Clinical trials: Neurogenetic Diseases
HSP, Hereditary Spastic Paraplegia
Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)
Safety and Efficacy of AAV9/AP4B1 For Patients with AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1/2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration.
- Conditions
- HSP, Hereditary Spastic Paraplegia, Hereditary Spastic Paraparesis, Hereditary Spastic Paraplegia Type 50
- Trial name
- NCT06948019
- Testing
- BFB-101 (AAV9-CBh-AP4B1)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- BlackfinBio Ltd
Alzheimer's Disease
Neurogenetic Pharmaceuticals (NGP) 555 in Healthy Young Volunteers (Single-ascending Dose)
NGP 555 is a small molecule preventative therapy aimed at reducing Alzheimer's disease amyloid buildup by targeting Abeta 42 production.
- Trial name
- NCT02534480
- Testing
- NGP 555
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- NeuroGenetic Pharmaceuticals Inc
Spinal Muscular Atrophy (SMA)
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
This is a global, prospective, multi-center study that is designed to assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial.
- Trial name
- SPECTRUM (NCT05335876)
- Testing
- onasemnogene abeparvovec
- Where
- 32 sites in United States, Australia and 17 more
- Volunteers
- 20 people
- Sponsor
- Novartis Pharmaceuticals
Alzheimer's Disease
Neurogenetic Pharmaceuticals (NGP) 555 in Healthy Volunteers (14 Day Multiple Ascending Dose)
This study involves the use of an investigational drug called NGP 555. In each group of healthy subjects, 2 people will receive placebo and 6 people will receive NGP 555.
- Trial name
- NCT02537938
- Testing
- NGP 555
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- NeuroGenetic Pharmaceuticals Inc
Charcot-Marie-Tooth Disease
A Study to Assess the Safety and Effectiveness of FLX-787 in Subjects With Charcot-Marie-Tooth Disease Experiencing Muscle Cramps.
The COMMIT Study will assess the safety and effectiveness of FLX-787 in men and women with Charcot-Marie-Tooth disease (CMT) experiencing muscle cramps.
- Trial name
- COMMIT (NCT03254199)
- Testing
- FLX-787-ODT (orally disintegrating tablet)
- Where
- 20 sites in United States
- Volunteers
- 27 people
- Sponsor
- Flex Pharma, Inc.