Duchenne Muscular DystrophyPhase 1NCT02056808
A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD)
A Phase 1 study testing SMT C1100 in people with Duchenne Muscular Dystrophy, run by Summit Therapeutics at 4 sites in United Kingdom.
Trial summary
The purpose of this study is to determine whether increasing doses of SMT C1100 are safe, well tolerated and achieve appropriate blood levels in patients with Duchenne Muscular Dystrophy (DMD). Read more about this study
- Who is it for?
- Ages 5 to 11 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- 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
- Nov 2013to Jul 2014
- 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 11 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 11 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, as this is the only language the consent/assent forms are written in, and understand the requirements for reporting of any adverse event to the Investigator.
Who cannot join
- Enrollment or participation in any therapeutic clinical trial within the prior 3 months or 5 times the half-life (whichever is longer).
- Initiation or change (other than dose modifications for body weight) of systemic corticosteroid therapy within 2 months prior to the start of dose administration or discontinuation of corticosteroids within 30 days prior to the start of dose administration.
- Known hypersensitivity to the excipients of the study drug or a previous history of drug allergy.
- Use of the following therapies is prohibited during the study and for at least 5 half-lives prior to the start of dose administration: Inducers of cytochrome P450 CYP1A2 (eg, carbamazepine, phenytoin, primidone, rifampin, omeprazole, and barbiturates), and moderate and strong inhibitors of CYP1A2 (e.g., fluvoxamine, ciprofloxacin, enoxacin, mexiletine; propafenone, zileuton). Substrates of CYP1A2 with narrow therapeutic windows (e.g., tacrine, theophylline, methadone, mexiletine). Nicotine, including exposure to daily passive smoking to minimize cytochrome P450 CYP 1A induction. Chargrilled food, cruciferous vegetables, caffeine, tea, and any xanthine containing foods, and drinks are prohibited from 36 hours prior to check-in until final discharge from study. Herbal supplements and homeopathic preparations (unless approved by medical monitor).
- Need for mechanical ventilation.
- Non ambulatory.
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- Any clinically significant acute illness within 4 weeks of the start of dose administration.
- Any co-morbidity 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 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, psychiatric condition or behavioral disorder).
- Exposure to daily passive smoking (including parent/legal guardian, siblings) so as to minimize environmental factors causing cytochrome P450 CYP 1A induction. For information SMT C1100 is metabolized by cytochrome P450 CYP 1A.
- 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 for Children NHS Foundation Trust
London
Central Manchester University Hospitals NHS Foundation Trust- Royal Manchester Children's Hospital
Manchester
About this study
Official title: SMT C1100 - A Phase 1b, Open-label, Single and Multiple Oral Dose, Safety, Tolerability and Pharmacokinetic Study in Paediatric Patients With Duchenne Muscular Dystrophy
The purpose of this study is to determine whether increasing doses of SMT C1100 are safe, well tolerated and achieve appropriate blood levels in patients with Duchenne Muscular Dystrophy (DMD).
Read the detailed description
Primary Objective: To determine the safety and tolerability of single and multiple oral doses of SMT C1100 in patients with Duchenne Muscular Dystrophy (DMD).
Secondary Objectives: To determine the single and multiple oral dose pharmacokinetics of SMT C1100 and its metabolites in patients with DMD.
- Conditions
- Duchenne Muscular Dystrophy
- 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
Comparison of safety and pharmacokinetic of different dosages of drug
| Study group | Role | What they receive |
|---|---|---|
| SMT C1100 | Experimental | Patients will be studied in 3 groups (Groups A to C), with each group consisting of 4 patients aged between 5 to 11 years. It is planned that doses for Groups A to C will be administered in an escalating manner after safety review for each dose group. Receives: Drug: SMT C1100 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability
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.
Measured: After 10 days of treatment phase
Secondary outcomes (1)
Pharmacokinetic parameters at different dose levels
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.
Measured: After single oral dose and after 10 days of treatment phase
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Feb 2014 (estimated)
- Study startNov 2013
- Primary completionMay 2014
- Study completionJul 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Summit Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Stefan Spinty, MD, Principal investigator, Alder Hey Children's NHS Foundation TrustHelen Roper, MD, Principal investigator, Heart of England NHS Foundation Trust - Heartlands HospitalImelda Hughes, MD, Principal investigator, Central Manchester University Hospitals NHS Foundation Trust - Royal Manchester Childrens HospitalFranceso Muntoni, MD, Principal investigator, Great Ormond Street Hospital for Children NHS Foundation Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02056808
- Sponsor's protocol ID
- SMT C11002
- Status last verified
- Aug 2014
- First submitted
- 21 Nov 2013
- Last updated
- 27 Aug 2014
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 27 Aug 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- SMT C1100 - A Phase 1b, Open-label, Single and Multiple Oral Dose, Safety, Tolerability and Pharmacokinetic Study in Paediatric Patients With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT02056808
- Acronym / sponsor ID
- SMT C11002
- Conditions
- Duchenne Muscular Dystrophy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Summit TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Nov 2013
- Primary completion
- May 2014 (actual)
- Study completion
- Jul 2014 (actual)
- First posted
- 6 Feb 2014 (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.
The purpose of this study is to determine whether increasing doses of SMT C1100 are safe, well tolerated and achieve appropriate blood levels in patients with Duchenne Muscular Dystrophy (DMD).
Detailed description
Primary Objective: To determine the safety and tolerability of single and multiple oral doses of SMT C1100 in patients with Duchenne Muscular Dystrophy (DMD).
Secondary Objectives: To determine the single and multiple oral dose pharmacokinetics of SMT C1100 and its metabolites in patients with DMD.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| SMT C1100 | Experimental | Drug: SMT C1100Patients will be studied in 3 groups (Groups A to C), with each group consisting of 4 patients aged between 5 to 11 years. It is planned that doses for Groups A to C will be administered in an escalating manner after safety review for each dose group. |
Who may qualify
- Ages
- 5 to 11 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Patients will be males of any ethnic origin with a genetic diagnosis of DMD.
- Children between 5 and 11 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, as this is the only language the consent/assent forms are written in, and understand the requirements for reporting of any adverse event to the Investigator.
Key exclusion criteria (13 listed)
- Enrollment or participation in any therapeutic clinical trial within the prior 3 months or 5 times the half-life (whichever is longer).
- Initiation or change (other than dose modifications for body weight) of systemic corticosteroid therapy within 2 months prior to the start of dose administration or discontinuation of corticosteroids within 30 days prior to the start of dose administration.
- Known hypersensitivity to the excipients of the study drug or a previous history of drug allergy.
- Use of the following therapies is prohibited during the study and for at least 5 half-lives prior to the start of dose administration: Inducers of cytochrome P450 CYP1A2 (eg, carbamazepine, phenytoin, primidone, rifampin, omeprazole, and barbiturates), and moderate and strong inhibitors of CYP1A2 (e.g., fluvoxamine, ciprofloxacin, enoxacin, mexiletine; propafenone, zileuton). Substrates of CYP1A2 with narrow therapeutic windows (e.g., tacrine, theophylline, methadone, mexiletine). Nicotine, including exposure to daily passive smoking to minimize cytochrome P450 CYP 1A induction. Chargrilled food, cruciferous vegetables, caffeine, tea, and any xanthine containing foods, and drinks are prohibited from 36 hours prior to check-in until final discharge from study. Herbal supplements and homeopathic preparations (unless approved by medical monitor).
- Need for mechanical ventilation.
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 C1100Comparison of safety and pharmacokinetic of different dosages of drug | Drug | SMT C1100 |
Primary outcome
Safety and tolerability
Time frame: After 10 days of treatment phase
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.
1 secondary outcome
Pharmacokinetic parameters at different dose levels
Time frame: After single oral dose and after 10 days of treatment phase
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 27 Aug 2014.
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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.
Stefan Spinty, MD principal investigator
Alder Hey Children's NHS Foundation Trust
Helen Roper, MD principal investigator
Heart of England NHS Foundation Trust - Heartlands Hospital
Imelda Hughes, MD principal investigator
Central Manchester University Hospitals NHS Foundation Trust - Royal Manchester Childrens Hospital
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 Duchenne Muscular Dystrophy, 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 5 inclusion and 13 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 27 Aug 2014. 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?
Safety and tolerability (time frame: After 10 days of treatment phase). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 27 Aug 2014.