Clinical trials: GBA Gene Mutation
Parkinson's Disease
Study Assessing Tolerability and Safety and Exploring the Immunogenicity and Therapeutic Activity of AFFITOPE® PD01A in PD-GBA Patients
This is a randomized, placebo-controlled, parallel group, patient-blind, single-center phase I clinical trial of repeated once every 4 weeks administration by subcutaneous injection of AFFITOPE® PD01A, adsorbed to aluminium oxide…
- Trial name
- NCT02758730
- Testing
- AFFITOPE® PD01A + Adjuvant, Adjuvant without active component
- Where
- Germany
- Sponsor
- Affiris AG
Parkinson's Disease
A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of Venglustat (GZ/SAR402671) in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene Mutation
Primary Objectives: Part 1: To determine the safety and tolerability of 4, 8, and 15 milligrams of GZ/SAR402671 (venglustat) administered orally for 4 weeks, as compared to placebo in participants with early-stage Parkinson's…
- Trial name
- MOVES-PD (NCT02906020)
- Testing
- venglustat GZ/SAR402671
- Where
- 52 sites in United States, Austria and 14 more
- Volunteers
- 273 people
- Sponsor
- Genzyme, a Sanofi Company
GBA1 Parkinson Disease
A Clinical Trial of CAP-003 Gene Therapy in Adult Patients With GBA1 Associated Parkinson's Disease
The goal of this clinical trial is to learn about the safety of CAP-003 gene therapy in adults with GBA1 associated Parkinson's Disease.
- Trial name
- NCT07011771
- Testing
- gene therapy
- Where
- 5 sites in United States
- Sponsor
- Capsida Biotherapeutics, Inc.
Gaucher Disease, Type 2
Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
J3Z-MC-OJAB is an open-label, Phase 1/2, multicenter study to evaluate the safety and efficacy of single-dose LY3884961 (formerly PR001) in infants diagnosed with Type 2 Gaucher disease (GD2).
- Trial name
- NCT04411654
- Testing
- LY3884961, Methylprednisolone, Sirolimus
- Where
- 6 sites in United States, United Kingdom
- Volunteers
- 7 people
- Sponsor
- Prevail Therapeutics
Parkinson Disease, GBA Gene Mutation
High-Dose Ambroxol in GBA1-Related Parkinson
Parkinson's disease (PD), affecting 10 million people globally, lacks a cure, and current therapies only manage symptoms. A link between Gaucher disease (GD) and PD, particularly in carriers of glucocerebrosidase (GBA1)…
- Trial name
- NCT06193421
- Testing
- Ambroxol Hydrochloride
- Where
- Israel
- Volunteers
- 40 people
- Sponsor
- Agyany Pharma LTD
Parkinson Disease
Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)
Study J3Z-MC-OJAA is a Phase 1/2a, multicenter, open-label, ascending dose, first in-human study that will evaluate the safety of intracisternal LY3884961 administration in patients with moderate to severe Parkinson's disease…
- Trial name
- NCT04127578
- Testing
- LY3884961, Methylprednisolone, Sirolimus
- Where
- 15 sites in United States, Israel
- Volunteers
- 32 people
- Sponsor
- Prevail Therapeutics