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History of Changes for Study: NCT03893240
Neutralizing Antibody Screening Study With a Retrospective Component in Participants With Late-Onset Pompe Disease
Latest version (submitted July 14, 2021) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 March 26, 2019 None (earliest Version on record)
2 July 19, 2019 Recruitment Status, Study Status, Contacts/Locations and Study Identification
3 September 9, 2019 Contacts/Locations, Study Status and Eligibility
4 December 3, 2019 Study Status and Contacts/Locations
5 May 14, 2020 Recruitment Status, Study Status and Contacts/Locations
6 August 25, 2020 Recruitment Status, Study Status, Contacts/Locations, Outcome Measures, Arms and Interventions and Study Description
7 July 14, 2021 Recruitment Status, Study Status, Contacts/Locations and Study Design
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Study NCT03893240
Submitted Date:  March 26, 2019 (v1)

Open or close this module Study Identification
Unique Protocol ID: SPK-GAA-100
Brief Title: Neutralizing Antibody Screening Study With a Retrospective Component in Participants With Late-Onset Pompe Disease
Official Title: A Multi-Center, Low-Interventional Study With a Retrospective Component in Participants With Late-Onset Pompe Disease
Secondary IDs:
Open or close this module Study Status
Record Verification: March 2019
Overall Status: Not yet recruiting
Study Start: April 2019
Primary Completion: February 2022 [Anticipated]
Study Completion: February 2022 [Anticipated]
First Submitted: March 11, 2019
First Submitted that
Met QC Criteria:
March 26, 2019
First Posted: March 28, 2019 [Actual]
Last Update Submitted that
Met QC Criteria:
March 26, 2019
Last Update Posted: March 28, 2019 [Actual]
Open or close this module Sponsor/Collaborators
Sponsor: Spark Therapeutics
Responsible Party: Sponsor
Collaborators:
Open or close this module Oversight
U.S. FDA-regulated Drug: No
U.S. FDA-regulated Device: No
Data Monitoring: No
Open or close this module Study Description
Brief Summary: The purpose of this study is to obtain information pertaining to the occurrence of antibodies to investigational SPK-3006 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:
Open or close this module Conditions
Conditions: Pompe Disease
Pompe Disease (Late-onset)
Glycogen Storage Disease Type 2
LOPD
Lysosomal Storage Diseases
Acid Maltase Deficiency
Keywords:
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Screening
Study Phase: Not Applicable
Interventional Study Model: Single Group Assignment
Number of Arms: 1
Masking: None (Open Label)
Allocation: N/A
Enrollment: 60 [Anticipated]
Open or close this module Arms and Interventions
Arms Assigned Interventions
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: SPK-3006 Neutralizing Antibody
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.
Open or close this module Outcome Measures
Primary Outcome Measures:
1. SPK-3006 Neutralizing Antibodies Titer
[ Time Frame: 1 day ]

The SPK-3006 neutralizing antibodies titer is measured once prospectively at one site visit.
2. Occurrence of SPK-3006 Neutralizing Antibodies
[ Time Frame: 1 day ]

The proportion of participants who have Neutralizing Antibodies to SPK-3006.
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.
Open or close this module Eligibility
Minimum Age: 18 Years
Maximum Age:
Sex: All
Gender Based:
Accepts Healthy Volunteers: No
Criteria:

Inclusion Criteria:

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

Exclusion Criteria:

  1. History of HIV infection
  2. Requires any invasive ventilation (other than BiPAP at night) or noninvasive ventilation while awake and upright
  3. Previously received SPK-3006
  4. 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)
  5. 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
  6. Unable or unwilling to comply with the schedule of visits and/or study assessments described in the clinical protocol.
Open or close this module Contacts/Locations
Central Contact Person: Clinical Director
Telephone: +1 215-220-9300
Email: clinicaltrials@sparktx.com
Central Contact Backup: Clinical Director
Email: clinicaltrials@sparktx.com
Locations:
Open or close this module IPDSharing
Plan to Share IPD: No
Open or close this module References
Citations:
Links:
Available IPD/Information:

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