This is a Phase 1 study designed to assess the relative bioavailability (BA), safety and tolerability and PK of the pediatric and adult formulations of branaplam.
This study is to compare the pharmacokinetics, safety and tolerability of the pediatric and adult branaplam formulation in healthy adults. The study will also clarify if dosing with food can affect the PK of the adult formulation in order to guide recommendations on dosing relative to meals in subsequent studies. It is two-part, two-period, cross-over study in healthy participants which means that participants will receive both doses and take the treatment with and without food. The total study duration for each participant is expected to be up to approximately 88 days, including the Screening period and safety FU call. Participants will be required to be stay at the site overnight for 6 days during each period to receive dose and have multiple blood draws.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
9
Novartis Investigative Site
Mere Way, Nottingham, United Kingdom
Part 1: Plasma Cmax
Assess relative bioavailability of adult vs pediatric formulation of branaplam under fasting conditions
Time frame: Day 1, 2, 3, 4, 5, 8, 11 and 15
Part 1: Plasma AUClast
Assess relative bioavailability of adult vs pediatric formulation of branaplam under fasting conditions
Time frame: Day 1, 2, 3, 4, 5, 8, 11 and 15 in each period
Part 1: Plasma AUCinf
Assess relative bioavailability of adult vs pediatric formulation of branaplam under fasting conditions
Time frame: Day 1, 2, 3, 4, 5, 8, 11 and 15 in each period
Part 2:Plasma Cmax
Investigate food effect on the pharmacokinetics of the adult formulation of branaplam
Time frame: Day 1, 2, 3, 4, 5, 8, 11 and 15 in each period
Part 2: Plasma AUClast
Investigate food effect on the pharmacokinetics of the adult formulation of branaplam
Time frame: Day1, 2, 3, 4, 5, 8, 11 and 15 in each period
Part 2: Plasma AUCinf
Investigate food effect on the pharmacokinetics of the adult formulation of branaplam
Time frame: Day 1, 2, 3, 4, 5, 8, 11 and 15 in each period
Number of Adverse events and Serious adverse events
Any clinically significant events from other safety assessments will be recorded as adverse events as evaluated by investigator.
Time frame: Day 1 up to 30 days after last drug administration
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Oral solution given in fed state
Parts 1 & 2: Plasma Cmax
Assess the Pharmacokinetic profile of branaplam and its metabolite UFB112
Time frame: Day 1, 2, 3, 4, 5, 8, 11 and 15 in each period
Parts 1 & 2: Plasma AUClast
Assess the Pharmacokinetic profile of branaplam and its metabolite UFB112
Time frame: Day 1, 2, 3, 4, 5, 8, 11 and 15 in each period
Parts 1 &2: Plasma AUCinf
Assess the Pharmacokinetic profile of branaplam and its metabolite UFB112
Time frame: Day 1, 2, 3, 4, 5, 8, 11 and 15 in each period
Parts 1 & 2: Plasma Tmax
Assess the Pharmacokinetic profile of branaplam and its metabolite UFB112
Time frame: Day 1, 2, 3, 4, 5, 8, 11 and 15 in each period
Parts 1 & 2: T1/2
Assess the Pharmacokinetic profile of branaplam and its metabolite UFB112
Time frame: Day 1, 2, 3, 4, 5, 8, 11 and 15 in each period
Parts 1 & 2: Plasma Vz/F
Assess the Pharmacokinetic profile of branaplam and its metabolite UFB112
Time frame: Day 1, 2, 3, 4, 5, 8, 11 and 15 in each period
Parts 1 & 2: Plasma CL/F
Assess the Pharmacokinetic profile of branaplam and its metabolite UFB112
Time frame: Day 1, 2, 3, 4, 5, 8, 11 and 15 in each period