EUCTR2017-001629-41-NL进行中（未招募）1 期
A randomised, double-blind, placebo-controlled study to evaluate the micro-macroscopic effects on muscles, the safety and tolerability, and the efficacy ofgivinostat in patients with Becker Muscular Dystrophy - NA
ITALFARMACO S.P.A.0 个研究点目标入组 48 人开始时间: 2018年5月31日最近更新:
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 48
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
- 性别
- Male
入选标准
- •1. Ambulant male patients aged =18 years to < 65 years at randomization with BMD diagnosis confirmed by genetic testing.
- •2. Able and willing to give informed consent in writing.
- •3. Able to perform 6MWT at screening with a minimum distance of 200 m and maximum distance of 450 m.
- •4. If in treatment with systemic corticosteroids and/or ACE inhibitor, and/or ß or a adrenergic receptor blocker, no significant
- •change in dosage or dosing regimen (excluding changes related to body weight change) for a minimum of 6 months immediately
- •prior to start of study treatment.
- •5. Patients must be willing to use adequate contraception. Contraceptive methods must be used from Randomization through 3
- •months after the last dose of study treatment.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 48
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Exposure to another investigational drug within 3 months prior to the start of study treatment.
- •2. Use of any pharmacologic treatment, other than corticosteroids, that might have an effect on muscle strength or function within
- •3 months prior to the start of study treatment (e.g., growth hormone). Vitamin D, calcium, and any other supplements will be
- •3. Surgery that might affect muscle strength or function within 3 months before study entry or planned surgery at any time during
- •4. Presence of other clinically significant disease that in the Investigator’s opinion could adversely affect the safety of the patient,
- •making it unlikely that the course of treatment or follow-up is completed, or could impair the assessment of study results.
- •5. A diagnosis of other uncontrolled neurological diseases or presence of relevant somatic disorders not related to BMD that may interfere with the ability to perform the muscle function tests and/or to comply with the study protocol procedures.
- •6. Platelet count, WBC count and hemoglobin at screening < Lower Limit of Normal (LLN). If laboratory screening results are < LLN,
- •platelet count, WBC count and hemoglobin are to be repeated once, and if again < LLN become exclusionary.
- •7. Symptomatic cardiomyopathy or heart failure (New York Heart Association Class III or IV) or left ventricular ejection fraction <
- •50% at screening or with heart transplant.
- •8. Current liver disease or impairment, including but not limited to elevated total bilirubin (> 1.5 x ULN), unless secondary to
- •Gilbert’s disease or pattern consistent with Gilbert's disease.
- •9. Inadequate renal function, as defined by serum Cystatin C > 2 x the upper limit of normal (ULN). If the value is > 2 x ULN,
- •serum Cystatin C will be repeated once, and if again > 2 x ULN becomes exclusionary.
- •10. Positive test for hepatitis B surface antigen, hepatitis C antibody, or human immunodeficiency virus at screening.
- •11. Baseline corrected QT interval, Fredericia’s correction (QTcF) > 450 msec, (as the mean of 3 consecutive readings 5 minutes
- •apart) or history of additional risk factors for torsades de pointes (e.g., heart failure, hypokalemia, or family history of long QT
- •12. Current psychiatric illness/social situations rendering the potential patient unable to understand and comply with the muscle function
- •tests and/or with the study protocol procedures.
- •13. Hypersensitivity to the components of study medication.
- •14. Sorbitol intolerance or sorbitol malabsorption, or the hereditary form of fructose intolerance.
- •15. Contraindications to muscle biopsy.
- •16. Contraindications to MRI/MRS (e.g., claustrophobia, metal implants, or seizure disorder).
- •17. Hypertriglyceridemia (> 1.5 x upper limit of normal [ULN])*
- •* At screening, patients with hypertriglyceridemia can be enrolled if in stable treatment and with controlled levels of triglycerides (i.e. within normal range) for at least six months.
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