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

Late-onset Pompe Disease Clinical Trial

Official title:

A Prospective Study in Subjects With Late Onset Pompe Disease Who Are Currently Being Treated With Enzyme Replacement Therapy

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03347253
• Other study ID #: POM-003
• Status: Terminated
• Start date: December 8, 2017
• Est. completion date: November 30, 2018

Study information

• Verified date: February 2019
• Source: Amicus Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Description:

The objective of this study is to evaluate the baseline characteristics and degree of change over time in clinical outcome measures commonly used to evaluate patients with LOPD.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 12
• Est. completion date: November 30, 2018
• Est. primary completion date: November 30, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

Inclusion Criteria:

1. Subject has a diagnosis of Pompe disease based on documented deficiency of GAA activity and a documented GAA mutation.

2. Male and female subjects between 18 years and 75 years, inclusive and = 50 kg.

3. Subject must be currently receiving standard-of-care ERT (alglucosidase alfa) at a dose of 20 mg/kg dose every other week.

4. Subject must have been on ERT for the preceding 2 years or more.

5. 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

6. Subject is able to walk at least 100 m in the 6MWT and the assessment is noted as valid.

Exclusion Criteria:

1. 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

2. 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)

3. Subject requires use of invasive or non-invasive ventilatory support for > 6 hours a day while awake.

4. 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.

5. Subject is breastfeeding, or is pregnant or planning to become pregnant within the next 2 years.

6. Other exclusion criteria according to the Lumizyme/Myozyme instructions for use.

Related Conditions & MeSH terms

• Glycogen Storage Disease Type II
• Late-onset Pompe Disease

Locations

Country	Name	City	State
Australia	Royal Adelaide Hospital	Adelaide
Belgium	Antwerp University Hospital	Edegem
Canada	Alberta Children's Hospital	Calgary	Alberta
Canada	McMaster University Medical Center	Hamilton	Ontario
United States	Cincinnati Children's Hospital	Cincinnati	Ohio
United States	Ohio State University	Columbus	Ohio
United States	Emory University	Decatur	Georgia
United States	Duke University Medical Center	Durham	North Carolina
United States	University of Florida Clinical Research Center	Gainesville	Florida
United States	Northwell Health	Great Neck	New York
United States	Hackensack University Medical Center	Hackensack	New Jersey
United States	Penn State Hershey Medical Center	Hershey	Pennsylvania
United States	University of California, Irvine	Irvine	California
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	University of Minnesota Medical Center	Minneapolis	Minnesota
United States	Jacobs & Levy Genomic Medicine and Research Program	Morristown	New Jersey
United States	NYU Neurogenetics	New York	New York
United States	University of Pittsburgh	Pittsburgh	Pennsylvania
United States	Oregon Health& Science University	Portland	Oregon
United States	The University of Texas Health Science Center San Antonio	San Antonio	Texas
United States	Baystate Medical Center	Springfield	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Amicus Therapeutics

Countries where clinical trial is conducted

United States,  Australia,  Belgium,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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