ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)



Status:Active, not recruiting
Conditions:Gastrointestinal
Therapuetic Areas:Gastroenterology
Healthy:No
Age Range:12 - Any
Updated:3/14/2019
Start Date:November 1, 2017
End Date:September 2021

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ENVISION: A Phase 3 Randomized, Double-blind, Placebo-Controlled Multicenter Study With an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients With Acute Hepatic Porphyrias

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).


Inclusion Criteria:

- ≥ 12 years of age

- Diagnosed with Acute Hepatic Porphyria (Acute Intermittent Porphyria, Hereditary
Corproporhyria, Variegate Porphyria, ALA dehydratase deficient porphyria)

- Elevated urinary or plasma 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 Criteria:

- 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
We found this trial at
12
sites
Miami, Florida
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Miami, FL
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Ann Arbor, Michigan 48109
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Ann Arbor, MI
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Boston, Massachusetts 02114
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Boston, MA
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Galveston, Texas
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Galveston, TX
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Little Rock, Arkansas
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Little Rock, AR
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New York, New York 10032
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New York, NY
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Parkville, Victoria
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Parkville,
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Philadelphia, Pennsylvania 19104
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Philadelphia, PA
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Salt Lake City, Utah
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Salt Lake City, UT
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San Francisco, California
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San Francisco, CA
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Seattle, Washington
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Seattle, WA
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1901 South Hawthorne Road
Winston-Salem, North Carolina 27103
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Winston-Salem, NC
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