Cerebral PalsyNCT05384951
HMB Cerebral Palsy Pilot Study
A study testing HMB + Vitamin D3 in people with Cerebral Palsy, run by Gillette Children's Specialty Healthcare at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 13 to 17 yearsHealthy volunteers can take part
- Conditions the study is for
- Cerebral Palsy
- What is being tested?
- HMB + Vitamin D3
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteUnited States
- Study dates
- 15 May 2022to 31 May 2024
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 13 to 17 years
- All sexes
- Healthy volunteers accepted
- Child
Who may be able to join
- 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
Who cannot join
- 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
Show all 9Show fewer
- 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)
Trial locations and contacts
1 site in 1 country.
United States1 site
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, 55101
About this study
Official title: 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.
- Conditions
- Cerebral Palsy
- Being tested
- HMB + Vitamin D3
- Keywords
- HMB, Vitamin D, Safety, Compliance, Strength, Muscle Mass, Mobility, β-hydroxy-β-methylbutyrate, Calcium β-hydroxy-β-methylbutyrate
- Medical subject headings
- Cerebral Palsy, Patient Compliance
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HMB + Vitamin D3Dietary 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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Dietary Supplement: HMB + Vitamin D3 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 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).
Measured: 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).
Measured: 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.
Measured: 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).
Measured: 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).
Measured: 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).
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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).
Measured: 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.
Measured: 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).
Measured: 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).
Measured: Week 12 of supplementation
Secondary outcomes (5)
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).
Measured: Pre-supplementation (12 wks), post-supplementation (12 wks)
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.
Measured: Pre-supplementation (12 wks), post-supplementation (12 wks)
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.
Measured: Pre-supplementation (12 wks), post-supplementation (12 wks)
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.
Measured: Pre-supplementation (12 wks), post-supplementation (12 wks)
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.
Measured: Pre-supplementation (12 wks), post-supplementation (12 wks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 May 2022
- Study start15 May 2022
- Primary completion31 May 2024 (estimated)
- Study completion31 May 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cerebral PalsyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- 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)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gillette Children's Specialty Healthcare
- Sponsor type
- University, hospital or other
- Collaborators
- University of Minnesota, Metabolic Technologies, LLC
- Responsible party
- Principal investigator, Elizabeth Boyer, Gillette Children's Specialty Healthcare
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05384951
- Sponsor's protocol ID
- STUDY00015262
- Status last verified
- Feb 2024
- First submitted
- 2 May 2022
- Last updated
- 22 Feb 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- β-hydroxy-β-methylbutyrate (HMB) Pilot Feasibility and Efficacy Study in Cerebral Palsy (CP)
- ClinicalTrials.gov ID
- NCT05384951
- Acronym / sponsor ID
- STUDY00015262
- Conditions
- Cerebral Palsy
- Keywords
- HMB, Vitamin D, Safety, Compliance, Strength, Muscle Mass, Mobility, β-hydroxy-β-methylbutyrate
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Gillette Children's Specialty HealthcareUniversity, hospital or other
- Collaborators
- University of Minnesota, Metabolic Technologies, LLC
- Study start
- 15 May 2022 (actual)
- Primary completion
- 31 May 2024 (estimated)
- Study completion
- 31 May 2024 (estimated)
- First posted
- 23 May 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Single group repeated measures design with 12 weeks of no intervention (control period) followed by 12 weeks of intervention.
| Arm | Type | Receives |
|---|---|---|
| HMB + Vitamin D3 Supplement | Experimental | Dietary Supplement: HMB + Vitamin D3Supplement 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. |
Who may qualify
- Ages
- 13 to 17 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (9 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HMB + Vitamin D3The 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. | Dietary supplement | HMB + Vitamin D3 Supplement |
Primary outcome
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)
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
Time frame: Pre-supplementation (12 wks), post-supplementation (12 wks)
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
Time frame: Pre-supplementation (12 wks), post-supplementation (12 wks)
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).
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)
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
Time frame: Pre-supplementation (12 wks), post-supplementation (12 wks)
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
Time frame: Pre-supplementation (12 wks), post-supplementation (12 wks)
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
Time frame: Pre-supplementation (12 wks), post-supplementation (12 wks)
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
Time frame: Pre-supplementation (12 wks), post-supplementation (12 wks)
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
Time frame: Pre-supplementation (12 wks), post-supplementation (12 wks)
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
Time frame: Post-supplementation (12 wks)
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
Time frame: Week 1 of supplementation
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
Time frame: Week 1 of supplementation
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
Time frame: Week 12 of supplementation
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
Time frame: Week 12 of supplementation
A question will measure if participants felt the frequency (2 times per day) was acceptable (yes or no).
5 secondary outcomes
Change in lower extremity strength with supplementation as assessed using a Biodex isokinetic system
Time frame: Pre-supplementation (12 wks), post-supplementation (12 wks)
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)
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)
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)
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)
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Feb 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing HMB + Vitamin D3 in people with Cerebral Palsy, run by Gillette Children's Specialty Healthcare at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 22 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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)). The study lists 14 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Feb 2024.