β-hydroxy-β-methylbutyrate (HMB) Pilot Feasibility and Efficacy Study in Cerebral Palsy (CP)
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 7
- 试验地点
- 2
- 主要终点
- Difference in satisfaction of supplement dose frequency as assessed by survey
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 13 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •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
排除标准
- •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)
研究组 & 干预措施
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.
干预措施: HMB + Vitamin D3 (Dietary Supplement)
结局指标
主要结局
Difference in satisfaction of supplement dose frequency as assessed by survey
时间窗: Week 12 of supplementation
A question will measure if participants felt the frequency (2 times per day) was acceptable (yes or no).
Difference in the incidence of Treatment-Emergent Adverse Events before and after supplementation as assessed by adverse events form
时间窗: Pre-supplementation (12 wks), post-supplementation (12 wks)
Adverse events will be recorded using the NIH's Adverse Events Form, ver 2.
Palatability of HMB supplement as assessed by the visual 5 faces hedonic scale
时间窗: 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.
Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by renal (kidney) function - pH
时间窗: 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
时间窗: 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
时间窗: 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
时间窗: 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 before and after supplementation as assessed by checklist of changes to major organ systems
时间窗: 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.
Difference in the incidence of Treatment-Emergent Adverse Events with supplementation as assessed by renal (kidney) function - specific gravity
时间窗: 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 hepatic (liver) function - enzymes
时间窗: 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
时间窗: 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).
Ability to comply with HMB supplementation as assessed by a daily diary & compliance check-ins
时间窗: 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
时间窗: 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).
Satisfaction of supplement dose volume as assessed by survey
时间窗: Week 12 of supplementation
A question will measure if participants felt the dose volume (number of tablets) were acceptable (yes or no).
次要结局
- 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 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 6 minute walk test (6MWT)(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 lower extremity strength with supplementation as assessed using a Biodex isokinetic system(Pre-supplementation (12 wks), post-supplementation (12 wks))
研究者
Elizabeth Boyer
Clinical Scientist
Gillette Children's Specialty Healthcare