The purpose of this study is to provide expanded access of givosiran to patients with Acute Hepatic Porphyria (AHP).
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.
Drug: Givosiran
givosiran (ALN-AS1) administered as a subcutaneous (SC) injection
Other Name: ALN-AS1
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
Medical Director, Study Director
Alnylam Pharmaceuticals