Open Label Extension Trial of Idebenone for Primary Progressive Multiple Sclerosis
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 61
- 试验地点
- 1
- 主要终点
- Area Under the Curve (AUC) of the Combinatorial Weight-Adjusted Disability Score (CombiWISE)
研究概览
简要总结
Background:
- The Idebenone in Patients With Primary Progressive Multiple Sclerosis (IPPoMS) trial tested a new drug for multiple sclerosis. In the IPPoMS trial, participants took either idebenone or placebo. Researchers want to give idebenone to all participants for 1 year. It is still not certain whether idebenone can slow the progression of multiple sclerosis, but this study may help answer that question.
Objectives:
- To provide idebenone to all participants on the IPPoMS trial.
- To collect data on the safety and effectiveness of idebenone for primary progressive multiple sclerosis.
Eligibility:
- Individuals at least 18 years of age who have completed 3 years in the IPPoMS trial.
Design:
- The first study visit for this trial will happen on the same day as the last visit for the IPPoMS trial.
- Participants will provide a blood samples and will have a lumbar puncture. They will also receive a new supply of idebenone to take three times a day with food. They will keep a diary to report on any side effects.
- After this first treatment visit, participants will have two follow-up visits to the NIH 6 months apart. These visits may be scheduled over multiple days. Participants will provide blood and urine samples. They will also have imaging studies of the brain and spine.
- Participants will have phone calls with the study researchers to provide updates on their condition and any side effects.
详细描述
OBJECTIVE:
A Phase I/II clinical trial is being conducted to investigate the safety, therapeutic efficacy and mechanism of action of idebenone in primary-progressive multiple sclerosis (PP-MS) patients (IPPoMS (Protocol Number 09-N-0197). Patients who have completed the 2-year treatment period of IPPoMS, may enter into this open-label extension study (IPPoMS-E) if they are found to be eligible by the Investigator and desire treatment with idebenone despite remaining blinded as to their allocation to active treatment versus placebo during the IPPoMS trial. The aim of this open-label extension study is gather additional data on safety, efficacy and effects of idebenone on cerebrospinal fluid (CSF) biomarkers in these patients over a period of 1 year. This study will provide open-label idebenone for patients with PP-MS, previously randomized to idebenone or to placebo in the blinded phase of IPPoMS.
STUDY POPULATION:
Patients who were previously enrolled in the IPPoMS (Protocol Number 09-N-0197) will be invited to participate in the trial. The same idebenone dose used in the randomized clinical trial (2250 mg/day) will be used in this study.
DESIGN:
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Active treatment
Idebenone (150mg tablets) administered orally as five tablets, three times per day with food.
干预措施: Idebenone (Drug)
结局指标
主要结局
Area Under the Curve (AUC) of the Combinatorial Weight-Adjusted Disability Score (CombiWISE)
时间窗: 1-year pre-treatment baseline vs 2-year randomized double-blind phased vs 1-year treatment period
CombiWISE is a composite scale derived from Expanded Disability Status Scale (EDSS) , Scripps Neurological Disability Scale (SNRS), times 25 foot walk (25FW), and non-dominant hand of 9 hole peg test (9HPT) with a minimum value of 0 (no disability) and maximum value of 100 (maximum disability). The AUC values were calculated for both study groups (Active treatment group in the 09-I-0197 trial and Placebo group in the 09-I-0197) as follows: 1. pre-treatment baseline during the 09-I-0197 trial (from Months -12, -6, and 0) 2. double-blind phase during the 09-I-0197 trial (from Months 0, 6, 12, 18, and 24) 3. extension phase during the 13-I-0088 trial (from Months 24, 30, and 36) Because the follow-up times varied from patient to patient, the AUC values were made comparable by scaling them by dividing the AUC value by the square of the actual duration (in years) of each of the phases.
次要结局
- Slopes of 25 Foot Walk (25FW) Time(3-years double-blind phase and 1-year extension phase)
- Slopes of 9 Hole Peg Test (9HPT) Time(3-years double-blind phase and 1-year extension phase)
- Slopes of Expanded Disability Status Scale (EDSS) Score(3-years double-blind phase and 1-year extension phase)
- Change in Slopes of Scripps Neurological Rating Scale (SNRS) Score(3-years double-blind phase and 1-year extension phase)