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Home › Conditions › idiopathic short stature

Idiopathic short stature

2 published articles · 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 101 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 · 101 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.
Patient Mode — summaries use plain language, avoiding clinical jargon.
FGFR3 N540S variant exhibits phenotypic heterogeneity between proportionate short stature and hypochondroplasia
Diabetes & Endocrinology Sys. Review
FGFR3 N540S variant exhibits phenotypic heterogeneity between proportionate short stature and hypochondroplasia Genetic Testing Reveals New Variety of Short Stature Cause
This case report describes a 3-year-old male with proportionate short stature and his relatives who all harbored the heterozygous FGFR3 N540…
A new genetic finding shows that the same gene mutation can cause different types of short stature in different people.
Frontiers Sep 3, 2026
Meta-analysis finds growth hormone therapy shows no significant ocular biometric changes in idiopathic short stature
Pediatrics Meta-analysis
Meta-analysis finds growth hormone therapy shows no significant ocular biometric changes in idiopathic short stature Growth hormone therapy shows possible link to refractive changes in children with short stature
This systematic review and meta-analysis of nine studies evaluated growth hormone therapy in children with idiopathic short stature.
Growth hormone therapy in children with short stature may change how their eyes focus light, though other eye measurements remain stable.
Apr 17, 2026