Trials / Approved For Marketing
Approved For MarketingNCT04552691
Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients
Expanded Access Treatment With Open-Label Pegunigalsidase Alfa for Fabry Patients
- Status
- Approved For Marketing
- Phase
- —
- Study type
- Expanded Access
- Enrollment
- —
- Sponsor
- Chiesi Farmaceutici S.p.A. · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- —
Summary
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Pegunigalsidase Alfa | Pegunigalsidase alfa is a recombinant ERT (enzyme replacement therapy) used to treat Fabry disease (dosage: 1 mg/kg body weight every 2 weeks). |
Timeline
- First posted
- 2020-09-17
- Last updated
- 2024-07-31
Locations
11 sites across 1 country: United States
Source: ClinicalTrials.gov record NCT04552691. Inclusion in this directory is not an endorsement.