A multicenter, Phase 3, randomized, open-label, active-controlled, parallel-group trial investigating the safety, tolerability, and efficacy of TransCon hGH administered once a week versus standard daily hGH replacement therapy over 52 weeks in prepubertal children with growth hormone deficiency (GHD)
- Children’s Medical Center (Dallas, Plano, Southlake)
Grace Tannin, M.D.
This is a phase 3, randomized, open-label, active-controlled trial of TransCon hGH as compared to standard daily growth hormone, over 52 weeks. The trial will be conducted at approximately 100 sites in approximately 20 countries in north and South america, europe, Middle east and north africa, and oceania. all centers will be specialized treatment centers in the management of pediatric GHD.
The trial consists of:
* Screening period - up to 6 weeks (plus a maximum of 2 weeks until V1)
* Treatment period - 52 weeks of dosing
The total duration of participation for each subject in the trial will therefore be up to about 60 weeks.
all subjects who successfully complete the 52-week randomized trial will be invited to participate in an extension trial after the treatment period of this 52-week trial has ended. This extension period is to assess long-term safety and efficacy. Subjects on Genotropin treatment (Cohort 2) will be switched over to TransCon hGH treatment for this extension trial.
1. Prepubertal children with GHD in Tanner stage 1
- Boys: 3 -12 years, inclusive
-Girls: 3 -11 years, inclusive
2. Impaired height defined as at least 2.0 standard deviations below the mean height for chronological age and sex
3. BMI within (+ or -)2.0 of the mean BMI for chronological age and sex.
4. Diagnosis of GHD confirmed by 2 different GH stimulation tests, defined as a peak GH level of <=10 ng/mL, determined with a validated assay. One or 2 well documented historical tests performed within 6 months prior to Screening can be accepted to replace 1 or both GH stimulation tests. The highest GH level determines eligibility
5. Bone age (BA) at least 6 months less than the chronological age.
6. Baseline IGF-1 level of at least 1.0 SD below the mean IGF-1 level standardized for age and sex. According to the central laboratory reference values
7. Normal fundoscopy at Screening.
8. Children with multiple hormonal deficiencies must be on stable replacement therapy for other hypothalamic-pituitary axes for at least 3 months. Thyroid replacement therapy for thyroid hormone deficiency must be instituted at least 6 months prior to Screening. Temporary adjustment of glucocorticoid replacement therapy, as appropriate, is acceptable.
9. Normal 46 XX karyotype for girls
10. Written, signed informed consent of the parent(s) or legal guardian(s) of the subject and written assent of the subject.