A Widely Inclusive, Hybrid-Decentralized Pilot Trial Utilizing β-hydroxy-β-methylbutyrate to Lower IGFBP7 Levels in People With ALS
2026년 5월 11일 업데이트: Duke University
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.
연구 개요
상세 설명
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.
연구 유형
중재적
등록 (추정된)
100
단계
- 1단계
연락처 및 위치
이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.
연구 연락처
- 이름: Richard Bedlack, MD, PhD
- 전화번호: 919-613-2681
- 이메일: alsresearch@duke.edu
연구 연락처 백업
- 이름: Michelle Ward, RN
- 전화번호: 919-613-2681
- 이메일: alsresearch@duke.edu
연구 장소
-
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North Carolina
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Durham, North Carolina, 미국, 27705
- Duke University Medical Center
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수석 연구원:
- Richard Bedlack, MD, PhD
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연락하다:
- Richard Bedlack, MD. PhD
- 이메일: alsresearch@duke.edu
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Pennsylvania
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Philadelphia, Pennsylvania, 미국, 19122
- Temple University
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수석 연구원:
- Terry Heiman-Patterson, MD
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연락하다:
- John Furey
- 이메일: john.furey0001@temple.edu
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연락하다:
- Christopher Pizzica
- 이메일: christopher.pizzica@temple.edu
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참여기준
연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.
자격 기준
공부할 수 있는 나이
- 성인
- 고령자
건강한 자원 봉사자를 받아들입니다
아니
설명
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
공부 계획
이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 해당 없음
- 중재 모델: 단일 그룹 할당
- 마스킹: 없음(오픈 라벨)
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
|---|---|
|
실험적: β-hydroxy-β-methylbutyrate (HMB) administration
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Our source of HMB will be Life Extension's "Wellness Code Muscle Strength & Restore Formula.
The dose will be 3g daily, which has previously been shown to be safe and well-tolerated, and to reduce IGFBP7 blood levels in humans.
|
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
ALS Functional Rating Scale, Revised (ALSFRS-R)
기간: 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.
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Baseline, month 3, month 9
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Speech (Listener Effort)
기간: 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.
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Baseline, month 3, month 9
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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Neurofilament Light Chain levels (blood)
기간: Baseline, month 3, and month 6
|
Neurofilaments are neuron-specific components of the cytoskeleton.
They exist in heavy, medium, and light chain forms.
Neurofilament light chain levels are elevated in the spinal fluid and the blood of patients with ALS and other neurodegenerative diseases, and higher levels predict more severe disease progression.
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Baseline, month 3, and month 6
|
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Neurofilament Light Chain levels (Cerebrospinal fluid (CSF))
기간: Baseline, month 3
|
Neurofilaments are neuron-specific components of the cytoskeleton.
They exist in heavy, medium, and light chain forms.
Neurofilament light chain levels are elevated in the spinal fluid and the blood of patients with ALS and other neurodegenerative diseases, and higher levels predict more severe disease progression.
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Baseline, month 3
|
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Insulin-like growth-factor binding protein 7 (IGFBP7) levels (blood)
기간: Baseline, month 3 and month 6
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Non-competitive inhibitor of the IGF-1 receptor
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Baseline, month 3 and month 6
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Insulin-like growth-factor binding protein 7 (IGFBP7) levels (Cerebrospinal fluid (CSF))
기간: Baseline, month 3
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Non-competitive inhibitor of the IGF-1 receptor
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Baseline, month 3
|
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Frequency of ALS reversals
기간: 6 months
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Frequency of ALS reversals will be calculated as the number of participants who have an ALSFRS-R score that improves by 4 points or more over 6 months of treatment.
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6 months
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공동 작업자 및 조사자
여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.
연구 기록 날짜
이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.
연구 주요 날짜
연구 시작 (추정된)
2026년 7월 1일
기본 완료 (추정된)
2026년 7월 1일
연구 완료 (추정된)
2026년 8월 1일
연구 등록 날짜
최초 제출
2026년 5월 11일
QC 기준을 충족하는 최초 제출
2026년 5월 11일
처음 게시됨 (실제)
2026년 5월 15일
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
2026년 5월 15일
QC 기준을 충족하는 마지막 업데이트 제출
2026년 5월 11일
마지막으로 확인됨
2026년 5월 1일
추가 정보
이 연구와 관련된 용어
추가 관련 MeSH 약관
기타 연구 ID 번호
- Pro00120054
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
아니요
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
예
미국 FDA 규제 기기 제품 연구
아니
미국에서 제조되어 미국에서 수출되는 제품
아니
이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .
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