RP-A601 for Arrhythmogenic Cardiomyopathy

Recruiting at 2 trial locations

Overseen By Clinical Information

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Rocket Pharmaceuticals Inc.

Disqualifiers: Severe RV dysfunction, NYHA Class IV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 13

Duration: 1 day

9 Participants Needed

Trial Summary

What is the purpose of this trial?

This Phase 1 dose escalation trial will assess the safety and preliminary efficacy of a single dose intravenous infusion of RP-A601 in high-risk adult patients with PKP2-ACM.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Research Team

Barry Greenberg, MD

Principal Investigator

University of California, San Diego

Eligibility Criteria

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.

Inclusion Criteria

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

I am willing and able to sign the consent form.

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

Exclusion Criteria

My heart's right side is not working properly.

I have the most severe form of heart failure.

My heart condition is genetic but not caused by the PKP2 gene.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single dose intravenous infusion of RP-A601

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and preliminary efficacy, including cardiac biomarkers and myocardial PKP2 protein expression

12 months

Regular visits (in-person and virtual) over 12 months

Treatment Details

Interventions

• RP-A601

Trial Overview 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.

Participant Groups

Group I: RP-A601 Experimental Treatment 1 Intervention

Single ascending dose of RP-A601 in 2 consecutive cohorts

RP-A601 is already approved in European Union, United States for the following indications:

Approved in European Union as RP-A601 for:

• PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM) - Orphan Medicinal Product Designation

Approved in United States as RP-A601 for:

• PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM) - Fast Track and Orphan Drug Designations

Who Is Running the Clinical Trial?

Rocket Pharmaceuticals Inc.

Lead Sponsor

Trials

17

Recruited

430+