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Rocket Pharmaceuticals clinical trials

Source: ClinicalTrials.gov, U.S. National Library of Medicine — current trials in our USA and UK registers with Rocket Pharmaceuticals as lead sponsor. Synced 26 Sep 2026
8Current trials
5Recruiting now
1Drug in these trials
2Trial registers
Maintained by Sreepriya Prasannan
MSc Digital Transformation of Life Sciences (Innopharma Education / Griffith College); MSc & BSc Botany HSE Spark Ignite 2026 — Top 14 finalist
Compiled from ClinicalTrials.gov and CTIS registry records · Last verified · Editorial standards
At a glance

Rocket Pharmaceuticals is the lead sponsor of 8 current clinical trials in our USA and UK registers. 5 are recruiting patients now. The most common phase is Observational. The drugs named most often in its trials are RP-L102. The most-studied condition is Danon Disease.

ClinicalTrials.gov trials by status

  • Recruiting563%
  • Active, closed225%
  • By invitation113%

Trials by phase

  • Observational450%
  • Phase 2225%
  • Phase 1225%
01

Clinical trials sponsored by Rocket Pharmaceuticals

8 on ClinicalTrials.gov
StudyPhaseStatusStartRegisters
Danon Disease Natural History Study
NCT06214507 · ClinicalTrials.gov ↗ · Danon Disease
Observational Recruiting 2023-12 UK, USA
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
NCT06092034 · ClinicalTrials.gov ↗ · Danon Disease
Phase 2 Recruiting 2023-09 UK, USA
A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)
NCT05885412 · ClinicalTrials.gov ↗ · PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)
Phase 1 Recruiting 2023-08 USA
A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy
NCT07137338 · ClinicalTrials.gov ↗ · Dilated Cardiomyopathy (DCM)
Phase 1 Recruiting 2026-06 USA
PKP2-ACM Natural History Study
NCT06644742 · ClinicalTrials.gov ↗ · Cardiomyopathies, Heart Diseases, Cardiovascular Diseases
Observational Recruiting 2026-03 USA
Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102
NCT07527975 · Fanconi Anemia
Drug: RP-L102
Observational By invitation 2022-02 UK, USA
Gene Therapy for Fanconi Anemia, Complementation Group A
NCT04248439 · Fanconi Anemia Complementation Group A
Drug: RP-L102
Phase 2 Active, closed 2020-07 USA
Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I)
NCT06282432 · ClinicalTrials.gov ↗ · Leukocyte Adhesion Deficiency
Observational Active, closed 2022-03 UK, USA

Registers list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Titles and statuses are copied from the registry as published.

02

Drugs in Rocket Pharmaceuticals trials

1 compound

Drugs and biologicals named as interventions in these trials, with the number of this sponsor's trials that name each one. Placebos and generic comparators are excluded.

03

Conditions in Rocket Pharmaceuticals trials

10 conditions
Danon Disease2Fanconi Anemia1Leukocyte Adhesion Deficiency1Fanconi Anemia Complementation Group A1PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)1Dilated Cardiomyopathy (DCM)1Cardiomyopathies1Heart Diseases1Cardiovascular Diseases1Genetic Diseases1
04

By trial register

RegisterTrialsRecruiting
Clinical trials in USA85
Clinical trials in UK42

A trial with sites in more than one country appears in each of those registers.

05

Questions about Rocket Pharmaceuticals trials

How many clinical trials does Rocket Pharmaceuticals sponsor?

Rocket Pharmaceuticals is the lead sponsor of 8 current trials on ClinicalTrials.gov in our USA and UK registers, as of the registry sync on 26 September 2026. Completed and withdrawn studies are not counted.

Are Rocket Pharmaceuticals trials recruiting?

Yes. 5 of its 8 ClinicalTrials.gov trials are recruiting. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.

Which drugs is Rocket Pharmaceuticals testing?

Its current trials name 1 drug or biological as interventions. The most frequent are RP-L102 (2). The number in brackets is how many of its trials name each drug.

Where does this information come from?

Every trial, status, phase and condition on this page is copied from public ClinicalTrials.gov and EU CTIS records by a daily sync, grouped by the sponsor name each registry records. It says nothing about the organisation beyond its registered trials, and it is not medical advice.

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How this page is built

Everything on this page is copied from public registry records — it is not written from memory, and nothing here describes the organisation beyond its registered trials. Each trial links to its own record so you can check it yourself.

This page is built automatically: a daily sync job reads the registry records behind our trial registers and groups every current trial by its lead sponsor name, ignoring legal-form suffixes such as Inc. or Ltd. A small, hand-checked list joins the same organisation's different registry names (for example its EU legal entity). There is no AI drafting and no per-sponsor editorial pass. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The the registries data on this page was last retrieved on 26 Sep 2026.

Please verify before you rely on this. This page is general information for life-science and pharmaceutical professionals. It is not medical advice, and it has not been reviewed by a clinician — our editorial team holds life-science qualifications, not clinical ones. It is not exhaustive and may not reflect the most recent label change. Always check the official prescribing information (US Prescribing Information or EU SmPC) and speak to your doctor or pharmacist before acting on anything here. Drugs in the same class are not automatically interchangeable, and approvals, brand names and indications differ between the US, the EU/Ireland (EMA/HPRA) and other regions. Spotted an error? Tell us — we correct promptly and log it.