parallel randomized trial

NCT02527200

Effect of Liraglutide for Weight Management in Paediatric Subjects With Prader-Willi Syndrome.

Design

Randomized: 56
Start year: 2015
Treatment: 52 weeks, follow-up 54 weeks
Countries: multinational (20 sites)
Sponsor: Novo Nordisk (industry)
Eligibility: age 6 to 17; genetically confirmed Prader-Willi syndrome; BMI at least adult 30 equivalent and 95th percentile

Arm	Treatment	Background	Randomized	Analyzed
A1	Liraglutide 3.0 mg subcutaneous once daily Liraglutide, adolescents	diet and physical activity counseling; growth hormone allowed; lifestyle: standard	20	19
A2	Placebo Placebo, adolescents	diet and physical activity counseling; growth hormone allowed; lifestyle: standard	12	12
A3	Liraglutide 3.0 mg subcutaneous once daily Liraglutide, children	diet and physical activity counseling; growth hormone allowed; lifestyle: standard	17	17
A4	Placebo Placebo, children	diet and physical activity counseling; growth hormone allowed; lifestyle: standard	7	7

As printed in the trial reports or registry, per arm or for the whole trial.

Characteristic	Statistic	Liraglutide 3.0 mg subcutaneous once daily A1	Placebo A2	Liraglutide 3.0 mg subcutaneous once daily A3	Placebo A4
age	mean (years)	14.4	14.1	8.1	9.4
age	sd (years)	1.9	1.9	1.7	1.6
asian	n	0	0	0	0
black	n	2	0	1	0
black	percent	10.5		5.9
bmi sds	mean (SDS)	3.35	4.02	4.89	4.17
bmi sds	sd (SDS)	1.07	1.61	2.37	2.11
bmi	mean (kg/m2)	36.3	40.2	32.4	30.3
bmi	sd (kg/m2)	6.5	10.7	7.5	5.5
female	n	10	5	11	2
female	percent	52.6	41.7	64.7	28.6
hba1c	mean (percent)	5.5	5.5	5.6	5.4
hba1c	sd (percent)	0.4	0.4	0.3	0.3
hispanic	n	1	0	3	1
other race american indian alaska native	n	0	0	0	0
other race multiple	n	0	0	0	0
other race native hawaiian pacific islander	n	0	0	1	0
weight	mean (kg)	90.1	102.0	57.1	55.5
white	n	11	6	10	7
white	percent	57.9	50.0	58.8	100

59 adjudicated result lines, grouped by outcome. Open an outcome to see its values.

Percent body weight change (8 lines; O10)

Week	Arm	Statistic	Estimate	SE or SD	Events / N	Detail
16	A1	percent change	-1.7 percent	3.8	18	not stated; full analysis; registry:outcomes
16	A2	percent change	-0.7 percent	3.7	12	not stated; full analysis; registry:outcomes
16	A3	percent change	-1.1 percent	4.1	16	not stated; full analysis; registry:outcomes
16	A4	percent change	-2.2 percent	5.5	7	not stated; full analysis; registry:outcomes
52	A1	percent change	-0.4 percent	6.5	17	not stated; full analysis; registry:outcomes
52	A2	percent change	2.3 percent	5.7	11	not stated; full analysis; registry:outcomes
52	A3	percent change	6.3 percent	12.1	14	not stated; full analysis; registry:outcomes
52	A4	percent change	4.9 percent	8.6	7	not stated; full analysis; registry:outcomes

BMI standard deviation score change (20 lines; O13A)

Week	Arm	Statistic	Estimate	SE or SD	95% CI	Events / N	Detail
16	A1	change	-0.20 SDS	0.05		19	not stated; full analysis; R0021634:Table 2
16	A1	change	-0.18 SDS	0.21		18	not stated; full analysis; registry:outcomes
16	A1 vs A2	md	-0.07 SDS		-0.23 to 0.09		not stated; full analysis; R0021634:Table 2
16	A2	change	-0.13 SDS	0.06		12	not stated; full analysis; R0021634:Table 2
16	A2	change	-0.18 SDS	0.23		12	not stated; full analysis; registry:outcomes
16	A3 vs A4	md	-0.06 SDS		-1.06 to 0.93		not stated; full analysis; R0021634:Table 2
16	A3	change	-0.50 SDS	0.28		16	not stated; full analysis; R0021634:Table 2
16	A3	change	-0.50 SDS	0.65		16	not stated; full analysis; registry:outcomes
16	A4	change	-0.44 SDS	0.33		7	not stated; full analysis; R0021634:Table 2
16	A4	change	-0.48 SDS	0.39		7	not stated; full analysis; registry:outcomes
52	A1	change	-0.31 SDS	0.13		19	not stated; full analysis; R0021634:Table 2
52	A1	change	-0.27 SDS	0.37		17	not stated; full analysis; registry:outcomes
52	A1 vs A2	md	-0.14 SDS		-0.62 to 0.34		not stated; full analysis; R0021634:Table 2
52	A2	change	-0.17 SDS	0.21		12	not stated; full analysis; R0021634:Table 2
52	A2	change	-0.13 SDS	0.25		11	not stated; full analysis; registry:outcomes
52	A3	change	-0.73 SDS	0.21		16	not stated; full analysis; R0021634:Table 2
52	A3	change	-0.79 SDS	1.21		14	not stated; full analysis; registry:outcomes
52	A3 vs A4	md	-0.07 SDS		-0.89 to 0.76		not stated; full analysis; R0021634:Table 2
52	A4	change	-0.67 SDS	0.32		7	not stated; full analysis; R0021634:Table 2
52	A4	change	-0.71 SDS	0.68		7	not stated; full analysis; registry:outcomes

Serious adverse events (6 lines; O25)

Arm	Statistic	Events / N	Detail
A1	events	2 / 20	treatment policy; safety; R0021634:Table 4
A1	events	1 / 20	while on treatment; safety; registry:adverse_events
A2	events	2 / 12	treatment policy; safety; R0021634:Table 4
A2	events	0 / 12	while on treatment; safety; registry:adverse_events
A3	events	2 / 17	treatment policy; safety; R0021634:Table 4
A4	events	1 / 7	treatment policy; safety; R0021634:Table 4

Discontinuation due to adverse events (4 lines; O26)

Arm	Statistic	Events / N	Detail
A1	events	2 / 20	treatment policy; safety; R0021634:Table 4
A2	events	0 / 12	treatment policy; safety; R0021634:Table 4
A3	events	1 / 17	treatment policy; safety; R0021634:Table 4
A4	events	0 / 7	treatment policy; safety; R0021634:Table 4

Nausea (4 lines; O27A)

Arm	Statistic	Events / N	Detail
A1	events	1 / 20	while on treatment; safety; registry:adverse_events
A2	events	0 / 12	while on treatment; safety; registry:adverse_events
A3	events	1 / 17	while on treatment; safety; registry:adverse_events
A4	events	0 / 7	while on treatment; safety; registry:adverse_events

Vomiting (4 lines; O27B)

Arm	Statistic	Events / N	Detail
A1	events	2 / 20	treatment policy; safety; R0021634:Table 4
A2	events	0 / 12	treatment policy; safety; R0021634:Table 4
A3	events	3 / 17	treatment policy; safety; R0021634:Table 4
A4	events	0 / 7	treatment policy; safety; R0021634:Table 4

Diarrhea (5 lines; O27C)

Arm	Statistic	Events / N	Detail
A1	events	10 / 20	treatment policy; safety; R0021634:Table 4
A2	events	2 / 12	treatment policy; safety; R0021634:Table 4
A3	events	7 / 17	treatment policy; safety; R0021634:Table 4
A4	events	1 / 7	treatment policy; safety; R0021634:Table 4
A4	events	0 / 7	while on treatment; safety; registry:adverse_events

Constipation (4 lines; O27D)

Arm	Statistic	Events / N	Detail
A1	events	3 / 20	treatment policy; safety; R0021634:Table 4
A2	events	0 / 12	treatment policy; safety; R0021634:Table 4
A3	events	0 / 17	treatment policy; safety; R0021634:Table 4
A4	events	0 / 7	treatment policy; safety; R0021634:Table 4

Gallbladder and biliary disease (4 lines; O28)

Arm	Statistic	Events / N	Detail
A1	events	1 / 20	while on treatment; safety; cholelithiasis; registry:adverse_events
A2	events	0 / 12	while on treatment; safety; cholelithiasis; registry:adverse_events
A3	events	0 / 17	while on treatment; safety; cholelithiasis; registry:adverse_events
A4	events	0 / 7	while on treatment; safety; cholelithiasis; registry:adverse_events