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

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ClinicalTrials.gov Identifier: NCT03338816
Recruitment Status : Recruiting
First Posted : November 9, 2017
Last Update Posted : June 19, 2018
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

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).

Condition or disease Intervention/treatment Phase
Acute Hepatic Porphyria Acute Intermittent Porphyria Porphyria, Acute Intermittent Acute Porphyria Hereditary Coproporphyria (HCP) Variegate Porphyria (VP) ALA Dehydratase Deficient Porphyria (ADP) Drug: Givosiran Drug: Placebo Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 74 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : November 1, 2017
Estimated Primary Completion Date : March 2019
Estimated Study Completion Date : September 2021


Arm Intervention/treatment
Experimental: Givosiran Drug: Givosiran
by subcutaneous (SC) injection

Placebo Comparator: Placebo
Normal Saline (0.9% NaCl)
Drug: Placebo
Sterile Normal Saline (0.9% NaCl)




Primary Outcome Measures :
  1. The annualized rate of porphyria attacks in patients with AIP. [ Time Frame: at 6 months ]

Secondary Outcome Measures :
  1. The pharmacodynamic (PD) effect of Givosiran on urine levels of delta-aminolevulinic acid (ALA) in patients with AIP. [ Time Frame: at 3 and 6 months ]
  2. The pharmacodynamic (PD) effect of Givosiran on urine levels of Porphobilinogen (PBG) in patients with AIP. [ Time Frame: at 6 months ]
  3. Annualized rate of hemin administrations in AIP patients. [ Time Frame: through month 6 ]
  4. Annualized rate of porphyria attacks in patients with AHP. [ Time Frame: through month 6 ]
  5. Pain as measured by the Brief Pain Inventory-Short Form numeric rating scale, a 0 to 10 point scale with 10 rated as worst pain. [ Time Frame: through month 6 ]
  6. Nausea as measured by the numeric rating scale, a 0 to 10 point scale with 10 rated as worst nausea. [ Time Frame: through month 6 ]
  7. Fatigue as measured by the Brief Fatigue Inventory-Short Form numeric rating scale, a 0 to 10 point scale with 10 rated as worst fatigue. [ Time Frame: through month 6 ]
  8. Change from baseline in the Physical Component Summary of the 12-Item Short Form Survey (SF-12). [ Time Frame: through month 6 ]


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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03338816


Contacts
Contact: Alnylam Clinical Trial Information Line 1-877-ALNYLAM clinicaltrials@alnylam.com
Contact: Alnylam Clinical Trial Information Line 1-877-256-9526

  Show 40 Study Locations
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
Study Director: Amy Simon, MD Alnylam Pharmaceuticals

Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03338816     History of Changes
Other Study ID Numbers: ALN-AS1-003
First Posted: November 9, 2017    Key Record Dates
Last Update Posted: June 19, 2018
Last Verified: June 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Alnylam Pharmaceuticals:
Acute Intermittent Porphyria (AIP)
Acute Hepatic Porphyria (AHP)
Hereditary Coproporphyria (HCP)
Variegate Porphyria (VP)
ALA dehydratase deficient porphyria (ADP) (ALAD)
RNAi therapeutic
Porphyria

Additional relevant MeSH terms:
Porphyrias
Porphyria, Erythropoietic
Porphyria, Acute Intermittent
Porphyrias, Hepatic
Coproporphyria, Hereditary
Porphyria, Variegate
Metabolic Diseases
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Liver Diseases
Digestive System Diseases