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

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

Brief Summary

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


Study Phase

Phase 1/Phase 2

Study Type

Interventional


Primary Outcome

The safety of givosiran evaluated by the proportion of patients experiencing adverse events

Secondary Outcome

 The pharmacodynamic (PD) effect of givosiran on urine levels of delta-aminolevulinic acid (ALA)

Condition

Acute Intermittent Porphyria

Intervention

givosiran (ALN-AS1)

Study Arms / Comparison Groups

 givosiran (ALN-AS1)
Description:  

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

17

Start Date

October 2016

Completion Date

October 2021

Primary Completion Date

October 2020

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
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Amy Simon, MD, , 

Location Countries

Sweden

Location Countries

Sweden

Administrative Informations


NCT ID

NCT02949830

Organization ID

ALN-AS1-002


Responsible Party

Sponsor

Study Sponsor

Alnylam Pharmaceuticals


Study Sponsor

Amy Simon, MD, Study Director, Alnylam Pharmaceuticals


Verification Date

February 2020