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NCT04552691
Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients
trial testing Pegunigalsidase Alfa in Fabry Disease. Approved for marketing.
Quick facts
| Lead sponsor | Chiesi Farmaceutici S.p.A. |
|---|---|
| Status | APPROVED FOR MARKETING |
| Study type | EXPANDED_ACCESS |
| Sites | 11 locations across United States |
Drugs / interventions tested
- Pegunigalsidase Alfa (PEGUNIGALSIDASE ALFA) — full drug profile →
Conditions studied
- Fabry Disease — all drugs for Fabry Disease →
Sponsor
Chiesi Farmaceutici S.p.A. — full company profile →
Who can join
18 and older, any sex, with Fabry Disease. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
The objective of this treatment protocol is to provide guidance to Treating Physicians who seek access to pegunigalsidase alfa for Fabry patients whose clinical condition, in the opinion of the Treating Physician, requires treatment with enzyme replacement therapy (ERT) with pegunigalsidase alfa and a) cannot be adequately treated with currently approved FDA products and/or b) are not able or willing to participate in any of the on-going clinical trials in the United States.
Publications & conference data
2 peer-reviewed publications reference this trial (live from Europe PMC):
-
Anderson-Fabry disease cardiomyopathy: an update on epidemiology, diagnostic approach, management and monitoring strategies.
Averbuch T, White JA, Fine NM. · · 2023 · cited 23× · PMID 37332587 · DOI 10.3389/fcvm.2023.1152568 -
Overcoming Resistance in Anderson-Fabry Disease: Current Therapeutic Challenges and Future Perspectives.
Carella MC, Forleo C, Caretto P, Naccarati ML, et al · · 2024 · cited 5× · PMID 39685654 · DOI 10.3390/jcm13237195
Verify or expand the search:
- PubMed search for NCT04552691
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other recruiting trials for Fabry Disease
Currently open trials in the same condition.
- NCT07187440 — A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease · recruiting
- NCT06776419 — the Role of cArdiac Inflammation, endoThelial Dysfunction, and FIbrosis in fabrY Disease · recruiting
- NCT06539624 — Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease · NA · recruiting
- NCT07277361 — Study of the Quality of Life of Patients With Fabry Disease Aged 65 and Over With and Without Specific Treatment · recruiting
- NCT06270316 — Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease · Phase 1, PHASE2 · recruiting
Other Chiesi Farmaceutici S.p.A. trials
Trials by the same sponsor.
- NCT07516951 — A Study to Find an Efficacious and Safe Dose of CHF10067 (Zampilimab) in Participants With Idiopathic Pulmonary Fibrosis · Phase 2 · not yet recruiting
- NCT07301736 — Therapeutic Equivalence of CHF5993 pMDI 100/6/12.5 µg HFA-152a in Subjects With Mild to Moderate Asthma · Phase 2 · recruiting
- NCT07163182 — Evaluation of Safety, Side Effects and How the Drug CHF6467 Administered Via Intranasal Route is Absorbed, Modified and · Phase 1 · recruiting
- NCT06900816 — A Comparison Study Between Adolescents With Asthma and Adults With Asthma on How They Absorb, Metabolise and Eliminate C · Phase 2 · completed
- NCT06892756 — Effect of Cyclosporine Drug Interaction on the Absorption, Metabolism and Elimination of CHF6001 in Healthy Volunteers. · Phase 1 · 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 NCT04552691 (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 Chiesi Farmaceutici S.p.A.
- Last refreshed: 31 July 2024
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/NCT04552691.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing