Clinical trial
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
Name
SPK-3006-101
Description
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.
Trial arms
Trial start
2020-10-01
Estimated PCD
2032-04-01
Trial end
2032-04-01
Status
Active (not recruiting)
Phase
Early phase I
Treatment
SPK-3006
adeno-associated viral (AAV) vector
Arms:
SPK-3006
Size
4
Primary endpoint
Number of adverse and serious adverse events (AEs/SAEs), including clinically significant abnormal laboratory values.
Up to 5 years
Occurrence of immune response against AAV capsid
Up to 5 years
Occurrence of immune response against GAA transgene
Up to 5 years
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.
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SEQUENTIAL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 4, 'type': 'ACTUAL'}}
Updated at
2023-11-01
1 organization
1 product
5 indications
Organization
Spark TherapeuticsProduct
SPK-3006Indication
Pompe DiseaseIndication
Glycogen Storage Disease Type 2Indication
Late-Onset Pompe Disease (LOPD)Indication
Lysosomal Storage DiseasesIndication
Glycogen Storage Disease Type II