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Virus Therapy

Gene Therapy for Danon Disease

Phase 2

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

Age ≥8 years

New York Heart Association (NYHA) Class II to III

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 60 months post infusion

Awards & highlights

Study Summary

This trial tests a new treatment for Danon Disease in male patients to see if it is effective and safe.

Who is the study for?

This trial is for male patients with Danon Disease, a rare genetic condition. Participants must have a specific gene mutation, good heart pump function (LVEF ≥ 50%), elevated heart stress levels (hsTnI), be at least 8 years old, show signs of thickened heart muscle but normal pumping ability, and be able to follow the study's procedures. They should not need mechanical breathing support or have had certain serious cardiovascular events.Check my eligibility

What is being tested?

The trial is testing RP-A501, a gene therapy delivered via a virus vector designed to introduce a healthy version of the LAMP2B gene into patients' cells. It aims to assess how effective and safe this treatment is in improving symptoms or halting progression of Danon Disease in males.See study design

What are the potential side effects?

Potential side effects may include immune reactions to the viral vector like fever or fatigue, injection site reactions, changes in liver enzymes indicating liver inflammation, and other responses related to gene therapy which will be closely monitored.

Eligibility Criteria

Inclusion Criteria

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

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I am 8 years old or older.

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My heart condition limits my physical activity but I can still perform light tasks.

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My tests show a genetic change in the LAMP2 gene.

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I am male.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 60 months post infusion

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~60 months post infusion

This trial's timeline: 3 weeks for screening, Varies for treatment, and 60 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

Co-Primary endpoint including myocardial tissue expression of LAMP2 protein and decrease in left ventricular mass index (LVMI).

Secondary outcome measures

Event free survival with events defined as death, heart transplant, mechanical circulatory support (MCS) or heart failure hospitalization.

Incidence, severity and duration of treatment emergent safety events.

KCCQ and NYHA class

+4 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: RP-A501Experimental Treatment1 Intervention

One planned dose of RP-A501 in cohorts of subjects with a confirmed diagnosis of Danon Disease.

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

Rocket Pharmaceuticals Inc.Lead Sponsor

14 Previous Clinical Trials

368 Total Patients Enrolled

4 Trials studying Danon Disease

312 Patients Enrolled for Danon Disease

Barry Greenberg, MDPrincipal InvestigatorUniversity of California San Diego (UCSD)

6 Previous Clinical Trials

484 Total Patients Enrolled

1 Trials studying Danon Disease

7 Patients Enrolled for Danon Disease

Joseph Rossano, MDPrincipal InvestigatorChildren's Hospital of Philadelphia

1 Previous Clinical Trials

7 Total Patients Enrolled

1 Trials studying Danon Disease

7 Patients Enrolled for Danon Disease

Frequently Asked Questions

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

To what extent do the risks of RP-A501 outweigh its potential benefits?

"RP-A501 is estimated to be relatively safe, receiving a rating of 2. This assessment was based on the fact that this medication is in Phase 2 development, meaning safety data exists but efficacy data does not yet exist."

Answered by AI

Is there still an opportunity to enrol in this study?

"Correct. Data from clinicaltrials.gov verifies that this medical study, which first appeared on September 5th 2023, is presently recruiting patients. To successfully complete the trial, 12 volunteers need to be recruited across 2 sites."

Answered by AI

How many people have been enrolled in this experiment thus far?

"Affirmative. Data hosted on clinicaltrials.gov validates that this medical research project, which was originally advertised on September 5th 2023, is actively recruiting test subjects. A total of 12 participants are needed from 2 distinct locations."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Multi-Center, Open Label Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

2023. "A Multi-Center, Open Label Gene Therapy Study of RP-A501 in Male Patients With Danon Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06092034.

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