BridgeMD
Multi-Center, Open Label Gene Therapy Study of RP-A501 in Male Patients with Danon Disease
EU CTIS ID: 2023-506480-34-00
What this study is testing
The primary objective of this study is to evaluate whether RP-A501 (an AAV9 capsid containing the LAMP2B transgene; AAV9.LAMP2B) will result in significant improvement as assessed via the primary endpoint comprised of LAMP2 myocardial tissue expression and LVMI.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene.
- Male gender.
- Age ≥8 years.
- Evidence of left ventricular hypertrophy with preserved systolic function phenotype as defined by each of the following: a. For subjects < 18 years, z-score of the left ventricular posterior wall or interventricular septum at end diastole ≥+ 2, and for subjects ≥18 years, left ventricular posterior wall or interventricular septum at end diastole >13 mm (>12 mm if family history of clinically significant Danon disease), b. Left ventricular ejection fraction (LVEF) ≥ 50%.
- New York Heart Association (NYHA) Class II to III.
- hsTnI ≥20% above the ULN
You likely can't join if
- Anti-AAV9 neutralizing antibody titer >1:40.
- Intravenous inotropic, vasodilator, or diuretic therapy within the 30 days prior to enrollment.
- Presence or requirement for mechanical circulatory support (MCS).
- Presence or requirement for mechanical ventilation.
- History of intracardiac thrombosis or arterial thromboembolic events including stroke, transient ischemic attack (TIA), acute coronary syndrome, myocardial infarction or unstable angina.
- Prior cardiovascular (CV) surgery, percutaneous coronary intervention (PCI), or valvuloplasty.
See the full eligibility criteria
- Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene.
- Male gender.
- Age ≥8 years.
- Evidence of left ventricular hypertrophy with preserved systolic function phenotype as defined by each of the following: a. For subjects < 18 years, z-score of the left ventricular posterior wall or interventricular septum at end diastole ≥+ 2, and for subjects ≥18 years, left ventricular posterior wall or interventricular septum at end diastole >13 mm (>12 mm if family history of clinically significant Danon disease), b. Left ventricular ejection fraction (LVEF) ≥ 50%.
- New York Heart Association (NYHA) Class II to III.
- hsTnI ≥20% above the ULN
- Ability to comply with study procedures including investigational therapy and follow-up evaluations.
- Has received approved vaccination against Neisseria meningitidis ≥ 6 weeks before administration of RP-A501.
- Anti-AAV9 neutralizing antibody titer >1:40.
- Intravenous inotropic, vasodilator, or diuretic therapy within the 30 days prior to enrollment.
- Presence or requirement for mechanical circulatory support (MCS).
- Presence or requirement for mechanical ventilation.
- History of intracardiac thrombosis or arterial thromboembolic events including stroke, transient ischemic attack (TIA), acute coronary syndrome, myocardial infarction or unstable angina.
- Prior cardiovascular (CV) surgery, percutaneous coronary intervention (PCI), or valvuloplasty.
- Greater than moderate valvular stenosis or regurgitation on most recent echocardiographic assessment.
- Prior cardiac or other organ (lung, liver, other) transplantation.
The study team makes the final eligibility decision.
Where it's taking place
- United Kingdom
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 0-17 years, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United Kingdom; United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.