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: 

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.

Available
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: 
All
Eligibility Age: 
Minimum: 12 Years
Contacts: 

Alnylam EAP Hotline
1-877-256-9526
eap@alnylam.com

Mary-Jean Fanelli
Study Director
Alnylam Pharmaceuticals

Alnylam Pharmaceuticals
EA Number: 
Keywords: 
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
Disclaimer:
Expanded Access program information for companies listed in the Expanded Access Company Directory is pulled daily from ClinicalTrials.gov, a resource provided by the National Institutes of Health in cooperation with the U.S. Food and Drug Administration. Click here for more information about ClinicalTrials.gov.