Phase 1 Cardiomyopathy Trial, Recruiting NCT05885412 Sponsor: Rocket Pharmaceuticals Inc. Condition: Cardiomyopathy
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Phase 1 Cardiomyopathy Trial, Recruiting

NCT05885412
Recruiting Phase 1

Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

  • Adults aged 18 or older at the time of signing the consent form
  • People who are able and willing to sign a consent form agreeing to take part
  • People who have been clinically diagnosed with ACM (arrhythmogenic cardiomyopathy) based on a specific set of recognised diagnostic criteria from 2010
  • People who have a confirmed harmful or likely harmful gene change (mutation) in a gene called PKP2
  • People who have had an ICD (a small device implanted in the chest to help control dangerous heart rhythms) for at least 6 months before joining the trial
  • People whose heart monitoring shows at least 500 extra or irregular heartbeats (PVCs) within a 24-hour period
  • People whose left side of the heart is pumping at a normal or near-normal level (at least 50%), as measured by an ultrasound or special heart scan

Who may not be able to join:

  • People whose blood tests show a high level of antibodies against a specific component used in the treatment (anti-AAVrh.74 antibody levels above a certain threshold)
  • People whose heart muscle disease is caused by a genetic reason other than a PKP2 gene mutation
  • People who have previously taken part in a gene therapy or gene editing study
  • People who have severe dysfunction of the right side of the heart
  • People who have the most severe level of heart failure symptoms (classified as NYHA Class IV, meaning symptoms occur even at rest)

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.
Last synced 23 July 2026
Phase 1: approximately ~10% of drugs entering this phase reach regulatory approval, based on published industry-wide historical data. This is not specific to this trial.
Phase success rates shown are historical industry-wide averages based on published data. They are not a prediction for this specific trial or your individual situation.

Contact this trial

Principal Investigator: Barry Greenberg, MD, University of California, San Diego

Phone: 646-627-0033

Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.

GP referral letter

Print a one-page summary to share with your doctor.

Trial details

Status
Recruiting
Phase
Phase 1
Sponsor
Rocket Pharmaceuticals Inc.
Registry
ClinicalTrials.gov
Start date
29 August 2023
Est. completion
1 September 2028

Where this trial is recruiting

🇺🇸 United States

Primary endpoints

Evaluation of safety associated with RP-A601

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 23 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov