Last reviewed · How we verify
NCT03865836
Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease
trial testing ATB200 in Pompe Disease. Available.
Quick facts
| Lead sponsor | Amicus Therapeutics |
|---|---|
| Status | AVAILABLE |
| Study type | EXPANDED_ACCESS |
Drugs / interventions tested
Conditions studied
- Pompe Disease — all drugs for Pompe Disease →
Sponsor
Amicus Therapeutics — full company profile →
Who can join
Eligibility, any sex, with Pompe Disease. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.
Publications & conference data
2 peer-reviewed publications reference this trial (live from Europe PMC):
-
Pompe disease: pathogenesis, molecular genetics and diagnosis.
Taverna S, Cammarata G, Colomba P, Sciarrino S, et al · · 2020 · cited 61× · PMID 32745073 · DOI 10.18632/aging.103794 -
Cipaglucosidase Alfa: First Approval.
Blair HA. · · 2023 · cited 27× · PMID 37184753 · DOI 10.1007/s40265-023-01886-5
Verify or expand the search:
- PubMed search for NCT03865836
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other trials of ATB200
Trials testing the same drug.
- NCT04138277 — A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LO · Phase 3 · completed
- NCT04327973 — Expanded Access for ATB200/AT2221 for the Treatment of IOPD · available
Other recruiting trials for Pompe Disease
Currently open trials in the same condition.
- NCT07123155 — Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD) · Phase 2 · recruiting
- NCT06539169 — FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases · recruiting
- NCT06121011 — A Global Prospective Observational Registry of Patients With Pompe Disease · recruiting
- NCT05943678 — Novel Metabolic Muscular Biomarkers in Pompe Disease - a Non-invasive Magnetic Resonance Exploratory Pilot Study. · active not recruiting
- NCT05734521 — Avalglucosidase Alfa Pregnancy Study · recruiting
Other Amicus Therapeutics trials
Trials by the same sponsor.
- NCT06121011 — A Global Prospective Observational Registry of Patients With Pompe Disease · recruiting
- NCT04804566 — Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients · completed
- NCT04020055 — A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease · Phase 3 · active not recruiting
- NCT04281537 — A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme R · completed
- NCT04138277 — A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LO · Phase 3 · completed
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT03865836 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Amicus Therapeutics
- Last refreshed: 15 September 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03865836.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing