Back to resultsHMB Cerebral Palsy Pilot Study
Primary Purpose
Cerebral Palsy
Status
Enrolling by invitation
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
HMB + Vitamin D3
Sponsored by
About this trial
This is an interventional other trial for Cerebral Palsy focused on measuring HMB, Vitamin D, Safety, Compliance, Strength, Muscle Mass, Mobility, β-hydroxy-β-methylbutyrate, Calcium β-hydroxy-β-methylbutyrate
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)
Sites / Locations
- Gillette Children's Specialty Healthcare
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
HMB + Vitamin D3 Supplement
Arm 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.
Outcomes
Primary Outcome Measures
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).
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).
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: mg/dL).
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.
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).
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).
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).
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.
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.
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.
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).
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.
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).
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).
Secondary Outcome Measures
Change in lower extremity strength with supplementation as assessed using a Biodex isokinetic system
Isokinetic dynamometry will be used to measure peak torque of hip extensors & flexors, knee extensors & flexors, and ankle plantarflexors and dorsiflexors (Newton-meters/body mass).
Change in muscle mass with supplementation as assessed by dual-energy x-ray absorptiometry (DXA)
Skeletal muscle mass (kg) will be measured using a whole body DXA scan.
Change in functional mobility with supplementation as assessed by the 10-meter walk test (10MWT)
The 10MWT will be performed at the participant's fastest walking speed over a 14-m walkway, with 2-m on each end for acceleration and deceleration. Time to travel the middle 10-m will be recorded by stopwatch and average speed (m/s) calculated. Usual orthoses and assistive devices will be permitted and used at each repeat assessment.
Change in functional mobility with supplementation as assessed by the Timed-up-and-go test (TUG)
The TUG will be performed at self-selected speed using a standard height chair with arms. Participants will stand up, walk 3-m, and return to their seat. Time (seconds) will be measured. Usual orthoses and assistive devices will be permitted and used at each repeat assessment.
Change in functional mobility with supplementation as assessed by the 6 minute walk test (6MWT)
The 6MWT will be performed at the participant's fastest walking speed on an indoor, oval walking path. Distance travelled (m) will be recorded and average speed (m/s) calculated. Usual orthoses and assistive devices will be permitted and used at each repeat assessment.
Full Information
NCT ID
NCT05384951
First Posted
May 2, 2022
Last Updated
August 31, 2023
Sponsor
Gillette Children's Specialty Healthcare
Collaborators
University of Minnesota, Metabolic Technologies, LLC
1. Study Identification
Unique Protocol Identification Number
NCT05384951
Brief Title
HMB Cerebral Palsy Pilot Study
Official Title
β-hydroxy-β-methylbutyrate (HMB) Pilot Feasibility and Efficacy Study in Cerebral Palsy (CP)
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
May 15, 2022 (Actual)
Primary Completion Date
May 31, 2024 (Anticipated)
Study Completion Date
May 31, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Gillette Children's Specialty Healthcare
Collaborators
University of Minnesota, Metabolic Technologies, LLC
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
Yes
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cerebral Palsy
Keywords
HMB, Vitamin D, Safety, Compliance, Strength, Muscle Mass, Mobility, β-hydroxy-β-methylbutyrate, Calcium β-hydroxy-β-methylbutyrate
7. Study Design
Primary Purpose
Other
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Model Description
Single group repeated measures design with 12 weeks of no intervention (control period) followed by 12 weeks of intervention.
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
HMB + Vitamin D3 Supplement
Arm Type
Experimental
Arm 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.
Intervention Type
Dietary Supplement
Intervention Name(s)
HMB + Vitamin D3
Intervention 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.
Primary Outcome Measure Information:
Title
Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by renal (kidney) function - specific gravity
Description
Specific gravity will be measured via urinalysis with microscopy (unitless; ratio of urine density [g/cm^3] divided by density of pure water).
Time Frame
Pre-supplementation (12 wks), post-supplementation (12 wks)
Title
Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by renal (kidney) function - pH
Description
pH will be measured via urinalysis with microscopy (usually presented unitless; moles H+ per liter).
Time Frame
Pre-supplementation (12 wks), post-supplementation (12 wks)
Title
Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by renal (kidney) function - microscopy
Description
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: mg/dL).
Time Frame
Pre-supplementation (12 wks), post-supplementation (12 wks)
Title
Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by renal (kidney) function - BUN
Description
Blood urea nitrogen (BUN; units: mg/dL) will be measured using a blood sample.
Time Frame
Pre-supplementation (12 wks), post-supplementation (12 wks)
Title
Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by renal (kidney) function - creatinine
Description
Creatinine will be measured using a blood sample. It will be used to estimate glomerular filtration rate (mL/min/m^2 body surface area).
Time Frame
Pre-supplementation (12 wks), post-supplementation (12 wks)
Title
Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by hepatic (liver) function - enzymes
Description
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).
Time Frame
Pre-supplementation (12 wks), post-supplementation (12 wks)
Title
Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by hepatic (liver) function
Description
Hepatic function will be measured with a blood sample. Outcomes of interest include bilirubin, albumin, and total protein (all units: g/dL).
Time Frame
Pre-supplementation (12 wks), post-supplementation (12 wks)
Title
Difference in the incidence of Treatment-Emergent Adverse Events before and after supplementation as assessed by adverse events form
Description
Adverse events will be recorded using the NIH's Adverse Events Form, ver 2.
Time Frame
Pre-supplementation (12 wks), post-supplementation (12 wks)
Title
Difference in the incidence of Treatment-Emergent Adverse Events before and after supplementation as assessed by checklist of changes to major organ systems
Description
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.
Time Frame
Pre-supplementation (12 wks), post-supplementation (12 wks)
Title
Ability to comply with HMB supplementation as assessed by a daily diary & compliance check-ins
Description
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.
Time Frame
Post-supplementation (12 wks)
Title
Ability to swallow HMB supplement as assessed by the PILL-5 survey
Description
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).
Time Frame
Week 1 of supplementation
Title
Palatability of HMB supplement as assessed by the visual 5 faces hedonic scale
Description
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.
Time Frame
Week 1 of supplementation
Title
Satisfaction of supplement dose volume as assessed by survey
Description
A question will measure if participants felt the dose volume (number of tablets) were acceptable (yes or no).
Time Frame
Week 12 of supplementation
Title
Difference in satisfaction of supplement dose frequency as assessed by survey
Description
A question will measure if participants felt the frequency (2 times per day) was acceptable (yes or no).
Time Frame
Week 12 of supplementation
Secondary Outcome Measure Information:
Title
Change in lower extremity strength with supplementation as assessed using a Biodex isokinetic system
Description
Isokinetic dynamometry will be used to measure peak torque of hip extensors & flexors, knee extensors & flexors, and ankle plantarflexors and dorsiflexors (Newton-meters/body mass).
Time Frame
Pre-supplementation (12 wks), post-supplementation (12 wks)
Title
Change in muscle mass with supplementation as assessed by dual-energy x-ray absorptiometry (DXA)
Description
Skeletal muscle mass (kg) will be measured using a whole body DXA scan.
Time Frame
Pre-supplementation (12 wks), post-supplementation (12 wks)
Title
Change in functional mobility with supplementation as assessed by the 10-meter walk test (10MWT)
Description
The 10MWT will be performed at the participant's fastest walking speed over a 14-m walkway, with 2-m on each end for acceleration and deceleration. Time to travel the middle 10-m will be recorded by stopwatch and average speed (m/s) calculated. Usual orthoses and assistive devices will be permitted and used at each repeat assessment.
Time Frame
Pre-supplementation (12 wks), post-supplementation (12 wks)
Title
Change in functional mobility with supplementation as assessed by the Timed-up-and-go test (TUG)
Description
The TUG will be performed at self-selected speed using a standard height chair with arms. Participants will stand up, walk 3-m, and return to their seat. Time (seconds) will be measured. Usual orthoses and assistive devices will be permitted and used at each repeat assessment.
Time Frame
Pre-supplementation (12 wks), post-supplementation (12 wks)
Title
Change in functional mobility with supplementation as assessed by the 6 minute walk test (6MWT)
Description
The 6MWT will be performed at the participant's fastest walking speed on an indoor, oval walking path. Distance travelled (m) will be recorded and average speed (m/s) calculated. Usual orthoses and assistive devices will be permitted and used at each repeat assessment.
Time Frame
Pre-supplementation (12 wks), post-supplementation (12 wks)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
13 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
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)
Facility Information:
Facility Name
Gillette Children's Specialty Healthcare
City
Saint Paul
State/Province
Minnesota
ZIP/Postal Code
55101
Country
United States
12. IPD Sharing Statement
Learn more about this trial
HMB Cerebral Palsy Pilot Study
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