Official Title
Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria (AHP)
Brief Summary

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

Detailed Description

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Approved for marketing
Treatment IND/Protocol
Acute Hepatic Porphyria

Drug: Givosiran

givosiran (ALN-AS1) administered as a subcutaneous (SC) injection
Other Name: ALN-AS1

Eligibility Criteria

Inclusion Criteria:

- 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 Criteria:

- Previously or currently participating in a givosiran clinical trial

- Has any of the following laboratory parameter assessments at Screening:

1. Alanine aminotransferase (ALT) >2×ULN

2. 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

3. Estimated Glomerular Filtration Rate (eGFR) <15 mL/min/1.73 m2

- On an active liver transplantation waiting list

Eligibility Gender
Eligibility Age
Minimum: 12 Years ~ Maximum: N/A

Medical Director, Study Director
Alnylam Pharmaceuticals

Alnylam Pharmaceuticals
NCT Number
Acute Hepatic Porphyria (AHP)
Acute Intermittent Porphyria (AIP)
Acute Intermittent
Acute Porphyria
Hereditary Coproporphyria (HCP)
Variegate Porphyria (VP)
ALA Dehydratase Deficient Porphyria (ADP)
Expanded Access
MeSH Terms
Porphyrias, Hepatic
Coproporphyria, Hereditary
Porphyria, Erythropoietic