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TransCon hGH

Phase 3

Growth Hormone Deficiency, Pediatric | Small molecule | Endocrine |Ascendis Pharma A/S|Last Updated: May 8, 2024

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDDMC
Total Trials2
Total Enrollment444
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03344458A Long-Term Trial Investigating Safety and Efficacy of TransCon hGH in Children With Growth Hormone Deficiency Who Have Completed a Prior TransCon hGH Clinical TrialPHASE3 COMPLETED 298Dec 19, 2017Feb 21, 2023May 8, 202442 United States, Armenia +9
NCT03305016A Safety, Tolerability and Efficacy Study of TransCon hGH in Children With Growth Hormone DeficiencyPHASE3 COMPLETED 146Nov 13, 2017Mar 19, 2019Jan 4, 202224 United States, Australia +2
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Study Endpoints
Primary Endpoints
Number of Subjects With Treatment-Emergent Adverse Events [Long-Term Safety and Tolerability]
Up to Week 208

Long-term safety and tolerability of weekly lonapegsomatropin (TransCon hGH) treatment

Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability]
26 weeks

Safety and tolerability of weekly lonapegsomatropin (TransCon hGH) treatment

Secondary Endpoints
Annualized Height Velocity by Visit
Up to Week 208
IGF-1 SDS by Visit
Up to Week 208
Height SDS - Change From Baseline
Up to Week 208
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
TransCon hGHEXPERIMENTALOnce weekly subcutaneous injection of TransCon hGH
Interventions
NameTypeDescription
TransCon hGHDRUGOnce weekly subcutaneous injection of TransCon hGH
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Eligibility Criteria
Age Range1 Year — 18 Years
SexALL
Healthy VolunteersNo
Study Sites42

Inclusion Criteria: 1. Children who have completed a prior phase 3 TransCon hGH trial 2. Children who have not permanently discontinued study drug in the prior trial 3. Written, signed, informed consent of the parent or legal guardian of the subject and written assent of the subject as required by ...

Countries:United StatesArmeniaAustraliaBelarusBulgariaGeorgiaGreeceNew ZealandPolandRussiaUkraineCanada
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