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

Sponsor

Alnylam Pharmaceuticals (Industry)

Overall Status

Completed

CT.gov ID

NCT02949830

Collaborator

(none)

Enrollment

Locations

Arm

61.1

Actual Duration (Months)

3.2

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

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

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

Study Design

Study Type:

Interventional

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 :

Oct 1, 2016

Actual Primary Completion Date :

Nov 5, 2021

Actual Study Completion Date :

Nov 5, 2021

Arms and Interventions

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

Arm

Intervention/Treatment

Experimental: givosiran (ALN-AS1)

Drug: givosiran (ALN-AS1)

givosiran by subcutaneous (SC) injection

Outcome Measures

Primary Outcome Measures

The safety of givosiran evaluated by the proportion of patients experiencing adverse events [Through Month 49]

Secondary Outcome Measures

The pharmacodynamic (PD) effect of givosiran on urine levels of delta-aminolevulinic acid (ALA) [Through Month 49]
The pharmacodynamic (PD) effect of givosiran on urine levels of Porphobilinogen (PBG) [Through Month 49]
The frequency of porphyria attacks as measured by number of attacks [Through Month 49]
Frequency of hematin administrations [Through Month 49]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country
1	Clinical Trial Site	San Francisco	California	United States
2	Clinical Trial Site	New York	New York	United States
3	Clinical Trial Site	Galveston	Texas	United States
4	Clinical Trial Site	Stockholm		Sweden
5	Clinical Trial Site	London		United Kingdom