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Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease

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: NCT03893240
Recruitment Status : Completed
First Posted : March 28, 2019
Last Update Posted : July 15, 2021
Sponsor:
Information provided by (Responsible Party):
Spark Therapeutics

Tracking Information
First Submitted Date  ICMJE March 11, 2019
First Posted Date  ICMJE March 28, 2019
Last Update Posted Date July 15, 2021
Actual Study Start Date  ICMJE June 12, 2019
Actual Primary Completion Date October 27, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 25, 2020)
  • Neutralizing Antibodies Titer to SPK-3006 capsid [ Time Frame: 1 day ]
    The neutralizing antibodies titer to SPK-3006 is measured once prospectively at one site visit.
  • Occurrence of Neutralizing Antibodies to SPK-3006 capsid [ Time Frame: 1 day ]
    The proportion of participants who have Neutralizing Antibodies to SPK-3006 capsid.
Original Primary Outcome Measures  ICMJE
 (submitted: March 26, 2019)
  • SPK-3006 Neutralizing Antibodies Titer [ Time Frame: 1 day ]
    The SPK-3006 neutralizing antibodies titer is measured once prospectively at one site visit.
  • Occurrence of SPK-3006 Neutralizing Antibodies [ Time Frame: 1 day ]
    The proportion of participants who have Neutralizing Antibodies to SPK-3006.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 26, 2019)
  • Anti-GAA binding antibodies Titer [ Time Frame: 1 day ]
    Anti-GAA binding antibodies titer is measured once prospectively at one site visit.
  • Occurrence of Anti-GAA binding antibodies across participants [ Time Frame: 1 day ]
    The proportion of participants who have Anti-GAA binding antibodies.
  • Neutralizing antibodies to circulating GAA Titer [ Time Frame: 1 day ]
    Neutralizing antibodies to circulating GAA titer is measured once prospectively at one site visit and, if available, retrospectively from medical records that are within 24 months of signing the informed consent.
  • Occurrence of Neutralizing antibodies to circulating GAA [ Time Frame: 1 day ]
    The proportion of participants who have neutralizing antibodies to circulating GAA.
  • GAA activity level [ Time Frame: 1 day ]
    GAA activity level (percent of normal) is measured once prospectively at one site visit and, if available, retrospectively from medical records that are within 24 months of signing the informed consent.
  • GAA antigen level [ Time Frame: 1 day ]
    GAA antigen level (percent of normal) is measured once prospectively at one site visit and, if available, retrospectively from medical records that are within 24 months of signing the informed consent.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease
Official Title  ICMJE A Multi-Center, Low-Interventional Study With a Retrospective Component in Participants With Late-Onset Pompe Disease
Brief Summary The purpose of this study is to obtain information pertaining to the occurrence of antibodies to investigational SPK-3006 capsid and GAA, GAA activity and GAA antigen levels in the usual care setting of Late-Onset Pompe Disease (LOPD) participants on an enzyme replacement regimen. Additionally, a careful evaluation of laboratory and functional testing in patients with LOPD may provide information to better understand the disease features and better drive the design of a future interventional investigational gene therapy trial. An understanding of the underlying status of liver and muscle health in individuals with LOPD may also inform best surveillance during the conduct of gene therapy trials.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Screening
Condition  ICMJE
  • Pompe Disease
  • Pompe Disease (Late-onset)
  • Glycogen Storage Disease Type 2
  • LOPD
  • Lysosomal Storage Diseases
  • Acid Maltase Deficiency
Intervention  ICMJE Diagnostic Test: Neutralizing Antibody to SPK-3006 capsid
Collected during a single study visit to establish the occurrence of neutralizing antibodies to SPK-3006 capsid in participants with LOPD on an enzyme replacement regimen.
Study Arms  ICMJE Participants with Late Onset Pompe disease
This is a multi-center, low-interventional study with a retrospective component in participants with LOPD. During a single study visit, assessments including but not limited to, liver health, neutralizing antibodies to SPK-3006 capsid and GAA, anti-GAA binding antibodies, GAA activity and GAA antigen levels will be performed. Additional information will be collected to provide retrospective evaluations relating to muscle and liver inflammation and/or injury. Historic data relating to Pompe disease will be collected from medical records. The retrospective and laboratory data collected may assist in providing baseline information for a future investigational gene therapy study.
Intervention: Diagnostic Test: Neutralizing Antibody to SPK-3006 capsid
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 Completed
Actual Enrollment  ICMJE
 (submitted: July 14, 2021)
61
Original Estimated Enrollment  ICMJE
 (submitted: March 26, 2019)
60
Actual Study Completion Date  ICMJE October 27, 2020
Actual Primary Completion Date October 27, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Provide written informed consent and authorization to use protected health information in accordance with national and local privacy regulations
  • Male or females ≥18 years of age
  • Currently on ERT using regular recombinant human GAA infusions for at least 18 months prior to screening
  • Documented history of clinically moderate late-onset Pompe disease.

Exclusion Criteria:

  • History of HIV infection
  • Requires any invasive ventilation (other than BiPAP at night) or noninvasive ventilation while awake and upright
  • Previously received SPK-3006
  • Previously dosed with any investigational or approved gene therapy product at any time or treated with an investigational drug within the last 12 weeks (vaccination studies are accepted)
  • Any concurrent clinically significant condition that would not allow the potential participant to complete the Day 1 examinations, or other condition that, in the opinion of the Investigator and/or Sponsor, makes the subject unsuitable for participation in the study
  • Unable or unwilling to comply with the schedule of visits and/or study assessments described in the clinical protocol.
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 information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France,   Germany,   Italy,   Netherlands,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03893240
Other Study ID Numbers  ICMJE SPK-GAA-100
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Current Responsible Party Spark Therapeutics
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Spark Therapeutics
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Tahseen Mozaffar, MD University of California Irvine Health
PRS Account Spark Therapeutics
Verification Date July 2021

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