- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05384951
HMB Cerebral Palsy Pilot Study
February 21, 2024 updated by: Elizabeth Boyer, Gillette Children's Specialty Healthcare
β-hydroxy-β-methylbutyrate (HMB) Pilot Feasibility and Efficacy Study in Cerebral Palsy (CP)
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.
Study Overview
Status
Active, not recruiting
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
7
Phase
- Not Applicable
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Minnesota
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Saint Paul, Minnesota, United States, 55101
- Gillette Children's Specialty Healthcare
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
13 years to 17 years (Child)
Accepts Healthy Volunteers
Yes
Description
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)
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Other
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: HMB + Vitamin D3 Supplement
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.
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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.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by renal (kidney) function - specific gravity
Time Frame: Pre-supplementation (12 wks), post-supplementation (12 wks)
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Specific gravity will be measured via urinalysis with microscopy (unitless; ratio of urine density [g/cm^3] divided by density of pure water).
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Pre-supplementation (12 wks), post-supplementation (12 wks)
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Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by renal (kidney) function - pH
Time Frame: Pre-supplementation (12 wks), post-supplementation (12 wks)
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pH will be measured via urinalysis with microscopy (usually presented unitless; moles H+ per liter).
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Pre-supplementation (12 wks), post-supplementation (12 wks)
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Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by renal (kidney) function - BUN
Time Frame: Pre-supplementation (12 wks), post-supplementation (12 wks)
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Blood urea nitrogen (BUN; units: mg/dL) will be measured using a blood sample.
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Pre-supplementation (12 wks), post-supplementation (12 wks)
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Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by renal (kidney) function - creatinine
Time Frame: Pre-supplementation (12 wks), post-supplementation (12 wks)
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Creatinine will be measured using a blood sample.
It will be used to estimate glomerular filtration rate (mL/min/m^2 body surface area).
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Pre-supplementation (12 wks), post-supplementation (12 wks)
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Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by hepatic (liver) function - enzymes
Time Frame: Pre-supplementation (12 wks), post-supplementation (12 wks)
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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).
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Pre-supplementation (12 wks), post-supplementation (12 wks)
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Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by hepatic (liver) function
Time Frame: Pre-supplementation (12 wks), post-supplementation (12 wks)
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Hepatic function will be measured with a blood sample.
Outcomes of interest include bilirubin, albumin, and total protein (all units: g/dL).
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Pre-supplementation (12 wks), post-supplementation (12 wks)
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Difference in the incidence of Treatment-Emergent Adverse Events before and after supplementation as assessed by adverse events form
Time Frame: Pre-supplementation (12 wks), post-supplementation (12 wks)
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Adverse events will be recorded using the NIH's Adverse Events Form, ver 2.
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Pre-supplementation (12 wks), post-supplementation (12 wks)
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Difference in the incidence of Treatment-Emergent Adverse Events before and after supplementation as assessed by checklist of changes to major organ systems
Time Frame: Pre-supplementation (12 wks), post-supplementation (12 wks)
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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.
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Pre-supplementation (12 wks), post-supplementation (12 wks)
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Ability to comply with HMB supplementation as assessed by a daily diary & compliance check-ins
Time Frame: Post-supplementation (12 wks)
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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.
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Post-supplementation (12 wks)
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Ability to swallow HMB supplement as assessed by the PILL-5 survey
Time Frame: Week 1 of supplementation
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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).
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Week 1 of supplementation
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Palatability of HMB supplement as assessed by the visual 5 faces hedonic scale
Time Frame: Week 1 of supplementation
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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.
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Week 1 of supplementation
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Satisfaction of supplement dose volume as assessed by survey
Time Frame: Week 12 of supplementation
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A question will measure if participants felt the dose volume (number of tablets) were acceptable (yes or no).
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Week 12 of supplementation
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Difference in satisfaction of supplement dose frequency as assessed by survey
Time Frame: Week 12 of supplementation
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A question will measure if participants felt the frequency (2 times per day) was acceptable (yes or no).
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Week 12 of supplementation
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Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by renal (kidney) function - microscopy
Time Frame: Pre-supplementation (12 wks), post-supplementation (12 wks)
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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).
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Pre-supplementation (12 wks), post-supplementation (12 wks)
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in lower extremity strength with supplementation as assessed using a Biodex isokinetic system
Time Frame: Pre-supplementation (12 wks), post-supplementation (12 wks)
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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).
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Pre-supplementation (12 wks), post-supplementation (12 wks)
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Change in muscle mass with supplementation as assessed by dual-energy x-ray absorptiometry (DXA)
Time Frame: Pre-supplementation (12 wks), post-supplementation (12 wks)
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Skeletal muscle mass (kg) will be measured using a whole body DXA scan.
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Pre-supplementation (12 wks), post-supplementation (12 wks)
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Change in functional mobility with supplementation as assessed by the 10-meter walk test (10MWT)
Time Frame: Pre-supplementation (12 wks), post-supplementation (12 wks)
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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.
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Pre-supplementation (12 wks), post-supplementation (12 wks)
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Change in functional mobility with supplementation as assessed by the Timed-up-and-go test (TUG)
Time Frame: Pre-supplementation (12 wks), post-supplementation (12 wks)
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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.
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Pre-supplementation (12 wks), post-supplementation (12 wks)
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Change in functional mobility with supplementation as assessed by the 6 minute walk test (6MWT)
Time Frame: Pre-supplementation (12 wks), post-supplementation (12 wks)
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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.
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Pre-supplementation (12 wks), post-supplementation (12 wks)
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
May 15, 2022
Primary Completion (Estimated)
May 31, 2024
Study Completion (Estimated)
May 31, 2024
Study Registration Dates
First Submitted
May 2, 2022
First Submitted That Met QC Criteria
May 18, 2022
First Posted (Actual)
May 23, 2022
Study Record Updates
Last Update Posted (Estimated)
February 22, 2024
Last Update Submitted That Met QC Criteria
February 21, 2024
Last Verified
February 1, 2024
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- STUDY00015262
Drug and device information, study documents
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
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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