /ID# 139052

location_facility: Accurate Clinical Management /ID# 139338

location_facility: Wilhelminenspital der Stadt Wien /ID# 138788

location_facility: University of Arizona Cancer Center - North Campus /ID# 140451

location_facility: Chonnam National University Hospital /ID# 138651

location_facility: Ctr Arthritis & Rheumatic Dise /ID# 141696

location_facility: Optimus Clinical Research Pty. /ID# 138769

location_facility: Instituto Portugues De Reumatologia /ID# 148315

location_facility: Diag Consult Ctr 17 Sofia EOOD /ID# 141006

location_facility: Far Eastern Memorial Hospital /ID# 140871

location_facility: Arizona Research Center, Inc. /ID# 140448

location_facility: Physician Res. Collaboration /ID# 138533

location_facility: JSC Nat Scientific Med Res Ctr /ID# 140575

location_facility: Pacific Arthritis Ctr Med Grp /ID# 138744

location_facility: China Medical University Hosp /ID# 139232

location_facility: Yaroslavi State Medical Univer /ID# 139908

location_facility: Winelands Medical Research Ctr /ID# 138944

location_facility: School of Medicine University of Puerto Rico-Medical Sciences Campus /ID# 139328

location_facility: Slovak research center Team Member, Thermium s.r.o. /ID# 139924

location_facility: Inst. Rheumatologic Strusberg /ID# 145648

location_facility: South Karelia Central Hospital /ID# 139973

location_facility: Clinical Res of West FL, Inc. /ID# 138854

location_facility: SunValley Arthritis Center, Lt /ID# 140452

location_facility: Hanyang University Seoul Hospi /ID# 138655

location_facility: Universita di Catanzaro Magna Graecia /ID# 139316

location_facility: Ankara Numune Training and Res /ID# 139237

location_facility: Daegu Catholic University Med /ID# 139249

location_facility: Articularis Healthcare Group, Inc d/b/a Low Country Rheumatology /ID# 138689

location_facility: Spitalul Clinic Judetean de Ur /ID# 138393

location_facility: St. Augustine's Medical Centre /ID# 141352

location_facility: Diagnostic Consultative Center /ID# 138882

location_facility: Narodny ustav reumatickych chorob Piestany /ID# 138427

location_facility: Comprehensive Arthritis Care, a division of Comprehensive Rheumatology Care PLLC /ID# 141021

location_facility: Aurora Rheumatology and Immunotherapy Center /ID# 139306

location_facility: Samara Regional Clinical Hosp /ID# 148642

location_facility: Kaohsiung Medical University Chung-Ho Memorial Hospital /ID# 140869

location_facility: HFR Fribourg - Hopital Canton /ID# 139155

location_facility: Southampton General Hospital /ID# 139169

location_facility: AZ Arthritis and Rheum Assoc /ID# 148651

location_facility: Odessa National Medical Univ /ID# 139179

location_facility: Taipei Veterans General Hosp /ID# 139234

location_facility: Groupe de Recherche en Maladies Osseuses /ID# 138906

location_facility: Helsinki Univ Central Hospital /ID# 140381

location_facility: Voronezh State Medical Univers /ID# 148431

location_facility: University General Hospital of Heraklion "PA.G.N.I" /ID# 139115

location_facility: Spitalul Clinic Judetean de Urgenta /ID# 138407

location_facility: National Cheng Kung University Hospital /ID# 140868

location_facility: Medallion Clinical Research Institute, LLC /ID# 140074

location_facility: The Center for Rheumatology & /ID# 139203

location_facility: Houston Institute for Clin Res /ID# 138716

location_facility: University Clinical Centre of the Republic of Srpska /ID# 138819

location_facility: St Vincent's University Hosp /ID# 138562

location_facility: Warrington + Halton Hosp NHS /ID# 139195

location_facility: Arth and Osteo Clin Brazo Valley /ID# 147809

location_facility: Kazan State Medical University /ID# 138413

location_facility: Klaipeda University Hospital /ID# 141416

location_facility: Hospital General Univ de Elche /ID# 138991


study_start_date_type: Actual

intervention_type: Drug

lead_sponsor: MedImmune LLC

summary: This is a multicenter, open-label, dose-escalation, dose-exploration and dose-expansion study to evaluate the safety, tolerability, antitumor activity, pharmacokinetic (PK), pharmacodynamics, and immunogenicity of durvalumab (MEDI4736) in combination with monalizumab (IPH2201) in adult participants with selected advanced solid tumors and the combination of durvalumab and monalizumab (IPH2201) standard of care systemic therapy with or without biological agent and monalizumab (IPH2201) with biological agent administered to participants with recurrent or metastatic colorectal cancer (CRC).

status: Active, not recruiting

title: A Phase 1/2 Study of Durvalumab and Monalizumab in Adult Subjects With Select Advanced Solid Tumors

primary_purpose: Treatment

type: Other Grant/Funding Number

type: EudraCT Number

source: euctr

enrollment_type: Actual

ipd_sharing_description: Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal. All request will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure

design_allocation: Randomized

study_completion_date_type: Anticipated

location_country:
Italy
New Zealand
Hungary
United Kingdom
Australia
Belgium
United States
Korea, Republic of
Spain
Canada
France

study_type: Interventional

ipd_sharing: Yes

lead_sponsor_type: industriel

status_simplified: Ongoing


title: A Phase 1/1b, Open Label, Multiple Dose, Dose Escalation and Expansion Study to Investigate the Safety, Pharmacokinetics and Antitumor Activity of the Anti-PD-1 Monoclonal Antibody BGB-A317 in Combination With the PARP Inhibitor BGB-290 in Subjects With Advanced Solid Tumors

summary: This trial studied the safety, pharmacokinetics, and antitumor activity of the anti-programmed cell death 1 (PD-1) monoclonal antibody (mAb) BGB-A317 (tislelizumab) in combination with the poly(adenosine diphosphate ribose) polymerase (PARP) inhibitor BGB-290 (pamiparib) in participants with advanced solid tumors.

status: Completed

study_completion_date_type: Actual

design_allocation: Non-Randomized

ReferenceType: result

ReferenceCitation: Friedlander M, Meniawy T, Markman B, Mileshkin L, Harnett P, Millward M, Lundy J, Freimund A, Norris C, Mu S, Wu J, Paton V, Gao B. Pamiparib in combination with tislelizumab in patients with advanced solid tumours: results from the dose-escalation stage of a multicentre, open-label, phase 1a/b trial. Lancet Oncol. 2019 Sep;20(9):1306-1315. doi: 10.1016/S1470-2045(19)30396-1. Epub 2019 Aug 1.

oa_host_type: closed

repositories_concat: closed

publisher_dissemination: Elsevier

lead_sponsor: BeiGene

sponsor_collaborators:
BeiGene
Myriad Genetic Laboratories, Inc.

location_country:
New Zealand
United Kingdom
Australia
United States
Spain
France

location_facility: Hospital Clínico Universitario de Valencia

status_simplified: Completed


type: Other Identifier

title: An Open-Label, Multicenter, Dose Escalation and Expansion Phase Ib Study to Evaluate the Safety, Pharmacokinetics, and Therapeutic Activity of RO6958688 in Combination With Atezolizumab in Patients With Locally Advanced and/or Metastatic CEA-Positive Solid Tumors

summary: This is an open-label, multicenter, dose-escalation and expansion Phase Ib clinical study of RO6958688 in combination with atezolizumab. Part I of the study is subdivided into parts IA and IB. Part IA is dose escalation with a starting dose of 5 mg of RO6958688 given QW (once a week) and a fixed, flat dose of 1200 mg given Q3W (every 3 weeks) of atezolizumab, to evaluate the safety and determine the MTD of RO6958688 in combination with atezolizumab. Part IB is a dose/schedule finding part that will explore different administration schedules of RO6958688 in combination with atezolizumab (1200 mg Q3W) to establish the appropriate dose/schedule of RO6958688 in combination with atezolizumab.

ReferenceType: derived

ReferenceCitation: Martinez-Sabadell A, Morancho B, Rius Ruiz I, Roman Alonso M, Ovejero Romero P, Escorihuela M, Chicote I, Palmer HG, Nonell L, Alemany-Chavarria M, Klein C, Bacac M, Arribas J, Arenas EJ. The target antigen determines the mechanism of acquired resistance to T cell-based therapies. Cell Rep. 2022 Oct 18;41(3):111430. doi: 10.1016/j.celrep.2022.111430.

oa_colors: green gold

oa_host_type: publisher;repository

evidence: oa journal (via doaj)

host_type: publisher

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

version: publishedVersion

evidence: oa repository (via OAI-PMH doi match)

host_type: repository

repository_institution: Universitat Pompeu Fabra - Repositori digital de la UPF

unpaywall_oa_status: gold

lead_sponsor: Hoffmann-La Roche

location_country:
Italy
Denmark
Netherlands
United States
Spain
Canada
France

location_facility: Clinica Universitaria de Navarra; Servicio de Oncologia

location_facility: Smilow Cancer Hospital at Yale- New Haven Oncology Investigational Drug Pharmacy

location_facility: Centre Leon Berard; Departement Oncologie Medicale

location_facility: Antoni Van Leeuwenhoek Ziekenhuis; Gastro-Enterologie

location_facility: Hospital Universitario 12 de Octubre; Servicio de Oncologia

location_facility: START Madrid. Centro Integral Oncologico Clara Campal; CIOCC

location_facility: Azienda Ospedaliera Universitaria Senese, U.O.C. Immunoterapia Oncologica

location_facility: Hospital Univ Vall d'Hebron; Servicio de Oncologia

location_facility: IRCCS IST. Tumori Fondaz. Pascale; S.C. Oncologia Medica,Melanoma,Immunoterapia E Terapie Innovative

location_facility: START Madrid-FJD, Hospital Fundacion Jimenez Diaz


acronym: RainbowExt

lead_sponsor: Novartis Pharmaceuticals

summary: The purpose of this study was to evaluate the long term efficacy and safety of intravitreal ranibizumab compared with laser ablation therapy in patients who were treated for retinopathy of prematurity (ROP) in the core study CRFB002H2301 (NCT02375971)

ReferenceCitation: Marlow N, Stahl A, Lepore D, Fielder A, Reynolds JD, Zhu Q, Weisberger A, Stiehl DP, Fleck B; RAINBOW investigators group. 2-year outcomes of ranibizumab versus laser therapy for the treatment of very low birthweight infants with retinopathy of prematurity (RAINBOW extension study): prospective follow-up of an open label, randomised controlled trial. Lancet Child Adolesc Health. 2021 Oct;5(10):698-707. doi: 10.1016/S2352-4642(21)00195-4. Epub 2021 Aug 13.

title: RAINBOW Extension Study: an Extension Study to Evaluate the Long Term Efficacy and Safety of RAnibizumab Compared With Laser Therapy for the Treatment of INfants BOrn Prematurely With Retinopathy of Prematurity

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:
Germany
Japan
Turkey
Croatia
Czechia
France
Lithuania
Slovakia
Hungary
Denmark
United States
India
Romania
Belgium
Malaysia
Estonia
Egypt
Italy
United Kingdom
Austria
Saudi Arabia
Greece
Taiwan
Russian Federation


acronym: SCOUT

lead_sponsor: Bayer

summary:
The study is being done to test the safety of a cancer drug called larotrectinib in children. The cancer must have a change in a particular gene (NTRK1, NTRK2 or NTRK3). Larotrectinib blocks the actions of these NTRK genes in cancer cells and can therefore be used to treat cancer.

The first study part (Phase 1) is done to determine what dose level of larotrectinib is safe for children, how the drug is absorbed and changed by their bodies and how well the cancer responds to the drug. The main purpose of the second study part (Phase 2) is to investigate how well and how long different cancer types respond to the treatment with larotrectininb.

ReferenceCitation: Doz F, van Tilburg CM, Geoerger B, Hojgaard M, Ora I, Boni V, Capra M, Chisholm J, Chung HC, DuBois SG, Gallego-Melcon S, Gerber NU, Goto H, Grilley-Olson JE, Hansford JR, Hong DS, Italiano A, Kang HJ, Nysom K, Thorwarth A, Stefanowicz J, Tahara M, Ziegler DS, Gavrilovic IT, Norenberg R, Dima L, De La Cuesta E, Laetsch TW, Drilon A, Perreault S. Efficacy and safety of larotrectinib in TRK fusion-positive primary central nervous system tumors. Neuro Oncol. 2022 Jun 1;24(6):997-1007. doi: 10.1093/neuonc/noab274.

oa_colors: green hybrid

evidence: open (via crossref license)

license: cc-by-nc

license_normalized: cc-by-nc

repository_institution: Yonsei University Medical Library - YUHSpace

repository_institution: University of Zurich - Zurich Open Repository and Archive

evidence: oa repository (via pmcid lookup)

license_normalized: no license

repository_normalized: PubMed Central

unpaywall_oa_status: hybrid

publisher_dissemination: Oxford University Press

ReferenceCitation: Bebb DG, Banerji S, Blais N, Desmeules P, Gill S, Grin A, Feilotter H, Hansen AR, Hyrcza M, Krzyzanowska M, Melosky B, Noujaim J, Purgina B, Ruether D, Simmons CE, Soulieres D, Torlakovic EE, Tsao MS. Canadian Consensus for Biomarker Testing and Treatment of TRK Fusion Cancer in Adults. Curr Oncol. 2021 Jan 15;28(1):523-548. doi: 10.3390/curroncol28010053.

license: cc-by

license_normalized: cc-by

repositories_concat: PubMed Central

publisher_dissemination: MDPI

ReferenceCitation: Perreault S, Chami R, Deyell RJ, El Demellawy D, Ellezam B, Jabado N, Morgenstern DA, Narendran A, Sorensen PHB, Wasserman JD, Yip S. Canadian Consensus for Biomarker Testing and Treatment of TRK Fusion Cancer in Pediatric Patients. Curr Oncol. 2021 Jan 9;28(1):346-366. doi: 10.3390/curroncol28010038.

ReferenceCitation: Bielack SS, Cox MC, Nathrath M, Apel K, Blattmann C, Holl T, Jenewein R, Klenk U, Klothaki P, Muller-Abt P, Ortega-Lawerenz S, Reynolds M, Scheer M, Simon-Klingenstein K, Stegmaier S, Tupper R, Vokuhl C, von Kalle T. Rapid, complete and sustained tumour response to the TRK inhibitor larotrectinib in an infant with recurrent, chemotherapy-refractory infantile fibrosarcoma carrying the characteristic ETV6-NTRK3 gene fusion. Ann Oncol. 2019 Nov;30 Suppl 8:viii31-viii35. doi: 10.1093/annonc/mdz382. Epub 2019 Dec 24.

repository_institution: PubMed Central - Europe PMC

ReferenceCitation: Hong DS, DuBois SG, Kummar S, Farago AF, Albert CM, Rohrberg KS, van Tilburg CM, Nagasubramanian R, Berlin JD, Federman N, Mascarenhas L, Geoerger B, Dowlati A, Pappo AS, Bielack S, Doz F, McDermott R, Patel JD, Schilder RJ, Tahara M, Pfister SM, Witt O, Ladanyi M, Rudzinski ER, Nanda S, Childs BH, Laetsch TW, Hyman DM, Drilon A. Larotrectinib in patients with TRK fusion-positive solid tumours: a pooled analysis of three phase 1/2 clinical trials. Lancet Oncol. 2020 Apr;21(4):531-540. doi: 10.1016/S1470-2045(19)30856-3. Epub 2020 Feb 24.

oa_host_type: repository

version: acceptedVersion

evidence: oa repository (via OAI-PMH title and first author match)

repository_institution: University of California - eScholarship University of California

version: submittedVersion

repositories_concat: PubMed Central;escholarship.org

unpaywall_oa_status: green

ReferenceCitation: Bielack SS, Cox MC, Nathrath M, Apel K, Blattmann C, Holl T, Jenewein R, Klenk U, Klothaki P, Muller-Abt P, Ortega-Lawerenz S, Reynolds M, Scheer M, Simon-Klingenstein K, Stegmaier S, Tupper R, Vokuhl C, von Kalle T. Rapid, complete and sustained tumour response to the TRK inhibitor larotrectinib in an infant with recurrent, chemotherapy-refractory infantile fibrosarcoma carrying the characteristic ETV6-NTRK3 gene fusion. Ann Oncol. 2019 Nov 1;30(Suppl_8):viii31-viii35. doi: 10.1093/annonc/mdz382.

ReferenceCitation: DuBois SG, Laetsch TW, Federman N, Turpin BK, Albert CM, Nagasubramanian R, Anderson ME, Davis JL, Qamoos HE, Reynolds ME, Cruickshank S, Cox MC, Hawkins DS, Mascarenhas L, Pappo AS. The use of neoadjuvant larotrectinib in the management of children with locally advanced TRK fusion sarcomas. Cancer. 2018 Nov 1;124(21):4241-4247. doi: 10.1002/cncr.31701. Epub 2018 Sep 11.

repository_institution: California Digital Library - eScholarship

ReferenceCitation: Laetsch TW, DuBois SG, Mascarenhas L, Turpin B, Federman N, Albert CM, Nagasubramanian R, Davis JL, Rudzinski E, Feraco AM, Tuch BB, Ebata KT, Reynolds M, Smith S, Cruickshank S, Cox MC, Pappo AS, Hawkins DS. Larotrectinib for paediatric solid tumours harbouring NTRK gene fusions: phase 1 results from a multicentre, open-label, phase 1/2 study. Lancet Oncol. 2018 May;19(5):705-714. doi: 10.1016/S1470-2045(18)30119-0. Epub 2018 Mar 29. Erratum In: Lancet Oncol. 2018 May;19(5):e229.

ReferenceCitation: Drilon A, Laetsch TW, Kummar S, DuBois SG, Lassen UN, Demetri GD, Nathenson M, Doebele RC, Farago AF, Pappo AS, Turpin B, Dowlati A, Brose MS, Mascarenhas L, Federman N, Berlin J, El-Deiry WS, Baik C, Deeken J, Boni V, Nagasubramanian R, Taylor M, Rudzinski ER, Meric-Bernstam F, Sohal DPS, Ma PC, Raez LE, Hechtman JF, Benayed R, Ladanyi M, Tuch BB, Ebata K, Cruickshank S, Ku NC, Cox MC, Hawkins DS, Hong DS, Hyman DM. Efficacy of Larotrectinib in TRK Fusion-Positive Cancers in Adults and Children. N Engl J Med. 2018 Feb 22;378(8):731-739. doi: 10.1056/NEJMoa1714448.

location_facility: Lucille Salter Packard Children's Hospital at Stanford

location_facility: Beijing Children's Hospital, Capital Medical University

location_facility: Cincinnati Children's Hospital and Medical Center

location_facility: Institut Gustave Roussy - Département de Médecine Oncologique

location_facility: Clalit Health Services Schneider Children's Medical Center

location_facility: Tianjin Medical University Cancer Institiute & Hospital

location_facility: The Hospital for Sick Children (SickKids)

location_facility: Fondazione IRCCS Istituto Nazionale dei Tumori

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

location_facility: UCLA Jonsson Comprehensive Cancer Center

location_facility: Western Ukrainian Specialized Pediatric Medical Centre

location_facility: Karolinska Universitetssjukhuset i Solna

location_facility: Rigshospitalet, Dept Pediatrics & Adelescent Med.

location_facility: University of Texas Southwestern Medical Center

location_facility: Istanbul Universitesi Istanbul Tip Fakultesi

title: A Phase 1/2 Study of the Oral TRK Inhibitor Larotrectinib in Pediatric Patients With Advanced Solid or Primary Central Nervous System Tumors

ipd_sharing_description:
Availability of this study's data will later 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.vivli.org 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 member section of the portal.

location_country:
Germany
Ireland
Poland
Australia
Japan
Turkey
Czechia
Canada
France
Denmark
Switzerland
United States
Ukraine
China
Italy
United Kingdom
Sweden
Netherlands
Korea, Republic of
Spain
Israel


title: Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector

summary: This is a multi-center, long-term safety and efficacy follow-up study for subjects with transfusion-dependent β-thalassemia (TDT) who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored parent clinical studies. After completing the parent clinical study (approximately 2 years), eligible subjects will be followed for an additional 13 years for a total of 15 years post-drug product infusion. No investigational drug product will be administered in the study.

study_type: Observational

time_perspective: Prospective

ReferenceCitation: Magrin E, Semeraro M, Hebert N, Joseph L, Magnani A, Chalumeau A, Gabrion A, Roudaut C, Marouene J, Lefrere F, Diana JS, Denis A, Neven B, Funck-Brentano I, Negre O, Renolleau S, Brousse V, Kiger L, Touzot F, Poirot C, Bourget P, El Nemer W, Blanche S, Treluyer JM, Asmal M, Walls C, Beuzard Y, Schmidt M, Hacein-Bey-Abina S, Asnafi V, Guichard I, Poiree M, Monpoux F, Touraine P, Brouzes C, de Montalembert M, Payen E, Six E, Ribeil JA, Miccio A, Bartolucci P, Leboulch P, Cavazzana M. Long-term outcomes of lentiviral gene therapy for the beta-hemoglobinopathies: the HGB-205 trial. Nat Med. 2022 Jan;28(1):81-88. doi: 10.1038/s41591-021-01650-w. Epub 2022 Jan 24.

publisher_dissemination: Springer-Nature

ipd_sharing_description: Bluebird bio is committed to transparency and appropriately de-identified subject-level datasets and supporting documents may be shared after all participants have completed study participation and following attainment of applicable marketing approvals associated with a given study and consistent with criteria established by bluebird bio and/or industry best practices to maintain the privacy of study participants. For enquiries, please contact us at datasharing@bluebirdbio.com.

lead_sponsor: bluebird bio

location_country:
Germany
Italy
United Kingdom
Australia
United States
Thailand
Greece
France

intervention_type: Other


title: A Prospective, 5-Year Global Study on MAST™ Minimally Invasive Fusion Procedures for the Treatment of the Degenerative Lumbar Spine

summary:
The purpose of this study is

to evaluate the effectiveness of MAST techniques for anterior/lateral and posterior approaches in Degenerative Disc Disease (DDD) patients with spondylolisthesis (≥ grade I).
To assess how single or double level MAST(Minimal Access Spinal Technologies) fusion procedures PLIF (Posterior Lumbar Interbody Fusion), TLIF (Transforaminal Lumbar Interbody Fusion), DLIF (Direct Lateral Interbody Fusion), OLIF (Oblique Lumbar Interbody Fusion), ALIF (Anterior Lumbar Interbody Fusion), or MIDLF (Midline Lumbar Interbody Fusion) are used in surgical practice and to describe long-term safety and effectiveness in a broad patient population of patients with degenerative lumbar disc disease .

lead_sponsor: Medtronic Spinal and Biologics

location_country:
Germany
Italy
Portugal
Slovakia
United Kingdom
Argentina
Denmark
Brazil
Austria
Belgium
Mexico
Korea, Republic of
China
Spain
Czechia
Russian Federation
France

location_facility: The First Affiliated Hospital of Dalian Medical University

location_facility: Department of Neurological Surgery, Chung-Ang University Hospital

location_facility: Hospital Naval General de Alta Especialidad

location_facility: Department of Orthopedics, Korea University Ansan Hospital

location_facility: Hospital Universitario Marques de Valdecilla

location_facility: Nizhny Novgorod Research Institute of Traumatology and Orthopedics of Public Health Ministry

location_facility: East Suffolk and North Essex NHS Foundation Trust (was formerly Ipswich Hospital NHS)

location_facility: Scientific Research Institute of Traumatology and Orthopedics

location_facility: National Health Insurance Corporation Ilsan Hospital

intervention_type: Device


title: Methodology Study of Novel Electrophysiological, Physical, and Imaging Outcome Measures to Assess the Progression of Amyotrophic Lateral Sclerosis

summary: The primary objectives of the study are to estimate and rank-order the longitudinal standardized mean changes over 6 months and over 12 months, for a set of outcome measures administered to participants with amyotrophic lateral sclerosis (ALS), in order to identify measures that are more sensitive to disease progression than Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R). The secondary objectives of this study are: To evaluate the test-retest reproducibility of each outcome measure; To determine correlations between 6 and 12-month changes in all exploratory measures with 18 and 24-month changes in ALSFRS-R and survival; To assess correlations between/among the various measures; To obtain biological samples in order to identify molecular correlates to the clinical measures and to further characterize previously identified and novel molecular biomarkers of disease progression for incorporation into future clinical studies.

lead_sponsor: Biogen

location_country:
Germany
Ireland
United Kingdom
Netherlands
Switzerland
Belgium
United States
Canada
France

location_facility: University of California San Diego Medical Center

location_facility: Penn State Milton S. Hershey Medical Center

location_facility: Hopital Gui de Chauliac, Service de Neurologie

location_facility: Washington University School of Medicine

location_facility: Montreal Neurological Institute Clinical Research Unit


title: A Phase 1 Study of the EZH2 Inhibitor Tazemetostat in Pediatric Subjects With Relapsed or Refractory INI1-Negative Tumors or Synovial Sarcoma

summary:
This is a Phase I, open-label, dose escalation and dose expansion study with BID (suspension) and TID (tablet) oral dose of tazemetostat. Subjects will be screened for eligibility within 14 days of the planned first dose of tazemetostat. A treatment cycle will be 28 days. Response assessment will be evaluated after 8 weeks of treatment and subsequently every 8 weeks while on study.

The study has two parts: Dose Escalation and Dose Expansion.

Dose escalation for subjects with the following relapsed/refractory malignancies:

Rhabdoid tumors:
Atypical teratoid rhabdoid tumor (ATRT)
Malignant rhabdoid tumor (MRT)
Rhabdoid tumor of kidney (RTK)
Selected tumors with rhabdoid features
INI1-negative tumors:
Epithelioid sarcoma
Epithelioid malignant peripheral nerve sheath tumor
Extraskeletal myxoid chondrosarcoma
Myoepithelial carcinoma
Renal medullary carcinoma
Other INI1-negative malignant tumors (e.g., dedifferentiated chordoma) (with Sponsor approval)
Synovial Sarcoma with a SS18-SSX rearrangement Dose Escalation cohorts are closed to enrollment.

Dose Expansion at the MTD or the RP2D

Cohort 1 - ATRT (closed to enrollment)
Cohort 2 - MRT/RTK/selected tumors with rhabdoid features (closed to enrollment)

Cohort 3 - INI-negative tumors:

Epithelioid sarcoma
Epithelioid malignant peripheral nerve sheath tumor
Extraskeletal myxoid chondrosarcoma
Myoepithelial carcinoma
Renal medullary carcinoma
Chordoma (poorly differentiated or de-differentiated)
Other INI1-negative malignant tumors (e.g., dedifferentiated chordoma) with Sponsor approval
Cohort 4 - Tumor types eligible for Cohorts 1 through 3 or synovial sarcoma with SS18-SSX rearrangement (closed to enrollment)

lead_sponsor: Epizyme, Inc.

location_country:
Germany
Italy
United Kingdom
Denmark
Australia
Netherlands
United States
Canada
France

location_facility: Rigshospitalet Department of Oncology Blegdamsvej

location_facility: Oregon Health & Science University (OHSU)

location_facility: Cincinnati Children's Hospital Medical Center

location_facility: Massachusetts General Hospital - Cancer Center

location_facility: Fondazione IRCCS Istituto Nazionale dei Tumori

location_facility: Texas Children's Cancer and Hematology Center

location_facility: Westfalische Wilhelms - Universitat Munster Padiatrische

location_facility: Lady Cilento/Queensland Children's Hospital

location_facility: Prinses Maxima Centrum voor Kinderoncologie

location_facility: Central Manchester University Hospital - Royal Manchester Children's Hospital

location_facility: Istituto Giannina Gaslini- UOSD Centro di Neuro-Oncologia

location_facility: St. Jude Children's Research Hospital, Inc.

location_facility: University of California San Francisco - Benioff Children's Hospital

location_facility: The Childrens Hospital at Westmead Oncology Unit

location_facility: Erasmus University Medical Center - Sophia Children's Hospital

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

location_facility: Great Ormond Street Hospital for Children NHS Foundation Trust


title: A Randomized, Actively Controlled, Open-label, Multicenter Study of Efficacy and Safety of Evolocumab Compared With Low Density Lipoprotein Cholesterol (LDL-C) Apheresis, Followed by Single-Arm Evolocumab Administration in Subjects Receiving LDL-C Apheresis Prior to Study Enrollment

summary: To evaluate the efficacy of subcutaneous (SC) evolocumab, compared to regularly scheduled low-density lipoprotein cholesterol (LDL-C) apheresis, on reducing the need for future apheresis.

ReferenceType: background

ReferenceCitation: Baum SJ, Sampietro T, Datta D, Moriarty PM, Knusel B, Schneider J, Somaratne R, Kurtz C, Hohenstein B. Effect of evolocumab on lipoprotein apheresis requirement and lipid levels: Results of the randomized, controlled, open-label DE LAVAL study. J Clin Lipidol. 2019 Nov-Dec;13(6):901-909.e3. doi: 10.1016/j.jacl.2019.10.003. Epub 2019 Oct 11.

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

lead_sponsor: Amgen

location_country:
Germany
Italy
United Kingdom
Australia
United States
Spain
Czechia
France

intervention_type: Procedure


acronym: AQUA

summary: To evaluate the change in quality of life (NEI VFQ 25) in subjects with DME during the first year of treatment with aflibercept according to the EU Label.

title: Open-label Phase-4 Study to Examine the Change of Vision-related Quality of Life in Subjects With Diabetic Macular Edema (DME) During Treatment With Intravitreal Injections of 2 mg Aflibercept According to EU Label for the First Year of Treatment

location_country:
Germany
Italy
Lithuania
Poland
Portugal
Slovakia
Hungary
United Kingdom
Switzerland
Austria
Spain
Czechia
Canada
France


acronym: ARIES

summary: This study aims to evaluate the optimal use, efficacy, and safety of a Treat-and-Extend regimen with aflibercept in subjects with nAMD.

ReferenceCitation: Wolf S, Holz FG, Midena E, Souied EH, Lambrou G, Machewitz T, Allmeier H, Mitchell P; ARIES Study Investigators. Patients with Neovascular Age-Related Macular Degeneration Requiring Intensive Intravitreal Aflibercept Treatment: An ARIES Post Hoc Analysis. Ophthalmol Ther. 2022 Oct;11(5):1793-1803. doi: 10.1007/s40123-022-00541-8. Epub 2022 Jul 12.

repository_institution: University Library Bern - BORIS - Bern Open Repository and Information System

repositories_concat: PubMed Central;boris.unibe.ch

ReferenceCitation: Chaudhary V, Holz FG, Wolf S, Midena E, Souied EH, Allmeier H, Lambrou G, Machewitz T, Mitchell P; ARIES study investigators. Association Between Visual Acuity and Fluid Compartments with Treat-and-Extend Intravitreal Aflibercept in Neovascular Age-Related Macular Degeneration: An ARIES Post Hoc Analysis. Ophthalmol Ther. 2022 Jun;11(3):1119-1130. doi: 10.1007/s40123-022-00491-1. Epub 2022 Mar 18.

repository_institution: University of Bern - Bern Open Repository and Information System

ReferenceCitation: Tuerksever C, Somfai GM, Oesch S, Machewitz T, Hasler PW, Zweifel S. Hypothetical Switch of Anti-Vascular Endothelial Growth Factor in Neovascular Age-Related Macular Degeneration: An ARIES Post Hoc Analysis. Ophthalmol Ther. 2022 Apr;11(2):613-627. doi: 10.1007/s40123-021-00448-w. Epub 2022 Jan 23.

ReferenceCitation: Mitchell P, Holz FG, Hykin P, Midena E, Souied E, Allmeier H, Lambrou G, Schmelter T, Wolf S; ARIES study investigators. EFFICACY AND SAFETY OF INTRAVITREAL AFLIBERCEPT USING A TREAT-AND-EXTEND REGIMEN FOR NEOVASCULAR AGE-RELATED MACULAR DEGENERATION: The ARIES Study: A Randomized Clinical Trial. Retina. 2021 Sep 1;41(9):1911-1920. doi: 10.1097/IAE.0000000000003128. Erratum In: Retina. 2022 Sep 1;42(9):e43.

evidence: open (via page says license)

publisher_dissemination: Wolters Kluwer Health

sponsor_collaborators:
Bayer
Regeneron Pharmaceuticals

title: Managing Neovascular Age-related Macular Degeneration (nAMD) Over 2 Years With a Treat and Extend (T&E) Regimen of 2 mg Intravitreal Aflibercept - a Randomized, Open-label, Active-controlled, Parallel-group Phase IV/IIIb Study (ARIES)

location_country:
Germany
Italy
Hungary
United Kingdom
Australia
Spain
Canada
France


acronym: CheckMate 436

lead_sponsor: Bristol-Myers Squibb

summary: The purpose of this study is to determine whether Nivolumab, in combination with brentuximab vedotin, is safe and effective in patients with certain subtypes of non-Hodgkin's lymphomas with CD30 expression that have not responded to treatment or have come back. The subtypes we are studying are Diffuse Large B-Cell Lymphoma (DLBCL), Peripheral T-Cell Lymphoma (PTCL), Cutaneous T-Cell Lymphoma (CTCL), Primary Mediastinal Large B-Cell Lymphoma (PMBL) and Mediastinal Gray Zone Lymphoma (MGZL).

ReferenceCitation: Zinzani PL, Santoro A, Gritti G, Brice P, Barr PM, Kuruvilla J, Cunningham D, Kline J, Johnson NA, Mehta-Shah N, Manley T, Francis S, Sharma M, Moskowitz AJ. Nivolumab Combined With Brentuximab Vedotin for Relapsed/Refractory Primary Mediastinal Large B-Cell Lymphoma: Efficacy and Safety From the Phase II CheckMate 436 Study. J Clin Oncol. 2019 Nov 20;37(33):3081-3089. doi: 10.1200/JCO.19.01492. Epub 2019 Aug 9.

publisher_dissemination: American Society of Clinical Oncology (ASCO)

sponsor_collaborators:
Bristol-Myers Squibb
Seagen Inc.

location_facility: The Ohio State University Comprehensive Cancer Center

location_facility: University of Washington - Seattle Cancer Care Alliance

location_facility: University Of Miami Sylvester Comprehensive Cancer Center

location_facility: Washington University School Of Medicine

title: A Phase I/ II Study to Evaluate the Safety and Preliminary Efficacy of Nivolumab in Combination With Brentuximab Vedotin in Subjects With Relapsed Refractory Non Hodgkin Lymphomas With CD30 Expression (CheckMate 436: CHECKpoint Pathway and Nivolumab Clinical Trial Evaluation 436)

location_country:
Italy
United Kingdom
United States
Spain
Canada
France


title: A Phase 3 Open-Label Extension Study to Evaluate the Long-Term Safety of Omiganan Topical Gel in Subjects With Rosacea

summary: This study evaluates the long-term safety of once-daily application of Omiganan topical gel in subjects with severe papulopustular rosacea.

lead_sponsor: Maruho Co., Ltd.

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


acronym: JAVELIN Medley

lead_sponsor: Pfizer

summary: This is a Phase 1b/2 dose-optimization study to evaluate safety, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of avelumab (MSB0010718C) in combination with other cancer immunotherapies in patients with locally advanced or metastatic solid tumors. The primary purpose is to assess the safety and early signs of efficacy of various avelumab combinations with other cancer immunotherapies, optimizing dosing regimens as appropriate, in a limited series of indications.

location_facility: Weill Cornell Medical College/New York Presbyterian Hospital

location_facility: Investigational Drug Services, National Taiwan University Hospital

location_facility: Centre Hospitalier de l'Université de Montréal (CHUM)

location_facility: University of Pittsburgh Cancer Institute

location_facility: Karmanos Cancer Institute Weisberg Cancer Treatment Center

location_facility: Sanford Cancer Center Oncology Clinic & Pharmacy

location_facility: University of Texas Southwestern Medical Center

location_facility: UT Southwestern Simmons Comprehensive Cancer Center

location_facility: UCLA Clinical Research Unit (Adminstration Office)

location_facility: UC San Diego Medical Center - La Jolla (Jacobs Medical Center / Thornton Pavilion)

location_facility: NYU Laura and Isaac Perlmutter Cancer Center

location_facility: The Sarah Cannon Research Institute / Tennessee Oncology, PLLC

location_facility: Narodowy Instytut Onkologii im. Marii Sklodowskiej-Curie - Panstwowy Instytut Badawczy

location_facility: Investigational Pharmacy, Karmanos Cancer Center

location_facility: The University of Texas MD Anderson Cancer Center

location_facility: UCLA Hematology-Oncology Infusion Center

location_facility: H Lee Moffitt Cancer Center and Research Institute

location_facility: PH 145294, Centre Hospitalier de l'Universite de Montreal (CHUM), Oncology Research Pharmacy

location_facility: University of Iowa Hospitals and Clinics

location_facility: British Columbia Cancer Agency - Vancouver Centre

location_facility: Mount Sinai Comprehensive Cancer Center - Aventura

status: Terminated

title: A PHASE 1B/2 OPEN-LABEL STUDY TO EVALUATE SAFETY, CLINICAL ACTIVITY, PHARMACOKINETICS AND PHARMACODYNAMICS OF AVELUMAB (MSB0010718C) IN COMBINATION WITH OTHER CANCER IMMUNOTHERAPIES IN PATIENTS WITH ADVANCED MALIGNANCIES

ipd_sharing_description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

location_country:
Poland
United Kingdom
Australia
Japan
United States
Taiwan
Canada
France

status_simplified: Unknown


title: A Phase 1b Study to Evaluate the Safety and Efficacy of MEDI4736 as Monotherapy and in Combination With Tremelimumab or AZD9150 in Subjects With Relapsed or Refractory Diffuse Large B-cell Lymphoma.

summary: The purpose of this study is to evaluate the safety, tolerability, and efficacy of MEDI4736 (durvalumab) alone and in combination with either tremelimumab or AZD9150 in adult subjects with relapsed or refractory dIffuse large B-cell lymphoma.

location_country:
United States
Ireland
United Kingdom
France


intervention_type: Biological

lead_sponsor: Kyowa Kirin, Inc.

summary: The primary objective of this study is to establish the effect of KRN23 treatment on improvement in XLH-associated osteomalacia as determined by osteoid volume (osteoid volume/bone volume, OV/BV).

ReferenceCitation: Fratzl-Zelman N, Hartmann MA, Gamsjaeger S, Rokidi S, Paschalis EP, Blouin S, Zwerina J. Bone Matrix Mineralization and Response to Burosumab in Adult Patients With X-Linked Hypophosphatemia: Results From the Phase 3, Single-Arm International Trial. J Bone Miner Res. 2022 Sep;37(9):1665-1678. doi: 10.1002/jbmr.4641. Epub 2022 Aug 10.

oa_host_type: publisher

evidence: open (via free article)

unpaywall_oa_status: bronze

publisher_dissemination: Wiley

sponsor_collaborators:
Kyowa Kirin, Inc.
Kyowa Kirin Co., Ltd.

location_facility: Indiana University Department of Medicine University Hospital

location_facility: Aarhus University Hospital-Dept of Endocrinology and Internal Medicine

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

location_facility: Yale University School of Medicine - Yale New-Haven Hospital/Yale Center for Clinical Investigation

title: An Open-Label, Single-Arm, Phase 3 Study to Evaluate the Effects of KRN23 on Osteomalacia in Adults With X-linked Hypophosphatemia (XLH)

location_country:
Denmark
Japan
United States
Korea, Republic of
Canada
France


title: Phase 1b, Randomized, Double-blind, Placebo-controlled, Dose Escalation Study to Evaluate the Safety, Tolerability and Pharmacokinetics of QR-010 in Subjects With Homozygous ΔF508 Cystic Fibrosis

summary: A randomized, double-blind, placebo-controlled study of single and multiple ascending doses of QR-010 in adults homozygous for ΔF508 Cystic Fibrosis.

ReferenceCitation: Miah KM, Hyde SC, Gill DR. Emerging gene therapies for cystic fibrosis. Expert Rev Respir Med. 2019 Aug;13(8):709-725. doi: 10.1080/17476348.2019.1634547. Epub 2019 Jun 27.

repository_institution: University of Oxford - Oxford University Research Archive (ORA)

publisher_dissemination: Informa

lead_sponsor: ProQR Therapeutics

sponsor_collaborators:
ProQR Therapeutics
European Commission

location_country:
Germany
Italy
United Kingdom
Denmark
Belgium
United States
Spain
Czechia
Canada
France

location_facility: University of Calgary (Health Sciences Centre)

location_facility: Azienda Ospedaliera Universitaria Integrata di Verona

location_facility: University of Kansas Medical Center Research Institute

location_facility: Penn State Milton S. Hershey Medical Center

location_facility: Washington University School of Medicine

location_facility: University of Southern California USC - Keck School of Medicine

location_facility: Munich U. Hospital, Cystic Fibrosis Center for Adults

location_facility: University of Texas Southwestern Medical Center


title: A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel-group, Multicenter Study to Evaluate the Net Clinical Benefit of Sotagliflozin as Adjunct to Insulin Therapy in Type 1 Diabetes

summary: This Phase 3 study was designed to demonstrate the net benefit of sotagliflozin versus placebo in patients with Type 1 Diabetes (T1D).

ReferenceCitation: Garg SK, Henry RR, Banks P, Buse JB, Davies MJ, Fulcher GR, Pozzilli P, Gesty-Palmer D, Lapuerta P, Simo R, Danne T, McGuire DK, Kushner JA, Peters A, Strumph P. Effects of Sotagliflozin Added to Insulin in Patients with Type 1 Diabetes. N Engl J Med. 2017 Dec 14;377(24):2337-2348. doi: 10.1056/NEJMoa1708337. Epub 2017 Sep 13.

oa_colors: green other

repository_institution: University of North Carolina at Chapel Hill - Carolina Digital Repository

repository_institution: La Trobe University - OPAL (Open@LaTrobe)

repository_institution: University of Leicester - Leicester Research Archive

publisher_dissemination: Massachusetts Medical Society

lead_sponsor: Lexicon Pharmaceuticals

sponsor_collaborators:
Lexicon Pharmaceuticals
Sanofi

location_country:
Germany
Poland
New Zealand
Argentina
Australia
Czechia
Canada
France
Colombia
Slovakia
Hungary
United States
Belgium
Italy
United Kingdom
Spain
South Africa
Bulgaria
Israel


title: Single-Arm Study to Assess the Efficacy of UVADEX® (Methoxsalen) Sterile Solution in Conjunction With the THERAKOS® CELLEX® Photopheresis System in Pediatric Patients With Steroid-Refractory Acute Graft-vs-Host Disease (aGvHD)

summary: This is a single-arm, open-label, multicenter study of the efficacy of UVADEX® (methoxsalen) Sterile Solution in conjunction with THERAKOS® CELLEX® Photopheresis Systems (ECP) in pediatric participants with steroid-refractory aGvHD. The study is composed of Screening, Treatment, and Follow-up Periods.

ipd_sharing: No

ipd_sharing_description:
Because we work in the rare disease space, to eliminate risk of patient identification we do not share individual patient data.

We post summary aggregate results for applicable clinical trials in the registry, and statistical endpoints and discussion in publications; with each referencing the other.

lead_sponsor: Therakos, Inc., a Mallinckrodt Company

location_country:
Germany
Italy
Hungary
United Kingdom
Austria
United States
Spain
France

location_facility: U.O.C. Clinica di Oncoematologia Pediatrica Azienda Ospedaliera di Padova

location_facility: Klinikum rechts der Isar, TU München, Klinik- und Poliklinik für Kinder- und Jugendmedizin, Kinderklinik München Schwabing

location_facility: University Hospitals Rainbow Babies & Children's

location_facility: Universitaetsklinikum Ulm, Kinder- und Jugendmedizin

location_facility: Children's Healthcare of Atlanta, Emory - Children's Center

location_facility: Universitätsklinikum Leipzig, Klinik und Poliklinik für Kinder- und Jugendmedizin, Abteilung für Hämatologie und Internistische Onkologie

location_facility: Hospital Infantil Universitario "Nino Jesus"

location_facility: Vanderbilt University Medical Center - Ingram Cancer Institute


title: A Phase 1 Study of BLU-285 in Patients With Gastrointestinal Stromal Tumors (GIST) and Other Relapsed and Refractory Solid Tumors

summary: This is a Phase 1, open-label, first-in-human (FIH) study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and antineoplastic activity of avapritinib (formerly BLU-285), administered orally (PO), in adult patients with unresectable GIST or other relapsed or refractory solid tumors. The study consists of 2 parts, a dose-escalation part (Part 1) and an expansion part (Part 2).

ReferenceCitation: Kang YK, George S, Jones RL, Rutkowski P, Shen L, Mir O, Patel S, Zhou Y, von Mehren M, Hohenberger P, Villalobos V, Brahmi M, Tap WD, Trent J, Pantaleo MA, Schoffski P, He K, Hew P, Newberry K, Roche M, Heinrich MC, Bauer S. Avapritinib Versus Regorafenib in Locally Advanced Unresectable or Metastatic GI Stromal Tumor: A Randomized, Open-Label Phase III Study. J Clin Oncol. 2021 Oct 1;39(28):3128-3139. doi: 10.1200/JCO.21.00217. Epub 2021 Aug 3.

endpoint_id: fhisxppmrqpajgzqkvft

repository_institution: Alma Mater Studiorum Università di Bologna - Archivio istituzionale della ricerca - Alma Mater Studiorum Università di Bologna

repository_institution: KU Leuven - Lirias

repository_normalized: LIRIAS (KU Leuven)

repositories:
LIRIAS (KU Leuven)
PubMed Central
cris.unibo.it

repositories_concat: LIRIAS (KU Leuven);PubMed Central;cris.unibo.it

ReferenceCitation: von Mehren M, Heinrich MC, Shi H, Iannazzo S, Mankoski R, Dimitrijevic S, Hoehn G, Chiroli S, George S. Clinical efficacy comparison of avapritinib with other tyrosine kinase inhibitors in gastrointestinal stromal tumors with PDGFRA D842V mutation: a retrospective analysis of clinical trial and real-world data. BMC Cancer. 2021 Mar 19;21(1):291. doi: 10.1186/s12885-021-08013-1.

ReferenceCitation: Jones RL, Serrano C, von Mehren M, George S, Heinrich MC, Kang YK, Schoffski P, Cassier PA, Mir O, Chawla SP, Eskens FALM, Rutkowski P, Tap WD, Zhou T, Roche M, Bauer S. Avapritinib in unresectable or metastatic PDGFRA D842V-mutant gastrointestinal stromal tumours: Long-term efficacy and safety data from the NAVIGATOR phase I trial. Eur J Cancer. 2021 Mar;145:132-142. doi: 10.1016/j.ejca.2020.12.008. Epub 2021 Jan 16.

repositories:
LIRIAS (KU Leuven)
PubMed Central

repositories_concat: LIRIAS (KU Leuven);PubMed Central

ReferenceCitation: Joseph CP, Abaricia SN, Angelis MA, Polson K, Jones RL, Kang YK, Riedel RF, Schoffski P, Serrano C, Trent J, Tetzlaff ED, Si TD, Zhou T, Doyle A, Bauer S, Roche M, Havnaer T. Optimal Avapritinib Treatment Strategies for Patients with Metastatic or Unresectable Gastrointestinal Stromal Tumors. Oncologist. 2021 Apr;26(4):e622-e631. doi: 10.1002/onco.13632. Epub 2021 Jan 5.

ReferenceCitation: Heinrich MC, Jones RL, von Mehren M, Schoffski P, Serrano C, Kang YK, Cassier PA, Mir O, Eskens F, Tap WD, Rutkowski P, Chawla SP, Trent J, Tugnait M, Evans EK, Lauz T, Zhou T, Roche M, Wolf BB, Bauer S, George S. Avapritinib in advanced PDGFRA D842V-mutant gastrointestinal stromal tumour (NAVIGATOR): a multicentre, open-label, phase 1 trial. Lancet Oncol. 2020 Jul;21(7):935-946. doi: 10.1016/S1470-2045(20)30269-2. Erratum In: Lancet Oncol. 2020 Sep;21(9):e418.

ReferenceCitation: Gebreyohannes YK, Wozniak A, Zhai ME, Wellens J, Cornillie J, Vanleeuw U, Evans E, Gardino AK, Lengauer C, Debiec-Rychter M, Sciot R, Schoffski P. Robust Activity of Avapritinib, Potent and Highly Selective Inhibitor of Mutated KIT, in Patient-derived Xenograft Models of Gastrointestinal Stromal Tumors. Clin Cancer Res. 2019 Jan 15;25(2):609-618. doi: 10.1158/1078-0432.CCR-18-1858. Epub 2018 Oct 1.

publisher_dissemination: American Association for Cancer Research

location_country:
Germany
Italy
Poland
United Kingdom
Netherlands
Belgium
United States
Korea, Republic of
Spain
France

location_facility: Leuven Cancer Institute University Hospitals Leuven

location_facility: Centrum Onkologii-Instytut im. Marii Skłodowskiej-Curie w Warszawie

location_facility: Fondazione IRCCS - Istituto Nazinale dei Tumori

location_facility: Vall d' Hebron Institute of Oncology (VHIO)

location_facility: Scottsdale Healthcare Hospitals DBA HonorHealth


title: A Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of BLU-554 in Patients With Hepatocellular Carcinoma

summary: This is a Phase 1, open-label, first-in-human (FIH) study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary antineoplastic activity of fisogatinib (formerly known as BLU- 554) administered orally in patients with FGF19 IHC+ hepatocellular carcinoma (HCC). The study consists of 3 parts, a dose-escalation part (Part 1), an expansion part (Part 2) exploring a once daily (qd) dosing schedule at the recommended Phase 2 dose (RP2D), and a Part 3 expansion of the qd dosing schedule at the RP2D in TKI naive patients.

enrollment_type: Anticipated

lead_sponsor: Blueprint Medicines Corporation

location_country:
Germany
Italy
United Kingdom
Hong Kong
Singapore
Switzerland
United States
China
Korea, Republic of
Spain
Taiwan
France

location_facility: University of Liverpool - Clatterbridge Cancer Centre

location_facility: IRCCS Foundation - National Institute of Tumors

location_facility: Tianjin Medical University Cancer Institute & Hospital, Hepatobiliary Oncology Department

location_facility: The First Affiliated Hospital, College of Medicine, Zhejiang University

location_facility: Hunan Cancer Hospital, Radioactive Interventional Department

location_facility: Jilin University the First Affiliated Hospital

location_facility: University of Miami - Sylvester Comprehensive Cancer Center

location_facility: Johannes Gutenberg University Mainz - University Medical Center

location_facility: Harbin Medical University Cancer Hospital

location_facility: The Chinese People's Liberation Army 81 Hospital


acronym: Phase 1 dry powder

summary: To characterize the pharmacokinetic profile of rivaroxaban administered as granules for suspension formulation and to document safety and tolerability

sponsor_collaborators:
Bayer
Janssen Research & Development, LLC

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

location_facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

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

location_facility: Hospital General Universitario Gregorio Marañón

location_facility: Hôpital Arnaud de Villeneuve - Montpellier

location_facility: Hopital Necker les enfants malades - Paris

location_facility: Turun yliopistollinen keskussairaala, kantasairaala

location_facility: University of Semmelweis/ Semmelweis Egyetem

title: Single-dose Study Testing Rivaroxaban Granules for Oral Suspension Formulation in Children From 2 Months to 12 Years With Previous Thrombosis

primary_purpose: Other

location_country:
Finland
Italy
Hungary
Belgium
United States
Spain
Canada
France


title: A Randomised, Double-blind, Double-dummy, Parallel-group, Multicenter, Phase IIb Study to Evaluate the Effect of Ticagrelor Versus Placebo in Reducing the Number of Days With Pain in Young Adults With Sickle Cell Disease

summary: The purpose of this study is to determine whether ticagrelor is effective in reducing the number of days of pain, intensity of pain, and reducing the use of analgesics due to sickle cell disease

primary_purpose: Prevention

lead_sponsor: AstraZeneca

location_country:
Italy
United Kingdom
Lebanon
United States
Turkey
Kenya
Egypt
France


title: A Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of lGIV-C as a Corticosteroid Sparing Agent in Corticosteroid Dependent Patients With Generalized Myasthenia Gravis

summary: This is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of Immune Globulin (Human), 10% Caprylate/Chromatography Purified (IGIV-C) as a corticosteroid (CS)-sparing agent in subjects with CS-dependent Myasthenia Gravis (MG).

ReferenceCitation: Bril V, Szczudlik A, Vaitkus A, Rozsa C, Kostera-Pruszczyk A, Hon P, Bednarik J, Tyblova M, Kohler W, Toomsoo T, Nowak RJ, Mozaffar T, Freimer ML, Nicolle MW, Magnus T, Pulley MT, Rivner M, Dimachkie MM, Distad BJ, Pascuzzi RM, Babiar D, Lin J, Querolt Coll M, Griffin R, Mondou E. Randomized Double-Blind Placebo-Controlled Trial of the Corticosteroid-Sparing Effects of Immunoglobulin in Myasthenia Gravis. Neurology. 2023 Feb 14;100(7):e671-e682. doi: 10.1212/WNL.0000000000201501. Epub 2022 Oct 21. Erratum In: Neurology. 2023 Sep 12;101(11):501.

repositories_institution: University of California - eScholarship University of California

ReferenceCitation: Dalakas MC, Meisel A. Immunomodulatory effects and clinical benefits of intravenous immunoglobulin in myasthenia gravis. Expert Rev Neurother. 2022 Apr;22(4):313-318. doi: 10.1080/14737175.2022.2057223. Epub 2022 Apr 5.

repository_institution: Oxford - Oxford Academix

lead_sponsor: Grifols Therapeutics LLC

location_country:
Germany
Lithuania
Poland
Hungary
Belgium
United States
Czechia
Canada
Estonia
France

location_facility: Ohio State University Wexner Medical Center, Neurology Department

location_facility: Charité Universitaetsmedizin Berlin, Klinik für Neurologie

location_facility: Universitaetsklinikum Hamburg-Eppendorf, Klinik und Poliklinik

location_facility: Yale University School of Medicine, Department of Neurology

location_facility: Jahn Ferenc Del-pesti Korhaz es Rendelointezet, Neurologiai Osztaly

location_facility: Universitaetsklinikum Giessen und Marburg GmbH Standort Marburg

location_facility: London Health Sciences Centre- University Hospital

location_facility: Vseobecna fakultni nemocnice v Praze, Dept of Neurologicka klinika

location_facility: CHU de Toulouse - Hôpital Purpan, Service de Neurologie Générale

location_facility: Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont

location_facility: Houston Methodist Neurological Institute

location_facility: CHU Strasbourg - Nouvel Hôpital Civil, Clinique Neurologique

location_facility: Samodzielny Publiczny Centralny Szpital Kliniczny

location_facility: Fachkrankenhaus Hubertusburg gGmbH, Klinik f. Neurologie

location_facility: University Health Network (UHN) - Toronto General Hospital

location_facility: Krankenhaus Martha-Maria Halle-Doelau, Klinik fuer Neurologie

location_facility: University of Kansas Medical Center Research Institute, Inc.

location_facility: Hospital of Lithuanian University of Health Sciences Kaunas Clinics

location_facility: III Szpital Miejski w Lodzi im. Dr K. Jonschera

location_facility: Uniwersyteckie Centrum Kliniczne, Dept of Neurology

location_facility: Krakowska Akademia Neurologii Sp z o.o. Centrum Neurologii Klinicznej

location_facility: University of Florida at Shands Jacksonville

location_facility: Pest Megyei Flor Ferenc Korhaz, Neurologia es Stroke Osztaly

location_facility: Fakultni nemocnice Brno, Dept of Neurologicka klinika

location_facility: Universitaetsklinikum Jena, Klinik fuer Neurologie


title: A Phase Ib, Open-Label, Dose-Escalation Study of the Safety and Pharmacology of GDC-0919 Administered With Atezolizumab in Patients With Locally Advanced or Metastatic Solid Tumors

summary: This study will evaluate the safety, tolerability, and pharmacokinetics of the combination of GDC-0919 and atezolizumab in participants with locally advanced, recurrent, or metastatic incurable solid malignancy that has progressed after available standard therapy or for which standard therapy is ineffective, intolerable, or inappropriate. Participants will be enrolled in two stages, including a dose-escalation stage and an expansion stage.

ReferenceCitation: Jung KH, LoRusso P, Burris H, Gordon M, Bang YJ, Hellmann MD, Cervantes A, Ochoa de Olza M, Marabelle A, Hodi FS, Ahn MJ, Emens LA, Barlesi F, Hamid O, Calvo E, McDermott D, Soliman H, Rhee I, Lin R, Pourmohamad T, Suchomel J, Tsuhako A, Morrissey K, Mahrus S, Morley R, Pirzkall A, Davis SL. Phase I Study of the Indoleamine 2,3-Dioxygenase 1 (IDO1) Inhibitor Navoximod (GDC-0919) Administered with PD-L1 Inhibitor (Atezolizumab) in Advanced Solid Tumors. Clin Cancer Res. 2019 Jun 1;25(11):3220-3228. doi: 10.1158/1078-0432.CCR-18-2740. Epub 2019 Feb 15.

evidence: open (via free pdf)

lead_sponsor: Genentech, Inc.

location_country:
United States
Spain
Korea, Republic of
France

location_facility: Johns Hopkins Sidney Kimmel Comprehensive Cancer Center

location_facility: Hospital Clinico Universitario de Valencia

location_facility: The Angeles Clinic and Research Institute, Santa Monica Office

location_facility: Hospital Universitario HM Sanchinarro-CIOCC

location_facility: HonorHealth Research Institute - Bisgrove

location_facility: H. Lee Moffitt Cancer Center and Research Inst.


title: A 24 Week Off Drug Extension, Parallel Group, Study Assessing Durability of Effect on Skeletal Muscle Strength and Function Following a 6-month Double-blind, Placebo Controlled Study Evaluating Bimagrumab in Older Adults With Sarcopenia (InvestiGAIT Extension)

summary: This extension study was a 24-week off-drug follow-up of the core CBYM338E2202 (NCT ) study and the main objective was to determine the long-term durability of bimagrumab (BYM338) effect after a 6-month treatment period.

lead_sponsor: Novartis Pharmaceuticals

location_country:
Denmark
Australia
Japan
Switzerland
Belgium
United States
Korea, Republic of
Spain
Taiwan
Czechia
Russian Federation
France


lead_sponsor: Hoffmann-La Roche

summary: This study is to assess safety as defined by the frequency and severity of gastrointestinal (GI) perforation/fistula, GI-vaginal fistula and genitourinary (GU) fistula in participants treated with bevacizumab 15 milligrams per kilogram (mg/kg) in combination with paclitaxel and carboplatin, all repeated every 3 weeks, for recurrent, persistent or metastatic cervical cancer. In addition, this study will include evaluation of the overall safety profile of bevacizumab in combination with paclitaxel and carboplatin in this setting, assessment of GI perforation/fistula, GI-vaginal fistula and GU fistula events over time, and evaluation of efficacy.

ReferenceCitation: Redondo A, Colombo N, McCormack M, Dreosti L, Nogueira-Rodrigues A, Scambia G, Lorusso D, Joly F, Schenker M, Ruff P, Estevez-Diz M, Irahara N, Donica M, Gonzalez-Martin A. Primary results from CECILIA, a global single-arm phase II study evaluating bevacizumab, carboplatin and paclitaxel for advanced cervical cancer. Gynecol Oncol. 2020 Oct;159(1):142-149. doi: 10.1016/j.ygyno.2020.07.026. Epub 2020 Aug 4.

location_facility: Instituto Valenciano Oncologia; Oncologia Medica

location_facility: Instituto Nacional de Cancer - INCa; Pesquisa Clinica

location_facility: Instituto do Cancer do Estado de Sao Paulo - ICESP

location_facility: Institut Gustave Roussy; Oncologie Medicale

location_facility: Alexandras General Hospital of Athens; Oncology Department

location_facility: Complejo Hospitalario Universitario de Santiago (CHUS) ; Servicio de Oncologia

location_facility: St. Petersburg Oncology & Gynecology; City Clinical Oncology Dispensary

location_facility: Istituto Nazionale dei Tumori; Divisione Oncologia Chirurgica e Ginecologica

location_facility: Centre Francois Baclesse; Urologie Gynecologie

location_facility: Centro Oncologico MD Anderson International Espana

location_facility: Centro Oncologico Riojano Integral (CORI)

location_facility: Istituto Tumori Napoli;Unità Operativa Oncologia Medica Uro-Ginecologica

location_facility: University of Pretoria; Department of Medical Oncology

location_facility: Hospital Universitario La Paz; Servicio de Oncologia

location_facility: Complex Oncological Center - Plovdiv, EOOD

location_facility: Istanbul Uni of Medicine Faculty; Oncology Dept

location_facility: IPO do Porto; Servico de Oncologia Medica

location_facility: Instituto Nacional de Cancerologia; Oncology

location_facility: Russian Oncology Research Center n.a. N.N. Blokhin Dpt of Clinical Pharmacology and Chemotherapy

location_facility: Hospital Universitario la Fe; Servicio de Oncologia

location_facility: Centrum Onkologii - Instytut M.Sklodowskiej-Curie; Klinika Ginekologii Onkologicznej

location_facility: Ankara Baskent University Medicine Faculty; Gynaecology

location_facility: Universita' Cattolica Del Sacro Cuore; Reparto Ginecologia Oncologica

location_facility: Centrum Onkologii Instytut im. M.Sklodowskiej-Curie; Klinika Ginekologii Onkologicznej

location_facility: Irccs Istituto Europeo Di Oncologia (IEO); Oncologia Medica

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

title: A Multicenter Open-Label Single-Arm Phase II Study Evaluating the Safety and Efficacy of Bevacizumab in Combination With Carboplatin and Paclitaxel in Patients With Metastatic, Recurrent or Persistent Cervical Cancer

location_country:
Italy
Poland
Colombia
Portugal
Argentina
Romania
Brazil
Costa Rica
Mexico
Spain
Greece
Panama
South Africa
Turkey
Bulgaria
Serbia
Russian Federation
France


summary: This study was to characterize the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and anti-tumor activity of LAG525 as a single agent and in combination with PDR001 to adult patients with solid tumors. The study consists of a dose escalation (phase 1) to determine the maximum tolerated dose (MTD) or recommended Phase 2 dose (RP2D) for LAG525 as a single agent and in combination with PDR001, and a dose expansion (phase 2) which characterized treatment of LAG525 in combination with PDR001 at the MTD or RP2D.

ReferenceCitation: Schoffski P, Tan DSW, Martin M, Ochoa-de-Olza M, Sarantopoulos J, Carvajal RD, Kyi C, Esaki T, Prawira A, Akerley W, De Braud F, Hui R, Zhang T, Soo RA, Maur M, Weickhardt A, Krauss J, Deschler-Baier B, Lau A, Samant TS, Longmire T, Chowdhury NR, Sabatos-Peyton CA, Patel N, Ramesh R, Hu T, Carion A, Gusenleitner D, Yerramilli-Rao P, Askoxylakis V, Kwak EL, Hong DS. Phase I/II study of the LAG-3 inhibitor ieramilimab (LAG525) +/- anti-PD-1 spartalizumab (PDR001) in patients with advanced malignancies. J Immunother Cancer. 2022 Feb;10(2):e003776. doi: 10.1136/jitc-2021-003776.

location_facility: Cancer Therapy and Research Center UT Health Science Center CTRC 2

location_facility: University of Texas MD Anderson Cancer Center

location_facility: Columbia University Medical Center SC LAG X2101C

location_facility: Memorial Sloan Kettering Cancer Center SC

location_facility: Huntsman Cancer Institute Huntsman Cancer Institute

title: A Phase I/II, Open Label, Multicenter Study of the Safety and Efficacy of LAG525 Single Agent and in Combination With PDR001 Administered to Patients With Advanced Malignancies

location_country:
Germany
Italy
Hong Kong
Singapore
Australia
Japan
Belgium
United States
Spain
Taiwan
Canada
France

ipd_sharing: Undecided


title: A 52-Week Double-Blind, Placebo-Controlled, Parallel-Group Efficacy and Safety Study of Reslizumab 110 mg Fixed, Subcutaneous Dosing in Patients With Uncontrolled Asthma and Elevated Blood Eosinophils

summary: The primary objective of this study is to determine the effect of reslizumab (110 mg) administered subcutaneously every 4 weeks on clinical asthma exacerbations in adults and adolescents with asthma and elevated blood eosinophils who are inadequately controlled on standard-of-care asthma therapy.

ReferenceCitation: Bernstein JA, Virchow JC, Murphy K, Maspero JF, Jacobs J, Adir Y, Humbert M, Castro M, Marsteller DA, McElhattan J, Hickey L, Garin M, Vanlandingham R, Brusselle G. Effect of fixed-dose subcutaneous reslizumab on asthma exacerbations in patients with severe uncontrolled asthma and corticosteroid sparing in patients with oral corticosteroid-dependent asthma: results from two phase 3, randomised, double-blind, placebo-controlled trials. Lancet Respir Med. 2020 May;8(5):461-474. doi: 10.1016/S2213-2600(19)30372-8. Epub 2020 Feb 14.

lead_sponsor: Teva Branded Pharmaceutical Products R&D, Inc.

location_country:
Germany
Poland
New Zealand
Argentina
Australia
Japan
Turkey
Czechia
Canada
France
Hungary
United States
Ukraine
Romania
Belgium
Mexico
Korea, Republic of
Spain
South Africa
Russian Federation
Israel

location_facility:
Teva Investigational Site 53313
Teva Investigational Site 32564
Teva Investigational Site 53309
Teva Investigational Site 58226
Teva Investigational Site 50350
Teva Investigational Site 13205
Teva Investigational Site 13197
Teva Investigational Site 13238
Teva Investigational Site 13189
Teva Investigational Site 82042
Teva Investigational Site 11106
Teva Investigational Site 51218
Teva Investigational Site 13239
Teva Investigational Site 84038
Teva Investigational Site 13209
Teva Investigational Site 13223
Teva Investigational Site 20054
Teva Investigational Site 90027
Teva Investigational Site 50355
Teva Investigational Site 58216
Teva Investigational Site 84037
Teva Investigational Site 13207
Teva Investigational Site 54128
Teva Investigational Site 32555
Teva Investigational Site 51223
Teva Investigational Site 84045
Teva Investigational Site 13224
Teva Investigational Site 80073
Teva Investigational Site 54129
Teva Investigational Site 84053
Teva Investigational Site 58219
Teva Investigational Site 50351
Teva Investigational Site 13763
Teva Investigational Site 50349
Teva Investigational Site 82043
Teva Investigational Site 31154
Teva Investigational Site 32559
Teva Investigational Site 84031
Teva Investigational Site 58231
Teva Investigational Site 84032
Teva Investigational Site 13217
Teva Investigational Site 13769
Teva Investigational Site 31149
Teva Investigational Site 13201
Teva Investigational Site 58229
Teva Investigational Site 13183
Teva Investigational Site 78084
Teva Investigational Site 21085
Teva Investigational Site 58223
Teva Investigational Site 13764
Teva Investigational Site 84041
Teva Investigational Site 53308
Teva Investigational Site 58214
Teva Investigational Site 32566
Teva Investigational Site 53311
Teva Investigational Site 51224
Teva Investigational Site 32570
Teva Investigational Site 13243
Teva Investigational Site 13226
Teva Investigational Site 13212
Teva Investigational Site 50348
Teva Investigational Site 51219
Teva Investigational Site 50347
Teva Investigational Site 13251
Teva Investigational Site 84035
Teva Investigational Site 13240
Teva Investigational Site 58222
Teva Investigational Site 13254
Teva Investigational Site 51228
Teva Investigational Site 13211
Teva Investigational Site 58225
Teva Investigational Site 84034
Teva Investigational Site 78088
Teva Investigational Site 80079
Teva Investigational Site 51225
Teva Investigational Site 53315
Teva Investigational Site 13241
Teva Investigational Site 58234
Teva Investigational Site 13195
Teva Investigational Site 13242
Teva Investigational Site 51226
Teva Investigational Site 32569
Teva Investigational Site 13220
Teva Investigational Site 90032
Teva Investigational Site 58213
Teva Investigational Site 13210
Teva Investigational Site 20046
Teva Investigational Site 80078
Teva Investigational Site 84043
Teva Investigational Site 13765
Teva Investigational Site 58220
Teva Investigational Site 31152
Teva Investigational Site 13260
Teva Investigational Site 58253
Teva Investigational Site 20041
Teva Investigational Site 13247
Teva Investigational Site 13258
Teva Investigational Site 21087
Teva Investigational Site 80081
Teva Investigational Site 90029
Teva Investigational Site 80077
Teva Investigational Site 20036
Teva Investigational Site 58224
Teva Investigational Site 13194
Teva Investigational Site 79046
Teva Investigational Site 13225
Teva Investigational Site 84040
Teva Investigational Site 51222
Teva Investigational Site 51227
Teva Investigational Site 90031
Teva Investigational Site 13203
Teva Investigational Site 52107
Teva Investigational Site 13235
Teva Investigational Site 84036
Teva Investigational Site 32560
Teva Investigational Site 80094
Teva Investigational Site 58218
Teva Investigational Site 13257
Teva Investigational Site 13185
Teva Investigational Site 13252
Teva Investigational Site 84048
Teva Investigational Site 51255
Teva Investigational Site 35183
Teva Investigational Site 79047
Teva Investigational Site 80080
Teva Investigational Site 52108
Teva Investigational Site 32556
Teva Investigational Site 84044
Teva Investigational Site 32563
Teva Investigational Site 84039
Teva Investigational Site 13259
Teva Investigational Site 31147
Teva Investigational Site 21089
Teva Investigational Site 82041
Teva Investigational Site 13256
Teva Investigational Site 32565
Teva Investigational Site 82040
Teva Investigational Site 20032
Teva Investigational Site 32561
Teva Investigational Site 52109
Teva Investigational Site 13255
Teva Investigational Site 52105
Teva Investigational Site 13218
Teva Investigational Site 13200
Teva Investigational Site 53314
Teva Investigational Site 51221
Teva Investigational Site 20040
Teva Investigational Site 32567
Teva Investigational Site 51217
Teva Investigational Site 84049
Teva Investigational Site 20033
Teva Investigational Site 53312
Teva Investigational Site 58221
Teva Investigational Site 11109
Teva Investigational Site 13181
Teva Investigational Site 52104
Teva Investigational Site 52111
Teva Investigational Site 52110
Teva Investigational Site 13196
Teva Investigational Site 13222
Teva Investigational Site 13191
Teva Investigational Site 20053
Teva Investigational Site 11105
Teva Investigational Site 13229
Teva Investigational Site 21084
Teva Investigational Site 58230
Teva Investigational Site 13233
Teva Investigational Site 13232
Teva Investigational Site 87015
Teva Investigational Site 13246
Teva Investigational Site 13767
Teva Investigational Site 13253
Teva Investigational Site 37053
Teva Investigational Site 32551
Teva Investigational Site 78083
Teva Investigational Site 13766
Teva Investigational Site 20045
Teva Investigational Site 53310
Teva Investigational Site 58228
Teva Investigational Site 13204
Teva Investigational Site 13190
Teva Investigational Site 37055
Teva Investigational Site 21088
Teva Investigational Site 87014
Teva Investigational Site 13187
Teva Investigational Site 13202
Teva Investigational Site 50354
Teva Investigational Site 51220
Teva Investigational Site 90030
Teva Investigational Site 13184
Teva Investigational Site 13230
Teva Investigational Site 51231
Teva Investigational Site 13250
Teva Investigational Site 20037
Teva Investigational Site 13199
Teva Investigational Site 90026
Teva Investigational Site 20035
Teva Investigational Site 37054
Teva Investigational Site 54130
Teva Investigational Site 32562
Teva Investigational Site 82039
Teva Investigational Site 51229
Teva Investigational Site 87016
Teva Investigational Site 21086
Teva Investigational Site 13186
Teva Investigational Site 52106
Teva Investigational Site 80075
Teva Investigational Site 32568
Teva Investigational Site 32557
Teva Investigational Site 13215
Teva Investigational Site 80076
Teva Investigational Site 13216
Teva Investigational Site 78085
Teva Investigational Site 50352
Teva Investigational Site 51216
Teva Investigational Site 84047
Teva Investigational Site 58233
Teva Investigational Site 13188
Teva Investigational Site 13208
Teva Investigational Site 84046
Teva Investigational Site 90028
Teva Investigational Site 35182
Teva Investigational Site 58227
Teva Investigational Site 78087
Teva Investigational Site 13227
Teva Investigational Site 13249


title: A Phase II, Single Arm, Multicenter Trial to Determine the Efficacy and Safety of CTL019 in Pediatric Patients With Relapsed and Refractory B-cell Acute Lymphoblastic Leukemia

acronym: ELIANA

summary: This is a single arm, open-label, multi-center, phase II study to determine the efficacy and safety of CTL019 in pediatric patients with r/r B-cell ALL.

ReferenceCitation: Laetsch TW, Maude SL, Rives S, Hiramatsu H, Bittencourt H, Bader P, Baruchel A, Boyer M, De Moerloose B, Qayed M, Buechner J, Pulsipher MA, Myers GD, Stefanski HE, Martin PL, Nemecek E, Peters C, Yanik G, Khaw SL, Davis KL, Krueger J, Balduzzi A, Boissel N, Tiwari R, O'Donovan D, Grupp SA. Three-Year Update of Tisagenlecleucel in Pediatric and Young Adult Patients With Relapsed/Refractory Acute Lymphoblastic Leukemia in the ELIANA Trial. J Clin Oncol. 2023 Mar 20;41(9):1664-1669. doi: 10.1200/JCO.22.00642. Epub 2022 Nov 18.

repository_institution: Ghent University - Ghent University Academic Bibliography

repositories_concat: PubMed Central;archive.ugent.be

ReferenceCitation: Newman H, Li Y, Liu H, Myers RM, Tam V, DiNofia A, Wray L, Rheingold SR, Callahan C, White C, Baniewicz D, Winestone LE, Kadauke S, Diorio C, June CH, Getz KD, Aplenc R, Teachey DT, Maude SL, Grupp SA, Bona K, Leahy AB. Impact of poverty and neighborhood opportunity on outcomes for children treated with CD19-directed CAR T-cell therapy. Blood. 2023 Feb 9;141(6):609-619. doi: 10.1182/blood.2022017866.

publisher_dissemination: American Society of Hematology

ReferenceCitation: Laetsch TW, Maude SL, Balduzzi A, Rives S, Bittencourt H, Boyer MW, Buechner J, De Moerloose B, Qayed M, Phillips CL, Pulsipher MA, Hiramatsu H, Tiwari R, Grupp SA. Tisagenlecleucel in pediatric and young adult patients with Down syndrome-associated relapsed/refractory acute lymphoblastic leukemia. Leukemia. 2022 Jun;36(6):1508-1515. doi: 10.1038/s41375-022-01550-z. Epub 2022 Apr 14.

ReferenceCitation: Thudium Mueller K, Grupp SA, Maude SL, Levine JE, Pulsipher MA, Boyer MW, August KJ, Myers GD, Tam CS, Jaeger U, Foley SR, Borchmann P, Schuster SJ, Waller EK, Awasthi R, Potthoff B, Warren A, Waldron ER, McBlane F, Chassot-Agostinho A, Laetsch TW. Tisagenlecleucel immunogenicity in relapsed/refractory acute lymphoblastic leukemia and diffuse large B-cell lymphoma. Blood Adv. 2021 Dec 14;5(23):4980-4991. doi: 10.1182/bloodadvances.2020003844.

ReferenceCitation: Levine JE, Grupp SA, Pulsipher MA, Dietz AC, Rives S, Myers GD, August KJ, Verneris MR, Buechner J, Laetsch TW, Bittencourt H, Baruchel A, Boyer MW, De Moerloose B, Qayed M, Davies SM, Phillips CL, Driscoll TA, Bader P, Schlis K, Wood PA, Mody R, Yi L, Leung M, Eldjerou LK, June CH, Maude SL. Pooled safety analysis of tisagenlecleucel in children and young adults with B cell acute lymphoblastic leukemia. J Immunother Cancer. 2021 Aug;9(8):e002287. doi: 10.1136/jitc-2020-002287.

ReferenceCitation: Buechner J, Grupp SA, Hiramatsu H, Teachey DT, Rives S, Laetsch TW, Yanik GA, Wood P, Awasthi R, Yi L, Chassot-Agostinho A, Eldjerou LK, De Moerloose B. Practical guidelines for monitoring and management of coagulopathy following tisagenlecleucel CAR T-cell therapy. Blood Adv. 2021 Jan 26;5(2):593-601. doi: 10.1182/bloodadvances.2020002757.

evidence: oa journal (via observed oa rate)

ReferenceCitation: Laetsch TW, Myers GD, Baruchel A, Dietz AC, Pulsipher MA, Bittencourt H, Buechner J, De Moerloose B, Davis KL, Nemecek E, Driscoll T, Mechinaud F, Boissel N, Rives S, Bader P, Peters C, Sabnis HS, Grupp SA, Yanik GA, Hiramatsu H, Stefanski HE, Rasouliyan L, Yi L, Shah S, Zhang J, Harris AC. Patient-reported quality of life after tisagenlecleucel infusion in children and young adults with relapsed or refractory B-cell acute lymphoblastic leukaemia: a global, single-arm, phase 2 trial. Lancet Oncol. 2019 Dec;20(12):1710-1718. doi: 10.1016/S1470-2045(19)30493-0. Epub 2019 Oct 9.

ReferenceCitation: Maude SL, Laetsch TW, Buechner J, Rives S, Boyer M, Bittencourt H, Bader P, Verneris MR, Stefanski HE, Myers GD, Qayed M, De Moerloose B, Hiramatsu H, Schlis K, Davis KL, Martin PL, Nemecek ER, Yanik GA, Peters C, Baruchel A, Boissel N, Mechinaud F, Balduzzi A, Krueger J, June CH, Levine BL, Wood P, Taran T, Leung M, Mueller KT, Zhang Y, Sen K, Lebwohl D, Pulsipher MA, Grupp SA. Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia. N Engl J Med. 2018 Feb 1;378(5):439-448. doi: 10.1056/NEJMoa1709866.

location_country:
Germany
Italy
Norway
Australia
Japan
Austria
Belgium
United States
Spain
Canada
France

location_facility: Children s Mercy Hospital SC - CTL019B2205J

location_facility: Children's Healthcare of Atlanta SC CTL019

location_facility: Stanford Universtiy Medical Center SC - CTL019B2205J - B2206

location_facility: Oregon Health and Science University Doernbecher Children's Hosp.

location_facility: The Childrens Hospital of Philadelphia CHOP

location_facility: Childrens Hospital Los Angeles SC CTL019

location_facility: University of Utah Clinical Trials Office SC - CTL019B2205J

location_facility: University of Texas Southwestern Medical Center .


acronym: TNBC

summary: To determine whether MCS110 antibody therapy improves the efficacy of carboplatin and gemcitabine (carbo/gem) in advanced TNBC patients

location_facility: Massachusetts General Hospital Cancer Center SC

title: A Randomized Phase II Study of MCS110 Combined With Carboplatin and Gemcitabine in Advanced Triple Negative Breast Cancer (TNBC)

location_country:
Germany
Italy
Hong Kong
Australia
Austria
Belgium
United States
Korea, Republic of
Spain
Turkey
Taiwan
France


summary:
This is a randomized, open-label, multicenter, two arm, phase II study to evaluate treatment compliance and change in serum ferritin of a deferasirox granule formulation and a deferasirox DT formulation in children and adolescents aged ≥ 2 and < 18 years at enrollment with any transfusion-dependent anemia requiring chelation therapy due to iron overload, to demonstrate the effect of improved compliance on iron burden.

Randomization will be stratified by age groups (2 to <10 years, 10 to <18 years) and prior iron chelation therapy (Yes/ No). There will be two study phases which include a 1 year core phase where patients will be randomized to a 48 week treatment period to either Deferasirox DT or granules, and an optional extension phase where all patients will receive the granules up to 5 years. Patients who demonstrated benefit to granules or DT in the core phase, and/or express the wish to continue in the optional extension phase on granules, will be offered this possibility until there is local access to the new formulation (granules or FCT) or up to 5 years, whichever occurs first.

location_facility: Childrens Healthcare of Atlanta Onc Dept

location_facility: Childrens Hospital of Philadelphia Onc. Dept

location_facility: Medical University of South Carolina Medical Uni of South Carolina

location_facility: St. Jude Children's Research Hospital Memphis St Jude

title: A Randomized, Open-label, Multicenter, Two Arm, Phase II Study to Evaluate Treatment Compliance, Efficacy and Safety of an Improved Deferasirox Formulation (Granules) in Pediatric Patients With Iron Overload

location_country:
Italy
Russian Federation
India
Hungary
Tunisia
Lebanon
Belgium
United States
Philippines
Thailand
Malaysia
Panama
Turkey
Oman
Bulgaria
Egypt
France


title: An Open-label, Randomized, Controlled, Multicenter, Phase II Study Evaluating Safety and Efficacy of Intratumorally Administered Intuvax Pre-nephrectomy Followed by Sunitinib Post-nephrectomy, Compared to Sunitinib Post-nephrectomy in Metastatic Renal Cell Carcinoma Patients

acronym: MERECA

summary: The purpose of this study is to compare tumor response, progression free survival (PFS) and overall survival (OS) in newly diagnosed mRCC patients treated with Intuvax (INN: ilixadencel) pre-nephrectomy followed by Sunitinib post-nephrectomy vs Sunitinib post-nephrectomy patients.

lead_sponsor: Mendus

collaborators:
TFS Trial Form Support
Accelovance

sponsor_collaborators:
Mendus
TFS Trial Form Support
Accelovance

location_country:
Poland
Hungary
United Kingdom
Sweden
United States
Latvia
Spain
Czechia
France

location_facility: Hospital Universitario Puerta de Hierro Majadahonda

location_facility: Centre Hospitalier Universitaire de Toulouse-Hôpital Rangueil

location_facility: Niepubliczny Zakład Opieki Zdrowotnej Vesalius Sp. z o.o.

location_facility: Pauls Stradins Clinical University Hospital

location_facility: Hospital Universitari Germans Trias i Pujol

location_facility: Centre Hospitalier Universitaire d'Angers


acronym: BET

summary: The purpose of this study is to determine the safety, tolerability, pharmacokinetics, and pharmacodynamics of BMS-986158 in subjects with select advanced cancers

location_facility: Institute for Translational Oncology Research-ITOR

title: A Phase I/IIa Trial With BMS-986158, a Small Molecule Inhibitor of the Bromodomain and Extra-Terminal (BET) Proteins, as Monotherapy or in Combination With Nivolumab in Subjects With Selected Advanced Solid Tumors or Hematologic Malignancies

location_country:
Australia
United States
Spain
Canada
France


title: An Open-Label Study to Characterize the Incidence and Severity of Diarrhea in Patients With Early-Stage HER2+ Breast Cancer Treated With Neratinib and Loperamide

summary: An Open-Label Study to Characterize the Incidence and Severity of Diarrhea in Patients with Early-Stage HER2+ Breast Cancer Treated with Neratinib and Loperamide or other prophylactic measures.

ReferenceCitation: Barcenas CH, Hurvitz SA, Di Palma JA, Bose R, Chien AJ, Iannotti N, Marx G, Brufsky A, Litvak A, Ibrahim E, Alvarez RH, Ruiz-Borrego M, Chan N, Manalo Y, Kellum A, Trudeau M, Thirlwell M, Garcia Saenz J, Hunt D, Bryce R, McCulloch L, Rugo HS, Tripathy D, Chan A; CONTROL Study Investigators. Improved tolerability of neratinib in patients with HER2-positive early-stage breast cancer: the CONTROL trial. Ann Oncol. 2020 Sep;31(9):1223-1230. doi: 10.1016/j.annonc.2020.05.012. Epub 2020 May 25.

repository_institution: Washington University School of Medicine - Digital Commons@Becker

repository_institution: Spanish National Research Council (CSIC) - DIGITAL.CSIC

repository_normalized: DIGITAL.CSIC (Spain)

ipd_sharing_description:
Puma Biotechnology is committed to sharing clinical trial data and information to help physicians and patients make informed treatment decisions, and to help qualified researchers advance scientific knowledge.

In accordance with legal and regulatory requirements, Puma publishes study protocol information and clinical study results on clinical trial registries, including ClinicalTrials.gov and EU Clinical Trials Register. Puma also publishes information about clinical studies in peer-reviewed scientific journals and shares data in scientific meetings.

Puma commits to safeguarding confidentiality and patient privacy throughout the clinical trial data and information sharing process. Any patient-level data will be anonymized to protect personally identifiable information.

Qualified researchers and study participants may submit requests for other study documentation and clinical trial data to clinicaltrials@pumabiotechnology.com for consideration.

lead_sponsor: Puma Biotechnology, Inc.

location_country:
Germany
Australia
Austria
United States
Spain
Canada
France

location_facility: CHU Group Hospitalier Pitié-Salpêtrière, Service d'oncologie Médicale

location_facility: The Oncology Institute of Hope and Innovation

location_facility: Charleston Hematology Oncology Associates

location_facility: Good Samaritan Hospital Samaritan Pastega Regional Cancer Center

location_facility: Medical University of Vienna, Department of Oncology

location_facility: Medical University of Vienna,Department of Obstetrics and Gynecology

location_facility: Praxis für interdisziplinäre Onkologie & Hämatologie

location_facility: Medical University of Innsbruck-Department of Gynecology

location_facility: Mammazentrum HH am Krankenhaus Jerusalem

location_facility: UCSF Helen Diller Family Comprehensive Cancer Center

location_facility: Great Plains Health (Callahan Cancer Center)

location_facility: Cancer Center of Santa Barbara with Sansum Clinic

location_facility: Central Coast Medical Oncology Corporation

location_facility: Uniklinikum Salzburg, Landeskrankenhaus, Univ. Klinik fur Innere Medizin III der PMU

location_facility: Sana Klinikum Offenbach GmbH - Frauenklinik

location_facility: McGill University Health Centre, Cedars Cancer Centre

location_facility: Decatur Memorial Hospital Cancer Care Specialists of Central Illinois

location_facility: North Mississippi Medical Center Hematology and Oncology Services

location_facility: Torrance Memorial Physician Network Cancer Care Associates

location_facility: Universitaetsklinikum Schleswig-Holstein (UKSH), Klinik fuer Gynaekologie und Geburtshilfe, Studienzentrale Gynäkologische Onkologie (SGC) Kiel

location_facility: Hematology-Oncology Associates of the Treasure Coast

location_facility: Saint Joseph / Candler SC Cancer Specialists

location_facility: The University of Texas MD Anderson Cancer Center

location_facility: Univ. Klinik für Innere Medizin, Klin. Abt. Onkologie

location_facility: University of Maryland, Greenebaum Comprehensive Cancer Center


title: A Phase 1a/1b Multicenter, Single-Arm, Open-Label, Dose-Escalation Study to Determine the Maximum Tolerated Dose, Safety, and Preliminary Activity of Oral ACY-241 Alone and in Combination With Pomalidomide and Low-Dose Dexamethasone in Patients With Relapsed or Relapsed-and-Refractory Multiple Myeloma

summary: This is a phase 1a/1b, multicenter, single-arm, open-label, dose escalation study to determine the maximum tolerated dose (MTD) and evaluate the safety and preliminary antitumor activity of ACY-241 for oral administration as monotherapy and in combination therapy with orally administered pomalidomide and low-dose dexamethasone in eligible patients with relapsed or relapsed-and-refractory multiple myeloma (MM).

lead_sponsor: Celgene

location_country:
Germany
United States
Spain
Greece
France

location_facility: CTRC at The UT Health Science Center at San Antonio


title: A 12-Week, Multicenter, Randomized, Parallel-Group Study to Assess the Safety, Tolerability, Pharmacokinetics, Biomarker Effects, Efficacy, and Effect on Microglia Activation, as Measured by Positron Emission Tomography, of AZD3241 in Subjects With Multiple System Atrophy

summary: AZD3241 myeloperoxidase (MPO) inhibitor trial is assessing safety and tolerability, randomized trial, in patients with Multiple System Atrophy.

location_country:
Finland
Italy
United Kingdom
Sweden
Austria
United States
France


title: SHERLOC: A Phase 2 Study of MM-121 in Combination With Docetaxel Versus Docetaxel Alone in Patients With Heregulin Positive, Locally Advanced or Metastatic Non-Small Cell Lung Cancer (Merrimack Pharmaceuticals Inc.)

acronym: SHERLOC

summary: The purpose of this study is to determine whether the combination of MM-121 plus docetaxel is more effective than docetaxel alone in regards to PFS in patients with heregulin-positive NSCLC.

ReferenceCitation: Sequist LV, Gray JE, Harb WA, Lopez-Chavez A, Doebele RC, Modiano MR, Jackman DM, Baggstrom MQ, Atmaca A, Felip E, Provencio M, Cobo M, Adiwijaya B, Kuesters G, Kamoun WS, Andreas K, Pipas JM, Santillana S, Cho BC, Park K, Shepherd FA. Randomized Phase II Trial of Seribantumab in Combination with Erlotinib in Patients with EGFR Wild-Type Non-Small Cell Lung Cancer. Oncologist. 2019 Aug;24(8):1095-1102. doi: 10.1634/theoncologist.2018-0695. Epub 2019 Apr 11.

repositories_concat: PubMed Central;www.repositoriosalud.es

lead_sponsor: Elevation Oncology

sponsor_collaborators:
Elevation Oncology
Merrimack Pharmaceuticals

location_country:
Germany
Hungary
United States
Spain
Canada
France

location_facility:
CHI Creteil
Centre Léon Bérard


title: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sialic Acid Extended-Release Tablets in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

acronym: GNEM

summary: The primary objective of this study is to evaluate the effect of 6 g/day aceneuramic acid extended-release (Ace-ER) treatment of participants with GNEM on upper extremity muscle strength (upper extremity composite [UEC] score) as measured by dynamometry.

ReferenceCitation: Lochmuller H, Behin A, Caraco Y, Lau H, Mirabella M, Tournev I, Tarnopolsky M, Pogoryelova O, Woods C, Lai A, Shah J, Koutsoukos T, Skrinar A, Mansbach H, Kakkis E, Mozaffar T. A phase 3 randomized study evaluating sialic acid extended-release for GNE myopathy. Neurology. 2019 Apr 30;92(18):e2109-e2117. doi: 10.1212/WNL.0000000000006932. Epub 2019 Jan 25.

repository_institution: Newcastle Univesity - Newcastle University ePrints

ReferenceCitation: Tandon PK, Kakkis ED. The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases. Orphanet J Rare Dis. 2021 Apr 19;16(1):183. doi: 10.1186/s13023-021-01805-5.

lead_sponsor: Ultragenyx Pharmaceutical Inc

location_country:
France
Italy
United Kingdom
United States
Bulgaria
Canada
Israel

location_facility: Washington University School of Medicine

location_facility: Hadassah-Hebrew University Medical Center

location_facility: Institut de Myologie GH Pitié-Salpêtrière


lead_sponsor: Amgen

summary: The purpose of this study is to learn more about the role of etanercept alone or in combination with methotrexate on disease activity in adults with psoriatic arthritis.

ReferenceCitation: Mease PJ, Gladman DD, Samad AS, Coates LC, Liu LXH, Aras GA, Collier DH, Chung JB. Design and rationale of the Study of Etanercept and Methotrexate in Combination or as Monotherapy in Subjects with Psoriatic Arthritis (SEAM-PsA). RMD Open. 2018 Feb 3;4(1):e000606. doi: 10.1136/rmdopen-2017-000606. eCollection 2018.

ReferenceCitation: Mease PJ, Gladman DD, Collier DH, Ritchlin CT, Helliwell PS, Liu L, Kricorian G, Chung JB. Etanercept and Methotrexate as Monotherapy or in Combination for Psoriatic Arthritis: Primary Results From a Randomized, Controlled Phase III Trial. Arthritis Rheumatol. 2019 Jul;71(7):1112-1124. doi: 10.1002/art.40851. Epub 2019 May 28.

ReferenceCitation: Strand V, Mease PJ, Maksabedian Hernandez EJ, Stolshek BS, Liu LXH, Collier DH, Kricorian G, Merola JF. Patient-reported outcomes data in patients with psoriatic arthritis from a randomised trial of etanercept and methotrexate as monotherapy or in combination. RMD Open. 2021 Jan;7(1):e001484. doi: 10.1136/rmdopen-2020-001484.

ReferenceCitation: Coates LC, Merola JF, Mease PJ, Ogdie A, Gladman DD, Strand V, van Mens LJJ, Liu L, Yen PK, Collier DH, Kricorian G, Chung JB, Helliwell PS. Performance of composite measures used in a trial of etanercept and methotrexate as monotherapy or in combination in psoriatic arthritis. Rheumatology (Oxford). 2021 Mar 2;60(3):1137-1147. doi: 10.1093/rheumatology/keaa271.

ReferenceCitation: Helliwell PS, Mease PJ, Kavanaugh A, Coates LC, Ogdie A, Deodhar A, Strand V, Kricorian G, Liu LXH, Collier D, Gladman DD. Impact of clinical domains other than arthritis on composite outcomes in psoriatic arthritis: comparison of treatment effects in the SEAM-PsA trial. RMD Open. 2022 Jul;8(2):e002366. doi: 10.1136/rmdopen-2022-002366.

title: A Multicenter Double-Blind, Randomized Controlled Study of Etanercept and Methotrexate in Combination or as Monotherapy in Subjects With Psoriatic Arthritis

location_country:
Poland
Portugal
Hungary
Argentina
United Kingdom
Mexico
United States
Latvia
Spain
Greece
South Africa
Chile
Czechia
Bulgaria
Canada
Puerto Rico
Russian Federation
France