Clinical Trials Logo
  1. Home
  2. Acute Hepatic Porphyria
  3. NCT04056481
  4. Details
NCT04056481 Clinical Trial

Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria

Acute Hepatic Porphyria Clinical Trial

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

Clinical Trial Details — Status: Approved for marketing

Administrative data
NCT number NCT04056481
Other study ID # ALN-AS1-005
Secondary ID
Status Approved for marketing
Phase
First received
Last updated

Study information
Verified date May 2024
Source Alnylam Pharmaceuticals
Contact n/a
Is FDA regulated No
Health authority
Study type Expanded Access

Clinical Trial Summary

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

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.

Recruitment information / eligibility
Status Approved for marketing
Enrollment 0
Est. completion date
Est. primary completion date
Accepts healthy volunteers
Gender All
Age group 12 Years and older
Eligibility 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

Study Design

Related Conditions & MeSH terms

Intervention

Drug:
• Givosiran
givosiran (ALN-AS1) administered as a subcutaneous (SC) injection

Locations
Country Name City State
n/a

Sponsors (1)
Lead Sponsor Collaborator
Alnylam Pharmaceuticals
See also
  Status Clinical Trial Phase
Completed NCT03338816 - ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP) Phase 3
Terminated NCT03547297 - INSIGHT-AHP: A Study to Characterize the Prevalence of Acute Hepatic Porphyria (AHP) in Patients With Clinical Presentation and History Consistent With AHP
Not yet recruiting NCT04923516 - Prevalence of Acute Hepatic Porphyria
Recruiting NCT05344599 - Evaluating the Prevalence of Acute Hepatic Porphyria in Postural Tachycardia Syndrome
Completed NCT02240784 - EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)
Recruiting NCT04883905 - ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)