Alnylam Pharmaceuticals

Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria

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: 

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EA Type
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)

- Experienced a porphyria attack and be considered by their treating physician to be at risk for having an attack in the future.

- 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) <30 mL/min/1.73 m2

- On an active liver transplantation waiting list

Eligibility Gender: 
Eligibility Age: 
Minimum: 12 Years
Belgium, France, Germany, Netherlands, Spain

Alnylam EAP Hotline

Mary-Jean Fanelli
Study Director
Alnylam Pharmaceuticals

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