Trials Sponsors Conditions Drugs Pricing

Conditions

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

Outcome Measures

Primary Outcomes (2)

Neutralizing Antibodies Titer to SPK-3006 capsid
The neutralizing antibodies titer to SPK-3006 is measured once prospectively at one site visit.
1 day
Occurrence of Neutralizing Antibodies to SPK-3006 capsid
The proportion of participants who have Neutralizing Antibodies to SPK-3006 capsid.
1 day

Secondary Outcomes (6)

Anti-GAA binding antibodies Titer
1 day

Study Arms (1)

Participants with Late Onset Pompe disease

OTHER

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

Interventions

Neutralizing Antibody to SPK-3006 capsidDiagnostic test

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.

Participants with Late Onset Pompe disease

Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.

Eligibility Criteria

Age18 Years+

Sexall

Healthy VolunteersNo

Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

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

Sponsors & Collaborators

Spark Therapeutics, Inc.
Industrylead

Study Sites (18)

Barrow Neurological Institute

Phoenix, Arizona, 85013, United States

University of California Irvine Health

Orange, California, 92868, United States

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

University of Minnesota Medical School

Minneapolis, Minnesota, 55455, United States

Oregon Health & Science University

Portland, Oregon, 97239, United States

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15213, United States

Hôpital Raymond-Poincaré

MeSH Terms

Conditions

Glycogen Storage Disease Type IILysosomal Storage Diseases

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGlycogen Storage DiseaseCarbohydrate Metabolism, Inborn ErrorsMetabolic Diseases

Study Officials

Tahseen Mozaffar, MD
University of California Irvine Health
PRINCIPAL INVESTIGATOR

Enrollment

61 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: not applicable
Allocation: NA
Masking: NONE
Purpose: SCREENING
Intervention Model: SINGLE GROUP
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

March 11, 2019

First Posted

March 28, 2019

Study Start

June 12, 2019

Actual (reported)

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

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (2)

Secondary Outcomes (6)

Study Arms (1)

Participants with Late Onset Pompe disease

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (18)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (2)

Secondary Outcomes (6)

Study Arms (1)

Participants with Late Onset Pompe disease

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (18)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations