Safety and Tolerability Trial of Arimoclomol for Sporadic Inclusion Body Myositis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 24
- 试验地点
- 2
- 主要终点
- Count of Adverse Events Reported
研究概览
简要总结
Inclusion body myositis (IBM) is the most common progressive and debilitating muscle disease beginning in persons over 50 years of age. This study will assess the safety and tolerability of Arimoclomol in IBM as compared to placebo over 4 months of treatment.
详细描述
IBM is a chronic disorder in which muscles become inflamed (swollen) and cause muscle weakening. The cause is unknown. There is new evidence to suggest that the pathology in IBM results from cellular changes induced by a variety of stressful events and diseases. In response to these stressful events the body's normal response is to increase the levels of Heat Shock Proteins (HSP) to help counteract and stop these cellular changes. In people with IBM this increase does not appear sufficient enough to reverse these toxic cellular changes. Arimoclomol causes the body to make more of this HSP protein. By increasing HSP levels in IBM patients we hope to reverse the toxic cellular changes that might be responsible for the pathology of IBM.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 50 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Meet the diagnostic criteria for definite or probable IBM (Griggs 1995)
- •Muscle function adequate for quantitative muscle testing
- •Age > 50 years
- •Women must be postmenopausal or status post hysterectomy
- •For any patient currently taking medication for IBM, they must remain on current dosage for the extent of the study and last dosage change must be > 30 days previous to enrollment
排除标准
- •Presence of any one of the following medical conditions: diabetes mellitus or patients taking anti-diabetic medications, chronic infection, chronic renal insufficiency, cancer other than skin cancer less than 5 years previously, multiple sclerosis or prior episode or central nervous system demyelination, or other chronic serious medical illnesses
- •Presence of any of the following on routine blood screening: WBC < 3000, platelets < 100,000, hematocrit < 30%, BUN > 30 mg%, creatine > 1.5 mg%, symptomatic liver disease with serum albumin < 3 g/dl, PT or PTT > upper range of control values
- •Women who are pregnant or lactating
- •History of non-compliance with other therapies
- •Coexistence of other neuromuscular disease
- •Drug or alcohol abuse within the last 3 months
- •Inability to give informed consent
- •Known bleeding disorder
- •Use of potentially renal toxic drugs
- •Prior difficulties with local anesthetic
研究组 & 干预措施
1
Arimoclomol
干预措施: Arimoclomol (Drug)
2
干预措施: Placebo (Other)
结局指标
主要结局
Count of Adverse Events Reported
时间窗: Month 12
Measure reflects the total number of adverse events reported during course of the study.
次要结局
- Heat Shock Protein 70 (HSP70) Levels in the Tissue(Change from Baseline to Month 4)
- Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score(Change from Baseline to Month 12)
- Muscle Strength Testing(Change from Baseline to Month 12)
- Maximum Isometric Voluntary Contraction Testing (MVICT) Score(Change from Baseline to Month 12)
研究者
Richard Barohn, MD
Gertrude and Dewey Zeigler Professor of Neurology and Chair
University of Kansas Medical Center