Muscular Dystrophy, DuchennePhase 1NCT02383511
Modified Diet Trial: A Study of SMT C1100 in Paediatric Patients With DMD Who Follow a Balanced Diet
A Phase 1 study testing SMT C1100 in people with Muscular Dystrophy, Duchenne, run by Summit Therapeutics at 4 sites in United Kingdom.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 5 to 13 years, men only
- Conditions the study is for
- Muscular Dystrophy, Duchenne
- What is being tested?
- SMT C1100
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 4 sitesUnited Kingdom
- Study dates
- Feb 2015to Aug 2015
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 13 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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).
Trial locations and contacts
4 sites in 1 country.
United Kingdom4 sites
Heart of England NHS Foundation Trust - Heart Lands Hospital
Birmingham, B9 5SS
Alder Hey Children's NHS Foundation Trust
Liverpool, L12 2AP
Great Ormond Street Hospital for Children NHS Foundation Trust
London, WC1N 3JH
Central Manchester University Hospitals NHS Foundation Trust- Royal Manchester Children's Hospital
Manchester
About this study
Official title: 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.
Read the 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.
- Conditions
- Muscular Dystrophy, Duchenne
- Being tested
- SMT C1100
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SMT C1100Drug
Period 1, SMT C1100 1250 mg BID; Period 2, Placebo BID; Period 3, SMT C1100 2500 mg BID
SMT C1100Drug
Period 1, SMT C1100 1250 mg BID; Period 2, SMT C1100 2500 mg BID; Period 3, Placebo BID
SMT C1100Drug
Period 1, Placebo BID; Period 2, SMT C1100 1250 mg BID; Period 3, SMT C1100 2500 mg BID
| Study group | Role | What they receive |
|---|---|---|
| Sequence 1 | Other | Drug: SMT C1100 or placebo 3-treatment (Period 1,2 and 3) Receives: Drug: SMT C1100 |
| Sequence 2 | Other | Drug: SMT C1100 or placebo 3-treatment (Period 1,2, and 3) Receives: Drug: SMT C1100 |
| Sequence 3 | Other | Drug: SMT C1100 or placebo 3-treatment (Period 1,2 and 3) Receives: Drug: SMT C1100 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pharmacokinetic parameters at different dose levels of SMT C1100
To determine the plasma concentration of SMT C1100 parent and the major metabolites calculated at each time point for each subject.
Measured: 28 days
Secondary outcomes (5)
Safety and tolerability of SMT C1100
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.
Measured: 28 days
Evaluation of plasma CK levels
To evaluate reductions in plasma creatine phosphokinase as a potential pharmacodynamic (PD) marker of SMT C1100 activity and muscle benefit.
Measured: 42 days
Pharmacokinetic parameters at different dose levels of SMT C1100
To determine the plasma concentration of SMT C1100 major metabolites calculated at each time point for each subject.
Measured: 28 Days
Safety and tolerability of SMT C1100
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.
Measured: 28 Days
Pharmacokinetic parameters at different dose levels of SMT C1100
To evaluate the diurnal variability in the steady state PK of SMT C1100 calculated at each time point for each subject.
Measured: 28 Days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Mar 2015 (estimated)
- Study startFeb 2015
- Primary completionAug 2015
- Study completionAug 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Muscular Dystrophy, DuchenneStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Summit Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02383511
- Sponsor's protocol ID
- SMT C11003
- Status last verified
- Aug 2015
- First submitted
- 4 Feb 2015
- Last updated
- 26 Aug 2015
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 27055247
Study at a glance
From the registry record, last updated by the sponsor on 26 Aug 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT02383511
- Acronym / sponsor ID
- SMT C11003
- Conditions
- Muscular Dystrophy, Duchenne
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Summit TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Feb 2015
- Primary completion
- Aug 2015 (actual)
- Study completion
- Aug 2015 (actual)
- First posted
- 9 Mar 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Sequence 1 | Other | Drug: SMT C1100Drug: SMT C1100 or placebo 3-treatment (Period 1,2 and 3) |
| Sequence 2 | Other | Drug: SMT C1100Drug: SMT C1100 or placebo 3-treatment (Period 1,2, and 3) |
| Sequence 3 | Other | Drug: SMT C1100Drug: SMT C1100 or placebo 3-treatment (Period 1,2 and 3) |
Who may qualify
- Ages
- 5 to 13 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (16 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SMT C1100Period 1, SMT C1100 1250 mg BID; Period 2, Placebo BID; Period 3, SMT C1100 2500 mg BID | Drug | Sequence 1 |
| SMT C1100Period 1, SMT C1100 1250 mg BID; Period 2, SMT C1100 2500 mg BID; Period 3, Placebo BID | Drug | Sequence 2 |
| SMT C1100Period 1, Placebo BID; Period 2, SMT C1100 1250 mg BID; Period 3, SMT C1100 2500 mg BID | Drug | Sequence 3 |
Primary outcome
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.
5 secondary outcomes
Safety and tolerability of SMT C1100
Time frame: 28 days
Evaluation of plasma CK levels
Time frame: 42 days
Pharmacokinetic parameters at different dose levels of SMT C1100
Time frame: 28 Days
Safety and tolerability of SMT C1100
Time frame: 28 Days
Pharmacokinetic parameters at different dose levels of SMT C1100
Time frame: 28 Days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Aug 2015.
- Derived from this study: PubMed 27055247Ricotti 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing SMT C1100 in people with Muscular Dystrophy, Duchenne, run by Summit Therapeutics at 4 sites in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Aug 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Pharmacokinetic parameters at different dose levels of SMT C1100 (time frame: 28 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02383511, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Aug 2015.