lead_sponsor: UCB BIOSCIENCES GmbH

summary: Phase 3, multicenter, randomized, double-blind, parallel-group, placebo-controlled study to evaluate the efficacy and safety of Certolizumab Pegol (CZP) in subjects with adult onset active and progressive Psoriatic Arthritis (PsA).

ReferenceCitation: Mease PJ, Fleischmann R, Deodhar AA, Wollenhaupt J, Khraishi M, Kielar D, Woltering F, Stach C, Hoepken B, Arledge T, van der Heijde D. Effect of certolizumab pegol on signs and symptoms in patients with psoriatic arthritis: 24-week results of a Phase 3 double-blind randomised placebo-controlled study (RAPID-PsA). Ann Rheum Dis. 2014 Jan;73(1):48-55. doi: 10.1136/annrheumdis-2013-203696. Epub 2013 Aug 13.

ReferenceCitation: van der Heijde D, Fleischmann R, Wollenhaupt J, Deodhar A, Kielar D, Woltering F, Stach C, Hoepken B, Arledge T, Mease PJ. Effect of different imputation approaches on the evaluation of radiographic progression in patients with psoriatic arthritis: results of the RAPID-PsA 24-week phase III double-blind randomised placebo-controlled study of certolizumab pegol. Ann Rheum Dis. 2014 Jan;73(1):233-7. doi: 10.1136/annrheumdis-2013-203697. Epub 2013 Aug 13.

ReferenceCitation: van der Heijde D, Deodhar A, FitzGerald O, Fleischmann R, Gladman D, Gottlieb AB, Hoepken B, Bauer L, Irvin-Sellers O, Khraishi M, Peterson L, Turkiewicz A, Wollenhaupt J, Mease PJ. 4-year results from the RAPID-PsA phase 3 randomised placebo-controlled trial of certolizumab pegol in psoriatic arthritis. RMD Open. 2018 Mar 14;4(1):e000582. doi: 10.1136/rmdopen-2017-000582. eCollection 2018. Erratum In: RMD Open. 2018 Mar 26;4(1):

repositories_concat: PubMed Central;touroscholar.touro.edu

ReferenceCitation: Walsh JA, Gottlieb AB, Hoepken B, Nurminen T, Mease PJ. Efficacy of certolizumab pegol with and without concomitant use of disease-modifying anti-rheumatic drugs over 4 years in psoriatic arthritis patients: results from the RAPID-PsA randomized controlled trial. Clin Rheumatol. 2018 Dec;37(12):3285-3296. doi: 10.1007/s10067-018-4227-7. Epub 2018 Sep 6. Erratum In: Clin Rheumatol. 2018 Oct 11;:

ReferenceCitation: van der Heijde D, Deodhar A, Fleischmann R, Mease PJ, Rudwaleit M, Nurminen T, Davies O. Early Disease Activity or Clinical Response as Predictors of Long-Term Outcomes With Certolizumab Pegol in Axial Spondyloarthritis or Psoriatic Arthritis. Arthritis Care Res (Hoboken). 2017 Jul;69(7):1030-1039. doi: 10.1002/acr.23092. Epub 2017 Jun 2.

repositories_institution:
Leiden University - Leiden Repository
PubMed Central - Europe PMC

ReferenceCitation: Osterhaus JT, Purcaru O. Discriminant validity, responsiveness and reliability of the arthritis-specific Work Productivity Survey assessing workplace and household productivity in patients with psoriatic arthritis. Arthritis Res Ther. 2014 Jul 4;16(4):R140. doi: 10.1186/ar4602.

ReferenceCitation: Kavanaugh A, Gladman D, van der Heijde D, Purcaru O, Mease P. Improvements in productivity at paid work and within the household, and increased participation in daily activities after 24 weeks of certolizumab pegol treatment of patients with psoriatic arthritis: results of a phase 3 double-blind randomised placebo-controlled study. Ann Rheum Dis. 2015 Jan;74(1):44-51. doi: 10.1136/annrheumdis-2014-205198. Epub 2014 Jun 18.

repository_institution: University of Oxford - Oxford Academic Notes & Queries

ReferenceCitation: Gladman D, Fleischmann R, Coteur G, Woltering F, Mease PJ. Effect of certolizumab pegol on multiple facets of psoriatic arthritis as reported by patients: 24-week patient-reported outcome results of a phase III, multicenter study. Arthritis Care Res (Hoboken). 2014 Jul;66(7):1085-92. doi: 10.1002/acr.22256.

title: Phase 3, Multicenter, Randomized, Double-Blind, Parallel Group, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Certolizumab Pegol in Subjects With Adult-Onset Active and Progressive Psoriatic Arthritis (PsA)

location_country:
Germany
Ireland
Italy
Poland
Hungary
Argentina
United Kingdom
Brazil
Mexico
Belgium
United States
Spain
Czechia
Canada
France


type: EudraCT Number

title: A Randomized, Open-label, Multi-center Phase II Study to Compare AUY922 With Docetaxel or Irinotecan in Adult Patients With Advanced Gastric Cancer, Who Have Progressed After One Line of Chemotherapy

summary: A clinical trial to determine the effectiveness and safety of AUY922 compared to other drugs known to be effective against gastric cancer in second line therapy for patients who have failed one line of chemotherapy.

status: Completed

study_completion_date_type: Actual

study_type: Interventional

design_allocation: Randomized

primary_purpose: Treatment

enrollment_type: Actual

lead_sponsor: Novartis Pharmaceuticals

location_country:
Germany
Italy
United Kingdom
Singapore
Australia
Netherlands
Switzerland
United States
Korea, Republic of
Turkey
Taiwan
Canada
Russian Federation
France

location_facility: The Sidney Kimmel Cancer Center at Johns Hopkins Hospital Dept. of SKCC @ JHU

location_facility: Tyler Cancer Center Dept.ofTylerCancerCtr. (2)

location_facility: University of California at Los Angeles Dept. of UCLA (4)

location_facility: USC/Kenneth Norris Comprehensive Cancer Center USC/Norris

location_facility: Baylor Health Care System/Sammons Cancer Center Baylor Texas Oncology

location_facility: Clinical Research Alliance Dept.ofArenaOncologyAssoc(2)

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Completed


type: Other Identifier

title: A Phase I/2 Dose Escalation Safety, Pharmacokinetic and Efficacy Study of Multiple Intravenous Administrations of a Humanized Monoclonal Antibody (SAR650984) Against CD38 in Patients With Selected CD38+ Hematological Malignancies

summary:
Primary Objective:

Phase 1:

To determine the maximum tolerated dose (MTD)/maximum administered dose (MAD) of SAR650984 (Isatuximab).

Phase 2 (stage 1):

To evaluate the activity of single-agent Isatuximab at different doses/schedules and to select dose and regimen to further evaluate the overall response rate (ORR) of Isatuximab as single agent or in combination with dexamethasone.

Phase 2 (stage 2):

To evaluate the activity in terms of overall response rate (ORR) of Isatuximab at the selected dose/schedule from stage1, as single agent (ISA arm) and in combination with dexamethasone (ISAdex arm).

Secondary Objectives:

Phase 1:

To characterize the global safety profile including cumulative toxicities.
To evaluate the pharmacokinetic (PK) profile of Isatuximab in the proposed dosing schedule(s).
To assess the pharmacodynamics (PD), immune response, and preliminary disease response.

Phase 2 (stage 1): to evaluate the following objectives for Isatuximab as single agent:

Safety
Efficacy as measured by duration of response, clinical benefit rate, progression free survival, overall survival.

Phase 2 (stage 2): to evaluate the following objectives in each arm (ISA and ISAdex):

Safety
Efficacy as measured by duration of response, clinical benefit rate, progression free survival, overall survival.
Participant-reported changes in health-related quality of life, symptoms of multiple myeloma and generic health status.
Pharmacokinetic profile of Isatuximab.
Immunogenicity of Isatuximab.
Investigate the relationship between CD38 receptor density and CD38 receptor occupancy (Stage 1 only) on multiple myeloma cells and parameters of clinical response.

study_start_date_type: Actual

ReferenceType: derived

ReferenceCitation: Dimopoulos M, Bringhen S, Anttila P, Capra M, Cavo M, Cole C, Gasparetto C, Hungria V, Jenner M, Vorobyev V, Ruiz EY, Yin JY, Saleem R, Hellet M, Mace S, Paiva B, Vij R. Isatuximab as monotherapy and combined with dexamethasone in patients with relapsed/refractory multiple myeloma. Blood. 2021 Mar 4;137(9):1154-1165. doi: 10.1182/blood.2020008209.

oa_colors: green other

oa_host_type: publisher;repository

evidence: open (via free article)

host_type: publisher

license_normalized: no license

version: publishedVersion

evidence: oa repository (via pmcid lookup)

host_type: repository

repository_normalized: PubMed Central

repositories_concat: PubMed Central

unpaywall_oa_status: bronze

publisher_dissemination: American Society of Hematology

ReferenceCitation: Mikhael J, Richter J, Vij R, Cole C, Zonder J, Kaufman JL, Bensinger W, Dimopoulos M, Lendvai N, Hari P, Ocio EM, Gasparetto C, Kumar S, Oprea C, Chiron M, Brillac C, Charpentier E, San-Miguel J, Martin T. A dose-finding Phase 2 study of single agent isatuximab (anti-CD38 mAb) in relapsed/refractory multiple myeloma. Leukemia. 2020 Dec;34(12):3298-3309. doi: 10.1038/s41375-020-0857-2. Epub 2020 May 14.

oa_colors: green hybrid

evidence: open (via crossref license)

license: cc-by

license_normalized: cc-by

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

repository_institution: University of Navarra - Deposito Adademico Digital Universidad De Navarra

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

repository_institution: University of Cantabria - UCrea

unpaywall_oa_status: hybrid

publisher_dissemination: Springer-Nature

ipd_sharing: Yes

ipd_sharing_description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

lead_sponsor: Sanofi

location_country:
Finland
Italy
Israel
Argentina
United Kingdom
Brazil
Mexico
Belgium
United States
Spain
Greece
Turkey
Chile
Peru
Ukraine
Russian Federation
France

location_facility:
Investigational Site Number 840013
Investigational Site Number 792004
Investigational Site Number 840016
Investigational Site Number 826001
Investigational Site Number 032003
Investigational Site Number 840012
Investigational Site Number 724002
Investigational Site Number 300001
Investigational Site Number 250004
Investigational Site Number 840009
Investigational Site Number 840002
Investigational Site Number 840001
Investigational Site Number 792005
Investigational Site Number 076004
Investigational Site Number 724006
Investigational Site Number 840014
Investigational Site Number 724007
Investigational Site Number 032002
Investigational Site Number 724004
Investigational Site Number 484001
Investigational Site Number 840022
Investigational Site Number 724008
Investigational Site Number 250001
Investigational Site Number 484003
Investigational Site Number 380001
Investigational Site Number 246001
Investigational Site Number 840003
Investigational Site Number 076002
Investigational Site Number 826002
Investigational Site Number 724001
Investigational Site Number 376002
Investigational Site Number 792001
Investigational Site Number 840027
Investigational Site Number 643001
Investigational Site Number 152001
Investigational Site Number 376004
Investigational Site Number 840017
Investigational Site Number 643002
Investigational Site Number 643004
Investigational Site Number 804001
Investigational Site Number 840018
Investigational Site Number 804002
Investigational Site Number 804004
Investigational Site Number 032001
Investigational Site Number 840010
Investigational Site Number 604002
Investigational Site Number 643003
Investigational Site Number 840005
Investigational Site Number 724005
Investigational Site Number 380002
Investigational Site Number 076003
Investigational Site Number 840011
Investigational Site Number 250003
Investigational Site Number 076001
Investigational Site Number 246002
Investigational Site Number 604001
Investigational Site Number 840004
Investigational Site Number 056001
Investigational Site Number 792002


title: A Phase 1/2a Open-label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, Immunogenicity and Antitumor Activity of LAVA-051 in Patients With Relapsed or Refractory CLL, MM, or AML

summary: A phase 1/2a, first-in-human trial to evaluate the safety and tolerability of LAVA-051 in patients with relapsed or refractory Chronic Lymphocytic Leukemia (CLL), Multiple Myeloma (MM), or Acute Myeloid Leukemia (AML).

status: Active, not recruiting

study_completion_date_type: Anticipated

enrollment_type: Anticipated

ipd_sharing: No

lead_sponsor: Lava Therapeutics

location_country:
United States
Spain
Netherlands
France

location_facility: Hospital Clinico Universitario de Salamanca

intervention_type: Biological

status_simplified: Ongoing


acronym: SMART

intervention_type: Genetic

summary: To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene weighing ≥ 8.5 kg and ≤ 21 kg, over a 12 month period.

title: A Phase IIIb, Open-label, Single-arm, Single-dose, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Gene Replacement Therapy With Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)

source: euctr

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 https://www.clinicalstudydatarequest.com/.

location_country:
Italy
Portugal
United Kingdom
Australia
Belgium
United States
Taiwan
Canada
France


title: Open-label Uncontrolled Trial to Evaluate Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod in Children From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis

summary:
The purpose of this trial is to investigate the PK, PD, safety, and activity of efgartigimod IV in children and adolescents aged from 2 to less than 18 years of age with gMG.

Trial details include:

The maximum trial duration for each individual participant will be approximately 28 weeks
The treatment duration will be 8 weeks for the dose-confirmatory part (Part A) and 18 weeks for the treatment response-confirmatory part (Part B)

status: Recruiting

ipd_sharing: Undecided

lead_sponsor: argenx

location_country:
Germany
Italy
Poland
United Kingdom
Netherlands
Austria
Belgium
United States
Spain
Georgia
France

location_facility:
Investigator site - UK440035
Investigator Site - ES0340041
Investigator Site - US0010120
Investigator site UK0440027
Investigator Site - PL0480038
Investigator Site - US0010003
Investigator site - US0010142
Investigator Site - GEO9950018
Investigator Site - DE0490032
Investigator Site - IT0390048
Investigator Site - ES0340040
Investigator Site - GEO9950017
Investigator site - UK440024
Investigator Site - FR0330041
Investigator site DE0490034
Investigator Site - NL0310001
Investigator Site - PL0480035
Investigator Site - PL0480034
Investigator Site - FR0330032
Investigator Site - AT0430010
Investigator Site - IT0390047
Investigator Site - BE0320021


title: BIOTRONIK Dynetic-35 for the Treatment of Peripheral Iliac Lesions, Using the Cobalt Chromium Balloon-expandable Dynetic-35 Stent in Association With Passeo-35 Xeo Peripheral Dilation Catheter

acronym: BIONETIC-I

summary: BIOTRONIK cobalt chromium balloon-expandable Dynetic-35 stent in association with Passeo-35 Xeo peripheral dilation catheter for the treatment of peripheral iliac lesions

study_type: Observational

time_perspective: Prospective

lead_sponsor: Biotronik AG

location_country: Germany Hungary Austria Belgium Latvia France

location_facility: Pauls Stradins Clinical University Hospital

location_facility: LKH Univ.-Klinikum Graz, Ambulanz für Angiologie

intervention_type: Device


title: An Open-Label Phase 2 Study of Surufatinib in Patients With Neuroendocrine Tumours in Europe

summary: This is a Phase 2, open-label, multi-centre study of surufatinib in patients with low to intermediate grade (Grade 1 or Grade 2), well-differentiated neuroendocrine tumours (NETs).

lead_sponsor: Hutchmed

location_country:
Germany
Italy
Norway
United Kingdom
United States
Spain
France

location_facility: Azienda Universitaria Ospedaliera Consorziale - Policlinico Bari

location_facility: Universitatsklinikum Essen, Klinik fur Endokrinologie

location_facility: University of California Irvine Medical Center UCIMC - H.H. Chao Comprehensive Digestive Disease Center CDDC

location_facility: Institut Catala d'Oncologis (ICO) - Hospital Duran i Reynals

location_facility: Emory University, Winship Cancer Institute

location_facility: Universita degli Studi di Firenze - Azienda Ospedaliero Universitaria Careggi (AOUC)


title: A Phase 1, Multi-center, Open Label First-in-Human Study With ABBV-CLS-579 Alone and in Combination in Subjects With Locally Advanced or Metastatic Tumors

summary:
The purpose of this study is to see how safe and effective ABBV-CLS-579 is when used alone and in combination with a PD-1 target agent or with a VEGF TKI.

ABBV-CLS-579 is an investigational drug being developed for the treatment of tumors.

The trial aims to establish a safe, tolerable, and efficacious dose of ABBV-CLS-579 as monotherapy and in combination. The study will be conducted in three parts. Part 1 Monotherapy Dose Escalation, Part 2 Combination Dose Escalation, and Part 3 Combination Dose Expansion.

Part 1, ABBV-CLS-579 will be administered alone in escalating dose levels to eligible subjects who have advanced solid tumors.

Part 2, ABBV-CLS-579 will be administered at escalating dose levels in combination with a PD-1 targeting agent to eligible subjects who have advanced solid tumors.

Part 3, ABBV-CLS-579 will be administered at the determined recommended dose in combination with a PD-1 target agent or with a VEGFR TKI in subjects with locally advanced or metastatic, relapsed or refractory head and neck squamous cell carcinoma (HNSCC), relapsed or refractory non-small cell lung cancer (NSCLC), and advanced clear cell renal cell carcinoma (ccRCC).

Adult participants with a diagnosis of some solid tumors for which no effective standard therapy exists or has failed will be enrolled. Participants will receive study treatment until disease progresses or discontinued.

There may be a higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the course of the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects, and completing questionnaires.

design_allocation: Non-Randomized

lead_sponsor: Calico Life Sciences LLC

sponsor_collaborators:
Calico Life Sciences LLC
AbbVie

location_country:
France
Japan
United States
Korea, Republic of
Spain
Taiwan
Israel

location_facility: Hospital Universitario Fundacion Jimenez Diaz

location_facility: Hospital Universitario Virgen de la Victoria

location_facility: Fort Wayne Medical Oncology and Hematology


title: Neo-adjuvant Chemotherapy Combined With Stereotactic Body Radiotherapy to the Primary Tumour +/- Durvalumab, +/- Oleclumab in Luminal B Breast Cancer: a Phase ll Randomised Trial

acronym: Neo-CheckRay

summary:
Neo-CheckRay is a multicenter, open-label phase II study that randomizes luminal B breast cancer subjects candidate for neo-adjuvant chemotherapy in a 1:1:1 ratio in 3 arms:

the combination of weekly paclitaxel followed by dose-dense doxorubicin-cyclophosphamide (ddAC) and pre-operative radiation therapy (boost dose) on the primary tumour
arm 1 with the addition of the anti-PD-L1 antibody durvalumab
arm 2 with the addition of the anti-CD73 antibody oleclumab The primary tumour will be excised 2-6 weeks after completion of ddAC. A safety run-in is planned for the 6 first subjects before starting the randomized phase II trial. Those 6 subjects will receive the treatment given in Arm 3.

ReferenceCitation: De Caluwe A, Buisseret L, Poortmans P, Van Gestel D, Salgado R, Sotiriou C, Larsimont D, Paesmans M, Craciun L, Stylianos D, Vandekerckhove C, Reyal F, Isabelle V, Eiger D, Piccart M, Romano E, Ignatiadis M. Neo-CheckRay: radiation therapy and adenosine pathway blockade to increase benefit of immuno-chemotherapy in early stage luminal B breast cancer, a randomized phase II trial. BMC Cancer. 2021 Aug 6;21(1):899. doi: 10.1186/s12885-021-08601-1.

oa_colors: green gold

evidence: oa journal (via doaj)

repository_institution: Université Libre de Bruxelles - Dépôt institutionnel de l'Université libre de Bruxelles

repository_institution: University of Antwerp - Institutional Repository University of Antwerp

repository_normalized: irua

repositories:
PubMed Central
dipot.ulb.ac.be
irua

repositories_concat: PubMed Central;dipot.ulb.ac.be;irua

unpaywall_oa_status: gold

lead_sponsor: Jules Bordet Institute

collaborators:
Institut Curie
AstraZeneca

sponsor_collaborators:
Jules Bordet Institute
Institut Curie
AstraZeneca

location_country: Belgium France

location_facility: GZA - Ziekenhuizen (Campus St. Augustinus)


title: Follow-up in Early and Locally Advanced Breast Cancer Patients

summary: A cross-sectional follow-up study of Early and Locally Advanced Breast Cancer patients after primary treatment.

lead_sponsor: European Organisation for Research and Treatment of Cancer - EORTC

location_country:
Germany
Italy
Poland
United Kingdom
Netherlands
Switzerland
Belgium
Spain
Jordan
France

location_facility: Group Of Private Clinics Hirslanden - Hirslanden Klinik-Tumor Zentrum Aarau

location_facility: HELIOS Kliniken - Helios Klinikum Wuppertal - Klin. Univ. Witten / Herdecke

location_facility: Universitaetsklinikum Leipzig-Klinik fuer Strahlentherapie und Radioonkologie

location_facility: Barts Health NHS Trust - St. Bartholomew'S Hospital

location_facility: Assistance Publique Hopitaux Paris - Assistance Publique - Hopitaux de Paris - Hopital Tenon

location_facility: CHU Saint Pierre - CHU Saint-Pierre-Site Porte de Hal

location_facility: Leeds Teaching Hospitals NHS Trust - St. James's University Hospital

location_facility: The Netherlands Cancer Institute-Antoni Van Leeuwenhoekziekenhuis

location_facility: Maria Sklodowska-Curie Memorial Cancer Centre - Maria Sklodowska-Curie National Research Institute of Oncology

location_facility: Groupe Radiopole Artois - Centre de radiotherapie Marie Curie

location_facility: Tumor and Breast Center ZeTuP St. Gallen

location_facility: Universita Di Roma La Sapienza - Universita Degli Studi Di Roma La Sapienza - Ospedale Sant'Andrea

location_facility: CHU-UCL Namur - CHU Site Sainte-Elisabeth-UCL Namur


acronym: Stellar

lead_sponsor: ProQR Therapeutics

summary: The purpose of this study is to evaluate the safety and tolerability of QR-421a administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.

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.

oa_host_type: repository

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

version: submittedVersion

unpaywall_oa_status: green

publisher_dissemination: Informa

location_facility: Centre de maladies rares CHNO des Quinze Vingts

location_facility: Casey Eye Institute, Oregon Health & Science University

location_facility: University of Michigan, Kellogg Eye Center

location_facility: Centre for Innovative Medicine, Department of Paediatric Surgery, Montreal Children's Hospital at the McGill University Health Centre

location_facility: Center for Clinical Research Operations, Massachusetts Eye and Ear

location_facility: Hôpital Gui de Chauliac - CHRU de Montpellier - Maladies Sensorielles Génétique

title: A First-in-Human Study to Evaluate the Safety and Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene

location_country:
United States
Canada
France


acronym: ILLUMINATE-A

lead_sponsor: Alnylam Pharmaceuticals

summary: The purpose of this study is to evaluate the efficacy and safety of lumasiran in children and adults with primary hyperoxaluria type 1 (PH1).

ReferenceCitation: Garrelfs SF, Frishberg Y, Hulton SA, Koren MJ, O'Riordan WD, Cochat P, Deschenes G, Shasha-Lavsky H, Saland JM, Van't Hoff WG, Fuster DG, Magen D, Moochhala SH, Schalk G, Simkova E, Groothoff JW, Sas DJ, Meliambro KA, Lu J, Sweetser MT, Garg PP, Vaishnaw AK, Gansner JM, McGregor TL, Lieske JC; ILLUMINATE-A Collaborators. Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1. N Engl J Med. 2021 Apr 1;384(13):1216-1226. doi: 10.1056/NEJMoa2021712.

repository_institution: University College London - UCL Discovery

repository_normalized: UCL Discovery

repositories_concat: UCL Discovery

publisher_dissemination: Massachusetts Medical Society

title: ILLUMINATE-A: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study With an Extended Dosing Period to Evaluate the Efficacy and Safety of Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1

location_country:
Germany
France
United Kingdom
Netherlands
Switzerland
United Arab Emirates
United States
Israel


lead_sponsor: Bayer

summary: The purpose of this study was to assess the safety and tolerability of multiple doses of a human monoclonal antibody (BAY1093884) given under the skin in subjects with hemophilia A or B. This antibody was intended to protect from bleeds by inhibiting a substance (Tissue Factor Pathway Inhibitor, TFPI) that reduces the ability of the body to form blood clots.

location_facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

location_facility: Haematology Service, Canterbury Health Laboratories

location_facility: SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD

status: Terminated

title: Multiple Escalating Dose Study of BAY1093884 in Adults With Hemophilia A or B With or Without Inhibitors

location_country:
Italy
New Zealand
Hungary
United Kingdom
Australia
Japan
Austria
Korea, Republic of
Taiwan
Bulgaria
France

status_simplified: Unknown


title: A Non-Interventional Observational Study of Pegvaliase-Naïve Adults With Phenylketonuria (PKU): Concept Elicitation and Cognitive Interviews

summary: The 165-901 study is designed to identify the appropriate tools for use in future interventional studies on the neurocognitive effects of pegvaliase on adults with PKU.

time_perspective: Retrospective

lead_sponsor: BioMarin Pharmaceutical

location_country:
Germany
Italy
United Kingdom
United States
Turkey
Canada
France

location_facility: Bretonneau Hospital, Internal Medicine Department

location_facility: Rheumatology Unit, Clinic for Inborn Errors of Metabolism

location_facility: National Hospital for Neurology and Neurosurgery

location_facility: Emory University Department of Human Genetics

location_facility: University Klinik Jena, Klinik für Neuropädiatrie

location_facility: Adult Metabolic Diseases Clinic, Vancouver General Hospital

location_facility: Ann and Robert H Lurie Children's Hospital


type: Registry Identifier

title: A Phase 1/2 Open-label, Multi-center, Safety, Preliminary Efficacy and Pharmacokinetic (PK) Study of Isatuximab (SAR650984) in Combination With REGN2810, or Isatuximab Alone, in Patients With Advanced Malignancies

summary:
Primary Objectives:

To characterize the safety and tolerability of isatuximab in combination with REGN2810 in participants with metastatic, castration-resistant prostate cancer (mCRPC) who were naïve to anti-programmed cell death-1 (PD-1)/programmed cell death-ligand 1 (PD-L1)-containing therapy, or non-small cell lung cancer (NSCLC) who progressed on anti-PD-1/PD-L1-containing therapy, and to confirm the recommended Phase 2 dose (RP2D).
To assess the response rate of isatuximab in combination with REGN2810 in participants with either mCRPC who were anti-PD-1/PD-L1 therapy naive, or NSCLC who progressed on anti-PD-1/PD-L1 therapy, or of isatuximab as single agent in participants with mCRPC.

Secondary Objectives:

To evaluate the safety of the combination of isatuximab with REGN2810 or isatuximab monotherapy.
To evaluate the immunogenicity of isatuximab and REGN2810.
To characterize the pharmacokinetic (PK) profile of isatuximab single agent or in combination with REGN2810, and to characterize the PK of REGN2810 in combination with isatuximab.
To assess overall efficacy of isatuximab in combination with REGN2810 or as a single agent.

ReferenceCitation: Zucali PA, Lin CC, Carthon BC, Bauer TM, Tucci M, Italiano A, Iacovelli R, Su WC, Massard C, Saleh M, Daniele G, Greystoke A, Gutierrez M, Pant S, Shen YC, Perrino M, Meng R, Abbadessa G, Lee H, Dong Y, Chiron M, Wang R, Loumagne L, Lepine L, de Bono J. Targeting CD38 and PD-1 with isatuximab plus cemiplimab in patients with advanced solid malignancies: results from a phase I/II open-label, multicenter study. J Immunother Cancer. 2022 Jan;10(1):e003697. doi: 10.1136/jitc-2021-003697.

license: cc-by-nc

license_normalized: cc-by-nc

repository_institution: Newcastle Univesity - Newcastle University ePrints

repositories_concat: PubMed Central;eprints.ncl.ac.uk

publisher_dissemination: British Medical Journal

location_country:
Italy
United Kingdom
United States
Taiwan
France

location_facility:
Investigational Site Number 3800004
Investigational Site Number 3800001
Investigational Site Number 8400003
Investigational Site Number 2500002
Investigational Site Number 1580001
Investigational Site Number 8400005
Investigational Site Number 8400004
Investigational Site Number 3800006
Investigational Site Number 8400007
Investigational Site Number 1580002
Investigational Site Number 8260001
Investigational Site Number 8400002
Investigational Site Number 3800005
Investigational Site Number 3800003
Investigational Site Number 2500001
Investigational Site Number 8260002


title: Observational Registry Study of Leber Hereditary Optic Neuropathy (LHON) Affected Patients

acronym: REALITY

summary: This study is a multi-country retrospective and cross-sectional observational study of affected LHON subjects, based on retrospective subjects' medical chart abstractions and cross-sectional administration of patient-reported outcomes (PROs).

time_perspective: Cross-Sectional

ReferenceCitation: Newman NJ, Yu-Wai-Man P, Carelli V, Biousse V, Moster ML, Vignal-Clermont C, Sergott RC, Klopstock T, Sadun AA, Girmens JF, La Morgia C, DeBusk AA, Jurkute N, Priglinger C, Karanjia R, Josse C, Salzmann J, Montestruc F, Roux M, Taiel M, Sahel JA. Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A ND4 Mutation: Systematic Review and Indirect Comparison. Front Neurol. 2021 May 24;12:662838. doi: 10.3389/fneur.2021.662838. eCollection 2021.

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Université de Nantes

repository_normalized: HAL

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - memSIC

repository_institution: California Digital Library - eScholarship

repositories:
HAL
PubMed Central
UCL Discovery
escholarship.org

repositories_institution: University College London - UCL Discovery

publisher_dissemination: Frontiers

lead_sponsor: GenSight Biologics

location_country:
Italy
United Kingdom
United States
Spain
France

location_facility:
Emory University Hospital
Ospedale Bellaria
Wills Eye Institute
CHNO Les Quinze Vingts
Ospedale San Raffaele
Institut Catala de Retina
CHU d'Angers
Doheny Eye Center UCLA Pasadena
Alkek Eye Center
Moorfields Eye Hospital
Massachusetts Eye and Ear Infirmary

intervention_type: Other


title: A Phase 1, First-in-Human, Open-Label, Dose Escalation Study of Teclistamab, a Humanized BCMA x CD3 Bispecific Antibody in Subjects With Relapsed or Refractory Multiple Myeloma

summary: The purpose of this study is to identify the recommended Phase 2 dose(s) (RP2Ds) and schedule assessed to be safe for Teclistamab and to characterize the safety and tolerability of Teclistamab at the RP2Ds.

ReferenceCitation: Girgis S, Lin SXW, Pillarisetti K, Banerjee A, Stephenson T, Ma X, Shetty S, Yang TY, Hilder BW, Jiao Q, Hanna B, Adams HC 3rd, Sun YN, Sharma A, Smit J, Infante JR, Goldberg JD, Elsayed Y. Translational Modeling Predicts Efficacious Therapeutic Dosing Range of Teclistamab for Multiple Myeloma. Target Oncol. 2022 Jul;17(4):433-439. doi: 10.1007/s11523-022-00893-y. Epub 2022 Jun 24. Erratum In: Target Oncol. 2022 Aug 1;:

ReferenceCitation: Moreau P, Garfall AL, van de Donk NWCJ, Nahi H, San-Miguel JF, Oriol A, Nooka AK, Martin T, Rosinol L, Chari A, Karlin L, Benboubker L, Mateos MV, Bahlis N, Popat R, Besemer B, Martinez-Lopez J, Sidana S, Delforge M, Pei L, Trancucci D, Verona R, Girgis S, Lin SXW, Olyslager Y, Jaffe M, Uhlar C, Stephenson T, Van Rampelbergh R, Banerjee A, Goldberg JD, Kobos R, Krishnan A, Usmani SZ. Teclistamab in Relapsed or Refractory Multiple Myeloma. N Engl J Med. 2022 Aug 11;387(6):495-505. doi: 10.1056/NEJMoa2203478. Epub 2022 Jun 5.

version: acceptedVersion

ReferenceCitation: Usmani SZ, Garfall AL, van de Donk NWCJ, Nahi H, San-Miguel JF, Oriol A, Rosinol L, Chari A, Bhutani M, Karlin L, Benboubker L, Pei L, Verona R, Girgis S, Stephenson T, Elsayed Y, Infante J, Goldberg JD, Banerjee A, Mateos MV, Krishnan A. Teclistamab, a B-cell maturation antigen x CD3 bispecific antibody, in patients with relapsed or refractory multiple myeloma (MajesTEC-1): a multicentre, open-label, single-arm, phase 1 study. Lancet. 2021 Aug 21;398(10301):665-674. doi: 10.1016/S0140-6736(21)01338-6. Epub 2021 Aug 10.

oa_host_type: closed

repositories_concat: closed

publisher_dissemination: Elsevier

ReferenceCitation: Pillarisetti K, Powers G, Luistro L, Babich A, Baldwin E, Li Y, Zhang X, Mendonca M, Majewski N, Nanjunda R, Chin D, Packman K, Elsayed Y, Attar R, Gaudet F. Teclistamab is an active T cell-redirecting bispecific antibody against B-cell maturation antigen for multiple myeloma. Blood Adv. 2020 Sep 22;4(18):4538-4549. doi: 10.1182/bloodadvances.2020002393.

lead_sponsor: Janssen Research & Development, LLC

location_country:
France
Netherlands
United States
Spain
Sweden

location_facility: Icahn School of Medicine at Mount Sinai Program for the Protection of Human Subjects


title: An Open-Label, Multi-Center, Randomized, Dose-Escalation, Phase 1b Study to Evaluate Safety, Pharmacokinetics and Therapeutic Activity of RO6874281 in Combination With Atezolizumab ± Bevacizumab in Patients With Unresectable Advanced and/or Metastatic Renal Cell Carcinoma

summary: This is an open-label, multi-center, randomized, Phase 1b, adaptive, clinical study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary therapeutic activity of RO6874281 in combination with atezolizumab with/without bevacizumab in participants with unresectable advanced and/or metastatic RCC. The study will consist of a dose-escalation part and an extension part.

lead_sponsor: Hoffmann-La Roche

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

location_facility: Institut Claudius Regaud; Departement Oncologie Medicale

location_facility: University of Maryland Greenebaum Comprehensive Cancer Center

location_facility: Northwestern Center for Clinical Research; Cancer Center

location_facility: Clinica Universitaria de Navarra; Servicio de Oncologia

location_facility: Barts & London School of Med; Medical Oncology

location_facility: Fondazione IRCCS Policlinico San Matteo, Oncologia

location_facility: Hospital Clínic i Provincial; Servicio de Oncología

location_facility: Uniklinikum, Comprehensive Cancer Center Mainfranken; Interdisziplinäres Studienzentrum mit ECTU

location_facility: Southampton General Hospital; Medical Oncology

location_facility: Centre Léon Bérard - Centre régional de lutte contre le cancer Rhône-Alpes

location_facility: Universita di Modena e Reggio Emilia;Dipartimento di Oncologia ed Ematologia

location_facility: Hospital Ramon y Cajal; Servicio de Oncologia

location_facility: Universitätsklinikum Tübingen; Klinik für Urologie

location_facility: Herlev Hospital; Afdeling for Kræftbehandling

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

location_facility: Hospital Clinico Universitario de Valencia; Servicio de Onco-hematologia

location_facility: START Madrid-FJD, Hospital Fundacion Jimenez Diaz


title: Randomized Evaluation of BST-CarGel Versus Microfracture Alone On Recovery From Distal Femoral Cartilage Lesions

acronym: RECORD

summary: This multi-centre randomized, controlled trial will assess the impact of BST-CarGel scaffold with microfracture versus microfracture alone on short and long term clinical benefit in patients with cartilage lesions of the femoral condyle requiring operative management.

primary_purpose: Basic Science

ReferenceType: result

ReferenceCitation: Shive MS, Stanish WD, McCormack R, Forriol F, Mohtadi N, Pelet S, Desnoyers J, Methot S, Vehik K, Restrepo A. BST-CarGel(R) Treatment Maintains Cartilage Repair Superiority over Microfracture at 5 Years in a Multicenter Randomized Controlled Trial. Cartilage. 2015 Apr;6(2):62-72. doi: 10.1177/1947603514562064.

evidence: open (via free pdf)

license: unspecified-oa

license_normalized: unspecified-oa

repository_institution: PubMed Central - Europe PMC

publisher_dissemination: SAGE Publications

ReferenceCitation: Stanish WD, McCormack R, Forriol F, Mohtadi N, Pelet S, Desnoyers J, Restrepo A, Shive MS. Novel scaffold-based BST-CarGel treatment results in superior cartilage repair compared with microfracture in a randomized controlled trial. J Bone Joint Surg Am. 2013 Sep 18;95(18):1640-50. doi: 10.2106/JBJS.L.01345.

publisher_dissemination: Wolters Kluwer Health

lead_sponsor: Piramal Healthcare Canada Ltd

sponsor_collaborators:
Piramal Healthcare Canada Ltd
Smith & Nephew, Inc.
Global Research Solutions

location_country:
Germany
United Kingdom
Australia
Switzerland
Spain
Canada
France

location_facility:
Hospital Universitari del Mar
Fowler Kennedy Sport Medicine Clinic
Calvary Wakefield Hospital
Hopital de La Croix-Rousse
Hospital Quironsalud Barcelona
University Medical Centre Regensburg
Hospital Universitari Vall d'Hebron
SportClinic Zurich / Hirslanden Clinic
University Hospital Southampton
Banff Sport Medicine
Murdoch Orthopaedic Clinic
CHRU Nancy - Hospital Central
Hospital Universitario Miguel Servet
Hôpital Maisonneuve-Rosemont
The Royal Orthopaedic Hospital

intervention_type: Procedure


title: An Open-label, Multi-center Rollover Protocol for Patients Who Have Participated in a Novartis-sponsored Ribociclib (LEE011) Study and Are Continuing to Benefit From Ribociclib as Single Agent or in Combination With Other Investigational Treatments

summary: This study is to allow continued use of ribociclib (LEE011) as single agent or in combination with other investigational treatments in patients benefitting from treatment in an eligible Novartis-sponsored ribociclib (LEE011) study that has reached its primary objective(s) or has been halted for other reasons.

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

lead_sponsor: Novartis Pharmaceuticals

location_country:
Singapore
United States
Spain
Taiwan
France

location_facility: The Regents of the University of Michigan


title: A Post-Market, Prospective, Multi-Center, Single-Arm Clinical Investigation of Phasix™ Mesh for VHWG Grade 3 Midline Hernia Repair

summary: The purpose of this study is to collect additional data on safety and performance of Phasix™ Mesh in subjects requiring Ventral Hernia Working Group (VHWG) Grade 3 midline hernia repair.

ReferenceCitation: Van Rooijen MMJ, Tollens T, Jorgensen LN, de Vries Reilingh TS, Piessen G, Kockerling F, Miserez M, Windsor ACJ, Berrevoet F, Fortelny RH, Dousset B, Woeste G, van Westreenen HL, Gossetti F, Lange JF, Tetteroo GWM, Koch A, Jeekel J. Slowly resorbable biosynthetic mesh: 2-year results in VHWG grade 3 hernia repair. Hernia. 2022 Feb;26(1):131-138. doi: 10.1007/s10029-021-02453-1. Epub 2021 Jul 19.

repository_institution: Ghent University - Ghent University Academic Bibliography

repository_institution: KU Leuven - Lirias

repository_normalized: LIRIAS (KU Leuven)

repositories:
LIRIAS (KU Leuven)
PubMed Central
archive.ugent.be

repositories_concat: LIRIAS (KU Leuven);PubMed Central;archive.ugent.be

ReferenceCitation: van Rooijen MMJ, Jairam AP, Tollens T, Jorgensen LN, de Vries Reilingh TS, Piessen G, Kockerling F, Miserez M, Windsor ACJ, Berrevoet F, Fortelny RH, Dousset B, Woeste G, van Westreenen HL, Gossetti F, Lange JF, Tetteroo GWM, Koch A, Kroese LF, Jeekel J. A post-market, prospective, multi-center, single-arm clinical investigation of Phasix mesh for VHWG grade 3 midline incisional hernia repair: a research protocol. BMC Surg. 2018 Nov 20;18(1):104. doi: 10.1186/s12893-018-0439-7.

repository_institution: Goethe University Frankfurt - Publication Server of Goethe University Frankfurt am Main

repository_institution: University of Copenhagen - Research at the University of Copenhagen

repository_normalized: Pure (Denmark)

repository_institution: Erasmus University Rotterdam - EUR Research Repository

repository_institution: DANS - Data Archiving and Networked Services - NARCIS - National Academic Research and Collaborations Information System

repositories_concat: LIRIAS (KU Leuven);PubMed Central;Pure (Denmark);archive.ugent.be;publikationen.ub.uni-frankfurt.de;pure.eur.nl;repub.eur.nl

location_country:
Germany
Italy
United Kingdom
Denmark
Netherlands
Austria
Belgium
France

location_facility: University College London Hospitals NHS Trust

location_facility: Klinikum der Johann Wolfgang Goethe-Universität

location_facility: Centre Hospitalier Régional Universitaire de Lille


title: An Open-Label, Phase 2, Pilot Study Investigating the Safety, Clinical Activity, Pharmacokinetics, and Pharmacodynamics of Oral Treatment With the BTK Inhibitor PRN1008 in Patients With Newly Diagnosed or Relapsing Pemphigus Vulgaris

summary: Open-label cohort study in adult patients with newly diagnosed or relapsing pemphigus vulgaris, with intra-patient dose-adjustment based on clinical response and BTK occupancy, and with conventional immunosuppressive "rescue treatment", if indicated. The duration of therapy in Part A will be 12 weeks, followed by 12 weeks of follow up. The extension phase, Part B includes 24 weeks of therapy, followed by 4 weeks of follow-up.

lead_sponsor: Principia Biopharma, a Sanofi Company

sponsor_collaborators:
Principia Biopharma, a Sanofi Company
Principia Biopharma Australia Pty Ltd.

location_country: France Australia Greece Croatia Israel

location_facility: Hospital of Venereal and Skin Diseases A.Syggros

location_facility: Papageorgiou General Hospital of Thessaloniki


summary:
The purpose of this randomized controlled Phase II study is to assess the efficacy of PDR001 versus investigator's choice of chemotherapy in patients with advanced nasopharyngeal carcinoma (NPC).

By blocking the interaction between PD-1 and its ligands PD-L1 and PD-L2, PDR001 leads to the activation of a T-cell mediated antitumor immune response.

ReferenceCitation: Even C, Wang HM, Li SH, Ngan RK, Dechaphunkul A, Zhang L, Yen CJ, Chan PC, Chakrabandhu S, Ma BBY, Tanasanvimon S, Lee VHF, Lou PJ, Li Z, Spira AI, Sukari A, Guigay J, McCune S, Gonzalez-Maffe J, Szpakowski S, Yao Y, Liang H, Mataraza J, Sechaud R, Manenti L, Lim DW. Phase II, Randomized Study of Spartalizumab (PDR001), an Anti-PD-1 Antibody, versus Chemotherapy in Patients with Recurrent/Metastatic Nasopharyngeal Cancer. Clin Cancer Res. 2021 Dec 1;27(23):6413-6423. doi: 10.1158/1078-0432.CCR-21-0822. Epub 2021 Aug 25.

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

publisher_dissemination: American Association for Cancer Research

location_facility: Northwest Georgia Oncology Center NWGA Onc - Carrollton

location_facility: NYU Laura and Isaac Perlmutter Cancer Center Laura & Isaac Perlmutter Ctr

title: A Phase II, Open-label, Randomized Controlled Study of PDR001 in Patients With Moderately Differentiated/Undifferentiated Locally Advanced Recurrent or Metastatic Nasopharyngeal Carcinoma Who Progressed on Standard Treatment

location_country:
Singapore
Hong Kong
United States
China
Thailand
Taiwan
France


title: An Open-label, Multicenter, Dose Escalation Phase 1b Study to Assess the Safety and Pharmacokinetics of Subcutaneous Delivery of Daratumumab With the Addition of Recombinant Human Hyaluronidase (rHuPH20) for the Treatment of Subjects With Relapsed or Refractory Multiple Myeloma

summary: The purpose of the study is to evaluate the pharmacokinetics and safety from the mixture of daratumumab and rHuPH20 prepared immediately before administration via Subcutaneous (SC) delivery (Part 1) and CF (co-formulated daratumumab and rHuPH20 preparation) administration via SC delivery of daratumumab (Part 2) and to evaluate the safety of Dara-CF 1800 milligram (mg) SC delivery without pre-dose and post-dose corticosteroids (Part 3).

ReferenceCitation: Usmani SZ, Nahi H, Mateos MV, van de Donk NWCJ, Chari A, Kaufman JL, Moreau P, Oriol A, Plesner T, Benboubker L, Hellemans P, Masterson T, Clemens PL, Luo M, Liu K, San-Miguel J. Subcutaneous delivery of daratumumab in relapsed or refractory multiple myeloma. Blood. 2019 Aug 22;134(8):668-677. doi: 10.1182/blood.2019000667. Epub 2019 Jul 3.

location_country:
Denmark
Sweden
Netherlands
United States
Spain
France


lead_sponsor: Five Prime Therapeutics, Inc.

summary: This is a phase 1/2 single arm, open-label, safety, tolerability, and PK study of cabiralizumab in PVNS/dt-TGCT patients.

location_facility: University Hospitals Birmingham NHS Foundation Trust

location_facility: Klinika Nowotworow Tkanek Miekkich, Kosci i Czerniakow, Centrum Onkologii-Instytut im. M. Sklodowskiej-Curie

location_facility: Institut Bergonie- CRLCC de Bordeaux et du Sud-Ouest

location_facility: The University of Texas, MD Anderson Cancer Center

title: A Phase 1/2 Study of Cabiralizumab, an Anti-CSF1 Receptor Antibody, in Patients With Pigmented Villonodular Synovitis (PVNS)/ Diffuse Type Tenosynovial Giant Cell Tumor (Dt-TGCT)

location_country:
Poland
United Kingdom
Netherlands
United States
Korea, Republic of
France


title: A Phase Ib, Open-label, Multicenter, Dose Escalation and Expansion Study, to Evaluate the Safety, Pharmacokinetics and Activity of INC280 in Combination With Cetuximab in c-MET Positive CRC and HNSCC Patients Who Have Progressed After Anti-EGFR Monoclonal Antibody Therapy.

summary:
This was an open-label, phase Ib, multicenter clinical trial to determine the MTD/RDE of the orally administered c-MET inhibitor INC280 in combination with cetuximab. This combination was to be explored in c-MET positive mCRC and HNSCC patients whose disease progressed on cetuximab or panitumumab treatment. The dose escalation part was to be guided by a Bayesian Logistic Regression Model with overdose control. At MTD/RDE, additional mCRC and HNSCC patients who progressed on cetuximab or panitumumab treatment were to be enrolled in two expansion groups to further assess the anti-tumor activity and the safety and tolerability of the combination of INC280 and cetuximab. Patients were to receive INC280 on a continuous bid dosing regimen and cetuximab every week. A treatment cycle was defined as 28 days with no scheduled break between cycles.

The trial was terminated because of difficulties in identifying patients who met the eligibility criteria.

ReferenceCitation: Delord JP, Argiles G, Fayette J, Wirth L, Kasper S, Siena S, Mesia R, Berardi R, Cervantes A, Dekervel J, Zhao S, Sun Y, Hao HX, Tiedt R, Vicente S, Myers A, Siu LL. A phase 1b study of the MET inhibitor capmatinib combined with cetuximab in patients with MET-positive colorectal cancer who had progressed following anti-EGFR monoclonal antibody treatment. Invest New Drugs. 2020 Dec;38(6):1774-1783. doi: 10.1007/s10637-020-00928-z. Epub 2020 May 14.

location_country:
Germany
Italy
Belgium
United States
Spain
Canada
France

location_facility: University of Utah / Huntsman Cancer Institute Onc Dept

location_facility: Massachusetts General Hospital Head & Neck


acronym: RIALTO

lead_sponsor: Amgen

summary:
Primary Objective:

To estimate the incidence of treatment-emergent and treatment-related adverse events during treatment with blinatumomab in pediatric and adolescent subjects with B-precursor ALL in second or later bone marrow relapse, in any marrow relapse after alloHSCT, or refractory to other treatments

Secondary Objective(s):

To describe key efficacy outcomes, including incidence of complete response (CR) within 2 cycles of blinatumomab, minimal residual disease (MRD) remission within 2 cycles of blinatumomab, relapse free survival (RFS), overall survival (OS), incidence of alloHSCT, and 100-day mortality after alloHSCT.

Hypotheses:

A formal statistical hypothesis will not be tested. The incidence of treatment-emergent and treatment-related adverse events will be estimated.

Study Endpoints:

Incidence of treatment-emergent and treatment-related adverse events
Incidence of CR within 2 cycles of blinatumomab
MRD remission within 2 cycles of blinatumomab
RFS
OS
Incidence of alloHSCT
100-day mortality after alloHSCT

Study Design:

Multi-center, open-label, single-arm expanded access protocol

ReferenceType: background

ReferenceCitation: Locatelli F, Zugmaier G, Mergen N, Bader P, Jeha S, Schlegel PG, Bourquin JP, Handgretinger R, Brethon B, Rossig C, Kormany WN, Viswagnachar P, Chen-Santel C. Blinatumomab in pediatric relapsed/refractory B-cell acute lymphoblastic leukemia: RIALTO expanded access study final analysis. Blood Adv. 2022 Feb 8;6(3):1004-1014. doi: 10.1182/bloodadvances.2021005579.

ReferenceCitation: Locatelli F, Zugmaier G, Mergen N, Bader P, Jeha S, Schlegel PG, Bourquin JP, Handgretinger R, Brethon B, Rossig C, Chen-Santel C. Blinatumomab in pediatric patients with relapsed/refractory acute lymphoblastic leukemia: results of the RIALTO trial, an expanded access study. Blood Cancer J. 2020 Jul 24;10(7):77. doi: 10.1038/s41408-020-00342-x. No abstract available. Erratum In: Blood Cancer J. 2021 Feb 1;11(2):28. Blood Cancer J. 2021 Oct 27;11(10):173.

status: No longer available

title: An Open-Label, Multi-center, Expanded Access Protocol of Blinatumomab for the Treatment of Pediatric and Adolescent Subjects With Relapsed and/or Refractory B-precursor Acute Lymphoblastic Leukemia (ALL)

location_country:
Germany
Italy
United Kingdom
Switzerland
Austria
United States
France

study_type: Expanded Access


lead_sponsor: Octapharma

summary: The purpose of the study is to collect long-term data on the inhibitor development rate of Human-cl rhFVIII in previously untreated patients with severe Hemophilia A.

location_facility: Danylo Halytsky Lviv National Medical University

location_facility: Great Ormond Street Hospital for Children

location_facility: The National Children Specialized Hospital "OHMATDET"

location_facility: Institute of Hematology and Transfusiology

title: Extension Study for Patients Who Completed GENA-05 (NuProtect)- to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII

location_country:
Moldova, Republic of
Poland
India
United Kingdom
United States
Ukraine
Canada
Georgia
France


lead_sponsor: Novo Nordisk A/S

summary: This trial is conducted in Europe and Asia. The aim of the trial is to investigate safety, tolerability, pharmacokinetics (the exposure of the trial drug in the body) and pharmacodynamics (the effect of the investigated drug on the body) of a single dose of long-acting growth hormone (NNC0195-0092, somapacitan) compared to daily dosing of Norditropin® SimpleXx® (somatropin) in children with growth hormone deficiency.

ReferenceCitation: Juul Kildemoes R, Hojby Rasmussen M, Agerso H, Overgaard RV. Optimal Monitoring of Weekly IGF-I Levels During Growth Hormone Therapy With Once-Weekly Somapacitan. J Clin Endocrinol Metab. 2021 Jan 23;106(2):567-576. doi: 10.1210/clinem/dgaa775.

ReferenceCitation: Papathanasiou T, Agerso H, Damholt BB, Hojby Rasmussen M, Kildemoes RJ. Population Pharmacokinetics and Pharmacodynamics of Once-Daily Growth Hormone Norditropin(R) in Children and Adults. Clin Pharmacokinet. 2021 Sep;60(9):1217-1226. doi: 10.1007/s40262-021-01011-3. Epub 2021 Apr 17.

ReferenceCitation: Juul RV, Rasmussen MH, Agerso H, Overgaard RV. Pharmacokinetics and Pharmacodynamics of Once-Weekly Somapacitan in Children and Adults: Supporting Dosing Rationales with a Model-Based Analysis of Three Phase I Trials. Clin Pharmacokinet. 2019 Jan;58(1):63-75. doi: 10.1007/s40262-018-0662-5.

evidence: open (via page says license)

ReferenceCitation: Battelino T, Rasmussen MH, De Schepper J, Zuckerman-Levin N, Gucev Z, Savendahl L; NN8640-4042 Study Group. Somapacitan, a once-weekly reversible albumin-binding GH derivative, in children with GH deficiency: A randomized dose-escalation trial. Clin Endocrinol (Oxf). 2017 Oct;87(4):350-358. doi: 10.1111/cen.13409. Epub 2017 Aug 8.

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

repositories_institution: Ghent University - Ghent University Academic Bibliography

publisher_dissemination: Wiley

title: A Randomised, Open-labelled, Active-controlled, Multinational, Dose-escalation Trial Investigating Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of a Single Dose of Long-acting Growth Hormone (NNC0195-0092, Somapacitan) Compared to Daily Dosing of Norditropin® SimpleXx® in Children With Growth Hormone Deficiency

location_country:
France
Slovenia
North Macedonia
Norway
Sweden
Switzerland
Austria
Belgium
Spain
Israel


title: A Phase 2 Single Arm Study to Investigate the Efficacy of Autologous EBV-specific T-cells for the Treatment of Patients With Aggressive EBV Positive Extranodal NK/T-cell Lymphoma (ENKTCL)

acronym: CITADEL

summary: To investigate the efficacy of autologous EBV-specific T-cells for the treatment of patients with aggressive EBV positive extranodal NK/T-cell lymphoma

lead_sponsor: Cell Medica Ltd

location_country:
United States
United Kingdom
Korea, Republic of
France

location_facility: The Ohio State University Comprehensive Cancer Center


title: Phase Ib Dose Finding Study of Abiraterone Acetate Plus BEZ235 or BKM120 in Patients With Castration-resistant Prostate Cancer.

summary: This is an open label study of abiraterone acetate in combination with BEZ235 and abiraterone acetate in combination with BKM120 in CRPC patients with abiraterone acetate failure.

location_country:
United Kingdom
Belgium
United States
Spain
Canada
France

location_facility: Hackensack University Medical Center Hackensack Univ


summary: This study will evaluate the safety and efficacy of OnabotulinumtoxinA (BOTOX®) for the treatment of urinary incontinence due to NDO in non-catheterizing patients with MS.

ReferenceCitation: Tullman M, Chartier-Kastler E, Kohan A, Keppenne V, Brucker BM, Egerdie B, Mandle M, Nicandro JP, Jenkins B, Denys P. Low-dose onabotulinumtoxinA improves urinary symptoms in noncatheterizing patients with MS. Neurology. 2018 Aug 14;91(7):e657-e665. doi: 10.1212/WNL.0000000000005991. Epub 2018 Jul 20.

lead_sponsor: Allergan

location_country:
Poland
Portugal
Belgium
United States
Czechia
Canada
Russian Federation
France


summary: This non-drug, longitudinal, multi-center, multi-national study will evaluate the suitability of neurocognitive tests and functioning scales for the measurement of cognitive and functioning changes in individuals with Down Syndrome. Tests will be administered at clinic visits in Weeks 1, (4) and 24. The duration of the study for each individual will be between 24 and 27 weeks.

ReferenceCitation: Spiridigliozzi GA, Goeldner C, Edgin J, Hart SJ, Noeldeke J, Squassante L, Visootsak J, Heller JH, Khwaja O, Kishnani PS, Liogier d'Ardhuy X. Adaptive behavior in adolescents and adults with Down syndrome: Results from a 6-month longitudinal study. Am J Med Genet A. 2019 Jan;179(1):85-93. doi: 10.1002/ajmg.a.60685. Epub 2018 Dec 20.

location_country:
Italy
United Kingdom
Argentina
United States
Spain
Canada
France


title: An Open-label, Phase I, Dose Escalation Study Evaluating the Safety, Tolerability, and Pharmacokinetics of GDC-0575 Administered Alone and in Combination With Gemcitabine in Patients With Refractory Solid Tumors or Lymphoma

summary: This open-label, multicenter, Phase I, dose-escalation study will evaluate the safety, tolerability, and pharmacokinetics (PK) of GDC-0575 administered alone or in combination with gemcitabine in participants with refractory solid tumors or lymphoma. In Stage 1, cohorts of participants will receive multiple ascending oral doses of GDC-0575 alone or in combination with intravenous gemcitabine. In Stage 2, participants will receive GDC-0575 orally in combination with intravenous gemcitabine at or below the maximum tolerated dose determined in Stage 1. Anticipated time on study treatment is until disease progression or unacceptable toxicity occurs, up to approximately 5 years.

ReferenceCitation: Italiano A, Infante JR, Shapiro GI, Moore KN, LoRusso PM, Hamilton E, Cousin S, Toulmonde M, Postel-Vinay S, Tolaney S, Blackwood EM, Mahrus S, Peale FV, Lu X, Moein A, Epler J, DuPree K, Tagen M, Murray ER, Schutzman JL, Lauchle JO, Hollebecque A, Soria JC. Phase I study of the checkpoint kinase 1 inhibitor GDC-0575 in combination with gemcitabine in patients with refractory solid tumors. Ann Oncol. 2018 May 1;29(5):1304-1311. doi: 10.1093/annonc/mdy076.

oa_host_type: publisher

lead_sponsor: Genentech, Inc.

location_country:
United States
France

location_facility: Institut Gustave Roussy; Departement Oncologie Medicale

location_facility: University of Oklahoma Health Sciences Center


title: A Multicenter Pharmacogenomic Biomarker Study in Matched Patients With Severe Sepsis Treated With or Without Recombinant Human Activated Protein C [Xigris®, Drotrecogin Alfa (Activated)]

summary: The overall purpose of the study is to determine whether either of the Improved Response Polymorphisms (IRPs) individually predicts a differential DrotAA treatment effect in patients with severe sepsis and high risk of death. This will be an international, multicenter, "prospective-retrospective", nonrandomized, controlled, outcome-blinded, genotype-blinded, matched-patients study. No prospective enrollment or treatment of patients will occur under this protocol. Retrospectively collected clinical data and DNA samples will be analyzed for existing cohorts of patients with severe sepsis who were previously treated with DrotAA (treatment group) or not (control group) as part of their standard care in an ICU.

status: Unknown status

ReferenceCitation: Annane D, Mira JP, Ware LB, Gordon AC, Hinds CJ, Christiani DC, Sevransky J, Barnes K, Buchman TG, Heagerty PJ, Balshaw R, Lesnikova N, de Nobrega K, Wellman HF, Neira M, Mancini ADJ, Walley KR, Russell JA. Pharmacogenomic biomarkers do not predict response to drotrecogin alfa in patients with severe sepsis. Ann Intensive Care. 2018 Jan 31;8(1):16. doi: 10.1186/s13613-018-0353-2.

repository_institution: Harvard University - Digital Access to Scholarship at Harvard (DASH)

repository_institution: Imperial College London - Spiral

repository_institution: Queen Mary University of London - Queen Mary Research Online

lead_sponsor: Sirius Genomics Inc.

location_country:
United States
United Kingdom
Canada
France

location_facility: Université Paris Descartes, Sorbonne Paris Cité, Cochin Hotel-Dieu University Hospital

location_facility: University of British Columbia and Providence Health Care, St. Paul's Hospital

location_facility: Imperial College London, Charing Cross Hospital

location_facility: Johns Hopkins University, Bayview Medical Center

location_facility: University of Versailles, Hospital Raymond Poincaré (AP-HP)

location_facility: Harvard University School of Public Health


title: Ingenio Device Algorithm Study

acronym: IVORY

summary: The purpose of the IVORY Study is to gather data to support global submissions/approvals for some models of the Ingenio device family.

lead_sponsor: Boston Scientific Corporation

location_country:
Germany
Italy
United Kingdom
Hong Kong
Denmark
Sweden
Netherlands
Austria
Belgium
Spain
Malaysia
France

location_facility: Azienda Ospedaliera Mater Domini Policlinico Universitario


title: An Open Label, Multicentre, Single Arm Study to Characterise the Efficacy, Safety and Tolerability of Gefitinib 250 mg (IRESSA™) as First Line Treatment in Caucasian Patients, Who Have Epidermal Growth Factor Receptor (EGFR) Mutation Positive Locally Advanced or Metastatic Non-Small Cell Lung Cancer

acronym: IFUM

summary: This study is carried out to see how Caucasian patients with lung cancer which has EGFR mutation will respond to gefitinib (IRESSA™) as a first line treatment. Safety data will also be collected and analysed to confirm that treatment with gefitinib is safe and well tolerated.

ReferenceCitation: Douillard JY, Ostoros G, Cobo M, Ciuleanu T, McCormack R, Webster A, Milenkova T. First-line gefitinib in Caucasian EGFR mutation-positive NSCLC patients: a phase-IV, open-label, single-arm study. Br J Cancer. 2014 Jan 7;110(1):55-62. doi: 10.1038/bjc.2013.721. Epub 2013 Nov 21.

license: cc-by-nc-sa

license_normalized: cc-by-nc-sa

repositories_concat: PubMed Central;www.repositoriosalud.es

publisher_dissemination: Springer

lead_sponsor: AstraZeneca

location_country:
Italy
Poland
Portugal
Hungary
Norway
Romania
United Kingdom
Switzerland
Spain
Greece
Turkey
Bulgaria
France


lead_sponsor: Grünenthal GmbH

summary:
The main objective of the study is to evaluate the effectiveness, tolerability, and safety of tapentadol hydrochloride prolonged release in subjects suffering from severe chronic low back pain (LBP) who are taking WHO Step III analgesics and show lack of tolerability. This is a clinical effectiveness trial designed to establish a link between anticipated clinical outcomes and the clinical practice by means of selected measures of clinical and subject-reported outcome.

The trial will compare the effectiveness of previous analgesic treatment (WHO Step III) with that of tapentadol hydrochloride prolonged release treatment during defined periods of evaluation.

ReferenceCitation: Galvez R, Schafer M, Hans G, Falke D, Steigerwald I. Tapentadol prolonged release versus strong opioids for severe, chronic low back pain: results of an open-label, phase 3b study. Adv Ther. 2013 Mar;30(3):229-59. doi: 10.1007/s12325-013-0015-6. Epub 2013 Mar 7.

title: An Evaluation of the Effectiveness and Tolerability of Tapentadol Hydrochloride Prolonged Release, and Tapentadol Hydrochloride Immediate Release on Demand, in Subjects With Severe Chronic Nociceptive, Mixed or Neuropathic Low Back Pain Taking WHO Step III Analgesics But Showing a Lack of Tolerability

ipd_sharing_description: Information available on the Grünenthal Web Site.

location_country: Germany Poland Netherlands Switzerland Belgium Spain Czechia France


lead_sponsor: Pfizer

summary: The purpose of this study is to investigate the analgesic efficacy and safety of tanezumab added on to diclofenac SR in patients with osteoarthritis of the knee or hip currently experiencing partial benefit from, and are tolerating, diclofenac 150 mg/day therapy.

ReferenceCitation: Tive L, Bello AE, Radin D, Schnitzer TJ, Nguyen H, Brown MT, West CR. Pooled analysis of tanezumab efficacy and safety with subgroup analyses of phase III clinical trials in patients with osteoarthritis pain of the knee or hip. J Pain Res. 2019 Mar 19;12:975-995. doi: 10.2147/JPR.S191297. eCollection 2019. Erratum In: J Pain Res. 2020 Sep 14;13:2267-2268.

ReferenceCitation: Hochberg MC, Tive LA, Abramson SB, Vignon E, Verburg KM, West CR, Smith MD, Hungerford DS. When Is Osteonecrosis Not Osteonecrosis?: Adjudication of Reported Serious Adverse Joint Events in the Tanezumab Clinical Development Program. Arthritis Rheumatol. 2016 Feb;68(2):382-91. doi: 10.1002/art.39492.

ReferenceCitation: Balanescu AR, Feist E, Wolfram G, Davignon I, Smith MD, Brown MT, West CR. Efficacy and safety of tanezumab added on to diclofenac sustained release in patients with knee or hip osteoarthritis: a double-blind, placebo-controlled, parallel-group, multicentre phase III randomised clinical trial. Ann Rheum Dis. 2014 Sep;73(9):1665-72. doi: 10.1136/annrheumdis-2012-203164. Epub 2013 Jul 12.

location_facility: State Educational Institution of Additional Professional Education

location_facility: Niepubliczny Zaklad Opieki Zdrowotnej "POLIMEDICA"

location_facility: Clinical Emergency County Hospital Constanta

location_facility: County Emergency Clinic Hospital "Sf. Apostol Andrei"

location_facility: St. Petersburg State Healthcare Institution "City Outpatient Clinical #51"

location_facility: Communal Institution Ternopil regional council, "Ternopil Regional Clinical Hospital"

location_facility: Schmerz- und Palliativzentrum Goeppingen

location_facility: Klinische Forschung Hannover - Mitte GmbH

location_facility: Central City Clinical Hospital#1, Department of Therapy,

location_facility: Oleksandrivska Clinical Hospital in Kyiv-Department of Rheumatology # 1

location_facility: Apotheke des Ernst von Bergmann Klinikums

location_facility: State Healthcare Institution of Moscow city "City Clinical Hospital #15 n.a. O. M. Filatov"

location_facility: Chair of Hospital Therapy of Ryazan State Medical University

location_facility: Center polyclinic of Federal State Institution

location_facility: State Institution "Institute of Gerontology AMS of Ukraine"

location_facility: Institute of Urgent and Recovery Surgery named after V.K. Gusaka AMS Ukraine

location_facility: Institution of Russian Academy of Sciences "St. Petersburg Clinical Hospital of RAS"

location_facility: City Clinical Hospital #8, Department of reumatology,

location_facility: Road Clinical Hospital at Dnipropetrovsk station, Department of Rheumatology

location_facility: St. Petersburg State Healthcare Institution "City Pokrovskaya Hospital"

location_facility: 4th City Communal Clinical Hospital, Department of Rheumatology,

location_facility: Ivano-Frankivsk Regional Clinical Hospital

location_facility: Vinnytsya regional clinical hospital named after M.I. Pyrogova

location_facility: Chernivtsi Regional Clinical Hospital,Department of Rheumatology

location_facility: City communal clinical hospital #5, Dept. of Therapy, Danylo Galytskiy Lviv National Med. University

location_facility: Communal institution "City Hospital #7", Department of Therapy,

location_facility: Kyiv Central Basin Clinical Hospital, Department of cardiology,

location_facility: St. Petersburg State Healthcare Institution "City Hospital #25 City Rheumatology Center"

location_facility: "Centrum Medyczne MEDENS S.C. Niepubliczny Zaklad

location_facility: Clinical Emergency Military Hospital "Dr. Carol Davila"

title: A PHASE 3, RANDOMIZED, DOUBLE BLIND, CONTROLLED, MULTI CENTER STUDY OF THE ANALGESIC EFFICACY AND SAFETY OF TANEZUMAB ADDED ON TO DICLOFENAC SR IN PATIENTS WITH OSTEOARTHRITIS OF THE KNEE OR HIP

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:
Germany
Poland
United Kingdom
Romania
Sweden
Austria
Spain
Ukraine
Russian Federation
France


title: A Multicenter, Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid α-Glucosidase (rhGAA) in Patients With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy Studies

summary: This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in patients with infantile-onset Pompe disease who were previously treated with rhGAA derived from the Synpac cell line

lead_sponsor: Genzyme, a Sanofi Company

location_country:
United States
South Africa
France

location_facility: New York University [NYU] School of Medicine

location_facility: Institute for Genetic Medicine, Saint Peter's University Hospital


title: Randomized Trial of Fetoscopic Endoluminal Tracheal Occlusion (FETO) Versus Expectant Management During Pregnancy in Fetuses With Left Sided and Isolated Congenital Diaphragmatic Hernia and Moderate Pulmonary Hypoplasia.

acronym: TOTAL moderate

summary:
Isolated Congenital Diaphragmatic Hernia (CDH) can be diagnosed in the prenatal period, and remains associated with a 30 % chance of perinatal death and morbidity mainly because of pulmonary hypoplasia and pulmonary hypertension. In addition, in the survivors there is a high rate of morbidity with evidence of bronchopulmonary dysplasia in more than 70% of cases. The risk for these can be predicted prenatally by the ultrasonographic measurement of the observed/expected lung area to head circumference ratio (O/E LHR) which is a measure of pulmonary hypoplasia. Also position of the liver is predictive of outcome.

The proposing consortium has developed a prenatal therapeutic approach, which consists of percutaneous fetoscopic endoluminal tracheal occlusion (FETO) with subsequent removal of the balloon. Both procedures are performed percutaneously, there is now experience with more than 150 cases and it has been shown to be safe for the mother. We have witnessed an improvement of survival in fetuses with a predicted chance of survival of less than 30% (referred to as fetuses with severe pulmonary hypoplasia; O/E LHR <25% and liver herniation) to 55% on average. Also there is an apparent reduction in morbidity with the rate of bronchopulmonary dysplasia decreasing from the estimated rate of more than 70% to less than 40% in the same severity group.

Further we have shown that results of FETO are predicted by LHR measurement prior to the procedure, so that better results can be expected in fetuses with larger lung size. Therefore we now aim to offer FETO to fetuses with moderate CDH (=O/E LHR 25-34.9%, irrespective of the liver position as well as O/E LHR 35-44.9% with intrathoracic herniation of the liver). When managed expectantly the estimated rate of postnatal survival is 55%.

This trial will test whether temporary fetoscopic tracheal occlusion rather than expectant management during pregnancy, both followed by standardized postnatal management increases survival or decrease oxygen dependency at 6 months of age. The balloon will be placed between 30 and 31+6 weeks, and will be removed between 34 and 34+6 weeks.

ReferenceCitation: Jani J, Nicolaides KH, Keller RL, Benachi A, Peralta CF, Favre R, Moreno O, Tibboel D, Lipitz S, Eggink A, Vaast P, Allegaert K, Harrison M, Deprest J; Antenatal-CDH-Registry Group. Observed to expected lung area to head circumference ratio in the prediction of survival in fetuses with isolated diaphragmatic hernia. Ultrasound Obstet Gynecol. 2007 Jul;30(1):67-71. doi: 10.1002/uog.4052.

ReferenceCitation: Done E, Gucciardo L, Van Mieghem T, Jani J, Cannie M, Van Schoubroeck D, Devlieger R, Catte LD, Klaritsch P, Mayer S, Beck V, Debeer A, Gratacos E, Nicolaides K, Deprest J. Prenatal diagnosis, prediction of outcome and in utero therapy of isolated congenital diaphragmatic hernia. Prenat Diagn. 2008 Jul;28(7):581-91. doi: 10.1002/pd.2033.

ReferenceCitation: Deprest J, Jani J, Gratacos E, Vandecruys H, Naulaers G, Delgado J, Greenough A, Nicolaides K; FETO Task Group. Fetal intervention for congenital diaphragmatic hernia: the European experience. Semin Perinatol. 2005 Apr;29(2):94-103. doi: 10.1053/j.semperi.2005.04.006.

ReferenceCitation: Deprest J, Gratacos E, Nicolaides KH; FETO Task Group. Fetoscopic tracheal occlusion (FETO) for severe congenital diaphragmatic hernia: evolution of a technique and preliminary results. Ultrasound Obstet Gynecol. 2004 Aug;24(2):121-6. doi: 10.1002/uog.1711. Erratum In: Ultrasound Obstet Gynecol. 2004 Oct;24(5):594.

ReferenceCitation: Jani JC, Nicolaides KH, Gratacos E, Valencia CM, Done E, Martinez JM, Gucciardo L, Cruz R, Deprest JA. Severe diaphragmatic hernia treated by fetal endoscopic tracheal occlusion. Ultrasound Obstet Gynecol. 2009 Sep;34(3):304-10. doi: 10.1002/uog.6450.

ReferenceCitation: Deprest JA, Gratacos E, Nicolaides K, Done E, Van Mieghem T, Gucciardo L, Claus F, Debeer A, Allegaert K, Reiss I, Tibboel D. Changing perspectives on the perinatal management of isolated congenital diaphragmatic hernia in Europe. Clin Perinatol. 2009 Jun;36(2):329-47, ix. doi: 10.1016/j.clp.2009.03.004.

ReferenceCitation: Deprest JA, Hyett JA, Flake AW, Nicolaides K, Gratacos E. Current controversies in prenatal diagnosis 4: Should fetal surgery be done in all cases of severe diaphragmatic hernia? Prenat Diagn. 2009 Jan;29(1):15-9. doi: 10.1002/pd.2108. No abstract available.

ReferenceCitation: Gregoir C, Engels AC, Gomez O, DeKoninck P, Lewi L, Gratacos E, Deprest JA. Fertility, pregnancy and gynecological outcomes after fetoscopic surgery for congenital diaphragmatic hernia. Hum Reprod. 2016 Sep;31(9):2024-30. doi: 10.1093/humrep/dew160. Epub 2016 Jul 4.

ReferenceCitation: Snoek KG, Reiss IK, Greenough A, Capolupo I, Urlesberger B, Wessel L, Storme L, Deprest J, Schaible T, van Heijst A, Tibboel D; CDH EURO Consortium. Standardized Postnatal Management of Infants with Congenital Diaphragmatic Hernia in Europe: The CDH EURO Consortium Consensus - 2015 Update. Neonatology. 2016;110(1):66-74. doi: 10.1159/000444210. Epub 2016 Apr 15.

ReferenceCitation: Nawapun K, Eastwood MP, Diaz-Cobos D, Jimenez J, Aertsen M, Gomez O, Claus F, Gratacos E, Deprest J. In vivo evidence by magnetic resonance volumetry of a gestational age dependent response to tracheal occlusion for congenital diaphragmatic hernia. Prenat Diagn. 2015 Nov;35(11):1048-56. doi: 10.1002/pd.4642. Epub 2015 Sep 29.

ReferenceCitation: Deprest J, Brady P, Nicolaides K, Benachi A, Berg C, Vermeesch J, Gardener G, Gratacos E. Prenatal management of the fetus with isolated congenital diaphragmatic hernia in the era of the TOTAL trial. Semin Fetal Neonatal Med. 2014 Dec;19(6):338-48. doi: 10.1016/j.siny.2014.09.006. Epub 2014 Nov 11.

ReferenceCitation: Done E, Debeer A, Gucciardo L, Van Mieghem T, Lewi P, Devlieger R, De Catte L, Lewi L, Allegaert K, Deprest J. Prediction of neonatal respiratory function and pulmonary hypertension in fetuses with isolated congenital diaphragmatic hernia in the fetal endoscopic tracleal occlusion era: a single-center study. Fetal Diagn Ther. 2015;37(1):24-32. doi: 10.1159/000364805. Epub 2014 Nov 8.

ReferenceCitation: DeKoninck P, Gomez O, Sandaite I, Richter J, Nawapun K, Eerdekens A, Ramirez JC, Claus F, Gratacos E, Deprest J. Right-sided congenital diaphragmatic hernia in a decade of fetal surgery. BJOG. 2015 Jun;122(7):940-6. doi: 10.1111/1471-0528.13065. Epub 2014 Sep 17.

ReferenceCitation: Windrim R, Ryan G, Lebouthillier F, Campisi P, Kelly EN, Baud D, Yoo SJ, Deprest J. Development and use of a high-fidelity simulator for fetal endotracheal balloon occlusion (FETO) insertion and removal. Prenat Diagn. 2014 Feb;34(2):180-4. doi: 10.1002/pd.4284. Epub 2013 Dec 27.

ReferenceCitation: Engels AC, DeKoninck P, van der Merwe JL, Van Mieghem T, Stevens P, Power B, Nicolaides KH, Gratacos E, Deprest JA. Does website-based information add any value in counseling mothers expecting a baby with severe congenital diaphragmatic hernia? Prenat Diagn. 2013 Nov;33(11):1027-32. doi: 10.1002/pd.4190. Epub 2013 Aug 29.

ReferenceCitation: Dekoninck P, Gratacos E, Van Mieghem T, Richter J, Lewi P, Ancel AM, Allegaert K, Nicolaides K, Deprest J. Results of fetal endoscopic tracheal occlusion for congenital diaphragmatic hernia and the set up of the randomized controlled TOTAL trial. Early Hum Dev. 2011 Sep;87(9):619-24. doi: 10.1016/j.earlhumdev.2011.08.001.

ReferenceCitation: Deprest J, Flake A. How should fetal surgery for congenital diaphragmatic hernia be implemented in the post-TOTAL trial era: A discussion. Prenat Diagn. 2022 Mar;42(3):301-309. doi: 10.1002/pd.6091. Epub 2022 Jan 22.

ReferenceCitation: Van Calster B, Benachi A, Nicolaides KH, Gratacos E, Berg C, Persico N, Gardener GJ, Belfort M, Ville Y, Ryan G, Johnson A, Sago H, Kosinski P, Bagolan P, Van Mieghem T, DeKoninck PLJ, Russo FM, Hooper SB, Deprest JA. The randomized Tracheal Occlusion To Accelerate Lung growth (TOTAL)-trials on fetal surgery for congenital diaphragmatic hernia: reanalysis using pooled data. Am J Obstet Gynecol. 2022 Apr;226(4):560.e1-560.e24. doi: 10.1016/j.ajog.2021.11.1351. Epub 2021 Nov 19.

ReferenceCitation: Deprest JA, Benachi A, Gratacos E, Nicolaides KH, Berg C, Persico N, Belfort M, Gardener GJ, Ville Y, Johnson A, Morini F, Wielgos M, Van Calster B, DeKoninck PLJ; TOTAL Trial for Moderate Hypoplasia Investigators. Randomized Trial of Fetal Surgery for Moderate Left Diaphragmatic Hernia. N Engl J Med. 2021 Jul 8;385(2):119-129. doi: 10.1056/NEJMoa2026983. Epub 2021 Jun 8.

repository_institution: Griffith University, Queensland, Australia - Griffith Research Online

repository_institution: University of Antwerp - Institutional Repository University of Antwerp

repositories:
PubMed Central
irua
research-repository.griffith.edu.au

lead_sponsor: University Hospital, Gasthuisberg

collaborators: Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

collaborators: Ospedale Pediatrico Bambino Gesù, Rome (IT)

collaborators: The University of Texas Health Science Center, Houston

location_country:
Germany
Italy
Poland
United Kingdom
Australia
Belgium
United States
Spain
Canada
France

location_facility: 1st Department of Obstetrics and Gynecology, Medical University of Warsaw

location_facility: Baylor College of Medicine/Texas Children's Hospital

lead_sponsor_type: academique


lead_sponsor: Gilead Sciences

summary:
The primary purpose of the study is to evaluate the effectiveness, safety, and tolerability of tenofovir disoproxil fumarate (TDF) in adolescents (aged 12-17 years) with chronic hepatitis B virus (HBV) infection.

The optimal treatment for adolescents with chronic HBV infection is currently unknown. Treatment with interferon alfa, lamivudine, and adefovir dipivoxil in pediatric populations has been shown to be less than optimal. Further, the safety and efficacy of entecavir and telbivudine have not been established in patients < 16 years of age. A study evaluating TDF in adolescents (ages 12-17) was needed to assess the safety and efficacy of this agent in the treatment of chronic hepatitis B in this patient population. In addition, the study will help to further elucidate the pharmacokinetic (PK) and resistance profiles of TDF. Through their participation, study participants will help generate critical new information to help guide the most optimal treatment of chronic HBV infection in adolescents.

This is a randomized, double-blind study to evaluate the antiviral efficacy, safety, and tolerability of TDF versus placebo in adolescents with chronic HBV infection. TDF treatment-naive participants were randomized in a 1:1 ratio to TDF or placebo. After 72 weeks of blinded treatment, participants were to switch to open-label TDF for an additional 2.5 years of treatment, provided that no safety concerns are identified by the Independent Data Monitoring Committee monitoring the study.

ReferenceCitation: Murray KF, Szenborn L, Wysocki J, Rossi S, Corsa AC, Dinh P, McHutchison J, Pang PS, Luminos LM, Pawlowska M, Mizerski J. Randomized, placebo-controlled trial of tenofovir disoproxil fumarate in adolescents with chronic hepatitis B. Hepatology. 2012 Dec;56(6):2018-26. doi: 10.1002/hep.25818. Epub 2012 Aug 27.

location_facility: Samodzielny Publiczny Szpital Kliniczny im. Karola Johschera

location_facility: Specjalistyczny Zespol Opieki Zdrowotnej nad Matka i Dzieckiem

location_facility: Multiprofile Hospital for Active Treatment Sveti Georgi

location_facility: Wojewodzki Szpital Obserwacyjno-Zakazny im. T. Browicza

location_facility: Children's Hospital & Regional Medical Center, d/b/a Seattle Children's Research Institute

location_facility: Hosp Univ y Politecnico La Fe de Valencia

location_facility: Samodzielny Publiczny Dzieciecy Szpital Kliniczny Akademii Medycznej w Bialymstoku

location_facility: Krakowski Szpital Specjalistyczny im. Jana Pawla II

location_facility: Samodzielny Publiczny Szpital Kliniczny Nr 1

location_facility: Children's Hospital & Research Center at Oakland

location_facility: Clinic of Gastroenterology, Specialized Hospital for Active Treatment of Pediatric Diseases, Sofia

location_facility: Ege Universitesi Tip Fakultesi Hastanesi

location_facility: Wojewodzki Specjalistyczny Szpital im Bieganskiego

title: A Randomized, Double-Blind Evaluation of the Antiviral Efficacy, Safety, and Tolerability of Tenofovir Disoproxil Fumarate Versus Placebo in Adolescents With Chronic Hepatitis B Infection

location_country:
Poland
Romania
United States
Spain
Turkey
Bulgaria
France


title: A Multi-center, Open-label, Phase I Study of Single Agent R7112 Administered Orally in Patients With Advanced Malignancies, Except All Forms of Leukaemia

summary: This study will determine the maximum tolerated dose and the optimal associated 4 weekly dosing schedule of RO5045337, administered as monotherapy in patients with advanced solid tumors. A first cohort of patients will receive the starting dose of 20mg/m2/day, once daily for 10 days in each 28 day cycle. Subsequent cohorts of patients will receive dose escalations, and possible changes in dosing schedule, based on tolerability and pharmacokinetic knowledge gained from prior treatment cohorts. The anticipated time on study treatment is until disease progression or intolerable toxicity.

lead_sponsor: Hoffmann-La Roche


title: Phase II Study of Dasatinib (BMS-354825) for Advanced 'Triple-negative' Breast Cancer

summary: This study will determine whether the investigational drug dasatinib is effective in treatment of women with progressive advanced triple-negative breast cancer.

lead_sponsor: Bristol-Myers Squibb

location_country:
United States
Italy
Spain
France

location_facility: University Of Texas Md Anderson Cancer Ctr


title: Phase II Study Of SKI-606 In Subjects With Advanced Or Metastatic Breast Cancer

summary: The purpose of this study is to determine if SKI-606 (Bosutinib) is effective in the treatment of advanced or metastatic breast cancer. Patients must have current Stage IIIB, IIIC or IV breast cancer and have progressed after 1 to 3 prior chemotherapy regimens.

location_country:
Poland
Hong Kong
Australia
Malta
United States
Ukraine
Russian Federation
France


title: Follow-up of Thyroid Cancer Patients From Study THYR-008-00 Who Received Thyroid Remnant Ablation Using Either the Hypothyroid or the Thyrogen Method.

summary:
Patients diagnosed with thyroid cancer are commonly treated with surgery to remove their thyroid gland followed by radioiodine ablation to destroy any remaining parts of the thyroid gland that may have been missed during surgery. It is thought that ablation with radioiodine destroys normal remaining thyroid tissue as well as cancerous cells either in the thyroid area or at other sites.

Following successful treatment, patients are then monitored by their physicians at regular intervals with testing to detect any recurrence of thyroid cancer throughout the body. If thyroid cells are detected by these follow up tests, the physician will decide the best method to re-treat the patient.

In 2001-2003 Genzyme conducted a clinical study to test if Thyrogen® can be used to accomplish radioiodine ablation treatment. This study aimed to determine that the success rates of radioiodine ablation were comparable when patients were prepared for ablation with Thyrogen® while being maintained on their normal thyroid hormone therapy, or, alternatively, by thyroid hormone withdrawal. Thyroid hormone withdrawal commonly causes uncomfortable side effects for patients, and these might be avoided by the use of Thyrogen.

Eight months after the initial Thyrogen plus radioiodine treatment to achieve ablation, all patients in both groups were given Thyrogen® to test for any remaining thyroid tissue. The results of this testing showed that all patients (in both groups) had successfully achieved remnant ablation and had no detectable thyroid tissue remaining.

In order to confirm these remnant ablation results we will conduct follow up testing in this study for all patients that were enrolled in the previous study and we also will determine if their thyroid cancer has recurred. Only patients who completed this previous Thyrogen ablation study are eligible for entry into this study.

ReferenceCitation: Elisei R, Schlumberger M, Driedger A, Reiners C, Kloos RT, Sherman SI, Haugen B, Corone C, Molinaro E, Grasso L, Leboulleux S, Rachinsky I, Luster M, Lassmann M, Busaidy NL, Wahl RL, Pacini F, Cho SY, Magner J, Pinchera A, Ladenson PW. Follow-up of low-risk differentiated thyroid cancer patients who underwent radioiodine ablation of postsurgical thyroid remnants after either recombinant human thyrotropin or thyroid hormone withdrawal. J Clin Endocrinol Metab. 2009 Nov;94(11):4171-9. doi: 10.1210/jc.2009-0869. Epub 2009 Oct 22.

location_country:
Germany
Italy
United States
Canada
France

location_facility: University of Colorado Health Sciences Centre


title: An Open Label Extension, Investigator Initiated Trial to Examine Radiographic Progression , Efficacy and Safety of Long-Term Treatment With Infliximab in Patients With Ankylosing Spondylitis. EASIC (European Ankylosing Spondylitis Infliximab Cohort)

summary: Ankylosing spondylitis (AS) is a chronic inflammatory disease that involves the sacroiliac joints, axial skeleton, entheses and peripheral joints. Current therapy for AS is mainly NSAIDs and physiotherapy which are oft insufficient. Treatment with the TNF-alpha blocking agent infliximab was shown to have definite clinical efficacy in patients with active AS on a short- and a long-term-basis over 2 years. We want to show that treatment with infliximab on a long-term basis over 4 years is safe and efficient and can prevent radiographic progression over a long period of time. Further we want to learn about the outcome after discontinuation of anti-TNF-alpha therapy.

ReferenceCitation: Webers C, Essers I, van Tubergen A, Braun J, Heldmann F, Baraliakos X, Boonen A. Valuing Treatment With Infliximab for Ankylosing Spondylitis Using a Willingness-to-Pay Approach. Arthritis Care Res (Hoboken). 2018 Apr;70(4):608-616. doi: 10.1002/acr.23299. Epub 2018 Feb 18.

lead_sponsor: Rheumazentrum Ruhrgebiet

collaborators: Trial Coordination Center, 9713 GZ Groningen

location_country:
Finland
Germany
United Kingdom
Netherlands
Belgium
France

location_facility: Universitat R. Descartes, Hopital Cochin

location_facility: University Central Hospital, Division of Rheumatology

location_facility: Universitair Ziekenhuis, Afdeling Rheumatologie


title: A Pharmacokinetic Study to Assess Nevirapine Levels in HIV-infected Patients With Impaired Hepatic Function

summary: The objective of this study was to evaluate the steady-state clearance of nevirapine among HIV-1 positive patients with hepatic fibrosis, and to examine whether the degree of hepatic impairment influences clearance.

lead_sponsor: Boehringer Ingelheim

location_country:
United States
Spain
France

location_facility: Boehringer Ingelheim Investigational Site

location_facility: Hospital Clinico y Provincial de Barcelona - HIV


title: Dose Finding and Phase II Study of STI 571 in Advanced Soft Tissue Sarcoma

summary:
RATIONALE: STI571 may interfere with the growth of cancer cells and may be an effective treatment for soft tissue sarcoma.

PURPOSE: Phase I/II trial to study the effectiveness of STI571 in treating patients who have recurrent or refractory soft tissue sarcoma.

ReferenceCitation: van Erp N, Gelderblom H, van Glabbeke M, Van Oosterom A, Verweij J, Guchelaar HJ, Debiec-Rychter M, Peng B, Blay JY, Judson I. Effect of cigarette smoking on imatinib in patients in the soft tissue and bone sarcoma group of the EORTC. Clin Cancer Res. 2008 Dec 15;14(24):8308-13. doi: 10.1158/1078-0432.CCR-08-1303.

ReferenceCitation: Judson I, Ma P, Peng B, Verweij J, Racine A, di Paola ED, van Glabbeke M, Dimitrijevic S, Scurr M, Dumez H, van Oosterom A. Imatinib pharmacokinetics in patients with gastrointestinal stromal tumour: a retrospective population pharmacokinetic study over time. EORTC Soft Tissue and Bone Sarcoma Group. Cancer Chemother Pharmacol. 2005 Apr;55(4):379-386. doi: 10.1007/s00280-004-0876-0. Epub 2004 Dec 9.

ReferenceCitation: Debiec-Rychter M, Dumez H, Judson I, Wasag B, Verweij J, Brown M, Dimitrijevic S, Sciot R, Stul M, Vranck H, Scurr M, Hagemeijer A, van Glabbeke M, van Oosterom AT; EORTC Soft Tissue and Bone Sarcoma Group. Use of c-KIT/PDGFRA mutational analysis to predict the clinical response to imatinib in patients with advanced gastrointestinal stromal tumours entered on phase I and II studies of the EORTC Soft Tissue and Bone Sarcoma Group. Eur J Cancer. 2004 Mar;40(5):689-95. doi: 10.1016/j.ejca.2003.11.025.

ReferenceCitation: Verweij J, van Oosterom A, Blay JY, Judson I, Rodenhuis S, van der Graaf W, Radford J, Le Cesne A, Hogendoorn PC, di Paola ED, Brown M, Nielsen OS. Imatinib mesylate (STI-571 Glivec, Gleevec) is an active agent for gastrointestinal stromal tumours, but does not yield responses in other soft-tissue sarcomas that are unselected for a molecular target. Results from an EORTC Soft Tissue and Bone Sarcoma Group phase II study. Eur J Cancer. 2003 Sep;39(14):2006-11.

ReferenceCitation: van Oosterom AT, Judson IR, Verweij J, Stroobants S, Dumez H, Donato di Paola E, Sciot R, Van Glabbeke M, Dimitrijevic S, Nielsen OS; European Organisation for Research and Treatment of Cancer Soft Tissue and Bone Sarcoma Group. Update of phase I study of imatinib (STI571) in advanced soft tissue sarcomas and gastrointestinal stromal tumors: a report of the EORTC Soft Tissue and Bone Sarcoma Group. Eur J Cancer. 2002 Sep;38 Suppl 5:S83-7. doi: 10.1016/s0959-8049(02)80608-6.

ReferenceCitation: van Oosterom AT, Judson I, Verweij J, Stroobants S, Donato di Paola E, Dimitrijevic S, Martens M, Webb A, Sciot R, Van Glabbeke M, Silberman S, Nielsen OS; European Organisation for Research and Treatment of Cancer Soft Tissue and Bone Sarcoma Group. Safety and efficacy of imatinib (STI571) in metastatic gastrointestinal stromal tumours: a phase I study. Lancet. 2001 Oct 27;358(9291):1421-3. doi: 10.1016/s0140-6736(01)06535-7.

lead_sponsor: European Organisation for Research and Treatment of Cancer - EORTC

location_country:
United Kingdom
Denmark
Netherlands
Belgium
France

location_facility: Herlev Hospital - University Hospital of Copenhagen


title: Prognostic Value of the 5-SENSE Score to Predict Focality of the Seizure-onset Zone as Assessed by Stereo-electroencephalography - a Prospective Multicenter Validation Study

summary: The purpose of this study is to assess how well a new scoring system called the 5-SENSE score can predict where seizures start in the brain using Stereoelectroencephalography (SEEG). The 5-SENSE Score is a 5-point score based on routine presurgical work-up, designed to assist in predicting whether SEEG can identify a focal seizure onset zone, thereby sparing patients the risk of undergoing this invasive diagnostic procedure.

study_start_date_type: Anticipated

status: Not yet recruiting

lead_sponsor: Duke University

sponsor_collaborators:
Duke University
Christian Doppler University Hospital

location_country:
Germany
United Kingdom
Denmark
Australia
Japan
Romania
Austria
Switzerland
United States
Spain
Czechia
Canada
France

location_facility: University of Florida College of Medicine

location_facility: Christian Doppler University Hospital Paracelsus Medical University Salzburg and Centre for Cognitive Neuroscience Salzburg

location_facility: Grenoble Institute of Neurosciences, Centre Hospitalier Universitaire Grenoble Alpes

location_facility: Epilepsy Department University Hospitals of Marseille

location_facility: University College London Hospitals, NHS Foundation Trust

location_facility: Department of Clinical Neurosciences chez CHUV; Centre Hospitalier Universitaire Vaudois

location_facility: Hospital Universitario y Politecnico La Fe

location_facility: Danish Epilepsy Centre, Aarhus Universitets Hospital

location_facility: Departmet of Neurology, Masaryk University Brno

location_facility: "Carol Davila" University of Medicine and Pharmacy

location_facility: Epilepsy Centre, University Hospital Freiburg

location_facility: Epilepsy Department; Universitätsklinikum Erlangen


title: A Phase III, Randomized, Open-Label, Multi-Center, Global Study of Volrustomig (MEDI5752) as Sequential Therapy Versus Observation in Participants With Unresected Locally Advanced Head and Neck Squamous Cell Carcinoma, Who Have Not Progressed Following Definitive Concurrent Chemoradiotherapy (eVOLVE-HNSCC)

acronym: eVOLVE-HNSCC

summary: The main purpose of this study is to assess the efficacy and safety of volrustomig compared to observation in participants with unresected locally advanced head and neck squamous cell carcinoma (LA-HNSCC) who have not progressed after receiving definitive concurrent chemoradiotherapy (cCRT).

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

location_country:
Hungary
United Kingdom
Japan
Brazil
Belgium
United States
Korea, Republic of
Spain
Thailand
Turkey
Taiwan
Canada
France


title: A Continuation Study of Latozinemab in Participants With Neurodegenerative Disease

summary: Continuation study to provide continued access to latozinemab for participants who have previously participated in a latozinemab study

status: Enrolling by invitation

lead_sponsor: Alector Inc.

location_country:
Germany
Italy
Portugal
United Kingdom
Sweden
Netherlands
Belgium
United States
Spain
Canada
France

location_facility: Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico -Via Fracesco Sforza 35

location_facility: John Hopkins University School of Medicine

location_facility: University of Cincinnati Gardner Neuroscience Institute

location_facility: Universitätsklinikum Ulm - Leimgrubenweg 12-14

location_facility: Azienda Ospedaliero Universitaria Di Modena Policlinico

location_facility: Centro Hospitalar E Universitario de Coimbra EPE

location_facility: Centro Hospitalar Universitário Lisboa Norte, EPE - Hospital de Santa Maria

location_facility: Irving Institute for Clinical and Translational Research

location_facility: IRCCS - Centro S. Giovanni di Dio Fatebenefratelli

location_facility: Fondazione IRCCS Di Rilievo Nazionale Istituto Nazionale Neurologico Carlo Besta-VIA MANGIAGALLI 3


title: A Phase 3, Randomized, Open-label, Parallel-arm, Active-controlled, Multicenter Study to Evaluate Safety and Efficacy of ALXN1850 Versus Asfotase Alfa Administered Subcutaneously in Pediatric Participants (2 to < 12 Years of Age) With Hypophosphatasia (HPP) Previously Treated With Asfotase Alfa

acronym: CHESTNUT

summary: The primary purpose of this study is to assess the safety and tolerability of ALXN1850 versus asfotase alfa in pediatric participants with HPP previously treated with asfotase alfa.

ipd_sharing_description: Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

lead_sponsor: Alexion Pharmaceuticals, Inc.

location_country:
Germany
Italy
United Kingdom
Argentina
Australia
Japan
United States
Turkey
Canada
France


title: A Phase 3, Randomized, Double-blinded, Placebo-controlled, Multicenter Study to Evaluate Efficacy and Safety of ALXN1850 Versus Placebo Administered Subcutaneously in Pediatric (2 to < 12 Years of Age) Participants With Hypophosphatasia Who Have Not Received Previous Treatment With Asfotase Alfa

acronym: MULBERRY

summary: The primary purpose of this study is to evaluate the efficacy of ALXN1850 versus placebo on radiographic outcomes in pediatric participants with HPP who have not previously been treated with asfotase alfa.

location_country:
Germany
Poland
Argentina
Australia
Brazil
Thailand
Vietnam
Turkey
Canada
France
United States
Belgium
China
Italy
United Kingdom
Mexico
Korea, Republic of
Spain
Taiwan
Israel