A Study to Investigate the Interaction Between Givosiran and a 5-probe Drug Cocktail in Patients With Acute Intermittent Porphyria (AIP)
Phase 1
Completed
- Conditions
- Acute Intermittent Porphyria (AIP)Acute PorphyriaAcute Hepatic Porphyria (AHP)Porphyria, Acute Intermittent
- Interventions
- Drug: 5-probe cocktail
- Registration Number
- NCT03505853
- Lead Sponsor
- Alnylam Pharmaceuticals
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 10
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Givosiran with 5-probe cocktail Givosiran - Givosiran with 5-probe cocktail 5-probe cocktail -
- Primary Outcome Measures
Name Time Method Profile of Pharmacokinetics (PK) of Cytochrome P450 (CYP) probe cocktail Days 1 and 36 Maximum plasma concentration (Cmax)
- Secondary Outcome Measures
Name Time Method Profile of Pharmacokinetics (PK) of Cytochrome P450 (CYP) probe cocktail metabolites Days 1 and 36 Maximum plasma concentration (Cmax)
Safety as evaluated by the proportion of subjects experiencing adverse events (AEs) Day 1 - Day 92 Profile of Pharmacokinetics (PK) of Cytochrome P450 (CYP) probe cocktail Days 1 and 36 Volume of distribution (V/F)
The pharmacodynamic (PD) effect of givosiran on urine levels of delta-aminolevulinic acid (ALA) Days 1, 8, and 36 The pharmacodynamic (PD) effect of givosiran on urine levels of Porphobilinogen (PBG) in patients with AIP Days 1, 8, and 36
Trial Locations
- Locations (1)
Clinical Trial Site
🇸🇪Stockholm, Sweden