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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 : Recruiting
First Posted : March 28, 2019
Last Update Posted : September 10, 2019
Sponsor:
Information provided by (Responsible Party):
Spark Therapeutics

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.

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: SPK-3006 Neutralizing Antibody Not Applicable

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
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
Estimated Primary Completion Date : June 2022
Estimated Study Completion Date : June 2022


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: 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.




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.



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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.

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


Contacts
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Contact: Clinical Director +1 215-220-9300 clinicaltrials@sparktx.com
Contact: Clinical Director clinicaltrials@sparktx.com

Locations
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United States, Arizona
Barrow Neurological Institute Recruiting
Phoenix, Arizona, United States, 85013
Contact: Shafeeq Ladha, MD         
United States, California
University of California Irvine Health Recruiting
Orange, California, United States, 92868
Contact: Tahseen Mozaffar, MD         
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
Contact: Mazen Dimachkie, MD         
United States, Minnesota
University of Minnesota Medical School Not yet recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Chester Whitley, MD, PhD         
United States, Oregon
Oregon Health & Science University Recruiting
Portland, Oregon, United States, 97239
Contact: Chafic Karam, MD         
United States, Pennsylvania
University of Pittsburgh Medical Center Not yet recruiting
Pittsburgh, Pennsylvania, United States, 15213
Contact: Paula Clemens, MD         
France
Hôpital Raymond-Poincaré Recruiting
Garches, Hauts-de-Seine, France, 92380
Contact: Pascal Laforet, MD, PhD         
Assistance Publique Hôpitaux de Marseille Not yet recruiting
Marseille, France, 13385
Contact: Shahram Attarian, MD         
CHU Nice Not yet recruiting
Nice, France, 06001
Contact: Sabrina Sacconi, MD PhD         
Germany
Klinikum der Universität München Not yet recruiting
München, Germany, 80333
Contact: Benedikt Schoser, MD         
Italy
Università degli Studi di Messina Not yet recruiting
Messina, Italy, 98125
Contact: Antonio Toscano, MD         
Universita degli Studi di Milano - Clinica Oculistica I Not yet recruiting
Milano, Italy, 20122
Contact: Giacomo Comi, MD         
Università degli Studi di Napoli Federico II Not yet recruiting
Napoli, Italy, 80131
Contact: Giancarlo Parenti, MD         
Fondazione Mondino Istituto Neurologico Nazionale a Carattere Scientifico Not yet recruiting
Pavia, Italy, 27100
Contact: Sabrina Ravaglia, MD, PhD         
Azienda Ospedaliero Universitaria Pisana Not yet recruiting
Pisa, Italy, 56126
Contact: Gabriele Siciliano, MD, PhD         
Azienda Ospedaliera Universitaria Citta della Salute e della Scienza di Torino Not yet recruiting
Torino, Italy, 10126
Contact: Tiziana Mongini, MD         
Netherlands
Erasmus University Medical Center Not yet recruiting
Rotterdam, Netherlands, 3015 CE
Contact: Ans Van der Ploeg, MD, PhD         
United Kingdom
Salford Royal NHS Foundation Trust Not yet recruiting
Salford, United Kingdom, M6 8HD
Contact: Mark Roberts, MD         
Sponsors and Collaborators
Spark Therapeutics
Investigators
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Principal Investigator: Tahseen Mozaffar, MD University of California Irvine Health

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Responsible Party: Spark Therapeutics
ClinicalTrials.gov Identifier: NCT03893240     History of Changes
Other Study ID Numbers: SPK-GAA-100
First Posted: March 28, 2019    Key Record Dates
Last Update Posted: September 10, 2019
Last Verified: September 2019
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
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Carbohydrate Metabolism, Inborn Errors
Antibodies
Immunoglobulins
Antibodies, Blocking
Immunologic Factors
Physiological Effects of Drugs