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

Part of paid clinical trials in New York, New York.

Sponsor
Weill Medical College of Cornell University
Study ID
NCT05302271
Phase
PHASE1
Status
Recruiting
Apply to this trial

Conditions

  • Cardiac Hypertrophy
  • Cardiomyopathies
  • Friedreich Ataxia
  • Myocardial Fibrosis

Eligibility Criteria

Sex
ALL
Age
12 Years - 50 Years
Healthy Volunteers
Not accepted

Interventions

  • AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN — BIOLOGICAL
    AAVrh.10hFXN will be administered intravenously.
  • Prednisone — DRUG
    All participants will remain immunosuppression therapy with prednisone for a total of 14 weeks.

Study Details

The purpose of this study is to test the safety and preliminary efficacy of AAVrh.10hFXN to treat the cardiomyopathy associated with Friedreich's ataxia (FA). AAVrh.10hFXN is a serotype rh.10 adeno-associated virus gene transfer vector coding for Frataxin (FXN). The drug is administered intravenously. This is a phase 1, open label, dose escalation study with a total of 25 participants.

Key Dates

Start date
Feb 22, 2022
Status verified
Mar 2026
Primary completion
Dec 31, 2028
Completion
Dec 31, 2029

Study Design

Enrollment
25 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: First Dose Cohort
    AAVrh.10hFXN will be administered intravenously.
  • Experimental: Second Dose Cohort
    AAVrh.10hFXN will be administered intravenously.
  • Experimental: Third Dose Cohort
    AAVrh.10hFXN will be administered intravenously.
  • Experimental: Maximum Tolerated Dose Cohort
    AAVrh.10hFXN will be administered intravenously.

Primary Outcome Measure

Safety of AAVrh.10hFXN [ Time Frame: 5 Years ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Weill Cornell MedicineNew YorkNew York10021
Maddie Galbraith, BS
[email protected]
646-962-2672
Niamh Savage, BS
[email protected]
646-962-5527
Ronald G Crystal, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in New York, NY

By research site
Weill Cornell Medicine· New York, NY

Related Studies