2 and older, any sex, with Short Stature. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Height Standard Deviation ScorePrimary· Annually through three years of treatment
Height Standard Deviation Score is the standard deviation above or below the mean the height is for age and gender. Values were obtained by plotting heights on Centers for Disease Control and Prevention growth charts. An increase in Height Standard Deviation Score correlates with increase in height. Results are reported for 10 patients treated with recombinant human growth hormone.
Baseline
Group
Value
95% CI
Growth Hormone Treatment for Participants With ACAN Deficiency
-2.52
-4.27 – -1.07
Year 1
Group
Value
95% CI
Growth Hormone Treatment for Participants With ACAN Deficiency
-1.57
-3.89 – -0.46
Year 2
Group
Value
95% CI
Growth Hormone Treatment for Participants With ACAN Deficiency
-1.19
-3.55 – -0.08
Year 3
Group
Value
95% CI
Growth Hormone Treatment for Participants With ACAN Deficiency
-1.09
-3.45 – -0.02
Height Velocity After Three Years of Treatment With Recombinant Human Growth Hormone (rhGH)Primary· Annually through three years of treatment
A participants calculated height velocity derived from height measurements taken over a period of 36 months (baseline visit to 36 month visit). Only those in the treatment arm were treated with growth hormone and observed for response.
Baseline
Group
Value
95% CI
Growth Hormone Treatment for Participants With ACAN Deficiency
5.2
3.8 – 7.1
Year 1
Group
Value
95% CI
Growth Hormone Treatment for Participants With ACAN Deficiency
8.3
7.3 – 11.2
Year 2
Group
Value
95% CI
Growth Hormone Treatment for Participants With ACAN Deficiency
7.7
5.9 – 8.8
Year 3
Group
Value
95% CI
Growth Hormone Treatment for Participants With ACAN Deficiency
6.8
4.9 – 7.2
Number of Participants With Clinical Features of ACAN Deficiency - Osteochondritis DissecansSecondary· Baseline
Evidence of osteochondritis dissecans on MRI (in those who could cooperate to perform such unsedated between the ages of 6 years-old and 20 years-old) or radiograph examination of affected participant's knees.
Group
Value
95% CI
Pediatric Phenotype Group
3
Adult Phenotype Group
1
Number of Participants With Clinical Features of ACAN Deficiency - OsteoarthritisSecondary· Baseline
Evidence of early joint pathology evident (osteoarthritis) on MRI (in those who could cooperate to perform such unsedated between the ages of 6 years-old and 20 years-old) or radiograph examination of affected participant's knees, performed in those who were age appropriate and would cooperate.
Group
Value
95% CI
Pediatric Phenotype Group
0
Adult Phenotype Group
8
Adverse events — posted to ClinicalTrials.gov
Time frame: Adverse event data was collected for treatment group participants from the time of enrollment until 3 months post treatment discontinuation. Adverse event data for phenotype participants was collected from time of enrollment until one week after the completion of their one-time study visit..
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
This is an open-label, single-arm prospective pilot study to study the effects of a single dose regimen of daily growth hormone medication (Norditropin) on pre-pubertal children with Aggrecan deficiency. The growth response will be tracked over a 12 month period. A protocol extension has been approved to continue subjects on treatment for an additional 2 years.
Publications & conference data
2 peer-reviewed publications reference this trial (live from Europe PMC):
NCT05308927 — French Registry of Children Treated With Norditropin® for Short Stature Associated With Noonan Syndrome
· enrolling by invitation
NCT04513171 — Safety and Efficacy of Y-shape Pegylated Somatropin in Growth Hormone Deficiency Children
· Phase 2, PHASE3
· completed
NCT03075644 — A Trial to Evaluate the Safety of Once Weekly Dosing of Somapacitan (NNC0195-0092) and Daily Norditropin® FlexPro® for 5
· Phase 3
· completed
NCT00663611 — Growth Hormone Administration and the Human Immune System
· Phase 1
· completed
Other recruiting trials for Short Stature
Currently open trials in the same condition.
NCT04219007 — Vosoritide for Selected Genetic Causes of Short Stature
· Phase 2
· active not recruiting
Other Children's Hospital Medical Center, Cincinnati trials
Trials by the same sponsor.
NCT07430332 — GLP-1 RA for Stage 1 Type 1 Diabetes
· Phase 2
· not yet recruiting
NCT07077213 — 18F-Fibroblast Activation Protein Inhibitor (18F-FAPI-74) in Tuberculosis Patients
· recruiting
NCT07265141 — An Automated Virtual Reality Intervention to Enhance Firearm Safety Counseling in Pediatrics
· NA
· not yet recruiting
NCT07217444 — Improving Outcomes in Adolescent Inpatient Depression With Deep Transcranial Magnetic Stimulation
· NA
· recruiting
NCT07195123 — Evaluation of the SMART IBD App in Pediatric IBD
· NA
· recruiting
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Children's Hospital Medical Center, Cincinnati
Last refreshed: 30 July 2024
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03288103.