Trial · NCT02858362
Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrophy (DMD)
NCT02858362, Phase 2, Terminated, Summit Therapeutics; results first posted 2020-01-02
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrophy (DMD). Trials, record NCT02858362. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02858362
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrophy (DMD)
- NCT number
- NCT02858362
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 43 participants, actual
- Lead sponsor
- Summit Therapeutics
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Ezutromid
- First posted
- 2016-08-08
- Last updated
- 2020-01-02
- Other registries' IDs
- eudract:2015-004333-27
- Results first posted
- 2020-01-02
- Limitations and caveats
- Analysis of the data collected from Cohort 3 was limited as the study was terminated prior to the first participant's Week 24 visit.
Source ClinicalTrials.gov
Registered outcomes25
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in Magnetic Resonance Spectroscopy (MRS) Fat Fraction (FF) for Leg Muscles | Baseline, Week 12, Week 24, Week 36 and Week 48 | MRS FF was analysed for vastus lateralis and soleus leg muscles. The endpoints were measured for Cohorts 1 and 2 only. Results for Cohorts 1 and 2 are pooled as specified in the protocol. |
| Primary | Change From Baseline for Magnetic Resonance Spectroscopy (MRS) Water Transverse Relaxation Time (WTRT) for Leg Muscles | Baseline, Week 12, Week 24, Week 36 and Week 48 | MRS WTRT was analysed for the vastus lateralis and soleus leg muscles. The endpoints were measured for Cohorts 1 and 2 only. Results for Cohorts 1 and 2 are pooled as specified in the protocol. |
| Primary | Observed Trough Plasma Concentration (Ctrough) for SMT C1100, Dihydrodiol 1 (DHD1) and Dihydrodiol III (DHD 3) | Pre-dose at Weeks 1, 4, 8, 12, 24, 36 and 48 | Pharmacokinetic analysis is presented by cohort due to the use of different formulations. The median pre-dose concentration was derived for each participant and then summarized across participants. |
| Primary | Simulated Maximum Plasma Concentration (Cmax) for SMT C1100, Dihydrodiol 1 (DHD1) and Dihydrodiol III (DHD 3) | Pre-dose and 3 to 10 hours post-dose at Weeks 1, 4, 8, 12, 24, 36 and 48 | Pharmacokinetic analysis is presented by cohort due to the use of different formulations. |
| Primary | Simulated Average Plasma Concentration (Cav) for SMT C1100, Dihydrodiol 1 (DHD1) and Dihydrodiol III (DHD 3) | Pre-dose and 3 to 10 hours post-dose at Weeks 1, 4, 8, 12, 24, 36 and 48 | Pharmacokinetic analysis is presented by cohort due to the use of different formulations. |
| Primary | Number of Participants Reporting One or More Treatment-Emergent Adverse Events (TEAEs) | Day 1 to a maximum of Week 96 | An adverse event is defined as any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. A TEAE is defined as any event not present before exposure to study drug or any event already present that worsens in either intensity or frequency after exposure to study drug. |
| Secondary | Change From Baseline in Utrophin Intensity | Baseline, Week 24 and Week 48 | A maximum of two muscle biopsies were taken, one at baseline and the other at Week 24 or Week 48. Utrophin intensity was analyzed using a semiautomated quantitative assay on the biopsy samples. A positive change from baseline represents an increase in utrophin expression, no change from baseline represents maintenance of utrophin expression and a negative change from baseline represents a reduction in utrophin expression. The endpoint was measured for Cohorts 1 and 2 only. Results for Cohorts 1 and 2 are pooled as specified in the protocol. |
| Secondary | Change From Baseline in Developmental Heavy Chain Myosin (MHCd) Expression | Baseline, Week 24 and Week 48 | A maximum of two muscle biopsies were taken, one at baseline and the other at Week 24 or Week 48. MHCd expression was analyzed using a semiautomated quantitative assay on the biopsy samples. A positive change from baseline represents an increase in MHCd expression, no change from baseline represents maintenance of MHCd expression and a negative change from baseline represents a reduction in MHCd expression. The endpoint was measured for Cohorts 1 and 2 only. Results for Cohorts 1 and 2 are pooled as specified in the protocol. |
| Secondary | Change From Baseline in Muscle Fibre Diameter | Baseline, Week 24 and Week 48 | A maximum of two muscle biopsies were taken, one at baseline and the other at Week 24 or Week 48. Muscle fibre diameter was analyzed using a semiautomated quantitative assay on the biopsy samples. The endpoint was measured for Cohorts 1 and 2 only. Results for Cohorts 1 and 2 are pooled as specified in the protocol. |
| Secondary | Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) | Baseline, Week 12, Week 24, Week 36 and Week 48 | Results for Cohorts 1, 2 and 3 are pooled as specified in the protocol. |
1 to 10 of 25Source ClinicalTrials.gov
See all 25 registered outcomesOutcomes25
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in Magnetic Resonance Spectroscopy (MRS) Fat Fraction (FF) for Leg Muscles | Baseline, Week 12, Week 24, Week 36 and Week 48 | All participants who received at least one dose of study medication and had at least one assessment of the endpoint (which could be their baseline assessment). | Percentage of fat in the muscle | MEAN |
| PRIMARY | Change From Baseline for Magnetic Resonance Spectroscopy (MRS) Water Transverse Relaxation Time (WTRT) for Leg Muscles | Baseline, Week 12, Week 24, Week 36 and Week 48 | All participants who received at least one dose of study medication and had at least one assessment of the endpoint (which could be their baseline assessment). | Milliseconds | MEAN |
| PRIMARY | Observed Trough Plasma Concentration (Ctrough) for SMT C1100, Dihydrodiol 1 (DHD1) and Dihydrodiol III (DHD 3) | Pre-dose at Weeks 1, 4, 8, 12, 24, 36 and 48 | All participants in Cohorts 1 or 2 who received at least one dose of study medication and had at least one concentration measurement (which could be below the limit of quantification). No evaluable data was collected from Cohort 3 participants due to early study termination. | ng/mL | GEOMETRIC_MEAN |
| PRIMARY | Simulated Maximum Plasma Concentration (Cmax) for SMT C1100, Dihydrodiol 1 (DHD1) and Dihydrodiol III (DHD 3) | Pre-dose and 3 to 10 hours post-dose at Weeks 1, 4, 8, 12, 24, 36 and 48 | All participants in Cohorts 1 or 2 who received at least one dose of study medication and had at least one concentration measurement (which could be below the limit of quantification). No evaluable data was collected from Cohort 3 participants due to early study termination. | ng/mL | GEOMETRIC_MEAN |
| PRIMARY | Simulated Average Plasma Concentration (Cav) for SMT C1100, Dihydrodiol 1 (DHD1) and Dihydrodiol III (DHD 3) | Pre-dose and 3 to 10 hours post-dose at Weeks 1, 4, 8, 12, 24, 36 and 48 | All participants in Cohorts 1 or 2 who received at least one dose of study medication and had at least one concentration measurement (which could be below the limit of quantification). No evaluable data was collected from Cohort 3 participants due to early study termination. | ng/mL | GEOMETRIC_MEAN |
| PRIMARY | Number of Participants Reporting One or More Treatment-Emergent Adverse Events (TEAEs) | Day 1 to a maximum of Week 96 | All participants who received at least one dose of study medication. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Utrophin Intensity | Baseline, Week 24 and Week 48 | All participants who received at least one dose of study medication and had at least one assessment of the endpoint (which could be their baseline assessment). For the summaries of changes from baseline only those participants in this population who had measurements at both baseline and the relevant post-baseline visit were included. | Arbitrary units | MEAN |
| SECONDARY | Change From Baseline in Developmental Heavy Chain Myosin (MHCd) Expression | Baseline, Week 24 and Week 48 | All participants who received at least one dose of study medication and had at least one assessment of the endpoint (which could be their baseline assessment). For the summaries of changes from baseline only those participants in this population who had measurements at both baseline and the relevant post-baseline visit were included. | Percent of muscle fibres expressing MHCd | MEAN |
| SECONDARY | Change From Baseline in Muscle Fibre Diameter | Baseline, Week 24 and Week 48 | All participants who received at least one dose of study medication and had at least one assessment of the endpoint (which could be their baseline assessment). For the summaries of changes from baseline only those participants in this population who had measurements at both baseline and the relevant post-baseline visit were included. | Micrometers (μm) | MEAN |
| SECONDARY | Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) | Baseline, Week 12, Week 24, Week 36 and Week 48 | All participants who received at least one dose of study medication. No evaluable data was collected from Cohort 3 participants due to early study termination. | Percentage of FEV1 | MEAN |
1 to 10 of 25Source ClinicalTrials.gov
See all 25 outcomesResults by group126
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Magnetic Resonance Spectroscopy (MRS) Fat Fraction (FF) for Leg Muscles | Cohorts 1 and 2: SMT C1100 | Vastus Lateralis: Week 12 Change from Baseline | 1.779 | 95% Confidence Interval 0.939 to 2.620 | Percentage of fat in the muscle |
| Change From Baseline in Magnetic Resonance Spectroscopy (MRS) Fat Fraction (FF) for Leg Muscles | Cohorts 1 and 2: SMT C1100 | Vastus Lateralis: Week 24 Change from Baseline | 3.914 | 95% Confidence Interval 2.695 to 5.132 | Percentage of fat in the muscle |
| Change From Baseline in Magnetic Resonance Spectroscopy (MRS) Fat Fraction (FF) for Leg Muscles | Cohorts 1 and 2: SMT C1100 | Vastus Lateralis: Week 36 Change from Baseline | 5.238 | 95% Confidence Interval 3.563 to 6.913 | Percentage of fat in the muscle |
| Change From Baseline in Magnetic Resonance Spectroscopy (MRS) Fat Fraction (FF) for Leg Muscles | Cohorts 1 and 2: SMT C1100 | Vastus Lateralis: Week 48 Change from Baseline | 7.142 | 95% Confidence Interval 4.866 to 9.417 | Percentage of fat in the muscle |
| Change From Baseline in Magnetic Resonance Spectroscopy (MRS) Fat Fraction (FF) for Leg Muscles | Cohorts 1 and 2: SMT C1100 | Soleus: Week 12 Change from Baseline | 0.615 | 95% Confidence Interval 0.068 to 1.162 | Percentage of fat in the muscle |
| Change From Baseline in Magnetic Resonance Spectroscopy (MRS) Fat Fraction (FF) for Leg Muscles | Cohorts 1 and 2: SMT C1100 | Soleus: Week 24 Change from Baseline | 1.108 | 95% Confidence Interval 0.350 to 1.865 | Percentage of fat in the muscle |
| Change From Baseline in Magnetic Resonance Spectroscopy (MRS) Fat Fraction (FF) for Leg Muscles | Cohorts 1 and 2: SMT C1100 | Soleus: Week 36 Change from Baseline | 2.384 | 95% Confidence Interval 1.287 to 3.481 | Percentage of fat in the muscle |
| Change From Baseline in Magnetic Resonance Spectroscopy (MRS) Fat Fraction (FF) for Leg Muscles | Cohorts 1 and 2: SMT C1100 | Soleus: Week 48 Change from Baseline | 2.584 | 95% Confidence Interval 1.258 to 3.909 | Percentage of fat in the muscle |
| Change From Baseline for Magnetic Resonance Spectroscopy (MRS) Water Transverse Relaxation Time (WTRT) for Leg Muscles | Cohorts 1 and 2: SMT C1100 | Vastus Lateralis: Week 12 Change from Baseline | -0.559 | 95% Confidence Interval -1.190 to 0.072 | Milliseconds |
| Change From Baseline for Magnetic Resonance Spectroscopy (MRS) Water Transverse Relaxation Time (WTRT) for Leg Muscles | Cohorts 1 and 2: SMT C1100 | Vastus Lateralis: Week 24 Change from Baseline | -0.486 | 95% Confidence Interval -1.193 to 0.221 | Milliseconds |
1 to 10 of 126Source ClinicalTrials.gov
See all 126 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort 1: SMT C1100 Formulation 1 | 30 | |
| Overall Study | STARTED | Cohort 2: SMT C1100 Formulation 2 | 10 | |
| Overall Study | STARTED | Cohort 3: SMT C1100 Formulation 1 | 3 | |
| Overall Study | Did Not Enter Extension Phase | Cohort 1: SMT C1100 Formulation 1 | 1 | |
| Overall Study | Did Not Enter Extension Phase | Cohort 2: SMT C1100 Formulation 2 | 2 | |
| Overall Study | Did Not Enter Extension Phase | Cohort 3: SMT C1100 Formulation 1 | 0 | |
| Overall Study | COMPLETED | Cohort 1: SMT C1100 Formulation 1 | 28 | |
| Overall Study | COMPLETED | Cohort 2: SMT C1100 Formulation 2 | 9 | |
| Overall Study | COMPLETED | Cohort 3: SMT C1100 Formulation 1 | 0 | |
| Overall Study | NOT COMPLETED | Cohort 1: SMT C1100 Formulation 1 | 2 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events252
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Faeces pale | Gastrointestinal disorders | Cohort 1: SMT C1100 Formulation 1 | 18 | 30 | |
| other | Faeces pale | Gastrointestinal disorders | Cohort 2: SMT C1100 Formulation 2 | 3 | 10 | |
| other | Faeces pale | Gastrointestinal disorders | Cohort 3: SMT C1100 Formulation 1 | 3 | 3 | |
| other | Vomiting | Gastrointestinal disorders | Cohort 1: SMT C1100 Formulation 1 | 17 | 30 | |
| other | Vomiting | Gastrointestinal disorders | Cohort 2: SMT C1100 Formulation 2 | 6 | 10 | |
| other | Vomiting | Gastrointestinal disorders | Cohort 3: SMT C1100 Formulation 1 | 0 | 3 | |
| other | Diarrhoea | Gastrointestinal disorders | Cohort 1: SMT C1100 Formulation 1 | 12 | 30 | |
| other | Diarrhoea | Gastrointestinal disorders | Cohort 2: SMT C1100 Formulation 2 | 4 | 10 | |
| other | Diarrhoea | Gastrointestinal disorders | Cohort 3: SMT C1100 Formulation 1 | 2 | 3 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Cohort 1: SMT C1100 Formulation 1 | 8 | 30 |
1 to 10 of 252Source ClinicalTrials.gov
See all 252 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1: SMT C1100 Formulation 1 | 0 | 30 |
| Total, all-cause mortality | Cohort 2: SMT C1100 Formulation 2 | 0 | 10 |
| Total, all-cause mortality | Cohort 3: SMT C1100 Formulation 1 | 0 | 3 |
| Total, other adverse events | Cohort 1: SMT C1100 Formulation 1 | 30 | 30 |
| Total, other adverse events | Cohort 2: SMT C1100 Formulation 2 | 10 | 10 |
| Total, other adverse events | Cohort 3: SMT C1100 Formulation 1 | 3 | 3 |
| Total, serious adverse events | Cohort 1: SMT C1100 Formulation 1 | 4 | 30 |
| Total, serious adverse events | Cohort 2: SMT C1100 Formulation 2 | 0 | 10 |
| Total, serious adverse events | Cohort 3: SMT C1100 Formulation 1 | 0 | 3 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02858362
GET https://conductscience.org/api/trials/v1/NCT02858362/registered?page=2