Evicenter
P&T meetings

RP-A501 (AAV9.LAMP2B)

Danon disease

Regulatory submission
BLA planned for late 2028
Last updated
300 sources

Product information and disease descriptionProduct descriptionProduct informationDosing and administration

Administration

8 evidence topics · 14 sources

Route and number of administrations

“The study is designed to assess the efficacy and safety of a single intravenous dose of RP-A501 in treating the cardiomyopathy associated with Danon disease.” (opens the source at this quote in a new tab)

“A single intravenous infusion of RP-A501 in cohorts of subjects with a confirmed diagnosis of Danon disease.” (opens the source at this quote in a new tab)

“Pharmaceutical form SOLUTION FOR INFUSION Route of administration INTRAVENOUS INFUSION” (opens the source at this quote in a new tab)

Sequential administration with a minimum interval between participants

“Treatment proceeded sequentially, with a minimum of four weeks between infusions.” (opens the source at this quote in a new tab)

“Dosing proceeded sequentially, with at least four weeks between infusions.” (opens the source at this quote in a new tab)

“The revised protocol introduced sequential enrolment with safety review for initial participants, with a minimum four-week interval between administrations. This conservative approach was intended to allow early detection of dose-related toxicity signals.” (opens the source at this quote in a new tab)

“The pediatric run-in enrolled two patients in a sequential manner with a minimum three-month follow-up prior to subsequent enrollment.” (opens the source at this quote in a new tab)

“Evaluation of RP-A501 in pediatric patients (ages 8-14) will commence only pending determination of safety in the older (adult and age 15-17) population.” (opens the source at this quote in a new tab)

Pre-infusion requirements: antibody testing, baseline biopsy, and imaging

“STEP 1 Initial Testing This will occur at the study site and includes ECHO, EKG, cardiac MRI, heart biopsy* and additional lab assessments that include blood and urine tests.” (opens the source at this quote in a new tab)

“You will receive pre-treatment to prepare your immune system.” (opens the source at this quote in a new tab)

“All patients enrolled in the trial are required to have a three-months observational pre-treatment run-in to enable an assessment of troponin (and other biomarker) trajectories to optimally assess this key secondary endpoint.” (opens the source at this quote in a new tab)

“An immunomodulation regimen is initiated before RP-A501 administration to reduce the risk of gene therapy-related complications.” (opens the source at this quote in a new tab)

Care setting and duration of inpatient observation

“STEP 2 Treatment & Monitoring You will receive pre-treatment to prepare your immune system. You will be hospitalized for gene therapy treatment and for safety monitoring which will continue as an outpatient.” (opens the source at this quote in a new tab)

“Elevated GGT observed in 1 patient; resolved and patient discharged after 12 days. All 3 patients discharged by 1-2 weeks post-infusion” (opens the source at this quote in a new tab)

“All three completed at least four weeks of follow-up and had been discharged following protocol-specified observation.” (opens the source at this quote in a new tab)

Monitoring after infusion: laboratory and clinical surveillance schedule

“Daily, real-time monitoring of labs & clinical status Day -14 Month 3 Day 14” (opens the source at this quote in a new tab)

“Revised Immunomodulatory Protocol: More rigorous daily monitoring of labs in initial days following infusion with independent clinical review team” (opens the source at this quote in a new tab)

“Frequent monitoring for early signs of TMA” (opens the source at this quote in a new tab)

Monitoring after infusion: complement activation and platelet count

“Platelets and Terminal Complement Complex (sC5b9) of the initial 3 patients up to 3 months post-treatment” (opens the source at this quote in a new tab)

“The sC5b9 lower limit of detection per the validated assay at the clinical site is <170 ng/mL; reported values of <170 ng/mL are shown as 170 ng/mL.” (opens the source at this quote in a new tab)

“The modified protocol incorporates a recalibrated RP-A501 dose and an optimized immunomodulatory regimen comprised of rituximab, sirolimus and corticosteroids, together with enhanced eligibility criteria, safety monitoring and risk-mitigation measures.” (opens the source at this quote in a new tab)

Class context: laboratory surveillance recommended in the first 30 days after systemic AAV9 administration

“This report highlights the importance of frequent immunosurveillance during the first 30 days after AAV dosing, including comprehensive complement and hematology panels, D-dimer, and other indicators of endothelial activation.” (opens the source at this quote in a new tab)

“In addition, the total anti-AAV antibody levels should be measured before and after administration of AAV gene therapies since measuring only the titer of neutralizing antibodies provides very little information about the total amount of complement-activating antibodies, such as IgM ( 40 ).” (opens the source at this quote in a new tab)

“the interval between days 4 and 10 after AAV dosing is really the critical period, and values on day 7 alone may not enable even the most diligent clinician to conclude that the risk of TMA is low” (opens the source at this quote in a new tab)

Follow-up schedule after administration

“The trial will include a total of 5 years of follow-up after administration of RP-A501.” (opens the source at this quote in a new tab)

“All patients are planned to be followed for 36 months after investigational product administration. After the end of the follow-up period, patients will enter a Long-Term Follow-Up (LTFU) study enabling follow-up for an additional 2 to 5 years post-IP administration.” (opens the source at this quote in a new tab)

“Phase 1: 3 years (completed) Phase 1 LTFU: up to 10 years (ongoing)” (opens the source at this quote in a new tab)