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Active, Not Recruiting

NCT Number: NCT04093349

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Pompe Disease Acid Maltase Deficiency Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Carbohydrate Metabolism, Inborn Errors Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Glycogen Storage Disease Glycogen Storage Disease Type 2 Glycogen Storage Disease Type II LOPD Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Pompe Disease (Late-onset)

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Vancouver General Hospital, Vancouver, British Columbia, Canada

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

SPK-3006

Genetic

adeno-associated viral (AAV) vector

Primary outcomes

  1. Number of adverse and serious adverse events (AEs/SAEs), including clinically significant abnormal laboratory values.

    Time frame: Up to 5 years

    Adverse events.

  2. Occurrence of immune response against AAV capsid

    Time frame: Up to 5 years

  3. Occurrence of immune response against GAA transgene

    Time frame: Up to 5 years

Sponsors and collaborators

Lead sponsor

Spark Therapeutics, Inc.

Industry

Registry information

Official study 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

Important dates

Study start
2020
Primary completion
2032
Study completion
2032
First posted
Sep 18, 2019
Registry last updated
Nov 27, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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