🇮🇪Ireland
16°C Partly Cloudy · Dublin
Live Updates
--:--:-- IST
Writer Login
Latest
Priya Life Science Sponsor trial record
Trackers Companies Trial finder
Sponsor · clinical trials

Amicus Therapeutics clinical trials

Source: ClinicalTrials.gov, U.S. National Library of Medicine and the EU Clinical Trials Information System (CTIS) — current trials in our USA, UK and EU (CTIS) registers with Amicus Therapeutics as lead sponsor. Synced 26 Sep 2026
8Current trials
5Recruiting now
6Drugs in these trials
3Trial registers
Maintained by Sreepriya Prasannan
MSc Digital Transformation of Life Sciences (Innopharma Education / Griffith College); MSc & BSc Botany HSE Spark Ignite 2026 — Top 14 finalist
Compiled from ClinicalTrials.gov and CTIS registry records · Last verified · Editorial standards
At a glance

Amicus Therapeutics is the lead sponsor of 8 current clinical trials in our USA, UK and EU (CTIS) registers. 5 are recruiting patients now. The most common phase is Phase 3. The drugs named most often in its trials are Migalastat HCl, Cipaglucosidase alfa and Miglustat. The most-studied condition is Fabry Disease.

ClinicalTrials.gov trials by status

  • Recruiting571%
  • Active, closed229%

Trials by phase

  • Phase 3457%
  • Observational343%
01

Clinical trials sponsored by Amicus Therapeutics

7 on ClinicalTrials.gov
StudyPhaseStatusStartRegisters
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
NCT04808505 · Glycogen Storage Disease Type II Infantile Onset
Phase 3 Recruiting 2023-07 UK, USA
A Global Prospective Observational Registry of Patients With Pompe Disease
NCT06121011 · Pompe Disease
Observational Recruiting 2024-02 UK, USA
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
NCT06904261 · Fabry Disease
Phase 3 Recruiting 2026-01 UK, USA
A Study of Patients With Fabry Disease (US Specific)
NCT06906367 · ClinicalTrials.gov ↗ · Fabry Disease
Observational Recruiting 2026-02 USA
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
NCT04252066 · ClinicalTrials.gov ↗ · Fabry Disease
Observational Recruiting 2025-03 USA
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
NCT03911505 · Pompe Disease (Late-onset)
Phase 3 Active, closed 2020-02 USA
A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
NCT04020055 · ClinicalTrials.gov ↗ · Fabry Disease
Phase 3 Active, closed 2022-10 UK, USA

Registers list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Titles and statuses are copied from the registry as published.

02

Amicus Therapeutics in the EU trials register (CTIS)

1 trial

Authorised or ongoing trials in the EU Clinical Trials Information System with Amicus Therapeutics as sponsor. Some are the same study as a ClinicalTrials.gov record above; the two registries share no common ID. Trials with a site in Ireland are listed first.

StudyPhaseStatusCountries
An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12-month Treatment with Migalastat in Pediatric Subjects (aged 2 to < 12 years) with Fabry Disease and Amenable GLA Variants
2025-521244-38-00 · CTIS ↗ · Nutritional and Metabolic Diseases
Phase 3 Authorised Spain, Germany, Belgium
03

Drugs in Amicus Therapeutics trials

6 compounds

Drugs and biologicals named as interventions in these trials, with the number of this sponsor's trials that name each one. Placebos and generic comparators are excluded.

04

Conditions in Amicus Therapeutics trials

4 conditions
Fabry Disease4Glycogen Storage Disease Type II Infantile Onset1Pompe Disease1Pompe Disease (Late-onset)1
05

By trial register

RegisterTrialsRecruiting
Clinical trials in USA75
Clinical trials in UK43
EU register (CTIS)1—

A trial with sites in more than one country appears in each of those registers.

06

Other sponsors in Fabry Disease

SponsorTrials
Chiesi Farmaceutici3
Sangamo Therapeutics1
Idorsia Pharmaceuticals1
Shire1

Sponsors with the most current trials registered for this condition.

07

Questions about Amicus Therapeutics trials

How many clinical trials does Amicus Therapeutics sponsor?

Amicus Therapeutics is the lead sponsor of 7 current trials on ClinicalTrials.gov in our USA and UK registers, and 1 authorised or ongoing trial in the EU register (CTIS), as of the registry sync on 26 September 2026. Completed and withdrawn studies are not counted.

Are Amicus Therapeutics trials recruiting?

Yes. 5 of its 7 ClinicalTrials.gov trials are recruiting. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.

Which drugs is Amicus Therapeutics testing?

Its current trials name 6 drugs and biologicals as interventions. The most frequent are Migalastat HCl (4), Cipaglucosidase alfa (3), Miglustat (3), Alglucosidase alfa (1) and Avalglucosidase alfa (1). The number in brackets is how many of its trials name each drug.

Where does this information come from?

Every trial, status, phase and condition on this page is copied from public ClinicalTrials.gov and EU CTIS records by a daily sync, grouped by the sponsor name each registry records. It says nothing about the organisation beyond its registered trials, and it is not medical advice.

Approvals, shortages and trials, every Friday

One email a week with new EMA and FDA approvals, EU and US medicine shortages, how many trials are recruiting in Ireland, and the week's Irish pharma news.

Free. Unsubscribe from any email with one click. See our privacy policy.

How this page is built

Everything on this page is copied from public registry records — it is not written from memory, and nothing here describes the organisation beyond its registered trials. Each trial links to its own record so you can check it yourself.

This page is built automatically: a daily sync job reads the registry records behind our trial registers and groups every current trial by its lead sponsor name, ignoring legal-form suffixes such as Inc. or Ltd. A small, hand-checked list joins the same organisation's different registry names (for example its EU legal entity). There is no AI drafting and no per-sponsor editorial pass. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The the registries data on this page was last retrieved on 26 Sep 2026.

Please verify before you rely on this. This page is general information for life-science and pharmaceutical professionals. It is not medical advice, and it has not been reviewed by a clinician — our editorial team holds life-science qualifications, not clinical ones. It is not exhaustive and may not reflect the most recent label change. Always check the official prescribing information (US Prescribing Information or EU SmPC) and speak to your doctor or pharmacist before acting on anything here. Drugs in the same class are not automatically interchangeable, and approvals, brand names and indications differ between the US, the EU/Ireland (EMA/HPRA) and other regions. Spotted an error? Tell us — we correct promptly and log it.