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New Genotropin Pen

Phase 3

Growth Hormone Deficiency | Unknown | Endocrine |Pfizer, Inc.|Last Updated: Oct 14, 2021

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
RandomizedCONTROLLEDBiomarker
Total Trials2
Total Enrollment223
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03831880Patient Perception of Treatment Burden in Weekly Versus Daily Growth Hormone Injections in Children With GHDPHASE3 COMPLETED 87Feb 7, 2019Aug 28, 2020Oct 14, 202132 United States, Bulgaria +3
NCT00965484Genotropin Study Assessing Use of Injection PenPHASE3 COMPLETED 136Oct 1, 2009Jan 1, 2010Jan 28, 201125 United States
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Study Endpoints
Primary Endpoints
Total Score Related to Overall Life Interference Assessed at Baseline, Using Dyad Clinical Outcomes Assessment 1 (DCOA 1) Questionnaire
Baseline

Participants were assessed for their treatment burden using DCOA 1 questionnaire completed by participant/caregiver dyads. The participant life interference questionnaire component of the DCOA 1 had 7 questions (life interference \[5 questions\]: a measure of life interference \[daily activities/social activities/leisure/night away from home/travel\]; life interference-changes to life routine \[1 question\]: a measure of how often changes are made to life routine; and life interference-bother of growth hormone \[GH\] injections \[1 question\]: a measure of how often the growth hormone injections cause bother) and all questions used a 5-point scale: 1= never, 2= rarely, 3= sometimes, 4= often, 5= always. The overall life interference total score was sum of all 7 questions, scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score meant less life interference (better outcome).

Total Score Related to Overall Life Interference Assessed at Week 12, Using DCOA 1 Questionnaire
Week 12

Participants were assessed for their treatment burden using DCOA 1 questionnaire completed by participant/caregiver dyads. The participant life interference questionnaire component of the DCOA 1 had 7 questions (life interference \[5 questions\]: a measure of life interference \[daily activities/social activities/leisure/night away from home/travel\]; life interference-changes to life routine \[1 question\]: a measure of how often changes are made to life routine; and life interference-bother of growth hormone \[GH\] injections \[1 question\]: a measure of how often the growth hormone injections cause bother) and all questions used a 5-point scale: 1= never, 2= rarely, 3= sometimes, 4= often, 5= always. The overall life interference total score was sum of all 7 questions, scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score meant less life interference (better outcome).

Total Score Related to Overall Life Interference Assessed at Week 24, Using DCOA 1 Questionnaire
Week 24

Participants were assessed for their treatment burden using DCOA 1 questionnaire completed by participant/caregiver dyads. The participant life interference questionnaire component of the DCOA 1 had 7 questions (life interference \[5 questions\]: a measure of life interference \[daily activities/social activities/leisure/night away from home/travel\]; life interference-changes to life routine \[1 question\]: a measure of how often changes are made to life routine; and life interference-bother of growth hormone \[GH\] injections \[1 question\]: a measure of how often the growth hormone injections cause bother) and all questions used a 5-point scale: 1= never, 2= rarely, 3= sometimes, 4= often, 5= always. The overall life interference total score was sum of all 7 questions, scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score meant less life interference (better outcome).

Total Score Related to Overall Life Interference by Treatment in Overall Study, Using DCOA 1 Questionnaire
Baseline up to Week 24

Participants were assessed for their treatment burden using DCOA 1 questionnaire completed by participant/caregiver dyads. The participant life interference questionnaire component of the DCOA 1 had 7 questions (life interference \[5 questions\]: a measure of life interference \[daily activities/social activities/leisure/night away from home/travel\]; life interference-changes to life routine \[1 question\]: a measure of how often changes are made to life routine; and life interference-bother of growth hormone \[GH\] injections \[1 question\]: a measure of how often the growth hormone injections cause bother) and all questions used a 5-point scale: 1= never, 2= rarely, 3= sometimes, 4= often, 5= always. The overall life interference total score was sum of all 7 questions, scores were transformed from raw scores and converted to a 0 to 100 scale; a lower score meant less life interference (better outcome).

Percentage of Dyads (Participant and Caregiver or Parent) Reporting no Difference or Easier to Use for New Genotropin Mark VII Injection Pen Compared to Pre-study Experience With the Genotropin Pen®
2 months

Ease of use measured using the Injection Pen Assessment Questionnaire (IPAQ) patient-reported outcome (PRO) tool (based on 13 unique characteristics of injection pens). Section I measures ease of use of Genotropin® (very easy, somewhat easy, neither easy nor difficult, somewhat difficult, or very difficult). Section II measures ease of use of new Genotropin Mark VII Pen in comparison to pre-study experience with Genotropin® Pen (Genotropin® pen easier to use, new injection pen easier to use, or no difference) and preference (prefer Genotropin® Pen, prefer new injection pen, or no preference).

Secondary Endpoints
Total Score Related to Pen Ease of Use Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire
Baseline, Week 12, Week 24
Total Score Related to Pen Ease of Use by Treatment in Overall Study, Using DCOA 1 Questionnaire
Baseline up to Week 24
Total Score Related to Ease of the Injection Schedule Assessed at Baseline, Week 12 and Week 24, Using DCOA 1 Questionnaire
Baseline, Week 12, Week 24
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelCROSSOVER
PurposeOTHER
Treatment Arms
ArmTypeDescription
Daily to WeeklyOTHERGenotropin to somatrogon
Weekly to DailyOTHERsomatrogon to Genotropin
Genotropin penEXPERIMENTALAll subjects will receive genotropin pen to use for 2 months.
Interventions
NameTypeDescription
GenotropinDRUGGenotropin (dose \[mg\] at time of enrollment) given subcutaneously once daily
somatrogonDRUG0.66 mg/kg/week given subcutaneously once weekly
New Genotropin PenDEVICESubjects will use the genotropin pen for 2 months. After 2 months patients and caregiver will be asked to fill out a questionnaire to assess perception of the genotropin pen
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Eligibility Criteria
Age Range3 Years — 17 Years
SexALL
Healthy VolunteersNo
Study Sites32

Inclusion Criteria: 1. Children aged 3 years old and \<18 years with either isolated GHD, or GH insufficiency. 2. Currently on treatment with either Genotropin Pen®, Genotropin GoQuick Pen®, HumatroPen® (United States of America \[USA\] only), or Omnitrope® Pen (USA only) ≥3 months and have been co...

Countries:United StatesBulgariaCzechiaSlovakiaUnited Kingdom
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