Sponsor or institution
Clinical trials and research at Rocket Pharmaceuticals Inc.
As of November 12, 2026, Find a Trial lists 5 clinical trials run by Rocket Pharmaceuticals Inc. that are recruiting, at sites in 8 countries (most in United States (5), Germany (2), Italy (2)). By phase: Phase 1 (2), Phase 2 (1). Every study is explained in plain English. Data from ClinicalTrials.gov and NIH RePORTER, updated November 12, 2026.
- 5Clinical trials
- 8Countries
Newest trials
| Study | Phase | Where | Status | Registered |
|---|---|---|---|---|
| RP-A701 gene therapy for BAG3-related dilated cardiomyopathy | Phase 1 | San Diego, California and 2 other locations | Recruiting | Aug 22, 2025 |
| Natural history of PKP2-related arrhythmogenic cardiomyopathy | — | Boston, Massachusetts and 2 other locations | Recruiting | Oct 16, 2024 |
| Observing the natural history of Danon disease | — | Phoenix, Arizona and 15 other locations | Recruiting | Jan 19, 2024 |
| RP-A501 gene therapy for males with Danon disease | Phase 2 | La Jolla, California and 5 other locations | Recruiting | Oct 23, 2023 |
| Testing RP-A601 for heart condition caused by PKP2 gene variants | Phase 1 | La Jolla, California and 2 other locations | Recruiting | Jun 2, 2023 |
All recruiting trials (5)
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RP-A701 gene therapy for BAG3-related dilated cardiomyopathy
This Phase 1 study will try a one-time intravenous RP-A701 gene therapy in adults with BAG3-related dilated cardiomyopathy to see if it is safe and may improve heart function.
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Natural history of PKP2-related arrhythmogenic cardiomyopathy
This project will collect past and ongoing health information to see how PKP2-related arrhythmogenic cardiomyopathy progresses in people managed with standard care.
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Observing the natural history of Danon disease
This study looks at how Danon disease affects boys over 8 and girls under 30 over time to see changes in their heart health, symptoms, and quality of life.
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RP-A501 gene therapy for males with Danon disease
This gives a single IV dose of RP-A501 gene therapy to males aged 8 and older with Danon disease to see if it improves heart structure and symptoms.
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Testing RP-A601 for heart condition caused by PKP2 gene variants
This study is testing a new treatment called RP-A601 to see if it can help adults with a heart condition caused by changes in the PKP2 gene.
Quick answers
How many clinical trials run by Rocket Pharmaceuticals Inc. are recruiting right now?
5, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.
Where are these trials running?
In 8 countries. The most sites are in United States (5 trials), Germany (2 trials), Italy (2 trials), United Kingdom (2 trials), Netherlands (1 trial). Each study page lists its sites and which ones are recruiting.
What phases are they in?
Phase 1: 2, Phase 2: 1. Trials with no phase listed are usually device, behavioural or observational studies.
How do I join one?
Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.
Find the studies that fit you
A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.