A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT04093349 |
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Recruitment Status :
Active, not recruiting
First Posted : September 18, 2019
Last Update Posted : July 26, 2022
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Sponsor:
Spark Therapeutics
Information provided by (Responsible Party):
Spark Therapeutics
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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.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Pompe Disease Pompe Disease (Late-onset) Glycogen Storage Disease Type 2 Glycogen Storage Disease Type II LOPD Lysosomal Storage Diseases Acid Maltase Deficiency | Genetic: SPK-3006 | Phase 1 Phase 2 |
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 30 participants |
| Allocation: | N/A |
| Intervention Model: | Sequential Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| 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 |
| Actual Study Start Date : | October 1, 2020 |
| Estimated Primary Completion Date : | October 2023 |
| Estimated Study Completion Date : | October 2027 |
Resource links provided by the National Library of Medicine
| Arm | Intervention/treatment |
|---|---|
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Experimental: SPK-3006
All participants who meet the eligibility criteria will receive a single intravenous (i.v.) administration of SPK-3006.
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Genetic: SPK-3006
adeno-associated viral (AAV) vector |
Primary Outcome Measures :
- Number of adverse and serious adverse events (AEs/SAEs), including clinically significant abnormal laboratory values. [ Time Frame: 52 weeks ]Adverse events.
- Occurrence of immune response against AAV capsid [ Time Frame: 52 weeks ]
- Occurrence of immune response against GAA transgene [ Time Frame: 52 weeks ]
Information from the National Library of Medicine
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| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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.
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04093349
Locations
Show 29 study locations
Sponsors and Collaborators
Spark Therapeutics
Investigators
| Principal Investigator: | Tahseen Mozaffar, MD | University of California Irvine Health |
Additional Information:
| Responsible Party: | Spark Therapeutics |
| ClinicalTrials.gov Identifier: | NCT04093349 |
| Other Study ID Numbers: |
SPK-3006-101 2019-001283-30 ( EudraCT Number ) |
| First Posted: | September 18, 2019 Key Record Dates |
| Last Update Posted: | July 26, 2022 |
| Last Verified: | July 2022 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Additional relevant MeSH terms:
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Glycogen Storage Disease Type II Glycogen Storage Disease Lysosomal Storage Diseases Metabolic Diseases Disease Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic |
Brain Diseases Central Nervous System Diseases Nervous System Diseases Metabolism, Inborn Errors Genetic Diseases, Inborn Carbohydrate Metabolism, Inborn Errors Pathologic Processes |