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Active & Responsive

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy

for
Muscular Atrophy, Spinal
Location: 10 recruiting locations
Sponsor: Hoffmann-La Roche

Sex: All

Age: 0 - 2

Code: NCT05861999

Recruiting
Active & Responsive

A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations

for
Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF),
Location: 9 recruiting locations
Sponsor: Regeneron Pharmaceuticals

Sex: All

Age: 0 - 17

Code: NCT05788536

Phase1, Phase2, Recruiting
Active & Responsive

AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy

for
Mesial Temporal Lobe Epilepsy
Location: 16 recruiting locations
Sponsor: UniQure Biopharma B.V.

Sex: All

Age: 18 - 70+

Code: NCT06063850

Phase1, Phase2, Recruiting
Active & Responsive

A Study to Evaluate the Safety and Effect of AVB-101, a Gene Therapy Product, in Subjects With a Genetic Sub-type of Frontotemporal Dementia (FTD-GRN)

for
Frontotemporal Dementia, FTD, FTD-GRN, Dementia, Frontotemporal
Location: 4 recruiting locations
Sponsor: AviadoBio Ltd

Sex: All

Age: 30 - 70+

Code: NCT06064890

Phase1, Phase2, Recruiting
Active & Responsive

ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis

for
X-linked Retinoschisis
Location: 4 recruiting locations
Sponsor: Atsena Therapeutics Inc.

Sex: Male

Age: 6+

Code: NCT05878860

Phase1, Phase2, Recruiting

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Active & Responsive

A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

for
Danon Disease
Location: 3 recruiting locations
Sponsor: Rocket Pharmaceuticals Inc.

Sex: Male

Age: 8+

Code: NCT06092034

Phase2, Recruiting
Active & Responsive

Gene Therapy for Alpha-1 Antitrypsin Deficiency

for
Alpha 1-Antitrypsin Deficiency
Location: 1 recruiting location
Sponsor: Weill Medical College of Cornell University

Sex: All

Age: 18 - 70

Code: NCT06996756

Phase1, Recruiting
Active & Responsive

Study of EN-374 Gene Therapy in Participants With X-Linked Chronic Granulomatous Disease

for
X-Linked Chronic Granulomatous Disease
Location: 4 recruiting locations
Sponsor: Ensoma

Sex: Male

Age: 0+

Code: NCT06876363

Phase1, Phase2, Recruiting
Active & Responsive

Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia

for
Friedreich Ataxia, Cardiomyopathies, Cardiac Hypertrophy,
Location: 1 recruiting location
Sponsor: Weill Medical College of Cornell University

Sex: All

Age: 18 - 50

Code: NCT05302271

Phase1, Recruiting
Active & Responsive

Anti-VEGF Gene Therapy Trial for Vestibular Schwannoma

for
Vestibular Schwannoma
Location: 4 recruiting locations
Sponsor: Akouos, Inc.

Sex: All

Age: 18+

Code: NCT06517888

Phase1, Phase2, Recruiting