Modified Diet Trial: A Study of SMT C1100 in Paediatric Patients With DMD Who Follow a Balanced Diet
NCT ID: NCT02383511
Last Updated: 2015-08-26
Study Results
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Basic Information
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COMPLETED
PHASE1
12 participants
INTERVENTIONAL
2015-02-28
2015-08-31
Brief Summary
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Detailed Description
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To determine the 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).
Secondary Objectives:
1. 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.
2. To evaluate the diurnal variability in the steady state PK 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).
3. To evaluate reductions in creatine phosphokinase as a potential pharmacodynamic (PD) marker of SMT C1100 activity and clinical benefit.
Conditions
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Study Design
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RANDOMIZED
CROSSOVER
TREATMENT
QUADRUPLE
Study Groups
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Sequence 1
Drug: SMT C1100 or placebo 3-treatment (Period 1,2 and 3)
SMT C1100
Period 1, SMT C1100 1250 mg BID; Period 2, Placebo BID; Period 3, SMT C1100 2500 mg BID
Sequence 2
Drug: SMT C1100 or placebo 3-treatment (Period 1,2, and 3)
SMT C1100
Period 1, SMT C1100 1250 mg BID; Period 2, SMT C1100 2500 mg BID; Period 3, Placebo BID
Sequence 3
Drug: SMT C1100 or placebo 3-treatment (Period 1,2 and 3)
SMT C1100
Period 1, Placebo BID; Period 2, SMT C1100 1250 mg BID; Period 3, SMT C1100 2500 mg BID
Interventions
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SMT C1100
Period 1, SMT C1100 1250 mg BID; Period 2, Placebo BID; Period 3, SMT C1100 2500 mg BID
SMT C1100
Period 1, SMT C1100 1250 mg BID; Period 2, SMT C1100 2500 mg BID; Period 3, Placebo BID
SMT C1100
Period 1, Placebo BID; Period 2, SMT C1100 1250 mg BID; Period 3, SMT C1100 2500 mg BID
Eligibility Criteria
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Inclusion Criteria
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 local regulations. This person must understand the contents of the consent, requirements of the study and have had an opportunity to review questions with a medically trained member of the site study team.
4. The patient is willing to give verbal or written age appropriate assent to participate.
5. For safety reasons, the patient's parent/legal guardian must have a good understanding of the English language, which the consent/assent forms are available, and understand the requirements for reporting of any AE to the Investigator.
6. The patient has 6 months or more stable systemic (Patients using an intermittent regimen of steroid are allowed to be enrolled) corticosteroid therapy prior to Screening. Dose modifications for body weight are permitted.
7. The patient or parent is willing to adhere to a balanced diet from 1 week prior to dosing until the end of the follow-up period.
8. Patients must agree to not have sexual intercourse during the study treatment phases and until the end of their participation in the study.
Exclusion Criteria
2. Known hypersensitivity to the excipients of the study drug or a previous history of drug allergy.
3. The patient or parent is unwilling to adhere to a balanced diet from 1 week prior to dosing until the end of the follow-up period.
4. Is dairy or lactose intolerant, has an allergy to egg or nuts or any other dietary restrictions that might interfere with the conduct of the study.
5. Is unable to refrain from eating cruciferous vegetables and barbecued (chargrilled) meat for the duration of the study.
6. Use of prohibited medication within 5 half-lives prior to baseline assessments, unless otherwise stated in protocol.
7. Need for mechanical ventilation.
8. The patient experiences intermittent or continuous difficulties in swallowing.
9. Non ambulatory.
10. Any clinically significant acute illness within 4 weeks of the start of dose administration.
11. Any comorbidity that, in the opinion of the Investigator, increases the risk of participating in the study.
12. Symptomatic cardiomyopathy that in the opinion of the Investigator prohibits participation in this study.
13. Abnormality in the 12-lead ECG at the Screening visit that, in the opinion of the Investigator, increases the risk of participating in the study.
14. Any clinically significant medical condition, other than DMD that in the opinion of the Investigator may increase the risk of participating in the study or interfere with the interpretation of safety or efficacy evaluations (e.g., concomitant illness, severe reflux, psychiatric condition or behavioural disorder).
15. The Patient smokes or has exposure to daily passive smoking (including parent/legal guardian, siblings) so as to minimise environmental factors causing CYP 1A induction.
16. Excessive exercise (Investigator opinion).
5 Years
13 Years
MALE
No
Sponsors
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Summit Therapeutics
INDUSTRY
Responsible Party
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Locations
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Heart of England NHS Foundation Trust - Heart Lands Hospital
Birmingham, , United Kingdom
Alder Hey Children's NHS Foundation Trust
Liverpool, , United Kingdom
Great Ormond Street Hospital for Children NHS Foundation Trust
London, , United Kingdom
Central Manchester University Hospitals NHS Foundation Trust- Royal Manchester Children's Hospital
Manchester, , United Kingdom
Countries
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References
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Ricotti V, Spinty S, Roper H, Hughes I, Tejura B, Robinson N, Layton G, Davies K, Muntoni F, Tinsley J. Safety, Tolerability, and Pharmacokinetics of SMT C1100, a 2-Arylbenzoxazole Utrophin Modulator, following Single- and Multiple-Dose Administration to Pediatric Patients with Duchenne Muscular Dystrophy. PLoS One. 2016 Apr 7;11(4):e0152840. doi: 10.1371/journal.pone.0152840. eCollection 2016.
Other Identifiers
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SMT C11003
Identifier Type: -
Identifier Source: org_study_id
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