Trial Outcomes & Findings for Bone Health in Pediatric Crohn's Disease: A Low Magnitude Mechanical Stimulus Trial (NCT NCT00364130)

NCT ID: NCT00364130

Last Updated: 2017-04-14

Results Overview

We calculated the mean change in tibia trabecular volumetric BMD Z-score between baseline and 12 months, as measured by peripheral quantitative computed tomography (pQCT). The Z-score, or Standard Deviation Score, is a measure of the number of standard deviations that an individual is above or below the median value in a healthy child or adolescent of the same age, sex and race. For example, a Z-score of 0 means that an individual's result is equivalent to the 50th percentile in a healthy population. A Z-score of -1.0 means that an individual's result is equovalent to the 16th percentile in a healthy population.

• Recruitment status: COMPLETED
• Study phase: NA
• Target enrollment: 138 participants
• Primary outcome timeframe: 12 months
• Results posted on: 2017-04-14

Participant Flow

The recruitment period began May 17, 2007 and ended June 25, 2010. Participants were recruited from the Center for Inflammatory Bowel Disease at CHOP and through referrals from physicians outside CHOP. The study was also advertised on the Crohn's and Colitis Foundation Website.

Patients between the ages of 8-21 years with a diagnosis of Crohn disease for at least 6 months were eligible for a screening visit. Patients meeting the entry criteria (pQCT trabecular volumetric BMD z-score below the 25th percentile)were randomly allocated 1:1 to an active LMMS device or a placebo device.

Participant milestones

Measure	Active Active Low Magnitude Mechanical Stimulus Low magnitude mechanical stimulus : 10 minute daily treatment sessions standing on the low magnitude mechanical stimulus device	Pacebo Placebo (inactive) low magnitude mechanical stimulus : 10 minute daily treatments standing on a placebo version of a low magnitude mechanical stimulus device
Intervention Period STARTED	69	69
Intervention Period COMPLETED	60	61
Intervention Period NOT COMPLETED	9	8
Post Intervention Follow-up Period STARTED	60	61
Post Intervention Follow-up Period COMPLETED	55	50
Post Intervention Follow-up Period NOT COMPLETED	5	11

Reasons for withdrawal

Measure	Active Active Low Magnitude Mechanical Stimulus Low magnitude mechanical stimulus : 10 minute daily treatment sessions standing on the low magnitude mechanical stimulus device	Pacebo Placebo (inactive) low magnitude mechanical stimulus : 10 minute daily treatments standing on a placebo version of a low magnitude mechanical stimulus device
Intervention Period Lost to Follow-up	4	3
Intervention Period Pregnancy	1	0
Intervention Period Withdrawal by Subject	4	5
Post Intervention Follow-up Period Lost to Follow-up	5	11

Baseline Characteristics

Bone Health in Pediatric Crohn's Disease: A Low Magnitude Mechanical Stimulus Trial

Baseline characteristics by cohort

Measure	Active n=69 Participants Active Low Magnitude Mechanical Stimulus Low magnitude mechanical stimulus : 10 minute daily treatment sessions standing on the low magnitude mechanical stimulus device	Pacebo n=69 Participants Placebo (inactive) low magnitude mechanical stimulus : 10 minute daily treatments standing on a placebo version of a low magnitude mechanical stimulus device	Total n=138 Participants Total of all reporting groups
Age, Categorical <=18 years	63 Participants n=5 Participants	63 Participants n=7 Participants	126 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	6 Participants n=5 Participants	6 Participants n=7 Participants	12 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	14.11475 years STANDARD_DEVIATION 2.900821 • n=5 Participants	14.51452 years STANDARD_DEVIATION 2.720447 • n=7 Participants	14.3134 years STANDARD_DEVIATION 2.816848 • n=5 Participants
Sex: Female, Male Female	33 Participants n=5 Participants	33 Participants n=7 Participants	66 Participants n=5 Participants
Sex: Female, Male Male	36 Participants n=5 Participants	36 Participants n=7 Participants	72 Participants n=5 Participants
Region of Enrollment United States	69 participants n=5 Participants	69 participants n=7 Participants	138 participants n=5 Participants

PRIMARY outcome

Timeframe: 12 months

Population: The number was determined by the number of participants with measures at both baseline and 12 month time points. The analysis was intention to treat.

We calculated the mean change in tibia trabecular volumetric BMD Z-score between baseline and 12 months, as measured by peripheral quantitative computed tomography (pQCT).

The Z-score, or Standard Deviation Score, is a measure of the number of standard deviations that an individual is above or below the median value in a healthy child or adolescent of the same age, sex and race. For example, a Z-score of 0 means that an individual's result is equivalent to the 50th percentile in a healthy population. A Z-score of -1.0 means that an individual's result is equovalent to the 16th percentile in a healthy population.

Outcome measures

Measure	Active n=60 Participants Active Low Magnitude Mechanical Stimulus Low magnitude mechanical stimulus : 10 minute daily treatment sessions standing on the low magnitude mechanical stimulus device	Pacebo n=61 Participants Placebo (inactive) low magnitude mechanical stimulus : 10 minute daily treatments standing on a placebo version of a low magnitude mechanical stimulus device
Change in Tibia Trabecular Volumetric Bone Mineral Density (BMD) Z-score at 12 Months	0.15 Z-score Standard Deviation 0.55	0.23 Z-score Standard Deviation 0.70

PRIMARY outcome

Timeframe: 12 months

Population: The number was determined by the number of participants with measures at both baseline and 12 month time points. The analysis was intention to treat.

We calculated the mean change in tibia cortical area Z-score, as measured by pQCT, between baseline and 12 months.

Outcome measures

Measure	Active n=60 Participants Active Low Magnitude Mechanical Stimulus Low magnitude mechanical stimulus : 10 minute daily treatment sessions standing on the low magnitude mechanical stimulus device	Pacebo n=61 Participants Placebo (inactive) low magnitude mechanical stimulus : 10 minute daily treatments standing on a placebo version of a low magnitude mechanical stimulus device
Change in Tibia Cortical Area Z-score 12 Months	0.17 Z-score Standard Deviation 0.58	0.20 Z-score Standard Deviation 0.59

PRIMARY outcome

Timeframe: 12 months

Population: The number was determined by the number of participants with measures at both baseline and 12 month time points. The analysis was intention to treat.

We calculated the mean change in spine volumetric BMD Z-score, as measured by QCT, between baseline and 12 months

Outcome measures

Measure	Active n=60 Participants Active Low Magnitude Mechanical Stimulus Low magnitude mechanical stimulus : 10 minute daily treatment sessions standing on the low magnitude mechanical stimulus device	Pacebo n=61 Participants Placebo (inactive) low magnitude mechanical stimulus : 10 minute daily treatments standing on a placebo version of a low magnitude mechanical stimulus device
Change in Spine Volumetric BMD Z-score at 12 Months	0.22 Z-score Standard Deviation 0.59	-0.02 Z-score Standard Deviation 0.67

SECONDARY outcome

Timeframe: 12 months

We calculated the mean change in posterior anterior lumbar spine areal BMD Z-score between baseline and 12 months as measured by DXA

Outcome measures

Measure	Active n=60 Participants Active Low Magnitude Mechanical Stimulus Low magnitude mechanical stimulus : 10 minute daily treatment sessions standing on the low magnitude mechanical stimulus device	Pacebo n=61 Participants Placebo (inactive) low magnitude mechanical stimulus : 10 minute daily treatments standing on a placebo version of a low magnitude mechanical stimulus device
Change in Posteroanterior Lumbar Spine Areal BMD Z-score	0.20 Z-score Standard Deviation 0.44	0.09 Z-score Standard Deviation 0.44

SECONDARY outcome

Timeframe: 12months

We calculated the mean change in total hip bone mineral density z-score, as measured by DXA, between baseline and 12 months

Outcome measures

Measure	Active n=60 Participants Active Low Magnitude Mechanical Stimulus Low magnitude mechanical stimulus : 10 minute daily treatment sessions standing on the low magnitude mechanical stimulus device	Pacebo n=61 Participants Placebo (inactive) low magnitude mechanical stimulus : 10 minute daily treatments standing on a placebo version of a low magnitude mechanical stimulus device
Change in Total Hip Areal BMD Z-score Between Baseline and 12 Months	0.24 Z-score Standard Deviation 0.44	0.25 Z-score Standard Deviation 0.43

SECONDARY outcome

Timeframe: 12 months

We calculated the mean change in femoral neck areal bmd Z-score between baseline and 12 months as measured by DXA

Outcome measures

Measure	Active n=60 Participants Active Low Magnitude Mechanical Stimulus Low magnitude mechanical stimulus : 10 minute daily treatment sessions standing on the low magnitude mechanical stimulus device	Pacebo n=61 Participants Placebo (inactive) low magnitude mechanical stimulus : 10 minute daily treatments standing on a placebo version of a low magnitude mechanical stimulus device
Change in Femoral Neck Areal BMD Z-score Between Baseline and 12 Months	0.19 Z-score Standard Deviation 0.45	0.22 Z-score Standard Deviation 0.45

SECONDARY outcome

Timeframe: 12 months

We calculated the mean change in whole body bone mineral content Z-score, as measured by DXA, between baseline and 12 months

Outcome measures

Measure	Active n=60 Participants Active Low Magnitude Mechanical Stimulus Low magnitude mechanical stimulus : 10 minute daily treatment sessions standing on the low magnitude mechanical stimulus device	Pacebo n=61 Participants Placebo (inactive) low magnitude mechanical stimulus : 10 minute daily treatments standing on a placebo version of a low magnitude mechanical stimulus device
Change in Whole Body Bone Mineral Content Z-score Between Baseline and 12 Months	0.20 Z-score Standard Deviation 0.40	0.18 Z-score Standard Deviation 0.43

SECONDARY outcome

Timeframe: 12 months

We calculated the mean change in tibia trabecular volumetric BMDbetween baseline and 12 months as measured by (QCT)

Outcome measures

Measure	Active n=60 Participants Active Low Magnitude Mechanical Stimulus Low magnitude mechanical stimulus : 10 minute daily treatment sessions standing on the low magnitude mechanical stimulus device	Pacebo n=61 Participants Placebo (inactive) low magnitude mechanical stimulus : 10 minute daily treatments standing on a placebo version of a low magnitude mechanical stimulus device
Change in QCT Tibia Trabecular Volumetric BMD at 12 Months	1.05 cm^3 Standard Deviation 10.5	1.35 cm^3 Standard Deviation 12.93

Adverse Events

Active

Serious events: 7 serious events

Other events: 10 other events

Deaths: 0 deaths

Pacebo

Serious events: 6 serious events

Other events: 10 other events

Deaths: 0 deaths

Serious adverse events

Measure	Active n=69 participants at risk Active Low Magnitude Mechanical Stimulus Low magnitude mechanical stimulus : 10 minute daily treatment sessions standing on the low magnitude mechanical stimulus device	Pacebo n=69 participants at risk Placebo (inactive) low magnitude mechanical stimulus : 10 minute daily treatments standing on a placebo version of a low magnitude mechanical stimulus device
Infections and infestations hospitalization for infection	7.2% 5/69 • Number of events 6 • Adverse event data were collected at 6 months, 1 year and 2 years.	7.2% 5/69 • Number of events 5 • Adverse event data were collected at 6 months, 1 year and 2 years.
Surgical and medical procedures hospitalization for for appendectomy	1.4% 1/69 • Number of events 1 • Adverse event data were collected at 6 months, 1 year and 2 years.	0.00% 0/69 • Adverse event data were collected at 6 months, 1 year and 2 years.
Nervous system disorders Hospitalization for migraine headache	1.4% 1/69 • Number of events 4 • Adverse event data were collected at 6 months, 1 year and 2 years.	0.00% 0/69 • Adverse event data were collected at 6 months, 1 year and 2 years.
Gastrointestinal disorders hospitalization for small bowel overgrowth	0.00% 0/69 • Adverse event data were collected at 6 months, 1 year and 2 years.	1.4% 1/69 • Number of events 1 • Adverse event data were collected at 6 months, 1 year and 2 years.

Other adverse events

Measure	Active n=69 participants at risk Active Low Magnitude Mechanical Stimulus Low magnitude mechanical stimulus : 10 minute daily treatment sessions standing on the low magnitude mechanical stimulus device	Pacebo n=69 participants at risk Placebo (inactive) low magnitude mechanical stimulus : 10 minute daily treatments standing on a placebo version of a low magnitude mechanical stimulus device
Nervous system disorders headache	1.4% 1/69 • Number of events 1 • Adverse event data were collected at 6 months, 1 year and 2 years.	5.8% 4/69 • Number of events 4 • Adverse event data were collected at 6 months, 1 year and 2 years.
Gastrointestinal disorders abdominal pain	5.8% 4/69 • Number of events 4 • Adverse event data were collected at 6 months, 1 year and 2 years.	0.00% 0/69 • Adverse event data were collected at 6 months, 1 year and 2 years.
Gastrointestinal disorders stool changes	7.2% 5/69 • Number of events 5 • Adverse event data were collected at 6 months, 1 year and 2 years.	1.4% 1/69 • Number of events 1 • Adverse event data were collected at 6 months, 1 year and 2 years.
Musculoskeletal and connective tissue disorders lower extremity pain	5.8% 4/69 • Number of events 4 • Adverse event data were collected at 6 months, 1 year and 2 years.	8.7% 6/69 • Number of events 6 • Adverse event data were collected at 6 months, 1 year and 2 years.

Additional Information

Mary Leonard, MD, MSCE

Children's Hospital of Philadelphia

Phone: 215-590-0874

Email: leonard@email.chop.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place