Trial record 1 of 1 for:
pom-003
STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT
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| ClinicalTrials.gov Identifier: NCT03347253 |
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Recruitment Status :
Terminated
(As a result of change of clinical development plan.)
First Posted : November 20, 2017
Last Update Posted : February 8, 2019
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Sponsor:
Amicus Therapeutics
Information provided by (Responsible Party):
Amicus Therapeutics
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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.
| Condition or disease |
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| Late-onset Pompe Disease |
| Study Type : | Observational |
| Actual Enrollment : | 12 participants |
| Observational Model: | Case-Only |
| Time Perspective: | Prospective |
| Official Title: | A Prospective Study in Subjects With Late Onset Pompe Disease Who Are Currently Being Treated With Enzyme Replacement Therapy |
| Actual Study Start Date : | December 8, 2017 |
| Actual Primary Completion Date : | November 30, 2018 |
| Actual Study Completion Date : | November 30, 2018 |
Resource links provided by the National Library of Medicine
Primary Outcome Measures :
- Evaluate degree of change in muscle function and respiratory endpoints over time [ Time Frame: 6-15 month ]To evaluate the degree of change in muscle function and respiratory endpoints over time in patients with Late Onset Pompe disease
Biospecimen Retention: Samples With DNA
blood
Information from the National Library of Medicine
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| Ages Eligible for Study: | 18 Years to 75 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
Study Population
Male and female subjects with LOPD between 18 years and 75 years, inclusive and ≥ 50 kg.
Criteria
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
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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.
- Other exclusion criteria according to the Lumizyme/Myozyme instructions for use.
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03347253
Locations
Show 21 study locations
Sponsors and Collaborators
Amicus Therapeutics
| Responsible Party: | Amicus Therapeutics |
| ClinicalTrials.gov Identifier: | NCT03347253 |
| Other Study ID Numbers: |
POM-003 |
| First Posted: | November 20, 2017 Key Record Dates |
| Last Update Posted: | February 8, 2019 |
| Last Verified: | February 2019 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |
| Product Manufactured in and Exported from the U.S.: | No |
Additional relevant MeSH terms:
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Glycogen Storage Disease Type II Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases |
Metabolism, Inborn Errors Genetic Diseases, Inborn Glycogen Storage Disease Carbohydrate Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases |