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Idiopathic short stature

1 published article · Updated continuously

Clinical Trial Landscape

Clinical Trials for idiopathic short stature

7 trials tracked for idiopathic short stature: 6 in phase 3 or 4 and 3 with published results. The most-cited published study has 99 citations.

7Trials tracked
6Phase 3 & 4
0Recruiting
3With published results
Phase distribution
Phase 3 6 Other / NA 1
  1. Phase 3 Aromatase Inhibitors, Alone And In Combination With Growth Hormone In Adolescent Boys With Idiopathic Short Stature Completed · 99 cited
  2. Phase 3 A Phase 3 Study to Evaluate the Safety and Efficacy of Saizen® in Children With Idiopathic Short Stature (ISS) Completed · 12 cited
  3. Phase 3 Efficacy and Safety of Recombinant Human Growth Hormone on Height Velocity in Subjects With Idiopathic Short Stature Completed · 5 cited
  4. Phase 3 Efficacy and Safety of DA-3002 in Children With Idiopathic Short Stature Completed
  5. Phase 3 Genotropin Study Assessing Use of Injection Pen Completed
  6. Phase 3 Individualized, Target-Driven Treatment Of Children With Idiopathic Short Stature Completed
Show 1 more trials
  1. N/A Ghrelin Levels in Children With Poor Growth Completed

Showing the 7 most-cited and recently-updated of 7 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found For clinicians

Idiopathic short stature: what the trials found

Treatment with somatropin has been shown to significantly increase height velocity (Ht-V) compared to placebo, with observed increases of 12.02 and 6.87 respectively (p<0.0001) 3. Similarly, DA-3002 demonstrated an annualized height velocity of 10.68 cm/year versus 5.72 cm/year over a 26-week period 4.

Aromatase Inhibitor treatment resulted in significant increases in height (ranging from 14 to 18.9) with p<0.006, while also showing statistically significant improvements in lean body mass (p=0.015) 1. However, this intervention did not result in a statistically significant change in bone density z-scores adjusted for height (p=0.906) 1.

Saizen® demonstrated a significant increase in height velocity at month 6 compared to placebo (p<0.0001) 2. Clinical evaluations of Genotropin showed no statistically significant difference between treatment arms regarding absolute on-target difference (AOTD) at 24 months (p=0.5762), variability of height SDS (p=0.627), or time cost to reach a target SDS (p=0.8016) 5.

Regarding administration, the New Genotropin Mark VII Injection Pen was reported as easier to use by 63.16% of dyads compared to previous experiences, with over 65% of dyads reporting no preference or a preference for the new pen 6.

For the clinician treating this condition

  • Somatropin and DA-3002 are established interventions that significantly increase height velocity in patients with idiopathic short stature [3, 4].
  • Aromatase Inhibitor therapy provides significant gains in both height and lean body mass, though it does not appear to impact bone density z-scores 1.
  • Saizen® is associated with a statistically significant increase in height velocity at the 6-month mark 2.

AI synthesis of 6 cited trials, updated Jun 29, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
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