The following safety measures have been implemented into RESOLUTE® to help minimize the risk of COVID-19 (SARS-CoV-2) exposure to patients and their caregivers:
All patients and their caregivers must follow risk mitigation guidelines: face masks, social distancing, hand washing, etc.
Screening all patients for COVID-19 (SARS-CoV-2) Safety Measures Implemented active virus infection during the screening period of the study, prior to investigational product candidate SPK-3006 infusion, and after receiving SPK-3006 infusion.
Additional safety measures may be initiated, as per the judgment of the Investigator, per the institutional guideline and in consultation with the Sponsor Medical Monitor.
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://medlineplus.gov/genetics/understanding/therapy/procedures/ and https://medlineplus.gov/genesandgenetherapy.html
A thoughtful, diligent approach to educating a patient about fundamental factors regarding possible participation in an investigational gene therapy study is critical. Click on the adjacent video for more information.
LOPD is a lysosomal storage disorder that results from mutations in the gene responsible for making GAA enzyme leading to a partial deficiency or shortage of GAA enzyme. 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.
To evaluate safety and tolerability of a single intravenous dose of investigational product candidate SPK-3006 administered to participants with clinically moderate LOPD.
Open-label, non-randomized, Phase 1/2 dose-escalation study to evaluate the safety, tolerability, and efficacy of a single intravenous infusion of investigational product candidate SPK-3006 in adults with clinically moderate LOPD currently receiving ERT. Participants will be treated in sequential, dose-level cohorts. The number of participants in each cohort will be determined by safety, by levels of the circulating lysosomal enzyme GAA, and immunogenicity evaluations.
The planned sample size is approximately 20 participants with up to 30 participants potentially enrolled.
The planned number of sites is up to approximately 25 centers globally.