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RESOLUTE® is a Phase 1/2 Investigational Gene Transfer Study for Adults with Late-Onset Pompe Disease (LOPD)

COVID-19 (SARS-CoV-2) Safety Measures Implemented

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:

1

All patients and their caregivers must follow risk mitigation guidelines: face masks, social distancing, hand washing, etc.

2

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.

3

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.

What is the RESOLUTE® Trial?

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.

To learn more about the RESOLUTE® trial, please visit the listing on ClinicalTrials.gov or contact us at RESOLUTE@sparktx.com.

Clinical Trial Overview

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.

Doctor talking about LOPD with patient.
Doctor using microscope

Key Participant Criteria

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.

Where are the study locations?

For inquiries regarding study participation, please contact us at 1-866-MISPARK (1-866-647-7275) or https://sparktx.com/medinfo/.

Frequently Asked Questions:
Gene Therapy and Gene Therapy Clinical Trials

What is gene therapy?

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


What factors might a healthcare provider want to consider when thinking about referring a patient to a gene therapy clinical trial?

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.

Frequently Asked Questions:
The RESOLUTE® Study

What is late-onset Pompe disease (LOPD)?

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.


What is the primary study objective?

To evaluate safety and tolerability of a single intravenous dose of investigational product candidate SPK-3006 administered to participants with clinically moderate LOPD.


What is the study design?

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.


How many participants are being enrolled in the study?

The planned sample size is approximately 20 participants with up to 30 participants potentially enrolled.


How many sites are being opened for the study execution?

The planned number of sites is up to approximately 25 centers globally.


What are the different steps in the study?

  • Participants will need to contact a study center/site to set up a prescreening appointment.
  • If deemed eligible, the participant will be asked to sign the main informed consent form prior to the screening period. Screening assessments will begin and extend up to 14 weeks to permit sufficient time for assessment of all screening criteria and final confirmation of study eligibility. Assessments in the screening period include, but may not be limited to:
    • Bloodwork will be drawn
    • Evaluation of heart and liver function will be conducted
    • Functional assessments, such as:
      • A 6-minute walk test
      • Forced vital respiratory capacity
  • Eligible participants will be scheduled to receive a single intravenous dose of investigational product candidate SPK-3006.
  • A single infusion of SPK-3006 will be given and participant vital signs will be monitored at multiple timepoints for 24 hours after the infusion.
  • Follow-up observation will consist of home visits and site visits, per discretion of the investigator and participant.