Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria
- Conditions
- Acute Hepatic Porphyria
- Registration Number
- NCT04056481
- Lead Sponsor
- Alnylam Pharmaceuticals
- 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.
Recruitment & Eligibility
- Status
- APPROVED_FOR_MARKETING
- Sex
- All
- Target Recruitment
- Not specified
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:
- Alanine aminotransferase (ALT) >2×ULN
- 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
- Estimated Glomerular Filtration Rate (eGFR) <15 mL/min/1.73 m2
-
On an active liver transplantation waiting list
Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method