A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD)
|ClinicalTrials.gov Identifier: NCT02056808|
Recruitment Status : Completed
First Posted : February 6, 2014
Last Update Posted : August 27, 2014
|Condition or disease||Intervention/treatment||Phase|
|Duchenne Muscular Dystrophy||Drug: SMT C1100||Phase 1|
Primary Objective: To determine the safety and tolerability of single and multiple oral doses of SMT C1100 in patients with Duchenne Muscular Dystrophy (DMD).
Secondary Objectives: To determine the single and multiple oral dose pharmacokinetics of SMT C1100 and its metabolites in patients with DMD.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||12 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||SMT C1100 - A Phase 1b, Open-label, Single and Multiple Oral Dose, Safety, Tolerability and Pharmacokinetic Study in Paediatric Patients With Duchenne Muscular Dystrophy|
|Study Start Date :||November 2013|
|Actual Primary Completion Date :||May 2014|
|Actual Study Completion Date :||July 2014|
Experimental: SMT C1100
Patients will be studied in 3 groups (Groups A to C), with each group consisting of 4 patients aged between 5 to 11 years. It is planned that doses for Groups A to C will be administered in an escalating manner after safety review for each dose group.
Drug: SMT C1100
Comparison of safety and pharmacokinetic of different dosages of drug
- Safety and tolerability [ Time Frame: After 10 days of treatment phase ]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.
- Pharmacokinetic parameters at different dose levels [ Time Frame: After single oral dose and after 10 days of treatment phase ]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.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02056808
|Heart of England NHS Foundation Trust - Heart Lands Hospital|
|Birmingham, United Kingdom, B9 5SS|
|Alder Hey Children's NHS Foundation Trust|
|Liverpool, United Kingdom, L12 2AP|
|Great Ormond Street for Children NHS Foundation Trust|
|London, United Kingdom|
|Central Manchester University Hospitals NHS Foundation Trust- Royal Manchester Children's Hospital|
|Manchester, United Kingdom|
|Principal Investigator:||Stefan Spinty, MD||Alder Hey Children's NHS Foundation Trust|
|Principal Investigator:||Helen Roper, MD||Heart of England NHS Foundation Trust - Heartlands Hospital|
|Principal Investigator:||Imelda Hughes, MD||Central Manchester University Hospitals NHS Foundation Trust - Royal Manchester Childrens Hospital|
|Principal Investigator:||Franceso Muntoni, MD||Great Ormond Street Hospital for Children NHS Foundation Trust|