A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)

Brief Title

A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)

Official Title

A Phase 1, Single-ascending Dose, Multiple-ascending Dose, and Multi-dose Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN AS1 in Patients With Acute Intermittent Porphyria (AIP)

Brief Summary

      The purpose of this study is to evaluate the safety and tolerability of givosiran (ALN-AS1)
      in AIP patients as well as to characterize pharmacokinetics (PK) and pharmacodynamics (PD) of
      ALN-AS1 in AIP patients.
    


Study Phase

Phase 1

Study Type

Interventional


Primary Outcome

The safety of givosiran evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation

Secondary Outcome

 Profile of Pharmacokinetics (PK) of givosiran

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

40

Start Date

May 6, 2015

Completion Date

September 6, 2017

Primary Completion Date

September 6, 2017

Eligibility Criteria

        Parts A and B

        Inclusion Criteria:

          -  Diagnosis of AIP

          -  Urine PBG at Screening indicating patient is a high excreter

          -  No clinically significant health concerns

          -  Women of child bearing potential must have a negative pregnancy test, not be nursing,
             and use effective contraception

          -  Willing to provide written informed consent and willing to comply with study
             requirements.

        Exclusion Criteria:

          -  Porphyria attack within 6 months of screening

          -  Started a new prescription medication within 3 months of screening

          -  Clinically significant abnormal laboratory results

          -  Received an investigational agent 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

        Part C

        Inclusion Criteria:

          -  Diagnosis of AIP

          -  Patient experienced a porphyria attack or was taking medication to prevent attacks
             recently

          -  No clinically significant health concerns

          -  Women of child bearing potential must have a negative pregnancy test, not be nursing,
             and use effective contraception

          -  Willing to provide written informed consent and willing to comply with study
             requirements.

        Exclusion Criteria:

          -  Stared a new prescription medication within 3 months of screening

          -  Clinically significant abnormal laboratory results

          -  Received an investigational agent 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 - 65 Years

Accepts Healthy Volunteers

No

Contacts

Amy Simon, MD, , 

Location Countries

Sweden

Location Countries

Sweden

Administrative Informations


NCT ID

NCT02452372

Organization ID

ALN-AS1-001


Responsible Party

Sponsor

Study Sponsor

Alnylam Pharmaceuticals


Study Sponsor

Amy Simon, MD, Study Director, Alnylam Pharmaceuticals


Verification Date

June 2018