Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria
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The purpose of this study is to provide expanded access of givosiran to patients with Acute Hepatic Porphyria (AHP).
Condition or disease
Acute Hepatic Porphyria
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.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Layout table for eligibility information
Ages Eligible for Study:
12 Years and older (Child, Adult, Older Adult)
Sexes Eligible for Study:
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
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