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Trial record 2 of 2 for:    spark therapeutics | pompe

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04093349
Recruitment Status : Recruiting
First Posted : September 18, 2019
Last Update Posted : November 3, 2020
Sponsor:
Information provided by (Responsible Party):
Spark Therapeutics

Tracking Information
First Submitted Date  ICMJE September 16, 2019
First Posted Date  ICMJE September 18, 2019
Last Update Posted Date November 3, 2020
Actual Study Start Date  ICMJE October 1, 2020
Estimated Primary Completion Date October 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 16, 2019)
  • 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 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
Official Title  ICMJE 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
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Pompe Disease
  • Pompe Disease (Late-onset)
  • Glycogen Storage Disease Type 2
  • Glycogen Storage Disease Type II
  • LOPD
  • Lysosomal Storage Diseases
  • Acid Maltase Deficiency
Intervention  ICMJE Genetic: SPK-3006
adeno-associated viral (AAV) vector
Study Arms  ICMJE Experimental: SPK-3006
All participants who meet the eligibility criteria will receive a single intravenous (i.v.) administration of SPK-3006.
Intervention: Genetic: SPK-3006
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: September 16, 2019)
20
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE October 2023
Estimated Primary Completion Date October 2023   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Clinical Director +1 215-220-9300 Resolute@sparktx.com
Listed Location Countries  ICMJE France,   Germany,   Italy,   Netherlands,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04093349
Other Study ID Numbers  ICMJE SPK-3006-101
2019-001283-30 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Spark Therapeutics
Study Sponsor  ICMJE Spark Therapeutics
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Tahseen Mozaffar, MD University of California Irvine Health
PRS Account Spark Therapeutics
Verification Date October 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP