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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

16 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-10-31

Study Completion Date

2021-11-05

Brief Summary

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The purpose of this study is to determine the long-term safety, tolerability and pharmacokinetics of givosiran (ALN-AS1) in AIP patients who completed study ALN-AS1-001 (NCT02452372).

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Detailed Description

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Conditions

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Acute Intermittent Porphyria

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Givosiran

At the beginning of this study, participants received either givosiran 2.5 mg/kg subcutaneous (SC) injection once monthly(QM), givosiran 5.0 mg/kg SC injection QM, or givosiran 5.0 mg/kg SC injection once every 3 months (Q3M). Within a year, all participants were transitioned to givosiran 2.5 mg/kg SC injection QM.

Group Type EXPERIMENTAL

Givosiran

Intervention Type DRUG

Givosiran by subcutaneous (SC) injection.

Interventions

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Givosiran

Givosiran by subcutaneous (SC) injection.

Intervention Type DRUG

Other Intervention Names

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ALN-AS1 Givlaari

Eligibility Criteria

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Inclusion Criteria

* Completed participation in Part C of study ALN-AS1-001 (NCT02452372)
* 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

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No