Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B (NCT03882437) | Clinical Trial Compass
UnknownPhase 1
Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B
United States7 participantsStarted 2019-04-17
Plain-language summary
This is a non-randomized open-label Phase 1 study to evaluate the safety and toxicity of gene therapy using a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene (investigational product (IP), RP-A501) in male patients with Danon Disease (DD).
Who can participate
Age range8 Years
SexMALE
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Inclusion criteria
✓. DD diagnosis with any confirmed LAMP2 mutation(s).
✓. Cardiac involvement as documented by at least one abnormal finding on electrocardiogram (ECG), echocardiogram, gadolinium-enhanced cardiac magnetic resonance imaging (MRI), or electrophysiology study.
✓. Age ≥15 years for cohorts 1 and 2; 8-14 years for cohorts 1A.
✓. Male gender.
✓. New York Heart Association (NYHA) Class II or III.
✓. Adequate hematologic function as defined by hemoglobin, absolute neutrophil count (ANC), and platelet count ≥ lower limit of normal (LLN).
✓. Adequate hepatic function as defined by:
✓. AST and ALT ≤10.0×ULN or GGT ≤2.0×ULN (transaminase elevations in DD are considered extensively to result from muscle injury; hence the relatively high upper limit for transaminases and consideration of GGT level, and the presence of additional hepatic eligibility markers of bilirubin and PT/INR).
Exclusion criteria
✕. I.V. therapy with positive inotropes, vasodilators, or diuretics within the 30 days prior to enrollment (i.e., patient must be stable on oral medical therapy).
✕. Prior cardiac transplantation or prior transplant of other organ (lung, liver, other).
✕. Prior cardiac surgery and/or percutaneous cardiac intervention for arteriothrombotic complications, or valvuloplasty.
What they're measuring
1
Number of participants with treatment-related adverse events as assessed by United States (US) National Cancer Institute Common Terminology Criteria (NCI CTCAE)
Timeframe: 3 years
2
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)
Timeframe: 3 years
3
Evaluation of cardiomyocyte histologic correction following administration of RP-A501 via endomyocardial biopsy
Timeframe: 3 years
4
Preliminary evaluation of clinical stabilization of cardiomyopathy following administration of RP-A501 via cardiopulmonary testing