STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT (NCT03347253) | Clinical Trial Compass
TerminatedNot Applicable
STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT
Stopped: As a result of change of clinical development plan.
United States12 participantsStarted 2017-12-08
Plain-language 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.
Who can participate
Age range18 Years – 75 Years
SexALL
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Inclusion criteria
✓. 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.
Exclusion criteria
✕. 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:
✕. Subject requires use of invasive or non-invasive ventilatory support for \> 6 hours a day while awake.
What they're measuring
1
Evaluate degree of change in muscle function and respiratory endpoints over time
✕. 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.
✕. Other exclusion criteria according to the Lumizyme/Myozyme instructions for use.