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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: Philadelphia PA, Gainesville FL, Atlanta GA,
Sponsor: Hoffmann-La Roche

Sex: All

Age: 0 - 2

Code: NCT05861999

Recruiting
Featured

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

for
Muscular Atrophy, Spinal
Location: New York NY, Philadelphia PA, Gainesville FL,
Sponsor: Hoffmann-La Roche

Sex: All

Age: 0 - 2

Code: NCT05861986

Recruiting
Featured

A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease

for
Huntington Disease
Location: Boston MA, Philadelphia PA, Pittsburgh PA, Columbus OH,
Sponsor: Hoffmann-La Roche

Sex: All

Age: 25 - 65

Code: NCT06826612

Phase1, Phase2, Recruiting
Featured

A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A

for
Hemophilia A
Location: Orange CA, Vallejo CA, San Francisco CA
Sponsor: Hoffmann-La Roche

Sex: Male

Age: 18+

Code: NCT07226206

Phase1, Phase2, Recruiting
Featured

A Study Evaluating the Safety, Activity, and Pharmacokinetics of Divarasib as a Single Agent or in Combination With Other Anti-Cancer Therapies in Participants With Previously Untreated Advanced or Metastatic Non-Small Cell Lung Cancer With a KRAS G12C Mutation

for
Non-Small Cell Lung Cancer
Location: New Haven CT, Mineola NY, The Bronx NY, New York NY,
Sponsor: Hoffmann-La Roche

Sex: All

Age: 18+

Code: NCT05789082

Phase1, Phase2, Recruiting

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Featured

A Study of Multiple Therapies in Biomarker-selected Participants With Resectable Stages IB-III Non-small Cell Lung Cancer (NSCLC)

for
Non-small Cell Lung Cancer
Location: Boston MA, New Haven CT, Lebanon NH, New York NY,
Sponsor: Genentech, Inc.

Sex: All

Age: 18+

Code: NCT04302025

Phase2, Recruiting
Featured

A Study to Assess Anti-Tumor Activity of Intravenously (IV) Infused Carboplatin With Mirvetuximab Soravtansine in Participants With Newly Diagnosed Folate Receptor Alpha (FRα)Expressing Advanced-Stage Serous Epithelial Ovarian, Fallopian Tube or Primary Peritoneal Cancer.

for
Epithelial Ovarian Cancer, Fallopian Tube Cancer,
Location: Providence RI, New Haven CT, Lebanon NH, Bay Shore NY,
Sponsor: AbbVie

Sex: Female

Age: 18+

Code: NCT06890338

Phase2, Recruiting
Featured

A Study to Evaluate Efficacy and Safety of Giredestrant Compared With Fulvestrant (Plus a CDK4/6 Inhibitor), in Participants With ER-Positive, HER2-Negative Advanced Breast Cancer Resistant to Adjuvant Endocrine Therapy (pionERA Breast Cancer)

for
Estrogen Receptor-Positive, HER2-Negative Advanced Breast Cancer,
Location: Bensalem PA, Philadelphia PA, Annapolis MD, Raleigh NC,
Sponsor: Hoffmann-La Roche

Sex: All

Age: 18+

Code: NCT06065748

Phase3, Recruiting
Active & Responsive

A Study to Evaluate a Novel Gene Therapy in Patients With Relapsed and Refractory Multiple Myeloma

for
Multiple Myeloma in Relapse, Myeloma Multiple,
Location: Boston MA, Hackensack NJ, Tampa FL, Atlanta GA,
Sponsor: Kelonia Therapeutics, Inc.

Sex: All

Age: 18+

Code: NCT07075185

Phase1, Recruiting
Active & Responsive

Long-Term Follow-Up of Subjects Treated With Seattle Children's Therapeutics Gene Therapy Products

for
CAR T Cell, CAR T Cell Therapy
Location: Seattle WA
Sponsor: Seattle Children's Hospital

Sex: All

Age: 0+

Code: NCT07424157

Recruiting
Active & Responsive

A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)

for
Amyotrophic Lateral Sclerosis
Location: Worcester MA, Philadelphia PA, Baltimore MD,
Sponsor: Insmed Gene Therapy LLC

Sex: All

Age: 18 - 70+

Code: NCT07290062

Phase1, Recruiting
Active & Responsive

A Study of SGT-212 Gene Therapy in Friedreich's Ataxia

for
Friedreich's Ataxia (FA)
Location: Philadelphia PA, Columbus OH, Los Angeles CA
Sponsor: Solid Biosciences Inc.

Sex: All

Age: 18 - 40

Code: NCT07180355

Phase1, 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: New York NY
Sponsor: Weill Medical College of Cornell University

Sex: All

Age: 12 - 50

Code: NCT05302271

Phase1, Recruiting
Active & Responsive

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

for
Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF),
Location: Boston MA, New York NY, Orlando FL, Jacksonville FL,
Sponsor: Regeneron Pharmaceuticals

Sex: All

Age: 0+

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: Boston MA, Lebanon NH, Hackensack NJ, New Brunswick NJ,
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: Toronto, Columbus OH, Nashville TN, Houston TX
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: Boston MA, New York NY, Philadelphia PA, Durham NC,
Sponsor: Atsena Therapeutics Inc.

Sex: All

Age: 6+

Code: NCT05878860

Phase3, Recruiting
Active & Responsive

A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)

for
Duchenne Muscular Dystrophy
Location: Toronto, Calgary, Vancouver
Sponsor: Solid Biosciences Inc.

Sex: Male

Age: 7 - 11

Code: NCT07160634

Phase3, Recruiting
Active & Responsive

Study of an AAV Mediated Dual-Payload Gene Therapy in Patients With High Grade Glioma

for
Glioblastoma (GBM), High Grade Gliomas, Recurrent Glioblastoma,
Location: Columbus OH
Sponsor: Trogenix ltd

Sex: All

Age: 18 - 70

Code: NCT07346144

Phase1, Phase2, Recruiting
Active & Responsive

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

for
Danon Disease
Location: Boston MA, Philadelphia PA, La Jolla CA
Sponsor: Rocket Pharmaceuticals Inc.

Sex: Male

Age: 8+

Code: NCT06092034

Phase2, Recruiting