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

Alnylam Pharmaceuticals

Status and phase: Completed; Phase 2; Phase 1

Conditions: Acute Intermittent Porphyria

Treatments: Drug: Givosiran

Study type: Interventional

Funder types: Industry

Identifiers

NCT02949830

ALN-AS1-002

2016-002638-54 (EudraCT Number)

Details and patient eligibility

About

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).

Enrollment: 16 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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16 participants in 1 patient group

Givosiran

Experimental group

Description:

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.

Treatment:

Drug: Givosiran