Status:

APPROVED_FOR_MARKETING

Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria

Lead Sponsor:

Alnylam Pharmaceuticals

Conditions:

Acute Hepatic Porphyria

Eligibility:

All Genders

12+ years

Brief Summary

The purpose of this study is to provide expanded access of givosiran to patients with Acute Hepatic Porphyria (AHP).

Detailed Description

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Eligibility Criteria

Inclusion

  • Diagnosed with Acute Hepatic Porphyria (Acute Intermittent Porphyria, Hereditary Coproporhyria, Variegate Porphyria, ALA dehydratase deficient porphyria)
  • Have adequate venous access for program sample collections as judged by the Investigator for study sample collections

Exclusion

  • Previously or currently participating in a givosiran clinical trial
  • Has any of the following laboratory parameter assessments at Screening:
  • Alanine aminotransferase (ALT) \>2×ULN
  • Total bilirubin \>1.5×ULN. Patients with elevated total bilirubin that is secondary to documented Gilbert's syndrome are eligible if the total bilirubin is \<2×ULN
  • Estimated Glomerular Filtration Rate (eGFR) \<15 mL/min/1.73 m2
  • On an active liver transplantation waiting list

Key Trial Info

Start Date :

Trial Type :

EXPANDED_ACCESS

End Date :

Estimated Enrollment :

Patients enrolled

Trial Details

Trial ID

NCT04056481

Last Update

May 20 2024

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