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 with Acute Hepatic Porphyria (AHP).

Detailed Description

Choosing to participate in an expanded access program is an important personal decision. Talk

with your doctor and family members or friends about deciding to join a research study. To

learn more about this study, please have your doctor contact the study research staff using

the Contacts provided. For general information, see the link provided in More Information.

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

Eligibility Gender
Eligibility Age
Minimum: 12 Years

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