EUCTR2015-004333-27-GB进行中（未招募）1 期
A Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin Modulation with SMT C1100 in Ambulatory Paediatric Male Subjects with Duchenne Muscular Dystrophy (C11005) - PoC Study to Assess Activity and Safety of SMT C1100 in Boys with DMD
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 43
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
入选标准
- •Patients will be required to satisfy the following criteria at the screening visit:
- •1. Be able to provide written informed consent/assent as per local requirements.
- •2. Be male.
- •3.Be aged =5 years <10 years of age (from 5th birthday to 10th birthday)-not applicable for cohort 3
- •4. Have phenotypic evidence of dystrophinopathy based on the onset of characteristic clinical symptoms or signs (e.g., proximal muscle weakness, waddling gait, and Gowers' manoeuvre), an elevated serum creatinine kinase level, and ongoing difficulty with walking.
- •5. Have prior confirmation of the DMD diagnosis through:Documentation of the presence of a mutation in the dystrophin gene as determined by gene sequencing from a laboratory certified by the College of American Pathologists, the Clinical Laboratory Improvement Act/Amendment or an equivalent organization. Or Documentation of the absence of dystrophin in the muscle (via biopsy).
- •6. Be willing and able to comply with two muscle biopsy procedures (not applicable for cohort 3)
- •7. Be able to undergo MRI examination.
- •8. Patients must have used stable systemic corticosteroids (prednisone, prednisolone or deflazacort) for a minimum of 6 months immediately prior to the start of the Treatment Phase , with no significant change in dosage or dosing regimen (not related to body weight change) and a reasonable expectation that dosage and dosing regimen will not change significantly for the duration of the study.
- •9. Have the ability to walk at least 300 meters unassisted during the screening 6MWD and be below the protocol-specified threshold for 80%-predicted 6MWD (not applicable for cohort 3)
- •10. Have results of two 6MWD at Baseline determined as valid. The results of the second 6MWD must be within 20% of the first 6MWD (not applicable for cohort 3)
- •11. Have cardiac ECHO measurements showing an ejection fraction of = 55% and fractional shortening of =28% (not applicable for cohort 3)
- •12. Confirmed screening laboratory values within the central laboratory ranges (haematology, renal and serum electrolyte parameters and serum chemistry parameters) or considered not clinically significant in the opinion of the Investigator. Variations in specific parameters expected in a DMD population (e.g., aspartate aminotransferase, alanine aminotransferase, alkaline phosphatase, lactate dehydrogenase and creatinine phosphokinase) classed by the Investigator as not clinically significant will not exclude the patient
- •13.Be willing and able to comply with scheduled visits, drug administration plan, study procedures, laboratory tests and study restrictions.
- •14.Have taken part in a prior SMT C1100 study (not applicable for cohorts 1 and 2 )
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 40
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Patients will be excluded from the study if they satisfy the following
- •criteria at the screening visit
- •1. Have physical exam findings that in the investigator’s opinion should be exclusionary e.g., lower limb injury that may affect 6MWD performance.
- •2. Have any change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation or reinitiation) in prophylaxis/treatment for congestive heart failure (CHF) within 3 months prior to the start of study treatment.
- •3. Have uncontrolled clinical symptoms and signs of CHF (American College of Cardiology/American Heart Association Stage C or Stage D).
- •4.Have abnormal GLDH at baseline (>1.5 x ULN)
- •5.Have abnormal coagulation times at baseline (>1.5 x ULN)
- •6. Have an abnormal ECG e.g., a QTcF >500ms, left bundle-branch block or any other major conduction defect.
- •7. Use beta blockers (however, if during the course of the study they are clinically indicated they can be initiated; not applicable for cohort 3 )
- •8. Use herbal supplements and be unwilling to stop these for the duration of the study.
- •9. Have a known hypersensitivity to any of the ingredients or excipients of the IMP. (microfluidised oral suspension F3, cohort 1 : Poloxamer 188, Methylparaben, Propylparaben, Hydroxypropylmethyl cellulose, Glycerol, Non crystallizing sorbitol [70%], Xanthan gum, Strawberry cream flavour [PHS-132963]);
- •10.Have a known hypersensitivity to any of the ingredients or excipients of the IMP. Powder for oral suspension (F6): hypromellose acetate (not applicable to cohorts 1 and 3 )
- •11. Have been exposed to another investigational drug or DMD interventional agent within 3 months prior to start of the Treatment Phase. Prior exposure to SMT C1100 or participation in an approved deflazacort access program (e.g FOR-DMD or ACCESS DMD clinical trials ) within this period would not exclude the patient (provided they have been on stable treatment for 6 months )
- •12. Have a history of major surgical procedure within 12 weeks prior to the start of the Treatment Phase (week 1)
- •13. Be undertaking ongoing immunosuppressive therapy (other than corticosteroids).
- •14. Have an expectation of a major surgical procedure (e.g., scoliosis surgery) during the 12-month Treatment Phase of the study.
- •15. Require daytime ventilator assistance.
- •16. Have a prior or ongoing medical condition (e.g., concomitant illness, psychiatric condition, behavioural disorder, alcoholism, drug abuse), medical history, ECG findings, or laboratory abnormality that, in the Investigator's opinion, could adversely affect the safety of the subject, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.
- •17. Be dairy or lactose intolerant or have any other dietary restrictions that might interfere with the conduct of the study.
- •18. Be a smoker, use other tobacco or nicotine products or be exposed to daily passive smoking (including parent/legal guardian, siblings) so as to minimize environmental factors causing CYP1A induction.
- •19.Be using an approved DMD medication or anticipates using one during the duration of t
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