Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria

NCT04056481 | Unknown

• 1 other identifier: ALN-AS1-005
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

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

Conditions

Acute Hepatic Porphyria

Keywords

Acute Hepatic Porphyria (AHP); Acute Intermittent Porphyria (AIP); Porphyria; Acute Intermittent; Acute Porphyria; Hereditary Coproporphyria (HCP); Variegate Porphyria (VP); ALA Dehydratase Deficient Porphyria (ADP); Givosiran; Expanded Access; EAP

Interventions

Givosiran DRUG

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

Also known as: ALN-AS1

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Alnylam Pharmaceuticals: lead

MeSH Terms

Conditions

Coproporphyria, Hereditary; Porphyria, Acute Intermittent; Porphyrias; Porphyria, Variegate

Interventions

givosiran

Condition Hierarchy (Ancestors)

Porphyrias, Hepatic; Liver Diseases; Digestive System Diseases; Skin Diseases, Genetic; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Medical Director

  Alnylam Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 12, 2019
• First Posted: August 14, 2019
• Last Updated: May 20, 2024

Record last verified: 2024-05