Back to resultsA Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)
Primary Purpose
Acute Intermittent Porphyria
Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
givosiran (ALN-AS1)
Sponsored by
About this trial
This is an interventional treatment trial for Acute Intermittent Porphyria focused on measuring RNAi therapeutic, Porphyria, AIP, Heme/Haem Arginate
Eligibility Criteria
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
Sites / Locations
- Clinical Trial Site
- Clinical Trial Site
- Clinical Trial Site
- Clinical Trial Site
- Clinical Trial Site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
givosiran (ALN-AS1)
Arm Description
Outcomes
Primary Outcome Measures
The safety of givosiran evaluated by the proportion of patients experiencing adverse events
Secondary Outcome Measures
The pharmacodynamic (PD) effect of givosiran on urine levels of delta-aminolevulinic acid (ALA)
The pharmacodynamic (PD) effect of givosiran on urine levels of Porphobilinogen (PBG)
The frequency of porphyria attacks as measured by number of attacks
Frequency of hematin administrations
Full Information
NCT ID
NCT02949830
First Posted
October 28, 2016
Last Updated
November 9, 2021
Sponsor
Alnylam Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT02949830
Brief Title
A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)
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
Study Type
Interventional
2. Study Status
Record Verification Date
November 2021
Overall Recruitment Status
Completed
Study Start Date
October 2016 (Actual)
Primary Completion Date
November 5, 2021 (Actual)
Study Completion Date
November 5, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alnylam Pharmaceuticals
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
The purpose of this study is to determine the long-term safety, tolerability and pharmacokinetics of givosiran (ALN-AS1) in AIP patients
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Acute Intermittent Porphyria
Keywords
RNAi therapeutic, Porphyria, AIP, Heme/Haem Arginate
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
16 (Actual)
8. Arms, Groups, and Interventions
Arm Title
givosiran (ALN-AS1)
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
givosiran (ALN-AS1)
Intervention Description
givosiran by subcutaneous (SC) injection
Primary Outcome Measure Information:
Title
The safety of givosiran evaluated by the proportion of patients experiencing adverse events
Time Frame
Through Month 49
Secondary Outcome Measure Information:
Title
The pharmacodynamic (PD) effect of givosiran on urine levels of delta-aminolevulinic acid (ALA)
Time Frame
Through Month 49
Title
The pharmacodynamic (PD) effect of givosiran on urine levels of Porphobilinogen (PBG)
Time Frame
Through Month 49
Title
The frequency of porphyria attacks as measured by number of attacks
Time Frame
Through Month 49
Title
Frequency of hematin administrations
Time Frame
Through Month 49
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Alnylam Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Clinical Trial Site
City
San Francisco
State/Province
California
Country
United States
Facility Name
Clinical Trial Site
City
New York
State/Province
New York
Country
United States
Facility Name
Clinical Trial Site
City
Galveston
State/Province
Texas
Country
United States
Facility Name
Clinical Trial Site
City
Stockholm
Country
Sweden
Facility Name
Clinical Trial Site
City
London
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
Undecided
Learn more about this trial
A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)
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