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

Phase 1

Active, not recruiting

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

• Registration Number: NCT04093349
• Lead Sponsor: Spark Therapeutics, Inc.

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 available

Study Timeline

• Study First Submitted: 2019-09-16
• Study First Submitted QC: 2019-09-16
• Study First Posted: 2019-09-18
• Study Start: 2020-10-01
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-25
• Last Update Posted: 2024-11-27
• Primary Completion: 2032-04
• Study Completion: 2032-04

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 4

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Primary Outcome Measures

Name	Time	Method
Number of adverse and serious adverse events (AEs/SAEs), including clinically significant abnormal laboratory values.	Up to 5 years	Adverse events.
Occurrence of immune response against GAA transgene	Up to 5 years
Occurrence of immune response against AAV capsid	Up to 5 years

Trial Locations

Locations (29)

Barrow Neurological Institute; 🇺🇸 Phoenix, Arizona, United States

University of California Irvine Health; 🇺🇸 Orange, California, United States

Emory University School of Medicine; 🇺🇸 Atlanta, Georgia, United States

University of Kansas Medical Center Research Institute; 🇺🇸 Kansas City, Kansas, United States

University of Minnesota; 🇺🇸 Minneapolis, Minnesota, United States

Oregon Health & Science University; 🇺🇸 Portland, Oregon, United States

University of Pennsylvania; 🇺🇸 Philadelphia, Pennsylvania, United States

University of Pittsburgh Medical Center; 🇺🇸 Pittsburgh, Pennsylvania, United States

University of Utah; 🇺🇸 Salt Lake City, Utah, United States

Lysosomal and Rare Disorders Research & Treatment Center; 🇺🇸 Fairfax, Virginia, United States

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