Late-onset Pompe DiseaseNCT03347253
STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT
An observational study of Late-onset Pompe Disease, run by Amicus Therapeutics at 21 sites in 4 countries.
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). Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Late-onset Pompe Disease
- How many people
- 12 participants
- Where
- 21 sitesin 4 countries
- Study dates
- 8 Dec 2017to 30 Nov 2018
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Male and female subjects with LOPD between 18 years and 75 years, inclusive and ≥ 50 kg.
Who may be able to join
- 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.
Who cannot join
- 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)
Show all 10Show fewer
- 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.
Trial locations and contacts
21 sites in 4 countries.
21 sites in 4 countries
United States17 sites
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
Ohio State University
Columbus, Ohio, 43210
Emory University
Decatur, Georgia, 30033
Duke University Medical Center
Durham, North Carolina, 27710
University of Florida Clinical Research Center
Gainesville, Florida, 32611
Northwell Health
Great Neck, New York, 11021
Hackensack University Medical Center
Hackensack, New Jersey, 07601
Penn State Hershey Medical Center
Hershey, Pennsylvania, 17033
University of California, Irvine
Irvine, California, 92868
University of Kansas Medical Center
Kansas City, Kansas, 66160
University of Minnesota Medical Center
Minneapolis, Minnesota, 55455
Jacobs & Levy Genomic Medicine and Research Program
Morristown, New Jersey, 07960
NYU Neurogenetics
New York, New York, 10016
University of Pittsburgh
Pittsburgh, Pennsylvania, 15261
Oregon Health& Science University
Portland, Oregon, 97239
The University of Texas Health Science Center San Antonio
San Antonio, Texas, 78229
Baystate Medical Center
Springfield, Massachusetts, 01199
Canada2 sites
Alberta Children's Hospital
Calgary, Alberta
McMaster University Medical Center
Hamilton, Ontario
About this study
Official title: A Prospective Study in Subjects With Late Onset Pompe Disease Who Are Currently Being Treated With Enzyme Replacement Therapy
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.
Read the detailed 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.
- Conditions
- Late-onset Pompe Disease
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 6-15 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Nov 2017
- Study start8 Dec 2017
- Primary completion30 Nov 2018
- Study completion30 Nov 2018
Why it stopped: As a result of change of clinical development plan.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Late-onset Pompe DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amicus Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03347253
- Sponsor's protocol ID
- POM-003
- Status last verified
- Jul 2025
- First submitted
- 9 Nov 2017
- Last updated
- 8 Jul 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Study in Subjects With Late Onset Pompe Disease Who Are Currently Being Treated With Enzyme Replacement Therapy
- ClinicalTrials.gov ID
- NCT03347253
- Acronym / sponsor ID
- POM-003
- Conditions
- Late-onset Pompe Disease
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: As a result of change of clinical development plan.
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Amicus TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 8 Dec 2017 (actual)
- Primary completion
- 30 Nov 2018 (actual)
- Study completion
- 30 Nov 2018 (actual)
- First posted
- 20 Nov 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Male and female subjects with LOPD between 18 years and 75 years, inclusive and ≥ 50 kg.
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (10 listed)
- 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.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
Results and safety
The study was stopped early (reason given: As a result of change of clinical development plan.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jul 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
21 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Late-onset Pompe Disease, run by Amicus Therapeutics at 21 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 8 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 21 sites in United States, Canada, Australia, Belgium. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: As a result of change of clinical development plan.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate degree of change in muscle function and respiratory endpoints over time (time frame: 6-15 month). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jul 2025.