NCT02383511

Brief 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
12

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Feb 2015

Shorter than P25 for phase_1

Geographic Reach
1 country

4 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

February 1, 2015

Completed
3 days until next milestone

First Submitted

Initial submission to the registry

February 4, 2015

Completed
1 month until next milestone

First Posted

Study publicly available on registry

March 9, 2015

Completed
5 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2015

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2015

Completed
Last Updated

August 26, 2015

Status Verified

August 1, 2015

Enrollment Period

6 months

First QC Date

February 4, 2015

Last Update Submit

August 25, 2015

Conditions

Muscular Dystrophy, Duchenne

Outcome Measures

Primary Outcomes (1)

  • 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.

    28 days

Secondary Outcomes (5)

  • Safety and tolerability of SMT C1100

    28 days

  • Evaluation of plasma CK levels

    42 days

  • Pharmacokinetic parameters at different dose levels of SMT C1100

    28 Days

  • Safety and tolerability of SMT C1100

    28 Days

  • Pharmacokinetic parameters at different dose levels of SMT C1100

    28 Days

Study Arms (3)

Sequence 1

OTHER

Drug: SMT C1100 or placebo 3-treatment (Period 1,2 and 3)

Drug: SMT C1100

Sequence 2

OTHER

Drug: SMT C1100 or placebo 3-treatment (Period 1,2, and 3)

Drug: SMT C1100

Sequence 3

OTHER

Drug: SMT C1100 or placebo 3-treatment (Period 1,2 and 3)

Drug: SMT C1100

Interventions

SMT C1100DRUG

Period 1, SMT C1100 1250 mg BID; Period 2, Placebo BID; Period 3, SMT C1100 2500 mg BID

Sequence 1

Eligibility Criteria

Age5 Years - 13 Years
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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.

You may not qualify if:

  • 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).

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Heart of England NHS Foundation Trust - Heart Lands Hospital

Birmingham, B9 5SS, United Kingdom

Location

Alder Hey Children's NHS Foundation Trust

Liverpool, L12 2AP, United Kingdom

Location

Great Ormond Street Hospital for Children NHS Foundation Trust

London, WC1N 3JH, United Kingdom

Location

Central Manchester University Hospitals NHS Foundation Trust- Royal Manchester Children's Hospital

Manchester, United Kingdom

Location

Related Publications (1)

  • 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.

    PMID: 27055247DERIVED

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

SMT C1100

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 4, 2015

First Posted

March 9, 2015

Study Start

February 1, 2015

Primary Completion

August 1, 2015

Study Completion

August 1, 2015

Last Updated

August 26, 2015

Record last verified: 2015-08

Locations

GB(4)