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

Brief Title

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

Official Title

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

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).
    


Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Annualized Rate of Porphyria Attacks in Participants With Acute Intermittent Porphyria (AIP)

Secondary Outcome

 The Pharmacodynamic (PD) Effect of Givosiran on Urine Levels of Delta-aminolevulinic Acid (ALA) in Participants With AIP

Condition

Acute Hepatic Porphyria

Intervention

Givosiran

Study Arms / Comparison Groups

 Givosiran/Givosiran
Description:  Givosiran 2.5 mg/kg administered subcutaneously (SC), monthly (QM), for 6 months during the 6-Month Double-blind (DB) Period, followed by givosiran 2.5 mg/kg or 1.25 mg/kg SC, QM for 29 months during the Open-label Extension (OLE) Period.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

94

Start Date

November 16, 2017

Completion Date

May 2021

Primary Completion Date

January 31, 2019

Eligibility Criteria

        Inclusion Criteria:

          -  ≥ 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 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
      

Gender

All

Ages

12 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Medical Director, , 

Location Countries

Australia

Location Countries

Australia

Administrative Informations


NCT ID

NCT03338816

Organization ID

ALN-AS1-003


Responsible Party

Sponsor

Study Sponsor

Alnylam Pharmaceuticals


Study Sponsor

Medical Director, Study Director, Alnylam Pharmaceuticals


Verification Date

May 2021