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SMT C1100

Phase 1

Muscular Dystrophy, Duchenne | Small molecule | Neurology |Summit Therapeutics Inc.|Last Updated: Aug 26, 2015

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment12
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02383511Modified Diet Trial: A Study of SMT C1100 in Paediatric Patients With DMD Who Follow a Balanced DietPHASE1 COMPLETED 12Feb 1, 2015Aug 1, 2015Aug 26, 20154 United Kingdom
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Study Endpoints
Primary Endpoints
Pharmacokinetic parameters at different dose levels of SMT C1100
28 days

To determine the plasma concentration of SMT C1100 parent and the major metabolites calculated at each time point for each subject.

Secondary Endpoints
Safety and tolerability of SMT C1100
28 days
Evaluation of plasma CK levels
42 days
Pharmacokinetic parameters at different dose levels of SMT C1100
28 Days
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelCROSSOVER
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Sequence 1OTHERDrug: SMT C1100 or placebo 3-treatment (Period 1,2 and 3)
Sequence 2OTHERDrug: SMT C1100 or placebo 3-treatment (Period 1,2, and 3)
Sequence 3OTHERDrug: SMT C1100 or placebo 3-treatment (Period 1,2 and 3)
Interventions
NameTypeDescription
SMT C1100DRUGPeriod 1, SMT C1100 1250 mg BID; Period 2, Placebo BID; Period 3, SMT C1100 2500 mg BID
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Eligibility Criteria
Age Range5 Years — 13 Years
SexMALE
Healthy VolunteersNo
Study Sites4

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...

Countries:United Kingdom
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