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A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02949830
Recruitment Status : Completed
First Posted : October 31, 2016
Last Update Posted : November 17, 2021
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Brief Summary:
The purpose of this study is to determine the long-term safety, tolerability and pharmacokinetics of givosiran (ALN-AS1) in AIP patients

Condition or disease Intervention/treatment Phase
Acute Intermittent Porphyria Drug: givosiran (ALN-AS1) Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 16 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter, Open-label Extension Study to Evaluate the Long-term Safety and Clinical Activity of Subcutaneously Administered ALN-AS1 in Patients With Acute Intermittent Porphyria Who Have Completed a Previous Clinical Study With ALN-AS1
Actual Study Start Date : October 2016
Actual Primary Completion Date : November 5, 2021
Actual Study Completion Date : November 5, 2021

Arm Intervention/treatment
Experimental: givosiran (ALN-AS1) Drug: givosiran (ALN-AS1)
givosiran by subcutaneous (SC) injection

Primary Outcome Measures :
  1. The safety of givosiran evaluated by the proportion of patients experiencing adverse events [ Time Frame: Through Month 49 ]

Secondary Outcome Measures :
  1. The pharmacodynamic (PD) effect of givosiran on urine levels of delta-aminolevulinic acid (ALA) [ Time Frame: Through Month 49 ]
  2. The pharmacodynamic (PD) effect of givosiran on urine levels of Porphobilinogen (PBG) [ Time Frame: Through Month 49 ]
  3. The frequency of porphyria attacks as measured by number of attacks [ Time Frame: Through Month 49 ]
  4. Frequency of hematin administrations [ Time Frame: Through Month 49 ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Completed participation in Part C of study ALN-AS1-001
  • Not on a scheduled regimen of hemin
  • Women of child bearing potential must have a negative serum pregnancy test, not be nursing, and use acceptable contraception
  • Willing and able to comply with the study requirements and to provide written informed consent

Exclusion Criteria:

  • Clinically significant abnormal laboratory results
  • Received an investigational agent (other than ALN-AS1) within 90 days before the first dose of study drug or are in follow-up of another clinical study
  • History of multiple drug allergies or intolerance to subcutaneous injection

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): NCT02949830

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United States, California
Clinical Trial Site
San Francisco, California, United States
United States, New York
Clinical Trial Site
New York, New York, United States
United States, Texas
Clinical Trial Site
Galveston, Texas, United States
Clinical Trial Site
Stockholm, Sweden
United Kingdom
Clinical Trial Site
London, United Kingdom
Sponsors and Collaborators
Alnylam Pharmaceuticals
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Study Director: Medical Director Alnylam Pharmaceuticals
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Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02949830    
Other Study ID Numbers: ALN-AS1-002
First Posted: October 31, 2016    Key Record Dates
Last Update Posted: November 17, 2021
Last Verified: November 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Keywords provided by Alnylam Pharmaceuticals:
RNAi therapeutic
Heme/Haem Arginate
Additional relevant MeSH terms:
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Porphyria, Acute Intermittent
Porphyria, Erythropoietic
Metabolic Diseases
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Porphyrias, Hepatic
Liver Diseases
Digestive System Diseases