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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. Identifier: NCT04093349
Recruitment Status : Active, not recruiting
First Posted : September 18, 2019
Last Update Posted : July 26, 2022
Information provided by (Responsible Party):
Spark Therapeutics

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

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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

Arm Intervention/treatment
Experimental: SPK-3006
All participants who meet the eligibility criteria will receive a single intravenous (i.v.) administration of SPK-3006.
Genetic: SPK-3006
adeno-associated viral (AAV) vector

Primary Outcome Measures :
  1. Number of adverse and serious adverse events (AEs/SAEs), including clinically significant abnormal laboratory values. [ Time Frame: 52 weeks ]
    Adverse events.

  2. Occurrence of immune response against AAV capsid [ Time Frame: 52 weeks ]
  3. Occurrence of immune response against GAA transgene [ Time Frame: 52 weeks ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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 identifier (NCT number): NCT04093349

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Sponsors and Collaborators
Spark Therapeutics
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Principal Investigator: Tahseen Mozaffar, MD University of California Irvine Health
Additional Information:
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Responsible Party: Spark Therapeutics 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

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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
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