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Eine Gentransferstudie bei Morbus Pompe der späten Verlaufsform (RESOLUTE SM)
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
- Stoffwechselerkrankung
- Morbus Pompe (alpha-Glukosidase-Mangel)
- Typ II-Glykogenspeicherkrankheit (GSD II)
- Lysosomale Speicherkrankheit (LSK)
Bitte beachten Sie, dass der Rekrutierungsstatus der Studie an Ihrem Standort vom Gesamtstatus der Studie abweichen kann, da einige Studienzentren früher rekrutieren als andere.
Status der Studie:
Aktiv, keine Rekrutierung
Diese Studie läuft in
Stadt
- München
Studien-ID:
NCT04093349 2019-001283-30 SPK-3006-101
Studienzusammenfassung
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.
Spark Therapeutics
Sponsor
Phase 1/Phase 2
Phase
NCT04093349,SPK-3006-101,2019-001283-30
Studien-ID
SPK-3006
Treatments
Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, Glycogen Storage Disease Type II, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency
Condition
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
Einschlusskriterien
All
Gender
≥18 Years
Age
No
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;
- Pregnant or nursing women;
- Any evidence of active infection at the time of SPK-3006 infusion.
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