STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT

NCT03347253 | Terminated

• 1 other identifier: POM-003
• Study Type: observational
• Target: 12
• Locations: 4 countries
• Sites: 21

Brief Summary

The purpose of the study is to evaluate changes in key clinical outcome measures (eg, motor, respiratory, fatigue) in adult subjects with late-onset Pompe disease (LOPD) subjects receiving standard-of-care enzyme replacement therapy (ERT). Additionally, information gained may be used in the design and conduct of future studies in LOPD subjects.

Conditions

Late-onset Pompe Disease

Outcome Measures

Primary Outcomes (1)

• Evaluate degree of change in muscle function and respiratory endpoints over time

  To evaluate the degree of change in muscle function and respiratory endpoints over time in patients with Late Onset Pompe disease

  6-15 month

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Male and female subjects with LOPD between 18 years and 75 years, inclusive and ≥ 50 kg.

You may qualify if:

• Subject has a diagnosis of Pompe disease based on documented deficiency of GAA activity and a documented GAA mutation.
• Male and female subjects between 18 years and 75 years, inclusive and ≥ 50 kg.
• Subject must be currently receiving standard-of-care ERT (alglucosidase alfa) at a dose of 20 mg/kg dose every other week.
• Subject must have been on ERT for the preceding 2 years or more.
• Subject must have an upright forced vital capacity (FVC) within 35 to 90% of predicted normal (NHANES III reference values), based on the higher of the screening or baseline value, if their 6 minute walk distance (6MWD) is \> 200 m. Subject must have an upright FVC within 40 to 90% of predicted normal (NHANES III reference values), based on the higher of the screening or baseline value, if their 6MWD is ≤ 200 m. If FVC is between 80 and 90% of predicted normal, the subject may enter the study if the percent predicted FVC value drops by 10% predicted or more in supine position
• Subject is able to walk at least 100 m in the 6MWT and the assessment is noted as valid.

You may not qualify if:

• Subject has received any investigational therapy or pharmacological treatment for Pompe disease, other than alglucosidase alfa within 30 days or 5 half lives, whichever is shorter, prior to the Baseline Visit or is anticipated to do so during the course of the study
• Subject is on any of the following prohibited medications within 30 days of baseline:
• miglitol (eg, Glyset)
• miglustat (eg, Zavesca)
• acarbose (eg, Precose, Glucobay)
• voglibose (eg, Volix, Vocarb, Volibo)
• Subject requires use of invasive or non-invasive ventilatory support for \> 6 hours a day while awake.
• Subject has a medical or any other extenuating condition or circumstance that may, in the opinion of the investigator, pose an undue safety risk to the subject or compromise his/her ability to comply with protocol requirements. This includes clinical depression (as diagnosed by a psychiatrist or other mental health professional) with uncontrolled or poorly controlled symptoms.
• Subject is breastfeeding, or is pregnant or planning to become pregnant within the next 2 years.

Sponsors & Collaborators

• Amicus Therapeutics: lead

Study Sites (21)

University of California, Irvine

Irvine, California, 92868, United States

Location

University of Florida Clinical Research Center

Gainesville, Florida, 32611, United States

Location

Emory University

Decatur, Georgia, 30033, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Baystate Medical Center

Springfield, Massachusetts, 01199, United States

Location

University of Minnesota Medical Center

Minneapolis, Minnesota, 55455, United States

Location

Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

Location

Jacobs & Levy Genomic Medicine and Research Program

Morristown, New Jersey, 07960, United States

Location

Northwell Health

Great Neck, New York, 11021, United States

Location

NYU Neurogenetics

New York, New York, 10016, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Cincinnati Children's Hospital

Cincinnati, Ohio, 45229, United States

Location

Ohio State University

Columbus, Ohio, 43210, United States

Location

Oregon Health& Science University

Portland, Oregon, 97239, United States

Location

Penn State Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

University of Pittsburgh

Pittsburgh, Pennsylvania, 15261, United States

Location

The University of Texas Health Science Center San Antonio

San Antonio, Texas, 78229, United States

Location

Royal Adelaide Hospital

Adelaide, 5000, Australia

Location

Antwerp University Hospital

Edegem, 1650, Belgium

Location

Alberta Children's Hospital

Calgary, Alberta, Canada

Location

McMaster University Medical Center

Hamilton, Ontario, Canada

Location

Biospecimen

Retention: SAMPLES WITH DNA

blood

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 9, 2017
• First Posted: November 20, 2017
• Study Start: December 8, 2017
• Primary Completion: November 30, 2018
• Study Completion: November 30, 2018
• Last Updated: July 8, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1); BE (1); US (17); CA (2)