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Home › Conditions › achondroplasia

Achondroplasia

Part of Osteochondrodysplasias

2 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for achondroplasia

5 trials tracked for achondroplasia: 1 in phase 3 or 4 and 1 with published results. The most-cited published study has 43 citations.

5Trials tracked
1Phase 3 & 4
0Recruiting
1With published results
Phase distribution
Phase 3 1 Phase 2 3 Other / NA 1
  1. Phase 3 A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia Completed · 43 cited
  2. Phase 2 A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia Completed
  3. Phase 2 A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia Completed
  4. Phase 2 A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia Completed
  5. N/A A Study to Learn About Recifercept in Patients With Achondroplasia Completed

Showing the 5 most-cited and recently-updated of 5 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

Achondroplasia: what the trials found

Treatment with BMN 111 demonstrated a statistically significant increase in Annualized Growth Velocity (AGV) from baseline at week 52 (p < 0.0001) 1. Additionally, patients receiving BMN 111 showed a statistically significant improvement in Height Z-score at week 52 (p < 0.0001) 1.

Clinical evaluations of body proportions using BMN 111 showed no statistically significant change in the Upper to Lower Segment Body Ratio at week 52 (p = 0.506) 1. Other assessments of body proportion ratios, including Arm Span to Height, Upper Arm Length to Forearm Length, and Thigh to Tibia length, did not show statistically significant changes from baseline 2.

Data regarding BMN 111 also indicated consistent results across different study cohorts for upper-to-lower body ratio measurements during the initial 6 months and throughout the full study period 4. Safety data for BMN 111 noted various treatment-emergent adverse events (TEAEs) across trial participants 2.

Recent results — preliminary, needs further review

  • TransCon CNP showed no statistically significant difference in Annualized Height Velocity after 52 weeks of double-blind treatment (p = 0.6004) 3.
  • Recifercept was evaluated for changes in Arm Span to Height/Length, Knee Height: Lower Segment Ratio, and Height Z-score over a 12-month period, though these results are not yet corroborated 5.

For the clinician treating this condition

  • BMN 111 is associated with statistically significant improvements in both Annualized Growth Velocity and Height Z-score at 52 weeks 1.
  • While BMN 111 impacts growth velocity, it did not result in statistically significant changes to the Upper to Lower Segment Body Ratio or other specific limb proportion ratios [1, 2].

AI synthesis of 5 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.
Middle East Consensus Sets 25 Statements for Achondroplasia Care and Access
Diabetes & Endocrinology Guideline
Middle East Consensus Sets 25 Statements for Achondroplasia Care and Access New Guidelines Improve Care for Children with Achondroplasia
This consensus statement, developed for the Middle East, provides 25 statements on diagnosing and managing achondroplasia, including surveil…
New guidelines aim to standardize care for achondroplasia in the Middle East, focusing on early detection, family support, and access to new…
Frontiers Sep 17, 2026
Systematic review and meta-analysis of vosoritide in children with achondroplasia shows growth gains and injection site reactions
Pediatrics Meta-analysis
Systematic review and meta-analysis of vosoritide in children with achondroplasia shows growth gains and injection site reactions New Treatment Linked to Faster Growth in Children With Achondroplasia
This systematic review and single-arm meta-analysis evaluated vosoritide in children with genetically confirmed achondroplasia across 13 stu…
A new review shows a specific drug can help children with achondroplasia grow taller over one year.
Apr 24, 2026