Clinical trials: Gaucher's Disease
Gaucher Disease
A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
A protocol to extend the assessment of the safety and efficacy of taliglucerase alfa in pediatric subjects (2 to <18 years old) with symptoms and clinical manifestations of Gaucher disease who completed treatment in Protocols…
- Trial name
- NCT01411228
- Testing
- Taliglucerase alfa
- Where
- 3 sites in Israel, Paraguay and 1 more
- Volunteers
- 15 people
- Sponsor
- Pfizer
Gaucher Disease, Gaucher Disease, Type 1
A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)
Study J3Z-MC-OJAE is a Phase 1/2, multicenter, open-label, dose-finding study of LY3884961 evaluating the safety and tolerability in adults with peripheral manifestations of GD.
- Trial name
- NCT05487599
- Testing
- LY3884961
- Where
- 5 sites in United States, Australia and 2 more
- Volunteers
- 8 people
- Sponsor
- Prevail Therapeutics
Gaucher Disease
Omics Gaucher Study: Multiomic Approach
The study aims to investigate the transcriptomic and metabolomic changes in blood, plasma and isolated monocytes from Gaucher patients and healthy controls.
- Trial name
- OmicsGaucher (NCT05526664)
- Testing
- Genetic testing and Omics analysis
- Where
- Germany
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- CENTOGENE GmbH Rostock
Gaucher Disease, Type 1 Gaucher Disease
A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease
This study evaluated the safety and tolerability of afegostat tartrate in participants with type 1 Gaucher disease who were not receiving enzyme replacement therapy (ERT) or substrate reduction therapy (SRT).
- Conditions
- Gaucher Disease, Type 1 Gaucher Disease, Gaucher Disease, Type 1
- Trial name
- NCT00446550
- Testing
- afegostat tartrate
- Where
- 11 sites in United States, Israel and 2 more
- Volunteers
- 19 people
- Sponsor
- Amicus Therapeutics
Gaucher Disease, Type 1
A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to this deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular…
- Trial name
- NCT00430625
- Testing
- VPRIV ®,
- Where
- 5 sites in Argentina, Israel and 3 more
- Volunteers
- 25 people
- Sponsor
- Shire
Gaucher Disease
Gaucher Disease Outcome Survey (GOS)
The Gaucher Outcomes Survey (GOS) is an ongoing observational, international, multi-center, long-term Registry of Patients with Gaucher Disease irrespective of their treatment status or type of treatment received.
- Trial name
- NCT03291223
- Where
- United States
- Volunteers
- 1,257 people
- Sponsor
- Shire
Gaucher Disease, Splenomegaly
A National Study in Patients With Unexplained Splenomegaly
Primary Objective: To estimate the prevalence of Gaucher disease and of other etiologies, in patients of 15 years old or more presenting with unexplained splenomegaly after exclusion of first intention-diagnoses (e.g.
- Trial name
- SMS (NCT04430881)
- Where
- France
- Volunteers
- 506 people
- Sponsor
- Sanofi
Gaucher Disease
Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular…
- Trial name
- NCT00478647
- Testing
- GA-GCB (velaglucerase alfa)
- Where
- 15 sites in United States, Israel and 3 more
- Volunteers
- 40 people
- Sponsor
- Shire
Niemann-Pick Disease Type C, GM2 Gangliosidosis
Long-Term Safety and Tolerability of Nizubaglustat in Participants With Rare Lysosomal Disorders
This is an open-label extension trial to evaluate the long-term safety and tolerability of nizubaglustat (AZ-3102) in participants with rare lysosomal disorders who have participated in a previous nizubaglustat trial and wish to…
- Conditions
- Niemann-Pick Disease Type C, GM2 Gangliosidosis, GM1 Gangliosidosis, Gaucher Disease Type 3
- Trial name
- HARBOR (NCT07885917)
- Testing
- AZ-3102
- Volunteers
- 218 people
- Sponsor
- Azafaros B.V.
Gaucher Disease
Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies
Primary Objective: Evaluate long term skeletal response to eliglustat in adult participants who successfully completed one of the Phase 2 or Phase 3 eliglustat studies.
- Trial name
- EXOSKEL (NCT02536755)
- Testing
- Eliglustat, GZ385660
- Where
- 4 sites in Canada, Russia and 1 more
- Volunteers
- 31 people
- Sponsor
- Genzyme, a Sanofi Company
Type 3 Gaucher Disease
Managed Access Program of Venglustat in Pediatric and Adult Patients With Type 3 Gaucher Disease (GD3).
The objective of this program is to provide early access to venglustat for certain patients with Gaucher disease type 3 in response to unsolicited requests from healthcare providers.
- Trial name
- NCT07675031
- Testing
- GZ402671
- Sponsor
- Sanofi
Gaucher Disease, Type 1
Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular…
- Trial name
- NCT00954460
- Testing
- velaglucerase alfa
- Where
- 32 sites in United States
- Sponsor
- Shire
Gaucher Disease
A Phase I Study to Compare Abcertin and EU-sourced Cerezyme® in Healthy Volunteers
Primary Objective: To compare the pharmacokinetics of Abcertin to the reference product, EU-sourced Cerezyme, after single intravenous administration of 60 IU/kg.
- Trial name
- NCT04787887
- Testing
- Abcertin, EU-sourced Cerezyme
- Where
- Australia
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- ISU Abxis Co., Ltd.
Gaucher Disease Type 1, Gaucher Disease Type 3
Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension
Part 1: Biomarker evaluation/screening phase Primary Objectives: Evaluate cerebrospinal fluid (CSF) biomarkers in adult Gaucher disease Type 3 (GD3) participants that distinguish GD3 from adult Gaucher disease Type 1 (GD1)…
- Trial name
- LEAP (NCT02843035)
- Testing
- venglustat (GZ402671), imiglucerase
- Where
- 7 sites in United States, Germany and 2 more
- Volunteers
- 12 people
- Sponsor
- Genzyme, a Sanofi Company
Gaucher Disease, Splenomegaly, Acid SphingoMyelinase Deficiency
A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen
Primary Objective: To assess prevalence of Gaucher disease (GD) diagnosed in pediatric patients presenting with unexplained splenomegaly (SMG) after exclusion of first intention-diagnoses (e.g.
- Trial name
- OPPUS (NCT04845958)
- Where
- 40 sites in France
- Volunteers
- 60 people
- Sponsor
- Sanofi
Gaucher Disease, Type 1
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)
This Phase 3 study was designed to confirm the efficacy and safety of eliglustat tartrate (Genz-112638) in participants with Gaucher disease Type 1.
- Trial name
- NCT00891202
- Testing
- Eliglustat tartrate
- Where
- 18 sites in United States, Bulgaria and 10 more
- Volunteers
- 40 people
- Sponsor
- Genzyme, a Sanofi Company
Gaucher's Disease
Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ
Primary Objective To evaluate the efficacy on hematologic manifestations of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ To evaluate the safety profile of imiglucerase in maximum dose in…
- Trial name
- SEED (NCT04656600)
- Testing
- Cerezyme® / Imiglucerase
- Where
- 5 sites in China
- Volunteers
- 12 people
- Sponsor
- Sanofi
Gaucher Disease, Cerebroside Lipidosis Syndrome
International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
The ICGG Gaucher Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Gaucher disease, irrespective of treatment status.
- Conditions
- Gaucher Disease, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease
- Trial name
- NCT00358943
- Where
- 317 sites in United States, Argentina and 50 more
- Volunteers
- 12,000 people
- Sponsor
- Genzyme, a Sanofi Company
Gaucher Disease
Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease
This is an open-label expanded access trial of prGCD in patients with Gaucher disease who require enzyme replacement therapy (ERT) and who have been treated with imiglucerase but for whom the dose has been reduced or discontinued…
- Trial name
- NCT00962260
- Testing
- Plant cell expressed recombinant glucocerebrosidase (prGCD)
- Where
- 15 sites in United States, Israel
- Sponsor
- Pfizer
Gaucher Disease
The Safety and Efficacy Study of ISU302 in Patient With Type I Gaucher Disease
The purpose of this study is to compare and evaluate the efficacy and safety of ISU302, an investigational product, and Cerezyme®, comparator, for Type 1 Gaucher Disease patients
- Trial name
- NCT01161914
- Testing
- Cerezyme®, ISU302
- Sponsor
- ISU Abxis Co., Ltd.