AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN for Cardiac Hypertrophy

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Cardiac Hypertrophy+7 More
AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN - Biological
Eligibility
18 - 65
All Sexes
What conditions do you have?
Select

Study Summary

This trial is testing a gene therapy to treat Friedreich's ataxia, a disease that affects the heart. The therapy uses a virus to deliver the gene. It will be given intravenously to 10 people to see if it is safe and effective.

Eligible Conditions
  • Cardiac Hypertrophy
  • Cardiomyopathies
  • Myocardial Fibrosis
  • Friedreich Ataxia

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Cardiac Hypertrophy

Study Objectives

1 Primary · 0 Secondary · Reporting Duration: 5 Years

5 Years
Change in arrhythmias with 24-hour monitoring.
Change in cardiac-relevant parameters in cardiac-magnetic resonance scans
Change in cardiac-relevant parameters in echocardiograms
Change in cardiopulmonary exercise testing
Safety of AAVrh.10hFXN

Trial Safety

Safety Progress

1 of 3

Other trials for Cardiac Hypertrophy

Trial Design

2 Treatment Groups

First Dose Cohort
1 of 2
Second Dose Cohort
1 of 2
Experimental Treatment

10 Total Participants · 2 Treatment Groups

Primary Treatment: AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN · No Placebo Group · Phase 1

First Dose CohortExperimental Group · 2 Interventions: Prednisone, AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN · Intervention Types: Drug, Biological
Second Dose CohortExperimental Group · 2 Interventions: Prednisone, AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN · Intervention Types: Drug, Biological
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Prednisone
2020
Completed Phase 4
~2450

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 5 years

Who is running the clinical trial?

National Heart, Lung, and Blood Institute (NHLBI)NIH
3,576 Previous Clinical Trials
46,968,571 Total Patients Enrolled
Weill Medical College of Cornell UniversityLead Sponsor
983 Previous Clinical Trials
1,338,042 Total Patients Enrolled
Ronald G Crystal, MDPrincipal InvestigatorWeill Medical College of Cornell University
26 Previous Clinical Trials
5,941 Total Patients Enrolled

Eligibility Criteria

Age 18 - 65 · All Participants · 10 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You have a negative pregnancy test if you are male or female, age 18 to 40.
>600 GAA repeats in intron 1 in at least one allele.
You have the clinical phenotype of FA.
You have Friedreich's ataxia.
A cardiac MRI left ventricular mass index >2 standard deviations above the normal range (males >84 gm/m2, females >69 gm/m2) is a significant risk factor for left ventricular hypertrophy.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 25th, 2021

Last Reviewed: October 13th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.