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The purpose of this study is to determine whether increasing doses of SMT C1100 are safe, well tolerated and achieve appropriate blood levels in patients with Duchenne Muscular Dystrophy (DMD).
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| SMT C1100 | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SMT C1100 | Drug | Comparison of safety and pharmacokinetic of different dosages of drug |
|
| Measure | Description | Time Frame |
|---|---|---|
| Safety and tolerability | 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. | After 10 days of treatment phase |
| Measure | Description | Time Frame |
|---|---|---|
| Pharmacokinetic parameters at different dose levels | 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. | After single oral dose and after 10 days of treatment phase |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| 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 | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Heart of England NHS Foundation Trust - Heart Lands Hospital | Birmingham | B9 5SS | United Kingdom | |||
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| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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| ID | Term |
|---|---|
| C000610115 | SMT C1100 |
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| Alder Hey Children's NHS Foundation Trust |
| Liverpool |
| L12 2AP |
| United Kingdom |
| 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 |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |