HERV-K Suppression Using Antiretroviral Therapy in Volunteers With Amyotrophic Lateral Sclerosis (ALS)
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 122
- 试验地点
- 1
- 主要终点
- Percent Change in HERV-K Level
研究概览
简要总结
Background:
Some people with Amyotrophic Lateral Sclerosis (ALS) have a high level of the virus HERV-K in their blood. Researchers do not think this virus causes ALS. But they don t know why some people with ALS have a high level of it. They want to know if HERV-K can be suppressed by drugs that are used to treat HIV infection.
Objectives:
To learn how drugs usually taken for HIV infection affect people with Amyotrophic Lateral Sclerosis (ALS).
Eligibility:
Adults at least 18 years old with ALS and high levels of HERV-K but no HIV.
Design:
Interested participants can contact the study team and, if eligible, the study team will arrange for a screening blood draw to determine the HERV-K level.
Participants with a high HERV-K level will be screened with medical history, physical exam, questionnaires, nerve conduction test, lumbar puncture, and blood and breathing tests.
After screening, participants will start taking the 4 study drugs.
Participants will have up to 12 study visits over a period of 72 weeks. After starting study drugs, they will have study visits at Weeks 1 and 4 and then every 4 weeks until Week 28. They will be asked how they are feeling and have an exam and blood drawn. At 3 visits, they will have tests of nerve conduction, breathing, and their ALS symptoms.
At Week 24, they will stop taking the study drugs and may have a repeat lumbar puncture.
After the Week 48 visit, their participation is finished.
详细描述
Objective:
In this Phase I, proof-of-concept study, we aim to determine whether an antiretroviral regimen approved to treat human immunodeficiency virus (HIV) infection would also suppress levels of Human Endogenous Retrovirus-K (HERV-K) found to be activated in a subset of patients with amyotrophic lateral sclerosis (ALS). We propose to measure the blood levels of HERV-K before, during, and after treatment with an antiretroviral regimen. We will evaluate the safety of the antiretroviral regimen for participants with ALS and also explore clinical and neurophysiological outcomes of ALS symptoms, quality of life, and pulmonary function.
Study Population:
We will study a subset of ALS patients who have a ratio of HERV-K:RPP30 greater than or equal to 13. About 30% of ALS patients may have detectable levels of HERV-K; about 20% of patients with ALS have a level >1000 copies/ml. To show whether the HERV-K could be suppressed, we will recruit from the approximately 20% of patients with high levels so that the antiretroviral effect can be determined.
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
ALS
20 participants with ALS and a level of HERV-K:RPP30 greater than or equal to 13
干预措施: Ritonavir (Drug)
ALS
20 participants with ALS and a level of HERV-K:RPP30 greater than or equal to 13
干预措施: Darunavir (Drug)
ALS
20 participants with ALS and a level of HERV-K:RPP30 greater than or equal to 13
干预措施: Dolutegravir (Drug)
ALS
20 participants with ALS and a level of HERV-K:RPP30 greater than or equal to 13
干预措施: Tenofovir alafenamide (TAF) (Drug)
结局指标
主要结局
Percent Change in HERV-K Level
时间窗: 24 weeks
Blood samples were obtained during the screening visit and week 24 (post-treatment with antiretroviral drugs). The percent change in HERV-K level was measured using quantitative PCR: 100% x (screening visit - week 24 visit measurement) / screening visit.
次要结局
未报告次要终点