Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria

N/AApproved For MarketingNCT04056481

Alnylam Pharmaceuticals

Overview

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.

Study Type

EXPANDED_ACCESS

Conditions

Acute Hepatic Porphyria

Interventions

GivosiranDRUG

givosiran (ALN-AS1) administered as a subcutaneous (SC) injection

Eligibility

Sex: ALLMin age: 12 Years

Medical Language ↔ Plain English

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

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.