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ACTIVE_NOT_RECRUITINGPHASE1/PHASE2

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

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

NCT04093349•Spark Therapeutics, Inc.

View on ClinicalTrials.gov

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

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

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;
+2 more criteria

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

Key Dates

Start Date

October 1, 2020

Primary Completion Date

April 1, 2032

Completion Date

April 1, 2032

Last Updated

November 27, 2024

Enrollment

ACTUAL participants

Conditions

Pompe DiseasePompe Disease (Late-onset)Glycogen Storage Disease Type 2Glycogen Storage Disease Type IILOPDLysosomal Storage DiseasesAcid Maltase Deficiency

Interventions

SPK-3006

GENETIC

A Global Prospective Observational Registry of Patients With Pompe Disease

NCT06121011

Pompe Disease

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RECRUITING

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

NCT01665326

Pompe Disease

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ACTIVE_NOT_RECRUITINGEARLY_PHASE1

Study to Assess the Safety, Tolerability, PK and PD of ABX1100

NCT06109948

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: November 27, 2024Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

A Global Prospective Observational Registry of Patients With Pompe Disease

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

Study to Assess the Safety, Tolerability, PK and PD of ABX1100

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

Developing a Management Approach for Patients With "Late-Onset" Pompe Disease

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)