A Widely Inclusive, Hybrid-Decentralized Pilot Trial Utilizing β-hydroxy-β-methylbutyrate to Lower IGFBP7 Levels in People With ALS
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 100
- 试验地点
- 2
- 主要终点
- ALS Functional Rating Scale, Revised (ALSFRS-R)
研究概览
简要总结
This is an open label trial of a supplement called HMB in patients with ALS. The researchers are evaluating its safety and tolerability, as well as its ability to lower insulin-like growth-factor binding protein 7 (IGFBP7) and Neurofilament light chain levels (NFL) and to slow ALS Functional Rating Scale, Revised (ALSFRS-R) progression.
详细描述
This will be a widely inclusive, three-center, open-label pilot trial enrolling 100 people living with ALS. Duke (25 participants) and Temple (25 participants) will be traditional sites that consent, screen and follow participants in person, Everything ALS (50 participants) will be a decentralized site providing virtual consenting, screening and follow up. The total time commitment will be 9 months. All participants who pass screening will provide demographics, disease characteristics, co-morbidities, and concomitant medications. They will have a baseline ALSFRS-R score obtained, a baseline slope of Listener Effort Prediction Model (LEPM)-predicted Listener Effort (LE) progression, and Motor Movement Exam (MME) score. For those who enroll at Duke or Temple, blood will be drawn for neurofilament light chain (NfL) and IGFBP7 levels. For patients at Duke or Temple who consent to lumbar puncture, CSF will be drawn for NfL and IGFBP7 levels as well. Each month after baseline, participants will be contacted by phone by each site's study coordinator to review adverse events, new co-morbidities, and concomitant medications, and to generate a new clinician-administered ALSFRS-R score. For Duke and Temple participants, at month 1, 3 and 6 they will be asked to return for in person blood draws for NfL and IGFBP7 levels. Those who sign the additional consent for lumbar punctures will have CSF drawn as well and this will be checked for NfL and IGFBP7 levels at month 3, too. LSFRS-R is routinely measured as part of clinical care. All of the described blood tests and investigational treatments are being performed exclusively for research purposes. HMB will be given at a dose of 3g daily.
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
- •Male or female aged at least 18 years.
- •Sporadic or familial ALS diagnosed as per Gold Coast Criteria (37).
- •Patient is able to understand and express informed consent (in the opinion of the site investigator).
- •Patient is able to read and write English.
- •Patient is expected to survive for the duration of the trial.
- •Women must not be pregnant (will have evidence of a negative pregnancy test obtained by study team at baseline, or by local physician within past 7 days or be post-menopausal)
- •Women must not be able to become pregnant (e.g., post-menopausal, surgically sterile, or using adequate birth control methods) for the duration of the study and three months after study completion. Adequate contraception includes abstinence, hormonal contraception (oral contraception, implanted contraception, injected contraception, or other hormonal contraception, for example patch or contraceptive ring), intrauterine device (IUD) in place for ≥ 3 months, barrier method in conjunction with spermicide, or another adequate method.
排除标准
- •Actively or recently (within past 30 days) participating in another intervention trial.
- •Currently or recently (within 30 days) taking HMB
- •Prior side effects from HMB deemed to be significant by the investigator
- •Patient has a medical or psychiatric illness that could in the investigator's opinion interfere with the patient's ability to participate in this study.
- •Pregnant women or women currently breastfeeding.
- •Elevated serum calcium or vitamin D levels.
- •Life expectancy shorter than the duration of the trial.
研究组 & 干预措施
β-hydroxy-β-methylbutyrate (HMB) administration
干预措施: β-hydroxy-β-methylbutyrate (HMB) (Drug)
结局指标
主要结局
ALS Functional Rating Scale, Revised (ALSFRS-R)
时间窗: Baseline, month 3, month 9
A quickly administered (five minute) ordinal rating scale (ratings 0-4) used to determine patients' assessments of their capability and independence in 12 functional activities. All 12 activities are relevant to people living with ALS.The ALSFRS-R declines linearly with time over a wide range during the course of ALS and it has been validated for telephone use. The ALSFRS-R Range is 0-48. The more points, the better the person is functioning.
Speech (Listener Effort)
时间窗: Baseline, month 3, month 9
Listener effort (LE) is a perceptual rating of the amount of work necessary for a listener to understand speech, rated by an expert speech-language pathologist on a visual analog scale from 0 (easily understood) - 100 (unintelligible even with full effort) and it has proven to be one of the most robust overall measures of dysarthria.
次要结局
- Neurofilament Light Chain levels (blood)(Baseline, month 3, and month 6)
- Neurofilament Light Chain levels (Cerebrospinal fluid (CSF))(Baseline, month 3)
- Insulin-like growth-factor binding protein 7 (IGFBP7) levels (blood)(Baseline, month 3 and month 6)
- Insulin-like growth-factor binding protein 7 (IGFBP7) levels (Cerebrospinal fluid (CSF))(Baseline, month 3)
- Frequency of ALS reversals(6 months)