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A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD)

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ClinicalTrials.gov Identifier: NCT02056808
Recruitment Status : Completed
First Posted : February 6, 2014
Last Update Posted : August 27, 2014
Sponsor:
Information provided by (Responsible Party):
Summit Therapeutics

Brief 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).

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: SMT C1100 Phase 1

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 Type : Interventional  (Clinical Trial)
Actual Enrollment : 12 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Study Start Date : November 2013
Actual Primary Completion Date : May 2014
Actual Study Completion Date : July 2014


Arm Intervention/treatment
Experimental: SMT C1100
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.
Drug: SMT C1100
Comparison of safety and pharmacokinetic of different dosages of drug




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. Pharmacokinetic parameters at different dose levels [ Time Frame: After single oral dose and after 10 days of treatment phase ]
    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.



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Ages Eligible for Study:   5 Years to 11 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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.
  • 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).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02056808


Locations
United Kingdom
Heart of England NHS Foundation Trust - Heart Lands Hospital
Birmingham, United Kingdom, B9 5SS
Alder Hey Children's NHS Foundation Trust
Liverpool, United Kingdom, L12 2AP
Great Ormond Street for Children NHS Foundation Trust
London, United Kingdom
Central Manchester University Hospitals NHS Foundation Trust- Royal Manchester Children's Hospital
Manchester, United Kingdom
Sponsors and Collaborators
Summit Therapeutics
Investigators
Principal Investigator: Stefan Spinty, MD Alder Hey Children's NHS Foundation Trust
Principal Investigator: Helen Roper, MD Heart of England NHS Foundation Trust - Heartlands Hospital
Principal Investigator: Imelda Hughes, MD Central Manchester University Hospitals NHS Foundation Trust - Royal Manchester Childrens Hospital
Principal Investigator: Franceso Muntoni, MD Great Ormond Street Hospital for Children NHS Foundation Trust

Responsible Party: Summit Therapeutics
ClinicalTrials.gov Identifier: NCT02056808     History of Changes
Other Study ID Numbers: SMT C11002
First Posted: February 6, 2014    Key Record Dates
Last Update Posted: August 27, 2014
Last Verified: August 2014

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked