Achondroplasia clinical trials
The registry lists 47 studies for Achondroplasia across more than 10 countries. 13 are recruiting or about to start. Per ClinicalTrials.gov, last updated 2026-11-09.
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Status
Who runs them
BioMarin Pharmaceutical (14) · QED Therapeutics, a BridgeBio company (5) · Ascendis Pharma A/S (5) · Pfizer (4) · Ascendis Pharma Growth Disorders A/S (3) · Abbisko Therapeutics Co, Ltd (2) · Novo Nordisk A/S (2) · SYSNAV (1)
What is being tested here
Drugs under test in Achondroplasia studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| Navepegritide | 2 | 2 | 6 |
| Infigratinib | 1 | 1 | 3 |
| ABSK061 | 1 | 0 | 1 |
| Dabogratinib | 1 | 0 | 1 |
| Vosoritide | 0 | 2 | 9 |
| Recifercept | 0 | 0 | 2 |
| Human Growth Hormone | 0 | 0 | 1 |
Drug classes tested here Tyrosine kinase inhibitors (TKIs) 2 open
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
The study
Who these studies take
Of the 9 Achondroplasia studies recruiting now, 2 (22%) set no upper age limit and 7 (78%) stop at 75 or younger. 9 (100%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 0 (0%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 7 (78%) | 7 (78%) |
| 18 | 4 (44%) | 4 (44%) |
| 30 | 3 (33%) | 3 (33%) |
| 40 | 3 (33%) | 3 (33%) |
| 50 | 3 (33%) | 3 (33%) |
| 65 | 3 (33%) | 3 (33%) |
| 75 | 2 (22%) | 2 (22%) |
| 85 | 2 (22%) | 2 (22%) |
Across all 47 studies, recruiting or not: 77% stop at 75 or younger and 74% at 65; 94% take children; 0% are for women only and 2% for men only; 4% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Oct 13, 2026.
Newly registered1
Studies
Recruiting first, then most recently updated. Showing 47 of 47. search them.
- Phase 3 Clinical Trial Evaluating the Efficacy and Safety of Navepegritide and Lonapegsomatropin in AchondroplasiaNot yet recruitingNCT07924059Phase 3
- Extension Study of Infigratinib in Children With Achondroplasia (ACH)Enrolling by invitationNCT05145010Phase 210 countries
- Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With AchondroplasiaRecruitingNCT07441876Phase 2/39 countries
- A Study of TYRA-300 in Children With Achondroplasia: BEACH301RecruitingNCT06842355Phase 28 countries
- Prospective Longitudinal Multicentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal DysplasiaRecruitingNCT073889663 countries
- A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With AchondroplasiaEnrolling by invitationNCT05929807Phase 2/312 countries
- Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)RecruitingNCT07169279Phase 27 countries
- VIrtual STudy in Achondroplasia for the US (VISTA)RecruitingNCT061682011 country
- A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With AchondroplasiaRecruitingNCT06079398Phase 215 countries
- Achondroplasia Natural History Multicenter Clinical StudyRecruitingNCT025978811 country
- A Study in Children With AchondroplasiaRecruitingNCT073014631 country
- A Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children With AchondroplasiaNot yet recruitingNCT07297875Phase 1/21 country
- Registry for Patients With Achondroplasia / Hypochondroplasia (OMPR-Ach/Hy)RecruitingNCT053280501 country
- A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.Active, not recruitingNCT06732895Phase 25 countries
- Evaluate the Efficacy and Safety of KK8398 in Patients With Achondroplasia（AOBA Study）Active, not recruitingNCT06926491Phase 31 country
- A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Multiple Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children With AchondroplasiaCompletedNCT05246033Phase 21 country
- A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With AchondroplasiaCompletedNCT05353192Phase 41 country
- A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With AchondroplasiaCompletedNCT05598320Phase 2/37 countries
- A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With AchondroplasiaCompletedNCT04085523Phase 28 countries
- Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)CompletedNCT0403581111 countries
- A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With AchondroplasiaCompletedNCT06164951Phase 310 countries
- A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With AchondroplasiaActive, not recruitingNCT04554940Phase 22 countries
- An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With AchondroplasiaActive, not recruitingNCT03424018Phase 37 countries
- An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With AchondroplasiaActive, not recruitingNCT03989947Phase 24 countries
- A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)Active, not recruitingNCT02724228Phase 24 countries
- A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With AchondroplasiaActive, not recruitingNCT06433557Phase 23 countries
- Study of Infigratinib in Children With AchondroplasiaCompletedNCT04265651Phase 26 countries
- Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.TerminatedNCT0379460914 countries
- A Study to Learn About Recifercept in Patients With AchondroplasiaCompletedNCT056597191 country
- A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With AchondroplasiaCompletedNCT03583697Phase 24 countries
- A Multi-center, Longitudinal, Observational Study of Children With AchondroplasiaCompletedNCT0387553415 countries
- A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With AchondroplasiaTerminatedNCT04638153Phase 28 countries
- Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in AchondroplasiaTerminatedNCT05116046Phase 27 countries
- Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy ParticipantsTerminatedNCT05813314Phase 11 country
- Adaption and Testing of the Quality of Life in Short Stature Youth (QoLISSY) Questionnaire for Parents With Children From 0-4Status unknownNCT056039361 country
- The Norwegian Adult Achondroplasia StudyCompletedNCT037801531 country
- A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With AchondroplasiaCompletedNCT03197766Phase 37 countries
- A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With AchondroplasiaCompletedNCT016030956 countries
- Lifetime Impact of Achondroplasia Study in Europe-LIAISECompletedNCT034493686 countries
- Lifetime Impact Study for AchondroplasiaCompletedNCT038725313 countries
- A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With AchondroplasiaCompletedNCT02055157Phase 24 countries
- Radiological Analysis on Patients With Achondroplasia DisorderStatus unknownNCT041848171 country
- A Survey Collecting Data on Adult Height in Patients With Achondroplasia Treated With SomatropinCompletedNCT014356291 country
- Special Survey for Long Term ApplicationCompletedNCT015162291 country
- C-Type Natriuretic Peptide and AchondroplasiaCompletedNCT015413061 country
- A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult VolunteersCompletedNCT01590446Phase 11 country
- Issues Surrounding Prenatal Genetic Testing for AchondroplasiaCompletedNCT000015361 country