A clinical trial to test if the drug SMT C1100 is safe and well absorbed compared to placebo when given to children with Duchenne Muscular Dystrophy (DMD), who follow a balanced diet.

• Register: EUCTR
• Last refreshed on: 30 June 2019
• Main ID: EUCTR2014-003100-78-GB
• Date of registration: 19/09/2014
• Prospective Registration: Yes
• Primary sponsor: Summit (Oxford) Limited
• Public title: A clinical trial to test if the drug SMT C1100 is safe and well absorbed compared to placebo when given to children with Duchenne Muscular Dystrophy (DMD), who follow a balanced diet.
• Scientific title: SMT C11003 - A Phase 1b placebo-controlled, multi-centre, randomized, double-blind 3-period dose escalation study to evaluate the pharmacokinetics (PK) and safety of SMT C1100 in paediatric patients with Duchenne Muscular Dystrophy (DMD) who follow a balanced diet.
• Date of first enrolment: 01/12/2014
• Target sample size: 12
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2014-003100-78
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: yes; Open: no; Single blind: no; Double blind: yes; Parallel group: no; Cross over: yes; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: yes; Other: no; Number of treatment arms in the trial: 3
• Phase: Human pharmacology (Phase I): yes Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

United Kingdom

Contacts

• Name: Clinical Trial Information
• Affiliation: Summit (Oxford) Limited

• Name: Clinical Trial Information
• Affiliation: Summit (Oxford) Limited

Key inclusion & exclusion criteria

Inclusion criteria:
Patients will be required to satisfy the following criteria at the screening visit (and at the beginning of each new Treatment Period) unless otherwise stated:

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 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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 12
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
Patients will be excluded from the study if they satisfy the following criteria at the screening visit unless otherwise stated:

1. Enrolment or participation in any therapeutic clinical trial within the prior 3 months or 5 times the half-life (whichever is longer). Prior exposure to SMT C1100 is NOT an exclusion criterion.

2. Known hypersensitivity to the excipients of the study drug (i.e. Poloxamer 188 [Lutrol F68], Methyl paraben, Propyl paraben, Hydroxypropylmethyl cellulose [Pharmacoat 645], Glycerol, Non crystallizing sorbitol [70%] [Neosorb 70/70B],
Xanthan gum, Strawberry cream flavour [PHS-132963]) 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 Section 6.2.1.3.

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).

Following pre-dose assessments, patients may be excluded from the study for the following reasons:

• Clinically significant vital signs or 12-lead ECG findings
• Intercurrent illness or clinically significant adverse events since Screening
• Deviation from study restrictions (see Section 6.2) will not be allowed except in prior agreement with Sponsor. Agreement may be given if in the opinion of the investigator and Sponsor these deviations will not interfere with

Age minimum:
Age maximum:
Gender:
Female: no
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Duchenne Muscular Dystrophy
MedDRA version: 17.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders

Intervention(s)

Product Code: SMT C1100
Pharmaceutical Form: Oral suspension
INN or Proposed INN: Not available
CAS Number: 945531-77-1
Current Sponsor code: SMT C1100
Other descriptive name: SMT C1100
Concentration unit: mg/g milligram(s)/gram
Concentration type: equal
Concentration number: 200-
Pharmaceutical form of the placebo: Oral suspension
Route of administration of the placebo: Oral use

Primary Outcome(s)

Primary end point(s): Pharmacokinetic assessments

Secondary Objective: (1) To determine the safety and tolerability of single and multiple oral doses of SMT C1100 and its metabolites in patients with DMD who follow a balanced diet.

(2) To evaluate the diurnal variability in the steady state PK of SMT C1100.

(3) To evaluate reductions in creatine phosphokinase as a potential pharmacodynamic (PD) marker of SMT C1100 activity and clinical benefit.

Exploratory Objective
To quantify putative biomarkers of disease activity and dietary metabolism from blood and urine

Main Objective: To determine the single and multiple oral dose PK of SMT C1100 and its metabolites in patients with DMD who follow a balanced diet

Timepoint(s) of evaluation of this end point: Day 1, day 14

Secondary Outcome(s)

Secondary end point(s): 1. safety and tolerability assessments
2. CPK levels

Timepoint(s) of evaluation of this end point: 1. Throughout the study
2. Screening, days 1, 7, 14 and follow-up

Secondary ID(s)

SMT_C11003

Source(s) of Monetary Support

Summit (Oxford) Limited

Ethics review

Status: Approved
Approval date:
Contact:

Results

• Results available: Yes
• Date Posted: 17/06/2016
• Date Completed: 06/07/2015
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2014-003100-78/results