HMB Cerebral Palsy Pilot Study

NCT05384951 | Active Not Recruiting

• 1 other identifier: STUDY00015262
• Study Type: interventional
• Target: 7
• Locations: 1 country
• Sites: 1

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.

Conditions

Cerebral Palsy

Keywords

HMB; Vitamin D; Safety; Compliance; Strength; Muscle Mass; Mobility; β-hydroxy-β-methylbutyrate; Calcium β-hydroxy-β-methylbutyrate

Outcome Measures

Primary Outcomes (14)

• Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by renal (kidney) function - specific gravity

  Specific gravity will be measured via urinalysis with microscopy (unitless; ratio of urine density \[g/cm\^3\] divided by density of pure water).

  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

  pH will be measured via urinalysis with microscopy (usually presented unitless; moles H+ per liter).

  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

  Molecular concentrations in urine will be measured via urinalysis with microscopy. The following molecular concentrations will be measured: total protein, glucose, ketones, blood, bilirubin, and urobilinogen (all units: unitless).

  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

  Blood urea nitrogen (BUN; units: mg/dL) will be measured using a blood sample.

  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

  Creatinine will be measured using a blood sample. It will be used to estimate glomerular filtration rate (mL/min/m\^2 body surface area).

  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

  Hepatic enzyme function will be measured with a blood sample. Outcomes include alkaline phosphatase \[ALP\], aspartate aminotransferase \[AST\], alanine aminotransferase \[ALT\] (all units: units per liter).

  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

  Hepatic function will be measured with a blood sample. Outcomes of interest include bilirubin, albumin, and total protein (all units: g/dL).

  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

  Adverse events will be recorded using the NIH's Adverse Events Form, ver 2.

  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

  Common complaints of major organ system experienced over the last 3 days will be self-reported as present or not present for: stomachache, nausea, dizziness, coughing, wheezing, chest pain, weakness, increased headache, negative mood, rash, dry scalp, dry skin, nail changes, ear pain, decreased memory, itching, swelling, diarrhea, stiff joints, nose bleeds, heart burn, numbness, nasal congestion, ringing in ears, increased stress, decreased libido, constipation, shortness of breath, loss of appetite, loss of energy, blood in urine, \& blood in stool.

  Pre-supplementation (12 wks), post-supplementation (12 wks)

• Ability to comply with HMB supplementation as assessed by a daily diary & compliance check-ins

  Participants will complete a daily paper or electronic diary to document taking their supplement. Compliance checks will be conducted by the study staff via a call or email. Unused supplements will be counted at the end of the study. Compliance will be calculated as a percent (# of pills taken on time/total # of pills that should have been taken) x 100.

  Post-supplementation (12 wks)

• Ability to swallow HMB supplement as assessed by the PILL-5 survey

  The PILL-5 survey is a 5 question survey that measures physical (e.g., pill sticks in my throat) and emotional (e.g., I have a fear of swallowing pills) swallowing ability. It will be self-reported using a 5-pt Likert scale (never, almost never, sometimes, almost always, always). A total score is calculated (range 0-20, with 20 representing maximum pill dysphasia).

  Week 1 of supplementation

• Palatability of HMB supplement as assessed by the visual 5 faces hedonic scale

  Whether participants like or dislike the taste of the supplement will be measured with a 5 faces hedonic scale with the numerical anchors ranging from 1 to 5 (best) and text anchors: dislike a lot; dislike a little; neither like nor dislike; like a little; like a lot.

  Week 1 of supplementation

• Satisfaction of supplement dose volume as assessed by survey

  A question will measure if participants felt the dose volume (number of tablets) were acceptable (yes or no).

  Week 12 of supplementation

• Difference in satisfaction of supplement dose frequency as assessed by survey

  A question will measure if participants felt the frequency (2 times per day) was acceptable (yes or no).

  Week 12 of supplementation

Secondary Outcomes (5)

• 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)

Study Arms (1)

HMB + Vitamin D3 Supplement

EXPERIMENTAL

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.

Dietary Supplement: HMB + Vitamin D3

Interventions

HMB + Vitamin D3 DIETARY_SUPPLEMENT

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.

HMB + Vitamin D3 Supplement

Eligibility Criteria

• Age: 13 Years - 17 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17)

You may qualify if:

• Diagnosed with cerebral palsy
• Spastic or mixed tone
• GMFCS Level I-III (i.e., ambulatory)
• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• Gillette Children's Specialty Healthcare: lead
• University of Minnesota: collaborator
• Metabolic Technologies, LLC: collaborator

Study Sites (1)

Gillette Children's Specialty Healthcare

Saint Paul, Minnesota, 55101, United States

Location

MeSH Terms

Conditions

Cerebral Palsy; Patient Compliance

Interventions

Cholecalciferol

Condition Hierarchy (Ancestors)

Brain Damage, Chronic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Patient Acceptance of Health Care; Treatment Adherence and Compliance; Health Behavior; Behavior

Intervention Hierarchy (Ancestors)

Cholestenes; Cholestanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Sterols; Vitamin D; Secosteroids; Membrane Lipids; Lipids

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Clinical Scientist

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

Study Record Dates

• First Submitted: May 2, 2022
• First Posted: May 23, 2022
• Study Start: May 15, 2022
• Primary Completion: May 31, 2024
• Study Completion: May 31, 2024
• Last Updated: February 22, 2024

Record last verified: 2024-02

Locations

US (1)