NCT02781727|CompletedPhase 3ResultsDMC

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

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)

Ascendis Pharma Endocrinology Division A/S ClinicalTrials.gov

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2 other identifiers

TransCon hGH CT-3012016-001145-11

Study Type

interventional

Target

162

Locations

14 countries

Sites

Timeline

RegisteredMay 2016

StartedDec 2016

CompletionJan 2019

Brief Summary

A 52 week trial of TransCon hGH, a long-acting growth hormone product, versus human growth hormone therapy. TransCon hGH will be given once-a-week, human growth hormone (hGH) will be given daily. Approximately 150 prepubertal, hGH-treatment naïve children (males and females) with GHD will be included. Randomization will occur in a 2:1 ratio (TransCon hGH : Genotropin). This is a global trial that will be conducted in Armenia, Australia, Belarus, Bulgaria, Georgia, Greece, Italy, New Zealand, Poland, Romania, Russia, Turkey, Ukraine, and the United States.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

162

participants targeted

Target at P25-P50 for phase_3

Timeline

Completed

Started Dec 2016

Geographic Reach

14 countries

53 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

2.1 years study duration

First Submitted

Initial submission to the registry

May 19, 2016

Completed

5 days until next milestone

First Posted

Study publicly available on registry

May 24, 2016

Completed

7 months until next milestone

Study Start

First participant enrolled

December 13, 2016

Completed

2.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 17, 2019

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 17, 2019

Completed

3 years until next milestone

Results Posted

Study results publicly available

January 4, 2022

Completed

Last Updated

January 4, 2022

Status Verified

December 1, 2021

Enrollment Period

2.1 years

First QC Date

May 19, 2016

Results QC Date

September 24, 2021

Last Update Submit

December 6, 2021

Conditions

Growth Hormone Deficiency, Pediatric hGH (Human Growth Hormone)Endocrine System Diseases Hormones Pituitary Diseases

Keywords

Human Growth HormonehGHGHDrHGHPediatric Growth Hormone DeficiencyLong Acting Growth HormoneSomatropinProdrugGrowth FailureGrowth Hormone Replacement TherapySustained ReleaseSustained Release Growth HormoneGrowth Hormone Deficiency

Outcome Measures

Primary Outcomes (1)

Annualized Height Velocity at 52 Weeks for Weekly Lonapegsomatropin and Daily hGH Treatment Groups
Annualized height velocity (AHV) at 52 weeks for weekly lonapegsomatropin (TransCon hGH) and daily hGH treatment groups
52 weeks

Secondary Outcomes (5)

Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability]
52 Weeks
Annualized Height Velocity Over 52 Weeks for Weekly Lonapegsomatropin and Daily hGH Treatment Groups
Week 5, Week 13, Week 26, Week 39 and Week 52
Change in Height Standard Deviation Score Over 52 Weeks for the Weekly Lonapegsomatropin and Daily hGH Treatment Groups
Week 5, Week 13, Week 26, Week 39 and Week 52
Average IGF-1 Standard Deviation Score Over 52 Weeks for the Weekly Lonapegsomatropin and Daily hGH Treatment Groups
Week 13, Week 26, Week 39, and Week 52
Number of Participants With Treatment Emergent Anti-hGH Binding Antibody Formation
Start of study treatment through Week 52

Study Arms (2)

TransCon hGH

EXPERIMENTAL

Once weekly subcutaneous injection of TransCon hGH

Drug: Once weekly subcutaneous injection of TransCon hGH

human growth hormone (Genotropin)

ACTIVE COMPARATOR

Once daily subcutaneous injection of Genotropin

Drug: Once daily subcutaneous injection of Genotropin

Interventions

Once weekly subcutaneous injection of TransCon hGHDRUG

Once weekly subcutaneous injection

TransCon hGH

Once daily subcutaneous injection of GenotropinDRUG

Once daily subcutaneous injection

human growth hormone (Genotropin)

Eligibility Criteria

Age3 Years - 12 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17)

You may qualify if:

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)

You may not qualify if:

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

Contact the study team to confirm eligibility.