Trial Outcomes & Findings for Safety and Efficacy of Xenical in Children and Adolescents With Obesity-Related Diseases (NCT NCT00001723)
NCT ID: NCT00001723
Last Updated: 2012-12-18
Results Overview
Body Mass index standard deviation score calculated for age and sex according to Centers for Disease Control standards. See: Kuczmarski RJ, Ogden CL, Guo SS, Grummer-Strawn LM, Flegal KM, Mei Z et al. 2000 CDC Growth Charts for the United States: methods and development. Vital Health Stat 11 2002; (246): 1-190.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
200 participants
Primary outcome timeframe
baseline to 6 months
Results posted on
2012-12-18
Participant Flow
Accrual for RCT began in 1999 and ended in 2008. All subjects were screened at the NIH Clinical Center.
Most subjects screened but not enrolled (101) did not have an obesity-related comorbid condition (i.e., hypertension or dyslipidemia). 16 met a medical exclusion, and 13 declined participation after undergoing initial evaluation.
Participant milestones
| Measure |
Orlistat
Orlistat 120 mg TID for 6 months plus a behavioral weight loss program
Orlistat : Subjects receive drug for 6 months plus a 12 week intensive behavioral weight los program. Subjects return for monthly visits for 3 more months.
|
Placebo
Matching placebo 120 mg TID x 6 months plus a behavioral weight loss program
Placebo : Subjects receive drug for 6 months plus a 12 week intensive behavioral weight los program. Subjects return for monthly visits for 3 more months.
|
|---|---|---|
|
Overall Study
STARTED
|
100
|
100
|
|
Overall Study
COMPLETED
|
87
|
84
|
|
Overall Study
NOT COMPLETED
|
13
|
16
|
Reasons for withdrawal
| Measure |
Orlistat
Orlistat 120 mg TID for 6 months plus a behavioral weight loss program
Orlistat : Subjects receive drug for 6 months plus a 12 week intensive behavioral weight los program. Subjects return for monthly visits for 3 more months.
|
Placebo
Matching placebo 120 mg TID x 6 months plus a behavioral weight loss program
Placebo : Subjects receive drug for 6 months plus a 12 week intensive behavioral weight los program. Subjects return for monthly visits for 3 more months.
|
|---|---|---|
|
Overall Study
Lack of Efficacy
|
6
|
7
|
|
Overall Study
Lack of family interest
|
4
|
5
|
|
Overall Study
Lost to Follow-up
|
2
|
2
|
|
Overall Study
medication intolerance
|
1
|
2
|
Baseline Characteristics
Safety and Efficacy of Xenical in Children and Adolescents With Obesity-Related Diseases
Baseline characteristics by cohort
| Measure |
Orlistat
n=100 Participants
Orlistat 120 mg TID for 6 months plus a behavioral weight loss program
Orlistat : Subjects receive drug for 6 months plus a 12 week intensive behavioral weight los program. Subjects return for monthly visits for 3 more months.
|
Placebo
n=100 Participants
Matching placebo 120 mg TID x 6 months plus a behavioral weight loss program
Placebo : Subjects receive drug for 6 months plus a 12 week intensive behavioral weight los program. Subjects return for monthly visits for 3 more months.
|
Total
n=200 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
100 Participants
n=5 Participants
|
100 Participants
n=7 Participants
|
200 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 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
|
14.65 years
STANDARD_DEVIATION 1.38 • n=5 Participants
|
14.52 years
STANDARD_DEVIATION 1.46 • n=7 Participants
|
14.59 years
STANDARD_DEVIATION 1.41 • n=5 Participants
|
|
Sex: Female, Male
Female
|
65 Participants
n=5 Participants
|
66 Participants
n=7 Participants
|
131 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
35 Participants
n=5 Participants
|
34 Participants
n=7 Participants
|
69 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
100 participants
n=5 Participants
|
100 participants
n=7 Participants
|
200 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: baseline to 6 monthsPopulation: Multiple imputation analysis
Body Mass index standard deviation score calculated for age and sex according to Centers for Disease Control standards. See: Kuczmarski RJ, Ogden CL, Guo SS, Grummer-Strawn LM, Flegal KM, Mei Z et al. 2000 CDC Growth Charts for the United States: methods and development. Vital Health Stat 11 2002; (246): 1-190.
Outcome measures
| Measure |
Orlistat
n=100 Participants
Orlistat 120 mg TID for 6 months plus a behavioral weight loss program
Orlistat : Subjects receive drug for 6 months plus a 12 week intensive behavioral weight los program. Subjects return for monthly visits for 3 more months.
|
Placebo
n=100 Participants
Matching placebo 120 mg TID x 6 months plus a behavioral weight loss program
Placebo : Subjects receive drug for 6 months plus a 12 week intensive behavioral weight los program. Subjects return for monthly visits for 3 more months.
|
Placebo - Non-Hispanic Blacks
Change in weight for Non-Hispanic Black participants treated with placebo
|
Placebo - Non-Hispanic Whites
Change in weight for Non-Hispanic White participants treated with placebo
|
|---|---|---|---|---|
|
Change in BMI Standard Deviation Score
|
-0.12 Standard Deviation Score
Standard Error 0.02
|
-0.06 Standard Deviation Score
Standard Error 0.02
|
—
|
—
|
SECONDARY outcome
Timeframe: baseline to 6 monthsPopulation: Multiple imputation analysis
Weight in kg
Outcome measures
| Measure |
Orlistat
n=100 Participants
Orlistat 120 mg TID for 6 months plus a behavioral weight loss program
Orlistat : Subjects receive drug for 6 months plus a 12 week intensive behavioral weight los program. Subjects return for monthly visits for 3 more months.
|
Placebo
n=100 Participants
Matching placebo 120 mg TID x 6 months plus a behavioral weight loss program
Placebo : Subjects receive drug for 6 months plus a 12 week intensive behavioral weight los program. Subjects return for monthly visits for 3 more months.
|
Placebo - Non-Hispanic Blacks
Change in weight for Non-Hispanic Black participants treated with placebo
|
Placebo - Non-Hispanic Whites
Change in weight for Non-Hispanic White participants treated with placebo
|
|---|---|---|---|---|
|
Change in Body Weight
|
-2.9 kg
Standard Error 0.7
|
-0.6 kg
Standard Error 0.7
|
—
|
—
|
SECONDARY outcome
Timeframe: baseline to 6 monthsPopulation: Muliple imputation analysis
BMI is calculated in kg/m2. Change from baseline to 6 months of treatment
Outcome measures
| Measure |
Orlistat
n=100 Participants
Orlistat 120 mg TID for 6 months plus a behavioral weight loss program
Orlistat : Subjects receive drug for 6 months plus a 12 week intensive behavioral weight los program. Subjects return for monthly visits for 3 more months.
|
Placebo
n=100 Participants
Matching placebo 120 mg TID x 6 months plus a behavioral weight loss program
Placebo : Subjects receive drug for 6 months plus a 12 week intensive behavioral weight los program. Subjects return for monthly visits for 3 more months.
|
Placebo - Non-Hispanic Blacks
Change in weight for Non-Hispanic Black participants treated with placebo
|
Placebo - Non-Hispanic Whites
Change in weight for Non-Hispanic White participants treated with placebo
|
|---|---|---|---|---|
|
Change in Body Mass Index
|
-1.44 kg per square meter
Standard Error 0.26
|
-0.50 kg per square meter
Standard Error 0.20
|
—
|
—
|
SECONDARY outcome
Timeframe: baseline to 6 monthsPopulation: Multiple Imputation analysis
body fat distribution measures obtained from Dual-energy X-ray Absorptiometry (DEXA)
Outcome measures
| Measure |
Orlistat
n=100 Participants
Orlistat 120 mg TID for 6 months plus a behavioral weight loss program
Orlistat : Subjects receive drug for 6 months plus a 12 week intensive behavioral weight los program. Subjects return for monthly visits for 3 more months.
|
Placebo
n=100 Participants
Matching placebo 120 mg TID x 6 months plus a behavioral weight loss program
Placebo : Subjects receive drug for 6 months plus a 12 week intensive behavioral weight los program. Subjects return for monthly visits for 3 more months.
|
Placebo - Non-Hispanic Blacks
Change in weight for Non-Hispanic Black participants treated with placebo
|
Placebo - Non-Hispanic Whites
Change in weight for Non-Hispanic White participants treated with placebo
|
|---|---|---|---|---|
|
Change in Body Fat (kg)
|
-3.2 kg
Standard Error 0.55
|
-1.7 kg
Standard Error 0.53
|
—
|
—
|
SECONDARY outcome
Timeframe: baseline to 6 monthsPopulation: Multiple imputation analysis
Difference in change of weight in kg according to race (Non-Hispanic White versus Non-Hispanic Black)
Outcome measures
| Measure |
Orlistat
n=63 Participants
Orlistat 120 mg TID for 6 months plus a behavioral weight loss program
Orlistat : Subjects receive drug for 6 months plus a 12 week intensive behavioral weight los program. Subjects return for monthly visits for 3 more months.
|
Placebo
n=37 Participants
Matching placebo 120 mg TID x 6 months plus a behavioral weight loss program
Placebo : Subjects receive drug for 6 months plus a 12 week intensive behavioral weight los program. Subjects return for monthly visits for 3 more months.
|
Placebo - Non-Hispanic Blacks
n=60 Participants
Change in weight for Non-Hispanic Black participants treated with placebo
|
Placebo - Non-Hispanic Whites
n=40 Participants
Change in weight for Non-Hispanic White participants treated with placebo
|
|---|---|---|---|---|
|
Effect of Race on Change in Weight (kg)
|
-2.126 kg
Standard Error 0.812
|
-3.742 kg
Standard Error 1.046
|
0.415 kg
Standard Error 0.835
|
-1.580 kg
Standard Error 1.013
|
Adverse Events
Orlistat
Serious events: 0 serious events
Other events: 95 other events
Deaths: 0 deaths
Placebo
Serious events: 2 serious events
Other events: 94 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Orlistat
n=100 participants at risk
Orlistat 120 mg TID for 6 months plus a behavioral weight loss program Orlistat : Subjects receive drug for 6 months plus a 12 week intensive behavioral weight los program. Subjects return for monthly visits for 3 more months.
|
Placebo
n=100 participants at risk
Matching placebo 120 mg TID x 6 months plus a behavioral weight loss program Placebo : Subjects receive drug for 6 months plus a 12 week intensive behavioral weight los program. Subjects return for monthly visits for 3 more months.
|
|---|---|---|
|
Endocrine disorders
Hypoglycemia - pharmacy error in preparing insulin
|
0.00%
0/100 • 6 months of randomized treatment
|
1.0%
1/100 • 6 months of randomized treatment
|
|
Gastrointestinal disorders
Left lower quadrant pain and vomiting - admitted overnight
|
0.00%
0/100 • 6 months of randomized treatment
|
1.0%
1/100 • 6 months of randomized treatment
|
Other adverse events
| Measure |
Orlistat
n=100 participants at risk
Orlistat 120 mg TID for 6 months plus a behavioral weight loss program Orlistat : Subjects receive drug for 6 months plus a 12 week intensive behavioral weight los program. Subjects return for monthly visits for 3 more months.
|
Placebo
n=100 participants at risk
Matching placebo 120 mg TID x 6 months plus a behavioral weight loss program Placebo : Subjects receive drug for 6 months plus a 12 week intensive behavioral weight los program. Subjects return for monthly visits for 3 more months.
|
|---|---|---|
|
Gastrointestinal disorders
fatty-appearing stools
|
61.0%
61/100 • 6 months of randomized treatment
|
6.0%
6/100 • 6 months of randomized treatment
|
|
Gastrointestinal disorders
flatulence (passage of gas)
|
60.0%
60/100 • 6 months of randomized treatment
|
47.0%
47/100 • 6 months of randomized treatment
|
|
Gastrointestinal disorders
flatus with discharge
|
43.0%
43/100 • 6 months of randomized treatment
|
11.0%
11/100 • 6 months of randomized treatment
|
|
Gastrointestinal disorders
frequent urge for bowel movement
|
19.0%
19/100 • 6 months of randomized treatment
|
3.0%
3/100 • 6 months of randomized treatment
|
|
Ear and labyrinth disorders
ear disorders (otitis, earache, ear pain)
|
7.0%
7/100 • 6 months of randomized treatment
|
7.0%
7/100 • 6 months of randomized treatment
|
|
Eye disorders
eye disorders (change in vision, conjunctivitis, styes)
|
8.0%
8/100 • 6 months of randomized treatment
|
9.0%
9/100 • 6 months of randomized treatment
|
|
Gastrointestinal disorders
abdominal pain or cramping
|
16.0%
16/100 • 6 months of randomized treatment
|
21.0%
21/100 • 6 months of randomized treatment
|
|
Gastrointestinal disorders
bloating or gas
|
18.0%
18/100 • 6 months of randomized treatment
|
5.0%
5/100 • 6 months of randomized treatment
|
|
Gastrointestinal disorders
BORBORYGMI
|
6.0%
6/100 • 6 months of randomized treatment
|
2.0%
2/100 • 6 months of randomized treatment
|
|
Gastrointestinal disorders
constipation
|
1.0%
1/100 • 6 months of randomized treatment
|
7.0%
7/100 • 6 months of randomized treatment
|
|
Gastrointestinal disorders
Controlled discharge of oil without stool
|
56.0%
56/100 • 6 months of randomized treatment
|
11.0%
11/100 • 6 months of randomized treatment
|
|
Gastrointestinal disorders
Decreased frequency of bowel movements
|
25.0%
25/100 • 6 months of randomized treatment
|
22.0%
22/100 • 6 months of randomized treatment
|
|
Gastrointestinal disorders
diarrhea
|
21.0%
21/100 • 6 months of randomized treatment
|
8.0%
8/100 • 6 months of randomized treatment
|
|
Gastrointestinal disorders
hiccups
|
1.0%
1/100 • 6 months of randomized treatment
|
3.0%
3/100 • 6 months of randomized treatment
|
|
Gastrointestinal disorders
Increased frequency of bowel movements
|
68.0%
68/100 • 6 months of randomized treatment
|
45.0%
45/100 • 6 months of randomized treatment
|
|
Gastrointestinal disorders
NAUSEA
|
10.0%
10/100 • 6 months of randomized treatment
|
9.0%
9/100 • 6 months of randomized treatment
|
|
Gastrointestinal disorders
oily spotting
|
6.0%
6/100 • 6 months of randomized treatment
|
0.00%
0/100 • 6 months of randomized treatment
|
|
Gastrointestinal disorders
rectal bleeding - hemorrhoids
|
4.0%
4/100 • 6 months of randomized treatment
|
2.0%
2/100 • 6 months of randomized treatment
|
|
Gastrointestinal disorders
soft or deliquescent stools
|
68.0%
68/100 • 6 months of randomized treatment
|
42.0%
42/100 • 6 months of randomized treatment
|
|
Gastrointestinal disorders
stomach pain or cramps
|
8.0%
8/100 • 6 months of randomized treatment
|
9.0%
9/100 • 6 months of randomized treatment
|
|
Gastrointestinal disorders
Stools almost all liquid with very few solid parts
|
64.0%
64/100 • 6 months of randomized treatment
|
34.0%
34/100 • 6 months of randomized treatment
|
|
Gastrointestinal disorders
Stools hard and in the shape of small pellets
|
11.0%
11/100 • 6 months of randomized treatment
|
10.0%
10/100 • 6 months of randomized treatment
|
|
Gastrointestinal disorders
Stools mixed with fat or with a separate oily layer
|
83.0%
83/100 • 6 months of randomized treatment
|
18.0%
18/100 • 6 months of randomized treatment
|
|
Gastrointestinal disorders
uncontrolled passage of stool or oil
|
60.0%
60/100 • 6 months of randomized treatment
|
11.0%
11/100 • 6 months of randomized treatment
|
|
Gastrointestinal disorders
Urgent, but controlled, need to produce stools
|
44.0%
44/100 • 6 months of randomized treatment
|
18.0%
18/100 • 6 months of randomized treatment
|
|
Gastrointestinal disorders
Vomiting
|
7.0%
7/100 • 6 months of randomized treatment
|
7.0%
7/100 • 6 months of randomized treatment
|
|
General disorders
dizziness
|
4.0%
4/100 • 6 months of randomized treatment
|
4.0%
4/100 • 6 months of randomized treatment
|
|
General disorders
epistaxis
|
5.0%
5/100 • 6 months of randomized treatment
|
2.0%
2/100 • 6 months of randomized treatment
|
|
General disorders
feeling cold
|
5.0%
5/100 • 6 months of randomized treatment
|
2.0%
2/100 • 6 months of randomized treatment
|
|
General disorders
headache
|
14.0%
14/100 • 6 months of randomized treatment
|
17.0%
17/100 • 6 months of randomized treatment
|
|
General disorders
increased sweating
|
3.0%
3/100 • 6 months of randomized treatment
|
4.0%
4/100 • 6 months of randomized treatment
|
|
General disorders
increased thirst
|
5.0%
5/100 • 6 months of randomized treatment
|
4.0%
4/100 • 6 months of randomized treatment
|
|
General disorders
sinusitis, post-nasal drip or nasal stuffiness
|
2.0%
2/100 • 6 months of randomized treatment
|
5.0%
5/100 • 6 months of randomized treatment
|
|
General disorders
unusual tiredness or weakness (fatigue)
|
1.0%
1/100 • 6 months of randomized treatment
|
5.0%
5/100 • 6 months of randomized treatment
|
|
Infections and infestations
Pharyngitis
|
6.0%
6/100 • 6 months of randomized treatment
|
12.0%
12/100 • 6 months of randomized treatment
|
|
Infections and infestations
sinusitis, post-nasal drip or nasal stuffiness
|
1.0%
1/100 • 6 months of randomized treatment
|
3.0%
3/100 • 6 months of randomized treatment
|
|
Metabolism and nutrition disorders
decrease in appetite
|
11.0%
11/100 • 6 months of randomized treatment
|
9.0%
9/100 • 6 months of randomized treatment
|
|
Musculoskeletal and connective tissue disorders
muscle pain, stiffness, cramps, or ache
|
16.0%
16/100 • 6 months of randomized treatment
|
12.0%
12/100 • 6 months of randomized treatment
|
|
Nervous system disorders
migraine headaches
|
3.0%
3/100 • 6 months of randomized treatment
|
0.00%
0/100 • 6 months of randomized treatment
|
|
Psychiatric disorders
mental depression
|
1.0%
1/100 • 6 months of randomized treatment
|
3.0%
3/100 • 6 months of randomized treatment
|
|
Renal and urinary disorders
dysuria or UTI
|
1.0%
1/100 • 6 months of randomized treatment
|
5.0%
5/100 • 6 months of randomized treatment
|
|
Renal and urinary disorders
nocturia
|
0.00%
0/100 • 6 months of randomized treatment
|
3.0%
3/100 • 6 months of randomized treatment
|
|
Respiratory, thoracic and mediastinal disorders
Asthma symptoms
|
5.0%
5/100 • 6 months of randomized treatment
|
3.0%
3/100 • 6 months of randomized treatment
|
|
Respiratory, thoracic and mediastinal disorders
cough
|
0.00%
0/100 • 6 months of randomized treatment
|
7.0%
7/100 • 6 months of randomized treatment
|
|
Respiratory, thoracic and mediastinal disorders
Upper Respiratory Infection
|
14.0%
14/100 • 6 months of randomized treatment
|
17.0%
17/100 • 6 months of randomized treatment
|
|
Skin and subcutaneous tissue disorders
skin rash
|
5.0%
5/100 • 6 months of randomized treatment
|
2.0%
2/100 • 6 months of randomized treatment
|
Additional Information
Jack A. Yanovski, MD, PhD, Chief SGO, PDEGEN
National Institute of Child Health and Human Development, NIH
Phone: 301-496-4686
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place