Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria

Alnylam Pharmaceuticals

Status

Conditions: Acute Hepatic Porphyria

Treatments: Drug: Givosiran

Study type: Expanded Access

Funder types: Industry

Identifiers

NCT04056481

ALN-AS1-005

Details and patient eligibility

About

The purpose of this study is to provide expanded access of givosiran to patients with Acute Hepatic Porphyria (AHP).

Full 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.

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

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