HMB Cerebral Palsy Pilot Study

β-hydroxy-β-methylbutyrate (HMB) Pilot Feasibility and Efficacy Study in Cerebral Palsy (CP)

Sponsors

Lead Sponsor: Gillette Children's Specialty Healthcare

Collaborator: University of Minnesota
Metabolic Technologies, LLC

Source Gillette Children's Specialty Healthcare
Brief Summary

This is a pilot study of β-hydroxy-β-methylbutyrate (HMB) + Vitamin D3 supplementation in adolescents with cerebral palsy. The primary objective is to quantify safety, compliance, and acceptability of daily combined HMB + Vitamin D3 supplementation for 12 weeks in adolescents with CP. The secondary objective is to quantify changes in lower extremity muscle mass, strength, and functional mobility after daily combined HMB + Vitamin D3 supplementation for 12 weeks.

Overall Status Enrolling by invitation
Start Date 2022-05-15
Completion Date 2024-05-31
Primary Completion Date 2024-05-31
Phase N/A
Study Type Interventional
Primary Outcome
Measure Time Frame
Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by renal (kidney) function - specific gravity Pre-supplementation (12 wks), post-supplementation (12 wks)
Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by renal (kidney) function - pH Pre-supplementation (12 wks), post-supplementation (12 wks)
Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by renal (kidney) function - microscopy Pre-supplementation (12 wks), post-supplementation (12 wks)
Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by renal (kidney) function - BUN Pre-supplementation (12 wks), post-supplementation (12 wks)
Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by renal (kidney) function - creatinine Pre-supplementation (12 wks), post-supplementation (12 wks)
Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by hepatic (liver) function - enzymes Pre-supplementation (12 wks), post-supplementation (12 wks)
Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by hepatic (liver) function Pre-supplementation (12 wks), post-supplementation (12 wks)
Difference in the incidence of Treatment-Emergent Adverse Events before and after supplementation as assessed by adverse events form Pre-supplementation (12 wks), post-supplementation (12 wks)
Difference in the incidence of Treatment-Emergent Adverse Events before and after supplementation as assessed by checklist of changes to major organ systems Pre-supplementation (12 wks), post-supplementation (12 wks)
Ability to comply with HMB supplementation as assessed by a daily diary & compliance check-ins Post-supplementation (12 wks)
Ability to swallow HMB supplement as assessed by the PILL-5 survey Week 1 of supplementation
Palatability of HMB supplement as assessed by the visual 5 faces hedonic scale Week 1 of supplementation
Satisfaction of supplement dose volume as assessed by survey Week 12 of supplementation
Difference in satisfaction of supplement dose frequency as assessed by survey Week 12 of supplementation
Secondary Outcome
Measure Time Frame
Change in lower extremity strength with supplementation as assessed using a Biodex isokinetic system Pre-supplementation (12 wks), post-supplementation (12 wks)
Change in muscle mass with supplementation as assessed by dual-energy x-ray absorptiometry (DXA) Pre-supplementation (12 wks), post-supplementation (12 wks)
Change in functional mobility with supplementation as assessed by the 10-meter walk test (10MWT) Pre-supplementation (12 wks), post-supplementation (12 wks)
Change in functional mobility with supplementation as assessed by the Timed-up-and-go test (TUG) Pre-supplementation (12 wks), post-supplementation (12 wks)
Change in functional mobility with supplementation as assessed by the 6 minute walk test (6MWT) Pre-supplementation (12 wks), post-supplementation (12 wks)
Enrollment 10
Condition
Intervention

Intervention Type: Dietary Supplement

Intervention Name: HMB + Vitamin D3

Description: The supplement will be taken orally twice daily. Participants will take 2 blended HMB + Vitamin D3 tablets in the morning and 2 tablets in the evening for 12 weeks.

Arm Group Label: HMB + Vitamin D3 Supplement

Eligibility

Criteria:

Inclusion Criteria: - Diagnosed with cerebral palsy - Spastic or mixed tone - GMFCS Level I-III (i.e., ambulatory) - 13-17 years old - Physical training level expected to remain relatively constant over the study period - Ability to follow directions, including swallowing multiple pills daily and complying with reproductive risk recommendations (post-menarchal females) - Within reasonable driving distance to the University of Minnesota - Twin Cities - Reads English Exclusion Criteria: - Pregnant, lactating, or trying to become pregnant - Surgery in the past 9 months - Botulinum toxin injections in past 3 months - Selective dorsal rhizotomy in the past 12 months - Upcoming invasive treatment within the study period that may affect strength or functional mobility (e.g., surgery, botulinum toxin injections, intrathecal baclofen pump or dosage change) - Liver disease or liver disorder - Kidney disease or disorder - Prescription drug or nutrition supplement contraindications - Excessive research or medical-related radiation exposure in the past 12 months (approximately 500 mrem or greater)

Gender:

All

Minimum Age:

13 Years

Maximum Age:

17 Years

Healthy Volunteers:

Accepts Healthy Volunteers

Overall Contact Contact information is only displayed when the study is recruiting subjects.
Location
Facility: Gillette Children's Specialty Healthcare
Location Countries

United States

Verification Date

2022-06-01

Responsible Party

Type: Principal Investigator

Investigator Affiliation: Gillette Children's Specialty Healthcare

Investigator Full Name: Elizabeth Boyer

Investigator Title: Clinical Scientist

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 1
Arm Group

Label: HMB + Vitamin D3 Supplement

Type: Experimental

Description: Supplement delivery will be a tablet containing both HMB & Vitamin D3. HMB will be administered in its calcium salt form. One tablet will contain 750 mg HMB + 250 IU of Vitamin D3. The target dosage is 3 g HMB + 1000 IU of Vitamin D3 per day.

Study Design Info

Allocation: N/A

Intervention Model: Single Group Assignment

Intervention Model Description: Single group repeated measures design with 12 weeks of no intervention (control period) followed by 12 weeks of intervention.

Primary Purpose: Other

Masking: None (Open Label)

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