A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
ConditionDanon Disease
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexMale
Age8+
SponsorRocket Pharmaceuticals Inc.
About this trial
This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene, in male patients with Danon Disease.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene.
Male.
Age ≥8 years.
Abnormal thickening of Left ventricular wall,
Disqualifiers
Anti-AAV9 neutralizing antibody titer >1:40.
Severe heart failure or requirement for advanced therapies.
History of intracardiac thrombosis or arterial thromboembolic events including stroke, transient ischemic attack (TIA), acute coronary syndrome, myocardial infarction or unstable angina.
Prior cardiac or other organ (lung, liver, other) transplantation.
Trial design
Design model
Single group
Treatments tested in this trial
RP-A501
GeneticRP-A501 is a gene therapy product consisting of a rAAV9 capsid containing the human LAMP2B transgene which will be administered as a single IV infusion.
Treatment groups
14 Participants
are divided into 1 treatment groupGroup A: RP-A501Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Evaluation of efficacy via primary endpoint comprised of LAMP2 myocardial tissue expression and left ventricular mass index
Increase of myocardial tissue expression of LAMP2 protein and decrease in left ventricular mass index (LVMI).
Time frame
12 Months post-infusionSecondary outcomes
1
Evaluation of efficacy via components of the primary endpoint - LAMP2
Increase in LAMP2 protein expression
Time frame
12 months post infusion2
Evaluation of efficacy via components of the primary endpoint - Left Ventricular Mass Index (LVMI)
Decrease in Left Ventricular Mass Index (LVMI)
Time frame
12 months post infusion3
Evaluation of efficacy via biomarker evidence of myocardial injury - High Sensitivity Troponin I (hsTnI)
Decrease in high sensitivity Troponin I (hsTnI)
Time frame
12 months post infusion4
Evaluation of efficacy via biomarker evidence of myocardial injury - N-Terminal Prohormone of Brain Natriuretic Peptide (NT-proBNP)
Decrease in N-terminal prohormone of brain natriuretic peptide (NT-proBNP)
Time frame
12 months post infusionSponsors and contacts
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Source ClinicalTrials.gov