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Once weekly of TransCon hGH

Phase 3

Growth Hormone Deficiency, Pediatric | Small molecule | Endocrine |Ascendis Pharma A/S|Last Updated: Jan 4, 2022

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
RandomizedACTIVE_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment162
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02781727A Phase 3 Trial of the Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD)PHASE3 COMPLETED 162 — — Dec 13, 2016Jan 17, 2019Jan 4, 202253 United States, Armenia +12
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Study Endpoints
Primary Endpoints
Annualized Height Velocity at 52 Weeks for Weekly Lonapegsomatropin and Daily hGH Treatment Groups
52 weeks

Annualized height velocity (AHV) at 52 weeks for weekly lonapegsomatropin (TransCon hGH) and daily hGH treatment groups

Secondary Endpoints
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
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
TransCon hGHEXPERIMENTALOnce weekly subcutaneous injection of TransCon hGH
human growth hormone (Genotropin)ACTIVE_COMPARATOROnce daily subcutaneous injection of Genotropin
Interventions
NameTypeDescription
Once weekly subcutaneous injection of TransCon hGHDRUGOnce weekly subcutaneous injection
Once daily subcutaneous injection of GenotropinDRUGOnce daily subcutaneous injection
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Eligibility Criteria
Age Range3 Years — 12 Years
SexALL
Healthy VolunteersNo
Study Sites53

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)...

Countries:United StatesArmeniaAustraliaBelarusBulgariaGeorgiaGreeceItalyNew ZealandPolandRomaniaRussiaTurkey (Türkiye)Ukraine
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