Treatment of FUS-Related ALS With Betamethasone - The TRANSLATE Study
试验速览
- 阶段
- 早期 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Betamethasone plasma levels
研究概览
简要总结
By doing this study the investigator hopes to learn more about a potential cause of amyotrophic lateral sclerosis (ALS) called "oxidative stress". Oxidative stress is essentially an imbalance between the production of certain chemicals in the body called "free radicals" and the ability of the body to counteract or detoxify their harmful effects through neutralization by antioxidants. It is thought that factors such as environmental exposure (chemicals and lead), diet, smoking,alcohol consumption, physical activity and psychological stress cause oxidative stress to occur inside the body.
By doing this study, the investigator hopes to learn whether the FDA-approved steroid medication called Betamethasone will restore overall antioxidant activity fALS patients with mutations in the Fused in Sarcoma gene (FUS gene).
Participants who agree to take part in this research study, agree to the following responsibilities:
- Attend all scheduled visits
- Notify the study doctor of any illnesses, unexpected or troublesome side effects, or any other medical problems that occur during the study
- Be completely honest with their answers to all questions
- Check with the study doctor before taking any new medications, whether prescribed or "over the counter," even vitamins and herbal supplements.
详细描述
This will be a single-blinded, pharmacokinetic and pharmacodynamics study of intramuscular (IM) betamethasone in ALS patients and non-ALS relatives from families with a mutation in the FUS gene. Participants remain blinded as to their genotype.
Betamethasone is a FDA-approved drug and is only available in an IM dosing form (Celestone®). This will be a proof-of-concept translational study built on discoveries made by our research team at the University of Kentucky. All participants will receive active drug.
The research procedures will be conducted at the University of Kentucky (UK) Albert B. Chandler Hospital, Pavilion H, 800 Rose Street, Lexington, KY. Participants will need to come to Center for Clinical and Translational Science (CCTS) on the 3rd Floor (Room C300) of Pavilion H, at UK Albert B. Chandler Hospital, for a Consent/Screening Visit. If found eligible and qualify for the study, participants will be asked to give voluntary written consent to participate.
Following signing the consent form, participants will be admitted to the UK Albert B. Chandler Hospital, CCTS Inpatient Unit, 5th Floor, 5 North Wing of the Hospital for 2 nights (approximately 48 hours). The following tests and procedures will take place:
- Neurological Exam
- Vital Signs (blood pressure, heart rate and respiratory rate) will be measured
- Medical and medication history will be collected
- Questionnaire
- blood specimen for Pharmacokinetic and Pharmacodynamic will be collected testing*
- Participants will also be asked to do some testing of their breathing and physical abilities.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Single (Participant)
盲法说明
Participants remain blinded as to their genotype.
入排标准
- 年龄范围
- 20 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of familial ALS (fALS)
- •Relative of a fALS person and carry the FUS gene
排除标准
- •Under 20 years or over 80 years of age
- •Cannot tolerate steroids, including betamethasone
- •Are unwilling or unable to attend all scheduled research visits
- •Currently participating in another clinical drug trial
- •Major neurological disease, other than ALS
研究组 & 干预措施
Arm 1 - Amyotrophic Lateral Sclerosis
Betamethasone sodium phosphate/betamethasone acetate (Celestone® Soluspan®), 30 mg IM once a day for four days
干预措施: Betamethasone sodium phosphate/betamethasone acetate (Celestone® Soluspan®), 30 mg IM once a day for four days (Drug)
Arm 2 - Familial Amyotrophic Lateral Sclerosis
Betamethasone sodium phosphate/betamethasone acetate (Celestone® Soluspan®), 30 mg IM once a day for four days
干预措施: Betamethasone sodium phosphate/betamethasone acetate (Celestone® Soluspan®), 30 mg IM once a day for four days (Drug)
结局指标
主要结局
Betamethasone plasma levels
时间窗: 14 days
Blood will be collected at multiple time points (baseline, 24 hours, 48 hours, 72 hours, day 7 and day 14) for analysis of Betamethasone levels. Data will be reported as the change in Betamethasone over time.
次要结局
- Protein carbonyl plasma levels(14 days)
- Superoxide dismutase plasma levels(14 days)
- Glutathione disulfide plasma levels(14 days)
- Peroxide plasma levels(14 days)
- Glutathione plasma levels(14 days)
研究者
Edward Kasaraskis
Principal Investigator
University of Kentucky