A Study to Investigate the Interaction Between Givosiran and a 5-probe Drug Cocktail in Patients With Acute Intermittent Porphyria (AIP)

Alnylam Pharmaceuticals

Status and phase: Completed; Phase 1

Conditions: Porphyria, Acute Intermittent; Acute Porphyria; Acute Intermittent Porphyria (AIP); Acute Hepatic Porphyria (AHP)

Treatments: Drug: Givosiran; Drug: 5-probe cocktail

Study type: Interventional

Funder types: Industry

Identifiers

NCT03505853

ALN-AS1-004

Details and patient eligibility

About

The purpose of this study is to evaluate the effect of givosiran on the pharmacokinetics of the 5-probe cocktail of midazolam, caffeine, losartan, omeprazole, and dextromethorphan, and their metabolites, in asymptomatic patients with Acute Intermittent Porphyria.

Enrollment: 10 patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 18 to 65 years old
• Weight between 45kg and 110kg, inclusive, if male
• Weight between 45kg and 100 kg, inclusive, if female
• Diagnosis of acute intermittent porphyria (AIP)
• Elevated urine PBG level
• Not be pregnant or breast feeding, and must be willing to use a highly effective method of contraception

Exclusion criteria

• Clinically significant abnormal laboratory results
• Experienced an acute porphyria attack within past 12 months
• History of multiple drug allergies, intolerances or sensitivities
• History of recurrent pancreatitis
• Received an experimental drug, within 3 months of dosing
• Donated or lost an excessive amount of blood within 60 days of dosing

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 1 patient group

Givosiran with 5-probe cocktail

Experimental group

Treatment:

Drug: Givosiran

Drug: 5-probe cocktail