Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Somatropin
Genotropin · 9 trials · 7 indications
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).
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).
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).
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).
Participants were asked the following question from Section II of the Injection Pen Assessment Questionnaire (IPAQ) patient-reported outcome (PRO) tool, "Thinking about the Genotropin pen and the new injection pen you used over the past few months, please compare both injection pens and choose which one is easier to use overall?" Choices included: Genotropin Pen® easier to use, new injection pen easier to use, or no difference.
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).
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).
Height was measured using a wall mounted device (example, Harpenden stadiometer). The standing height of the participant was measured two times and the mean of these measurements was recorded. Height SDS CA Yx = (height Yx - reference mean for CA Yx) / reference SD for CA Yx; Yx refers to the value at particular timepoint x. Height in SDS was calculated using Sempe reference means and SD for height. CA calculated as integer (Date of height measurement-Date of birth)/365.25\*12.
Height was measured using a wall mounted device (example, Harpenden stadiometer). The standing height of the participant was measured two times and the mean of these measurements was recorded. Height SDS CA Yx = (height Yx - reference mean for CA Yx) / reference SD for CA Yx; Yx refers to the value at particular timepoint x. Height in SDS was calculated using Sempe reference means and SD for height. CA calculated as integer (Date of height measurement-Date of birth)/365.25\*12.
Change in annual growth rate (AGR) standard deviation score (SDS) for chronological age (CA) derived by subtracting AGR SDS CA at baseline from each time point (Yx) value. AGR at Yx= (height Yx-height Y\[x-1\])/(\[date of Yx-date of Y{x-1}\]/365.25). AGR as SDS calculated using Sempe reference means and standard deviations (SD) for growth rate. AGR SDS CA (for both baseline and Yx)= (AGR-reference mean for growth rate CA)/reference SD for growth rate CA. CA calculated as integer (Date of height measurement-Date of birth)/365.25\*12. SDS indicates how similar participant was to reference population.
Change in annual growth rate (AGR) standard deviation score (SDS) for chronological age (CA) derived by subtracting AGR SDS CA at baseline from each time point (Yx) value. AGR at Yx= (height Yx-height Y\[x-1\])/(\[date of Yx-date of Y{x-1}\]/365.25). AGR as SDS calculated using Sempe reference means and standard deviations (SD) for growth rate. AGR SDS CA (for both baseline and Yx)= (AGR-reference mean for growth rate CA)/reference SD for growth rate CA. CA calculated as integer (Date of height measurement-Date of birth)/365.25\*12. SDS indicates how similar participant was to reference population.
Change in Height SDS after 1 year where SDS=height minus mean (age-and sex-matched reference) divided by SD (age and sex-matched reference).
Change in Growth Velocity (GV) SDS after 1 year where SDS=GV minus mean (age-and sex-matched reference) divided by SD (age and sex-matched reference).
| Arm | Type | Description |
|---|---|---|
| Daily to Weekly | OTHER | Genotropin to somatrogon |
| Weekly to Daily | OTHER | somatrogon to Genotropin |
| Mark VII/Current pen | OTHER | Subject will use Mark VII pen for 2 months followed by Current pen for 2 months |
| Current pen/Mark VII | OTHER | Subject will use current Genotropin pen for 2 months followed by Mark VII pen for 2 months |
| Genotropin pen | EXPERIMENTAL | All subjects will receive genotropin pen to use for 2 months. |
| Standard | ACTIVE_COMPARATOR | Standard daily HGH treatment |
| Formula-based | ACTIVE_COMPARATOR | Formula-based dose regimen |
| Somatropin | EXPERIMENTAL | - |
| Treatment Arm | ACTIVE_COMPARATOR | - |
| Control Arm | NO_INTERVENTION | - |
| Name | Type | Description |
|---|---|---|
| Genotropin | DRUG | Genotropin (dose \[mg\] at time of enrollment) given subcutaneously once daily |
| somatrogon | DRUG | 0.66 mg/kg/week given subcutaneously once weekly |
| Genotropin Pen | DEVICE | Pen provided in 5, 5.3 and 12 mg doses, Dose based on body weight, subcutaneous injection daily for 2 months while in study. |
| MARK VII pen | DEVICE | Pen provided in 5, 5.3 and 12mg doses, dose based on body weight, subcutaneous injection daily for 2 months while in study. |
| New Genotropin Pen | DEVICE | Subjects 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 |
| Somatropin | DRUG | Liquid, daily, during 3 years and extended to 5 years Dosage: 0,46 mg/kg/week - the maximum dose should not exceed 50 µg/kg/day |
| Control Arm | OTHER | Controlled, prospective, randomized, multicenter study with an untreated (control) group during the first year. The children will be randomized into treatment or untreated (control) group. After one year the control group will undergo GH-therapy, too. The study will end after 2 and 3 years of observation, respectively. Children randomized to the control group will get the possibility to continue treatment for a further year. |
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...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 2 | PHASE3 | Somapacitan, Norditropin FlexPro pen |
| Ascendis Pharma A/S | ASND | 1 | PHASE3 | Lonapegsomatropin |
Genotropin is used to treat growth disorders, including growth hormone deficiency and idiopathic short stature. It is a growth hormone therapy developed by Pfizer for pediatric and adult patients whose growth is affected by insufficient growth hormone. Its clinical program has studied its use in children with growth hormone deficiency and idiopathic short stature.
Genotropin is made by Pfizer, Inc., which trades on the New York Stock Exchange under the ticker PFE. Pfizer develops and markets the product, and it has sponsored the Phase 3 clinical trials evaluating Genotropin in growth hormone deficiency and idiopathic short stature.
Genotropin has been studied in Phase 3 clinical development across growth disorders, growth hormone deficiency, and idiopathic short stature. Its Phase 3 program includes completed trials, with no active trials currently listed. The studies enrolled a combined total of 343 participants and were conducted in the United States and several European countries.
Genotropin has been evaluated in completed Phase 3 trials including NCT03831880, which compared weekly versus daily growth hormone injections in children with growth hormone deficiency, NCT01112865, a crossover convenience and preference study of the Mark VII device versus the Genotropin Pen, NCT00965484, assessing use of an injection pen, and NCT00396097, a target-driven treatment study in children with idiopathic short stature.
The completed Phase 3 trials for Genotropin focused on treatment burden, device preference, and target-driven dosing rather than placebo-controlled efficacy. NCT03831880 examined patient perception of weekly versus daily growth hormone injections, while NCT01112865 and NCT00965484 compared injection pen devices. NCT00396097 studied individualized, target-driven treatment in children with idiopathic short stature.