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Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients

NCT04552691 · Status: APPROVED_FOR_MARKETING · Type: EXPANDED_ACCESS

Last updated 2024-07-31

No results posted yet for this study

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

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).

Sponsors & Collaborators

  • Chiesi USA, Inc.

    collaborator INDUSTRY

  • Chiesi Farmaceutici S.p.A.

    lead INDUSTRY

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04552691 on ClinicalTrials.gov