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

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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

Study Description
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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.

Condition or disease Intervention/treatment Phase
Pompe Disease Pompe Disease (Late-onset) Glycogen Storage Disease Type 2 LOPD Lysosomal Storage Diseases Acid Maltase Deficiency Diagnostic Test: Neutralizing Antibody to SPK-3006 capsid Not Applicable

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 61 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Screening
Official Title: A Multi-Center, Low-Interventional Study With a Retrospective Component in Participants With Late-Onset Pompe Disease
Actual Study Start Date : June 12, 2019
Actual Primary Completion Date : October 27, 2020
Actual Study Completion Date : October 27, 2020

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Arms and Interventions
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Arm Intervention/treatment
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.
 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.


Outcome Measures
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Primary Outcome Measures :
  1. 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.

  2. Occurrence of Neutralizing Antibodies to SPK-3006 capsid [ Time Frame: 1 day ]
    The proportion of participants who have Neutralizing Antibodies to SPK-3006 capsid.


Secondary Outcome Measures :
  1. Anti-GAA binding antibodies Titer [ Time Frame: 1 day ]
    Anti-GAA binding antibodies titer is measured once prospectively at one site visit.

  2. Occurrence of Anti-GAA binding antibodies across participants [ Time Frame: 1 day ]
    The proportion of participants who have Anti-GAA binding antibodies.

  3. 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.

  4. Occurrence of Neutralizing antibodies to circulating GAA [ Time Frame: 1 day ]
    The proportion of participants who have neutralizing antibodies to circulating GAA.

  5. 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.

  6. 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.


Eligibility Criteria
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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 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.
Contacts and Locations
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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): NCT03893240


Locations
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Sponsors and Collaborators
Spark Therapeutics
Investigators
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Principal Investigator: Tahseen Mozaffar, MD University of California Irvine Health
More Information
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Responsible Party: Spark Therapeutics
ClinicalTrials.gov Identifier: NCT03893240    
Other Study ID Numbers: SPK-GAA-100
First Posted: March 28, 2019    Key Record Dates
Last Update Posted: July 15, 2021
Last Verified: July 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
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
Antibodies
Antibodies, Blocking
Immunologic Factors
Physiological Effects of Drugs