NL-OMON50228已完成2 期
Protocol Title: A randomised, double-blind, placebo-controlled study to evaluate the micro-macroscopic effects on muscles, the safety and tolerability, and the efficacy of givinostat in patients with Becker Muscular Dystrophy - DSC/15/2357/53
Italfarmaco S.p.A.0 个研究点目标入组 15 人开始时间: 待定最近更新:
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 15
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 99（—）
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 *
- •or * 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.
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 allowed.
- •3. Surgery that might affect muscle strength or function within 3 months before
- •study entry or planned surgery at any time during the study.
- •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 followup 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 syndrome).
- •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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