A Widely Inclusive, Hybrid-Decentralized Pilot Trial Utilizing β-hydroxy-β-methylbutyrate to Lower IGFBP7 Levels in People With ALS
Duke University
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.
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 sl…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Male or female aged at least 18 years. 2. Sporadic or familial ALS diagnosed as per Gold Coast Criteria (37). 3. Patient is able to understand and express informed consent (in the opinion of the site investigator). 4. Patient is able to read and write English. 5. Patient is expected to survive for the duration of the trial. 6. 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) 7. Women must not be able to become pregnant (e.g., post-menopausal, su…
Interventions
- Drugβ-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.
Locations (2)
- Duke University Medical CenterDurham, North Carolina
- Temple UniversityPhiladelphia, Pennsylvania