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Virus Therapy
Gene Therapy for Danon Disease
Phase 1
Waitlist Available
Led By Matthew Taylor, MD, PhD
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)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 3 years
Awards & highlights
Study 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.Check my eligibility
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.See study design
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 belowSelect...
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.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 3 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~3 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
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 moreSecondary outcome measures
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 moreTrial 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.
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Who is running the clinical trial?
Rocket Pharmaceuticals Inc.Lead Sponsor
14 Previous Clinical Trials
373 Total Patients Enrolled
4 Trials studying Danon Disease
317 Patients Enrolled for Danon Disease
Matthew Taylor, MD, PhDPrincipal InvestigatorUniversity of Colorado, Anschutz Medical Ctr
Joseph Rossano, MDPrincipal InvestigatorChildren's Hospital of Philadelphia
1 Previous Clinical Trials
12 Total Patients Enrolled
1 Trials studying Danon Disease
12 Patients Enrolled for Danon Disease
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Eligibility Criteria:
This trial includes the following eligibility criteria:- Your body has a high level of antibodies that can neutralize AAV9.I have an active blood or solid organ cancer.I can walk more than 150 meters by myself in a walking test.I have had a heart attack or unstable chest pain before.I have had a heart or other organ transplant.I do not have HIV, active hepatitis, uncontrolled high blood pressure or diabetes, heart rhythm problems, or untreated infections.Your blood levels of hemoglobin, neutrophils, and platelets are within the normal range.You have a serious problem with the valves of your heart, as shown on an echocardiogram.I have been diagnosed with Danon Disease and have a confirmed LAMP2 mutation.I am a male diagnosed with Duchenne muscular dystrophy.I've had the meningococcal vaccine as recommended for my age and health, at least 6 weeks before starting the treatment.My kidney function is normal based on my creatinine levels.I have tuberculosis, whether active or not.Your heart's pumping ability is less than 40%.I have had heart surgery or a procedure for blood vessel issues.You have a heart condition that has been confirmed by tests like ECG, echocardiogram, MRI, or electrophysiology study.I have had blood clots in my heart or strokes.My heart condition moderately affects my daily activities.You currently have a hepatitis B or C infection that is not under control.I am at least 15 years old for certain study groups, or between 8-14 for a specific group.I am male.My liver is functioning well, without signs of serious damage or inflammation.I've had IV treatment with heart or blood pressure medication in the last 30 days.I am not allergic to sirolimus or its components.I need a machine to help me breathe.You have a heart pump called a Left Ventricular Assisted Device (LVAD).
Research Study Groups:
This trial has the following groups:- Group 1: RP-A501
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Is access to this research currently available for participants?
"Affirmative. According to the clinicaltrials.gov data, this medical study is actively seeking participants, having been debuted on April 17th 2019 and last updated on August 24th 2021. The team aims to recruit 10 individuals from 3 distinct sites."
Answered by AI
What are the primary goals of this medical experiment?
"This clinical trial will take about 3 years to come to completion. The primary objective of the study is to assess treatment-related adverse events using NCI CTCAE measurements. Secondary objectives include determining if RP-A501 can result in sustained improvement or stabilization of cardiovascular pathophysiology, calculating the percentage of patients with corrected LAMP2B genes/proteins and improved histologic abnormalities, and assessing potential immunogenicity when exposed to components of the investigational product."
Answered by AI
Has the Food and Drug Administration given clearance to utilize RP-A501?
"Due to the early-stage nature of this trial, RP-A501's safety has been rated as a 1."
Answered by AI
How many individuals are enrolled in this research endeavor?
"The study must come up with 10 suitable recruits meeting the criteria established. Rocket Pharmaceuticals Inc. is spearheading this trial from two sites, namely University of California in San Diego and Children's Hospital of Philadelphia located in Pennsylvania."
Answered by AI
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