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

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

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

location_facility: The Chinese People's Liberation Army 81 Hospital

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

location_facility: Jilin University the First Affiliated Hospital

location_facility: University of Miami - Sylvester Comprehensive Cancer Center

location_facility: Hunan Cancer Hospital, Radioactive Interventional Department

location_facility: IRCCS Foundation - National Institute of Tumors

location_facility: Johannes Gutenberg University Mainz - University Medical Center

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

location_facility: University of Liverpool - Clatterbridge Cancer Centre

location_facility: Harbin Medical University Cancer Hospital


intervention_type: Drug

status: Completed

study_start_date_type: Actual

study_type: Interventional

lead_sponsor: Bayer

enrollment_type: Actual

primary_purpose: Other

location_country:
Italy
United States
Finland
France
Hungary
Belgium
Spain
Canada

acronym: Phase 1 dry powder

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

location_facility: Hospital General Universitario Gregorio Marañón

location_facility: University of Semmelweis/ Semmelweis Egyetem

location_facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

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

location_facility: Turun yliopistollinen keskussairaala, kantasairaala

location_facility: Hôpital Arnaud de Villeneuve - Montpellier

location_facility: Hopital Necker les enfants malades - Paris

study_completion_date_type: Actual

type: EudraCT Number

source: euctr

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

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

lead_sponsor_type: industriel

status_simplified: Completed


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

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

design_allocation: Randomized

primary_purpose: Prevention

lead_sponsor: AstraZeneca

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


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

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

primary_purpose: Treatment

lead_sponsor: Grifols Therapeutics LLC

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

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

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

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

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

location_facility: University of Florida at Shands Jacksonville

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

location_facility: London Health Sciences Centre- University Hospital

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

location_facility: Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont

location_facility: Uniwersyteckie Centrum Kliniczne, Dept of Neurology

location_facility: Fakultni nemocnice Brno, Dept of Neurologicka klinika

location_facility: Houston Methodist Neurological Institute

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

location_facility: Universitaetsklinikum Giessen und Marburg GmbH Standort Marburg

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

location_facility: Samodzielny Publiczny Centralny Szpital Kliniczny

location_facility: Universitaetsklinikum Hamburg-Eppendorf, Klinik und Poliklinik

location_facility: Universitaetsklinikum Jena, Klinik fuer Neurologie

location_facility: Charité Universitaetsmedizin Berlin, Klinik für Neurologie

location_facility: Fachkrankenhaus Hubertusburg gGmbH, Klinik f. Neurologie

location_facility: CHU Strasbourg - Nouvel Hôpital Civil, Clinique Neurologique

location_facility: Hospital of Lithuanian University of Health Sciences Kaunas Clinics

location_facility: Ohio State University Wexner Medical Center, Neurology Department

location_facility: Yale University School of Medicine, Department of Neurology

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


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

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

design_allocation: Non-Randomized

ReferenceType: derived

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

family: Jung

full_name: Kyung Hae Jung

given: Kyung Hae

sequence: first

family: LoRusso

full_name: Patricia LoRusso

given: Patricia

sequence: additional

family: Burris

full_name: Howard Burris

given: Howard

family: Gordon

full_name: Michael Gordon

given: Michael

family: Bang

full_name: Yung-Jue Bang

given: Yung-Jue

family: Hellmann

full_name: Matthew D. Hellmann

given: Matthew D.

family: Cervantes

full_name: Andrés Cervantes

given: Andrés

family: Ochoa de Olza

full_name: Maria Ochoa de Olza

given: Maria

family: Marabelle

full_name: Aurelien Marabelle

given: Aurelien

family: Hodi

full_name: F. Stephen Hodi

given: F. Stephen

family: Ahn

full_name: Myung-Ju Ahn

given: Myung-Ju

family: Emens

full_name: Leisha A. Emens

given: Leisha A.

family: Barlesi

full_name: Fabrice Barlesi

given: Fabrice

family: Hamid

full_name: Omid Hamid

given: Omid

family: Calvo

full_name: Emiliano Calvo

given: Emiliano

family: McDermott

full_name: David McDermott

given: David

family: Soliman

full_name: Hatem Soliman

given: Hatem

family: Rhee

full_name: Ina Rhee

given: Ina

family: Lin

full_name: Ray Lin

given: Ray

family: Pourmohamad

full_name: Tony Pourmohamad

given: Tony

family: Suchomel

full_name: Julia Suchomel

given: Julia

family: Tsuhako

full_name: Amy Tsuhako

given: Amy

family: Morrissey

full_name: Kari Morrissey

given: Kari

publisher: American Association for Cancer Research (AACR)

location_country:
United States
Korea, Republic of
France
Spain

location_facility: Johns Hopkins Sidney Kimmel Comprehensive Cancer Center

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

location_facility: Hospital Universitario HM Sanchinarro-CIOCC

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

location_facility: HonorHealth Research Institute - Bisgrove

location_facility: Hospital Clinico Universitario de Valencia


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

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

ipd_sharing: Yes

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

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

lead_sponsor: Novartis Pharmaceuticals

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


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

family: Redondo

full_name: Andres Redondo

given: Andres

family: Colombo

full_name: Nicoletta Colombo

given: Nicoletta

family: McCormack

full_name: Mary McCormack

given: Mary

family: Dreosti

full_name: Lydia Dreosti

given: Lydia

family: Nogueira-Rodrigues

full_name: Angelica Nogueira-Rodrigues

given: Angelica

family: Scambia

full_name: Giovanni Scambia

given: Giovanni

family: Lorusso

full_name: Domenica Lorusso

given: Domenica

family: Joly

full_name: Florence Joly

given: Florence

family: Schenker

full_name: Michael Schenker

given: Michael

family: Ruff

full_name: Paul Ruff

given: Paul

family: Estevez-Diz

full_name: Maria Estevez-Diz

family: Irahara

full_name: Natsumi Irahara

given: Natsumi

family: Donica

full_name: Margarita Donica

given: Margarita

family: Gonzalez-Martín

full_name: Antonio Gonzalez-Martín

given: Antonio

bso_classification: Medical research

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: Gynecologic Oncology

publisher: Elsevier BV

publisher_dissemination: Elsevier

publisher_group: Elsevier

publisher_normalized: Elsevier

lead_sponsor: Hoffmann-La Roche

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

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

location_facility: Centro Oncologico MD Anderson International Espana

location_facility: Instituto Nacional de Cancerologia; Oncology

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

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

location_facility: IPO do Porto; Servico de Oncologia Medica

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

location_facility: Instituto Nacional de Cancer - INCa; Pesquisa Clinica

location_facility: Hospital Universitario la Fe; Servicio de Oncologia

location_facility: Hospital Universitario La Paz; Servicio de Oncologia

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

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

location_facility: Ankara Baskent University Medicine Faculty; Gynaecology

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

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

location_facility: Centro Oncologico Riojano Integral (CORI)

location_facility: University of Pretoria; Department of Medical Oncology

location_facility: Institut Gustave Roussy; Oncologie Medicale

location_facility: Alexandras General Hospital of Athens; Oncology Department

location_facility: Istanbul Uni of Medicine Faculty; Oncology Dept

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

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

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

location_facility: Instituto Valenciano Oncologia; Oncologia Medica

location_facility: Centre Francois Baclesse; Urologie Gynecologie

location_facility: Complex Oncological Center - Plovdiv, EOOD

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

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


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

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

ipd_sharing: Undecided

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

location_facility: Memorial Sloan Kettering Cancer Center SC

location_facility: Columbia University Medical Center SC LAG X2101C

location_facility: Huntsman Cancer Institute Huntsman Cancer Institute

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

location_facility: University of Texas MD Anderson Cancer Center


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

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

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

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

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


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

acronym: ELIANA

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

status: Active, not recruiting

study_completion_date_type: Anticipated

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

name: Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, United States

family: Mueller

full_name: Karen Thudium Mueller

given: Karen Thudium

name: University of Pennsylvania, United States

family: Grupp

full_name: Stephan A. Grupp

given: Stephan A.

family: Maude

full_name: Shannon L. Maude

given: Shannon L.

family: Levine

full_name: John E Levine

given: John E

name: Children's Hospital Los Angeles/USC Keck School of Medicine, Los Angeles, California, United States

family: Pulsipher

full_name: Michael A Pulsipher

given: Michael A

name: University of Utah, Salt Lake City, Utah, United States

family: Boyer

full_name: Michael W Boyer

given: Michael W

name: Children's Mercy Hospital, Kansas City, Missouri, United States

family: August

full_name: Keith August

given: Keith

family: Myers

full_name: G. Doug Myers

given: G. Doug

name: Peter MacCallum Cancer Center and The University of Melbourne, Melbourne, Australia

family: Tam

full_name: Constantine S. Tam

given: Constantine S.

name: Medical University of Vienna, Vienna, Austria

family: Jaeger

full_name: Ulrich Jaeger

given: Ulrich

family: Foley

full_name: Stephen Ronan Foley

given: Stephen Ronan

family: Borchmann

full_name: Peter Borchmann

given: Peter

name: University of Pennsylvania, Philadelphia, Pennsylvania, United States

family: Schuster

full_name: Stephen J Schuster

given: Stephen J

name: Winship Cancer Institute, Atlanta, Georgia, United States

family: Waller

full_name: Edmund K. Waller

given: Edmund K.

name: Novartis Institutes for BioMedical Research, East Hanover, New Jersey, United States

family: Awasthi

full_name: Rakesh Awasthi

given: Rakesh

family: Potthoff

full_name: Bernd Potthoff

given: Bernd

name: Salt River Integrated Bioanalysis GmbH, Basel, Switzerland

family: Warren

full_name: Andy Warren

given: Andy

name: Novartis, East Hanover, New Jersey, United States

family: Waldron

full_name: Edward Waldron

given: Edward

family: McBlane

full_name: Fraser McBlane

given: Fraser

family: Chassot-Agostinho

full_name: Andrea Chassot-Agostinho

given: Andrea

name: Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States

family: Laetsch

full_name: Theodore W. Laetsch

given: Theodore W.

journal_name: Blood Advances

oa_host_type: publisher

evidence: open (via free article)

title: Tisagenlecleucel Immunogenicity in Relapsed/Refractory Acute Lymphoblastic Leukemia and Diffuse Large B-Cell Lymphoma

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

repository_institution: Ghent University - Ghent University Academic Bibliography

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

name: Department of Pediatric Hematology and Oncology, Oslo University Hospital, Oslo, Norway;

name: Division of Oncology, Center for Childhood Cancer Research and Cancer Immunotherapy Program, Children’s Hospital of Philadelphia, Philadelphia, PA;

name: Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA;

name: Department of Pediatrics, Graduate School of Medicine, Kyoto University, Kyoto, Japan;

name: Department of Pediatric Hematology–Oncology and Institut de Recerca, Hospital Sant Joan de Déu Barcelona, Barcelona, Spain;

name: Department of Pediatrics and Harold C. Simmons Comprehensive Cancer Center, University of Texas Southwestern Medical Center and Children’s Health, Dallas, TX;

name: Department of Pediatrics, University of Michigan Medical Center, Ann Arbor, MI;

name: Novartis Pharmaceuticals Corporation, East Hanover, NJ; and

name: Department of Pediatric Hematology-Oncology and Stem Cell Transplantation, Ghent University Hospital, Ghent, Belgium

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

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

name: From the Departments of Pediatrics (S.L.M., S.A.G.) and Pathology and Laboratory Medicine (C.H.J., B.L.L.), Perelman School of Medicine, and Abramson Cancer Center (C.H.J., B.L.L.), University of Pennsylvania, and the Division of Oncology, Center for Childhood Cancer Research and Cancer Immunotherapy Program, Children’s Hospital of Philadelphia (S.L.M., S.A.G.) — all in Philadelphia; the Department of Pediatrics and Harold C. Simmons Comprehensive Cancer Center, University of Texas Southwestern Medical...

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

location_facility: The Childrens Hospital of Philadelphia CHOP

location_facility: Cincinnati Children's Hospital Medical Center

location_facility: Children's Healthcare of Atlanta SC CTL019

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

location_facility: University of Wisconsin Hospital and Clinics

location_facility: Childrens Hospital Los Angeles SC CTL019


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

acronym: TNBC

location_facility: Massachusetts General Hospital Cancer Center SC

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

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


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

location_facility: Childrens Healthcare of Atlanta Onc Dept

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

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

location_facility: Lurie Children's Hospital of Chicago Onc Dept

location_facility: Childrens Hospital of Philadelphia Onc. Dept

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

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

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

status_simplified: Ongoing


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

acronym: MERECA

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

ipd_sharing: No

lead_sponsor: Immunicum AB

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

location_facility: Hospital Universitari Germans Trias i Pujol

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

location_facility: Pauls Stradins Clinical University Hospital

location_facility: Centre Hospitalier Universitaire d'Angers

location_facility: Hospital Universitario Puerta de Hierro Majadahonda

location_facility: Centre Hospitalier Universitaire de Toulouse-Hôpital Rangueil


lead_sponsor: Bristol-Myers Squibb

enrollment_type: Anticipated

location_country:
United States
France
Australia
Spain
Canada

acronym: BET

location_facility: Institute for Translational Oncology Research-ITOR

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

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


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

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

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

family: Barcenas

full_name: C.H. Barcenas

family: Hurvitz

full_name: S.A. Hurvitz

family: Di Palma

full_name: J.A. Di Palma

family: Bose

full_name: R. Bose

given: R.

family: Chien

full_name: A.J. Chien

family: Iannotti

full_name: N. Iannotti

given: N.

family: Marx

full_name: G. Marx

given: G.

family: Brufsky

full_name: A. Brufsky

given: A.

family: Litvak

full_name: A. Litvak

family: Ibrahim

full_name: E. Ibrahim

given: E.

family: Alvarez

full_name: R.H. Alvarez

family: Ruiz-Borrego

full_name: M. Ruiz-Borrego

given: M.

family: Chan

full_name: N. Chan

family: Manalo

full_name: Y. Manalo

given: Y.

family: Kellum

full_name: A. Kellum

family: Trudeau

full_name: M. Trudeau

family: Thirlwell

full_name: M. Thirlwell

family: Garcia Saenz

full_name: J. Garcia Saenz

given: J.

family: Hunt

full_name: D. Hunt

given: D.

family: Bryce

full_name: R. Bryce

family: McCulloch

full_name: L. McCulloch

given: L.

family: Rugo

full_name: H.S. Rugo

family: Tripathy

full_name: D. Tripathy

full_name: A. Chan

family: Carcas

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

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

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

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

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

location_country:
Austria
United States
Germany
France
Australia
Spain
Canada

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

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

location_facility: Medical University of Innsbruck-Department of Gynecology

location_facility: Saint Joseph / Candler SC Cancer Specialists

location_facility: Universitaetsklinikum Schleswig-Holstein, Klinik fuer Gynaekologie und Geburtshilfe, Studienzentrale Gynäkologische Onkologie

location_facility: Great Plains Health (Callahan Cancer Center)

location_facility: Sana Klinikum Offenbach GmbH - Frauenklinik

location_facility: UCSF Helen Diller Family Comprehensive Cancer Center

location_facility: Washington University School of Medicine

location_facility: Uniklinikum Salzburg, Landeskrankenhaus, Universitätsklinik fur Innere Medizin III der PMU

location_facility: Universitätsklinik Klinik für Innere Medizin, Klinische Abteilung für Onkologie

location_facility: North Mississippi Medical Center Hematology and Oncology Services

location_facility: Praxis für interdisziplinäre Onkologie & Hämatologie

location_facility: Charleston Hematology Oncology Associates

location_facility: Good Samaritan Hospital Samaritan Pastega Regional Cancer Center

location_facility: The University of Texas MD Anderson Cancer Center

location_facility: Central Coast Medical Oncology Corporation

location_facility: Hematology-Oncology Associates of the Treasure Coast

location_facility: Cancer Center of Santa Barbara with Sansum Clinic

location_facility: Mammazentrum HH am Krankenhaus Jerusalem

location_facility: The Oncology Institute of Hope and Innovation

location_facility: McGill University Health Centre, Cedars Cancer Centre

location_facility: Medical University of Vienna, Department of Oncology

location_facility: Decatur Memorial Hospital Cancer Care Specialists of Central Illinois

location_facility: University of Maryland, Greenebaum Comprehensive Cancer Center

location_facility: Torrance Memorial Physician Network Cancer Care Associates


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

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

lead_sponsor: Celgene

location_country:
United States
France
Greece
Spain
Germany

location_facility: Winship Cancer Institute of Emory University

location_facility: Universitatsklinikum Heidelberg Medizinische Klinik Krehl-Klinik Haematologie, Onkologie, Rheumato

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

location_facility: CHRU de Lille-Hopital Claude Huriez Service des Maladies du Sang

location_facility: Alexandra Hospital, University of Athens


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

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

location_country:
Italy
Austria
Sweden
Finland
France
United States
United Kingdom


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

acronym: SHERLOC

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

status: Terminated

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

name: Massachusetts General Hospital Boston Massachusetts USA

family: Sequist

full_name: Lecia V. Sequist

given: Lecia V.

name: H. Lee Moffitt Cancer Center & Research Institute Tampa Florida USA

family: Gray

full_name: Jhanelle Elaine Gray

given: Jhanelle Elaine

name: Horizon Oncology Center Lafayette Indiana USA

family: Harb

full_name: Wael A. Harb

given: Wael A.

name: Knight Cancer Institute, Oregon Health and Science University Portland Oregon USA

family: Lopez‐Chavez

full_name: Ariel Lopez‐Chavez

given: Ariel

name: University of Colorado Cancer Center Aurora Colorado USA

family: Doebele

full_name: Robert C. Doebele

given: Robert C.

name: Arizona Clinical Research Center Tucson Arizona USA

family: Modiano

full_name: Manuel R. Modiano

given: Manuel R.

name: Dana‐Farber Cancer Institute Boston Massachusetts USA

family: Jackman

full_name: David Michael Jackman

given: David Michael

name: Division of Oncology, Washington University School of Medicine St. Louis Missouri USA

family: Baggstrom

full_name: Maria Quintos Baggstrom

given: Maria Quintos

name: Department of Hematology and Oncology, Institute of Clinical Research at Krankenhaus Nordwest, UCT‐University Cancer Center Frankfurt Germany

family: Atmaca

full_name: Akin Atmaca

given: Akin

name: Vall d'Hebron University Hospital Barcelona Spain

family: Felip

full_name: Enriqueta Felip

given: Enriqueta

name: Hospital Universitario Puerta de Hierro Majadahonda Madrid Spain

family: Provencio

full_name: Mariano Provencio

given: Mariano

name: Hospital Regional Universitario Málaga, Instituto de Investigación Biomédica de Málaga Málaga Spain

family: Cobo

full_name: Manuel Cobo

given: Manuel

name: Merrimack Pharmaceuticals, Inc. Cambridge Massachusetts USA

family: Adiwijaya

full_name: Bambang Adiwijaya

given: Bambang

family: Kuesters

full_name: Geoffrey Kuesters

given: Geoffrey

family: Kamoun

full_name: Walid S. Kamoun

given: Walid S.

family: Andreas

full_name: Karen Andreas

given: Karen

family: Pipas

full_name: J. Marc Pipas

given: J. Marc

family: Santillana

full_name: Sergio Santillana

given: Sergio

name: Yonsei University College of Medicine Seoul South Korea

family: Cho

full_name: Byoung Chul Cho

given: Byoung Chul

name: Department of Medicine, Division of Hematology‐Oncology, Samsung Medical Center, Sungkyunkwan University School of Medicine Seoul South Korea

family: Park

full_name: Keunchil Park

given: Keunchil

name: Princess Margaret Hospital Cancer Centre Toronto Ontario Canada

family: Shepherd

full_name: Frances A. Shepherd

given: Frances A.

journal_name: The Oncologist

oa_colors: green hybrid

oa_host_type: publisher;repository

title: Randomized Phase II Trial of Seribantumab in Combination with Erlotinib in Patients with EGFR Wild‐Type Non‐Small Cell Lung Cancer

location_country:
United States
France
Hungary
Spain
Germany
Canada


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

acronym: GNEM

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

ReferenceType: result

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

family: Lochmüller

full_name: Hanns Lochmüller

given: Hanns

family: Behin

full_name: Anthony Behin

given: Anthony

family: Caraco

full_name: Yoseph Caraco

given: Yoseph

family: Lau

full_name: Heather Lau

given: Heather

family: Mirabella

full_name: Massimiliano Mirabella

given: Massimiliano

family: Tournev

full_name: Ivailo Tournev

given: Ivailo

family: Tarnopolsky

full_name: Mark Tarnopolsky

given: Mark

family: Pogoryelova

full_name: Oksana Pogoryelova

given: Oksana

family: Woods

full_name: Catherine Woods

given: Catherine

family: Lai

full_name: Alexander Lai

given: Alexander

family: Shah

full_name: Jinay Shah

given: Jinay

family: Koutsoukos

full_name: Tony Koutsoukos

given: Tony

family: Skrinar

full_name: Alison Skrinar

given: Alison

family: Mansbach

full_name: Hank Mansbach

given: Hank

family: Kakkis

full_name: Emil Kakkis

given: Emil

family: Mozaffar

full_name: Tahseen Mozaffar

given: Tahseen

journal_name: Neurology

evidence: open (via page says license)

host_type: publisher

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

version: publishedVersion

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

host_type: repository

repository_institution: PubMed Central - Europe PMC

repository_normalized: PubMed Central

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

license: cc-by

license_normalized: cc-by

repository_institution: California Digital Library - eScholarship

evidence: oa repository (via pmcid lookup)

license_normalized: no license

publisher: Ovid Technologies (Wolters Kluwer Health)

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

location_country:
Italy
France
Bulgaria
Israel
United States
United Kingdom
Canada

location_facility: Washington University School of Medicine

location_facility: Institut de Myologie GH Pitié-Salpêtrière

location_facility: Hadassah-Hebrew University Medical Center


ReferenceType: background

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

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

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

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

lead_sponsor: Amgen

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

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

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


ReferenceCitation: Stahl A, Lepore D, Fielder A, Fleck B, Reynolds JD, Chiang MF, Li J, Liew M, Maier R, Zhu Q, Marlow N. Ranibizumab versus laser therapy for the treatment of very low birthweight infants with retinopathy of prematurity (RAINBOW): an open-label randomised controlled trial. Lancet. 2019 Oct 26;394(10208):1551-1559. doi: 10.1016/S0140-6736(19)31344-3. Epub 2019 Sep 12.

family: Stahl

full_name: Andreas Stahl

given: Andreas

family: Lepore

full_name: Domenico Lepore

given: Domenico

family: Fielder

full_name: Alistair Fielder

given: Alistair

family: Fleck

full_name: Brian Fleck

given: Brian

family: Reynolds

full_name: James D Reynolds

given: James D

family: Chiang

full_name: Michael F Chiang

given: Michael F

family: Li

full_name: Jun Li

given: Jun

family: Liew

full_name: Melissa Liew

given: Melissa

family: Maier

full_name: Rainer Maier

given: Rainer

family: Zhu

full_name: Qi Zhu

given: Qi

family: Marlow

full_name: Neil Marlow

given: Neil

journal_name: The Lancet

oa_host_type: repository

repository_institution: University College London - UCL Discovery

repository_normalized: UCL Discovery

version: submittedVersion

intervention_type: Procedure

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

acronym: RAINBOW

title: RAINBOW Study: a Randomized, Controlled Study Evaluating the Efficacy and Safety of RAnibizumab Compared With Laser Therapy for the Treatment of INfants BOrn Prematurely With Retinopathy of Prematurity

summary: The purpose of this study was to determine if intravitreal ranibizumab is superior to laser ablation therapy in the treatment of retinopathy of prematurity (ROP).


title: A Double-blind, Randomized, Placebo-controlled Phase 3 Study of Orally Administered PLX3397 in Subjects With Pigmented Villonodular Synovitis or Giant Cell Tumor of the Tendon Sheath

acronym: ENLIVEN

summary:
This is a Phase 3 clinical study, which aims to evaluate the effectiveness of an investigational drug called pexidartinib for the treatment of certain tumors for which surgical removal could cause more harm than good.

The main purpose of this study is to gather information about the investigational drug pexidartinib, which may help to treat tumors of pigmented villonodular synovitis (PVNS) or giant cell tumor of the tendon sheath (GCT-TS).

The study consists of two parts with a follow-up period. In Part 1, eligible study participants will be assigned to receive either pexidartinib or matching placebo for 24 weeks. A number of assessments will be carried out during the course of the study, including physical examinations, blood tests, imaging studies, electrocardiograms, and questionnaires. MRI scans will be used to evaluate the response of the tumors to the treatment. Some subjects, assigned to placebo in Part 1 transitioned to pexidartinib for Part 2.

Then a protocol amendment was written to allow only pexidartinib patients to continue into Part 2. Part 2 is a long-term treatment phase in which all participants receive open-label pexidartinib. There was also a follow-up period added to Part 2.

ReferenceCitation: Lewis JH, Gelderblom H, van de Sande M, Stacchiotti S, Healey JH, Tap WD, Wagner AJ, Pousa AL, Druta M, Lin CC, Baba HA, Choi Y, Wang Q, Shuster DE, Bauer S. Pexidartinib Long-Term Hepatic Safety Profile in Patients with Tenosynovial Giant Cell Tumors. Oncologist. 2021 May;26(5):e863-e873. doi: 10.1002/onco.13629. Epub 2020 Dec 24.

name: Georgetown University Hospital  Washington District of Columbia USA

family: Lewis

full_name: James H. Lewis

given: James H.

name: Leiden University Medical Center  Leiden The Netherlands

family: Gelderblom

full_name: Hans Gelderblom

given: Hans

family: Sande

full_name: Michiel Sande

given: Michiel

name: Fondazione IRCCS Istituto Nazionale dei Tumori  Milan Italy

family: Stacchiotti

full_name: Silvia Stacchiotti

given: Silvia

name: Memorial Sloan Kettering Cancer Center and Weill Cornell Medical College  New York New York USA

family: Healey

full_name: John H. Healey

given: John H.

family: Tap

full_name: William D. Tap

given: William D.

name: Dana‐Farber Cancer Institute  Boston Massachusetts USA

family: Wagner

full_name: Andrew J. Wagner

given: Andrew J.

name: Hospital de la Santa Creu I Sant Pau  Barcelona Spain

family: Pousa

full_name: Antonio Lopez Pousa

given: Antonio Lopez

name: Moffitt Cancer Center  Tampa Florida USA

family: Druta

full_name: Mihaela Druta

given: Mihaela

name: National Taiwan University Hospital  Taipei Taiwan

family: Lin

full_name: Chia‐Chi Lin

given: Chia‐Chi

name: University Hospital Essen, University of Duisburg‐Essen  Germany

family: Baba

full_name: Hideo A. Baba

given: Hideo A.

name: Daiichi Sankyo, Inc  Basking Ridge New Jersey USA

family: Choi

full_name: Youngsook Choi

given: Youngsook

family: Wang

full_name: Qiang Wang

given: Qiang

family: Shuster

full_name: Dale E. Shuster

given: Dale E.

family: Bauer

full_name: Sebastian Bauer

given: Sebastian

evidence: open (via crossref license)

publisher: Wiley

publisher_dissemination: Wiley

publisher_group: Wiley

publisher_normalized: Wiley

title: Pexidartinib Long‐Term Hepatic Safety Profile in Patients with Tenosynovial Giant Cell Tumors

ReferenceCitation: Tap W. ENLIVEN study: Pexidartinib for tenosynovial giant cell tumor (TGCT). Future Oncol. 2020 Sep;16(25):1875-1878. doi: 10.2217/fon-2020-0307. Epub 2020 Aug 5.

name: Memorial Sloan Kettering Cancer Center and Weill Cornell Medical College, New York, NY, USA

full_name: William Tap

given: William

ReferenceCitation: Tap WD, Gelderblom H, Palmerini E, Desai J, Bauer S, Blay JY, Alcindor T, Ganjoo K, Martín-Broto J, Ryan CW, Thomas DM, Peterfy C, Healey JH, van de Sande M, Gelhorn HL, Shuster DE, Wang Q, Yver A, Hsu HH, Lin PS, Tong-Starksen S, Stacchiotti S, Wagner AJ; ENLIVEN investigators. Pexidartinib versus placebo for advanced tenosynovial giant cell tumour (ENLIVEN): a randomised phase 3 trial. Lancet. 2019 Aug 10;394(10197):478-487. doi: 10.1016/S0140-6736(19)30764-0. Epub 2019 Jun 19.

ReferenceCitation: Gelhorn HL, Tong S, McQuarrie K, Vernon C, Hanlon J, Maclaine G, Lenderking W, Ye X, Speck RM, Lackman RD, Bukata SV, Healey JH, Keedy VL, Anthony SP, Wagner AJ, Von Hoff DD, Singh AS, Becerra CR, Hsu HH, Lin PS, Tap WD. Patient-reported Symptoms of Tenosynovial Giant Cell Tumors. Clin Ther. 2016 Apr;38(4):778-93. doi: 10.1016/j.clinthera.2016.03.008. Epub 2016 Apr 1.

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

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

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

location_facility: Istituto Nazionale Tumori-Fondazione IRCCS


title: A Six-Week, Double-Blind, Placebo-Controlled, Randomized- Withdrawal, Multicenter Study of the Safety and Efficacy of JZP-110 [(R)-2-amino-3-phenylpropylcarbamate Hydrochloride] in the Treatment of Excessive Sleepiness in Subjects With Obstructive Sleep Apnea (OSA)

acronym: OSA

summary: This trial is a 6-week, double-blind, placebo-controlled, randomized-withdrawal, multicenter study of safety and efficacy of JZP-110 in the treatment of excessive sleepiness in adult subjects with OSA.

ReferenceCitation: Strollo PJ Jr, Hedner J, Collop N, Lorch DG Jr, Chen D, Carter LP, Lu Y, Lee L, Black J, Pépin JL, Redline S; Tones 4 Study Investigators. Solriamfetol for the Treatment of Excessive Sleepiness in OSA: A Placebo-Controlled Randomized Withdrawal Study. Chest. 2019 Feb;155(2):364-374. doi: 10.1016/j.chest.2018.11.005. Epub 2018 Nov 22.

family: Strollo

full_name: Patrick J. Strollo

given: Patrick J.

suffix: Jr.

family: Hedner

full_name: Jan Hedner

given: Jan

family: Collop

full_name: Nancy Collop

given: Nancy

family: Lorch

full_name: Daniel G. Lorch

given: Daniel G.

family: Chen

full_name: Dan Chen

given: Dan

family: Carter

full_name: Lawrence P. Carter

given: Lawrence P.

family: Lu

full_name: Yuan Lu

given: Yuan

family: Lee

full_name: Lawrence Lee

given: Lawrence

family: Black

full_name: Jed Black

given: Jed

family: Pépin

full_name: Jean-Louis Pépin

given: Jean-Louis

family: Redline

full_name: Susan Redline

given: Susan

family: Partinen

full_name: Markku Partinen

given: Markku

family: Polo

full_name: Olli Polo

given: Olli

family: Saaresranta

full_name: Tarja Saaresranta

given: Tarja

family: Paschen

full_name: Christine Paschen

given: Christine

family: Leissner

full_name: Lena Leissner

given: Lena

family: Block

full_name: Bradley Block

given: Bradley

family: Doekel

full_name: Robert Doekel

given: Robert

family: Duntley

full_name: Stephen Duntley

given: Stephen

family: Giangreco

full_name: Guillermo Giangreco

location_country:
United States
Sweden
Finland
France
Germany

location_facility: Center for Sleep & Circadian Neurobiology

location_facility: The Research Center of Southern California

location_facility: Critical Care Pulmonary & Sleep Associates, LLC


ReferenceCitation: Lebwohl M, Blauvelt A, Paul C, Sofen H, Węgłowska J, Piguet V, Burge D, Rolleri R, Drew J, Peterson L, Augustin M. Certolizumab pegol for the treatment of chronic plaque psoriasis: Results through 48 weeks of a phase 3, multicenter, randomized, double-blind, etanercept- and placebo-controlled study (CIMPACT). J Am Acad Dermatol. 2018 Aug;79(2):266-276.e5. doi: 10.1016/j.jaad.2018.04.013. Epub 2018 Apr 14.

family: Lebwohl

full_name: Mark Lebwohl

given: Mark

family: Blauvelt

full_name: Andrew Blauvelt

given: Andrew

family: Paul

full_name: Carle Paul

given: Carle

family: Sofen

full_name: Howard Sofen

given: Howard

family: Węgłowska

full_name: Jolanta Węgłowska

given: Jolanta

family: Piguet

full_name: Vincent Piguet

given: Vincent

family: Burge

full_name: Daniel Burge

given: Daniel

family: Rolleri

full_name: Robert Rolleri

family: Drew

full_name: Janice Drew

given: Janice

family: Peterson

full_name: Luke Peterson

given: Luke

family: Augustin

full_name: Matthias Augustin

given: Matthias

journal_name: Journal of the American Academy of Dermatology

ReferenceCitation: Warren RB, Lebwohl M, Sofen H, Piguet V, Augustin M, Brock F, C Arendt, Fierens F, Blauvelt A. Three-year efficacy and safety of certolizumab pegol for the treatment of plaque psoriasis: results from the randomized phase 3 CIMPACT trial. J Eur Acad Dermatol Venereol. 2021 Dec;35(12):2398-2408. doi: 10.1111/jdv.17486. Epub 2021 Aug 17.

name: Dermatology Centre Salford Royal NHS Foundation Trust Manchester NIHR Biomedical Research Centre The University of Manchester Manchester UK

family: Warren

full_name: R. B. Warren

given: R. B.

name: Icahn School of Medicine at Mount Sinai New York NY USA

full_name: M. Lebwohl

given: M.

name: David Geffen School of Medicine at UCLA Los Angeles CA USA

full_name: H. Sofen

given: H.

name: Division of Dermatology Department of Medicine University of Toronto Toronto ON Canada

name: Division of Dermatology Department of Medicine Women’s College Hospital Toronto ON Canada

full_name: V. Piguet

given: V.

name: Institute for Health Services Research in Dermatology and Nursing (IVDP) University Medical Center Hamburg‐Eppendorf (UKE) Hamburg Germany

full_name: M. Augustin

family: Brock

full_name: F. Brock

given: F.

family: C. Arendt

full_name: C. Arendt

family: Fierens

full_name: F. Fierens

name: Oregon Medical Research Center Portland OR USA

full_name: A. Blauvelt

given: A.

journal_name: Journal of the European Academy of Dermatology and Venereology

title: Three‐year efficacy and safety of certolizumab pegol for the treatment of plaque psoriasis: results from the randomized phase 3 CIMPACT trial

ReferenceCitation: Blauvelt A, Paul C, van de Kerkhof P, Warren RB, Gottlieb AB, Langley RG, Brock F, Arendt C, Boehnlein M, Lebwohl M, Reich K. Long-term safety of certolizumab pegol in plaque psoriasis: pooled analysis over 3 years from three phase III, randomized, placebo-controlled studies. Br J Dermatol. 2021 Apr;184(4):640-651. doi: 10.1111/bjd.19314. Epub 2020 Sep 6.

name: Paul Sabatier University Toulouse France

name: Radboud University Nijmegen the Netherlands

name: Translational Research in Inflammatory Skin Diseases Institute for Health Services Research in Dermatology and Nursing University Medical Center Hamburg‐Eppendorf Hamburg Germany

name: Skinflammation<sup>®</sup> Center Hamburg Germany

title: Long‐term safety of certolizumab pegol in plaque psoriasis: pooled analysis over 3 years from three phase III, randomized, placebo‐controlled studies

location_country:
United States
Netherlands
France
Czechia
Bulgaria
Poland
Hungary
Germany
United Kingdom

title: A Phase 3, Multicenter, Randomized, Double-Blind, Parallel-Group, Placebo- and Active-Controlled Study Followed by a Placebo-Controlled Maintenance Period and Open-Label Follow-Up to Evaluate the Efficacy and Safety of Certolizumab Pegol in Subjects With Moderate to Severe Chronic Plaque Psoriasis

summary: The purpose of this study is to investigate the efficacy and safety of two dose levels of certolizumab pegol compared to active comparator and placebo in adults with moderate to severe chronic plaque psoriasis.


title: An Initial Evaluation of the Safety and Performance of the Transcatheter Mitral Valve Replacement System in Patients With Severe, Symptomatic Mitral Regurgitation

summary:
PILOT:

The study is pilot trial to evaluate the safety and performance of the Twelve TMVR System in very high risk mitral regurgitation patients.

EFS:

The study is an EFS trial to evaluate the safety and performance of the Medtronic Intrepid™ Transcatheter Mitral Valve Replacement System with Transfemoral Transseptal access in Patients with Severe, Symptomatic Mitral Regurgitation

status: Recruiting

ReferenceCitation: Bapat V, Rajagopal V, Meduri C, Farivar RS, Walton A, Duffy SJ, Gooley R, Almeida A, Reardon MJ, Kleiman NS, Spargias K, Pattakos S, Ng MK, Wilson M, Adams DH, Leon M, Mack MJ, Chenoweth S, Sorajja P; Intrepid Global Pilot Study Investigators. Early Experience With New Transcatheter Mitral Valve Replacement. J Am Coll Cardiol. 2018 Jan 2;71(1):12-21. doi: 10.1016/j.jacc.2017.10.061. Epub 2017 Nov 16.

family: Bapat

full_name: Vinayak Bapat

given: Vinayak

family: Rajagopal

full_name: Vivek Rajagopal

given: Vivek

family: Meduri

full_name: Christopher Meduri

given: Christopher

family: Farivar

full_name: R. Saeid Farivar

given: R. Saeid

family: Walton

full_name: Antony Walton

given: Antony

family: Duffy

full_name: Stephen J. Duffy

given: Stephen J.

family: Gooley

full_name: Robert Gooley

given: Robert

family: Almeida

full_name: Aubrey Almeida

given: Aubrey

family: Reardon

full_name: Michael J. Reardon

given: Michael J.

family: Kleiman

full_name: Neal S. Kleiman

given: Neal S.

family: Spargias

full_name: Konstantinos Spargias

given: Konstantinos

family: Pattakos

full_name: Stratis Pattakos

given: Stratis

family: Ng

full_name: Martin K. Ng

given: Martin K.

family: Wilson

full_name: Michael Wilson

given: Michael

family: Adams

full_name: David H. Adams

given: David H.

family: Leon

full_name: Martin Leon

given: Martin

family: Mack

full_name: Michael J. Mack

family: Chenoweth

full_name: Sharla Chenoweth

given: Sharla

family: Sorajja

full_name: Paul Sorajja

given: Paul

full_name: Michael Reardon

family: Bajwa

full_name: Tanvir Bajwa

given: Tanvir

family: O’Hair

full_name: Daniel O’Hair

journal_name: Journal of the American College of Cardiology

location_country:
Finland
France
Greece
Australia
Denmark
United States
United Kingdom

location_facility: Oregon Health Sciences University Hospital


type: Other Identifier

title: A PHASE 1, OPEN-LABEL, DOSE ESCALATION STUDY OF PF-04518600 AS A SINGLE AGENT AND IN COMBINATION WITH PF-05082566 IN PATIENTS WITH SELECTED LOCALLY ADVANCED OR METASTATIC CANCERS

summary: To assess the safety and tolerability at increasing dose levels of PF-04518600 alone or in combination wtih PF-05082566 in patients with select advanced or metastatic carcinoma in order to determine the maximum tolerated dose and select the recommended Phase 2 dose.

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

lead_sponsor: Pfizer

location_country:
United States
France
Japan
Netherlands

location_facility: Santa Monica UCLA Hematology & Oncology Clinic

location_facility: Mass General/North Shore Center for Outpatient Care

location_facility: Columbia University Medical Center - Herbert Irving Pavilion

location_facility: Beth Israel Deaconess Medical Center (BIDMC)

location_facility: Massachusetts General/Chelsea HealthCare Center

location_facility: The University of Texas MD Anderson Cancer Center

location_facility: Medical Imaging Center of Southern California, Inc.

location_facility: Slotervaart Hospital/Antoni van Leeuwenhoek

location_facility: The Angeles Clinic And Research Institute, A Cedars-Sinai Affiliate

location_facility: UConn Health, Neag Comprehensive Cancer Center

location_facility: H. Lee Moffitt Cancer Center & Research Institute


title: A Phase 4, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Effect of Obeticholic Acid on Clinical Outcomes in Patients With Primary Biliary Cholangitis

acronym: COBALT

summary: Primary Biliary Cholangitis (PBC) is a serious, life-threatening, bile acid related liver disease of unknown cause. Without treatment, it frequently progresses to liver fibrosis and eventual cirrhosis requiring liver transplantation or resulting in death. The investigational drug, Obeticholic Acid (OCA) is a modified bile acid and FXR agonist that is derived from the primary human bile acid chenodeoxycholic acid. The key mechanisms of action of OCA, including its choleretic, anti-inflammatory, and anti-fibrotic properties, underlie its hepatoprotective effects and result in attenuation of injury and improved liver function in a cholestatic liver disease such as PBC. The study will assess the effect of OCA compared to placebo, combined with stable standard care, on clinical outcomes in PBC patients.

ReferenceCitation: Lindor KD, Gershwin ME, Poupon R, Kaplan M, Bergasa NV, Heathcote EJ; American Association for Study of Liver Diseases. Primary biliary cirrhosis. Hepatology. 2009 Jul;50(1):291-308. doi: 10.1002/hep.22906.

ReferenceCitation: European Association for the Study of the Liver. EASL Clinical Practice Guidelines: management of cholestatic liver diseases. J Hepatol. 2009 Aug;51(2):237-67. doi: 10.1016/j.jhep.2009.04.009. Epub 2009 Jun 6.

lead_sponsor: Intercept Pharmaceuticals

location_country:
Finland
Australia
Belgium
Denmark
France
Poland
Lithuania
Argentina
Canada
Korea, Republic of
Turkey
Chile
Sweden
Hong Kong
Netherlands
Switzerland
Hungary
Germany
United States
United Kingdom
Italy
Austria
Brazil
Mexico
Israel
Spain
Estonia

location_facility: University of Colorado Denver and Hospital

location_facility: Saint Louis University Gastroenterology & Hepatology

location_facility: Aarhus University Hospital Department of Hepatology and Gastroenterology

location_facility: Klinikum der Johann-Wolfgang Goethe, Universitaet Frankfurt am Main

location_facility: University of Louisville, Medical Dental Complex

location_facility: UT Health The University of Texas Health Science Center at Houston

location_facility: University Health Network, Toronto General Hospital

location_facility: Azienda Ospedaliero Universitaria Careggi Universita di Firenze

location_facility: Ankara University School of Medicine Gastroenterology Dept.

location_facility: Kantonsspital St. Gallen, Clinic for Gastroeterologie and Hepatologie

location_facility: University of Alberta, Walter C. Mackenzie Health Sciences Centre (WMC)

location_facility: Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo-HCFMUSP

location_facility: The Liver Institute at Methodist Dallas Medical Center

location_facility: Azienda Ospedaliero - Universita di Padova

location_facility: Plymouth Hospitals NHS Trust, Derriford Hospital

location_facility: Medical University of Warsaw Samodzielny Publiczny Centralny Szpital Kliniczny w Warszawie (SPCSK in Warsaw

location_facility: Universität Leipzig KöR, Medizinische Fakultät

location_facility: AOU Ospedale Civile S. Agostino Estense - UO Medicina Metabolica

location_facility: Hospital Universitario Professor Edgard Santos

location_facility: Gastrocentro. Unidad Estadual de CAmpinas UNICAMP

location_facility: Schiff Center for Liver Diseases/University of Miami

location_facility: Azienda Ospedaliero - Universitaria di Cagliari - Centro per lo Studio delle Malattie del Fegato

location_facility: Békés Megyei Kozponti Korhaz - Dr. Rethy Pal Tagkorhaz

location_facility: Hospital of the University of Pennsylvania

location_facility: Azienda Socio Sanitaria Territoriale (ASST) di Monza

location_facility: Hospital das Clinicas da Universidade Federal de Minas Gerais (UFMG)

location_facility: Universidade Federal do Estado do Rio de Janeiro - Hospital Universitario Gaffree e Guinle/HUGG/UNIRIO

location_facility: AOU Policlinico Paolo Giaccone - Di.Bi.M.I.S

location_facility: Cambrigde University Hospitals NHS Foundation Trust

location_facility: Vrije Universiteit Medisch Centrum (VUMC)

location_facility: Hospital Privado Universitario de Cordoba S.A.

location_facility: Instituto Hospital de Base do Distrito Federal-IHBDF

location_facility: East Tallinn Central Hospital Gastroenterology Center

location_facility: Azienda Socio-Sanitarla Territoriale (ASST) Santi Paolo e Carlo

location_facility: Azienda Ospedaliero Universitaria Sant´ Orsola Malpighi

location_facility: Ege University School of Medicine Gastroenterology Dept.

location_facility: Turku University Central Hospital, Gastroenterology Outpatient Clinic

location_facility: Gastrocentro/ Universidade Estadual de Campinas (UNICAMP)

location_facility: Università Politecnica delle Marche - Azienda Ospedaliero

location_facility: Department of Gastroenterology & Hepatology, Nepean Hospital

location_facility: Seoul National University Bundang Hospital

location_facility: Inselspital, University Of Bern, UVCM, DMLL

location_facility: University Of Debrecen, Department Of Medicine, Division Of Gastroenterology

location_facility: Oddział Gastr. I Hepat. Uniwersyteckie Centrum Kliniczne Im. Prof. K. Gibińskiego Sum

location_facility: Odense University Hospital Afdeling for medicinske mave-tarmsygdomme S

location_facility: University Hospitals Birmingham NHS Foundation Trust

location_facility: Vilnius University Hospital Santaros klinikos

location_facility: University Hospitals Bristol NHS Foundation Trust

location_facility: Medizinische Hochschule Hannover, Klinik für Gastroenterologie, Hepatologie und Endokrinologie

location_facility: Fiona Stanley Hospital, Gastroenterology Department

location_facility: University of California Davis, Davis Medical Center

location_facility: Hospital de Gastroenterologia Dr. Carlos Bonorino Udaondo

location_facility: Southern Therapy and Advanced Research (STAR)

location_facility: Centrum Onkologii - Instytut im. Marii Skłodowskiej - Curie, Klinika Gastroenterologii Onkologicznej

location_facility: AW Morrow Gastroenterology and Liver Centre, Royal Prince Alfred Hospital

location_facility: Medicinsk klinik for mave, tarm-og leversygdomme

location_facility: Centro De Investigaciones Clínicas Viña Del Mar

location_facility: Amsterdam University Medical Centre (AMC)

location_facility: Hadassah Hebrew University Medical Center

location_facility: Policlinico Universitario Agostino Gemelli - Universita Cattolica del Sacro Cuore

location_facility: Department of Gastroenterology and Hepatology, Royal Adelaide Hospital

location_facility: American Research Corporation at Texas Liver Institute

location_facility: Wojewodzki Szpital Specjalistyczny im. J. Gromkowskiego, Pierwszy Oddzial Chorob Zakaznych

location_facility: University of Calgary Liver Unit (Heritage Medical Research Clinic)

location_facility: USZ, Klinik für Gastroenterologie und Hepatologie

location_facility: Hospital of Lithuanian University of Health Sciences, Kauno klinikos


title: Long-term Follow-up at 10 Years of Patients Enrolled in the Fingolimod Phase II Program in Relapsing Multiple Sclerosis (MS)

acronym: ACROSS

summary: This study collected follow-up data on approximately 90% of participants who were randomized and received one dose of study drug in FTY720D2201 (D2201). No study drug was given or required. Participants were required to be assessed at one or two visits, preferably at the original study site, but the option to be interviewed via phone or seen at home was provided. Information was gathered also on deceased participants. Assessments were performed only once within an 8 week period and included medical history, Multiple Sclerosis (MS) and Multiple Sclerosis Disease Modifying Therapy (MS DMT) history, Expanded Disability Status Scale (EDSS), Magnetic Resonance Imaging (MRI), and Multiple Sclerosis Functional Composite (MSFC).

location_country:
Italy
Switzerland
Spain
Germany
France
Poland
Portugal
Denmark
Canada
United Kingdom

intervention_type: Other


title: Attain Performa™ Left Ventricular Quadripolar Lead Post-Approval Study

summary: Lead survivability will be summarized.

study_type: Observational

time_perspective: Prospective

lead_sponsor: Medtronic

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

location_facility:
Hattiesburg Clinic
St Luke's University Health Network


ReferenceCitation: Pournara E, Kormaksson M, Nash P, Ritchlin CT, Kirkham BW, Ligozio G, Pricop L, Ogdie A, Coates LC, Schett G, McInnes IB. Clinically relevant patient clusters identified by machine learning from the clinical development programme of secukinumab in psoriatic arthritis. RMD Open. 2021 Nov;7(3). pii: e001845. doi: 10.1136/rmdopen-2021-001845.

family: Pournara

full_name: Effie Pournara

given: Effie

family: Kormaksson

full_name: Matthias Kormaksson

given: Matthias

family: Nash

full_name: Peter Nash

given: Peter

family: Ritchlin

full_name: Christopher T Ritchlin

given: Christopher T

family: Kirkham

full_name: Bruce W Kirkham

given: Bruce W

family: Ligozio

full_name: Gregory Ligozio

given: Gregory

family: Pricop

full_name: Luminita Pricop

given: Luminita

family: Ogdie

full_name: Alexis Ogdie

given: Alexis

family: Coates

full_name: Laura C Coates

given: Laura C

family: Schett

full_name: Georg Schett

given: Georg

family: McInnes

full_name: Iain B McInnes

given: Iain B

currency_apc_doaj: GBP

journal_name: RMD Open

evidence: oa journal (via doaj)

license: cc-by-nc

license_normalized: cc-by-nc

publisher: BMJ

publisher_dissemination: British Medical Journal

publisher_group: British Medical Journal

publisher_normalized: British Medical Journal

ReferenceCitation: Kivitz AJ, Nash P, Tahir H, Everding A, Mann H, Kaszuba A, Pellet P, Widmer A, Pricop L, Abrams K. Efficacy and Safety of Subcutaneous Secukinumab 150 mg with or Without Loading Regimen in Psoriatic Arthritis: Results from the FUTURE 4 Study. Rheumatol Ther. 2019 Sep;6(3):393-407. doi: 10.1007/s40744-019-0163-5. Epub 2019 Jun 21.

family: Kivitz

full_name: Alan J. Kivitz

given: Alan J.

family: Tahir

full_name: Hasan Tahir

given: Hasan

family: Everding

full_name: Andrea Everding

given: Andrea

family: Mann

full_name: Heřman Mann

given: Heřman

family: Kaszuba

full_name: Andrzej Kaszuba

given: Andrzej

family: Pellet

full_name: Pascale Pellet

given: Pascale

family: Widmer

full_name: Albert Widmer

given: Albert

family: Abrams

full_name: Ken Abrams

location_country:
Italy
United States
Sweden
Germany
France
Bulgaria
Czechia
Poland
Belgium
Australia
Russian Federation
Canada
United Kingdom

title: A Phase III, Randomized, Double-blind, Placebo-controlled Multicenter Study of Subcutaneous Secukinumab (150 mg) in Pre-filled Syringe, With or Without Loading Regimen, to Demonstrate Efficacy, Safety and Tolerability up to 2 Years in Patients With Active Psoriatic Arthritis (FUTURE 4)

summary: The purpose of this study was to provide 16-week efficacy, safety and tolerability data versus placebo to support the use of secukinumab 150 mg by subcutaneous (s.c.) self-administration with or without a loading regimen and maintenance dosing using pre-filled syringe (PFS) and to assess efficacy, safety and tolerability up to 2 years in subjects with active PsA despite current or previous NSAID or DMARD therapy


title: PV-10 Intralesional Injection vs Systemic Chemotherapy or Oncolytic Viral Therapy for Treatment of Locally Advanced Cutaneous Melanoma

summary: This is an international multicenter, open-label, randomized controlled trial (RCT) of single-agent intralesional PV-10 versus systemic chemotherapy or intralesional oncolytic viral therapy to assess treatment of locally advanced cutaneous melanoma in patients who (1) are not candidates for targeted therapy and (2) are not candidates for an immune checkpoint inhibitor. Subjects in the comparator arm will receive the Investigator's choice of dacarbazine (DTIC), temozolomide (TMZ) or intralesional talimogene laherparepvec as determined by Investigator preference and standard of care in the Investigator's country or region. Effectiveness will be assessed by comparison of progression-free survival (PFS) between all intent-to-treat (ITT) subjects in the two study treatment arms.

lead_sponsor: Provectus Biopharmaceuticals, Inc.

location_country:
Italy
United States
Mexico
France
Germany

location_facility: Azienda Sanitaria Azienda Ospedaliera Universitaria Senese

location_facility: Oklahoma Cancer Specialists and Research Institute

location_facility: Istituto Dermopatico dell'Immacolata (IDI IRCCS)

location_facility: Centro de Estudios y Prevención del Cancer A.C.

location_facility: Washington University School of Medicine - Dermatology

location_facility: Klinik für Dermatologie, Venerologie und Allergologie Charite Universitätsmedizin Berlin

location_facility: Moffitt Cancer Center and Research Institute

location_facility: Unité Cancéro-Dermatologie, Hôtel-Dieu CHU Nantes

location_facility: Sharp Memorial Hospital - Clinical Oncology Research

location_facility: Klinik für Dermatologie Universitätsklinikum Essen Studienzentrum

location_facility: Klinik für Dermatologie, Venerologie und Allergologie Universitätsklinikum Schleswig-Holstein Hautkrebszentrum

location_facility: IRCCS Instituto Nazionale Tumori "Fondazione Giovanni Pascale"

location_facility: Hautklinik Klinikum der Johannes Gutenberg Universität Hautkrebszentrum

location_facility: St Luke's University Hospital and Health Network

status_simplified: Unknown


title: A Phase Ib/II Multi-center, Open Label, Dose Escalation Study of WNT974, LGX818 and Cetuximab in Patients With BRAFV600-mutant KRAS Wild-type Metastatic Colorectal Cancer Harboring Wnt Pathway Mutations

summary:
The purpose of this study is to assess the safety and anti-tumor activity of the triple combination of WNT974, LGX818 and cetuximab in BRAFV600-mutant mCRC with RNF43 mutations or RSPO fusions.

The design of this study is based upon the translational and pre-clinical data that suggest that Wnt pathway signals, increased due to RNF43 mutations or RSPO fusions, cooperate with the EGFR and BRAF signals to maintain the growth of BRAFV600 CRCs. Inhibition of these signals with the triple combination of WNT974, LGX818 and cetuximab may result in anti-tumor activity.

lead_sponsor: Array BioPharma

location_country:
Italy
United States
Netherlands
France
Israel
Singapore
Belgium
Australia
Spain
Canada

location_facility: University of Wisconsin / Paul P. Carbone Comp Cancer Center Univ Wisc

location_facility: Medical University of South Carolina Oncology Dept

location_facility: Memorial Sloan-Kettering Cancer Center (MSKCC) MSKCC (3)

location_facility: University of Texas/MD Anderson Cancer Center Onc. Dept,


title: A Phase 1/2, Multicenter, Open-Label, Dose-Escalation Study of AG-221 in Subjects With Advanced Solid Tumors, Including Glioma, and With Angioimmunoblastic T-cell Lymphoma, That Harbor an IDH2 Mutation

summary: The purpose of this study is to evaluate the safety, pharmacokinetics, and clinical activity of enasidenib in adults with advanced solid tumors, including glioma, or with angioimmunoblastic T-cell lymphoma (AITL), with an isocitrate dehydrogenase-2 (IDH2) mutation.

location_country:
United States
France


title: ImThera Medical Targeted Hypoglossal Neurostimulation Study #3

summary: The purpose of this study is to evaluate the benefits and risks of hypoglossal nerve stimulation with the ImThera Medical aura6000 System as a potential therapeutic option for individuals with moderate to severe OSA that have failed or do not tolerate PAP.

lead_sponsor: ImThera Medical, Inc.

location_country:
United States
France
Israel
Belgium
Portugal
Germany

location_facility: Ear, Nose, and Throat Associates of South Florida

intervention_type: Device


location_country:
Austria
Hong Kong
Netherlands
Switzerland
Germany
Finland
France
Czechia
Korea, Republic of
Israel
Poland
Norway
Ireland
Singapore
Spain
Denmark
United States
United Kingdom
Canada
Taiwan

title: A Phase II Randomized, Double-blind, Placebo-controlled Trial of Radium-223 Dichloride Versus Placebo When Administered to Metastatic HER2 Negative Hormone Receptor Positive Breast Cancer Subjects With Bone Metastases Treated With Hormonal Treatment Background Therapy

summary: The objective of this study was to assess efficacy and safety of radium-223 dichloride in subjects with human epidermal growth factor receptor 2 negative (HER2 negative) hormone receptor positive breast cancer with bone metastases treated with hormonal treatment background therapy


title: A Multicenter, Randomized, Double Blind, Placebo Controlled, Multiple Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RO6885247 Following 12 Weeks of Treatment in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH).

summary: This multicenter, randomized, double-blind, 12-week, placebo-controlled multiple dose study will investigate the safety and tolerability of RO6885247 in adult and pediatric patients with spinal muscular atrophy (SMA).

location_country:
Italy
Sweden
Switzerland
Netherlands
France
Turkey
United States
United Kingdom
Canada

location_facility: Fondazione IRCCS Istituto Neurologico "Carlo Besta"; UO di Neurologia dello Sviluppo

location_facility: MRC Neuromuscular Centre - Institute of Genetic Medicine

location_facility: Ch Pitie Salpetriere; Institut de Myologie

location_facility: Universitäts-Kinderspitalbeider Basel_Abteilung für Neuro- und Entwicklungspädiatrie

location_facility: UCL; GAP Unit, Institute of Child Health, UCL

location_facility: Drottning Silvias Barn- och ungdomssjukhus; Kliniken för barnmedicin

location_facility: UMC Utrecht; Polkliniek Neuromusculaire ziekten

location_facility: Policlinico Agostino Gemelli; Dipartimento di Neuropsichiatria Infantile

location_facility: Hacettepe University, School of Medicine; Pediatrics Department; Pediatrics Child Neurology Unit


ReferenceCitation: Nolan B, Klukowska A, Shapiro A, Rauch A, Recht M, Ragni M, Curtin J, Gunawardena S, Mukhopadhyay S, Jayawardene D, Winding B, Fischer K, Liesner R. Final results of the PUPs B-LONG study: evaluating safety and efficacy of rFIXFc in previously untreated patients with hemophilia B. Blood Adv. 2021 Jul 13;5(13):2732-2739. doi: 10.1182/bloodadvances.2020004085.

name: Children’s Health Ireland at Crumlin, Dublin, Ireland;

family: Nolan

full_name: Beatrice Nolan

given: Beatrice

name: Department of Pediatrics, Hematology and Oncology, Medical University of Warsaw, Warsaw, Poland;

family: Klukowska

full_name: Anna Klukowska

given: Anna

name: Indiana Hemophilia and Thrombosis Center, Indianapolis, IN;

family: Shapiro

full_name: Amy Shapiro

given: Amy

name: Department of Hematology and Transfusion, Centre Hospitalier Universitaire de Lille, Lille, France;

family: Rauch

full_name: Antoine Rauch

given: Antoine

name: Division of Hematology and Oncology, Department of Pediatrics, Oregon Health & Science University, Portland, OR;

family: Recht

full_name: Michael Recht

given: Michael

name: University of Pittsburgh Medical Center, Pittsburgh, PA;

family: Ragni

full_name: Margaret Ragni

given: Margaret

name: The Children’s Hospital at Westmead, Westmead, NSW, Australia;

family: Curtin

full_name: Julie Curtin

given: Julie

family: Gunawardena

full_name: Sriya Gunawardena

given: Sriya

family: Mukhopadhyay

full_name: Sutirtha Mukhopadhyay

given: Sutirtha

family: Jayawardene

full_name: Deepthi Jayawardene

given: Deepthi

family: Winding

full_name: Bent Winding

given: Bent

name: University Medical Center Utrecht, Utrecht, The Netherlands; and

family: Fischer

full_name: Kathelijn Fischer

given: Kathelijn

name: Great Ormond Street Hospital, London, United Kingdom

family: Liesner

full_name: Raina Liesner

given: Raina

oa_colors: green gold

publisher: American Society of Hematology

publisher_dissemination: American Society of Hematology

publisher_group: American Society of Hematology

publisher_normalized: American Society of Hematology

intervention_type: Biological

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

lead_sponsor: Bioverativ, a Sanofi company

location_country:
Italy
Sweden
Netherlands
France
Poland
Ireland
Australia
New Zealand
Denmark
United States
United Kingdom

acronym: PUPs B-LONG

location_facility:
Hopital Cardiologique - CHU Lille
Research Site

title: An Open-Label, Multicenter Evaluation of the Safety and Efficacy of Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc; BIIB029) in the Prevention and Treatment of Bleeding in Previously Untreated Patients With Severe Hemophilia B

summary: The primary objective of the study was to evaluate the safety of recombinant coagulation factor IX Fc fusion protein (rFIXFc, BIIB029) in previously untreated patients (PUPs) with severe hemophilia B. Secondary objectives were to evaluate the efficacy of rFIXFc in the prevention and treatment of bleeding episodes in PUPs, and to evaluate rFIXFc consumption for prevention and treatment of bleeding episodes in PUPs.


location_country:
Peru
Guatemala
Finland
Malaysia
Romania
Belgium
Russian Federation
Denmark
Taiwan
India
France
Panama
Lithuania
Poland
Slovakia
Thailand
Argentina
Canada
Korea, Republic of
Turkey
South Africa
Chile
Sweden
Hong Kong
Netherlands
Philippines
Bulgaria
Singapore
Hungary
Portugal
Germany
United States
United Kingdom
Italy
Brazil
Mexico
Czechia
Israel
Spain
Estonia
Latvia

title: A Multicenter Study to Evaluate Safety and Tolerability in Patients With Chronic Heart Failure and Reduced Ejection Fraction From PARADIGM-HF Receiving Open Label LCZ696

summary: The purpose of this study was to collect safety and tolerability data on LCZ696 in eligible PARADIGM-HF patients who received open-label investigational drug. The parent PARADIGM-HF (NCT01035255) trial was terminated early due to compelling efficacy of LCZ696 in patients with heart failure with reduced ejection fraction (HFrEF) after the final pre-specified interim analysis in March 2014.