First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
Completed · Phase 1/Phase 2
Conditions studied: Pompe Disease
In brief
This is an international, multi-center, open-label study designed to evaluate if the co-administration of investigational new drugs ATB200 and AT2221 is safe in adults with Pompe disease.
Key facts
- Study ID
- NCT02675465
- Run by
- Amicus Therapeutics
- People needed
- 29
- Starts
- 2016-04-01
- Expected to finish
- 2024-08-22
- Last updated by the study team
- 2025-10-23
Who can join
Age: 18 and older, up to 75. Sex: any. Healthy volunteers: not accepted.
You may not qualify if…
- Received treatment with prohibited medications within 30 days of Baseline Visit
- Subject, if female, was pregnant or breastfeeding at screening
- Subject, whether male or female, planned to conceive a child during the study
- Had a medical or any other extenuating condition or circumstance that may, in opinion of investigator, pose an undue safety risk to the subject or compromise his/her ability to comply with protocol requirements
- Had a history of allergy or sensitivity to alglucosidase alfa, miglustat or other iminosugars (Cohorts 1, 2, and 4)
- Required invasive ventilatory support, or used noninvasive ventilatory support ≥ 6 hours a day while awake (Cohorts 1, 3, and 4)
- Had active systemic autoimmune disease such as lupus, scleroderma, or rheumatoid arthritis; subjects with autoimmune disease must have been discussed with the Amicus Medical Monitor
- Had active bronchial asthma; subjects with bronchial asthma must have been discussed with the Amicus Medical Monitor
Where it is running
- Neuromuscular Research Centre — Phoenix, Arizona, United States
- University of California Irvine — Orange, California, United States
- University of Florida — Gainesville, Florida, United States
- Emory University Division of Medical Genetics — Decatur, Georgia, United States
- Infusion Associates — Grand Rapids, Michigan, United States
- Great Falls Clinic, LLP — Great Falls, Montana, United States
- Rutgers New Jersey Medical School — Newark, New Jersey, United States
- Duke University Medical Center — Durham, North Carolina, United States
- Perelman Center for Advanced Medicine — Philadelphia, Pennsylvania, United States
- University of Pittsburgh — Pittsburgh, Pennsylvania, United States
- Abramson Cancer Center Chester County Hospital — West Chester, Pennsylvania, United States
- Lysosomal & Rare Disorders Research & Treatment Center (LDRTC) — Fairfax, Virginia, United States
- Womens & Childrens Hospital, Adelaide — North Adelaide, South Australia, Australia
- University Children's Hospital Department of Neuropediatrics and Inborn Metabolic Disorders, St. Josefs-Hospital — Bochum, Germany
- Friedrich-Baur-Institure, Dep of Neurology - University Munich — Munich, Germany
- Erasmus Medical Center — Rotterdam, Netherlands
- School of Medicine, University of Auckland — Auckland, New Zealand
- University Hospital Birmingham NHS Foundation Trust, Queen Elizabeth Medical Center — Birmingham, United Kingdom
- Salford Royal NHS Foundation Trust — Salford, United Kingdom
Full record on ClinicalTrials.gov
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