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

• ClinicalTrials.gov Identifier: NCT04093349
• Recruitment Status: Active, not recruiting
• First Posted: September 18, 2019
• Last Update Posted: July 26, 2022
• Sponsor: Spark Therapeutics
• Information provided by (Responsible Party): Spark Therapeutics

Tracking Information

• First Submitted Date: September 16, 2019
• First Posted Date: September 18, 2019
• Last Update Posted Date: July 26, 2022
• Actual Study Start Date: October 1, 2020
• Estimated Primary Completion Date: October 2023 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: September 16, 2019)

• Number of adverse and serious adverse events (AEs/SAEs), including clinically significant abnormal laboratory values. [ Time Frame: 52 weeks ]
  Adverse events.
• Occurrence of immune response against AAV capsid [ Time Frame: 52 weeks ]
• Occurrence of immune response against GAA transgene [ Time Frame: 52 weeks ]

• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
• 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
• Brief 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 1; Phase 2
• Study Design: Allocation: N/A; Intervention Model: Sequential Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

• Pompe Disease
• Pompe Disease (Late-onset)
• Glycogen Storage Disease Type 2
• Glycogen Storage Disease Type II
• LOPD
• Lysosomal Storage Diseases
• Acid Maltase Deficiency

Intervention

Genetic: SPK-3006

adeno-associated viral (AAV) vector

Study Arms

Experimental: SPK-3006

All participants who meet the eligibility criteria will receive a single intravenous (i.v.) administration of SPK-3006.

Intervention: Genetic: SPK-3006

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Active, not recruiting
• Estimated Enrollment (submitted: January 7, 2022): 30
• Original Estimated Enrollment (submitted: September 16, 2019): 20
• Estimated Study Completion Date: October 2027
• Estimated Primary Completion Date: October 2023 (Final data collection date for primary outcome measure)

Eligibility Criteria

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Canada, Denmark, France, Germany, Italy, Netherlands, United Kingdom, United States
• Removed Location Countries: Spain

Administrative Information

• NCT Number: NCT04093349
• Other Study ID Numbers: SPK-3006-101; 2019-001283-30 ( EudraCT Number )
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Spark Therapeutics
• Original Responsible Party: Same as current
• Current Study Sponsor: Spark Therapeutics
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Principal Investigator: Tahseen Mozaffar, MD; University of California Irvine Health

• PRS Account: Spark Therapeutics
• Verification Date: July 2022