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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT03893240
Other study ID # SPK-GAA-100
Secondary ID
Status Completed
Phase N/A
First received
Last updated
Start date June 12, 2019
Est. completion date October 27, 2020

Study information

Verified date March 2023
Source Spark Therapeutics
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

Status Completed
Enrollment 61
Est. completion date October 27, 2020
Est. primary completion date October 27, 2020
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility 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.

Study Design


Intervention

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.

Locations

Country Name City State
France Hôpital Raymond-Poincaré Garches Hauts-de-Seine
France Assistance Publique Hôpitaux de Marseille Marseille
France CHU Nice Nice
Germany Klinikum der Universität München München
Italy Università degli Studi di Messina Messina
Italy Universita degli Studi di Milano - Clinica Oculistica I Milano
Italy Università degli Studi di Napoli Federico II Napoli
Italy Fondazione Mondino Istituto Neurologico Nazionale a Carattere Scientifico Pavia
Italy Azienda Ospedaliero Universitaria Pisana Pisa
Italy Azienda Ospedaliera Universitaria Citta della Salute e della Scienza di Torino Torino
Netherlands Erasmus University Medical Center Rotterdam
United Kingdom Salford Royal NHS Foundation Trust Salford
United States University of Kansas Medical Center Kansas City Kansas
United States University of Minnesota Medical School Minneapolis Minnesota
United States University of California Irvine Health Orange California
United States Barrow Neurological Institute Phoenix Arizona
United States University of Pittsburgh Medical Center Pittsburgh Pennsylvania
United States Oregon Health & Science University Portland Oregon

Sponsors (1)

Lead Sponsor Collaborator
Spark Therapeutics

Countries where clinical trial is conducted

United States,  France,  Germany,  Italy,  Netherlands,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Neutralizing Antibodies Titer to SPK-3006 capsid The neutralizing antibodies titer to SPK-3006 is measured once prospectively at one site visit. 1 day
Primary Occurrence of Neutralizing Antibodies to SPK-3006 capsid The proportion of participants who have Neutralizing Antibodies to SPK-3006 capsid. 1 day
Secondary Anti-GAA binding antibodies Titer Anti-GAA binding antibodies titer is measured once prospectively at one site visit. 1 day
Secondary Occurrence of Anti-GAA binding antibodies across participants The proportion of participants who have Anti-GAA binding antibodies. 1 day
Secondary Neutralizing antibodies to circulating GAA Titer 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. 1 day
Secondary Occurrence of Neutralizing antibodies to circulating GAA The proportion of participants who have neutralizing antibodies to circulating GAA. 1 day
Secondary GAA activity level 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. 1 day
Secondary GAA antigen level 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. 1 day
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