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Also known as Ezutromid
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 Duchenne Muscular Dystrophy, a neuromuscular condition in the neurology therapeutic area. It has been studied in Phase 1 clinical trials in male patients aged 5 years and older. It is not approved for any indication.
SMT C1100 targets utrophin, a protein. Utrophin is a structural relative of dystrophin, the protein missing in Duchenne Muscular Dystrophy, and the drug is designed to modulate utrophin as a potential therapeutic approach in this disease.
SMT C1100 is developed by Summit Therapeutics Inc., which trades under the ticker SMMT. The company has sponsored the clinical studies of the compound in Duchenne Muscular Dystrophy.
SMT C1100 is in Phase 1 development. It is an investigational drug and has not been approved by the FDA. Two Phase 1 trials have been completed, and no trials are currently active.
SMT C1100 has been evaluated in two completed Phase 1 trials: NCT02383511, a modified diet study in pediatric DMD patients, and NCT02056808, a Phase 1b study in subjects with Duchenne Muscular Dystrophy. Both enrolled 12 participants in the United Kingdom, for a total of 24.
Both SMT C1100 trials were Phase 1 studies conducted in the United Kingdom in male participants aged 5 years and older with Duchenne Muscular Dystrophy. The trials were randomized, double blind, and controlled, and each enrolled 12 subjects. Neither trial used biomarker selection.