A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) With Open-Label Extension (OLE)
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 76
- 试验地点
- 17
- 主要终点
- Number of Participants Reporting Serious Treatment Emergent Adverse Events (Serious TEAEs) and TEAEs
研究概览
简要总结
This study is an open-label extension to evaluate the safety and tolerability of long-term dosing of Losmapimod in patients with FSHD1 who participated in the ReDux4 study.
详细描述
This study is an open-label extension study to evaluate the safety and tolerability of long-term dosing of Losmapimod in patients with FSHD1 who participated in the ReDux4 study.
This study is a multi-center clinical trial. It will be conducted in North America, Canada and Europe. Only patients who participated and competed all study procedures in the ReDUX4 Study treatment period will be eligible to participate in this open label extension study.
Patients who complete the randomized, placebo-controlled portion of the study will have the option to roll over into the open-label extension study.
Patients will receive 15 mg of losmapimod by mouth twice daily for a total of 30 mg by mouth daily. All patients will attend clinic visits approximately every 12 weeks.
Participation in this open-label extension study will continue until 90 days after losmapimod becomes commercially available, the patient withdraws from the study, or the Sponsor decides to close the study.
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
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The patient must have consented to participate and must have provided signed, dated and witnessed an IRB-approved informed consent form that conforms to federal and institutional guidelines.
- •Male or female subjects
- •Patients must be between 18 and 65 years of age, inclusive
- •Must be willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines and other study procedures.
- •Will practice an approved method of birth control
排除标准
- •Has a history of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject. This may include, but is not limited to, a history of relevant drug or food allergies; history of cardiovascular or central nervous system disease; neuromuscular diseases except FSHD (eg, myopathy, neuropathy, neuromuscular junction disorders); or clinically significant history of mental disease.
- •For subjects who are on drug(s) or supplements that may affect muscle function, as determined by the treating physician, or that are included in the list of drugs presented in the protocol, subjects must be on a stable dose of that drug(s) or supplement for at least 3 months prior to the first dose of study drug and remain on that stable dose for the duration of the study. Changes to the dose or treatment discontinuation during the study can only be done for strict medical reasons by the treating physician with clear documentation and notification to the sponsor.
研究组 & 干预措施
Losmapimod
FSHD1 patients with genetic confirmation will receive Losmapimod 15 mg by mouth twice daily for a total of 30 mg daily until 90 days after commercial drug is available post regulatory approval or until the study is discontinued by the Sponsor.
干预措施: Losmapimod (Drug)
结局指标
主要结局
Number of Participants Reporting Serious Treatment Emergent Adverse Events (Serious TEAEs) and TEAEs
时间窗: Up to 57 months
A TEAE is an adverse event that begins on or after the first dose of study drug and on or before the stop of study drug plus 7 days or begins before the first dose of study drug and worsens on or after the first dose of study drug and on or before the stop of study drug plus 7 days. An adverse event with completely missing onset and end dates was considered as TEAE. An adverse event with missing onset date but the end date is on or after the first dose of study drug and before the stop of study drug plus 7 days was considered a TEAE.
Number of Participants With Clinically Significant Changes in Chemistry Parameters
时间窗: Up to 57 months
Blood samples were collected for the analysis of chemistry parameters: Glucose, sodium, potassium, calcium, inorganic phosphate, total protein, albumin, blood urea nitrogen, creatinine, total bilirubin, alkaline phosphatase, aspartate aminotransferase, alanine aminotransferase, gamma glutamyl transferase and creatine phosphokinase.
Number of Participants With Clinically Significant Changes in Hematology Parameters
时间窗: Up to 57 months
Blood samples were collected for the analysis of hematology parameters: hemoglobin (including mean corpuscular volume), mean corpuscular hemoglobin, mean corpuscular hemoglobin concentration, hematocrit, red blood cell count, total white blood cell count, platelet count. Differential blood counts, including basophils, eosinophils, neutrophils, lymphocytes, and monocytes
Number of Participants With Clinically Significant Changes in Electrocardiogram (ECG) Parameters
时间窗: Up to 57 months
Twelve-lead ECGs was performed after participants has been recumbent for at least 5 minutes.
Number of Participants With Clinically Significant Changes in Serum Coagulation Parameters
时间窗: Up to 57 months
Blood samples were collected for the analysis of Serum coagulation parameters: International normalized ratio, prothrombin time, partial thromboplastin time.
Number of Participants With Clinically Significant Changes in Urinalysis
时间窗: Up to 57 months
Urine samples were collected for the analysis of urinalysis parameters: Leucocytes, blood, nitrite, protein, urobilinogen, bilirubin, pH, specific gravity, ketones, glucose.
Number of Participants With Clinically Significant Changes in Vital Parameters
时间窗: Up to 57 months
Vital parameters including pulse rate, respiration rate, blood pressure, and temperature were measured in seated or recumbent for at least 5 minutes. Data for number of participants with abnormal clinically significant changes for vital signs have been presented.
Number of Participants With Clinically Significant Changes in Physical Examinations
时间窗: Up to 57 months
Physical examinations included an evaluation of body systems, including but not limited to the following: skin; head, eyes, ears, nose, and throat; respiratory system; cardiovascular system; abdomen (liver, spleen); lymph nodes; neurological system; and musculoskeletal system.
次要结局
未报告次要终点