Placebo-controlled, multi-centre, randomized, double-blind dose escalation study. The aim is to evaluate the pharmacokinetics (PK) and safety of SMT C1100 in paediatric patients with Duchenne Muscular Dystrophy (DMD) who follow a balanced diet.
Primary Objective: To determine the plasma concentration of SMT C1100 calculated at each time point for each subject (sample size (n), mean, standard deviation (SD), percentage of coefficient of variation (%CV), geometric mean, median, minimum, and maximum for the parent and the major metabolites). Secondary Objectives: 1. To determine the safety and tolerability of single and multiple oral doses of SMT C1100 in patients with Duchenne Muscular Dystrophy (DMD) by assessing the participants adverse events, ECG results, vital signs and laboratory tests. 2. To evaluate the diurnal variability in the steady state PK of SMT C1100 calculated at each time point for each subject (sample size (n), mean, standard deviation (SD), percentage of coefficient of variation (%CV), geometric mean, median, minimum, and maximum for the parent and the major metabolites). 3. To evaluate reductions in creatine phosphokinase as a potential pharmacodynamic (PD) marker of SMT C1100 activity and clinical benefit.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
12
Period 1, SMT C1100 1250 mg BID; Period 2, Placebo BID; Period 3, SMT C1100 2500 mg BID
Period 1, SMT C1100 1250 mg BID; Period 2, SMT C1100 2500 mg BID; Period 3, Placebo BID
Period 1, Placebo BID; Period 2, SMT C1100 1250 mg BID; Period 3, SMT C1100 2500 mg BID
Heart of England NHS Foundation Trust - Heart Lands Hospital
Birmingham, United Kingdom
Alder Hey Children's NHS Foundation Trust
Liverpool, United Kingdom
Great Ormond Street Hospital for Children NHS Foundation Trust
London, United Kingdom
Central Manchester University Hospitals NHS Foundation Trust- Royal Manchester Children's Hospital
Manchester, United Kingdom
Pharmacokinetic parameters at different dose levels of SMT C1100
To determine the plasma concentration of SMT C1100 parent and the major metabolites calculated at each time point for each subject.
Time frame: 28 days
Safety and tolerability of SMT C1100
To determine the safety and tolerability of single and multiple oral doses of SMT C1100 in patients with Duchenne Muscular Dystrophy (DMD) by assessing the participants adverse events.
Time frame: 28 days
Evaluation of plasma CK levels
To evaluate reductions in plasma creatine phosphokinase as a potential pharmacodynamic (PD) marker of SMT C1100 activity and muscle benefit.
Time frame: 42 days
Pharmacokinetic parameters at different dose levels of SMT C1100
To determine the plasma concentration of SMT C1100 major metabolites calculated at each time point for each subject.
Time frame: 28 Days
Safety and tolerability of SMT C1100
To determine the safety and tolerability of single and multiple oral doses of SMT C1100 in patients with Duchenne Muscular Dystrophy (DMD) composite assessment of the participant's ECG results and laboratory tests.
Time frame: 28 Days
Pharmacokinetic parameters at different dose levels of SMT C1100
To evaluate the diurnal variability in the steady state PK of SMT C1100 calculated at each time point for each subject.
Time frame: 28 Days
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