A Phase 3 Trial of the Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD)

• Register: ClinicalTrials.gov
• Last refreshed on: 13 September 2021
• Main ID: NCT02781727
• Date of registration: 19/05/2016
• Prospective Registration: Yes
• Primary sponsor: Ascendis Pharma Endocrinology Division A/S
• Public title: A Phase 3 Trial of the Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD)
• Scientific title: 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)
• Date of first enrolment: December 13, 2016
• Target sample size: 161
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02781727
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Armenia, Australia, Belarus, Bulgaria, Georgia, Greece, Italy, New Zealand, Poland, Romania, Russian Federation, Turkey, Ukraine, United States

Contacts

• Name: Michael Beckert, MD
• Affiliation: Ascendis Pharma A/S

• Name: Aimee D Shu, MD
• Affiliation: Ascendis Pharma, Inc.

Key inclusion & exclusion criteria

Inclusion Criteria:

- Prepubertal children with GHD (either isolated or as part of a multiple pituitary
hormone deficiency) in Tanner stage 1 (Tanner 1982) aged:

- Boys: 3-12 years, inclusive

- Girls: 3-11 years, inclusive

- Impaired height (HT) defined as at least 2.0 standard deviations (SD) below the mean
height for chronological age and sex (HT SDS = -2.0) according to the 2000 CDC Growth
Charts for the United States Methods and Development, available at
http://www.cdc.gov/growthcharts/

- 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

- Bone age (BA) at least 6 months less than chronological age

- Baseline IGF-1 level of at least 1 SD below the mean IGF-1 level standardized for age
and sex (IGF-1 SDS =-1)

- Written, signed informed consent of the parent(s) or legal guardian(s) of the subject
and written assent of the subject (if the subject is able to read, understand, and
sign)

Exclusion Criteria:

- Children with a body weight below 12 kg

- Prior exposure to recombinant hGH or IGF-1 therapy

- Children with past or present intracranial tumor growth as confirmed by a sellar MRI
scan (with contrast) at Screening (MRI results from up to 6 months prior to Screening
may be accepted)

- Children with psychosocial dwarfism

- Children with idiopathic short stature

- History or presence of malignant disease; any evidence of present tumor growth

- Closed epiphyses

- Major medical conditions and/or presence of contraindication to hGH treatment

- Participation in any other trial of an investigational agent within 3 months prior to
Screening



Age minimum: 3 Years
Age maximum: 12 Years
Gender: All

Health Condition(s) or Problem(s) studied

Hormones

Growth Hormone Deficiency, Pediatric

Endocrine System Diseases

Pituitary Diseases

hGH (Human Growth Hormone)

Intervention(s)

Drug: Once daily subcutaneous injection of Genotropin

Drug: Once weekly subcutaneous injection of TransCon hGH

Primary Outcome(s)

Annualized height velocity at 52 weeks for weekly TransCon hGH treatment and the daily hGH treatment groups [Time Frame: 52 weeks]

Secondary Outcome(s)

Annualized height velocity for the TransCon hGH and the daily hGH treatment groups over 52 weeks [Time Frame: 52 Weeks]

Evaluate serum IGF-1 and IGFBP-3 and IGF-1 SDS and IGFBP-3 SDS over 52 weeks for the TransCon hGH and the daily hGH treatment groups [Time Frame: 52 Weeks]

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [Time Frame: 52 Weeks]

Change in height standard deviation score (SDS) over 52 weeks for the TransCon hGH and the daily hGH treatment groups [Time Frame: 52 Weeks]

Incidence of anti-hGH antibodies including neutralizing antibodies [Time Frame: 52 Weeks]

Secondary ID(s)

2016-001145-11

TransCon hGH CT-301

Source(s) of Monetary Support

Please refer to primary and secondary sponsors