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

Pompe Disease Clinical Trial

Official title:

A Multi-Center, Low-Interventional Study With a Retrospective Component in Participants With Late-Onset Pompe Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03893240
• Other study ID #: SPK-GAA-100
• Status: Completed
• Phase: N/A
• 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
• 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.

Related Conditions & MeSH terms

• Acid Maltase Deficiency
• Glycogen Storage Disease
• Glycogen Storage Disease Type 2
• Glycogen Storage Disease Type II
• LOPD
• Lysosomal Storage Diseases
• Metabolic Diseases
• Pompe Disease
• Pompe Disease (Late-onset)

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