NCT01805024已完成1 期
Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety and Tolerability of Omigapil (CALLISTO)
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Pharmacokinetic Profile of Omigapil:Maximum Observed Plasma Concentration (Cmax) of Omigapil
研究概览
简要总结
The purpose of the study is to establish the pharmacokinetic profile of omigapil in paediatric and adolescent patients with CMD and to evaluate the safety and tolerability of omigapil.
Funding source - FDA OOPD
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 5 Years 至 16 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Ambulatory and non-ambulatory patients from age 5 - 16 years (5 years old and <17 years old) at time of screening with a clinical picture (see below) consistent with COL6-related dystrophy (COL6-RD) or LAMA2-related dystrophy (LAMA2-RD)
- •Under regular review at a neuromuscular center
- •On adequate double-barrier contraception (if of child-bearing potential)
- •Stable on any allowed concomitant medications for 1 month prior to run in Phase
- •Forced Vital Capacity (FVC) 30-80% of the predicted value and confirmed at Screening and Baseline visit(s)
- •For patients with Collagen VI-related dystrophy (COL6-RD)- required clinical picture:
- •Muscle weakness: inability to walk or, if patient is still ambulatory, inability to run and > 5 s for 10 m walk
- •Genetic and Pathology:
- •Molecular diagnosis of COL6-RD, defined by one dominant or two recessive mutation(s) in COL6A1, COL6A2 or COL6A3 known to cause the clinical picture,,
- •Histological diagnosis showing (i) absent or significantly decreased expression of collagen VI in muscle (overall reduction or basal lamina specific) or (ii) absent or significantly abnormal matrix in skin fibroblast culture
- •For patients with Laminin alpha 2 related dystrophy (LAMA2-RD) - required clinical picture:
- •Muscle weakness: Inability to walk; if patient is still ambulatory, inability to run and > 5 s for 10 m walk.
- •Genetics and Pathology:
- •Either: 2 identified pathologic or probable pathologic mutations in LAMA2 gene
- •1 identified pathologic or probable pathologic mutation in LAMA 2 gene with evidence of decrease in laminin alpha 2 staining on muscle or skin biopsy
- •Evidence of decrease in laminin alpha 2 staining on muscle or skin biopsy with matching clinical phenotype and no suspicion of alpha dystroglycanopathy (aDG-RD) (clinically or by staining on muscle biopsy)
排除标准
- •Use of any investigational drug other than the study medication within 12 weeks of study start.
- •Recurrent hospitalisation for chest infections in previous 2 years (≥2 per year)
- •Patients with respiratory parameters (eg: low pulmonary function test value i.e. <30% or need for brief course of daytime non-invasive ventilation) currently affected by short term medications, or acute illness/ conditions (conduct baseline assessments when the patient has recovered and no longer taking acute medication)
- •Any need for surgery (scoliosis, gastrostomy, other) in the preceding 24 weeks or foreseen during the course of the study.
- •Patient has an intercurrent significant medical condition or situation which in the opinion of the Investigator or the study Medical Monitor may put the patient at significant risk, confound the study results or interfere significantly with the patient's participation in the study
- •Failure to thrive, defined as:
- •Falling 20 percentiles (20/100) in body weight in the 12 weeks preceding Screening/Baseline (based on family report of weight loss and acquiring relevant medical records)
- •In patients below the 3rd percentile, any further drop in body weight percentile in the 12 weeks preceding Screening/Baseline (based on family report of weight loss and acquiring relevant medical records)
- •Weight less than 17kg at Baseline
- •Morbidly obese or grossly overweight (≥86 percentile BMI in children)
- •History of epilepsy or on antiepileptic medication at Screening/Baseline
- •On daytime Non Invasive Ventilation (NIV)
- •Intake of prohibited medication (as listed in Appendix I)
- •Anticipated need for anesthesia during the course of this study
- •Patients with renal impairment defined as urinary protein concentration ≥ 0.2 g/L
- •Patients with moderate to severe hepatic impairment
- •ALT ≥ 8x upper limit of normal (ULN) and total bilirubin 2x ULN (plus >35% 'direct' bilirubin), or
- •ALT ≥ 8x ULN and INR >1.5 or ALT >2x baseline levels, and total bilirubin > 2x ULN (plus >35% 'direct' bilirubin), or
- •ALT >2x baseline levels, and INR greater than 1.5,
研究组 & 干预措施
Omigapil
Experimental
Omigapil treatment oral administration once per day after breakfast Cohort 1 Cohort 1 0.02 mg/kg/day Cohort 2 0.08 mg/kg/day Cohort 3a 0.04 mg/kg/day Cohort 3b 0.06 mg/kg/day
干预措施: Omigapil (Drug)
结局指标
主要结局
Pharmacokinetic Profile of Omigapil:Maximum Observed Plasma Concentration (Cmax) of Omigapil
时间窗: 0.5, 1, 1.5, 2, 4 and 8 hours post-dose on Day 1, Week 4 and Week 12
Pharmacokinetic Profile of Omigapil: Time at Which Cmax Was Apparent (Tmax) of Omigapil
时间窗: 0.5, 1, 1.5, 2, 4 and 8 hours post-dose on Day 1, Week 4 and Week 12
Pharmacokinetic Profile of Omigapil Area Under the Plasma Concentration Versus Time Curve From Time Zero to 8h Post-dose (AUC0-8)
时间窗: 0 to 8 hours post-dose on Day 1, Week 4, Week 12
次要结局
- Summary of All Treatment-emergent Adverse Events (TEAEs)(12 weeks)
研究者
研究点 (1)
Loading locations...
相似试验
已完成
不适用
A Study to Test Lung Stretch Therapy (Hyperinsufflation) in Children With Collagen VI Muscular DystrophyCongenital Muscular DystrophyNCT01836627Children's Hospital Medical Center, Cincinnati34
已完成
4 期
A Pilot Study of Dexmedetomidine-Propofol in Children Undergoing Magnetic Resonance ImagingDevelopmental DelayEpilepsyCerebral PalsyNCT02633241Joseph Cravero71
招募中
不适用
Myotonic Dystrophy Type 1 Congenital and Juvenile Form: From Diagnosis to Rehabilitation [MDCJ-NeuBeRe]Myotonic Dystrophy 1NCT06378216IRCCS Eugenio Medea30
尚未招募
2 期
Repurposing Empagliflozin for DMD-associated Cardiomyopathy in Children 6-18 Years of AgeDMD-associated Dilated CardiomyopathyNCT06643442Sebastiano Lava12
已完成
1 期
Gentamicin Treatment of Muscular DystrophyBecker Muscular DystrophyDuchenne Muscular DystrophyNCT00005574National Institute of Neurological Disorders and Stroke (NINDS)4