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Virus Therapy

Gene Therapy for Danon Disease

Phase 1

Waitlist Available

Led By Joseph Rossano, 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

Able to walk >150 meters unassisted during the 6MWT

DD diagnosis with any confirmed LAMP2 mutation(s)

Must not have

Prior cardiac transplantation or prior transplant of other organ (lung, liver, other)

Active hematologic or solid organ malignancy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 3 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a gene therapy to treat men with Danon Disease. The therapy uses a virus to deliver a healthy gene to patients.

Who is the study for?

This trial is for male patients with Danon Disease, a genetic condition affecting the heart and muscles. Participants must be males aged 8 years or older with cardiac involvement, able to walk over 150 meters unassisted, have normal blood counts and liver function, and not have had recent heart surgery or other serious health conditions.

What is being tested?

The study tests RP-A501 gene therapy in males with Danon Disease. It's an open-label Phase 1 trial using AAV9.LAMP2B to deliver the LAMP2 gene. The goal is to assess safety and toxicity of this treatment in affected individuals without randomizing participants into different groups.

What are the potential side effects?

Potential side effects are not detailed here but may include typical risks associated with gene therapies such as immune reactions to the viral vector, injection site reactions, and possible unintended effects on other genes or systems.

Eligibility Criteria

Inclusion Criteria

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

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I can walk more than 150 meters by myself in a walking test.

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I have been diagnosed with Danon Disease and have a confirmed LAMP2 mutation.

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I am a male diagnosed with Duchenne muscular dystrophy.

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My kidney function is normal based on my creatinine levels.

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My heart condition moderately affects my daily activities.

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I am male.

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My liver is functioning well, without signs of serious damage or inflammation.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had a heart or other organ transplant.

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I have an active blood or solid organ cancer.

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I have had a heart attack or unstable chest pain before.

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I do not have HIV, active hepatitis, uncontrolled high blood pressure or diabetes, heart rhythm problems, or untreated infections.

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I have tuberculosis, whether active or not.

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I have had heart surgery or a procedure for blood vessel issues.

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I have had blood clots in my heart or strokes.

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I am not allergic to sirolimus or its components.

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I need a machine to help me breathe.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 3 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~3 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 3 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Evaluation of cardiomyocyte histologic correction following administration of RP-A501 via endomyocardial biopsy

Number of participants with treatment-related adverse events as assessed by United States (US) National Cancer Institute Common Terminology Criteria (NCI CTCAE)

Number of participants within each dose level cohort with treatment-related adverse events as assessed by United States (US) National Cancer Institute Common Terminology Criteria (NCI CTCAE)

+1 more

Secondary study objectives

Determination and characterization of immunologic response to RP-A501

Determination of the percentage of patients in whom RP-A501 resulted in a sustained improvement or stabilization in cardiovascular pathophysiology

Determination of the percentage of patients in whom cardiomyocytes corrected LAMP2B gene and/or protein

+2 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: RP-A501Experimental Treatment1 Intervention

RP-A501 is a gene therapy product consisting of a rAAV9 capsid containing the human LAMP2B transgene which will be administered as a single intravenous (IV) infusion. Subjects will receive one of three dose levels depending on the cohort.

0 / 16Eligibility criteria met