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Genotropin

Phase 3

Idiopathic Short Stature | Small molecule | Other |Pfizer, Inc.|Last Updated: Apr 5, 2016

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_CONTROLLED
Total Trials1
Total Enrollment316
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00396097Individualized, Target-Driven Treatment Of Children With Idiopathic Short StaturePHASE3 COMPLETED 316Dec 1, 2006Aug 1, 2012Apr 5, 201643 United States
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Study Endpoints
Primary Endpoints
Absolute On-target Difference (AOTD) at 24 Months
2 years

This was defined as an absolute difference between the 24-month height standard deviation score (SDS) and targeted 24-month height SDS (10th percentile (%), or -1.3 SDS). SDS indicates how similar the participant was to the reference population. These were calculated using 2000 Center for the Disease Control (CDC) growth reference tables (by age and gender).

Secondary Endpoints
Variability of Height SDS at 24 Months
2 years
Time Cost (Months Until >= -2 SDS)
2 years
Computed Cost of Height Gain at 48 Months
4 years
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
StandardACTIVE_COMPARATORStandard daily HGH treatment
Formula-basedACTIVE_COMPARATORFormula-based dose regimen
Interventions
NameTypeDescription
GenotropinDRUGCompare daily injections of formula-based HGH treatment to daily injections of standard HGH treatment in subjects with Idiopathic Short Stature over 24 months period followed by an exploratory 24 months period.
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Eligibility Criteria
Age Range3 Years — 10 Years
SexALL
Healthy VolunteersNo
Study Sites43

Inclusion Criteria: * Prepubertal children with bone ages between 3 and 10 years of age for males and 3 and 9 years of age for females * Naive to Growth Hormone treatment Exclusion Criteria: * Abnormal karyotype. Small Gestational Age and Skeletal dysplasia.

Countries:United States
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