- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02383511
Modified Diet Trial: A Study of SMT C1100 in Paediatric Patients With DMD Who Follow a Balanced Diet
August 25, 2015 updated by: Summit Therapeutics
A Phase 1b Placebo-controlled, Multi-centre, Randomized, Double-blind Dose Escalation Study to Evaluate the Pharmacokinetics (PK) and Safety of SMT C1100 in Patients With Duchenne Muscular Dystrophy (DMD) Who Follow a Balanced Diet
Placebo-controlled, multi-centre, randomized, double-blind dose escalation study.
The aim is to evaluate the pharmacokinetics (PK) and safety of SMT C1100 in paediatric patients with Duchenne Muscular Dystrophy (DMD) who follow a balanced diet.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Detailed Description
Primary Objective:
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:
- 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.
- 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).
- To evaluate reductions in creatine phosphokinase as a potential pharmacodynamic (PD) marker of SMT C1100 activity and clinical benefit.
Study Type
Interventional
Enrollment (Actual)
12
Phase
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
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Birmingham, United Kingdom, B9 5SS
- Heart of England NHS Foundation Trust - Heart Lands Hospital
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Liverpool, United Kingdom, L12 2AP
- Alder Hey Children's NHS Foundation Trust
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London, United Kingdom, WC1N 3JH
- Great Ormond Street Hospital For Children NHS Foundation Trust
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Manchester, United Kingdom
- Central Manchester University Hospitals NHS Foundation Trust- Royal Manchester Children's Hospital
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-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
5 years to 13 years (Child)
Accepts Healthy Volunteers
No
Genders Eligible for Study
Male
Description
Inclusion Criteria:
- Patients will be males of any ethnic origin with a genetic diagnosis of DMD.
- Children between 5 and 13 years of age.
- 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.
- The patient is willing to give verbal or written age appropriate assent to participate.
- 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.
- 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.
- 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.
- 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:
- 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.
- Known hypersensitivity to the excipients of the study drug or a previous history of drug allergy.
- 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.
- 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.
- Is unable to refrain from eating cruciferous vegetables and barbecued (chargrilled) meat for the duration of the study.
- Use of prohibited medication within 5 half-lives prior to baseline assessments, unless otherwise stated in protocol.
- Need for mechanical ventilation.
- The patient experiences intermittent or continuous difficulties in swallowing.
- Non ambulatory.
- Any clinically significant acute illness within 4 weeks of the start of dose administration.
- Any comorbidity that, in the opinion of the Investigator, increases the risk of participating in the study.
- Symptomatic cardiomyopathy that in the opinion of the Investigator prohibits participation in this study.
- 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.
- 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).
- 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.
- Excessive exercise (Investigator opinion).
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Crossover Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Other: Sequence 1
Drug: SMT C1100 or placebo 3-treatment (Period 1,2 and 3)
|
Period 1, SMT C1100 1250 mg BID; Period 2, Placebo BID; Period 3, SMT C1100 2500 mg BID
Period 1, SMT C1100 1250 mg BID; Period 2, SMT C1100 2500 mg BID; Period 3, Placebo BID
Period 1, Placebo BID; Period 2, SMT C1100 1250 mg BID; Period 3, SMT C1100 2500 mg BID
|
Other: Sequence 2
Drug: SMT C1100 or placebo 3-treatment (Period 1,2, and 3)
|
Period 1, SMT C1100 1250 mg BID; Period 2, Placebo BID; Period 3, SMT C1100 2500 mg BID
Period 1, SMT C1100 1250 mg BID; Period 2, SMT C1100 2500 mg BID; Period 3, Placebo BID
Period 1, Placebo BID; Period 2, SMT C1100 1250 mg BID; Period 3, SMT C1100 2500 mg BID
|
Other: Sequence 3
Drug: SMT C1100 or placebo 3-treatment (Period 1,2 and 3)
|
Period 1, SMT C1100 1250 mg BID; Period 2, Placebo BID; Period 3, SMT C1100 2500 mg BID
Period 1, SMT C1100 1250 mg BID; Period 2, SMT C1100 2500 mg BID; Period 3, Placebo BID
Period 1, Placebo BID; Period 2, SMT C1100 1250 mg BID; Period 3, SMT C1100 2500 mg BID
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Pharmacokinetic parameters at different dose levels of SMT C1100
Time Frame: 28 days
|
To determine the plasma concentration of SMT C1100 parent and the major metabolites calculated at each time point for each subject.
|
28 days
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Safety and tolerability of SMT C1100
Time Frame: 28 days
|
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.
|
28 days
|
Evaluation of plasma CK levels
Time Frame: 42 days
|
To evaluate reductions in plasma creatine phosphokinase as a potential pharmacodynamic (PD) marker of SMT C1100 activity and muscle benefit.
|
42 days
|
Pharmacokinetic parameters at different dose levels of SMT C1100
Time Frame: 28 Days
|
To determine the plasma concentration of SMT C1100 major metabolites calculated at each time point for each subject.
|
28 Days
|
Safety and tolerability of SMT C1100
Time Frame: 28 Days
|
To determine the safety and tolerability of single and multiple oral doses of SMT C1100 in patients with Duchenne Muscular Dystrophy (DMD) composite assessment of the participant's ECG results and laboratory tests.
|
28 Days
|
Pharmacokinetic parameters at different dose levels of SMT C1100
Time Frame: 28 Days
|
To evaluate the diurnal variability in the steady state PK of SMT C1100 calculated at each time point for each subject.
|
28 Days
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
February 1, 2015
Primary Completion (Actual)
August 1, 2015
Study Completion (Actual)
August 1, 2015
Study Registration Dates
First Submitted
February 4, 2015
First Submitted That Met QC Criteria
March 3, 2015
First Posted (Estimate)
March 9, 2015
Study Record Updates
Last Update Posted (Estimate)
August 26, 2015
Last Update Submitted That Met QC Criteria
August 25, 2015
Last Verified
August 1, 2015
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- SMT C11003
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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