Clinical trial NCT05384951: HMB Cerebral Palsy Pilot Study
Official title: β-hydroxy-β-methylbutyrate (HMB) Pilot Feasibility and Efficacy Study in Cerebral Palsy (CP)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: NA
Conditions: Cerebral Palsy
Keywords: HMB
Vitamin D
Safety
Compliance
Strength
Muscle Mass
Mobility
β-hydroxy-β-methylbutyrate
Calcium β-hydroxy-β-methylbutyrate
Lead sponsor: Gillette Children's Specialty Healthcare
Sponsor class: OTHER
Collaborators: name: University of Minnesota
name: Metabolic Technologies, LLC
First posted: 2022-05-23
Enrollment: 7
Sex: ALL
Minimum age (years): 13
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: yes
Allocation: NA
Intervention model: SINGLE_GROUP
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

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.


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)

Primary outcomes:
measure: Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by renal (kidney) function - specific gravity
timeFrame: Pre-supplementation (12 wks), post-supplementation (12 wks)
measure: Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by renal (kidney) function - pH
timeFrame: Pre-supplementation (12 wks), post-supplementation (12 wks)
measure: Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by renal (kidney) function - microscopy
timeFrame: Pre-supplementation (12 wks), post-supplementation (12 wks)
measure: Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by renal (kidney) function - BUN
timeFrame: Pre-supplementation (12 wks), post-supplementation (12 wks)
measure: Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by renal (kidney) function - creatinine
timeFrame: Pre-supplementation (12 wks), post-supplementation (12 wks)
measure: Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by hepatic (liver) function - enzymes
timeFrame: Pre-supplementation (12 wks), post-supplementation (12 wks)
measure: Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by hepatic (liver) function
timeFrame: Pre-supplementation (12 wks), post-supplementation (12 wks)
measure: Difference in the incidence of Treatment-Emergent Adverse Events before and after supplementation as assessed by adverse events form
timeFrame: Pre-supplementation (12 wks), post-supplementation (12 wks)
measure: Difference in the incidence of Treatment-Emergent Adverse Events before and after supplementation as assessed by checklist of changes to major organ systems
timeFrame: Pre-supplementation (12 wks), post-supplementation (12 wks)
measure: Ability to comply with HMB supplementation as assessed by a daily diary & compliance check-ins
timeFrame: Post-supplementation (12 wks)
measure: Ability to swallow HMB supplement as assessed by the PILL-5 survey
timeFrame: Week 1 of supplementation
measure: Palatability of HMB supplement as assessed by the visual 5 faces hedonic scale
timeFrame: Week 1 of supplementation
measure: Satisfaction of supplement dose volume as assessed by survey
timeFrame: Week 12 of supplementation
measure: Difference in satisfaction of supplement dose frequency as assessed by survey
timeFrame: Week 12 of supplementation
Secondary outcomes:
measure: Change in lower extremity strength with supplementation as assessed using a Biodex isokinetic system
timeFrame: Pre-supplementation (12 wks), post-supplementation (12 wks)
measure: Change in muscle mass with supplementation as assessed by dual-energy x-ray absorptiometry (DXA)
timeFrame: Pre-supplementation (12 wks), post-supplementation (12 wks)
measure: Change in functional mobility with supplementation as assessed by the 10-meter walk test (10MWT)
timeFrame: Pre-supplementation (12 wks), post-supplementation (12 wks)
measure: Change in functional mobility with supplementation as assessed by the Timed-up-and-go test (TUG)
timeFrame: Pre-supplementation (12 wks), post-supplementation (12 wks)
measure: Change in functional mobility with supplementation as assessed by the 6 minute walk test (6MWT)
timeFrame: Pre-supplementation (12 wks), post-supplementation (12 wks)
Other outcomes:
Locations:
city: Saint Paul
country: United States
facility: Gillette Children's Specialty Healthcare
state: Minnesota
Study officials:
References: