A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
|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|
Recruitment Status : Active, not recruiting
First Posted : September 18, 2019
Last Update Posted : July 26, 2022
|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|
|Intervention Model:||Sequential Assignment|
|Masking:||None (Open Label)|
|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|
All participants who meet the eligibility criteria will receive a single intravenous (i.v.) administration of SPK-3006.
adeno-associated viral (AAV) vector
- 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 ]
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
|Principal Investigator:||Tahseen Mozaffar, MD||University of California Irvine Health|