A Clinical Trial Looking at the Effects of a Single Dose of gene therapy SPK-3006 (Investigational Study Drug) Given Into a Vein (‘Intravenous Infusion’) of Adult Patients with Late-Onset Pompe Disease (LOPD) Who Have Been Receiving Enzyme Replacement Therapy (ERT).

      Trial Status: Suspended

      The source of the below information is the publicly available website ClinicalTrials.gov. It has been summarised and edited into simpler language.

      The below information is taken directly from the publicly available website ClinicalTrials.gov and has not been edited.

      Results Disclaimer

      How does the RESOLUTE℠ trial work?

      The RESOLUTE clinical trial is recruiting men and women who have LOPD. In order to take part in the trial, people must have clinical symptoms of LOPD affecting daily living, must be 18 years of age or older, agree to use reliable birth control, have received ERT for at least the past 24 months (2 years), do not have any liver disease, and have not received any gene therapy before. This is not a complete list of requirements to take part in the RESOLUTE clinical trial.

      The purpose of the RESOLUTE clinical trial is to study the effects of gene therapy SPK-3006 (investigational study drug). For example, to see if it works to treat your Pompe disease and if you develop any serious and non-serious side effects.

      How do I take part in the RESOLUTE clinical trial?

      If you think the RESOLUTE clinical trial may be right for you and you would like to take part, please talk to your doctor. If your doctor thinks that you might be able to take part in this clinical trial, he/she may refer you to the closest clinical trial doctor who will give you all the information you need to make your decision about taking part in the clinical trial. You will also find the clinical trial locations at the top of this page. Before starting the RESOLUTE clinical trial, you will be told about any potential risks and benefits of taking part in the trial and will need to provide written informed consent to participate. You will also be told what other treatments are available so that you may decide if you still want to take part.

      What treatment will be given if I participate in the RESOLUTE clinical trial?

      Everyone who signs an approved informed consent form and is deemed eligible by the study doctor for the RESOLUTE clinical trial will receive the investigational study drug (gene therapy SPK-3006), which is given into a vein as a one-time intravenous infusion. You might receive other study treatments to help manage certain side effects.

      How long is the RESOLUTE clinical trial?

      After receiving the study drug, you will be monitored for up to 52 weeks (1 year).

      What happens if I’m unable to take part in the RESOLUTE clinical trial?

      If the RESOLUTE clinical trial is not suitable for you, you will not be able to take part. Your doctor may suggest other clinical trials that you may be able to take part in or other treatments that you can be given. You will not lose access to any of your regular care.

      For more information about the RESOLUTE clinical trial follow this link to ClinicalTrials.gov.

      Trial-identifier: NCT04093349

      Overview

      Condition
      Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, Glycogen Storage Disease Type II, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency
      Treatments
      SPK-3006
      Phase
      Phase 1/Phase 2
      Sponsor
      Spark Therapeutics
      Trial Identifier
      NCT04093349, SPK-3006-101, 2019-001283-30
      Official Title
      Phase 1/2, Dose-escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of SPK-3006 in Adults With Late-onset Pompe Disease

      Summary

      The purpose of this study is to evaluate the safety, tolerability, and efficacy of a single intravenous infusion of SPK-3006 in adults with clinically moderate, late-onset Pompe disease receiving enzyme replacement therapy (ERT). Participants will be treated in sequential, dose-level cohorts.

      Eligibility Criteria

      • Gender All
      • Age ≥18 Years
      • Healthy Volunteers No

      Inclusion Criteria

      • Provide written informed consent;
      • Males and Females ≥18 years of age with late-onset Pompe disease;
      • Received ERT for at least the previous 24 months
      • Have clinically moderate, late-onset Pompe disease characteristics;
      • Agree to use reliable contraception.

      Exclusion Criteria

      • Active hepatitis B and/or C;
      • Significant underlying liver disease;
      • Human immunodeficiency virus (HIV) infection;
      • Prior hypersensitivity to rhGAA;
      • Pre-existing anti-AAV neutralizing antibody titers;
      • High titer antibody responses to rhGAA;
      • Requires any invasive ventilation or requires noninvasive ventilation while awake and upright;
      • Received any prior vector or gene transfer agent;
      • Active malignancy (except non-melanoma skin cancer);
      • History of liver cancer;
      • Pregnant or nursing women;
      • Any evidence of active infection at the time of SPK-3006 infusion.

      Trial information was received from ClinicalTrials.gov and was last updated in July 2020. Information provided to ClinicalTrials.gov by Hoffmann-La Roche.