Status:
COMPLETED
ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)
Lead Sponsor:
Alnylam Pharmaceuticals
Conditions:
Acute Hepatic Porphyria
Acute Intermittent Porphyria
Eligibility:
All Genders
12+ years
Phase:
PHASE3
Brief Summary
The purpose of this study is to evaluate the effect of subcutaneous givosiran (ALN-AS1), compared to placebo, on the rate of porphyria attacks in patients with Acute Hepatic Porphyrias (AHP).
Eligibility Criteria
Inclusion
- ≥ 12 years of age
- Diagnosed with Acute Hepatic Porphyria (Acute Intermittent Porphyria, Hereditary Corproporhyria, Variegate Porphyria, aminolevulinic acid (ALA) dehydratase deficient porphyria)
- Elevated urinary or plasma porphobilinogen (PBG) or ALA values within the past year,
- Have active disease, with at least 2 documented porphyria attacks within the last 6 months
- Willing to discontinue or not initiate the use of prophylactic hemin throughout the study.
- Women of child bearing potential must have a negative serum pregnancy test, not be nursing, and use acceptable contraception
Exclusion
- Clinically significant abnormal laboratory results
- Anticipated liver transplantation
- History of multiple drug allergies or intolerance to subcutaneous injections
- Active HIV, hepatitis C virus, or hepatitis B virus infection(s)
- History of recurrent pancreatitis
Key Trial Info
Start Date :
November 16 2017
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 31 2021
Estimated Enrollment :
94 Patients enrolled
Trial Details
Trial ID
NCT03338816
Start Date
November 16 2017
End Date
May 31 2021
Last Update
April 22 2024
Active Locations (36)
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1
Clinical Trial Site
Little Rock, Arkansas, United States, 72205
2
Clinical Trial Site
San Francisco, California, United States, 94143
3
Clinical Trial Site
Boston, Massachusetts, United States, 02114
4
Clinical Trial Site
Ann Arbor, Michigan, United States, 48109