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Gene Therapy

RP-A601 for Arrhythmogenic Cardiomyopathy

Phase 1

Recruiting

Led By Barry Greenberg, MD

Research Sponsored by Rocket Pharmaceuticals Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Clinical diagnosis of ACM as defined by the 2010 revised Task Force Criteria (TFC)

Male or female ≥18 years at the time of signing the informed consent

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 months post-infusion

Awards & highlights

Study Summary

This trial will test the safety and effectiveness of a new drug in high-risk adults with a specific condition.

Who is the study for?

Adults over 18 with a specific heart condition called PKP2-ACM, who have had an ICD implanted for at least 6 months can join. They must have a certain genetic variant and low levels of antibodies against the treatment virus. People with severe heart issues, other genetic causes of cardiomyopathy, or those in advanced stages of heart failure cannot participate.Check my eligibility

What is being tested?

The trial is testing RP-A601, given as a one-time intravenous infusion to see if it's safe and might help patients with PKP2-ACM. It starts with small doses that increase gradually to find the right balance between effectiveness and safety.See study design

What are the potential side effects?

Since this is an early-phase trial for RP-A601, potential side effects are being studied; however, common risks may include reactions at the infusion site, flu-like symptoms, immune responses or worsening of underlying conditions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with ACM according to the 2010 criteria.

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I am 18 years old or older.

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My tests show a specific genetic change in the PKP2 gene.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months post-infusion

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 months post-infusion

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months post-infusion for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Identification of Dose Limiting Toxicities (DLTs)

Incidence of Serious Adverse Events (SAEs)

Incidence of treatment emergent adverse events (TEAEs)

Secondary outcome measures

Change in PKP2 myocardial tissue expression

Change in the frequency of clinical markers of life threatening arrhythmias

Trial Design

1Treatment groups

Experimental Treatment

Group I: RP-A601Experimental Treatment1 Intervention

Single ascending dose of RP-A601 in 2 consecutive cohorts

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

Rocket Pharmaceuticals Inc.Lead Sponsor

14 Previous Clinical Trials

371 Total Patients Enrolled

Barry Greenberg, MDPrincipal InvestigatorUniversity of California, San Diego

6 Previous Clinical Trials

487 Total Patients Enrolled

Media Library

RP-A601 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05885412 — Phase 1

Arrhythmogenic Cardiomyopathy Clinical Trial 2023: RP-A601 Highlights & Side Effects. Trial Name: NCT05885412 — Phase 1

RP-A601 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05885412 — Phase 1

Arrhythmogenic Cardiomyopathy Research Study Groups: RP-A601

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Has RP-A601 gained endorsement from the FDA?

"Our team at Power assigned RP-A601 a safety score of 1, as this is an initial Phase I trial and there are limited data on the efficacy and protection provided by the medication."

Answered by AI

Are the doors still open for people to join this experiment?

"According to the data hosted by clinicaltrials.gov, this medical research has ceased its participant recruitment phase; it was initially posted on June 1st 2023 and last updated on May 22nd 2023. Nevertheless, there are still 170 active trials seeking patients currently."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)

2023. "A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05885412.

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