publications_result: Oluwole OO, Shah BD, Baer MR, Bishop MR, Holmes H, Schiller GJ, et al. Outcomes of Patients with Relapsed/Refractory Acute Lymphoblastic Leukemia Treated with Prior Blinatumomab in ZUMA-3, a Study of KTE-C19, an Anti-CD19 Chimeric Antigen Receptor (CAR) T Cell Therapy [Abstract S1569]. The 23rd European Hematology Association (EHA) Congress 2018 14-17 June; Stockholm, Sweden.

ReferenceCitation: Sabatino M, Choi K, Chiruvolu V, Better M. Production of Anti-CD19 CAR T Cells for ZUMA-3 and -4: Phase 1/2 Multicenter Studies Evaluating KTE-C19 in Patients With Relapsed/Refractory B-Precursor Acute Lymphoblastic Leukemia (R/R ALL) [Abstract 711]. Blood 2016;128 (22):1227.

ReferenceCitation: Shah B, Castro J, Gokbuget N, Kersten MJ, Hagenbeek T, Wierda W, et al. ZUMA-3: A Phase 1/2 Multi-center Study Evaluating the Safety and Efficacy of KTE-C19 Anti-CD19 CAR T Cells in Adult Subjects with Relapsed/Refractory B Precursor Acute Lymphoblastic Leukemia (r/r ALL). European Society for Medical Oncology (ESMO) Congress 2016;Abstract 3713.

ReferenceCitation: Shah B, Huynh V, Sender LS, Lee DW, Castro JE, Wierda WG, et al. High Rates of Minimal Residual Disease-Negative (MRD-) Complete Responses (CR) in Adult and Pediatric and Patients With Relapsed/Refractory Acute Lymphoblastic Leukemia (R/R ALL) Treated With KTE-C19 (Anti-CD19 Chimeric Antigen Receptor [CAR] T Cells): Preliminary Results of the ZUMA-3 and ZUMA-4 Trials. Blood 2016;128 (22):2803.

ReferenceCitation: Shah B, Stock W, Wierda W, Topp M, Kersten MJ, Houot R, et al. KTE-C19, an Anti-CD19 Chimeric Antigen Receptor (CAR) T cell Therapy in Adult Patients (Pts) With Relapsed/ Refractory Acute Lymphoblastic Leukemia (R/R ALL) in the ZUMA-3 Trial: Preliminary Results of Novel Safety Interventions [Abstract ALL-025]. Clinical lymphoma, myeloma & leukemia 2017;17:S253.

ReferenceCitation: Shah B, Stock W, Wierda W, Topp MS, Kersten MJ, Houot R, et al. Preliminary Results of Novel Safety Interventions in Adult Patients (Pts) With Relapsed/Refractory Acute Lymphoblastic Leukemia (R/R ALL) in the ZUMA-3 Trial. European Society for Medical Oncology (ESMO) Congress 2017.

ReferenceCitation: Shah B, Wierda WG, Schiller GJ, Bishop MR, Castro JE, Sabatino M, et al. KTE-C19 Chimeric Antigen Receptor (CAR) T Cell Therapy in Adults with High-Burden Relapsed/Refractory Acute Lymphoblastic Leukemia (R/R ALL): Updated Results from Phase 1/2 of ZUMA-3 [Abstract P523]. The 22nd European Hematology Association (EHA) Congress 2017 22-25 June; Madrid, Spain.

ReferenceCitation: Shah BD, Bishop MR, Oluwole OO, Logan A, Baer MR, Donnellan WB, et al. End of Phase I Results of ZUMA-3, A Phase 1/2 Study of KTE-X19, Anti-CD19 Chimeric Antigen Receptor (CAR) T Cell Therapy, in Adult Patients (pts) with Relapsed/Refractory (R/R) Acute Lymphoblastic Leukemia (ALL) [Abstract]. J Clin Oncol 2019;37 (15):7006.

ReferenceCitation: Shah BD, Bishop MR, Oluwole OO, Logan AC, Baer MR, Donnellan WB, et al. KTE-X19, an Anti-CD19 Chimeric Antigen Receptor T Cell Therapy, in Adult Patients with Relapsed/Refractory Acute Lymphoblastic Leukemia: End of Phase 1 Results of ZUMA-3 [Abstract PS945]. HemaSphere 2019;3 (S1):426.

ReferenceCitation: Shah BD, Oluwole OO, Baer MR, Bishop MR, Holmes H, Schiller GJ, et al. KTE-C19, an Anti-CD19 Chimeric Antigen Receptor (CAR) T Cell Therapy, in Adult Patients with Relapsed/ Refractory Acute Lymphoblastic Leukemia (R/R ALL): Outcomes in Patients Who Were Treated with Prior Blinatumomab in ZUMA-3 [Abstract ALL-128]. Clinical lymphoma, myeloma & leukemia 2018;18 (Supplement 1):S184.

ReferenceCitation: Shah BD, Oluwole OO, Baer MR, Bishop MR, Holmes H, Schiller GJ, et al. Outcomes of Patients Treated With Prior Blinatumomab in ZUMA-3, a Study of KTE-C19, an Anti-CD19 Chimeric Antigen Receptor T Cell Therapy, in Adult Patients With Relapsed/Refractory Acute Lymphoblastic Leukemia. ASCO; 2018 01-05 June; Chicago, IL.

ReferenceCitation: Shah BD, Stock W, Wierda WG, Oluwole O, Holmes H, Schiller GJ, et al. Phase 1 Results of ZUMA-3: KTE-C19, an Anti-CD19 Chimeric Antigen Receptor (CAR) T Cell Therapy, in Adult Patients with Relapsed/Refractory Acute Lymphoblastic Leukemia (R/R ALL) [Abstract 612]. Blood 2017;130 (Supplement 1):888.

ReferenceCitation: Shah BD, Wierda WG, Schiller GJ, Bishop MR, Castro JE, Sabatino M, et al. Updated results from ZUMA- 3, a phase 1/2 study of KTE-C19 chimeric antigen receptor (CAR) T cell therapy, in adults with high-burden relapsed/refractory acute lymphoblastic leukemia (R/R ALL) [Abstract 3024]. American Society of Clinical Oncology (ASCO) Annual Meeting; 2017 02-06 June; Chicago, Illinois.

ReferenceCitation: Wierda WG, Bishop MR, Oluwole O, Logan AC, Baer MR, Donnellan WB, et al. Updated Phase 1 Results of Zuma-3: Kte-X19, an Anti-CD19 Chimeric Antigen Receptor T Cell Therapy, in Adult Patients with Relapsed/Refractory Acute Lymphoblastic Leukemia [Abstract 256]. Biol Blood Marrow Transplant 2019;25 (3):S185.

ReferenceCitation: Wierda WG, Bishop MR, Oluwole OO, Logan AC, Baer MR, Donnellan WB, et al. Updated Phase 1 Results of Zuma-3: Kte-C19, an Anti-CD19 Chimeric Antigen Receptor T Cell Therapy, in Adult Patients with Relapsed/Refractory Acute Lymphoblastic Leukemia [Abstract]. Blood 2018;132 (Supplement 1):897.

ReferenceCitation: Shah BD, Bishop MR, Oluwole OO, Logan AC, Baer MR, Donnellan WB, O'Dwyer KM, Holmes H, Arellano ML, Ghobadi A, Pagel JM, Lin Y, Cassaday RD, Park JH, Abedi M, Castro JE, DeAngelo DJ, Malone AK, Mawad R, Schiller GJ, Rossi JM, Bot A, Shen T, Goyal L, Jain RK, Vezan R, Wierda WG. KTE-X19 anti-CD19 CAR T-cell therapy in adult relapsed/refractory acute lymphoblastic leukemia: ZUMA-3 phase 1 results. Blood. 2021 Jul 8;138(1):11-22. doi: 10.1182/blood.2020009098.

name: Division of Hematology/Oncology, Department of Malignant Hematology, Moffitt Cancer Center, Tampa, FL;

family: Shah

full_name: Bijal D. Shah

given: Bijal D.

name: Division of Hematology/Oncology, The University of Chicago Medicine, Chicago, IL;

family: Bishop

full_name: Michael R. Bishop

given: Michael R.

name: Division of Hematology/Oncology, Vanderbilt-Ingram Cancer Center, Vanderbilt University School of Medicine, Nashville, TN;

family: Oluwole

full_name: Olalekan O. Oluwole

given: Olalekan O.

name: Division of Hematology/Oncology, Helen Diller Comprehensive Cancer Center, University of California San Francisco, San Francisco, CA;

family: Logan

full_name: Aaron C. Logan

given: Aaron C.

name: Marlene and Stewart Greenebaum Cancer Center, University of Maryland School of Medicine, Baltimore, MD;

family: Baer

full_name: Maria R. Baer

given: Maria R.

name: Sarah Cannon Research Institute, Nashville, TN;

family: Donnellan

full_name: William B. Donnellan

given: William B.

name: Acute Leukemia Program, Wilmot Cancer Institute, University of Rochester Medical Center, Rochester, NY;

family: O’Dwyer

full_name: Kristen M. O’Dwyer

given: Kristen M.

name: Texas Oncology-Baylor Charles A. Sammons Cancer Center, Dallas, TX;

family: Holmes

full_name: Houston Holmes

given: Houston

name: Division of Hematology/Oncology, Winship Cancer Institute, Emory University, Atlanta, GA;

family: Arellano

full_name: Martha L. Arellano

given: Martha L.

name: Section of Stem Cell Transplant and Leukemia, Division of Medical Oncology, Washington University School of Medicine–Siteman Cancer Center, St Louis, MO;

family: Ghobadi

full_name: Armin Ghobadi

given: Armin

name: Center for Blood Disorders and Stem Cell Transplantation, Swedish Cancer Institute, Seattle, WA;

family: Pagel

full_name: John M. Pagel

given: John M.

name: Division of Hematology, Department of Internal Medicine, Mayo Clinic, Rochester, MN;

family: Lin

full_name: Yi Lin

given: Yi

name: Division of Hematology and Clinical Research, University of Washington School of Medicine–Fred Hutchinson Cancer Research Center, Seattle, WA;

family: Cassaday

full_name: Ryan D. Cassaday

given: Ryan D.

name: Division of Hematologic Malignancies, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY;

family: Park

full_name: Jae H. Park

given: Jae H.

name: Department of Internal Medicine, University of California Davis Medical Center, Sacramento, CA;

family: Abedi

full_name: Mehrdad Abedi

given: Mehrdad

name: Division of Hematology-Oncology, Mayo Clinic, Phoenix, AZ;

family: Castro

full_name: Januario E. Castro

given: Januario E.

name: Division of Hematologic Oncology, Dana-Farber Cancer Institute, Boston, MA;

family: DeAngelo

full_name: Daniel J. DeAngelo

given: Daniel J.

name: Division of Hematology and Medical Oncology, Icahn School of Medicine at Mount Sinai, New York, NY;

family: Malone

full_name: Adriana K. Malone

given: Adriana K.

name: Center for Blood Disorders and Stem Cell Transplantation, Swedish Medical Center, Seattle, WA;

family: Mawad

full_name: Raya Mawad

given: Raya

name: Division of Hematology/Oncology, Department of Medicine, David Geffen School of Medicine, University of California at Los Angeles, Los Angeles, CA;

family: Schiller

full_name: Gary J. Schiller

given: Gary J.

name: Kite, a Gilead Company, Santa Monica, CA; and

family: Rossi

full_name: John M. Rossi

name: Division of Cancer Medicine, Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX

ReferenceCitation: Shah BD, Ghobadi A, Oluwole OO, Logan AC, Boissel N, Cassaday RD, Leguay T, Bishop MR, Topp MS, Tzachanis D, O'Dwyer KM, Arellano ML, Lin Y, Baer MR, Schiller GJ, Park JH, Subklewe M, Abedi M, Minnema MC, Wierda WG, DeAngelo DJ, Stiff P, Jeyakumar D, Feng C, Dong J, Shen T, Milletti F, Rossi JM, Vezan R, Masouleh BK, Houot R. KTE-X19 for relapsed or refractory adult B-cell acute lymphoblastic leukaemia: phase 2 results of the single-arm, open-label, multicentre ZUMA-3 study. Lancet. 2021 Aug 7;398(10299):491-502. doi: 10.1016/S0140-6736(21)01222-8. Epub 2021 Jun 4.

location_country:
United States
Netherlands
France
Germany
Canada

location_facility: University of MD Greenbaum Comprehensive Cancer Center

location_facility: University Health Network - Princess Margaret

location_facility: University of California Irvine Medical Center

location_facility: Hopital Pontchaillou - CHU de Rennes - Service d'Hematologie

location_facility: University of California Los Angeles (UCLA)

location_facility: University of California Davis Comprehensive Cancer Center

title: A Phase 1/2 Multi-Center Study Evaluating the Safety and Efficacy of KTE-X19 in Adult Subjects With Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia (r/r ALL) (ZUMA-3)

summary: The primary objectives of this study are to determine the safety and efficacy of brexucabtagene autoleucel (KTE-X19) in adult participants with relapsed/refractory (r/r) B-precursor acute lymphoblastic leukemia (ALL).


design_allocation: Randomized

intervention_type: Biological

status: Completed

study_start_date_type: Actual

study_type: Interventional

lead_sponsor: Bioeq GmbH

enrollment_type: Actual

primary_purpose: Treatment

location_country:
Italy
Austria
France
Czechia
Poland
Israel
Hungary
Russian Federation
Spain
Germany
United Kingdom
Ukraine

acronym: COLUMBUS-AMD

location_facility: University of Bonn, Department of Ophthalmology

ipd_sharing: No

study_completion_date_type: Actual

source: euctr

title: Efficacy and Safety of the Biosimilar Ranibizumab FYB201 in Comparison to Lucentis in Patients With Neovascular Age-related Macular Degeneration

summary: The purpose of this study is to determine the efficacy and safety of the biosimilar ranibizumab FYB201 in comparison to Lucentis in patients with neovascular age-related macular degeneration.

lead_sponsor_type: industriel

status_simplified: Completed


type: EudraCT Number

title: A Phase 3 Efficacy and Safety Study of Ataluren (PTC124®) in Patients With Nonsense Mutation Cystic Fibrosis

summary: This is a Phase 3, international, multicenter, randomized, double-blind, placebo-controlled, efficacy and safety study of ataluren in patients with nonsense mutation cystic fibrosis (nmCF) not receiving chronic inhaled aminoglycosides.

ReferenceType: derived

ReferenceCitation: VanDevanter DR, Hamblett NM, Simon N, McIntosh J, Konstan MW. Evaluating assumptions of definition-based pulmonary exacerbation endpoints in cystic fibrosis clinical trials. J Cyst Fibros. 2021 Jan;20(1):39-45. doi: 10.1016/j.jcf.2020.07.008. Epub 2020 Jul 15.

family: VanDevanter

full_name: Donald R. VanDevanter

given: Donald R.

sequence: first

family: Hamblett

full_name: Nicole Mayer Hamblett

given: Nicole Mayer

sequence: additional

family: Simon

full_name: Noah Simon

given: Noah

family: McIntosh

full_name: Joseph McIntosh

given: Joseph

family: Konstan

full_name: Michael W. Konstan

given: Michael W.

bso_classification: Medical research

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: Journal of Cystic Fibrosis

oa_colors: green hybrid

oa_host_type: publisher;repository

evidence: open (via free pdf)

host_type: publisher

license_normalized: no license

version: publishedVersion

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

host_type: repository

license: implied-oa

license_normalized: implied-oa

repository_institution: PubMed Central - Europe PMC

repository_normalized: PubMed Central

evidence: oa repository (via pmcid lookup)

publisher: Elsevier BV

publisher_dissemination: Elsevier

publisher_group: Elsevier

publisher_normalized: Elsevier

ReferenceCitation: Konstan MW, VanDevanter DR, Rowe SM, Wilschanski M, Kerem E, Sermet-Gaudelus I, DiMango E, Melotti P, McIntosh J, De Boeck K; ACT CF Study Group. Efficacy and safety of ataluren in patients with nonsense-mutation cystic fibrosis not receiving chronic inhaled aminoglycosides: The international, randomized, double-blind, placebo-controlled Ataluren Confirmatory Trial in Cystic Fibrosis (ACT CF). J Cyst Fibros. 2020 Jul;19(4):595-601. doi: 10.1016/j.jcf.2020.01.007. Epub 2020 Jan 23.

full_name: M.W. Konstan

full_name: D.R. VanDevanter

family: Rowe

full_name: S.M. Rowe

family: Wilschanski

full_name: M. Wilschanski

given: M.

family: Kerem

full_name: E. Kerem

given: E.

family: Sermet-Gaudelus

full_name: I. Sermet-Gaudelus

given: I.

family: DiMango

full_name: E. DiMango

family: Melotti

full_name: P. Melotti

given: P.

full_name: J. McIntosh

given: J.

family: De Boeck

full_name: K. De Boeck

given: K.

location_country:
Italy
Brazil
Netherlands
Germany
France
Bulgaria
Greece
Israel
Poland
Belgium
Argentina
Australia
Spain
United States
United Kingdom
Canada

location_facility: University of Texas Health Science Center

location_facility: Birmingham Children's Hospital NHS Foundation Trust

location_facility: Hospital Regional Universitario de Malaga

location_facility: New York University Langone Medical Center

location_facility: Children's Hospital and Research Center at Oakland

location_facility: NZOZ Podkarpacki Osrodek Pulmonologii i Alergologii

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

location_facility: University Multiprofile Hospital for Active Treatment Aleksandrovska EAD

location_facility: General Hospital of Thessaloniki Ippokration

location_facility: Hospital de Niños Superiora Sor Maria Ludovica

location_facility: University Mulitiprofile Hospital for Active Treatment Sveti Georgi EAD

location_facility: Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul

location_facility: Instituto da Criança - Hospital das Clínicas

location_facility: Hôpital Universitaire des Enfants Reine Fabiola

location_facility: Szpital Dzieciecy Polanki im Macieja Plazynskiego w Gdansku

location_facility: Penn State Milton S. Hershey Medical Center

location_facility: University of Toronto Hospital for Sick Children

location_facility: Hospital de Sabadell, Consorci Sanitari Parc Tauli

location_facility: Southampton University Hospitals NHS Trust


title: A Phase 3, Randomized, Double-Blind, Switch Study to Evaluate F/TAF in HIV-1 Positive Subjects Who Are Virologically Suppressed on Regimens Containing FTC/TDF

summary:
This study will evaluate the efficacy of switching from emtricitabine/tenofovir disoproxil fumarate (FTC/TDF) fixed dose combination (FDC) to emtricitabine/tenofovir alafenamide (F/TAF) FDC in HIV-1 positive participants who are virologically suppressed on regimens containing FTC/TDF.

This study will consist of a 96 week double-blind treatment period. After Week 96, all participants will continue on blinded study drug treatment and attend visits every 12 weeks until treatment assignments are unblinded. All participants will return for an unblinding visit and will be given the option to receive open-label F/TAF and attend visits every 12 weeks until F/TAF is commercially available, or the sponsor terminates the F/TAF clinical development program.

ReferenceCitation: Gallant JE, Daar ES, Raffi F, Brinson C, Ruane P, DeJesus E, Johnson M, Clumeck N, Osiyemi O, Ward D, Morales-Ramirez J, Yan M, Abram ME, Plummer A, Cheng AK, Rhee MS. Efficacy and safety of tenofovir alafenamide versus tenofovir disoproxil fumarate given as fixed-dose combinations containing emtricitabine as backbones for treatment of HIV-1 infection in virologically suppressed adults: a randomised, double-blind, active-controlled phase 3 trial. Lancet HIV. 2016 Apr;3(4):e158-65. doi: 10.1016/S2352-3018(16)00024-2. Epub 2016 Mar 14.

family: Gallant

full_name: Joel E Gallant

given: Joel E

family: Daar

full_name: Eric S Daar

given: Eric S

family: Raffi

full_name: François Raffi

given: François

family: Brinson

full_name: Cynthia Brinson

given: Cynthia

family: Ruane

full_name: Peter Ruane

given: Peter

family: DeJesus

full_name: Edwin DeJesus

given: Edwin

family: Johnson

full_name: Margaret Johnson

given: Margaret

family: Clumeck

full_name: Nathan Clumeck

given: Nathan

family: Osiyemi

full_name: Olayemi Osiyemi

given: Olayemi

family: Ward

full_name: Doug Ward

given: Doug

family: Morales-Ramirez

full_name: Javier Morales-Ramirez

given: Javier

family: Yan

full_name: Mingjin Yan

given: Mingjin

family: Abram

full_name: Michael E Abram

given: Michael E

family: Plummer

full_name: Andrew Plummer

given: Andrew

family: Cheng

full_name: Andrew K Cheng

given: Andrew K

family: Rhee

full_name: Martin S Rhee

given: Martin S

journal_name: The Lancet HIV

ipd_sharing: Yes

ipd_sharing_description: Qualified external researchers may request IPD for this study after study completion. For more information, please visit our website at https://www.gilead.com/science-and-medicine/research/clinical-trials-transparency-and-data-sharing-policy

lead_sponsor: Gilead Sciences

location_country:
Italy
United States
France
Belgium
Puerto Rico
Canada
United Kingdom

location_facility: Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center

location_facility: Kaiser Permanente Sacramento Medical Center

location_facility: Maple Leaf Medical Clinic/Maple Leaf Research

location_facility: AIDS Research & Treatment Center of the Treasure Coast

location_facility: Vancouver Infectious Disease Research and Care Centre

location_facility: Kaiser Permanente Medical Group San Francisco

location_facility: AIDS Arms, Inc./Trinity Health & Wellness Center

location_facility: Tarrant County Infectious Disease Associates

location_facility: North Texas Infectious Diseases Consulants

location_facility: University Health Network/Toronto General Hospital

location_facility: Brighton and Sussex University Hospitals

location_facility: IRCCS Ospedale San Raffaele, Centro San Luigi

location_facility: Prime Healthcare Services - St Michael's LLC d/b/a Saint Michael's Medical Center

location_facility: La Playa Medical Group and Clinical Research

location_facility: University Hospital of Montpellier (CHU-Gui de Chauliac)

location_facility: Highland Hospital - Alameda Health System (formerly Alameda County medical Center)

location_facility: Community Research Initiative of New England

location_facility: Southwest Infectious Disease Clinical Research, Inc

location_facility: University of Puerto Rico School of Medicine

intervention_type: Drug


ReferenceCitation: Hurwitz H, Van Cutsem E, Bendell J, Hidalgo M, Li CP, Salvo MG, Macarulla T, Sahai V, Sama A, Greeno E, Yu KH, Verslype C, Dawkins F, Walker C, Clark J, O'Reilly EM. Ruxolitinib + capecitabine in advanced/metastatic pancreatic cancer after disease progression/intolerance to first-line therapy: JANUS 1 and 2 randomized phase III studies. Invest New Drugs. 2018 Aug;36(4):683-695. doi: 10.1007/s10637-018-0580-2. Epub 2018 Mar 6.

family: Hurwitz

full_name: Herbert Hurwitz

given: Herbert

family: Van Cutsem

full_name: Eric Van Cutsem

given: Eric

family: Bendell

full_name: Johanna Bendell

given: Johanna

family: Hidalgo

full_name: Manuel Hidalgo

given: Manuel

family: Li

full_name: Chung-Pin Li

given: Chung-Pin

family: Salvo

full_name: Marcelo Garrido Salvo

given: Marcelo Garrido

family: Macarulla

full_name: Teresa Macarulla

given: Teresa

family: Sahai

full_name: Vaibhav Sahai

given: Vaibhav

family: Sama

full_name: Ashwin Sama

given: Ashwin

family: Greeno

full_name: Edward Greeno

given: Edward

family: Yu

full_name: Kenneth H. Yu

given: Kenneth H.

family: Verslype

full_name: Chris Verslype

given: Chris

family: Dawkins

full_name: Fitzroy Dawkins

given: Fitzroy

family: Walker

full_name: Chris Walker

family: Clark

full_name: Jason Clark

given: Jason

family: O’Reilly

full_name: Eileen M. O’Reilly

given: Eileen M.

journal_name: Investigational New Drugs

oa_host_type: repository

version: acceptedVersion

publisher: Springer Science and Business Media LLC

publisher_dissemination: Springer-Nature

publisher_group: Springer-Nature

publisher_normalized: Springer

status: Terminated

lead_sponsor: Incyte Corporation

location_country:
Austria
Chile
Sweden
Switzerland
Mexico
Netherlands
France
Israel
Ireland
Puerto Rico
Portugal
Denmark
United States
Colombia

acronym: The JANUS 2 Study

title: A Randomized, Double-Blind, Phase 3 Study of the JAK 1/2 Inhibitor Ruxolitinib or Placebo in Combination With Capecitabine in Subjects With Advanced or Metastatic Adenocarcinoma of the Pancreas Who Have Failed or Are Intolerant to First-Line Chemotherapy (The JANUS 2 Study)

summary: This was to determine the efficacy, based upon overall survival, of ruxolitinib added to capecitabine for the treatment of metastatic pancreatic cancer.

status_simplified: Unknown


title: A Phase 3, Double-Blind, Placebo Controlled Multicenter Study to Determine the Efficacy and Safety of Luspatercept (ACE-536) in Adults With Transfusion Dependent Beta (B)-Thalassemia

acronym: BELIEVE

summary:
This is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) plus Best supportive care (BSC) versus placebo plus BSC in adults who require regular red blood cell transfusion due to (β)-thalassemia.

The study is divided into the following periods:

Historical Period,
Screening/Run-in Period,
Double-blind Treatment Period (48 weeks),
Double-blind Long-term Treatment Period, (at the investigator's discretion an additional 48 weeks),
Open-Label Phase post unblinding and upon Data Monitoring Committee positive recommendation
Post-treatment Follow-up Period

status: Active, not recruiting

study_completion_date_type: Anticipated

ReferenceType: result

ReferenceCitation: Porter J. Beyond transfusion therapy: new therapies in thalassemia including drugs, alternate donor transplant, and gene therapy. Hematology Am Soc Hematol Educ Program. 2018 Nov 30;2018(1):361-370. doi: 10.1182/asheducation-2018.1.361. Review.

name: University College London, London, United Kingdom

family: Porter

full_name: John Porter

given: John

journal_name: Hematology

oa_colors: green other

evidence: open (via free article)

publisher: American Society of Hematology

publisher_dissemination: American Society of Hematology

publisher_group: American Society of Hematology

publisher_normalized: American Society of Hematology

ReferenceCitation: Piga A, Perrotta S, Gamberini MR, Voskaridou E, Melpignano A, Filosa A, Caruso V, Pietrangelo A, Longo F, Tartaglione I, Borgna-Pignatti C, Zhang X, Laadem A, Sherman ML, Attie KM. Luspatercept improves hemoglobin levels and blood transfusion requirements in a study of patients with β-thalassemia. Blood. 2019 Mar 21;133(12):1279-1289. doi: 10.1182/blood-2018-10-879247. Epub 2019 Jan 7.

name: Department of Clinical and Biological Sciences, Turin University, Turin, Italy;

family: Piga

full_name: Antonio Piga

given: Antonio

name: Dipartimento della Donna, del Bambino e della Chirurgia Generale e Specialistica, Università degli Studi della Campania “Luigi Vanvitelli,” Naples, Italy;

family: Perrotta

full_name: Silverio Perrotta

given: Silverio

name: Thalassemia Unit, Arcispedale S. Anna, Ferrara, Italy;

family: Gamberini

full_name: Maria Rita Gamberini

given: Maria Rita

family: Voskaridou

full_name: Ersi Voskaridou

given: Ersi

family: Melpignano

full_name: Angela Melpignano

given: Angela

name: Rare Red Blood Cell Disease Unit, Cardarelli Hospital, Naples, Italy;

family: Filosa

full_name: Aldo Filosa

given: Aldo

name: Azienda Ospedaliera di Rilievo Nazionale e di Alta Specializzazione Garibaldi, Catania, Italy;

family: Caruso

full_name: Vincenzo Caruso

given: Vincenzo

name: Centro Emocromatosi e Malattie Eredometaboliche del Fegato, Medicina 2, Modena, Italy;

family: Pietrangelo

full_name: Antonello Pietrangelo

given: Antonello

family: Longo

full_name: Filomena Longo

given: Filomena

family: Tartaglione

full_name: Immacolata Tartaglione

given: Immacolata

name: Section of Pediatrics, Department of Medical Sciences, University of Ferrara, Ferrara, Italy;

family: Borgna-Pignatti

full_name: Caterina Borgna-Pignatti

given: Caterina

family: Zhang

full_name: Xiaosha Zhang

given: Xiaosha

family: Laadem

full_name: Abderrahmane Laadem

given: Abderrahmane

family: Sherman

full_name: Matthew L. Sherman

given: Matthew L.

family: Attie

full_name: Kenneth M. Attie

given: Kenneth M.

repository_institution: University of Turin - Institutional Research Information System University of Turin

ReferenceCitation: Cappellini MD, Taher AT. The use of luspatercept for thalassemia in adults. Blood Adv. 2021 Jan 12;5(1):326-333. doi: 10.1182/bloodadvances.2020002725.

name: Department of Clinical Sciences and Community, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca’ Granda Policlinico Hospital, University of Milan, Milan, Italy; and

name: Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon

ReferenceCitation: Cappellini MD, Viprakasit V, Taher AT, Georgiev P, Kuo KHM, Coates T, Voskaridou E, Liew HK, Pazgal-Kobrowski I, Forni GL, Perrotta S, Khelif A, Lal A, Kattamis A, Vlachaki E, Origa R, Aydinok Y, Bejaoui M, Ho PJ, Chew LP, Bee PC, Lim SM, Lu MY, Tantiworawit A, Ganeva P, Gercheva L, Shah F, Neufeld EJ, Thompson A, Laadem A, Shetty JK, Zou J, Zhang J, Miteva D, Zinger T, Linde PG, Sherman ML, Hermine O, Porter J, Piga A; BELIEVE Investigators. A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-Thalassemia. N Engl J Med. 2020 Mar 26;382(13):1219-1231. doi: 10.1056/NEJMoa1910182.

name: From the Department of Clinical Sciences and Community, University of Milan, IRCCS Ca’ Granda Foundation Maggiore Policlinico Hospital, Milan (M.D.C.), Centro della Microcitemia e Anemie Congenite e del Dismetabolismo del Ferro, E.O. Ospedali Galliera, Genoa (G.L.F.), Ospedale Pediatrico Microcitemico A. Cao, Azienda Ospedaliera G. Brotzu, Cagliari (R.O.), Ematologia e Oncologia Pediatrica, Università della Campania L. Vanvitelli, Caserta (S.P.), and the Department of Clinical and Biological Sciences,...

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

repository_institution: University College London - UCL Discovery

location_country:
Italy
United States
Lebanon
France
Bulgaria
Greece
Israel
Malaysia
Thailand
Tunisia
Australia
Turkey
Canada
United Kingdom
Taiwan

location_facility: Multiprofile Hospital for Active Treatment Sveta Marina EAD

location_facility: Hospital of the University of Pennsylvania

location_facility: GH de Institut Catholique St. VincentHématologie

location_facility: Universita degli Studi di Cagliari - ASL8

location_facility: Royal Adelaide Hospital Institute of Medical and Veterinary Science

location_facility: Childrens' Hospital of Athens Aghoa Sofia

location_facility: Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi

location_facility: Maharaj Nakorn Chiang Mai Chiang Mai University

location_facility: Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello

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

location_facility: Azienda Ospedaliera Universitaria Integrata Di Verona

location_facility: Seconda Universita Degli Studi Di Napoli

location_facility: Ege Universitesi Tip Fakultesi Hastanesi

location_facility: Azienda Ospedaliero Universitaria S. Luigi Gonzaga

location_facility: Ann and Robert H Lurie Childrens Hospital of Chicago

location_facility: Children's Hospital and Research Center at Oakland

location_facility: Azienda Ospedaliera Universitaria Di Ferrara Arcispedale Sanna

location_facility: Cukurova University Medical Faculty Balcali Hospital

location_facility: General Hospital Georgios Gennimatas of Athens

location_facility: Hospital Pulau Pinang c/o Penang Medical College

location_facility: Fondazione Ca Granda IRCCS Ospedale Maggiore

location_facility: University Mulitiprofile Hospital for Active Treatment Sveti Georgi EAD


ReferenceCitation: Mok T, Camidge DR, Gadgeel SM, Rosell R, Dziadziuszko R, Kim DW, Pérol M, Ou SI, Ahn JS, Shaw AT, Bordogna W, Smoljanović V, Hilton M, Ruf T, Noé J, Peters S. Updated overall survival and final progression-free survival data for patients with treatment-naive advanced ALK-positive non-small-cell lung cancer in the ALEX study. Ann Oncol. 2020 Aug;31(8):1056-1064. doi: 10.1016/j.annonc.2020.04.478. Epub 2020 May 11.

family: Mok

full_name: T. Mok

given: T.

family: Camidge

full_name: D.R. Camidge

family: Gadgeel

full_name: S.M. Gadgeel

family: Rosell

full_name: R. Rosell

given: R.

family: Dziadziuszko

full_name: R. Dziadziuszko

family: Kim

full_name: D.-W. Kim

family: Pérol

full_name: M. Pérol

given: M.

family: Ou

full_name: S.-H.I. Ou

family: Ahn

full_name: J.S. Ahn

family: Shaw

full_name: A.T. Shaw

family: Bordogna

full_name: W. Bordogna

given: W.

family: Smoljanović

full_name: V. Smoljanović

given: V.

family: Hilton

full_name: M. Hilton

family: Ruf

full_name: T. Ruf

family: Noé

full_name: J. Noé

given: J.

family: Peters

full_name: S. Peters

given: S.

journal_name: Annals of Oncology

oa_host_type: publisher

evidence: open (via crossref license)

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

ReferenceCitation: Gadgeel S, Peters S, Mok T, Shaw AT, Kim DW, Ou SI, Pérol M, Wrona A, Novello S, Rosell R, Zeaiter A, Liu T, Nüesch E, Balas B, Camidge DR. Alectinib versus crizotinib in treatment-naive anaplastic lymphoma kinase-positive (ALK+) non-small-cell lung cancer: CNS efficacy results from the ALEX study. Ann Oncol. 2018 Nov 1;29(11):2214-2222. doi: 10.1093/annonc/mdy405.

full_name: S. Gadgeel

full_name: D.W. Kim

full_name: S.I. Ou

repositories_institution: University of Turin - Institutional Research Information System University of Turin

ReferenceCitation: Burudpakdee C, Wong W, Seetasith A, Corvino FA, Yeh W, Gubens M. Economic impact of preventing brain metastases with alectinib in ALK-positive non-small cell lung cancer. Lung Cancer. 2018 May;119:103-111. doi: 10.1016/j.lungcan.2018.03.008. Epub 2018 Mar 9.

ReferenceCitation: Peters S, Camidge DR, Shaw AT, Gadgeel S, Ahn JS, Kim DW, Ou SI, Pérol M, Dziadziuszko R, Rosell R, Zeaiter A, Mitry E, Golding S, Balas B, Noe J, Morcos PN, Mok T; ALEX Trial Investigators. Alectinib versus Crizotinib in Untreated ALK-Positive Non-Small-Cell Lung Cancer. N Engl J Med. 2017 Aug 31;377(9):829-838. doi: 10.1056/NEJMoa1704795. Epub 2017 Jun 6.

title: Alectinib versus Crizotinib in Untreated ALK-Positive Non–Small-Cell Lung Cancer

ReferenceCitation: Gainor JF, Shaw AT. J-ALEX: alectinib versus crizotinib in ALK-positive lung cancer. Lancet. 2017 Jul 1;390(10089):3-4. doi: 10.1016/S0140-6736(17)31074-7. Epub 2017 May 10.

title: J-ALEX: alectinib versus crizotinib in ALK -positive lung cancer

location_country:
Guatemala
Australia
Russian Federation
Taiwan
Ukraine
Costa Rica
Spain
France
Poland
Thailand
Canada
Korea, Republic of
Chile
Hong Kong
Switzerland
Singapore
China
Portugal
United States
Germany
United Kingdom
Italy
Brazil
Bosnia and Herzegovina
Serbia
Mexico
Israel
Egypt
New Zealand
Turkey

location_facility: A.O. Universitaria Di Parma; Oncologia Medica

location_facility: Ms Clinsearch Specjalistyczny Niepubliczny Zaklad Opieki Zdrowotnej

location_facility: Chu Grenoble - Hopital Albert Michallon; Departement de Cancero-Hematologie

location_facility: University Clinical Center Sarajevo;Clinic for Pulmonary disease

location_facility: Samodzielny Publiczny Zespol Gruzlicy i Chorob Pluc; Oddzial V Chemioterapii Nowotworow Pluc

location_facility: Queen Mary Hospital; Medicine & Respiratory

location_facility: Hospital Universitari Germans Trias i Pujol; Servicio de Oncologia

location_facility: UniversitätsSpital Zürich; Zentrum für Hämatologie und Onkologie, Klinik für Onkologie

location_facility: Ospedale Provinciale Santa Maria Delle Croci; Oncologia Medica

location_facility: Scientific Research Oncology Institute named after N.N. Petrov; Oncology

location_facility: Mazowieckie Centrum Leczenia Chorob Pluc I Gruzlicy; Oddzial Iii

location_facility: Hospital Universitario Puerta de Hierro; Servicio de Oncologia

location_facility: University Clinical Centre of the Republic of Srpska; Clinic for Pulmonary Diseases

location_facility: N.N.Burdenko Main Military Clinical Hospital; Oncology Dept

location_facility: Az. Osp. S. Luigi Gonzaga; Malattie Apparato Respiratorio 5 Ad Indirizzo Oncologico

location_facility: Calvary Mater Newcastle; Medical Oncology

location_facility: Centre Leon Berard; Departement Oncologie Medicale

location_facility: Trakya University Medical Faculty Research And Practice Hospital Medical Oncology Department

location_facility: Lviv State Oncology Regional Treatment and Diagnostic Centre; Department of hemotherapy

location_facility: Inonu University Medical Faculty Turgut Ozal Medical Center Medical Oncology Department

location_facility: Centro Internacional de Estudios Clínicos (CIEC)

location_facility: North Valley Hem Onc Med Grp; Thomas&Dorothy Leavey Can Ctr

location_facility: Az Ospedaliera Nuovo Garibaldi Quartiere Nesima; Oncologia Medica

location_facility: Prince of Wales Hosp; Dept. Of Clinical Onc

location_facility: King Chulalongkorn Memorial Hospital; Faculty of Medicine Chulalongkorn University

location_facility: Severance Hospital, Yonsei University Health System

location_facility: St. Vincentius Kliniken Karlsruhe; Abteilung Hämatologie / Onkologie

location_facility: Medical Radiological Research Centre Rams; Dept. of Radiotherapy & Chemotherapy of Hemoblastosis

location_facility: CHUC - Unidade de Pneumologia Oncológica; Hospital de Dia de Oncologia Edificio Sao Jeronimo

location_facility: Irccs Istituto Nazionale Dei Tumori (Int);S.C. Medicina Oncologica 1

location_facility: Birmingham Heartlands Hospital; Dept of Oncology

location_facility: University of Texas M.D. Anderson Cancer Center

location_facility: Dnipropetrovsk State Medical Academy; Chemotherapy Department

location_facility: National Cancer Centre; Medical Oncology

location_facility: National University Hospital; National University Cancer Institute, Singapore (NCIS)

location_facility: Kasr Eieny Uni Hospital; Oncology (Nemrock)

location_facility: Ankara University Medical Faculty; Medikal Onkoloji

location_facility: Guys & St Thomas Hospital; Department of Oncology

location_facility: Instituto Nacional De Enfermedades Respiratorias;Unidad de Investigación

location_facility: Institute for pulmonary diseases of Vojvodina

location_facility: Northwest Georgia Oncology Centers, a Service of WellStar Cobb Hospital

location_facility: Chao Family Comprehensive Cancer Center; UC Irvine Medical Center

location_facility: Azienda Ospedaliera Di Perugia Ospedale s. Maria Della Misericordia; Oncologia Medica

location_facility: IPO do Porto; Servico de Oncologia Medica

location_facility: Songklanagarind Hospital; Department of Internal Medicine, Division of Respiratory

location_facility: Monash Health Translational Precinct; Clinical Trials Centre, Level 3

location_facility: Seoul National University Bundang Hospital

location_facility: Irccs Ist. Tumori Giovanni Paolo Ii; Dipartimento Oncologia Medica

location_facility: Massachusetts General Hospital Cancer Center

location_facility: Sarah Cannon Cancer Center - Tennessee Oncology, Pllc

location_facility: Saskatoon Cancer Centre; Uni of Saskatoon Campus

location_facility: Centrum Onkologii - Inst.Im. Marii Sklodowskiej-Curie; Oncology

location_facility: Uniwersyteckie Centrum Kliniczne; Klinika Onkologii i Radioterapii

location_facility: Baskent University Adana Dr. Turgut Noyan Practice and Research Hospital; Medical Oncology

location_facility: Hospital Universitario Virgen del Rocio; Servicio de Oncologia

location_facility: Hospital General Univ. de Alicante; Servicio de Oncologia

location_facility: Chiang Rai Prachanukroh Hospital; Department Of Medicine

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

location_facility: Pamela Youde Nethersole Eastern Hospital; Clinical Oncology

location_facility: Policlinico Umberto i di Roma; dip. Scienze Radiologiche, Oncologiche, Anatomopatologiche

location_facility: Kinghorn Cancer Centre; St Vincents Hospital

location_facility: University Clinical Center Sarajevo;Institute of oncology

location_facility: Klinik Löwenstein gGmbH Medizinische Klinik II

location_facility: St. Mary's Hospital Regional Cancer Center

location_facility: Seconda Universita' Degli Studi; Divsione Di Oncologia Medica

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

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

location_facility: Inselspital Bern; Universitätsklinik für medizinische Onkologie

location_facility: Queen Elizabeth Hospital; Medical Oncology

title: Randomized, Multicenter, Phase III, Open-Label Study of Alectinib Versus Crizotinib in Treatment-Naive Anaplastic Lymphoma Kinase-Positive Advanced Non-Small Cell Lung Cancer

summary: This randomized, active controlled, multicenter phase III open-label study is designed to evaluate the efficacy and safety of alectinib compared with crizotinib treatment in participants with treatment-naive anaplastic lymphoma kinase-positive (ALK-positive) advanced non-small cell lung cancer (NSCLC). Participants will be randomized in a 1:1 ratio to receive either alectinib, 600 milligrams (mg) orally twice daily (BID), or crizotinib, 250 mg orally BID. Participants will receive treatment until disease progression, unacceptable toxicity, consent withdrawal or death. The study is expected to last approximately 144 months.


title: A 52-week, Multicenter, Randomized, Double-blind Study of Subcutaneous Secukinumab to Demonstrate Efficacy as Assessed by Psoriasis Area and Severity Index at 16 Weeks of Treatment Compared to Ustekinumab and to Assess Long-term Safety, Tolerability and Efficacy in Subjects With Moderate to Severe Plaque Psoriasis (CLEAR)

summary: This study assessed efficacy of secukinumab, compared to ustekinumab, in patients that have plaque-type psoriasis

ReferenceCitation: Augustin M, Thaci D, Eyerich K, Pinter A, Radtke M, Lauffer F, Mrowietz U, Gerdes S, Pariser D, Lebwohl M, Sieder C, Melzer N, Reich K. Continued treatment with secukinumab is associated with high retention or regain of response. Br J Dermatol. 2020 Jan;182(1):67-75. doi: 10.1111/bjd.17991. Epub 2019 Jul 17.

name: Institute of Health Services Research in Dermatology and Nursing University Medical Center of Hamburg Eppendorf Germany

family: Augustin

full_name: M. Augustin

name: Comprehensive Centre of Inflammation Medicine University of Lübeck Lübeck Germany

family: Thaci

full_name: D. Thaci

given: D.

name: Department of Dermatology and Allergy Technical University of Munich Munich Germany

family: Eyerich

full_name: K. Eyerich

given: K.

name: University Hospital Frankfurt Frankfurt am Main Germany

family: Pinter

full_name: A. Pinter

given: A.

family: Radtke

full_name: M. Radtke

family: Lauffer

full_name: F. Lauffer

given: F.

name: Psoriasis‐Center Department of Dermatology University Medical Center Schleswig‐Holstein Kiel Germany

family: Mrowietz

full_name: U. Mrowietz

given: U.

family: Gerdes

full_name: S. Gerdes

name: Eastern Virginia Medical School and Virginia Clinical Research, Inc. Norfolk VA U.S.A.

family: Pariser

full_name: D. Pariser

name: Department of Dermatology Icahn School of Medicine at Mount Sinai New York U.S.A.

family: Lebwohl

full_name: M. Lebwohl

family: Sieder

full_name: C. Sieder

given: C.

family: Melzer

full_name: N. Melzer

given: N.

name: Dermatologikum Berlin and SCIderm Research Institute Hamburg Germany

family: Reich

full_name: K. Reich

journal_name: British Journal of Dermatology

publisher: Wiley

publisher_dissemination: Wiley

publisher_group: Wiley

publisher_normalized: Wiley

ReferenceCitation: Thaçi D, Blauvelt A, Reich K, Tsai TF, Vanaclocha F, Kingo K, Ziv M, Pinter A, Hugot S, You R, Milutinovic M. Secukinumab is superior to ustekinumab in clearing skin of subjects with moderate to severe plaque psoriasis: CLEAR, a randomized controlled trial. J Am Acad Dermatol. 2015 Sep;73(3):400-9. doi: 10.1016/j.jaad.2015.05.013. Epub 2015 Jun 17.

family: Thaçi

full_name: Diamant Thaçi

given: Diamant

family: Blauvelt

full_name: Andrew Blauvelt

given: Andrew

full_name: Kristian Reich

given: Kristian

family: Tsai

full_name: Tsen-Fang Tsai

given: Tsen-Fang

family: Vanaclocha

full_name: Francisco Vanaclocha

given: Francisco

family: Kingo

full_name: Külli Kingo

given: Külli

family: Ziv

full_name: Michael Ziv

given: Michael

journal_name: Journal of the American Academy of Dermatology

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

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

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

location_country:
Greece
Australia
Belgium
Denmark
Taiwan
France
Slovakia
Norway
Canada
Korea, Republic of
Turkey
Switzerland
Netherlands
Bulgaria
Hungary
Portugal
Germany
United States
United Kingdom
Italy
Austria
Israel
Spain
Estonia


title: A Controlled, Parallel Group, Open-label, Multicenter Extension Study to Investigate Efficacy and Safety of Oral BAY85-3934 and Darbepoetin Alfa Comparator in the Long Term Treatment of Anemia in Pre-dialysis Subjects With Chronic Kidney Disease in Europe and Asia Pacific

acronym: DIALOGUE 3

summary:
Anaemia is a condition in which blood has a lower than normal number of red blood cells. It can also occur if red blood cells do not contain enough haemoglobin, an oxygen carrying part of blood. Anaemia is common in patients with chronic kidney disease. Healthy kidneys produce a hormone called erythropoietin, which stimulates the bone marrow to produce the proper number of red blood cells needed to carry oxygen to vital organs. Chronic kidney disease is a general term that means that the kidneys are not functioning to their full potential. The study drug, BAY85-3934, is being evaluated as a drug to increase the body's ability to produce erythropoietin.

The purpose of this extension study is to find out if the study drug, a tablet taken orally, is safe and effective for the treatment of anaemia associated with chronic kidney disease.

The extension study will enroll up to 240 patients at multiple locations in Europe, Asia and Australia. Patients who participated in Studies 15141 or 15261 may be eligible to take part in the extension study. The study consists of the Haemoglobin (Hb) Stabilisation Phase and the Main Phase. The Hb Stabilisation Phase involves up to 10 study visits scheduled over 16 weeks. The Main Phase will last for at least 6 months and up to a maximum of 36 months, with visits every 4 weeks. During these scheduled visits patients will undergo a number of procedures to confirm efficacy and safety of the study drug, including measurement of heart rate and blood pressure, physical examination, Electrocardiogram and blood/urine sample collection for laboratory tests.

The study will be conducted at 5 hospitals in the UK. Bayer HealthCare AG is funding this research. This study will include subjects who either completed the treatment period in their respective Phase 2 parent study (i.e., Study 15141 or Study 15261) or experienced a stopping event in the fixed dose parent study (Study 15141). As Study 15141 is a double-blind study, subjects will be unblinded as per the Study 15141 protocol prior to entry into the extension study.

lead_sponsor: Bayer

location_country:
Italy
France
Bulgaria
Japan
Israel
Poland
Romania
Hungary
Australia
Spain
Turkey
Germany
United Kingdom
Korea, Republic of

location_facility:
Baskent University Medical Faculty
Ankara Univ. Medical Faculty
Sifa University Medical Faculty


type: Other Identifier

type: Registry Identifier

title: A Multi-centre Non-interventional Study of Safety and Efficacy of Turoctocog Alfa (rFVIII) During Long-Term Treatment of Severe and Moderately Severe Haemophilia A (FVIII =<2%)

acronym: guardian™ 5

summary: This study is conducted in Europe, and North and South America. The aim of this study is to provide additional documentation of the immunogenicity, and obtain additional clinical data, of turoctocog alfa in the setting of normal clinical practise in patients previously treated with a factor VIII agent (FVIII).

study_type: Observational

time_perspective: Prospective

lead_sponsor: Novo Nordisk A/S

location_country:
Italy
Austria
Sweden
Switzerland
Netherlands
Germany
France
Czechia
Greece
Poland
Slovakia
Hungary
Spain
United States
Slovenia


title: A Double-blind, Randomised, Placebo-controlled Study to Investigate the Efficacy and Safety of Mepolizumab in the Treatment of Eosinophilic Granulomatosis With Polyangiitis in Subjects Receiving Standard of Care Therapy

summary: The purpose of this randomized, double-blind study is to investigate the efficacy and safety of mepolizumab (300 milligram [mg] administered subcutaneously [SC] every 4 weeks) compared with placebo over a 52-week study treatment period in subjects with relapsing or refractory Eosinophilic Granulomatosis with Polyangiitis (EGPA) receiving standard of care therapy including background corticosteroid therapy with or without immunosuppressive therapy. During the treatment period, in accordance with standard of care, corticosteroid dose will be tapered. The key outcomes in the study focus on evaluation of clinical remission, defined as Birmingham Vasculitis Activity Score (BVAS)=0 with a corticosteroid dose of <=4 mg/day prednisolone/prednisone, reduction in disease relapse and reduction in corticosteroid requirement.

ReferenceCitation: Wechsler ME, Akuthota P, Jayne D, Khoury P, Klion A, Langford CA, Merkel PA, Moosig F, Specks U, Cid MC, Luqmani R, Brown J, Mallett S, Philipson R, Yancey SW, Steinfeld J, Weller PF, Gleich GJ; EGPA Mepolizumab Study Team. Mepolizumab or Placebo for Eosinophilic Granulomatosis with Polyangiitis. N Engl J Med. 2017 May 18;376(20):1921-1932. doi: 10.1056/NEJMoa1702079.

family: Wechsler

full_name: Michael E. Wechsler

given: Michael E.

family: Akuthota

full_name: Praveen Akuthota

given: Praveen

family: Jayne

full_name: David Jayne

given: David

family: Khoury

full_name: Paneez Khoury

given: Paneez

family: Klion

full_name: Amy Klion

given: Amy

family: Langford

full_name: Carol A. Langford

given: Carol A.

family: Merkel

full_name: Peter A. Merkel

given: Peter A.

family: Moosig

full_name: Frank Moosig

given: Frank

family: Specks

full_name: Ulrich Specks

given: Ulrich

family: Cid

full_name: Maria C. Cid

given: Maria C.

family: Luqmani

full_name: Raashid Luqmani

given: Raashid

family: Brown

full_name: Judith Brown

given: Judith

family: Mallett

full_name: Stephen Mallett

given: Stephen

family: Philipson

full_name: Richard Philipson

given: Richard

family: Yancey

full_name: Steve W. Yancey

given: Steve W.

family: Steinfeld

full_name: Jonathan Steinfeld

given: Jonathan

family: Weller

full_name: Peter F. Weller

given: Peter F.

family: Gleich

full_name: Gerald J. Gleich

given: Gerald J.

journal_name: New England Journal of Medicine

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

location_country:
Italy
United States
Germany
France
Japan
Belgium
Spain
Canada
United Kingdom


title: A Phase III, Open Label, Randomised, Controlled, Multi-centre Study to Assess the Efficacy and Safety of Olaparib Monotherapy Versus Physicians Choice Chemotherapy in the Treatment of Metastatic Breast Cancer Patients With Germline BRCA1/2 Mutations.

acronym: OlympiAD

summary: This open label, randomised, controlled, multi-centre phase III study will assess the efficacy and safety of single agent olaparib vs standard of care based on physician's choice of capecitabine, vinorelbine or eribulin in metastatic breast cancer patients with gBRCA 1/2 mutations.

ReferenceCitation: Robson M, Im SA, Senkus E, Xu B, Domchek SM, Masuda N, Delaloge S, Li W, Tung N, Armstrong A, Wu W, Goessl C, Runswick S, Conte P. Olaparib for Metastatic Breast Cancer in Patients with a Germline BRCA Mutation. N Engl J Med. 2017 Aug 10;377(6):523-533. doi: 10.1056/NEJMoa1706450. Epub 2017 Jun 4. Erratum in: N Engl J Med. 2017 Oct 26;377(17 ):1700.

family: Robson

full_name: Mark Robson

given: Mark

family: Im

full_name: Seock-Ah Im

given: Seock-Ah

family: Senkus

full_name: Elżbieta Senkus

given: Elżbieta

family: Xu

full_name: Binghe Xu

given: Binghe

family: Domchek

full_name: Susan M. Domchek

given: Susan M.

family: Masuda

full_name: Norikazu Masuda

given: Norikazu

family: Delaloge

full_name: Suzette Delaloge

given: Suzette

family: Li

full_name: Wei Li

given: Wei

family: Tung

full_name: Nadine Tung

given: Nadine

family: Armstrong

full_name: Anne Armstrong

given: Anne

family: Wu

full_name: Wenting Wu

given: Wenting

family: Goessl

full_name: Carsten Goessl

given: Carsten

family: Runswick

full_name: Sarah Runswick

given: Sarah

family: Conte

full_name: Pierfranco Conte

given: Pierfranco

repository_institution: University of Padova - Padua Research Archive

publisher: Massachusetts Medical Society

publisher_dissemination: Massachusetts Medical Society

publisher_group: Massachusetts Medical Society

publisher_normalized: Massachusetts Medical Society

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

lead_sponsor: AstraZeneca

location_country:
Italy
Peru
Switzerland
Mexico
Spain
France
Czechia
Bulgaria
Japan
Poland
Romania
Hungary
Russian Federation
China
Turkey
United States
United Kingdom
Korea, Republic of
Taiwan

status_simplified: Ongoing


title: A Randomized, Global, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Once-daily Oral Avatrombopag for the Treatment of Adults With Thrombocytopenia Associated With Liver Disease Prior to an Elective Procedure

summary: This is a global, multicenter, randomized, double-blind, placebo-controlled, parallel group study using avatrombopag to treat adults with thrombocytopenia associated with liver disease. The study will evaluate avatrombopag in the treatment of thrombocytopenia associated with liver disease prior to an elective procedure to reduce the need for platelet transfusions or any rescue procedure for bleeding due to procedural and post-procedural bleeding complications. Participants will be enrolled into 2 cohorts according to mean baseline platelet count and, within each baseline platelet count cohort will be further stratified by risk of bleeding associated with the elective procedure (low, moderate, or high) and hepatocellular carcinoma (HCC) status (Yes or No).

ReferenceCitation: Poordad F, Terrault NA, Alkhouri N, Tian W, Allen LF, Rabinovitz M. Avatrombopag, an Alternate Treatment Option to Reduce Platelet Transfusions in Patients with Thrombocytopenia and Chronic Liver Disease-Integrated Analyses of 2 Phase 3 Studies. Int J Hepatol. 2020 Jan 25;2020:5421632. doi: 10.1155/2020/5421632. eCollection 2020.

name: Texas Liver Institute and University of Texas Health San Antonio, San Antonio, TX, USA

family: Poordad

full_name: Fred Poordad

given: Fred

name: Keck Medicine at University of Southern California,  Los Angeles, CA, USA

family: Terrault

full_name: Norah A. Terrault

given: Norah A.

name: Texas Liver Institute, San Antonio, TX, USA

family: Alkhouri

full_name: Naim Alkhouri

given: Naim

name: Dova Pharmaceuticals, Inc., Durham, NC, USA

family: Tian

full_name: Wei Tian

family: Allen

full_name: Lee F. Allen

given: Lee F.

name: University of Pittsburgh, Pittsburgh, PA, USA

family: Rabinovitz

full_name: Mordechai Rabinovitz

given: Mordechai

currency_apc_doaj: USD

journal_name: International Journal of Hepatology

oa_colors: green gold

evidence: oa journal (via doaj)

license: cc-by

license_normalized: cc-by

publisher: Hindawi Limited

publisher_dissemination: Hindawi

publisher_group: Hindawi

publisher_normalized: Hindawi

ReferenceCitation: Terrault N, Chen YC, Izumi N, Kayali Z, Mitrut P, Tak WY, Allen LF, Hassanein T. Avatrombopag Before Procedures Reduces Need for Platelet Transfusion in Patients With Chronic Liver Disease and Thrombocytopenia. Gastroenterology. 2018 Sep;155(3):705-718. doi: 10.1053/j.gastro.2018.05.025. Epub 2018 May 17.

full_name: Norah Terrault

given: Norah

family: Chen

full_name: Yi-Cheng Chen

given: Yi-Cheng

family: Izumi

full_name: Namiki Izumi

given: Namiki

family: Kayali

full_name: Zeid Kayali

given: Zeid

family: Mitrut

full_name: Paul Mitrut

given: Paul

family: Tak

full_name: Won Young Tak

given: Won Young

family: Hassanein

full_name: Tarek Hassanein

given: Tarek

journal_name: Gastroenterology

location_country:
Italy
Brazil
Mexico
Spain
Germany
France
Czechia
Japan
Israel
Romania
Belgium
Argentina
Australia
China
Russian Federation
United States
Canada


title: An Open Label, Phase Ia/Ib Dose Finding Study With BI 894999 Orally Administered Once a Day in Patients With Advanced Malignancies, With Repeated Administration in Patients With Clinical Benefit

summary:
This study is open to adults with different types of advanced cancer (solid tumours). The study is also open to patients with diffuse large B-cell lymphoma in whom previous treatment was not successful. In some countries, adolescents who are at least 15 years old and who are diagnosed with NUT carcinoma can also participate. No standard treatment exists for this rare and aggressive form of cancer.

The purpose of this study is to find out the highest dose of BI 894999 that people can tolerate.

BI 894999 is tested for the first time in humans. Participants take tablets once daily. The study also tests whether participants can tolerate BI 894999 better when taken continuously or with breaks in between.

Participants can stay in the study as long as they benefit from the treatment and can tolerate it.

The doctors also regularly check the general health of the participants.

design_allocation: Non-Randomized

ipd_sharing_description:
After the study is completed and the primary manuscript is accepted for publishing, researchers can use this following link https://www.mystudywindow.com/msw/datasharing to request access to the clinical study documents regarding this study, and upon a signed "Document Sharing Agreement".

Also, Researchers can use the following link https://www.mystudywindow.com/msw/datasharing to find information in order to request access to the clinical study data, for this and other listed studies, after the submission of a research proposal and according to the terms outlined in the website.

The data shared are the raw clinical study data sets.

lead_sponsor: Boehringer Ingelheim

location_country:
United States
France
Belgium
Spain
Germany
Korea, Republic of

location_facility: The University of Texas MD Anderson Cancer Center


title: A Phase 3 Double-blind, Randomized, Placebo-controlled, Parallel-group Study To Assess The Efficacy, Long-term Safety And Tolerability Of Pf-04950615 In Subjects With Primary Hyperlipidemia Or Mixed Dyslipidemia At Risk Of Cardiovascular Events

acronym: SPIRE-LDL

summary: This study is a multicenter, randomized study in subjects with high cholesterol receiving highly effective statins to assess the efficacy, safety and tolerability of Bococizumab (PF-04950615;RN316) to lower LDL-C.

ReferenceCitation: Ridker PM, Rose LM, Kastelein JJP, Santos RD, Wei C, Revkin J, Yunis C, Tardif JC, Shear CL; Studies of PCSK9 Inhibition and the Reduction of vascular Events (SPIRE) Investigators. Cardiovascular event reduction with PCSK9 inhibition among 1578 patients with familial hypercholesterolemia: Results from the SPIRE randomized trials of bococizumab. J Clin Lipidol. 2018 Jul - Aug;12(4):958-965. doi: 10.1016/j.jacl.2018.03.088. Epub 2018 Apr 3.

family: Ridker

full_name: Paul M Ridker

given: Paul M

family: Rose

full_name: Lynda M. Rose

given: Lynda M.

family: Kastelein

full_name: John J.P. Kastelein

given: John J.P.

family: Santos

full_name: Raul D. Santos

given: Raul D.

family: Wei

full_name: Caimiao Wei

given: Caimiao

family: Revkin

full_name: James Revkin

given: James

family: Yunis

full_name: Carla Yunis

given: Carla

family: Tardif

full_name: Jean-Claude Tardif

given: Jean-Claude

family: Shear

full_name: Charles L. Shear

given: Charles L.

journal_name: Journal of Clinical Lipidology

ReferenceCitation: Ridker PM, Tardif JC, Amarenco P, Duggan W, Glynn RJ, Jukema JW, Kastelein JJP, Kim AM, Koenig W, Nissen S, Revkin J, Rose LM, Santos RD, Schwartz PF, Shear CL, Yunis C; SPIRE Investigators. Lipid-Reduction Variability and Antidrug-Antibody Formation with Bococizumab. N Engl J Med. 2017 Apr 20;376(16):1517-1526. doi: 10.1056/NEJMoa1614062. Epub 2017 Mar 17.

family: Amarenco

full_name: Pierre Amarenco

given: Pierre

family: Duggan

full_name: William Duggan

given: William

family: Glynn

full_name: Robert J. Glynn

given: Robert J.

family: Jukema

full_name: J. Wouter Jukema

given: J. Wouter

family: Kim

full_name: Albert M. Kim

given: Albert M.

family: Koenig

full_name: Wolfgang Koenig

given: Wolfgang

family: Nissen

full_name: Steven Nissen

given: Steven

location_country:
Mexico
France
Lithuania
Romania
Hungary
Russian Federation
Spain
United States
United Kingdom
Canada
Taiwan
Colombia

location_facility: Southern Health & Social Care Trust, Craigavon Area Hospital

location_facility: Spitalul Clinic Judetean de Urgenta Craiova, Cardiologie

location_facility: Northmed Clinical Research Ltd, Randalstown Health Centre

location_facility: National Clinical Research-Richmond, Inc.

location_facility: East Coast Institute for Research, LLC at Northeast Florida Cardiology Clinic

location_facility: Hospital Clinico Universitario Santiago de Compostela

location_facility: Saint-Petersburg State Budgetary Institution of Healthcare "Municipal Hospital #15"

location_facility: Clinical Research Advantage, Inc./Colorado Springs Health Partners, SW

location_facility: Arch Health Partners Clinical Research Department

location_facility: Synexus Thames Valley Clinical Research Centre

location_facility: University of North Texas Health Science Center at Fort Worth

location_facility: Borsod-Abauj-Zemplen Megyei Korhaz, es Egyetemi Oktatokorhaz

location_facility: North Texas Healthcare System, Dallas VAMC

location_facility: Federal State Budgetary Institution "Research Institute of Complex problems of Cardio-vascular

location_facility: East Coast Institute for Research, LLC at Jacksonville Cardiovascular Center

location_facility: Synexus Scotland Clinical Research Centre

location_facility: Synexus Midlands Clinical Research Centre

location_facility: Metabolic and Atherosclerosis Research Center

location_facility: Municipal Institution of Healthcare "City Clinical Hospital 12

location_facility: California Medical Research Associates Inc.

location_facility: Asociacion IPS Medicos InterniStas de Caldas

location_facility: Centrul Medical de Diagnostic si Tratament Ambulator Neomed, Medicina Interna

location_facility: Spitalul Universitar de Urgenta Militar Central, Dr. Carol Davila"

location_facility: Vizel Cardiac Research, Saul Vizel Professional Medicine Corporation

location_facility: Instituto Jalisciense de Investigacion en Diabetes y Obesidad, S.C.

location_facility: Atlanta Center for Clinical Research / Internal Medicine

location_facility: Centro de Atención e Investigación Cardiovascular del Potosí S.C.

location_facility: FSAEI of Higher Education "People's Friendship University of Russia",

location_facility: Sentral Clinical Research Services - Admin Only

location_facility: Vilnius University Hospital Santariskiu Klinikos

location_facility: Florida Institute For Clinical Research, LLC

location_facility: Centro de Medicina del Ejercicio y Rehabilitacion Cardiaca S.A. CEMDE S.A.

location_facility: Hospital Clinico Universitario Miguel Servet; Medicina Interna

location_facility: Synexus Manchester Clinical Research Centre

location_facility: Synexus North East Clinical Research Centre-Hexham General Hospital

location_facility: Synexus Lancashire Clinical Research Centre

location_facility: The University of Iowa - College of Public Health - Preventive Intervention Center

location_facility: Drug Shipment and Study Conducted at Address: University of Alabama at Birmingham

location_facility: Synexus North East Clinical Research Centre

location_facility: Synexus Merseyside Clinical Research Centre

location_facility: Magyar Imre Korhaz, Belgyogyaszati Szakrendeles

location_facility: Saint-Petersburg State Budgetary Institution of Healthcare Municipal hospital # 40 of the Kurortnyi

location_facility: East Coast Institute for Research, LLC at Northeast Florida Endocrine & Diabetes Associates

location_facility: The Bradford on Avon & Melksham Health Partnership

location_facility: Centro de Investigacion Cardiometabolica de Aguascalientes SA de CV

location_facility: New Orleans Center for Clinical Research

location_facility: LMC Research Inc. d.b.a. Manna Research Inc.

location_facility: Fleming Island Center for Clinical Research

location_facility: Clinical Research Institute of Southern Oregon, PC

location_facility: Hospital General de Naucalpan "Dr. Maximiliano Ruiz Castaneda" (Emergencies)

location_facility: Instituto de Investigacion Endocrinologia y Prevencion Metabolica ENDOCARE LTDA

location_facility: Centrul Medical Unirea, Diabet Nutritie si Boli Metabolice

location_facility: Fundacion del Caribe para la Investigacion Biomedica-Fundacion Bios

location_facility: Hospital Universitario La Paz; Unidad Metabolico-Vascular

location_facility: South Miami Clinical Research Center, LLC

location_facility: The Lindner Center for Research and Education at The Christ Hospital

location_facility: Instituto de Diabetes, Obesidad y Nutricion S.C.

location_facility: HealthEast St. Joseph's Hospital Pharmacy

location_facility: Centro de Investigaciones Clinicas S.A.S

location_facility: Centro Hospitalario Mac, S.A. De C.V. (Emergencies)

location_facility: Albany Medical College, Division of Community Endocrinology

location_facility: KCUMB Center for Community and Clinical Research

location_facility: IPS Centro Cientifico Asistencial Jose Luis Accini S.A.S.

location_facility: Administradora de Clinicas S.A. de C.V. Hospital Inovamed Cuernavaca

location_facility: Hospital Angeles Centro Medico del Potosi (Emergencies)

location_facility: Stroke Prevention & Atherosclerosis Research Centre (SPARC)

location_facility: London Road Diagnostic Clinic and Medical Centre

location_facility: Centro de Diagnostico Cardiologico LTDA.

location_facility: Rutgers Biomedical and Health Sciences- Robert Wood Johnson Medical School

location_facility: Detweiler Family Medicine and Associates, PC

location_facility: Fundacion Centro de Investigaciones Biomedicas RIESCARD

location_facility: Hospital Universitario Sant Joan de Reus

location_facility: Morton Plant Mease Health Care, Inc. Cardiovascular Research

location_facility: Metabolic Research Institute Incorporated

location_facility: East Coast Institute for Research, LLC at Baker-Gilmour Cardiovascular Institute, PA


lead_sponsor: Bristol-Myers Squibb

location_country:
Italy
Austria
United States
Switzerland
Netherlands
Germany
Finland
France
Japan
Norway
Australia
Spain
Denmark
Canada
United Kingdom

location_facility: Advocate Health and Hospitals Corporation

location_facility: Hospital Universitario Virgen De La Victoria

location_facility: H Lee Moffitt Cancer Center and Research Institute

location_facility: Sidney Kimmel Comprehensive Cancer Center At Johns Hopkins

location_facility: Istituto Nazionale Tumori Fondazione Pascale

location_facility: University Of Washington Cancer Care Alliance

location_facility: Washington University School Of Medicine

location_facility: Lehigh Valley Hospital and Health Network

title: A Phase I/2a Dose Escalation and Cohort Expansion Study of the Safety, Tolerability, and Efficacy of Anti-LAG-3 Monoclonal Antibody (BMS-986016) Administered Alone and in Combination With Anti-PD-1 Monoclonal Antibody (Nivolumab, BMS-936558) in Advanced Solid Tumors

summary:
The purpose of the study is to assess the safety, tolerability and effectiveness of experimental medication BMS-986016 administered alone and in combination with nivolumab in patients with solid tumors that have spread and/or cannot be removed by surgery.

The following tumor types are included in this study:

Non-Small Cell Lung Cancer (NSCLC), gastric cancer, hepatocellular carcinoma, renal cell carcinoma, bladder cancer, squamous cell carcinoma of the head and neck, and melanoma, that have NOT previously been treated with immunotherapy. NSCLC and melanoma that HAVE previously been treated with immunotherapy.


ReferenceType: background

ReferenceCitation: Landewé RB, van der Heijde D, Dougados M, Baraliakos X, Van den Bosch FE, Gaffney K, Bauer L, Hoepken B, Davies OR, de Peyrecave N, Thomas K, Gensler LS. Maintenance of clinical remission in early axial spondyloarthritis following certolizumab pegol dose reduction. Ann Rheum Dis. 2020 Jul;79(7):920-928. doi: 10.1136/annrheumdis-2019-216839. Epub 2020 May 7. Erratum in: Ann Rheum Dis. 2020 Sep;79(9):e120.

ReferenceCitation: Landewé R, van der Heijde D, Dougados M, Baraliakos X, Van den Bosch F, Gaffney K, Bauer L, Hoepken B, de Peyrecave N, Thomas K, Gensler LS. Induction of Sustained Clinical Remission in Early Axial Spondyloarthritis Following Certolizumab Pegol Treatment: 48-Week Outcomes from C-OPTIMISE. Rheumatol Ther. 2020 Sep;7(3):581-599. doi: 10.1007/s40744-020-00214-7. Epub 2020 Jun 11.

family: Landewé

full_name: Robert Landewé

given: Robert

family: van der Heijde

full_name: Désirée van der Heijde

given: Désirée

family: Dougados

full_name: Maxime Dougados

given: Maxime

family: Baraliakos

full_name: Xenofon Baraliakos

given: Xenofon

family: Van den Bosch

full_name: Filip Van den Bosch

given: Filip

family: Gaffney

full_name: Karl Gaffney

given: Karl

family: Bauer

full_name: Lars Bauer

given: Lars

family: Hoepken

full_name: Bengt Hoepken

given: Bengt

family: de Peyrecave

full_name: Natasha de Peyrecave

given: Natasha

family: Thomas

full_name: Karen Thomas

given: Karen

family: Gensler

full_name: Lianne S. Gensler

given: Lianne S.

journal_name: Rheumatology and Therapy

license: cc-by-nc

license_normalized: cc-by-nc

repository_institution: Ghent University - Ghent University Academic Bibliography

repository_institution: University of East Anglia - UEA Digital Repository

intervention_type: Other

lead_sponsor: UCB BIOSCIENCES GmbH

location_country:
Netherlands
Germany
France
Czechia
Bulgaria
Poland
Romania
Hungary
Belgium
Spain
Turkey
United States
United Kingdom
Taiwan

acronym: C-OPTIMISE

title: A Multicenter, Open-label (Part A) Followed by a Randomized, Double-blind, Parallel-group, Placebo Controlled Study (Part B) to Evaluate Maintenance of Remission in Subjects With Active Axial Spondyloarthritis (axSpA) Receiving Either Certolizumab Pegol 200 mg Q2W or 200 mg Q4W as Compared to Placebo

summary: Patients receive study drug for one year (Part A). If, after the initial run-in phase, a sustained remission is reached they will be randomly split into one of three dose groups for another year (Part B). The maintenance of the sustained remission will be analyzed.


location_country:
Peru
Finland
Romania
Australia
Russian Federation
Qatar
Taiwan
France
Japan
Poland
Argentina
Canada
Monaco
Korea, Republic of
Colombia
South Africa
Chile
Sweden
Netherlands
Singapore
Hungary
Germany
United States
United Kingdom
Italy
Austria
Brazil
Mexico
Czechia
Israel
Spain

location_facility: Rheumatologische Schwerpunktpraxis Dr. Zinke

location_facility: Clinical Research Center Of Reading, Llp

location_facility: Rheumatology Associates Of North Alabama, P.C.

location_facility: Oklahoma Ctr For Arthritis Therapy And Research, Inc.

location_facility: Organizacion Medica De Investigacion S.A. (Omi)

location_facility: Hospital Privado-Centro Medico De Cordoba S.A.

location_facility: Valerius Medical Group and Research Center

location_facility: Slaski Szpital Reumatologiczno - Rehabilitacyjny

location_facility: Instituto de Asistencia Reumatologica Integral

location_facility: Schwerpunktpraxis fuer Rheumatologie an den Kreiskliniken

location_facility: Rheumatology Associates Of Central Florida, P.A.

location_facility: Arthritis And Osteoporosis Associates, Pa

location_facility: SANUS Szpital Specjalistyczny Sp. z o.o.

location_facility: Fundacion Instituto de Reumatologia Fernando Chalem

location_facility: Instytut Reumatologii, I Klinika Reumatologii

location_facility: Immanuel Krankenhaus Berlin/Rheumaklinik Berlin Buch

location_facility: CHI St. Vincent Medical Group Hot Springs

location_facility: Arizona Arthritis & Rheumatology Research PLLC

location_facility: Centro de Investigacion en Reumatologia y Especialidades Medicas S.A.S

location_facility: Gemeinschaftspraxis Drs. von Hinueber u Demary

location_facility: Instituto De Ginecologia Y Reproduccion Inv. Clinical Sac

location_facility: Hospital General De Agudos J.M. Ramos Mejia

location_facility: Centro De Investigaciones Reumatologicas

location_facility: Inland Rheumatology Clinical Trials Inc.

location_facility: Centre De Recherche Musculo-Squelettique

location_facility: Arthritis Clinic Of Northern Virginia, P.C.

title: A Phase 3B, Randomized, Double-Blind Clinical Trial to Evaluate the Efficacy and Safety of Abatacept SC in Combination With Methotrexate Compared to Methotrexate Monotherapy in Achieving Clinical Remission in Adults With Early Rheumatoid Arthritis Who Are Methotrexate Naive

summary: The purpose of this study is to determine if abatacept is effective in the treatment of early rheumatoid arthritis.


title: A Phase 3, 24-Week Double-Blind, Placebo-Controlled, Parallel-Group, Efficacy and Safety Study of Reslizumab Subcutaneous Dosing (110 mg Every 4 Weeks) in Patients With Oral Corticosteroid Dependent Asthma and Elevated Blood Eosinophils

summary: The primary objective of the study is to determine the ability of reslizumab administered by subcutaneous injection to produce a corticosteroid-sparing effect in patients with oral corticosteroid (OCS)-dependent asthma and elevated blood eosinophils, without loss of asthma control.

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

family: Bernstein

full_name: Jonathan A Bernstein

given: Jonathan A

family: Virchow

full_name: J Christian Virchow

given: J Christian

family: Murphy

full_name: Kevin Murphy

given: Kevin

family: Maspero

full_name: Jorge Fernando Maspero

given: Jorge Fernando

family: Jacobs

full_name: Joshua Jacobs

given: Joshua

family: Adir

full_name: Yochai Adir

given: Yochai

family: Humbert

full_name: Marc Humbert

given: Marc

family: Castro

full_name: Mario Castro

given: Mario

family: Marsteller

full_name: Douglas A Marsteller

given: Douglas A

family: McElhattan

full_name: Jennifer McElhattan

given: Jennifer

family: Hickey

full_name: Lisa Hickey

given: Lisa

family: Garin

full_name: Margaret Garin

given: Margaret

family: Vanlandingham

full_name: Rebecca Vanlandingham

given: Rebecca

family: Brusselle

full_name: Guy Brusselle

given: Guy

journal_name: The Lancet Respiratory Medicine

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

location_country:
Italy
Mexico
Netherlands
Germany
Spain
France
Czechia
Poland
Israel
Hungary
Belgium
Argentina
Australia
Russian Federation
United States
Korea, Republic of
Ukraine

location_facility:
Teva Investigational Site 13363
Teva Investigational Site 58244
Teva Investigational Site 50418
Teva Investigational Site 13344
Teva Investigational Site 80091
Teva Investigational Site 53320
Teva Investigational Site 54132
Teva Investigational Site 50385
Teva Investigational Site 20050
Teva Investigational Site 53323
Teva Investigational Site 80085
Teva Investigational Site 13348
Teva Investigational Site 20066
Teva Investigational Site 58240
Teva Investigational Site 21093
Teva Investigational Site 20058
Teva Investigational Site 30154
Teva Investigational Site 35186
Teva Investigational Site 35189
Teva Investigational Site 20055
Teva Investigational Site 13365
Teva Investigational Site 80083
Teva Investigational Site 87020
Teva Investigational Site 53318
Teva Investigational Site 54133
Teva Investigational Site 20059
Teva Investigational Site 50383
Teva Investigational Site 13369
Teva Investigational Site 50356
Teva Investigational Site 87024
Teva Investigational Site 20087
Teva Investigational Site 32580
Teva Investigational Site 21101
Teva Investigational Site 31158
Teva Investigational Site 13346
Teva Investigational Site 20056
Teva Investigational Site 53321
Teva Investigational Site 58246
Teva Investigational Site 78097
Teva Investigational Site 21105
Teva Investigational Site 21091
Teva Investigational Site 21102
Teva Investigational Site 58241
Teva Investigational Site 51232
Teva Investigational Site 50417
Teva Investigational Site 35185
Teva Investigational Site 78091
Teva Investigational Site 13343
Teva Investigational Site 80084
Teva Investigational Site 58248
Teva Investigational Site 58251
Teva Investigational Site 87023
Teva Investigational Site 21094
Teva Investigational Site 53322
Teva Investigational Site 13352
Teva Investigational Site 58243
Teva Investigational Site 58235
Teva Investigational Site 32576
Teva Investigational Site 13357
Teva Investigational Site 87021
Teva Investigational Site 13371
Teva Investigational Site 13342
Teva Investigational Site 13367
Teva Investigational Site 80082
Teva Investigational Site 20051
Teva Investigational Site 32578
Teva Investigational Site 21103
Teva Investigational Site 37059
Teva Investigational Site 58239
Teva Investigational Site 58249
Teva Investigational Site 30152
Teva Investigational Site 51233
Teva Investigational Site 53319
Teva Investigational Site 31160
Teva Investigational Site 50382
Teva Investigational Site 58242
Teva Investigational Site 21106
Teva Investigational Site 58245
Teva Investigational Site 13354
Teva Investigational Site 50357
Teva Investigational Site 13350
Teva Investigational Site 38084
Teva Investigational Site 35187
Teva Investigational Site 21104
Teva Investigational Site 87025
Teva Investigational Site 31159
Teva Investigational Site 13372
Teva Investigational Site 13362
Teva Investigational Site 21090
Teva Investigational Site 38085
Teva Investigational Site 13356
Teva Investigational Site 78093
Teva Investigational Site 78090
Teva Investigational Site 51254
Teva Investigational Site 53358
Teva Investigational Site 58250
Teva Investigational Site 20052
Teva Investigational Site 32574
Teva Investigational Site 53317
Teva Investigational Site 50384
Teva Investigational Site 13377
Teva Investigational Site 58238
Teva Investigational Site 50386
Teva Investigational Site 32573
Teva Investigational Site 21100
Teva Investigational Site 32579
Teva Investigational Site 78092
Teva Investigational Site 87022
Teva Investigational Site 13366
Teva Investigational Site 32621
Teva Investigational Site 32622
Teva Investigational Site 13368
Teva Investigational Site 20057
Teva Investigational Site 53316
Teva Investigational Site 51253
Teva Investigational Site 13358
Teva Investigational Site 50419
Teva Investigational Site 13347
Teva Investigational Site 50358
Teva Investigational Site 58237
Teva Investigational Site 37058
Teva Investigational Site 78089
Teva Investigational Site 13351
Teva Investigational Site 13349
Teva Investigational Site 31161
Teva Investigational Site 13345
Teva Investigational Site 13370


ReferenceCitation: Aubert O, Divard G, Pascual J, Oppenheimer F, Sommerer C, Citterio F, Tedesco H, Chadban S, Henry M, Vincenti F, Srinivas T, Watarai Y, Legendre C, Bernhardt P, Loupy A. Application of the iBox prognostication system as a surrogate endpoint in the TRANSFORM randomised controlled trial: proof-of-concept study. BMJ Open. 2021 Oct 7;11(10):e052138. doi: 10.1136/bmjopen-2021-052138.

family: Aubert

full_name: Olivier Aubert

given: Olivier

family: Divard

full_name: Gillian Divard

given: Gillian

family: Pascual

full_name: Julio Pascual

given: Julio

family: Oppenheimer

full_name: Federico Oppenheimer

given: Federico

family: Sommerer

full_name: Claudia Sommerer

given: Claudia

family: Citterio

full_name: Franco Citterio

given: Franco

family: Tedesco

full_name: Helio Tedesco

given: Helio

family: Chadban

full_name: Steve Chadban

given: Steve

family: Henry

full_name: Mitchell Henry

given: Mitchell

family: Vincenti

full_name: Flavio Vincenti

given: Flavio

family: Srinivas

full_name: Titte Srinivas

given: Titte

family: Watarai

full_name: Yoshihiko Watarai

given: Yoshihiko

family: Legendre

full_name: Christophe Legendre

given: Christophe

family: Bernhardt

full_name: Peter Bernhardt

given: Peter

family: Loupy

full_name: Alexandre Loupy

given: Alexandre

currency_apc_doaj: GBP

journal_name: BMJ Open

publisher: BMJ

publisher_dissemination: British Medical Journal

publisher_group: British Medical Journal

publisher_normalized: British Medical Journal

ReferenceCitation: Watarai Y, Danguilan R, Casasola C, Chang SS, Ruangkanchanasetr P, Kee T, Wong HS, Kenmochi T, Amante AJ, Shu KH, Ingsathit A, Bernhardt P, Hernandez-Gutierrez MP, Han DJ, Kim MS. Everolimus-facilitated calcineurin inhibitor reduction in Asian de novo kidney transplant recipients: 2-year results from the subgroup analysis of the TRANSFORM study. Clin Transplant. 2021 Jul 3. doi: 10.1111/ctr.14415. [Epub ahead of print]

name: Department of Transplant Surgery, Kidney Disease Center Nagoya Daini Red Cross Hospital  Nagoya Japan

name: National Kidney and Transplant Institute  Quezon City Philippines

family: Danguilan

full_name: Romina Danguilan

given: Romina

family: Casasola

full_name: Concesa Casasola

given: Concesa

name: National Cheng Kung University College of Medicine and Hospital  Tainan Taiwan

family: Chang

full_name: Shen‐Shin Chang

given: Shen‐Shin

name: Phramongkutklao Hospital  Bangkok Thailand

name: Department of Renal Medicine Singapore General Hospital  Singapore Singapore

name: Department of Nephrology & Clinical Research Centre Hospital Selayang Selangor Darul Ehsan  Batu Caves Malaysia

name: Fujita Health University Hospital  Toyoake Japan

name: Taichung Veterans General Hospital  Taichung Taiwan

name: Department of Clinical Epidemiology and Biostatistics Faculty of Medicine, Ramathibodi Hospital Mahidol University  Bangkok Thailand

name: Asan Medical Center  Seoul Republic of Korea

name: Department of Surgery Yonsei University College of Medicine  Seoul Republic of Korea

title: Everolimus‐facilitated calcineurin inhibitor reduction in Asian de novo kidney transplant recipients: Two‐year results from the subgroup analysis of the TRANSFORM study

ReferenceCitation: Citterio F, Henry M, Kim DY, Kim MS, Han DJ, Kenmochi T, Mor E, Tisone G, Bernhardt P, Hernandez Gutierrez MP, Watarai Y. Wound healing adverse events in kidney transplant recipients receiving everolimus with reduced calcineurin inhibitor exposure or current standard-of-care: insights from the 24-month TRANSFORM study. Expert Opin Drug Saf. 2020 Oct;19(10):1339-1348. doi: 10.1080/14740338.2020.1792441. Epub 2020 Jul 20.

name: Department of Surgery, Fondazione Policlinico Universitario A. Gemelli, IRCCS, Rome, Italy

name: Department of Surgery, The Comprehensive Transplant Center, The Ohio State University, Wexner Medical Center, Columbus, OH, USA

name: Department of Transplant and Hepatobiliary Surgery, Henry Ford Hospital, Detroit, USA

name: Department of Surgery, Yonsei University College of Medicine, Seoul, Republic of Korea

name: Department of Surgery, Asan Medical Center, Seoul, Republic of South Korea

name: Department of Transplant Surgery, Fujita Health University, Toyoake, Japan

name: Department of Surgery, Transplant Center at Sheba Medical Center, Ramat-Gan, Israel

name: Department of Surgery HPB and Transplant Unit, University of Tor Vergata, Rome, Italy

name: Department of Research and Development, Novartis Pharma AG, Basel, Switzerland

name: Department of Transplant Surgery and Nephrology, Nagoya Daini Red Cross Hospital, Nagoya-City, Japan

ReferenceCitation: Berger SP, Sommerer C, Witzke O, Tedesco H, Chadban S, Mulgaonkar S, Qazi Y, de Fijter JW, Oppenheimer F, Cruzado JM, Watarai Y, Massari P, Legendre C, Citterio F, Henry M, Srinivas TR, Vincenti F, Gutierrez MPH, Marti AM, Bernhardt P, Pascual J; TRANSFORM investigators. Two-year outcomes in de novo renal transplant recipients receiving everolimus-facilitated calcineurin inhibitor reduction regimen from the TRANSFORM study. Am J Transplant. 2019 Nov;19(11):3018-3034. doi: 10.1111/ajt.15480. Epub 2019 Jul 1.

name: Division of Nephrology University of GroningenUniversity Medical Center Groningen Groningen The Netherlands

name: Department of Nephrology Heidelberg University Hospital Heidelberg Germany

name: Department of Infectious Diseases University Hospital EssenUniversity Duisburg‐Essen Essen Germany

name: Department of Nephrology University Hospital EssenUniversity Duisburg‐Essen Essen Germany

name: Nephrology Division Hospital do RimUniversidade Federal de São Paulo (UNIFESP) São Paulo Brazil

name: Department of Renal Medicine and Transplantation Royal Prince Alfred Hospital Camperdown New South Wales Australia

name: Renal and Pancreas DivisionSaint Barnabas Medical CenterLivingston New Jersey

name: Division of Nephrology Keck School of Medicine Renal Transplant Program University of Southern California Los Angeles California

name: LUMC Leids Universitair Medisch Centrum Leiden The Netherlands

name: Department of Nephrology and Renal Transplantation Renal Transplant UnitHospital Clinic of Barcelona Barcelona Spain

name: Hospital Universitari De BellvitgeIDIBELL, Hospitalet de Llobregat Barcelona Spain

name: Department of Transplant Surgery Nagoya Daini Red Cross Hospital Nagoya‐City, Aichi Japan

name: Hospital Privado Centro Medico de Cordoba Cordoba Argentina

name: Department of Kidney Transplantation Adult Transplantation Service Paris Descartes University and Necker Hospital Paris France

name: Agostino Gemelli University Polyclinic FoundationCatholic University of the Sacred Heart Rome Italy

name: Department of Surgery The Comprehensive Transplant CenterThe Ohio State University Wexner Medical Center Columbus Ohio

name: Division of Nephrology Medical University of South Carolina Mount Pleasant South Carolina

name: Department of Surgery Kidney Transplant Service University of California San Francisco California

name: Department of Research and Development Novartis Pharma AG Basel Switzerland

name: Department of Nephrology Hospital del Mar Barcelona Spain

title: Two‐year outcomes in de novo renal transplant recipients receiving everolimus‐facilitated calcineurin inhibitor reduction regimen from the TRANSFORM study

ReferenceCitation: Pascual J, Berger SP, Witzke O, Tedesco H, Mulgaonkar S, Qazi Y, Chadban S, Oppenheimer F, Sommerer C, Oberbauer R, Watarai Y, Legendre C, Citterio F, Henry M, Srinivas TR, Luo WL, Marti A, Bernhardt P, Vincenti F; TRANSFORM Investigators. Everolimus with Reduced Calcineurin Inhibitor Exposure in Renal Transplantation. J Am Soc Nephrol. 2018 Jul;29(7):1979-1991. doi: 10.1681/ASN.2018010009. Epub 2018 May 11.

name: on behalf of the TRANSFORM Investigators

journal_name: Journal of the American Society of Nephrology

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.clinicalstudydata request.com

location_country:
Malaysia
Greece
Australia
Belgium
Russian Federation
Taiwan
India
Croatia
France
Japan
Poland
Kuwait
Norway
Slovakia
Argentina
Thailand
Slovenia
Korea, Republic of
Colombia
South Africa
Chile
Sweden
Saudi Arabia
Switzerland
Netherlands
Philippines
Bulgaria
Singapore
Portugal
Germany
United States
Italy
Austria
Brazil
Serbia
Lebanon
Mexico
Czechia
Israel
Egypt
Spain
Turkey

title: A 24 Month, Multicenter, Randomized, Open-label Safety and Efficacy Study of Concentration-controlled Everolimus With Reduced Calcineurin Inhibitor vs Mycophenolate With Standard Calcineurin Inhibitor in de Novo Renal Transplantation

summary: This is a 2-year, randomized, multicenter, open-label, 2-arm study evaluating the graft function of everolimus and reduced CNI versus MPA and standard CNI in adult de novo renal transplant recipients.


lead_sponsor: Hoffmann-La Roche

location_country:
Italy
Austria
Brazil
Switzerland
Netherlands
Germany
Finland
France
Poland
Norway
Ireland
Russian Federation
Spain
Turkey
Denmark
United States
United Kingdom
Canada

acronym: Basket

location_facility: Herlev Hospital; Afdeling for Kræftbehandling

location_facility: Centre Leon Berard; Departement Oncologie Medicale

location_facility: St Vincent'S Uni Hospital; Medical Oncology

location_facility: Fondazione IRCCS Istituto Nazionale dei Tumori

location_facility: St James' Hospital; Cancer Clinical Trials Office

location_facility: Clinica Universitaria de Navarra; Servicio de Oncologia

location_facility: Odense Universitetshospital, Onkologisk Afdeling, Klinisk Forsknings Enhed

location_facility: Istanbul Uni Cerrahpasa Medical Faculty Hospital; Medical Oncology

location_facility: LKH-UNIV. KLINIKUM GRAZ; Klinische Abteilung für Onkologie

location_facility: S-Pb clinical scientific practical center of specialized kinds of medical care (oncological)

location_facility: Klinikum Mutterhaus der Borromaeerinnen gGmbH; Haematologie/Onkologie

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

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

location_facility: Hopital Saint Louis, Service D Oncologie Medicale

location_facility: Universitatsklinik Heidelberg; Universitätshautklinik und Nationales Centrum für Tumorerkrankungen

location_facility: Helsinki University Central Hospital; Dept of Oncology

location_facility: Narodowy Instytut Onkologii im. M.Sklodowskiej-Curie; Klinika Now. Tkanek Miekkich,Kosci i Czer.

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

location_facility: Prof. Dr. Cemil Tascioglu City Hospital; Med Onc

location_facility: Sarah Cannon Cancer Center and Research Institute

location_facility: Oslo Universitetssykehus HF; Radiumhospitalet

location_facility: Kantonsspital St. Gallen; Onkologie/Hämatologie

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

location_facility: Haukeland Universitetssjukehus; Klinisk forskningspost

location_facility: Southampton General Hospital; Medical Oncology

location_facility: Uniklinik-Eppendorf; Zentren F. Innere Medizin-Klinik U. Poliklinik

location_facility: Istituto Nazionale Tumori Fondazione G. Pascale

location_facility: Uniwersyteckie Centrum Kliniczne; Klinika Onkologii i Radioterapii

location_facility: Medizinische Universität Wien; Univ.Klinik für Innere Medizin I - Chemotherapie & Infektionskrankhei

location_facility: Hacettepe Uni Medical Faculty Hospital; Oncology Dept

location_facility: University Health Network; Princess Margaret Hospital; Medical Oncology Dept

title: An Open-Label, Multicohort, Phase II Study of Atezolizumab in Advanced Solid Tumors

summary: The primary efficacy objective for this study is to evaluate non-progression rate (NPR) at 18 weeks in participants with advanced solid tumors treated with atezolizumab, defined as the percentage of participants with complete response (CR), partial response (PR), or stable disease (SD) as assessed by the investigator according to Response Evaluation Criteria in Solid Tumors (RECIST) Version (v) 1.1, or according to disease-specific criteria for prostate cancer and malignant pleural mesothelioma.


lead_sponsor: PTC Therapeutics

location_country:
Italy
Brazil
Netherlands
Germany
France
Bulgaria
Greece
Israel
Poland
Belgium
Argentina
Australia
Spain
United States
United Kingdom
Canada

location_facility: Penn State Milton S. Hershey Medical Center

location_facility: University of Texas Health Science Center

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

location_facility: University Multiprofile Hospital for Active Treatment Aleksandrovska EAD

location_facility: University of Toronto Hospital for Sick Children

location_facility: Ippokratio General Hospital Of Thessaloniki

location_facility: Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul

location_facility: Hospital Regional Universitario de Malaga

location_facility: Azienda Ospedaliero Universitaria Ospedall Riuniti di Acona-Umberto I G.M. Lancisi G. Salesi

location_facility: Hospital de Sabadell, Consorci Sanitari Parc Tauli

location_facility: New York University Langone Medical Center

location_facility: Hopital Universitaire des Enfants Reine Fabiola

location_facility: Children's Hospital and Research Center at Oakland

location_facility: Szpital Dzieciecy Polanki im Macieja Plazynskiego w Gdansku

location_facility: University of Cologne Children's Hospital

location_facility: University Mulitiprofile Hospital for Active Treatment Sveti Georgi EAD

title: Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Cystic Fibrosis

summary: This is an open-label extension study for participants who completed a Phase 3, placebo-controlled study of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) not receiving chronic inhaled aminoglycosides.


title: A Phase 1, First-in-Human, Open-Label, Dose Escalation Study of Duvortuxizumab, A Humanized CD19 x CD3 Dual-Affinity Re-Targeting (DART®) Protein in Subjects With Relapsed or Refractory B-cell Malignancies

summary: The purpose of this study is to evaluate the safety, tolerability, dose-limiting toxicities (any harmful effect of a drug) (DLT), maximum tolerated dose (MTD), recommended Phase 2 dose (RP2D) and preliminary clinical activity of duvortuxizumab when administered intravenously to participants with relapsed or refractory B-cell malignancies [diffuse-large B cell lymphoma (DLBCL), follicular lymphoma (FL), mantle-cell lymphoma (MCL), chronic lymphocytic leukemia (CLL), and acute lymphoblastic leukemia (ALL)].

lead_sponsor: Janssen Research & Development, LLC

location_country:
France
Israel
Belgium
Spain
United States


title: Amplatzer™Amulet™ Observational Post-Market Study

acronym: Amulet™PMS

summary: This was a prospective, multicenter, observational, nonrandomized study to compile real world outcome data on the use of the Amulet™ device in non-valvular atrial fibrillation (NVAF) subjects. The study was designed to follow the Instructions For Use (IFU) to gather data on the implant procedure through two years of follow up with the Amulet™ device in a commercial clinical setting."

ReferenceCitation: De Caterina AR, Nielsen-Kudsk JE, Schmidt B, Mazzone P, Fischer S, Lund J, Montorfano M, Gage R, Lam SCC, Berti S. Gender difference in left atrial appendage occlusion outcomes: Results from the Amplatzer™ Amulet™ Observational Study. Int J Cardiol Heart Vasc. 2021 Jul 26;35:100848. doi: 10.1016/j.ijcha.2021.100848. eCollection 2021 Aug.

family: De Caterina

full_name: Alberto Ranieri De Caterina

given: Alberto Ranieri

family: Nielsen-Kudsk

full_name: Jens Erik Nielsen-Kudsk

given: Jens Erik

family: Schmidt

full_name: Boris Schmidt

given: Boris

family: Mazzone

full_name: Patrizio Mazzone

given: Patrizio

family: Fischer

full_name: Sven Fischer

given: Sven

family: Lund

full_name: Juha Lund

given: Juha

family: Montorfano

full_name: Matteo Montorfano

given: Matteo

family: Gage

full_name: Ryan Gage

given: Ryan

family: Lam

full_name: Simon Cheung Chi Lam

given: Simon Cheung Chi

family: Berti

full_name: Sergio Berti

given: Sergio

currency_apc_doaj: EUR

journal_name: IJC Heart & Vasculature

ReferenceCitation: Piayda K, Hellhammer K, Nielsen-Kudsk JE, Schmidt B, Mazzone P, Berti S, Fischer S, Lund J, Montorfano M, Della Bella P, Gage R, Zeus T. Clinical outcomes of patients undergoing percutaneous left atrial appendage occlusion in general anaesthesia or conscious sedation: data from the prospective global Amplatzer Amulet Occluder Observational Study. BMJ Open. 2021 Mar 24;11(3):e040455. doi: 10.1136/bmjopen-2020-040455.

family: Piayda

full_name: Kerstin Piayda

given: Kerstin

family: Hellhammer

full_name: Katharina Hellhammer

given: Katharina

ReferenceCitation: Aminian A, De Backer O, Nielsen-Kudsk JE, Mazzone P, Berti S, Fischer S, Lund J, Montorfano M, Lam SCC, Freixa X, Gage R, Diener HC, Schmidt B. Incidence and clinical impact of major bleeding following left atrial appendage occlusion: insights from the Amplatzer Amulet Observational Post-Market Study. EuroIntervention. 2021 Oct 20;17(9):774-782. doi: 10.4244/EIJ-D-20-01309.

ReferenceCitation: Tarantini G, D'Amico G, Schmidt B, Mazzone P, Berti S, Fischer S, Lund J, Montorfano M, Della Bella P, Lam SCC, Cruz-Gonzalez I, Gage R, Zhao H, Omran H, Odenstedt J, Nielsen-Kudsk JE. The Impact of CHA(2)DS(2)-VASc and HAS-BLED Scores on Clinical Outcomes in the Amplatzer Amulet Study. JACC Cardiovasc Interv. 2020 Sep 28;13(18):2099-2108. doi: 10.1016/j.jcin.2020.06.003.

title: The Impact of CHA2DS2-VASc and HAS-BLED Scores on Clinical Outcomes in the Amplatzer Amulet Study

location_country:
Italy
Chile
Sweden
Netherlands
Finland
France
Poland
Israel
Norway
Ireland
Belgium
Australia
Spain
Denmark
Germany
United Kingdom

location_facility: Hospital Clinic I Provincial de Barcelona

location_facility: Internistische Klinik Dr. Müller, München-Thalkirchen

location_facility: Zentrum fur Herzgesundheit/Kardiologie am Alice Hospital Darmstadt

location_facility: Charité - Universitätsmedizin Berlin (CBF)

location_facility: Nuovo Ospedale Civile Sant'Agostino Estense

location_facility: Ospedale dell'Angelo and Ospedale SS Giovanni e Paolo Venezia

location_facility: The Cardinal Stefan Wyszynski Institute of Cardiology

location_facility: Harzklinikum Dorothea Christiane Erxleben GmbH

location_facility: Ospedale del Cuoro Fondazione C. N. R. Regione Toscana G. Monasterio

location_facility: Azienda Ospedaliera S. Anna e S. Sebastiano

location_facility: Cardiac Arrhythmia Research Center, Centro Cardiologico Monzino, Unveristiy of Milan