After careful review of the emerging epidemiological data and ongoing mitigation measures to prevent the spread of COVID-19 (SARS-CoV-2), and in consultation with the RESOLUTE Steering Committee, Spark Therapeutics will restart enrollment into RESOLUTE in August 2020.
The following safety measures will be implemented into RESOLUTE to help minimize the risk of COVID-19 (SARS-CoV-2) exposure to patients and their caregivers:
Spark Therapeutics remains committed to the investigation of a potential gene therapy for Pompe disease and to safely restarting RESOLUTE. For more information, please email RESOLUTE@sparktx.com.
Decision Details: On 16 March 2020, Spark Therapeutics voluntarily suspended enrollment into RESOLUTE out of an abundance of caution for the health and safety of patients with Pompe Disease, who may have compromised respiratory function, to minimize their risk of exposure to COVID-19 (SARS-CoV-2), as a result of traveling to the investigational sites.This decision was based on:
The RESOLUTE trial is a Phase 1/2, dose-escalation gene transfer study being conducted to evaluate the safety, tolerability, and efficacy of a single intravenous infusion of investigational product candidate SPK-3006, adeno-associated virus (AAV) vector-based gene therapy, in adults with clinically moderate, late-onset Pompe disease (LOPD) currently receiving enzyme replacement therapy.
The RESOLUTE study is the first in human clinical research study evaluating investigational product candidate SPK-3006 in LOPD patients. Patients who sign the consent form and meet all eligibility criteria will receive a single intravenous administration of investigational product candidate SPK-3006. Participants will be assessed for safety parameters, including physical examination, collection and monitoring of adverse events (AEs)/serious AEs, and occurrence of immune response against the AAV capsid and the GAA transgene. Physical and functional testing, including 6-minute Walk Test (6MWT), forced vital capacity (FVC), and acid alpha-glucosidase (GAA) enzyme levels will also be measured to assess effectiveness of investigational product candidate SPK-3006.
Key eligibility criteria for enrollment into the RESOLUTE study include: participants at least 18 years of age with clinically moderate LOPD and no active hepatitis B and/or C, HIV, significant underlying liver disease, or pre-existing anti-AAV neutralizing antibodies. Participants must have received enzyme replacement therapy (ERT) for at least the previous 24 months and maintained a stable dose and frequency for the past 6 months. For more information, please visit the listing on ClinicalTrials.gov.
Gene therapy is a potential therapeutic approach involving the transfer of genetic material, through the administration of nucleic acids, viruses, or genetically engineered microorganisms, to cells in the body in order to treat or prevent genetic disease. One goal of gene therapy research is to determine whether a functional gene can be used to restore the function of, or inactivate, a mutated gene. For more information, please visit https://ghr.nlm.nih.gov/primer/therapy/genetherapy.
Late-onset (or juvenile/adult) Pompe disease (LOPD) is a lysosomal storage disorder that results from mutations in the gene responsible for making acid alpha glucosidase (GAA) leading to a partial deficiency or shortage of GAA. Mutations in the gene prevent the GAA enzyme from effectively breaking down the energy storage molecule, glycogen, which allows it to build up to toxic or poisonous levels in lysosomes. In LOPD, the buildup of glycogen can lead to muscle weakness or difficulty breathing. The heart is usually not involved.
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