Cipaglucosidase alfa clinical trials
Cipaglucosidase alfa is named as an intervention in 3 current clinical trials in our USA, Germany, Italy, UK, Netherlands, Taiwan, Belgium, Denmark, Poland, Austria, Canada, Japan and Australia registers. 2 are recruiting patients now. The most common phase is Phase 3. The lead sponsor is Amicus Therapeutics. The most-studied condition is Glycogen Storage Disease Type II Infantile Onset. The EU clinical trials register (CTIS) separately lists 1 authorised or ongoing trial.
Where Cipaglucosidase alfa is approved
All regulators →Of the three regulators tracked here, the first to approve Cipaglucosidase alfa was the EU, on 20 Mar 2023. It is approved in 3 of the 3 regulators tracked here.
From the regulators' own registers: FDA Drugs@FDA, the EMA medicines list and Health Canada's Notice of Compliance database. Dates are the first listed approval of a product containing only this substance; combination products and nationally authorised EU medicines are not counted.
What health systems spend on Cipaglucosidase alfa
All medicines →From NHSBSA Prescription Cost Analysis (Open Government Licence v3.0) and CMS State Drug Utilization Data. Both are gross amounts: neither system publishes its discounts or rebates per drug. Products combining several substances are not counted here.
NICE appraisals of Cipaglucosidase alfa
All NICE appraisals →NICE, which decides whether the NHS in England should fund new medicines, has published 1 technology appraisal of Cipaglucosidase alfa. 1 is current, and 1 of it recommends it for at least some patients.
| Appraisal | NICE recommendation | Published | Status |
|---|---|---|---|
| Cipaglucosidase alfa with miglustat for treating late-onset Pompe disease NICE TA912 | Recommended | 15 Aug 2023 | Current |
NICE decisions apply to the NHS in England. In Ireland the HSE decides on reimbursement, advised by the National Centre for Pharmacoeconomics. Titles, categories and dates link to the guidance on nice.org.uk. © NICE 2026 technology appraisal guidance. Available from www.nice.org.uk/guidance. All rights reserved. Subject to Notice of rights. NICE guidance is prepared for the National Health Service in England. All NICE guidance is subject to regular review and may be updated or withdrawn. NICE accepts no responsibility for the use of its content in this product/publication.
Patient leaflets for Cipaglucosidase alfa
The package leaflet is the official guide for patients that comes with every medicine. These links go to the regulators that publish it.
- IEIreland: 1 product on the HPRA listEach product record links to the HPRA, which publishes the leaflet and SmPC.
- EUEuropean Union: PombilitiEMA product information: package leaflet and summary of product characteristics in every EU language.
- UKUnited Kingdom: Search MHRA ProductsPatient information leaflets and SmPCs for UK-licensed products.
- USUnited States: Search DailyMedFDA-approved labels, including patient and medication guides.
Leaflets differ between brands and countries; always read the one that comes with your own medicine. This is regulatory information, not medical advice.
Trials by status
Trials by phase
Clinical trials of Cipaglucosidase alfa
3 trials| Study | Phase | Status | Lead sponsor | Registers |
|---|---|---|---|---|
|
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 | Amicus Therapeutics | UK, USA, Germany, Italy, Netherlands, Taiwan |
|
A Global Prospective Observational Registry of Patients With Pompe Disease
NCT06121011 · Pompe Disease
|
Observational | Recruiting | Amicus Therapeutics | UK, USA, Germany, Italy, Netherlands, Belgium, Denmark, Poland, Austria |
|
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 | Amicus Therapeutics | USA, Germany, Italy, Canada, Japan, Australia |
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. Trial titles and statuses are copied from the registry as published.
Cipaglucosidase alfa in the EU trials register (CTIS)
1 trialAuthorised or ongoing trials in the EU Clinical Trials Information System that list a product containing Cipaglucosidase alfa. Many trials are registered in both CTIS and ClinicalTrials.gov, so these are shown separately rather than added to the count above. Trials with a site in Ireland are listed first.
| Study | Phase | Status | Sponsor | Countries |
|---|---|---|---|---|
|
A long-term prospective monitoring study of all patients with Pompe Disease. Evaluating natural history, treatment response, disease markers and novel outcome measures.
2024-520365-31-01 · Nutritional and Metabolic Diseases
|
Phase 4 | Authorised | Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC) | Netherlands |
Cipaglucosidase alfa across our data
Ireland · EU · USThe same active substance in our other registers, matched by its exact name. Each entry links to the register record it comes from.
Licensed in Ireland (HPRA)
1 single-substance product containing Cipaglucosidase alfa on the HPRA medicines list.
- Pombiliti 105 mg powder for concentrate for solution for infusionAmicus Therapeutics Europe Limited · EU/1/22/1714/001-003
Supply shortages
No entry for Cipaglucosidase alfa in the EMA, FDA or German BfArM shortage lists we track.
Conditions studied with Cipaglucosidase alfa
3 conditionsSponsors
lead sponsor per trial| Lead sponsor | Trials |
|---|---|
| Amicus Therapeutics | 3 |
By country register
A trial with sites in more than one country appears in each of those registers. CTIS does not publish a recruiting flag; its trials may also appear in the registers above.
Related compounds
Questions about Cipaglucosidase alfa trials
How many clinical trials are testing Cipaglucosidase alfa?
3 current trials in our USA, Germany, Italy, UK, Netherlands, Taiwan, Belgium, Denmark, Poland, Austria, Canada, Japan and Australia registers name Cipaglucosidase alfa (also registered as ATB200) as a drug or biological intervention, as of the registry sync on 10 November 2026. Completed and withdrawn studies are not counted.
Are Cipaglucosidase alfa trials recruiting patients?
Yes. 2 of the 3 trials are recruiting: 2 with sites in USA, 2 with sites in Germany, 2 with sites in Italy, 2 with sites in UK, 2 with sites in Netherlands, 1 with sites in Taiwan, 1 with sites in Belgium, 1 with sites in Denmark, 1 with sites in Poland and 1 with sites in Austria. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.
Who sponsors Cipaglucosidase alfa clinical trials?
The lead sponsor is Amicus Therapeutics. The number in brackets is how many trials each organisation leads.
Is Cipaglucosidase alfa licensed in Ireland?
Yes. The HPRA medicines list includes 1 single-substance product containing Cipaglucosidase alfa, for example Pombiliti 105 mg powder for concentrate for solution for infusion (Amicus Therapeutics Europe Limited). A licence does not mean a product is currently marketed or reimbursed; see each product's record.
Is there a Cipaglucosidase alfa shortage?
Not in the lists we track: Cipaglucosidase alfa has no entry in the EMA, FDA or German BfArM shortage registers as of their latest sync.
Where does this information come from?
Every trial, status, phase and sponsor on this page is copied from public ClinicalTrials.gov records by a daily sync. Nothing here describes how Cipaglucosidase alfa works or whether it is effective, and it is not medical advice.
How this page is built
Everything on this page is copied from public ClinicalTrials.gov registry records — it is not written from memory, and nothing here describes how Cipaglucosidase alfa works or whether it is effective. Each trial links to its own record so you can check it yourself.
- ClinicalTrials.gov (U.S. National Library of Medicine) ↗ Trial IDs, titles, intervention names and synonyms, phase, overall status, lead sponsor and conditions for every trial on this page
This page is built automatically: a daily sync job reads the registry records behind our country trial registers, collects every trial that lists Cipaglucosidase alfa (or a registered synonym) as a drug or biological intervention, and tabulates the registry fields directly. Placebos and generic comparators are excluded. There is no AI drafting and no per-compound editorial pass, and the page has not been clinically reviewed. 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 ClinicalTrials.gov data on this page was last retrieved on 10 Nov 2026. How every register is built: methodology · fixes we have made: corrections log.