Amyotrophic Lateral Sclerosis ALSPhase 1ROAR HMBNCT07589764
Pilot Trial Utilizing β-hydroxy-β-methylbutyrate to Lower IGFBP7 Levels in People With ALS
A Phase 1 study testing β-hydroxy-β-methylbutyrate (HMB) in people with Amyotrophic Lateral Sclerosis ALS, run by Duke University at 2 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis ALS
- What is being tested?
- β-hydroxy-β-methylbutyrate (HMB)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 2 sitesUnited States
- Study dates
- 1 Oct 2026to 1 Oct 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites
Duke University Medical Center
Durham, North Carolina, 27705
Richard Bedlack, MD. PhD, alsresearch@duke.edu
Richard Bedlack, MD, PhD
Temple University
Philadelphia, Pennsylvania, 19122
John Furey, john.furey0001@temple.edu
Christopher Pizzica, christopher.pizzica@temple.edu
About this study
Official title: A Widely Inclusive, Hybrid-Decentralized Pilot Trial Utilizing β-hydroxy-β-methylbutyrate to Lower IGFBP7 Levels in People With ALS
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.
Read the detailed description
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.
- Conditions
- Amyotrophic Lateral Sclerosis ALS
- Being tested
- β-hydroxy-β-methylbutyrate (HMB)
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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.
| Study group | Role | What they receive |
|---|---|---|
| β-hydroxy-β-methylbutyrate (HMB) administration | Experimental | Not described Receives: Drug: β-hydroxy-β-methylbutyrate (HMB) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: Baseline, month 3, month 9
Secondary outcomes (5)
Neurofilament Light Chain levels (blood)
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.
Measured: Baseline, month 3, and month 6
Neurofilament Light Chain levels (Cerebrospinal fluid (CSF))
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.
Measured: Baseline, month 3
Insulin-like growth-factor binding protein 7 (IGFBP7) levels (blood)
Non-competitive inhibitor of the IGF-1 receptor
Measured: Baseline, month 3 and month 6
Insulin-like growth-factor binding protein 7 (IGFBP7) levels (Cerebrospinal fluid (CSF))
Non-competitive inhibitor of the IGF-1 receptor
Measured: Baseline, month 3
Frequency of ALS reversals
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.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 May 2026
- Study start1 Oct 2026 (estimated)
- Primary completion1 Oct 2027 (estimated)
- Study completion1 Oct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral Sclerosis ALSStudies of the same condition, open and under way.
- More trials run by Duke UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- 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).
- Masking
- None (open label)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Duke University
- Sponsor type
- University, hospital or other
- Collaborators
- Temple University
- Responsible party
- Sponsor
- Investigators
- Richard Bedlack, MD, PhD, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07589764
- Sponsor's protocol ID
- Pro00120054
- Acronym
- ROAR HMB
- Status last verified
- Sep 2026
- First submitted
- 11 May 2026
- Last updated
- 8 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Widely Inclusive, Hybrid-Decentralized Pilot Trial Utilizing β-hydroxy-β-methylbutyrate to Lower IGFBP7 Levels in People With ALS
- ClinicalTrials.gov ID
- NCT07589764
- Acronym / sponsor ID
- ROAR HMB / Pro00120054
- Conditions
- Amyotrophic Lateral Sclerosis ALS
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Duke UniversityUniversity, hospital or other
- Collaborators
- Temple University
- Study start
- 1 Oct 2026 (estimated)
- Primary completion
- 1 Oct 2027 (estimated)
- Study completion
- 1 Oct 2027 (estimated)
- First posted
- 15 May 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- 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).
| Arm | Type | Receives |
|---|---|---|
| β-hydroxy-β-methylbutyrate (HMB) administration | Experimental | Drug: β-hydroxy-β-methylbutyrate (HMB) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| β-hydroxy-β-methylbutyrate (HMB)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. | Drug | β-hydroxy-β-methylbutyrate (HMB) administration |
Primary outcome
ALS Functional Rating Scale, Revised (ALSFRS-R)
Time frame: 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)
Time frame: 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.
5 secondary outcomes
Neurofilament Light Chain levels (blood)
Time frame: Baseline, month 3, and month 6
Neurofilament Light Chain levels (Cerebrospinal fluid (CSF))
Time frame: Baseline, month 3
Insulin-like growth-factor binding protein 7 (IGFBP7) levels (blood)
Time frame: Baseline, month 3 and month 6
Insulin-like growth-factor binding protein 7 (IGFBP7) levels (Cerebrospinal fluid (CSF))
Time frame: Baseline, month 3
Frequency of ALS reversals
Time frame: 6 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Richard Bedlack, MD, PhD principal investigator
Duke University
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing β-hydroxy-β-methylbutyrate (HMB) in people with Amyotrophic Lateral Sclerosis ALS, run by Duke University at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 8 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Richard Bedlack, MD, PhD (919-613-2681, alsresearch@duke.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
ALS Functional Rating Scale, Revised (ALSFRS-R) (time frame: Baseline, month 3, month 9). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Sep 2026.