Status:
ACTIVE_NOT_RECRUITING
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
Lead Sponsor:
Spark Therapeutics, Inc.
Conditions:
Pompe Disease
Pompe Disease (Late-onset)
Eligibility:
All Genders
18+ years
Phase:
PHASE1
PHASE2
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 enzy...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
October 1 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 1 2032
Estimated Enrollment :
4 Patients enrolled
Trial Details
Trial ID
NCT04093349
Start Date
October 1 2020
End Date
April 1 2032
Last Update
November 27 2024
Active Locations (29)
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1
Barrow Neurological Institute
Phoenix, Arizona, United States, 85013
2
University of California Irvine Health
Orange, California, United States, 92868
3
Emory University School of Medicine
Atlanta, Georgia, United States, 30329
4
University of Kansas Medical Center Research Institute
Kansas City, Kansas, United States, 66160