Clinical trial NCT03347253: STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT
Official title: A Prospective Study in Subjects With Late Onset Pompe Disease Who Are Currently Being Treated With Enzyme Replacement Therapy
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Late-onset Pompe Disease
Lead sponsor: Amicus Therapeutics
Sponsor class: INDUSTRY
First posted: 2017-11-20
Enrollment: 12
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Male and female subjects with LOPD between 18 years and 75 years, inclusive and ≥ 50 kg.

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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Evaluate degree of change in muscle function and respiratory endpoints over time
timeFrame: 6-15 month
Secondary outcomes:
Other outcomes:
Locations:
city: Irvine
country: United States
facility: University of California, Irvine
state: California
city: Gainesville
country: United States
facility: University of Florida Clinical Research Center
state: Florida
city: Decatur
country: United States
facility: Emory University
state: Georgia
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Springfield
country: United States
facility: Baystate Medical Center
state: Massachusetts
city: Minneapolis
country: United States
facility: University of Minnesota Medical Center
state: Minnesota
city: Hackensack
country: United States
facility: Hackensack University Medical Center
state: New Jersey
city: Morristown
country: United States
facility: Jacobs & Levy Genomic Medicine and Research Program
state: New Jersey
city: Great Neck
country: United States
facility: Northwell Health
state: New York
city: New York
country: United States
facility: NYU Neurogenetics
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital
state: Ohio
city: Columbus
country: United States
facility: Ohio State University
state: Ohio
city: Portland
country: United States
facility: Oregon Health& Science University
state: Oregon
city: Hershey
country: United States
facility: Penn State Hershey Medical Center
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh
state: Pennsylvania
city: San Antonio
country: United States
facility: The University of Texas Health Science Center San Antonio
state: Texas
city: Adelaide
country: Australia
facility: Royal Adelaide Hospital
city: Edegem
country: Belgium
facility: Antwerp University Hospital
city: Calgary
country: Canada
facility: Alberta Children's Hospital
state: Alberta
city: Hamilton
country: Canada
facility: McMaster University Medical Center
state: Ontario
Study officials:
References: