title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of ISIS 304801 Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)

summary: The purpose of this study is to evaluate the efficacy and safety of volanesorsen given for 52 weeks in participants with Familial Chylomicronemia Syndrome

ReferenceCitation: Witztum JL, Gaudet D, Freedman SD, Alexander VJ, Digenio A, Williams KR, Yang Q, Hughes SG, Geary RS, Arca M, Stroes ESG, Bergeron J, Soran H, Civeira F, Hemphill L, Tsimikas S, Blom DJ, O'Dea L, Bruckert E. Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome. N Engl J Med. 2019 Aug 8;381(6):531-542. doi: 10.1056/NEJMoa1715944. + Collaborators/Trial investigators: Alvarez-Sala L, Averna M, Ballantyne C, Baum S, Corder C, Diaz Diaz J, DuFour R, Dunbar R, Francis G, Gavish D , Goldberg I, Issa B, Izar M, Jones A, Julius A, Kane J, Krempf M, Lovell C, Martin S, Moriarty O, Muniz O, Neely R, Osamah H, Pauker Z, Quigley J, Rados D , Roeters van Lenneo J, Rosenblit R, Santos R, Soran H, Stefanutti C, Steinhagen-Thiessen E, Subramanian S, Valdivielso P, Valero R.

name: From the Department of Medicine, University of California San Diego, La Jolla (J.L.W., S.T.), and Ionis Pharmaceuticals, Carlsbad (V.J.A., Q.Y., S.G.H., R.S.G., S.T.) — both in California; the Department of Medicine, Université de Montréal and ECOGENE 21, Chicoutimi, QC (D.G.), and the Department of Medicine and Laboratory Medicine, Centre Hospitalier Universitaire de Québec–University Laval, Quebec, QC (J.B.) — both in Canada; the Department of Medicine, Beth Israel Deaconess Medical Center (S.D.F.),...

family: Witztum

full_name: Joseph L. Witztum

given: Joseph L.

family: Gaudet

full_name: Daniel Gaudet

given: Daniel

family: Freedman

full_name: Steven D. Freedman

given: Steven D.

family: Alexander

full_name: Veronica J. Alexander

given: Veronica J.

family: Digenio

full_name: Andres Digenio

given: Andres

family: Williams

full_name: Karren R. Williams

given: Karren R.

family: Yang

full_name: Qingqing Yang

given: Qingqing

family: Hughes

full_name: Steven G. Hughes

given: Steven G.

family: Geary

full_name: Richard S. Geary

given: Richard S.

family: Arca

full_name: Marcello Arca

given: Marcello

family: Stroes

full_name: Erik S.G. Stroes

given: Erik S.G.

family: Bergeron

full_name: Jean Bergeron

given: Jean

family: Soran

full_name: Handrean Soran

given: Handrean

family: Civeira

full_name: Fernando Civeira

given: Fernando

family: Hemphill

full_name: Linda Hemphill

given: Linda

family: Tsimikas

full_name: Sotirios Tsimikas

given: Sotirios

family: Blom

full_name: Dirk J. Blom

given: Dirk J.

family: O’Dea

full_name: Louis O’Dea

given: Louis

family: Bruckert

full_name: Eric Bruckert

given: Eric

journal_name: New England Journal of Medicine

oa_colors: green other

evidence: open (via free article)

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

repository_institution: University of Zaragoza Repository - Zaguan

publisher: Massachusetts Medical Society

ReferenceCitation: Hegele RA, Berberich AJ, Ban MR, Wang J, Digenio A, Alexander VJ, D'Erasmo L, Arca M, Jones A, Bruckert E, Stroes ES, Bergeron J, Civeira F, Witztum JL, Gaudet D. Clinical and biochemical features of different molecular etiologies of familial chylomicronemia. J Clin Lipidol. 2018 Jul - Aug;12(4):920-927.e4. doi: 10.1016/j.jacl.2018.03.093. Epub 2018 Apr 4.

ReferenceCitation: Digenio A, Dunbar RL, Alexander VJ, Hompesch M, Morrow L, Lee RG, Graham MJ, Hughes SG, Yu R, Singleton W, Baker BF, Bhanot S, Crooke RM. Antisense-Mediated Lowering of Plasma Apolipoprotein C-III by Volanesorsen Improves Dyslipidemia and Insulin Sensitivity in Type 2 Diabetes. Diabetes Care. 2016 Aug;39(8):1408-15. doi: 10.2337/dc16-0126. Epub 2016 Jun 6.

location_country:
Italy
Brazil
South Africa
Netherlands
Germany
France
Israel
Hungary
Spain
United States
United Kingdom
Canada


type: EudraCT Number

title: A Phase 1b Study of ABT-199 (GDC-0199) in Combination With Azacitidine or Decitabine in Treatment-Naive Subjects With Acute Myelogenous Leukemia Who Are Greater Than or Equal to 60 Years of Age and Who Are Not Eligible for Standard Induction Therapy

summary: This is a Phase 1b, open-label, non-randomized, multicenter study to evaluate the safety and pharmacokinetics of orally administered venetoclax (ABT-199) combined with decitabine or azacitidine and the preliminary efficacy of these combinations. In addition, there is a drug-drug interaction (DDI) sub-study only at a single site, to assess the pharmacokinetics and safety of venetoclax (ABT-199) in combination with posaconazole.

study_start_date_type: Actual

status: Active, not recruiting

study_completion_date_type: Anticipated

study_type: Interventional

design_allocation: Non-Randomized

primary_purpose: Treatment

enrollment_type: Actual

ReferenceType: derived

ReferenceCitation: DiNardo CD, Pratz K, Pullarkat V, Jonas BA, Arellano M, Becker PS, Frankfurt O, Konopleva M, Wei AH, Kantarjian HM, Xu T, Hong WJ, Chyla B, Potluri J, Pollyea DA, Letai A. Venetoclax combined with decitabine or azacitidine in treatment-naive, elderly patients with acute myeloid leukemia. Blood. 2019 Jan 3;133(1):7-17. doi: 10.1182/blood-2018-08-868752. Epub 2018 Oct 25.

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

family: DiNardo

full_name: Courtney D. DiNardo

given: Courtney D.

sequence: first

name: The Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University, Baltimore, MD;

family: Pratz

full_name: Keith Pratz

given: Keith

sequence: additional

name: Department of Hematology and Hematopoietic Cell Transplantation and

name: Gehr Family Center for Leukemia Research, City of Hope National Medical Center, Duarte, CA;

family: Pullarkat

full_name: Vinod Pullarkat

given: Vinod

name: University of California Davis Comprehensive Cancer Center, Sacramento, CA;

family: Jonas

full_name: Brian A. Jonas

given: Brian A.

name: Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta, GA;

family: Arellano

full_name: Martha Arellano

given: Martha

name: Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA;

name: Division of Hematology, Department of Medicine, University of Washington School of Medicine, Seattle, WA;

family: Becker

full_name: Pamela S. Becker

given: Pamela S.

name: Northwestern University Feinberg School of Medicine, Chicago, IL;

family: Frankfurt

full_name: Olga Frankfurt

given: Olga

family: Konopleva

full_name: Marina Konopleva

given: Marina

name: The Alfred Hospital and Monash University, Melbourne, Australia;

family: Wei

full_name: Andrew H. Wei

given: Andrew H.

family: Kantarjian

full_name: Hagop M. Kantarjian

given: Hagop M.

family: Xu

full_name: Tu Xu

given: Tu

family: Hong

full_name: Wan-Jen Hong

given: Wan-Jen

family: Chyla

full_name: Brenda Chyla

given: Brenda

family: Potluri

full_name: Jalaja Potluri

given: Jalaja

name: Blood Cancer and Bone Marrow Transplant Program, University of Colorado School of Medicine, Aurora, CO; and

family: Pollyea

full_name: Daniel A. Pollyea

given: Daniel A.

name: Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA

family: Letai

full_name: Anthony Letai

given: Anthony

bso_classification: Medical research

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: Blood

oa_colors: green hybrid

oa_host_type: publisher;repository

host_type: publisher

license_normalized: no license

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 pmcid lookup)

publisher: American Society of Hematology

publisher_dissemination: American Society of Hematology

publisher_group: American Society of Hematology

publisher_normalized: American Society of Hematology

ReferenceCitation: DiNardo CD, Pratz KW, Letai A, Jonas BA, Wei AH, Thirman M, Arellano M, Frattini MG, Kantarjian H, Popovic R, Chyla B, Xu T, Dunbar M, Agarwal SK, Humerickhouse R, Mabry M, Potluri J, Konopleva M, Pollyea DA. Safety and preliminary efficacy of venetoclax with decitabine or azacitidine in elderly patients with previously untreated acute myeloid leukaemia: a non-randomised, open-label, phase 1b study. Lancet Oncol. 2018 Feb;19(2):216-228. doi: 10.1016/S1470-2045(18)30010-X. Epub 2018 Jan 12.

ReferenceCitation: Agarwal SK, DiNardo CD, Potluri J, Dunbar M, Kantarjian HM, Humerickhouse RA, Wong SL, Menon RM, Konopleva MY, Salem AH. Management of Venetoclax-Posaconazole Interaction in Acute Myeloid Leukemia Patients: Evaluation of Dose Adjustments. Clin Ther. 2017 Feb;39(2):359-367. doi: 10.1016/j.clinthera.2017.01.003. Epub 2017 Feb 1.

location_facility: Universitaetsklinikum Carl Gustav Carus an der TU Dresden /ID# 130342

location_facility: Duplicate_Hopital Universitaire Purpan /ID# 134389

location_facility: University of California, Davis Comprehensive Cancer Center /ID# 129719

location_facility: Univ of Colorado Cancer Center /ID# 127859

location_facility: University of Texas MD Anderson Cancer Center /ID# 127860

location_facility: University of Texas MD Anderson Cancer Center /ID# 141581

location_facility: Northwestern University Feinberg School of Medicine /ID# 128741

location_facility: Duplicate_Klinikum Rechts der Isar /ID# 130347

location_facility: Winship Cancer Institute of Emory University /ID# 129715

location_facility: The University of Chicago Medical Center /ID# 128742

location_facility: Dana-Farber Cancer Institute /ID# 127857

location_facility: Duplicate_Hopital Haut-Lévêque /ID# 134388

location_facility: Columbia University Medical Center /ID# 130289

location_facility: Universitaetsklinikum Leipzig /ID# 130346


ReferenceCitation: Liu BP, Rosenberg M, Saverio P, Weon YC, Peters S, Ardellier FD, Boeckenhoff A, Endrikat J. Clinical Efficacy of Reduced-Dose Gadobutrol Versus Standard-Dose Gadoterate for Contrast-Enhanced MRI of the CNS: An International Multicenter Prospective Crossover Trial (LEADER-75). AJR Am J Roentgenol. 2021 Nov;217(5):1195-1205. doi: 10.2214/AJR.21.25924. Epub 2021 Jun 16.

name: Division of Neuroradiology, Departments of Radiology and Radiation Oncology, Feinberg School of Medicine of Northwestern University, Northwestern Memorial Hospital, Chicago, Illinois, USA

family: Liu

full_name: Benjamin P. Liu

given: Benjamin P.

name: Bayer Pharmaceuticals, Radiology, Whippany, New Jersey, USA

family: Rosenberg

full_name: Martin Rosenberg

given: Martin

name: Ospedale “L. Bonomo” – Andria, Viale Istria 1, Italy

family: Saverio

full_name: Pollice Saverio

given: Pollice

name: Department of Radiology, Ulsan University Hospital, University of Ulsan, Korea

family: Weon

full_name: Young Cheol Weon

given: Young Cheol

name: Department of Radiology and Neuroradiology, University Hospital of Schleswig-Holstein, Campus Kiel, Arnold-Heller-Straße 3; D-24105 Kiel, Germany

family: Peters

full_name: Soenke Peters

given: Soenke

name: Hôpitaux Universitaires de Strasbourg - Hôpital de Hautepierre, Avenue Molière, Strasbourg 67200, France

family: Ardellier

full_name: François-Daniel Ardellier

given: François-Daniel

name: Bayer AG, Statistics and Data Insights, D-42113 Wuppertal, Germany

family: Boeckenhoff

full_name: Annette Boeckenhoff

given: Annette

name: Bayer AG, Radiology, D-13353 Berlin, Germany and University Medical School of Saarland, Dept of Gynecology, Obstetrics and Reproductive Medicine, D-66421 Homburg/Saar, Germany

family: Endrikat

full_name: Jan Endrikat

given: Jan

journal_name: American Journal of Roentgenology

publisher: American Roentgen Ray Society

publisher_dissemination: American Roentgen Ray Society

publisher_group: American Roentgen Ray Society

publisher_normalized: American Roentgen Ray Society

title: Clinical Efficacy of Reduced Dose Gadobutrol Versus Standard Dose Gadoterate for Contrast-Enhanced MRI of the CNS: An International Multicenter Prospective Crossover Trial (LEADER-75)

intervention_type: Drug

status: Completed

lead_sponsor: Bayer

primary_purpose: Diagnostic

location_country:
Italy
United States
Switzerland
France
Germany
United Kingdom
Korea, Republic of

acronym: LEADER 75

location_facility: Penn State Milton S. Hershey Medical Center

location_facility: Universität Rostock - Medizinische Fakultät

location_facility: Universitätsklinikum Schleswig-Holstein / AÖR

location_facility: University of Texas Southwestern Medical Center

study_completion_date_type: Actual

source: euctr

title: LowEr Administered Dose With highEr Relaxivity: Gadovist vs Dotarem (LEADER 75)

summary:
The study was conducted to gain knowledge about a new dose of a diagnostic drug that is used for contrast-enhanced Magnetic Resonance Imaging (MRI) of the human central nervous system (CNS). MRI can visualize the anatomy of the body and is used to detect medical conditions. Diagnostic drugs like gadobutrol and gadoterate contain an element called gadolinium that is applied to improve the analysability of MRI-images.

The purpose of this study was to examine if contrast-enhanced MRI using a reduced dose of the gadolinium-based contrast agent gadobutrol delivers images of similar quality to those obtained when a full dose of the gadolinium-based contrast agent gadoterate was used.

lead_sponsor_type: industriel

status_simplified: Completed


title: 10-day Decitabine Versus Conventional Chemotherapy ("3+7") Followed by Allografting in AML Patients ≥ 60 Years: a Randomized Phase III Study of the EORTC Leukemia Group, CELG, GIMEMA and German MDS Study Group

summary:
Older patients with acute myeloid leukemia (AML) have a small (< 10%) chance of long-term survival. Despite the treatment of elderly AML patients with intensive chemotherapy, the survival has not been improved during the last decades.

The purpose of this study is to determine whether frontline therapy with a 10-day decitabine schedule provides a better survival than standard intensive combination chemotherapy in elderly AML patients (>= 60 years).

design_allocation: Randomized

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

location_country: Italy Netherlands Croatia Bulgaria France Lithuania Belgium Portugal Germany

location_facility: Universitätsklinikum Aachen AÖR - Medizinische Fakultät der RWTH

location_facility: Universita Degli Studi Di Bari - Policlinico

location_facility: Assistance Publique - Hôpitaux de Paris - Hôpital Saint Antoine

location_facility: Universitaetklinikum Halle - Martin Luther Universitaet - Universitaetsklinikum Halle (Saale)

location_facility: Azienda Ospedaliero - Universitaria Policlinico di Modena

location_facility: Helios Kliniken - Helios Klinikum Berlin-Buch

location_facility: Universitaet Rostock - Medizinische Fakultaet

location_facility: Universitaetsklinikum Tuebingen-Uni Kliniken Berg

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

location_facility: National Specialized Hospital for Active Treatment of Haematological Diseases

location_facility: Klinikum Der Phillips - Universität Marburg

location_facility: Amedeo Avogadro University of Eastern Piedmont-Ospedale Maggiore della Carita

location_facility: Azienda Ospedaliero-Universitaria "Santa Maria della Misericordia" di Udine

location_facility: AUSL Romagna - Ospedale Infermi di Rimini

location_facility: Radboud University Medical Center Nijmegen

location_facility: AUSL Romagna - Ospedale Santa Maria dell Croci

location_facility: Instituto Regina Elena / Instituti Fisioterapici Ospitalieri

location_facility: Vilnius University Hospital Santariskiu Clinics

location_facility: Azienda Ospedallera Universitaria - Policlinico Tor Vergata

location_facility: Clinica Ematologica dell'Universita di Roma La Sapienza

location_facility: Azienda Ospedaliera Città della Salute e della Scienza di Torino - Ospedale Molinette

location_facility: Azienda Ospedaliero Universitaria - Ospedali Riuniti

status_simplified: Ongoing


type: Other Identifier

type: Registry Identifier

title: An Open-label Single-arm Multicentre Non-controlled Phase 3 a Trial Investigating Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Prophylaxis and Treatment of Bleeding Episodes in Previously Untreated Patients With Haemophilia B (FIX Activity Below or Equal to 2 Percent)

summary: This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of nonacog beta pegol (N9-GP) in previously untreated patients with Haemophilia B.

enrollment_type: Anticipated

ReferenceType: background

ReferenceCitation: Chan AK, Alamelu J, Barnes C, Chuansumrit A, Garly ML, Meldgaard RM, Young G. Nonacog beta pegol (N9-GP) in hemophilia B: First report on safety and efficacy in previously untreated and minimally treated patients. Res Pract Thromb Haemost. 2020 Jul 29;4(7):1101-1113. doi: 10.1002/rth2.12412. eCollection 2020 Oct.

lead_sponsor: Novo Nordisk A/S

location_country:
Italy
Austria
Netherlands
Germany
France
Japan
Malaysia
Israel
Thailand
Australia
Argentina
Algeria
Spain
United States
United Kingdom
Canada
Taiwan


location_country:
Italy
Austria
Thailand
Germany
France
Bulgaria
Greece
Israel
Japan
Malaysia
Romania
Australia
Argentina
Algeria
Spain
United States
Canada
Taiwan

title: An Open-label Single-arm Multicentre Non-controlled Phase 3a Trial Investigating Safety and Efficacy of N8-GP in Prophylaxis and Treatment of Bleeding Episodes in Previously Untreated Paediatric Patients With Severe Haemophilia A

summary: This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of turoctocog alfa pegol (N8-GP) in previously untreated patients (PUPs) with haemophilia A.


title: A Randomized, Double-Blind, Phase 2 Study of Ruxolitinib or Placebo in Combination With Capecitabine in Subjects With Advanced or Metastatic HER2-Negative Breast Cancer

summary: This was a randomized, double-blind, placebo-controlled phase 2 clinical trial comparing the overall survival of women with advanced or metastatic HER2-negative breast cancer who received treatment with capecitabine in combination with ruxolitinib versus those who received treatment with capecitabine alone.

status: Terminated

lead_sponsor: Incyte Corporation

location_country:
Italy
France
Spain
United States
United Kingdom
Portugal

status_simplified: Unknown


title: A Randomized, Placebo-controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effects on Heterotopic Bone Formation of REGN2477 in Patients With Fibrodysplasia Ossificans Progressiva

summary:
This is a three period study design consisting of a 6-month, randomized, double-blind placebo-controlled treatment (period 1) followed by a 6-month, open-label treatment (period 2) and a follow-up treatment period (period 3).

Primary safety objective of the study is to assess the safety and tolerability of REGN2477 in male and female patients with fibrodysplasia ossificans progressiva (FOP).

Primary efficacy objective of the study is to assess the effect of REGN2477 versus placebo on the change from baseline in heterotopic ossification (HO) in patients with FOP, as determined by 18-NaF uptake in HO lesions by positron emission tomography (PET) and in total volume of HO lesions by computed tomography (CT).

Key Secondary objectives are:

To compare the effect of REGN2477 versus placebo on pain due to FOP, as measured by the area under the curve (AUC) for pain based on daily pain numeric rating scale (NRS) scores
To assess the effect of REGN2477 versus placebo on the change from baseline in HO, as determined by the number of new HO lesions identified by 18F-NaF PET or by CT
To assess the effect of REGN2477 versus placebo on the change from baseline in 18F-NaF standardized uptake value maximum (SUVmax) of individual active HO site(s) by PET
To assess the effect of REGN2477, between week 28 and week 56, on the number, activity, and volume of HO lesions identified by 18F-NaF PET or by CT in patients who switch from placebo to REGN2477 at week 28 versus the same patients between baseline and week 28
To assess the effect of REGN2477 versus placebo on the change from baseline in biochemical markers of bone formation
To characterize the concentrations of total activin A at baseline and over time following the first dose of study drug
To characterize the concentration-time profile (pharmacokinetics [PK]) of REGN2477 in patients with FOP
To assess the immunogenicity of REGN2477

ReferenceCitation: Vanhoutte F, Liang S, Ruddy M, Zhao A, Drewery T, Wang Y, DelGizzi R, Forleo-Neto E, Rajadhyaksha M, Herman G, Davis JD. Pharmacokinetics and Pharmacodynamics of Garetosmab (Anti-Activin A): Results From a First-in-Human Phase 1 Study. J Clin Pharmacol. 2020 Nov;60(11):1424-1431. doi: 10.1002/jcph.1638. Epub 2020 Jun 18.

name: SGS Clinical Pharmacology Unit  Antwerp Belgium

family: Vanhoutte

full_name: Frédéric Vanhoutte

given: Frédéric

name: Regeneron Pharmaceuticals, Inc.  Tarrytown New York USA

family: Liang

full_name: Su Liang

given: Su

family: Ruddy

full_name: Marcella Ruddy

given: Marcella

family: Zhao

full_name: An Zhao

given: An

family: Drewery

full_name: Tiera Drewery

given: Tiera

family: Wang

full_name: Yuhuan Wang

given: Yuhuan

family: DelGizzi

full_name: Richard DelGizzi

given: Richard

family: Forleo‐Neto

full_name: Eduardo Forleo‐Neto

given: Eduardo

family: Rajadhyaksha

full_name: Manoj Rajadhyaksha

given: Manoj

family: Herman

full_name: Gary Herman

given: Gary

family: Davis

full_name: John D. Davis

given: John D.

journal_name: The Journal of Clinical Pharmacology

evidence: open (via crossref license)

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

publisher: Wiley

publisher_dissemination: Wiley

publisher_group: Wiley

publisher_normalized: Wiley

title: Pharmacokinetics and Pharmacodynamics of Garetosmab (Anti‐Activin A): Results From a First‐in‐Human Phase 1 Study

ReferenceCitation: Aykul S, Corpina RA, Goebel EJ, Cunanan CJ, Dimitriou A, Kim HJ, Zhang Q, Rafique A, Leidich R, Wang X, McClain J, Jimenez J, Nannuru KC, Rothman NJ, Lees-Shepard JB, Martinez-Hackert E, Murphy AJ, Thompson TB, Economides AN, Idone V. Activin A forms a non-signaling complex with ACVR1 and type II Activin/BMP receptors via its finger 2 tip loop. Elife. 2020 Jun 9;9. pii: e54582. doi: 10.7554/eLife.54582.

name: Regeneron Pharmaceuticals, Tarrytown, United States

family: Aykul

full_name: Senem Aykul

given: Senem

family: Corpina

full_name: Richard A Corpina

given: Richard A

name: University of Cincinnati, Cincinnati, United States

family: Goebel

full_name: Erich J Goebel

given: Erich J

family: Cunanan

full_name: Camille J Cunanan

given: Camille J

family: Dimitriou

full_name: Alexandra Dimitriou

name: Michigan State University, East Lansing, United States

location_country:
Italy
Netherlands
France
Poland
Spain
United States
United Kingdom
Canada

location_facility: Royal National Orthopaedic Hospital, Brockley Hill

location_facility: Hopital Lariboisiere,Hospitalier Universitaire Nord


intervention_type: Biological

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: Italy Austria Germany France Japan Norway Belgium Spain Canada

acronym: Expanded treatment protocol for CTL019.

ipd_sharing: Yes

title: Phase IIIb Study for Relapsed/Refractory Pediatric/Young Adult Acute Lymphoblastic Leukemia Patients to be Treated With CTL019

summary: This is a single arm, open-label, multi-center, phase III B study to determine the safety and efficacy of CTL019 in pediatric/young adult patients with r/r B-cell Acute Lymphoblastic Leukemia (ALL).


title: A Real World Evaluation of the ELUVIA Drug Eluting Stent in All-Comers With Superficial Femoral Artery and Proximal Popliteal Artery Disease

acronym: REGAL

summary: The REGAL study is a European, prospective, multi-center Post-Market Clinical Follow-up (PMCF) trial providing additional data including health economics data to support the use of the ELUVIA stent in the treatment of lesions located in the femoropopliteal arteries.

study_type: Observational

time_perspective: Prospective

ipd_sharing: Undecided

lead_sponsor: Boston Scientific Corporation

location_country: Italy Austria France Belgium Spain

location_facility: Hospital Azienda Ospedaliera Universitaria Senese

location_facility: Centre Hospitalier Universitaire Grenoble-Alpes

location_facility: Hosp. Complejo Hosp Universitario (CHUO)

location_facility: Foundation for the Aragonese Healthcare Research Institute

intervention_type: Device


title: MERIT - A Phase II Marker Identification Trial for Tarceva in Second Line NSCLC Patients

acronym: MERIT

summary: This study will assess potentially predictive markers of efficacy in participants with NSCLC receiving oral erlotinib (Tarceva) therapy. The anticipated time on study treatment is until disease progression, unacceptable toxicity or death.

lead_sponsor: Hoffmann-La Roche

location_country:
Italy
Hong Kong
France
Bulgaria
Poland
Singapore
Ireland
Belgium
Russian Federation
Spain
Estonia
Germany
United Kingdom
Taiwan


title: A Phase I Dose Finding Study of Oral LXH254 in Adult Patients With Advanced Solid Tumors Harboring MAPK Pathway Alterations

summary: A Phase I Study of LXH254 in Patients With Advanced Solid Tumors That Harbor MAPK Pathway Alterations.

ipd_sharing: No

location_country:
Italy
United States
Switzerland
Netherlands
France
Japan
Korea, Republic of
Spain
Germany
Canada

location_facility: UT M.D Anderson Cancer Center SC - LXH254X2101

location_facility: Massachusetts General Hospital MGH Cancer Center

location_facility: Memorial Sloan Kettering Cancer Center SC - LXH254X2101


title: An Open-Label, Intrapatient Dose-Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients With Early Onset and Other Pediatric Onset Facioscapulohumeral Muscular Dystrophy

acronym: FSHD

summary: The purpose of this study is to assess the safety and biological activity of ATYR1940 in patients with early onset facioscapulohumeral muscular dystrophy (FSHD).

lead_sponsor: aTyr Pharma, Inc.

location_country:
Italy
United States
France

location_facility: Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta

location_facility: Centre d'nvestigation Clinique - Centre de Pharmacologie Clinique et d'Evaluations Thérapeutiques (CICCPCET)


title: A Phase I, Multicenter, Open-label, Single-sequence Drug-drug Interaction Study to Assess the Effect of INC280 on the Pharmacokinetics of Midazolam and Caffeine in Patients With cMET-dysregulated Advanced Solid Tumors

summary: Drg-drug Interaction (DDI) study to assess the effect of INC280 on the pharmacokinetics of midazolam and caffeine in patients with cMET-dysregulated advanced solid tumors

location_country:
Italy
France
Bulgaria
Spain
Denmark
United States
United Kingdom

location_facility: Washington University School of Medicine

location_facility: Emory University School of Medicine/Winship Cancer Institute SC-2


ReferenceCitation: Bisschops R, Manning J, Clayton LB, Ng Kwet Shing R, Álvarez-González M; MORA Study Group. Colon cleansing efficacy and safety with 1 L NER1006 versus 2 L polyethylene glycol + ascorbate: a randomized phase 3 trial. Endoscopy. 2019 Jan;51(1):60-72. doi: 10.1055/a-0638-8125. Epub 2018 Jul 19.

name: KU Leuven, University Hospitals Leuven, Leuven, Belgium

family: Bisschops

full_name: Raf Bisschops

given: Raf

name: Borders General Hospital, NHS Borders, Melrose, United Kingdom

family: Manning

full_name: Jonathan Manning

given: Jonathan

name: Clinical Development, Norgine Ltd, Harefield, United Kingdom

family: Clayton

full_name: Lucy Clayton

given: Lucy

family: Ng Kwet Shing

full_name: Richard Ng Kwet Shing

family: Álvarez-González

full_name: Marco Álvarez-González

given: Marco

journal_name: Endoscopy

oa_host_type: repository

repository_institution: KU Leuven - Lirias

repository_normalized: LIRIAS (KU Leuven)

version: acceptedVersion

publisher: Georg Thieme Verlag KG

publisher_dissemination: Georg Thieme

publisher_group: Georg Thieme

publisher_normalized: Georg Thieme

lead_sponsor: Norgine

primary_purpose: Screening

location_country:
Italy
France
Poland
Belgium
Spain
Germany
United Kingdom

acronym: MORA

location_facility: Uniwersytecki Szpital Kliniczny w Bialymstoku

location_facility: Centro di Riferimento Oncologico (C.R.O.) S.O.C Gastroenterologia

location_facility: Royal Albert Edward Infirmary Department

location_facility: Gabinet Internistyczny dr n. med. Krzysztof Janik

location_facility: A.O.U. di Bologna - Policlinico S. Orsola-Malpighi

location_facility: Kliniken Essen-Mitte; Abteilung für Gastroenterologie

location_facility: Ospedale Valduce U.O. Gastroenterologia e Endoscopia

location_facility: Hôpital Avicenne- Service de Gastro-Entérologie

location_facility: Hospital Universitari Germans Trias i Pujol

location_facility: Centralny Szpital Kliniczny Ministerstwa Spraw Wewnetrznych

location_facility: Hospital General Universitario de Alicante

location_facility: Praxis für Innere Medizin, Gastroenterologie und Allg. Medizin

location_facility: NZOZ Centrum Medyczne-Szpital Swietej Rodziny

location_facility: Klinikum der Friedrich Schiller Universität Jena

location_facility: Pol. Univ. A. Gemelli U.O. di Endoscopia Digestiva Chirurgica

location_facility: Hospital Ramon y Cajal - Ctra. De Colmenar km. 9, 100

title: A Multicenter Randomized Parallel Group Phase III Study Comparing the Bowel Cleansing Efficacy, Safety and Tolerability of NER1006 (a Low Volume Bowel Cleansing Solution) Versus MOVIPREP Using 2-Day Split-Dosing and 1-Day Morning Split-Dosing Regimen in Adults.

summary: This study evaluates the efficacy, safety and tolerability of NER1006 versus MOVIPREP in adult patients requiring bowel cleansing prior to any procedure that requires a clean bowel, using a 2-Day evening/morning Split-Dosing and 1-Day morning only Split-Dosing regimens. Approximately 810 patients will be randomised with the aim of achieving a minimum of 245 patients in each of the 3 groups.


title: A Prospective Colorectal Liver Metastasis Database With an Integrated Quality Assurance Program

acronym: CLIMB

summary:
This prospective database has two main objectives;

to evaluate the complication rates, 30-day and 90-day mortality from different surgical strategies for unresectable, borderline resectable or initially unresectable liver metastasis from colorectal cancer.
to establish baseline quality parameters for different surgical strategies for unresectable, borderline and initially unresectable colorectal liver metastasis (CRLM) patients.

location_country:
Italy
Austria
Sweden
Serbia
Switzerland
Netherlands
France
Belgium
Spain
Denmark
Germany
United Kingdom

location_facility: The Netherlands Cancer Institute-Antoni Van Leeuwenhoekziekenhuis

location_facility: Der Wiener Krankenanstaltenverbund - Krankenanstalt Rudolfstiftung

intervention_type: Other


title: An Open Label Study to Evaluate the Effects of Ezogabine/Retigabine Added to Existing Anti-epileptic Drug(s) on Urinary Voiding Function in Subjects With Partial Onset Seizures

summary:
This is a multicentre, open label study to examine the effect of ezogabine/retigabine on the voiding function of adult subjects with drug-resistant partial onset seizures (POS). Subjects fulfilling the study entry criteria at Screening and at Baseline including a comprehensive eye examination by an ophthalmologist or retina specialist and a skin assessment by the investigator will receive ezogabine/retigabine.

The starting dose of ezogabine/retigabine will be 300 mg/day. Subjects will be up titrated by 150 mg/day weekly up to the maximum ezogabine/retigabine daily dose of 1200 mg (or the highest tolerated dose). During the 49 days of the treatment phase, subjects will undergo three repeat non-invasive assessments of voiding function. In addition, subjects who meet pre-determined criteria for voiding dysfunction will undergo multichannel cystometry in order to characterise bladder hypocontractility, bladder outlet obstruction or a combination of events which clinically is manifest with difficulty emptying the bladder or acute urinary retention.

At the end of the Treatment Phase, all subjects will enter the Taper Phase, a 3-week down titration period. Subjects who have new findings of abnormal pigmentation of the retina, unexplained vision loss, pigmentation of non-retinal ocular tissue or discoloration of skin, lip, nail, or mucosa since baseline will be asked to enter the Safety Follow-Up / Continuation Phase. All subjects will undergo 6-monthly comprehensive eye examinations during the Safety Follow-Up / Continuation Phase. Subjects who have not developed abnormal discoloration of the skin, lips, nails or mucosa will continue to undergo skin assessments by the investigator. Any subject who has developed abnormal discoloration of the skin, lips, nails or mucosa since baseline will be referred to a dermatologist for evaluation and 6-monthly follow up assessments. All subjects will continue to be followed until the pigmentation and/or discoloration has resolved or stabilised, as defined by no change over 2 consecutive 6-monthly assessments conducted over at least 12 months after discontinuation of ezogabine/retigabine.

lead_sponsor: GlaxoSmithKline

location_country:
United States
France
Poland


title: An Open Label, 52-week, Safety and Tolerability Extension to a Randomized, Double-blind, Placebo Controlled Study of LCQ908 in Subjects With Familial Chylomicronemia Syndrome.

summary: This study was to determine long-term safety and tolerability, and continued efficacy in lowering triglycerides of LCQ908 in subjects with Familial Chylomicronemia Syndrome (FCS) (HLP type I).

location_country:
United States
South Africa
Netherlands
France
Germany
United Kingdom
Canada


title: An Open-label, Multi-center Phase I Dose-finding Study of RAD001 (Everolimus, Afinitor®) in Combination With BEZ235 in Patients With Advanced Solid Tumors

summary:
Study has two parts:

Dose-finding: to determine the maximum tolerated dose (MTD) and to evaluate the safety and tolerability of RAD001 (everolimus , Afinitor®) in combination with BEZ235 in patients with advanced solid tumors.
Dose-expansion: to assess safety and tolerability of RAD001 and BEZ235 at the MTD in patients with ER+/HER2- metastatic breast cancer and metastatic renal cell cancer

location_country:
Italy
France
Belgium
New Zealand
Spain
United States
United Kingdom

location_facility: Highlands Oncology Group Dept of Highlands Oncology Grp

location_facility: Washington University School of Medicine Washington University (16)


title: A Multi-arm Dose-finding Phase Ib Multicenter Study of Imatinib in Combination With the Oral Phosphatidyl-inositol 3-kinase (PI3-K) Inhibitor BKM120 in Patients With Gastrointestinal Stromal Tumor (GIST) Who Failed Prior Therapy With Imatinib and Sunitinib

summary: The purpose of this study is to determine a maximum tolerated dose and/or recommended phase 2 dose of a combination of imatinib and BKM120 in the treatment of 3rd line GIST patients.

ReferenceCitation: Gelderblom H, Jones RL, George S, Valverde Morales C, Benson C, Jean-Yves Blay, Renouf DJ, Doi T, Le Cesne A, Leahy M, Hertle S, Aimone P, Brandt U, Schӧffski P. Imatinib in combination with phosphoinositol kinase inhibitor buparlisib in patients with gastrointestinal stromal tumour who failed prior therapy with imatinib and sunitinib: a Phase 1b, multicentre study. Br J Cancer. 2020 Apr;122(8):1158-1165. doi: 10.1038/s41416-020-0769-y. Epub 2020 Mar 9.

family: Gelderblom

full_name: Hans Gelderblom

given: Hans

family: Jones

full_name: Robin L. Jones

given: Robin L.

family: George

full_name: Suzanne George

given: Suzanne

family: Valverde Morales

full_name: Claudia Valverde Morales

given: Claudia

family: Benson

full_name: Charlotte Benson

given: Charlotte

family: Jean-Yves Blay

full_name: Jean-Yves Blay

family: Renouf

full_name: Daniel J. Renouf

given: Daniel J.

family: Doi

full_name: Toshihiko Doi

given: Toshihiko

family: Le Cesne

full_name: Axel Le Cesne

given: Axel

family: Leahy

full_name: Michael Leahy

given: Michael

family: Hertle

full_name: Sabine Hertle

given: Sabine

family: Aimone

full_name: Paola Aimone

given: Paola

family: Brandt

full_name: Ulrike Brandt

given: Ulrike

family: Schӧffski

full_name: Patrick Schӧffski

given: Patrick

journal_name: British Journal of Cancer

license: cc-by

license_normalized: cc-by

repositories:
LIRIAS (KU Leuven)
PubMed Central

publisher: Springer Science and Business Media LLC

publisher_dissemination: Springer-Nature

publisher_group: Springer-Nature

publisher_normalized: Springer

location_country:
United States
Netherlands
France
Japan
Belgium
Spain
Canada
United Kingdom

location_facility:
Seattle Cancer Care Alliance Onc
Novartis Investigative Site
Dana Farber Cancer Institute SC (2)


ReferenceCitation: Schwartz GJ, Feld LG, Langford DJ. A simple estimate of glomerular filtration rate in full-term infants during the first year of life. J Pediatr. 1984 Jun;104(6):849-54.

ipd_sharing_description: Information available on the Grünenthal Web Site

lead_sponsor: Grünenthal GmbH

location_country:
Germany
Croatia
Czechia
Bulgaria
France
Poland
Hungary
Spain
United States
United Kingdom

title: An Evaluation of the Efficacy and Safety of Tapentadol Oral Solution in the Treatment of Post-operative Acute Pain Requiring Opioid Treatment in Pediatric Subjects Aged From Birth to Less Than 18 Years Old

summary: The purpose of the study was to evaluate the efficacy of tapentadol oral solution, based on the total amount of supplemental opioid analgesic used over 12 hours or 24 hours after initiation of investigational medicinal product (IMP) in children and adolescents who had undergone surgery that would produce moderate to severe pain during opioid treatment.


lead_sponsor: Actelion

location_country:
Switzerland
Mexico
France
Czechia
Lithuania
Poland
Thailand
Hungary
Belgium
Russian Federation
China
Turkey
Germany
United Kingdom
Ukraine

title: MERIT-2 : Long Term, Multicenter, Single-arm, Open-label Extension Study of the MERIT-1 Study, to Assess the Safety, Tolerabilty and Efficacy of Macitentan in Subjects With Inoperable Chronic Thromboembolic Pulmonary Hypertension (CTEPH)

summary: Long-term study to evaluate if macitentan is safe, tolerable and efficient enough to be used for treatment of inoperable chronic thromboembolic pulmonary hypertension (CTEPH)


lead_sponsor: LEO Pharma

location_country:
United States
France
Poland
Romania
Germany
United Kingdom
Canada

acronym: Not Applicable

location_facility: Policlinica de Diagnostic Rapid S.A. Clinic

location_facility: Cabinet Medical Individual Tatu G. Alin Laurentiu

location_facility: Spitalul Clinic Judetean de Urgenta Cluj-Napoca

location_facility: Clinical Research Center, Morsani Center for Advanced Healthcare

location_facility: Dermatology and Laser Center of Charleston, PA

location_facility: Specjalistyczny Ofrodek Leczniczo Clinic

location_facility: Spitalul Clinic Municipal de Urgenta Timisoara

title: Effect of Calcipotriol Plus Betamethasone Dipropionate Gel on the HPA Axis and Calcium Metabolism in Adolescent Subjects (Aged 12 to 16 Years, 11 Months) With Scalp and Body Psoriasis

summary: An international, multi-centre, prospective, non-controlled, open, single-group, 8-week trial in adolescent subjects (aged 12 to 16 years, 11 months) with scalp and body psoriasis.


title: A Multicentre, Open-Label, Long-Term, Safety and Tolerability Study of Retigabine Immediate Release (IR) in Adults With Partial-Onset Seizures (Extension of Study RGB113905)

acronym: IR

summary: The purpose of this Phase III study is to assess the long-term safety, tolerability and efficacy of flexibly dosed retigabine Immediate Release (IR) as adjunctive therapy in adult subjects with partial-onset seizures. In addition, those subjects who successfully completed 20 weeks of adjunctive treatment with retigabine IR in the parent study, RGB113905, and who were thought to have benefitted from treatment will be provided continued access to retigabine IR.

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

location_country:
Italy
Netherlands
France
Bulgaria
Poland
Thailand
Belgium
Russian Federation
Germany
Ukraine


title: A Multicenter, Long-term Extension Study to Further Evaluate the Safety and Tolerability of Telotristat Etiprate (LX1606)

acronym: TELEPATH

summary: The primary objective of this study is to evaluate the long-term safety and tolerability of orally administered telotristat etiprate.

lead_sponsor: Lexicon Pharmaceuticals

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

location_facility:
Lexicon Investgational Site
Lexicon Investigational Site


title: A Three Arm Randomized, Open-label Phase II Study of Radium-223 Dichloride 50 kBq/kg (55 kBq/kg After Implementation of NIST Update) Versus 80 kBq/kg (88 kBq/kg After Implementation of NIST Update), and Versus 50 kBq/kg (55 kBq/kg After Implementation of NIST Update) in an Extended Dosing Schedule in Subjects With Castration-resistant Prostate Cancer Metastatic to the Bone

summary: This study will assess different doses and regimens of radium-223 dichloride on the incidence of symptomatic skeletal events. Eligible subjects must have castration resistant prostate cancer with 2 or more skeletal metastases documented within 8 weeks of randomization. Subjects will be randomized to one of 3 treatment arms in a 1:1:1 fashion: a standard regimen of radium-223 dichloride of 50 kBq/kg (55 kBq/kg after implementation of NIST update) injections every month for 6 months, a high dose regimen of 80 kBq/kg (88 kBq/kg after implementation of NIST update)injections every month for 6 months or an extended duration regimen of 50 kBq/kg (55 kBq/kg after implementation of NIST update) injections every month for 12 months. Following the treatment phase, subjects will be followed up every 12 weeks for a minimum of 2 years, at which point they will enter a long term follow-up period during which they are seen every 6 months for up to 7 years after the last dose of radium dichloride. Symptomatic skeletal event and safety endpoints will be assessed at each clinic visit. Pain and analgesic use data will be collected every 4 weeks through Week 48. Additionally, radiological assessments including MRI/CT of the abdomen and pelvis and chest CT, as well as technetium-99 bone scans will be performed at Weeks 8, 16, and 24 and continue every 12 weeks thereafter until disease progression is documented in either the bone or in soft tissue. Radiological imaging will be evaluated by blinded central review.

ReferenceCitation: Sternberg CN, Saad F, Graff JN, Peer A, Vaishampayan UN, Leung E, Rosenbaum E, Gurney H, Epstein RJ, Davis ID, Wu B, Trandafir L, Wagner VJ, Hussain M. A randomised phase II trial of three dosing regimens of radium-223 in patients with bone metastatic castration-resistant prostate cancer. Ann Oncol. 2020 Feb;31(2):257-265. doi: 10.1016/j.annonc.2019.10.025. Epub 2019 Dec 23.

family: Sternberg

full_name: C.N. Sternberg

family: Saad

full_name: F. Saad

given: F.

family: Graff

full_name: J.N. Graff

family: Peer

full_name: A. Peer

given: A.

family: Vaishampayan

full_name: U.N. Vaishampayan

family: Leung

full_name: E. Leung

given: E.

family: Rosenbaum

full_name: E. Rosenbaum

family: Gurney

full_name: H. Gurney

given: H.

family: Epstein

full_name: R.J. Epstein

family: Davis

full_name: I.D. Davis

family: Wu

full_name: B. Wu

given: B.

family: Trandafir

full_name: L. Trandafir

given: L.

family: Wagner

full_name: V.J. Wagner

family: Hussain

full_name: M. Hussain

given: M.

journal_name: Annals of Oncology

oa_host_type: publisher

publisher: Elsevier BV

publisher_dissemination: Elsevier

publisher_group: Elsevier

publisher_normalized: Elsevier

location_country:
Italy
Brazil
United States
Chile
Sweden
Germany
France
Czechia
Israel
Australia
Spain
Canada
United Kingdom
Korea, Republic of
Taiwan

location_facility: Irmandade Santa Casa de Misericordia de Porto Alegre

location_facility: IBCC - Instituto Brasileiro de Controle do Cancer

location_facility: Instituto do Câncer do Estado de São Paulo


title: A Phase 1/2 Combined Dose Ranging and Randomized, Open-label, Comparative Study of the Efficacy and Safety of Plerixafor in Addition to Standard Regimens for Mobilization of Haematopoietic Stem Cells Into Peripheral Blood, and Subsequent Collection by Apheresis, Versus Standard Mobilization Regimens Alone in Pediatric Patients, Aged 1 to <18 Years, With Solid Tumours Eligible for Autologous Transplants.

summary:
This is a multi-site study with plerixafor in pediatric cancer patients. The study will be conducted in 2 stages:

Stage 1 is a dose-escalation study.
Stage 2 is an open-label, randomized, comparative study using the appropriate dosing regimen identified in the Stage 1 dose-escalation study.

All participating patients will receive a standard mobilization regimen as per study site practice guidelines (either chemotherapy plus once daily granulocyte-colony stimulating factor (G-CSF) or once daily G-CSF alone). The only change to the standard mobilization regimen is the addition of plerixafor treatment prior to apheresis for all patients in Stage 1 (dose escalation), and for those patients randomized to the plerixafor plus standard mobilization treatment arm in Stage 2 (randomized, comparative).

Stage 1 will enroll at least 27 patients. Stage 2 will enroll at least 40 patients.

ReferenceCitation: Morland B, Kepak T, Dallorso S, Sevilla J, Murphy D, Luksch R, Yaniv I, Bader P, Rößler J, Bisogno G, Maecker-Kolhoff B, Lang P, Zwaan CM, Sumerauer D, Kriván G, Bernard J, Liu Q, Doyle E, Locatelli F. Plerixafor combined with standard regimens for hematopoietic stem cell mobilization in pediatric patients with solid tumors eligible for autologous transplants: two-arm phase I/II study (MOZAIC). Bone Marrow Transplant. 2020 Sep;55(9):1744-1753. doi: 10.1038/s41409-020-0836-2. Epub 2020 Mar 3.

family: Morland

full_name: Bruce Morland

given: Bruce

family: Kepak

full_name: Tomas Kepak

given: Tomas

family: Dallorso

full_name: Sandro Dallorso

given: Sandro

family: Sevilla

full_name: Julian Sevilla

given: Julian

family: Murphy

full_name: Dermot Murphy

given: Dermot

family: Luksch

full_name: Roberto Luksch

given: Roberto

family: Yaniv

full_name: Isaac Yaniv

given: Isaac

family: Bader

full_name: Peter Bader

given: Peter

family: Rößler

full_name: Jochen Rößler

given: Jochen

family: Bisogno

full_name: Gianni Bisogno

given: Gianni

family: Maecker-Kolhoff

full_name: Britta Maecker-Kolhoff

given: Britta

family: Lang

full_name: Peter Lang

family: Zwaan

full_name: C. Michel Zwaan

given: C. Michel

family: Sumerauer

full_name: David Sumerauer

given: David

family: Kriván

full_name: Gergely Kriván

given: Gergely

family: Bernard

full_name: John Bernard

given: John

family: Liu

full_name: Qianying Liu

given: Qianying

family: Doyle

full_name: Eileen Doyle

given: Eileen

family: Locatelli

full_name: Franco Locatelli

given: Franco

journal_name: Bone Marrow Transplantation

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

location_country:
Italy
Netherlands
France
Czechia
Poland
Israel
Hungary
Belgium
Spain
Denmark
Germany
United Kingdom

location_facility:
Investigational Site Number 33
Investigational Site Number 81
Investigational Site Number 83
Investigational Site Number 92
Investigational Site Number 11
Investigational Site Number 23
Investigational Site Number 22
Investigational Site Number 43
Investigational Site Number 85
Investigational Site Number 51
Investigational Site Number 82
Investigational Site Number 26
Investigational Site Number 93
Investigational Site Number 61
Investigational Site Number 36
Investigational Site Number 42
Investigational Site Number 24
Investigational Site Number 35
Investigational Site Number 31
Investigational Site Number 34
Investigational Site Number 72
Investigational Site Number 94
Investigational Site Number 21
Investigational Site Number 13
Investigational Site Number 71
Investigational Site Number 84
Investigational Site Number 91


title: European Ankylosing Spondylitis Infliximab Cohort (EASIC) Follow up Registration Study

acronym: EASIC registry

summary: Long term data on efficacy and safety of anti-TNF treatment with infliximab in patients with ankylosing spondylitis (AS) beyond 5 years is lacking. These data are important because patients with AS usually are younger and withdrawal of anti-TNF therapy in these patients almost always leads to a disease relapse. Furthermore it is still unclear whether long term anti-TNF treatment in AS patients can inhibit radiographic progression. Patients who participated in the EASIC and the DIKAS trial respectively who were treated with infliximab within these studies for 7 and 10 years respectively are followed up by using clinical outcome parameters every 6 months assessing efficacy and safety of long term treatment. Furthermore radiographs of the spine, if done for clinical indication, are analyzed. It is hypothesized that anti-TNF treatment with infliximab is effective and safe over a time period of 9 and 12 years respectively and that long term anti-TNF therapy may inhibit radiographic progression of the spine.

status: Unknown status

ReferenceCitation: Zochling J, van der Heijde D, Burgos-Vargas R, Collantes E, Davis JC Jr, Dijkmans B, Dougados M, Géher P, Inman RD, Khan MA, Kvien TK, Leirisalo-Repo M, Olivieri I, Pavelka K, Sieper J, Stucki G, Sturrock RD, van der Linden S, Wendling D, Böhm H, van Royen BJ, Braun J; 'ASsessment in AS' international working group; European League Against Rheumatism. ASAS/EULAR recommendations for the management of ankylosing spondylitis. Ann Rheum Dis. 2006 Apr;65(4):442-52. Epub 2005 Aug 26.

ReferenceCitation: Baraliakos X, Listing J, Brandt J, Zink A, Alten R, Burmester G, Gromnica-Ihle E, Kellner H, Schneider M, Sörensen H, Zeidler H, Rudwaleit M, Sieper J, Braun J. Clinical response to discontinuation of anti-TNF therapy in patients with ankylosing spondylitis after 3 years of continuous treatment with infliximab. Arthritis Res Ther. 2005;7(3):R439-44. Epub 2005 Feb 21. Erratum in: Arthritis Res Ther. 2005;7(3):113. Zink, Angela [added];Alten, Rieke [added]; Burmester, Gerd [added]; Gromnica-Ihle, Erika [added]; Kellner, Herbert [added]; Schneider, Matthias [added]; Sörensen, Helmut [added]; Zeidler, Henning [added]; Rudwaleit, Martin [added].

ReferenceCitation: Baraliakos X, Listing J, Rudwaleit M, Brandt J, Alten R, Burmester G, Gromnica-Ihle E, Haibel H, Schewe S, Schneider M, Sörensen H, Zeidler H, Visvanathan S, Sieper J, Braun J. Safety and efficacy of readministration of infliximab after longterm continuous therapy and withdrawal in patients with ankylosing spondylitis. J Rheumatol. 2007 Mar;34(3):510-5. Epub 2007 Feb 1.

ReferenceCitation: Braun J, Baraliakos X, Listing J, Fritz C, Alten R, Burmester G, Krause A, Schewe S, Schneider M, Sörensen H, Zeidler H, Sieper J. Persistent clinical efficacy and safety of anti-tumour necrosis factor alpha therapy with infliximab in patients with ankylosing spondylitis over 5 years: evidence for different types of response. Ann Rheum Dis. 2008 Mar;67(3):340-5. Epub 2007 Oct 29.

ReferenceCitation: Dijkmans B, Emery P, Hakala M, Leirisalo-Repo M, Mola EM, Paolozzi L, Salvarani C, Sanmarti R, Sibilia J, Sieper J, Van Den Bosch F, van der Heijde D, van der Linden S, Wajdula J. Etanercept in the longterm treatment of patients with ankylosing spondylitis. J Rheumatol. 2009 Jun;36(6):1256-64. doi: 10.3899/jrheum.081033. Epub 2009 May 1. Erratum in: J Rheumatol. 2010 Oct;37(10):2198.

ReferenceCitation: Baraliakos X, Listing J, Rudwaleit M, Brandt J, Sieper J, Braun J. Radiographic progression in patients with ankylosing spondylitis after 2 years of treatment with the tumour necrosis factor alpha antibody infliximab. Ann Rheum Dis. 2005 Oct;64(10):1462-6. Epub 2005 Mar 18.

ReferenceCitation: van der Heijde D, Landewé R, Baraliakos X, Houben H, van Tubergen A, Williamson P, Xu W, Baker D, Goldstein N, Braun J; Ankylosing Spondylitis Study for the Evaluation of Recombinant Infliximab Therapy Study Group. Radiographic findings following two years of infliximab therapy in patients with ankylosing spondylitis. Arthritis Rheum. 2008 Oct;58(10):3063-70. doi: 10.1002/art.23901.

ReferenceCitation: Essers I, van Tubergen A, Heldmann F, Baraliakos X, Braun J, Kiltz U, Boonen A. Do patients with ankylosing spondylitis adapt to their disease? Evidence from a 'then-test' in patients treated with TNF inhibitors. RMD Open. 2015 Nov 17;1(1):e000164. doi: 10.1136/rmdopen-2015-000164. eCollection 2015.

family: Essers

full_name: I. Essers

given: I.

family: van Tubergen

full_name: A. van Tubergen

given: A.

family: Heldmann

full_name: F. Heldmann

given: F.

family: Baraliakos

full_name: X. Baraliakos

given: X.

family: Braun

full_name: J. Braun

given: J.

family: Kiltz

full_name: U. Kiltz

given: U.

family: Boonen

full_name: A. Boonen

currency_apc_doaj: GBP

journal_name: RMD Open

oa_colors: green gold

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

lead_sponsor: Rheumazentrum Ruhrgebiet

location_country:
Netherlands
Finland
France
Belgium
Germany
United Kingdom

location_facility: University Central Hospital, Division of Rheumatology

location_facility: Universitair Ziekenhuis, Afdeling Rheumatologie

location_facility: Klinik für Immunologie und Rheumatologie der MHH

location_facility: Klinikum der Universität München, Rheumaeinheit


title: A phase1 Multi-center, Open Label, Dose-escalation Study of Oral LEE011 in Patients With Advanced Solid Tumors or Lymphoma

summary:
LEE011 is a new oral drug designed to inhibit the activity of an enzyme known as CDK4/6. CDK4/6 is involved in the process that allows both normal and cancer cells to divide and multiply. Cancer cells are often driven to divide and multiply by abnormalities that increase the activity of CDK4. Hence there is hope that blocking the activity of CDK4 may slow the growth of some cancers. LEE011 has shown anti-cancer activity in several different tumor models in animals.

Because CDK4 is important in both normal and cancerous cells, LEE011 is expected to decrease the ability of the bone marrow to make white blood cells, platelets, and red blood cells. Although these effects are expected to be reversible, they can increase the risk of infection, bleeding and fatigue.

The primary purpose of this study is to find the highest dose of LEE011 that can be safely given to adult patients with advanced solid tumors or lymphomas for which no further effective standard treatment is available. It will provide information about the side effects that may occur following treatment. The study will also possibly provide early evidence for LEE011's anti-tumor activity.

location_country:
United States
France
Netherlands

location_facility: University of Michigan Comprehensive Cancer Center SC

location_facility: Sarah Cannon Research Institute DeptofSarahCannonRes.Inst. (2)


title: A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant Human GAA) in Patients With Late-onset Pompe Disease

summary: A Phase 1/2, open-label, multicenter, multiple dose escalation study of BMN 701 administered by intravenous infusion every 2 weeks over a 24-week treatment period to patients with late-onset Pompe disease.

lead_sponsor: BioMarin Pharmaceutical

location_country:
United States
France
Australia
Germany
United Kingdom

location_facility: Old Queen Elizabeth Hospital, Department of Medicine

location_facility: Univ of California San Diego School of Medicine

location_facility: University of Florida College of Medicine

location_facility: Hôpital de I´Archet- Centre Hospitalier Universitaire Nice


title: Double Blind Randomized Phase III Study of Maintenance Pazopanib Versus Placebo in NSCLC Patients Non Progressive After First Line Chemotherapy. MAPPING, an EORTC Lung Group Study.

summary:
RATIONALE: Pazopanib hydrochloride may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth and by blocking blood flow to the tumor. It is not yet known whether pazopanib hydrochloride is more effective than a placebo in treating patients with non-small cell lung cancer that has not progressed after first-line chemotherapy.

PURPOSE: This randomized phase II/III trial is studying how well giving pazopanib hydrochloride works and compares it with giving a placebo in treating patients with non-small cell lung cancer who have received first-line chemotherapy.

location_country:
United Kingdom
Netherlands
France
Greece
Belgium
Egypt
Germany
Slovenia

location_facility: Centre Hospitalier Regional De La Citadelle

location_facility: The Netherlands Cancer Institute-Antoni Van Leeuwenhoekziekenhuis

location_facility: Nottingham University Hospitals NHS Trust

location_facility: Assistance Publique - Hôpitaux de Marseille - Assistance Publique - Hôpitaux de Marseille - Hopital Nord


title: A Multi-Centre, Multi-National, Open-Label, Dose Escalation Trial Evaluating Safety and Pharmacokinetics of Ascending Intravenous Doses of 40K Pegylated Recombinant FIX in Non-Bleeding Patients With Haemophilia B.

summary:
This trial is conducted in Europe, Japan and the United States of America (USA).

The aim of this clinical trial is to investigate the safety and pharmacokinetics (the determination of the concentration of the administered medication in blood over time) of Pegylated Recombinant Factor IX (nonacog beta pegol) in Non-Bleeding Patients with Haemophilia B.

ReferenceCitation: Negrier C, Knobe K, Tiede A, Giangrande P, Møss J. Enhanced pharmacokinetic properties of a glycoPEGylated recombinant factor IX: a first human dose trial in patients with hemophilia B. Blood. 2011 Sep 8;118(10):2695-701. doi: 10.1182/blood-2011-02-335596. Epub 2011 May 9.

ReferenceCitation: Collins PW, Møss J, Knobe K, Groth A, Colberg T, Watson E. Population pharmacokinetic modeling for dose setting of nonacog beta pegol (N9-GP), a glycoPEGylated recombinant factor IX. J Thromb Haemost. 2012 Nov;10(11):2305-12. doi: 10.1111/jth.12000.

location_country:
Sweden
Germany
France
Japan
Spain
Denmark
United States
United Kingdom


title: A Phase 2 Single Arm, Multicenter Trial to Evaluate the Efficacy of the BiTE Antibody Blinatumomab in Adult Subjects With Relapsed/Refractory Philadelphia Positive B-precursor Acute Lymphoblastic Leukemia (Alcantara Study)

summary: The primary objective is to evaluate the rate of complete remission/complete remission with partial hematological recovery (CRh*) in adults with relapsed/refractory Philadelphia chromosome positive (Ph+) B-precursor acute lymphoblastic leukemia (ALL) who receive blinatumomab.

ReferenceCitation: Martinelli G, Boissel N, Chevallier P, Ottmann O, Gökbuget N, Topp MS, Fielding AK, Rambaldi A, Ritchie EK, Papayannidis C, Sterling LR, Benjamin J, Stein A. Complete Hematologic and Molecular Response in Adult Patients With Relapsed/Refractory Philadelphia Chromosome-Positive B-Precursor Acute Lymphoblastic Leukemia Following Treatment With Blinatumomab: Results From a Phase II, Single-Arm, Multicenter Study. J Clin Oncol. 2017 Jun 1;35(16):1795-1802. doi: 10.1200/JCO.2016.69.3531. Epub 2017 Mar 29. Erratum in: J Clin Oncol. 2017 Aug 10;35(23):2722. J Clin Oncol. 2017 Aug 20;35(24):2856.

ReferenceCitation: Kuchimanchi M, Zhu M, Clements JD, Doshi S. Exposure-response analysis of blinatumomab in patients with relapsed/refractory acute lymphoblastic leukaemia and comparison with standard of care chemotherapy. Br J Clin Pharmacol. 2019 Apr;85(4):807-817. doi: 10.1111/bcp.13864. Epub 2019 Feb 18.

ReferenceCitation: Kantarjian HM, Zugmaier G, Brüggemann M, Wood BL, Horst HA, Zeng Y, Martinelli G. Impact of Blinatumomab Treatment on Bone Marrow Function in Patients with Relapsed/Refractory B-Cell Precursor Acute Lymphoblastic Leukemia. Cancers (Basel). 2021 Nov 9;13(22). pii: 5607. doi: 10.3390/cancers13225607.

ReferenceCitation: Martinelli G, Boissel N, Chevallier P, Ottmann O, Gökbuget N, Rambaldi A, Ritchie EK, Papayannidis C, Tuglus CA, Morris JD, Stein A. Long-term follow-up of blinatumomab in patients with relapsed/refractory Philadelphia chromosome-positive B-cell precursor acute lymphoblastic leukaemia: Final analysis of ALCANTARA study. Eur J Cancer. 2021 Mar;146:107-114. doi: 10.1016/j.ejca.2020.12.022. Epub 2021 Feb 13.

family: Martinelli

full_name: Giovanni Martinelli

given: Giovanni

family: Boissel

full_name: Nicolas Boissel

given: Nicolas

family: Chevallier

full_name: Patrice Chevallier

given: Patrice

family: Ottmann

full_name: Oliver Ottmann

given: Oliver

family: Gökbuget

full_name: Nicola Gökbuget

given: Nicola

family: Rambaldi

full_name: Alessandro Rambaldi

given: Alessandro

family: Ritchie

full_name: Ellen K. Ritchie

given: Ellen K.

family: Papayannidis

full_name: Cristina Papayannidis

given: Cristina

family: Tuglus

full_name: Catherine A. Tuglus

given: Catherine A.

family: Morris

full_name: Joan D. Morris

given: Joan D.

family: Stein

full_name: Anthony Stein

given: Anthony

journal_name: European Journal of Cancer

repository_institution: Cardiff University - ORCA Online Research @Cardiff

repository_normalized: https

title: Long-term follow-up of blinatumomab in patients with relapsed/refractory Philadelphia chromosome–positive B-cell precursor acute lymphoblastic leukaemia: Final analysis of ALCANTARA study

lead_sponsor: Amgen

location_country:
Italy
United States
France
Germany
United Kingdom


title: A Phase 2, Open-label, Randomized Study to Evaluate the Safety and Efficacy of Momelotinib in Subjects With Polycythemia Vera or Essential Thrombocythemia

summary: This open-label study is to determine the safety and efficacy of momelotinib in participants with either polycythemia vera (PV) or essential thrombocythemia (ET) who have not yet received treatment with a Janus kinase (JAK) inhibitor.

ReferenceCitation: Verstovsek S, Courby S, Griesshammer M, Mesa RA, Brachmann CB, Kawashima J, Maltzman JD, Shao L, Xin Y, Huang D, Bajel A. A phase 2 study of momelotinib, a potent JAK1 and JAK2 inhibitor, in patients with polycythemia vera or essential thrombocythemia. Leuk Res. 2017 Sep;60:11-17. doi: 10.1016/j.leukres.2017.05.002. Epub 2017 May 30.

family: Verstovsek

full_name: Srdan Verstovsek

given: Srdan

family: Courby

full_name: Stephane Courby

given: Stephane

family: Griesshammer

full_name: Martin Griesshammer

given: Martin

family: Mesa

full_name: Ruben A. Mesa

given: Ruben A.

family: Brachmann

full_name: Carrie Baker Brachmann

given: Carrie Baker

family: Kawashima

full_name: Jun Kawashima

given: Jun

family: Maltzman

full_name: Julia D. Maltzman

given: Julia D.

family: Shao

full_name: Lixin Shao

given: Lixin

family: Xin

full_name: Yan Xin

given: Yan

family: Huang

full_name: Daniel Huang

given: Daniel

family: Bajel

full_name: Ashish Bajel

given: Ashish

journal_name: Leukemia Research

repository_institution: University of Melbourne - Minerva Access

lead_sponsor: Sierra Oncology, Inc.

location_country:
United States
Germany
France
Australia
Canada


title: Efficacy Evaluation of Intra-articular Hyaluronic Acid (Sinovial®) vs Synvisc® in the Treatment of Symptomatic Knee Osteoarthritis. A Double-blind, Controlled, Randomised, Parallel-group Non-inferiority Study

summary: The purpose of this study is to prove the non-inferiority of the intra-articular injection of hyaluronic acid (Sinovial®) in the symptomatic treatment of knee OA in comparison to Synvisc®.

ReferenceCitation: Theiler R, Brühlmann P. Overall tolerability and analgesic activity of intra-articular sodium hyaluronate in the treatment of knee osteoarthritis. Curr Med Res Opin. 2005 Nov;21(11):1727-33.

ReferenceCitation: Castellacci E, Polieri T. Antalgic effect and clinical tolerability of hyaluronic acid in patients with degenerative diseases of knee cartilage: an outpatient treatment survey. Drugs Exp Clin Res. 2004;30(2):67-73.

ReferenceCitation: Jordan KM, Arden NK, Doherty M, Bannwarth B, Bijlsma JW, Dieppe P, Gunther K, Hauselmann H, Herrero-Beaumont G, Kaklamanis P, Lohmander S, Leeb B, Lequesne M, Mazieres B, Martin-Mola E, Pavelka K, Pendleton A, Punzi L, Serni U, Swoboda B, Verbruggen G, Zimmerman-Gorska I, Dougados M; Standing Committee for International Clinical Studies Including Therapeutic Trials ESCISIT. EULAR Recommendations 2003: an evidence based approach to the management of knee osteoarthritis: Report of a Task Force of the Standing Committee for International Clinical Studies Including Therapeutic Trials (ESCISIT). Ann Rheum Dis. 2003 Dec;62(12):1145-55. Review.

ReferenceCitation: Recommendations for the medical management of osteoarthritis of the hip and knee: 2000 update. American College of Rheumatology Subcommittee on Osteoarthritis Guidelines. Arthritis Rheum. 2000 Sep;43(9):1905-15.

ReferenceCitation: Altman RD. Status of hyaluronan supplementation therapy in osteoarthritis. Curr Rheumatol Rep. 2003 Feb;5(1):7-14. Review.

ReferenceCitation: Lo GH, LaValley M, McAlindon T, Felson DT. Intra-articular hyaluronic acid in treatment of knee osteoarthritis: a meta-analysis. JAMA. 2003 Dec 17;290(23):3115-21.

ReferenceCitation: Wang CT, Lin J, Chang CJ, Lin YT, Hou SM. Therapeutic effects of hyaluronic acid on osteoarthritis of the knee. A meta-analysis of randomized controlled trials. J Bone Joint Surg Am. 2004 Mar;86(3):538-45.

ReferenceCitation: Bellamy N, Campbell J, Robinson V, Gee T, Bourne R, Wells G. Viscosupplementation for the treatment of osteoarthritis of the knee. Cochrane Database Syst Rev. 2006 Apr 19;(2):CD005321. Review.

ReferenceCitation: Recommendations for the registration of drugs used in the treatment of osteoarthritis. Group for the respect of ethics and excellence in science (GREES): osteoarthritis section. Ann Rheum Dis. 1996 Aug;55(8):552-7.

ReferenceCitation: Altman R, Brandt K, Hochberg M, Moskowitz R, Bellamy N, Bloch DA, Buckwalter J, Dougados M, Ehrlich G, Lequesne M, Lohmander S, Murphy WA Jr, Rosario-Jansen T, Schwartz B, Trippel S. Design and conduct of clinical trials in patients with osteoarthritis: recommendations from a task force of the Osteoarthritis Research Society. Results from a workshop. Osteoarthritis Cartilage. 1996 Dec;4(4):217-43.

ReferenceCitation: Strand V, Kelman A. Outcome measures in osteoarthritis: randomized controlled trials. Curr Rheumatol Rep. 2004 Feb;6(1):20-30. Review.

ReferenceCitation: Kirchner M, Marshall D. A double-blind randomized controlled trial comparing alternate forms of high molecular weight hyaluronan for the treatment of osteoarthritis of the knee. Osteoarthritis Cartilage. 2006 Feb;14(2):154-62. Epub 2005 Oct 19.

ReferenceCitation: Bellamy N, Buchanan WW, Goldsmith CH, Campbell J, Stitt LW. Validation study of WOMAC: a health status instrument for measuring clinically important patient relevant outcomes to antirheumatic drug therapy in patients with osteoarthritis of the hip or knee. J Rheumatol. 1988 Dec;15(12):1833-40.

ReferenceCitation: Bellamy N. WOMAC: a 20-year experiential review of a patient-centered self-reported health status questionnaire. J Rheumatol. 2002 Dec;29(12):2473-6. Review.

ReferenceCitation: KELLGREN JH, LAWRENCE JS. Radiological assessment of osteo-arthrosis. Ann Rheum Dis. 1957 Dec;16(4):494-502.

ReferenceCitation: Altman R, Asch E, Bloch D, Bole G, Borenstein D, Brandt K, Christy W, Cooke TD, Greenwald R, Hochberg M, et al. Development of criteria for the classification and reporting of osteoarthritis. Classification of osteoarthritis of the knee. Diagnostic and Therapeutic Criteria Committee of the American Rheumatism Association. Arthritis Rheum. 1986 Aug;29(8):1039-49.

ReferenceCitation: Lockman LE. Practice tips. Knee joint injections and aspirations: the triangle technique. Can Fam Physician. 2006 Nov;52(11):1403-4.

ReferenceCitation: Lequesne MG, Mery C, Samson M, Gerard P. Indexes of severity for osteoarthritis of the hip and knee. Validation--value in comparison with other assessment tests. Scand J Rheumatol Suppl. 1987;65:85-9. Erratum in: Scand J Rheumatol 1988;17(3):following 241. Scand J Rheumatol Suppl 1988;73:1.

ReferenceCitation: Lequesne M, Samson M, Gérard P, Mery C. [Pain-function indices for the follow-up of osteoarthritis of the hip and the knee]. Rev Rhum Mal Osteoartic. 1990 Oct 30;57(9 ( Pt 2)):32S-36S. French.

ReferenceCitation: Ali Y, Weinstein M, Jokl P. Acute pseudogout following intra-articular injection of high molecular weight hyaluronic acid. Am J Med. 1999 Dec;107(6):641-2.

ReferenceCitation: Luzar MJ, Altawil B. Pseudogout following intraarticular injection of sodium hyaluronate. Arthritis Rheum. 1998 May;41(5):939-40.

ReferenceCitation: Maillefert JF, Hirschhorn P, Pascaud F, Piroth C, Tavernier C. Acute attack of chondrocalcinosis after an intraarticular injection of hyaluronan. Rev Rhum Engl Ed. 1997 Oct;64(10):593-4.

ReferenceCitation: Allen E, Krohn K. Adverse reaction to Hylan GF-20. J Rheumatol. 2000 Jun;27(6):1572.

ReferenceCitation: Chen AL, Desai P, Adler EM, Di Cesare PE. Granulomatous inflammation after Hylan G-F 20 viscosupplementation of the knee : a report of six cases. J Bone Joint Surg Am. 2002 Jul;84(7):1142-7.

ReferenceCitation: Zardawi IM, Chan I. Synvisc perisynovitis. Pathology. 2001 Nov;33(4):519-20.

ReferenceCitation: Hamburger MI, Lakhanpal S, Mooar PA, Oster D. Intra-articular hyaluronans: a review of product-specific safety profiles. Semin Arthritis Rheum. 2003 Apr;32(5):296-309. Review.

ReferenceCitation: Ehrich EW, Davies GM, Watson DJ, Bolognese JA, Seidenberg BC, Bellamy N. Minimal perceptible clinical improvement with the Western Ontario and McMaster Universities osteoarthritis index questionnaire and global assessments in patients with osteoarthritis. J Rheumatol. 2000 Nov;27(11):2635-41.

ReferenceCitation: Angst F, Aeschlimann A, Michel BA, Stucki G. Minimal clinically important rehabilitation effects in patients with osteoarthritis of the lower extremities. J Rheumatol. 2002 Jan;29(1):131-8.

ReferenceCitation: Bijlsma JW. Patient centred outcomes in osteoarthritis. Ann Rheum Dis. 2005 Jan;64(1):1-2.

ReferenceCitation: Tubach F, Ravaud P, Baron G, Falissard B, Logeart I, Bellamy N, Bombardier C, Felson D, Hochberg M, van der Heijde D, Dougados M. Evaluation of clinically relevant changes in patient reported outcomes in knee and hip osteoarthritis: the minimal clinically important improvement. Ann Rheum Dis. 2005 Jan;64(1):29-33. Epub 2004 Jun 18.

ReferenceCitation: Pavelka K, Uebelhart D. Efficacy evaluation of highly purified intra-articular hyaluronic acid (Sinovial(®)) vs hylan G-F20 (Synvisc(®)) in the treatment of symptomatic knee osteoarthritis. A double-blind, controlled, randomized, parallel-group non-inferiority study. Osteoarthritis Cartilage. 2011 Nov;19(11):1294-300. doi: 10.1016/j.joca.2011.07.016. Epub 2011 Aug 16.

family: Pavelka

full_name: K. Pavelka

given: K.

family: Uebelhart

full_name: D. Uebelhart

given: D.

journal_name: Osteoarthritis and Cartilage

license: elsevier-specific: oa user license

license_normalized: elsevier-specific

title: Efficacy evaluation of highly purified intra-articular hyaluronic acid (Sinovial®) vs hylan G-F20 (Synvisc®) in the treatment of symptomatic knee osteoarthritis. A double-blind, controlled, randomized, parallel-group non-inferiority study

lead_sponsor: IBSA Institut Biochimique SA

location_country:
Italy
Switzerland
France
Slovakia
Germany
Czech Republic

location_facility: Interni hemato-onkologická klinika, Fakultní nemocnice

location_facility: Orthopädische Universitätsklinik und Poliklinik der RWTH Aachen

location_facility: Centrum léčby pohybového aparátu spol. s.r.o.

location_facility: University Hospital Centre of Limoges - Rheumatology Service

location_facility: Department of Rheumatology and Institute of Physical Medicine - Zurich University

location_facility: National Institute of Rheumatic Diseases

location_facility: Medicina e Traumatologia dello Sport - AzUSL 2 Lucca

location_facility: Unità Operativa di Reumatologia - Az. Ospedaliera Universitaria Senese - Policlinico "Le Scotte"

location_facility: Sezione Dipartimentale a valenza provinciale di Reumatologia - AzUSL 8

location_facility: Unità Operativa di Reumatologia - Istituto Ortopedico Gaetano Pini

location_facility: University Hospital Centre Bois-Guillaume


title: Clinical Study on Tacrolimus Ointment Over the Long-term. "CONTROL Study - Children"

summary: A long-term 0.03% tacrolimus ointment based regimen comprising of up to 6 weeks of initial twice daily treatment and subsequent twice weekly prophylactic application can effectively treat active lesions of atopic dermatitis, and prevent, delay, and reduce flares.

ReferenceCitation: Thaçi D, Reitamo S, Gonzalez Ensenat MA, Moss C, Boccaletti V, Cainelli T, van der Valk P, Buckova H, Sebastian M, Schuttelaar ML, Ruzicka T; European Tacrolimus Ointment Study Group. Proactive disease management with 0.03% tacrolimus ointment for children with atopic dermatitis: results of a randomized, multicentre, comparative study. Br J Dermatol. 2008 Dec;159(6):1348-56. doi: 10.1111/j.1365-2133.2008.08813.x. Epub 2008 Sep 6.

lead_sponsor: Astellas Pharma Inc

location_country:
Italy
United Kingdom
Netherlands
Spain
Finland
France
Hungary
Belgium
Portugal
Germany
Czech Republic


title: A Double-blind, Randomised Study Comparing the Safety and Tolerance of SMOFlipid 20% in Long-term Treatment With Parenteral Nutrition

summary: The aim of this study is to evaluate the safety and tolerance of SMOFlipid 20% in comparison to a standard lipid emulsion Intralipid 20% in patients requiring long-term parenteral nutrition. The safety and tolerance will be evaluated by biochemistry, hematology and coagulation variables, vital signs and adverse events. Further objectives to evaluate are the influence of SMOFlipid 20% on inflammation processes, the efficacy of anti-oxidative properties of vitamin E supplemented to SMOFlipid 20%, and the fatty acid pattern in red blood cells and serum.

lead_sponsor: Fresenius Kabi

location_country:
Netherlands
France
Poland
Israel
Australia
Denmark
United Kingdom

location_facility: Wydzial Lekarski I Katedra Chirurgii Ogolnej

location_facility: Rigshospitalet, Department of Medical Gastroenterology

location_facility: Hopital Edouard Herriot, Unite de Nutrition a Domicile Lyon

location_facility: University Hospital, Queen's Medical Centre

location_facility: Beilinson Medical Center, Department of Surgery "B"

location_facility: Samodzielny Publiczny Szpital Kliniczny im. Prof. W. Orłowskiego

location_facility: Aalborg University Hospital, Department of Medical Gastroenterology


title: A Randomized, Double-Blind Study to Evaluate the Safety of Continued Treatment With CellCept in Patients With Well-Controlled Myasthenia Gravis Receiving a Stable Dose of Prednisone

summary: This 2 arm study will provide optional continuation of double-blind treatment with CellCept or placebo, in patients with myasthenia gravis who have achieved good symptom control in study WX17798. Patients who have completed 36 weeks of treatment in study WX17798, with stable prednisone dosing for the last 4 weeks, can continue on blinded treatment with CellCept (1g bid) or placebo until the database for WX17798 is locked and unblinded. The anticipated time on study treatment is 3-12 months, and the target sample size is 100-500 individuals.

location_country:
Italy
United States
Former Serbia and Montenegro
France
Germany
United Kingdom
Ukraine


title: A Phase I Dose Escalation Study of BMS-690514 in Patients With Advanced or Metastatic Solid Tumors

summary: The purpose of this research study is to determine the highest dose of the drug (BMS-690514) that can be safely given to patients with advanced cancer.

ReferenceCitation: Soria JC, Baselga J, Hanna N, Laurie SA, Bahleda R, Felip E, Calvo E, Armand JP, Shepherd FA, Harbison CT, Berman D, Park JS, Zhang S, Vakkalagadda B, Kurland JF, Pathak AK, Herbst RS. Phase I-IIa study of BMS-690514, an EGFR, HER-2 and -4 and VEGFR-1 to -3 oral tyrosine kinase inhibitor, in patients with advanced or metastatic solid tumours. Eur J Cancer. 2013 May;49(8):1815-24. doi: 10.1016/j.ejca.2013.02.012. Epub 2013 Mar 13.

family: Soria

full_name: Jean-Charles Soria

given: Jean-Charles

family: Baselga

full_name: José Baselga

given: José

family: Hanna

full_name: Nasser Hanna

given: Nasser

family: Laurie

full_name: Scott A. Laurie

given: Scott A.

family: Bahleda

full_name: Rastislav Bahleda

given: Rastislav

family: Felip

full_name: Enriqueta Felip

given: Enriqueta

family: Calvo

full_name: Emiliano Calvo

given: Emiliano

family: Armand

full_name: Jean-Pierre Armand

given: Jean-Pierre

family: Shepherd

full_name: Frances A. Shepherd

given: Frances A.

family: Harbison

full_name: Christopher T. Harbison

given: Christopher T.

family: Berman

full_name: David Berman

given: David

family: Park

full_name: Jong-Soon Park

given: Jong-Soon

family: Zhang

full_name: Steven Zhang

given: Steven

family: Vakkalagadda

full_name: Blisse Vakkalagadda

given: Blisse

family: Kurland

full_name: John F. Kurland

given: John F.

family: Pathak

full_name: Ashutosh K. Pathak

given: Ashutosh K.

family: Herbst

full_name: Roy S. Herbst

given: Roy S.

title: Phase I–IIa study of BMS-690514, an EGFR, HER-2 and -4 and VEGFR-1 to -3 oral tyrosine kinase inhibitor, in patients with advanced or metastatic solid tumours

lead_sponsor: Bristol-Myers Squibb

location_country:
United States
Canada
France
Spain

location_facility: The University Of Texas Md Anderson Cancer Center


title: An Extension to a Phase II Study to Determine the Safety and Anti-Leukemic Effects of STI571 in Adult Patients With Philadelphia Chromosome Positive Leukemia Including Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, and Accelerated Phase Chronic Myeloid Leukemia

summary:
The objectives of Part 1 of the study were:

To determine the rate of hematologic response (HR) lasting ≥4 weeks in participants with Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) in the accelerated phase (AP).
To evaluate duration of HR, overall survival, cytogenetic response (CyR), time to blast crisis in CML participants in the AP, improvement of symptomatic parameters, tolerability and safety of STI571 treatment.

The objective of the extension (Part 2) was:

-To enable participants to have access to study drug and continue study treatment and to decrease data collection to include only overall survival and serious adverse events.

lead_sponsor: Novartis Pharmaceuticals

location_facility: MD Anderson Cancer Center, University of Texas


title: A Randomized Phase II-III Trial Evaluating the Efficacy of Capecitabine and Vinorelbine in Anthracycline and Taxane Pre-Treated Metastatic Breast Cancer

summary:
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. It is not yet known if capecitabine is more effective than vinorelbine in treating metastatic breast cancer.

PURPOSE: Randomized phase II/III trial to compare the effectiveness of capecitabine with that of vinorelbine in treating women who have metastatic breast cancer that has been previously treated with chemotherapy.

ReferenceType: result

ReferenceCitation: Pajk B, Cufer T, Canney P, Ellis P, Cameron D, Blot E, Vermorken J, Coleman R, Marreaud S, Bogaerts J, Basaran G, Piccart M. Anti-tumor activity of capecitabine and vinorelbine in patients with anthracycline- and taxane-pretreated metastatic breast cancer: findings from the EORTC 10001 randomized phase II trial. Breast. 2008 Apr;17(2):180-5. Epub 2007 Oct 31.

ReferenceCitation: Hoon SN, Lau PK, White AM, Bulsara MK, Banks PD, Redfern AD. Capecitabine for hormone receptor-positive versus hormone receptor-negative breast cancer. Cochrane Database Syst Rev. 2021 May 26;5:CD011220. doi: 10.1002/14651858.CD011220.pub2.

name: Medical Oncology Department; St John of God Midland; Perth Australia

name: Medical Oncology Department; Sir Charles Gairdner Hospital; Perth Australia

family: Hoon

full_name: Siao-Nge Hoon

given: Siao-Nge

name: Medical Oncology Department; Peter MacCallum Cancer Centre; Melbourne Australia

family: Lau

full_name: Peter K H Lau

given: Peter K H

name: Murdoch Community Hospice; St John of God Hospital Murdoch; Perth Australia

name: Palliative Care Department; Royal Perth Hospital; Perth Australia

family: White

full_name: Alison M White

given: Alison M

name: Institute for Health Research; University of Notre Dame Australia; Fremantle Australia

name: School of Population and Global Health; The University of Western Australia; Perth Australia

name: Division of Surgery and Interventional Science; University College London; London UK

family: Bulsara

full_name: Max K Bulsara

given: Max K

name: Medical Oncology Department; University Hospital Geelong; Geelong Australia

family: Banks

full_name: Patricia D Banks

given: Patricia D

name: School of Medicine; University of Western Australia; Perth Australia

name: Medical Oncology Department; Fiona Stanley Hospital; Perth Australia

family: Redfern

full_name: Andrew D Redfern

given: Andrew D

journal_name: Cochrane Database of Systematic Reviews

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

location_country:
United Kingdom
France
Belgium
Germany
Slovenia

location_facility: Cancer Research Centre at Weston Park Hospital


title: Randomized Phase III Trial of Two Investigational Schedules of Ifosfamide vs. Standard Dose Doxorubicin in Patients With Advanced or Metastatic Soft Tissue Sarcoma

summary:
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. It is not yet known whether ifosfamide or doxorubicin is more effective for advanced or metastatic soft tissue sarcoma.

PURPOSE: Randomized phase III trial to compare the effectiveness of ifosfamide with that of doxorubicin in treating patients who have advanced or metastatic soft tissue sarcoma.

ReferenceCitation: Lorigan P, Verweij J, Papai Z, Rodenhuis S, Le Cesne A, Leahy MG, Radford JA, Van Glabbeke MM, Kirkpatrick A, Hogendoorn PC, Blay JY; European Organisation for Research and Treatment of Cancer Soft Tissue and Bone Sarcoma Group Study. Phase III trial of two investigational schedules of ifosfamide compared with standard-dose doxorubicin in advanced or metastatic soft tissue sarcoma: a European Organisation for Research and Treatment of Cancer Soft Tissue and Bone Sarcoma Group Study. J Clin Oncol. 2007 Jul 20;25(21):3144-50.

location_country:
Netherlands
France
Poland
Slovakia
Hungary
Belgium
Spain
Denmark
Germany
United Kingdom

location_facility: Middlesex Hospital- Meyerstein Institute

location_facility: Maria Sklodowska-Curie Memorial Cancer Center and Institute of Oncology


title: Phase II Study of the Efficacy of rH Interleukin-2 in Patients With Slowly Progressing Acute Myelogenous Leukemia (AML) and With Limited Bone Marrow Blastosis After Autologous Stem Cell Transplantation or Chemotherapy

summary:
RATIONALE: Interleukin-2 may stimulate a person's white blood cells to kill acute myelogenous leukemia cells.

PURPOSE: Phase II trial to study the effectiveness of interleukin-2 in treating patients with acute myelogenous leukemia that has relapsed following previous treatment.

location_country: Italy Austria Netherlands Croatia France Belgium

location_facility:
Leiden University Medical Center
Hotel Dieu de Paris
Ospedale San Eugenio
Institut Jules Bordet
Innsbruck Universitaetsklinik
Universitair Ziekenhuis Antwerpen
University Hospital Rebro
Azienda Policlinico Umberto Primo
University Medical Center Nijmegen
Institut Gustave Roussy


title: An Open-Label Extension Study to Evaluate the Safety and Tolerability of QR 421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene (Helia)

acronym: HELIA

summary: PQ-421a-002 (Helia) is an open-label, extension study to evaluate the safety, tolerability and efficacy of QR 421a administered via intravitreal (IVT) injection in one or both eyes, in subjects ≥ 18 years of age with RP due to mutations in exon 13 of the USH2A gene, for an anticipated period of 24 months, or until provision of continued treatment by other means is available, provided the subject's benefit-risk determination remains positive.

status: Enrolling by invitation

lead_sponsor: ProQR Therapeutics

location_country:
United States
Canada
France

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

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

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

location_facility: Casey Eye Institute, Oregon Health & Science University

location_facility: University of Michigan, Kellogg Eye Center

location_facility: Centre de maladies rares CHNO des Quinze Vingts


title: A Phase III, Multi-center, Open-label, Randomized Study of Oral Asciminib Versus Investigator Selected TKI in Patients With Newly Diagnosed Philadelphia Chromosome Positive Chronic Myelogenous Leukemia in Chronic Phase

summary: The study is designed to compare the efficacy of asciminib 80 mg QD versus Investigator selected TKI for the treatment of newly diagnosed, previously untreated patients with Ph+ CML-CP. The Investigator selected TKI will be one of the following treatment options for first-line treatment of CML-CP - imatinib 400 mg QD or nilotinib 300 mg BID or dasatinib 100 mg QD or bosutinib 400 mg QD.

status: Recruiting

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 are 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: France Czechia Singapore Hungary Australia Spain Germany


lead_sponsor: Mirum Pharmaceuticals, Inc.

location_country:
France
Poland
Belgium
United States
United Kingdom

acronym: RISE

location_facility:
Seattle Children's Hospital
Hopital Necker
Hôpital Kremlin Bicêtre
Cliniques Universitaires Saint-Luc
Medstar Georgetown University Hospital
King's College Hospital
Instytut Pomnik-Centrum Zdrowia Dziecka
Children's Hospital of Pittsburgh
Children Hospital LA

title: Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome

summary: This study is designed to assess whether the investigational drug maralixibat, is safe and well tolerated in children <12 months of age with Alagille Syndrome [ALGS] or Progressive Familial Intrahepatic Cholestasis [PFIC].


title: A Phase 1/2, Open-Label, Multicenter Study of INCB000928 Administered as a Monotherapy in Participants With Anemia Due to Myelodysplastic Syndromes or Multiple Myeloma

summary: This Phase 1/2, open-label, dose-finding study is intended to evaluate the safety and tolerability, PK, PD, and efficacy of INCB000928 administered as monotherapy in participants with MDS or MM who are transfusion-dependent or present with symptomatic anemia.

ipd_sharing_description: Incyte shares data with qualified external researchers after a research proposal is submitted. These requests are reviewed and approved by a 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.

lead_sponsor: Incyte Corporation

location_country:
Italy
United States
France

location_facility: Hospices Civils de Lyon Centre Hospitalier Lyon Sud

location_facility: Centre Hospitalier Universitaire de Nantes (Chu de Nantes) - Hotel-Dieu

location_facility: Azienda Ospedaliero-Universitaria Careggi (Aouc)


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

name: Gene Medicine Group, Nuffield Division of Clinical Laboratory Science, Radcliffe Department of Medicine, University of Oxford, Oxford, UK

family: Miah

full_name: Kamran M. Miah

given: Kamran M.

family: Hyde

full_name: Stephen C. Hyde

given: Stephen C.

family: Gill

full_name: Deborah R. Gill

given: Deborah R.

journal_name: Expert Review of Respiratory Medicine

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

version: submittedVersion

publisher: Informa UK Limited

publisher_dissemination: Informa

publisher_group: Informa

publisher_normalized: Informa

acronym: Stellar

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

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


lead_sponsor: Bayer

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

location_facility: Haematology Service, Canterbury Health Laboratories

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

location_facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

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

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


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

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

time_perspective: Retrospective

location_country:
Italy
United States
France
Turkey
Germany
United Kingdom
Canada

location_facility: Ann and Robert H Lurie Children's Hospital

location_facility: Bretonneau Hospital, Internal Medicine Department

location_facility: Emory University Department of Human Genetics

location_facility: University Klinik Jena, Klinik für Neuropädiatrie

location_facility: Adult Metabolic Diseases Clinic, Vancouver General Hospital

location_facility: National Hospital for Neurology and Neurosurgery

location_facility: Rheumatology Unit, Clinic for Inborn Errors of Metabolism


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

summary:
Primary Objectives:

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

Secondary Objectives:

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

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

lead_sponsor: Sanofi

location_country:
Italy
France
United States
United Kingdom
Taiwan

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


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

acronym: REALITY

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

time_perspective: Cross-Sectional

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

family: Newman

full_name: Nancy J. Newman

given: Nancy J.

family: Yu-Wai-Man

full_name: Patrick Yu-Wai-Man

given: Patrick

family: Carelli

full_name: Valerio Carelli

given: Valerio

family: Biousse

full_name: Valerie Biousse

given: Valerie

family: Moster

full_name: Mark L. Moster

given: Mark L.

family: Vignal-Clermont

full_name: Catherine Vignal-Clermont

given: Catherine

family: Sergott

full_name: Robert C. Sergott

given: Robert C.

family: Klopstock

full_name: Thomas Klopstock

given: Thomas

family: Sadun

full_name: Alfredo A. Sadun

given: Alfredo A.

family: Girmens

full_name: Jean-François Girmens

given: Jean-François

family: La Morgia

full_name: Chiara La Morgia

given: Chiara

family: DeBusk

full_name: Adam A. DeBusk

given: Adam A.

family: Jurkute

full_name: Neringa Jurkute

given: Neringa

family: Priglinger

full_name: Claudia Priglinger

given: Claudia

family: Karanjia

full_name: Rustum Karanjia

given: Rustum

family: Josse

full_name: Constant Josse

given: Constant

family: Salzmann

full_name: Julie Salzmann

given: Julie

family: Montestruc

full_name: François Montestruc

given: François

family: Roux

full_name: Michel Roux

given: Michel

family: Taiel

full_name: Magali Taiel

given: Magali

family: Sahel

full_name: José-Alain Sahel

given: José-Alain

currency_apc_doaj: USD

journal_name: Frontiers in Neurology

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

location_country:
Italy
France
Spain
United States
United Kingdom

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