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SMT C1100 · 2 trials · 2 indications
To determine the plasma concentration of SMT C1100 parent and the major metabolites calculated at each time point for each subject.
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.
| Arm | Type | Description |
|---|---|---|
| Sequence 1 | OTHER | Drug: SMT C1100 or placebo 3-treatment (Period 1,2 and 3) |
| Sequence 2 | OTHER | Drug: SMT C1100 or placebo 3-treatment (Period 1,2, and 3) |
| Sequence 3 | OTHER | Drug: SMT C1100 or placebo 3-treatment (Period 1,2 and 3) |
| 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 |
|---|---|---|
| SMT C1100 | DRUG | Period 1, SMT C1100 1250 mg BID; Period 2, Placebo BID; Period 3, SMT C1100 2500 mg BID |
Inclusion Criteria: 1. Patients will be males of any ethnic origin with a genetic diagnosis of DMD. 2. Children between 5 and 13 years of age. 3. A parent/legal guardian must date and sign a written consent on behalf of the patient, according to International Conference on Harmonisation (ICH) and l...
SMT C1100 is an investigational small molecule being developed for the treatment of Duchenne Muscular Dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
SMT C1100 is being developed by Summit Therapeutics Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol SMMT. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational drug in patients with Duchenne Muscular Dystrophy.
SMT C1100 is in Phase 1 clinical development. Two Phase 1 trials have been completed, both involving pediatric male patients with Duchenne Muscular Dystrophy. The drug remains investigational and has not received FDA approval for any indication.
SMT C1100 has been studied in two completed Phase 1 clinical trials: NCT02056808, a study in subjects with Duchenne Muscular Dystrophy, and NCT02383511, a modified diet trial in pediatric patients with DMD. Both trials were conducted in the United Kingdom and enrolled 12 male participants each.
SMT C1100 is the primary identifier for this investigational compound developed by Summit Therapeutics. No alternative names have been disclosed in the clinical trial records, so it is not known to be marketed under any other brand name.