Gaucher Disease: Clinical Trials

11 trials for Gaucher Disease, newest first

ClinicalTrials Daily  ·  Areas  ·  Conditions  ·  Drugs  ·  Sponsors  ·  

8 completed, 2 withdrawn, 1 unknown. 11 Phase 4.

By phase and status

PhaseTrialsStatus
Phase 411Completed 8, Withdrawn 2, Unknown 1

Follow Gaucher Disease: new trials by email Literature Review of Gaucher Disease trials Research Report on Gaucher Disease

Trials (filtered) newest first · largest first

All 11 trials

Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

TrialPhaseStatusSponsorEnrollmentStartUpdated
A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy
Digital Engagement Application (GD App), No Intervention - Gaucher Disease
Phase 4CompletedTakeda42021-04-222023-12-21
Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ
Cerezyme® / Imiglucerase - Gaucher's Disease
Phase 4CompletedSanofi122021-03-022025-09-15
A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease
Elelyso - Gaucher Disease, Type 3
Phase 4CompletedAri Zimran142020-11-202024-12-17
Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease
Elelyso 60 units/kg - Type 1 Gaucher Disease
Phase 4WithdrawnPfizer2019-07-312019-09-24
Chronic Hepatitis C Treatment in Egyptian Children With Gaucher Disease.
Ledipasvir/Sofosbuvir - Gaucher Disease, HCV
Phase 4UnknownMansoura University Children Hospital102018-04-032018-10-30
Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease
Velaglucerase alfa, Vitamin D - Gaucher Disease
Phase 4CompletedShire212016-06-292022-02-01
Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)
VPRIV - Gaucher Disease, Type 1
Phase 4CompletedShaare Zedek Medical Center152016-01-102022-10-28
The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease
Velaglucerase alfa - Gaucher Disease Type 1, Gaucher Disease Type 3
Phase 4WithdrawnBaylor Research Institute2015-07-012018-03-02
A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
Taliglucerase alfa - Gaucher Disease
Phase 4CompletedPfizer112010-08-012018-10-05
Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease
Cerezyme - Gaucher Disease, Type 1, Cerebroside Lipidosis Syndrome
Phase 4CompletedGenzyme, a Sanofi Company952001-12-012015-04-03
A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.
Cerezyme (imiglucerase for injection) - Gaucher Disease Type I, Cerebroside Lipidosis Syndrome
Phase 4CompletedGenzyme, a Sanofi Company401997-12-012015-03-05

Broader conditions

Brain Diseases (11)  ·  Brain Diseases, Metabolic (11)  ·  Brain Diseases, Metabolic, Inborn (11)  ·  Central Nervous System Diseases (11)  ·  Congenital, Hereditary, and Neonatal Diseases and Abnormalities (11)  ·  Genetic Diseases, Inborn (11)  ·  Lipid Metabolism Disorders (11)  ·  Lipid Metabolism, Inborn Errors (11)  ·  Lipidoses (11)  ·  Lysosomal Storage Diseases (11)  ·  Lysosomal Storage Diseases, Nervous System (11)  ·  Metabolic Diseases (11)  ·  Metabolism, Inborn Errors (11)  ·  Nervous System Diseases (11)  ·  Nutritional and Metabolic Diseases (11)

Interventions

Glucosylceramidase (3)  ·  imiglucerase (3)  ·  taliglucerase alfa (2)  ·  Injections (1)  ·  Vitamin D (1)  ·  ledipasvir, sofosbuvir drug combination (1)

Sponsors

Genzyme, a Sanofi Company (2)  ·  Pfizer (2)  ·  Ari Zimran (1)  ·  Baylor Research Institute (1)  ·  Mansoura University Children Hospital (1)  ·  Sanofi (1)  ·  Shaare Zedek Medical Center (1)  ·  Shire (1)  ·  Takeda (1)

Countries

United States (6)  ·  Israel (3)  ·  Spain (2)  ·  United Kingdom (2)  ·  Brazil (1)  ·  Canada (1)  ·  China (1)  ·  Egypt (1)  ·  India (1)  ·  Italy (1)  ·  Paraguay (1)  ·  Poland (1)  ·  South Africa (1)  ·  Turkey (Türkiye) (1)

Top investigators

Ari Zimran (1)  ·  Ari Zimran, Prof. (1)  ·  Edward Kaye, M.D. (1)  ·  Raphael Schiffmann, M.D.,M.H.Sc. (1)

Results publications

PMID 25453586, PMID 29989200

NIH funding

2015 (1)