Table 2: Summary of efficacy endpoints.

		Group A (n=36)	Group B (n=15)	Estimated treatment difference* (95% CI)	P value
Primary endpoint
Ht-V (cm/year)		12.02	6.87	5.15 (4.09, 6.21)	<0.0001
Confirmatory secondary endpoint
Ht-SDS change from baseline at 6 months		0.76	0.19	–	–
Ht-SDS at 6 months		-1.56	-2.13	0.57 (0.43, 0.71)	<0.0001
Supportive secondary endpoint
IGF-1, ng/mL	At 6 months	321.18	156.61	–	–
	Change from baseline	192.58	28.03	164.56 (112.04, 217.08)	<0.0001
IGFBP-3, μg/mL	At 6 months	4.29	3.62	–	–
	Change from baseline	0.89	0.22	0.67 (-0.04, 1.38)	0.06
Ht-V endpoint
		Ht-V at 6 months cm/year (SD)	Ht-V at 12 months cm/year (SD)	Estimated treatment difference (Ht-V at 0–6 months minus Ht-V at 6–12 months) cm/year (95% CI)	P value
Group A		12.0 (1.8)	10.6 (1.5)	2.80 (1.55, 4.04)	<0.0001
Group B		6.9 (1.3)†	9.2 (0.8)†	-4.60 (-6.12, -3.09)	<0.0001

*Least square means. Based on the full analysis set.

†Patients in group B were not treated during the first 6 months of the study and received growth hormone during the second 6 months of the study.

CI = confidence interval; Ht-SDS = height standard deviation score; Ht-V = height velocity; IGF-1 = insulin-like growth factor 1; IGFBP-3 = insulin-like growth factor-binding protein 3; SD = standard deviation.