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Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria

Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria (AHP)

Status
APPROVED_FOR_MARKETING
Phases
Unknown
Study type
Expanded Access
Source
ClinicalTrials.gov
Registry ID
NCT04056481
Enrollment
Unknown
Registered
2019-08-14
Start date
Unknown
Completion date
Unknown
Last updated
2024-05-20

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

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

Brief summary

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

Detailed 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.

Interventions

DRUGGivosiran

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

Sponsors

Alnylam Pharmaceuticals
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
ALL
Age
12 Years to No maximum

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

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026