Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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: Recruiting Phase 1 study of Cardiomyopathies and Friedreich Ataxia, sponsored by Weill Medical College of Cornell University.
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. According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
To determine the safety of AAVrh.10hFXN, as measured by the number of subjects with any treatment-related adverse events for 5 years.
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 |
What NCT05302271 shows while recruiting
NCT05302271 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 25 participants, a relatively small participant target, below the 634-participant average among 17 other Cardiomyopathies trials with a reported enrollment target (96% lower).
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 a single indexed study location in 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.
Learn More About Clinical Trials
Similar trials for Cardiomyopathies
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT05302271's enrollment target sits among peer trials
25 17th of 17 higher than 1 of 17 other Cardiomyopathies trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Cardiomyopathies trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
Risk-Guided Cardioprotection With Carvedilol in Breast Cancer Patients Treated With Doxorubicin and/or Trastuzumab
COMPLETED · Phase 1
-
Cardiovascular Multimodality Imaging Study
RECRUITING
-
The PIVATAL Study -Study of Ventricular Arrhythmia (VTA) Ablation in Left Ventricular Assist Device (LVAD) Patients
RECRUITING · Phase 4
-
PKP2-ACM Natural History Study
RECRUITING
-
Prospective Observational Study of the ICD in Sudden Cardiac Death Prevention
RECRUITING
-
Multicenter Trial of ECMO in Children With Severe Cardiac Failure Using the Cardiohelp System
RECRUITING · Phase 2
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT01166854 · 25 participants
Characterization of Familial Myopathy and Paget Disease of Bone
- NCT01913106 · 25 participants · Phase 1
HSV-tk + Valacyclovir Therapy in Combination With Brachytherapy for Recurrent Prostate Cancer
- NCT02138669 · 25 participants · NA
Intacs Prescription Inserts for Keratoconus Patients
- NCT02174549 · 25 participants · Phase 1
Dose-defining Study of Tirapazamine Combined With Embolization in Liver Cancer
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI