Clinical trials: Gaucher Disease
Gaucher Disease, Type 1
Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)
This is a multicenter, long-term, follow-up trial of participants with Gaucher disease type 1 who received FLT201 treatment in a preceding clinical trial. Participants will be followed for 5 years post-treatment.
- Trial name
- NCT06545136
- Testing
- FLT201
- Where
- 4 sites in United States, Brazil and 2 more
- Volunteers
- 75 people
- Sponsor
- Spur Therapeutics
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 Disease, Type 1
An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease
The purpose of this study is to evaluate the long-term safety of every other week dosing of Gene-Activated® human glucocerebrosidase (GA-GCB, velaglucerase alfa) intravenously in patients with type 1 Gaucher disease.
- Trial name
- NCT00635427
- Testing
- VPRIV®
- Where
- 21 sites in United States, Argentina and 9 more
- Volunteers
- 95 people
- Sponsor
- Shire
Gaucher Disease, Type 1, Gaucher Disease, Type 3
Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease
Gaucher disease is a rare lysosomal storage disorder caused by deficient activity of the enzyme acid β-glucosidase, causing glucosylceramide to accumulate within macrophages and leading to hepatosplenomegaly, anemia…
- Trial name
- NCT05447494
- Testing
- Low-dose CAN103, High-dose CAN103
- Where
- China
- Volunteers
- 40 people
- Sponsor
- CANbridge (Suzhou) Bio-pharma Co., Ltd.
Gaucher Disease Type 1
A Gaucher Disease Gene Therapy Trial With FLT201
This study is a Phase 3, non-randomized, multicenter, efficacy and safety study in adult patients with Gaucher disease Type 1, on stable treatment with enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at…
- Trial name
- GALILEO-3 (NCT07223944)
- Testing
- FLT201
- Where
- 32 sites in United States, Argentina and 8 more
- Volunteers
- 45 people
- Sponsor
- Spur Therapeutics
Gaucher Disease, Type 3
High-Dose Ambroxol in Pediatric Type III Gaucher Disease (GD3)
Type: Prospective, open-label, single center study Duration: 6 months with an optional 12-month extension phase Participants: 12 pediatric patients diagnosed with type III Gaucher disease (GD3) aged ≥3 to ≤18 years old treatment…
- Trial name
- NCT07285369
- Testing
- Ambroxol
- Where
- Pakistan
- Volunteers
- 12 people
- Sponsor
- Agyany Pharma LTD
Gaucher's Disease
A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth
A study to assess the absorption of eliglustat through the mouth in healthy subjects and the safety of any systemic exposure resulting from oral surface absorption of eliglustat in healthy subjects.
- Trial name
- Acronym (NCT06193304)
- Testing
- Eliglustat
- Where
- United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Sanofi
Type 1 Gaucher Disease
A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02
This is a multicenter, multinational long-term follow-up study to assess the long-term safety and durability of effect of AVR-RD-02 treatment in subjects who previously received AVR-RD-02 treatment (single dose administration).
- Trial name
- NCT04836377
- Testing
- Safety and efficacy assessments
- Where
- 3 sites in United States, Canada
- Volunteers
- 4 people
- Sponsor
- AVROBIO
Gaucher Disease
Phase I Single Dose-Escalation Safety Study of Human Glucocerebrosidase (prGCD)
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD)leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the…
- Trial name
- NCT00258778
- Testing
- Human Glucocerebrosidase (prGCD)
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Protalix
Gaucher Disease
A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home Infusion
The main purpose of this survey is to determine participants', caregivers', and nurses' understanding and use of educational materials (EM) on VPRIV home treatment.
- Trial name
- NCT05669729
- Volunteers
- 60 people
- Sponsor
- Takeda
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
Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease
Although miglustat has been approved as a treatment for mild to moderate type 1 Gaucher disease in patients who are unsuitable for enzyme replacement therapy (ERT), more data are required to establish the long term efficacy…
- Trial name
- NCT00319046
- Testing
- Miglustat
- Volunteers
- 42 people
- Sponsor
- Actelion
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
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
Safety and Pharmacokinetics of Oral PRX-112 in Gaucher Disease Patients
Absorption of therapeutic proteins taken orally has remained the major hurdle for treatment in humans. The proteins are generally degraded by enzymes in the stomach and intestine and the intestine lining that prevents absorption…
- Trial name
- NCT01747980
- Testing
- PRX-112
- Where
- 2 sites in Israel
- Volunteers
- 16 people
- Sponsor
- Protalix
Lysosomal Storage Diseases, Gaucher Disease
Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease
International, multicenter, epidemiological study to demonstrate the correlation and predictive value of lyso-Gb1 concentration with the clinical severity of naïve, initially non-ERT/SRT Gaucher disease type 1 and during the…
- Conditions
- Lysosomal Storage Diseases, Gaucher Disease, Sphingolipidoses
- Trial name
- LYSO-PROOF (NCT02416661)
- Where
- 8 sites in Albania, Greece and 5 more
- Volunteers
- 299 people
- Sponsor
- CENTOGENE GmbH Rostock
Gaucher Disease
Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease
Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB) that leads to progressive accumulation of glucocerebroside within macrophages and subsequent tissue and organ damage; typically of the…
- Trial name
- NCT01614574
- Testing
- velaglucerase alfa
- Where
- 3 sites in Japan
- Volunteers
- 6 people
- Sponsor
- Shire
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
Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease
Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB) that leads to progressive accumulation of glucocerebroside within macrophages and subsequent tissue and organ damage; typically of the…
- Trial name
- NCT01842841
- Testing
- velaglucerase alfa
- Where
- 5 sites in Japan
- Volunteers
- 5 people
- Sponsor
- Shire
Gaucher Disease Type I, Cerebroside Lipidosis Syndrome
A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.
This is a multicenter, open-label, prospective study of the efficacy of Cerezyme in treating patients with skeletal manifestations secondary to Type I Gaucher disease.
- Conditions
- Gaucher Disease Type I, Cerebroside Lipidosis Syndrome, Clucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease
- Trial name
- NCT00365131
- Testing
- Cerezyme (imiglucerase for injection)
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Genzyme, a Sanofi Company