Brief Title
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.
Study Type
Expanded Access
Condition
Acute Hepatic Porphyria
Intervention
Givosiran
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
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
Gender
All
Ages
12 Years - N/A
Contacts
Medical Director, ,
Administrative Informations
NCT ID
NCT04056481
Organization ID
ALN-AS1-005
Responsible Party
Sponsor
Study Sponsor
Alnylam Pharmaceuticals
Study Sponsor
Medical Director, Study Director, Alnylam Pharmaceuticals
Verification Date
July 2022