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Phase 1 clinical trial of an investigational gene therapy for cardiac disease associated with Friedreich Ataxia

The investigational gene therapy involves a single, intravenous administration of a modified adeno-associated virus that delivers the normal FXN gene to the heart (and other organs). The goal of this initial clinical study is to assess safety and initial estimates of efficacy in treating heart disease.

Participation in this study requires an overnight stay at The New York Presbyterian Hospital/Weill Cornell Medicine for observation after administration of the study drug, followed by a one week stay at the Hemsley Hotel on the Weill Cornell Medical campus. Participants will then be assessed periodically over 5 years including 4 visits to the study site at Weill Cornell Medical campus at 1, 3, 6, and 12 months following administration of the study drug.

This clinical trial is funded by the National Heart, Lung, and Blood Institute at no cost to participants including travel and accommodation expenses.

To learn more about gene therapy please visit:

  1. FARA’s Roadmap to Gene Therapy Readiness
  2. Materials from the Cornell/LEXEO community webinar:
    1. Slides
    2. Unanswered audience questions

Inclusion Criteria:

  • Males and females, age 18 to 40
  • Definitive diagnosis of FA, based on clinical phenotype and genotype (>600 GAA repeat expansion on both alleles)
  • Left ventricle ejection fraction (EF) measured by cardiac MRI of >45% to 70%
  • In the absence of other factors known to cause left ventricular hypertrophy (LVH), left ventricular mass index on cardiac MRI >2 standard deviations above the normal range
  • Stroke volume index (<45 mL/m2) and/or global longitudinal left ventricular strain (<20%) on cardiac MRI
  • <5% fibrosis in the left ventricular wall on late gadolinium enhancement cardiac MRI
  • Serum neutralizing anti-AAVrh.10 titer <1:40
  • Capable of undergoing cardiac MRI
  • No contraindications to receiving corticosteroid immunosuppression
  • Must be fully vaccinated against SARS-CoV2 (for Pfizer and Moderna 2 vaccinations + booster; for Johnson & Johnson / Janssen 1 vaccination + booster)

Exclusion Criteria:
 
  • Individuals receiving corticosteroids or other immunosuppressive medications
  • Individuals with uncontrolled diabetes (glycated hemoglobin, HbA1c levels >7%)
  • Genotype FA missense mutation on one or both alleles
  • Contraindication to cardiac MRI (e.g., non-MRI compatible pacemaker/defibrillator) or gadolinium
  • Any malignancy during the last five years, except basal cell skin cancer
  • Prior participation in any gene and/or cell therapy

Additional information can be found on clinicaltrials.gov (identifier: NCT05302271)

View the study flyer

If you are interested in participation and would like to learn more about the study, please contact:

Haley Bowe, BS
646 962 2672
hab4007@med.cornell.edu
   or
Noor Hasan, MBBS
646 962 5583
noh4004@med.cornell.edu

Anyone considering participating in a clinical trial should discuss the matter with his or her physician. FARA does not endorse or recommend any particular studies.
 
 
 

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