A Widely Inclusive, Hybrid-Decentralized Pilot Trial Utilizing β-hydroxy-β-methylbutyrate to Lower IGFBP7 Levels in People With ALS

NCT07589764 | Not Yet Recruiting Phase 1 FDA Drug

• 1 other identifier: Pro00120054
• Study Type: interventional
• Target: 100
• Locations: 1 country
• Sites: 2

Brief Summary

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.

Conditions

Amyotrophic Lateral Sclerosis ALS

Outcome Measures

Primary Outcomes (2)

• ALS Functional Rating Scale, Revised (ALSFRS-R)

  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.

  Baseline, month 3, month 9

• Speech (Listener Effort)

  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.

  Baseline, month 3, month 9

Secondary Outcomes (5)

• 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

Study Arms (1)

β-hydroxy-β-methylbutyrate (HMB) administration

EXPERIMENTAL

Drug: β-hydroxy-β-methylbutyrate (HMB)

Interventions

β-hydroxy-β-methylbutyrate (HMB) DRUG

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.

β-hydroxy-β-methylbutyrate (HMB) administration

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Duke University: lead
• Temple University: collaborator

Study Sites (2)

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

Temple University

Philadelphia, Pennsylvania, 19122, United States

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Richard Bedlack, MD, PhD

  Duke University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Richard Bedlack, MD, PhD

CONTACT

919-613-2681; alsresearch@duke.edu

Michelle Ward, RN

CONTACT

919-613-2681; alsresearch@duke.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Participants will be people with ALS who are cared for by Dr. Bedlack's team at Duke or Dr. Heiman-Patterson's team at Temple, or who contact our sites inquiring about this study. 100 total participants will be enrolled (25 at Duke, 25 at Temple, 50 at Everything ALS).

Study Record Dates

• First Submitted: May 11, 2026
• First Posted: May 15, 2026
• Study Start (Estimated): July 1, 2026
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): August 1, 2026
• Last Updated: May 15, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)