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NCT05302271 · ClinicalTrials.gov registry record · Phase 1
Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia
A Phase 1 study of Cardiomyopathies and Friedreich Ataxia, sponsored by Weill Medical College of Cornell University.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 25
- Enrollment target
- 1
- Study location
NCT05302271 is a Phase 1 study of Cardiomyopathies and Friedreich Ataxia that is actively recruiting participants, run by Weill Medical College of Cornell University. The registered enrollment target is 25 participants, below the 634-participant average among 17 other Cardiomyopathies trials with a reported enrollment target (96% lower). The trial reports 1 study location across 1 state.
The verdict
NCT05302271, a Phase 1 study of Cardiomyopathies and Friedreich Ataxia, is actively recruiting participants, sponsored by Weill Medical College of Cornell University.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 25 participants
- Enrollment target
- 1
- Study location
Study Summary
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.
Conditions Studied
Interventions
- DRUG Prednisone
- BIOLOGICAL AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN
Study Locations (1)
New York
- Weill Cornell Medicine - New York
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 25 participants |
| Start Date | 2022-02-22 |
| Est. Completion | 2029-12-31 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05302271
The ClinicalTrials.gov registry entry for NCT05302271 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 25 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 634-participant average among 17 other Cardiomyopathies trials with a reported enrollment target (96% lower). The listed sponsor is Weill Medical College of Cornell University, which has 701 total studies on file at ClinicalTrials.gov.
The record links to 4 conditions, with Cardiomyopathies appearing as the primary indexed condition, and to 2 interventions - of which Prednisone is the first listed.
NCT05302271 reports 1 study location spanning 1 distinct geographic area - top geographies include New York.
Frequently Asked Questions
What is clinical trial NCT05302271 about?
NCT05302271 is a clinical study titled "Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia". 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 intravenousl...
What is the current status of trial NCT05302271?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 25 participants. The study started on 2022-02-22. Estimated completion is 2029-12-31.
What conditions does trial NCT05302271 study?
This clinical trial studies the following conditions: Cardiomyopathies, Friedreich Ataxia, Myocardial Fibrosis, Cardiac Hypertrophy.
What interventions are being tested in trial NCT05302271?
The interventions under investigation include: Prednisone (DRUG), AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN (BIOLOGICAL).
Who is sponsoring clinical trial NCT05302271?
This trial is sponsored by Weill Medical College of Cornell University, which has 701 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05302271 being conducted?
This trial has 1 study location across New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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