21 completed, 9 recruiting, 7 active not recruiting, 4 terminated. 20 Phase 2, 8 Phase 3, 3 Phase 1, 1 Phase 4.
Follow Achondroplasia: new trials by email Literature Review of Achondroplasia trials Research Report on Achondroplasia
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Clinical Trial Evaluating the Efficacy and Safety of Navepegritide and Lonapegsomatropin in Achondroplasia Dual therapy with navepegritide and lonapegsomatropin, Dual therapy with navepegritide and placebo for lonapegsomatropin, Dual therapy with lonapegsomatropin and placebo for navepegritide - Achondroplasia | Phase 3 | Not yet recruiting | Ascendis Pharma A/S | 144 | 2026-11-01 | 2026-11-12 |
| Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia BMN 333, Vosoritide Injection [Voxzogo] - Achondroplasia | Phase 2 / Phase 3 | Recruiting | BioMarin Pharmaceutical | 160 | 2026-04-20 | 2026-10-22 |
| Prospective Longitudinal Multicentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia Achondroplasia, Hypochondroplasia | Recruiting | SYSNAV | 140 | 2026-02-06 | 2026-09-23 | |
| A Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children With Achondroplasia ABSK061 - Achondroplasia | Phase 1 / Phase 2 | Not yet recruiting | Abbisko Therapeutics Co, Ltd | 110 | 2025-12-10 | 2025-12-22 |
| Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH) Infigratinib is provided as a single dose of minitablets for oral administration, Infigratinib is provided as sprinkle capsules for daily oral administration, Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration - Achondroplasia | Phase 2 | Recruiting | QED Therapeutics, a BridgeBio company | 77 | 2025-11-19 | 2026-07-13 |
| A Study in Children With Achondroplasia No Interventions, complete a natural history observation of ACH for at least 6 months and up to 2 years - Achondroplasia | Recruiting | Abbisko Therapeutics Co, Ltd | 260 | 2025-06-20 | 2025-12-24 | |
| A Study of TYRA-300 in Children With Achondroplasia: BEACH301 TYRA-300 0.125 mg/kg, TYRA-300 0.25 mg/kg, TYRA-300 0.375 mg/kg - Achondroplasia | Phase 2 | Recruiting | Tyra Biosciences, Inc | 92 | 2025-03-04 | 2026-10-01 |
| A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia. Navepegritide, Placebo for navepegritide - Achondroplasia | Phase 2 | Active not recruiting | Ascendis Pharma A/S | 25 | 2024-12-10 | 2026-10-13 |
| Evaluate the Efficacy and Safety of KK8398 in Patients With Achondroplasia（AOBA Study） KK8398 - Achondroplasia | Phase 3 | Active not recruiting | Kyowa Kirin Co., Ltd. | 13 | 2024-12-01 | 2026-10-08 |
| A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia Combination of Navepegritide and Lonapegsomatropin administered as two separate s.c. injections - Achondroplasia | Phase 2 | Active not recruiting | Ascendis Pharma Growth Disorders A/S | 22 | 2024-07-26 | 2026-02-18 |
| A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia Navepegritide, Placebo for Navepegritide - Achondroplasia | Phase 2 | Recruiting | Ascendis Pharma A/S | 72 | 2024-01-23 | 2026-04-08 |
| A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia Infigratinib 0.25 mg/kg/day, Placebo Comparator 0.25 mg/kg/day - Achondroplasia | Phase 3 | Completed | QED Therapeutics, a BridgeBio company | 114 | 2023-11-10 | 2026-03-17 |
| A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia TransCon CNP - Achondroplasia | Phase 2 / Phase 3 | Enrolling by invitation | Ascendis Pharma Growth Disorders A/S | 140 | 2023-06-21 | 2026-08-17 |
| Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants BMN 111 administration via Injector Pen, BMN 111 administration via vial and syringe - Achondroplasia | Phase 1 | Terminated | BioMarin Pharmaceutical | 36 | 2023-03-15 | 2023-10-12 |
| A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia Navepegritide (TransCon CNP), Placebo for Navepegritide (TransCon CNP) - Achondroplasia | Phase 2 / Phase 3 | Completed | Ascendis Pharma Growth Disorders A/S | 84 | 2023-03-03 | 2026-07-14 |
| VIrtual STudy in Achondroplasia for the US (VISTA) Achondroplasia | Recruiting | BioMarin Pharmaceutical | 170 | 2023-02-21 | 2026-06-30 | |
| A Study to Learn About Recifercept in Patients With Achondroplasia Recifercept - Achondroplasia | Completed | Pfizer | 248 | 2022-10-19 | 2024-09-20 | |
| A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia Recombinant human growth hormone - Achondroplasia | Phase 4 | Completed | Changchun GeneScience Pharmaceutical Co., Ltd. | 39 | 2022-08-20 | 2026-08-06 |
| Adaption and Testing of the Quality of Life in Short Stature Youth (QoLISSY) Questionnaire for Parents With Children From 0-4 Questionnaire - Achondroplasia, Small for Gestational Age | Unknown | Universitätsklinikum Hamburg-Eppendorf | 168 | 2022-03-01 | 2022-11-03 | |
| A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Multiple Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children With Achondroplasia TransCon CNP, Placebo for TransCon CNP - Achondroplasia | Phase 2 | Completed | Visen Pharmaceuticals (Shanghai) Co., Ltd. | 24 | 2022-01-05 | 2026-08-10 |
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Dwarfism (19) · Bone Diseases (14) · Musculoskeletal Diseases (12) · Genetic Diseases, Inborn (11) · Osteochondrodysplasias (10) · Mucopolysaccharidosis IV (7) · Bone Diseases, Developmental (6) · Hypochondroplasia (3) · Dwarfism, Pituitary (1) · Growth Disorders (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (47) · Bone Diseases, Developmental (41) · Osteochondrodysplasias (37) · Genetic Diseases, Inborn (36) · Musculoskeletal Diseases (35) · Bone Diseases (33) · Dwarfism (28) · Endocrine System Diseases (20) · Carbohydrate Metabolism, Inborn Errors (7) · Connective Tissue Diseases (7) · Lysosomal Storage Diseases (7) · Metabolic Diseases (7) · Metabolism, Inborn Errors (7) · Mucinoses (7) · Mucopolysaccharidoses (7)
vosoritide (9) · infigratinib (4) · Human Growth Hormone (2) · Administration, Oral (1) · Growth Hormone (1) · Registries (1) · Saline Solution (1) · Surveys and Questionnaires (1) · Syringes (1) · lonapegsomatropin (1)
BioMarin Pharmaceutical (14) · Ascendis Pharma A/S (5) · QED Therapeutics, a BridgeBio company (5) · Pfizer (4) · Ascendis Pharma Growth Disorders A/S (3) · Abbisko Therapeutics Co, Ltd (2) · Novo Nordisk A/S (2) · Changchun GeneScience Pharmaceutical Co., Ltd. (1) · Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico (1) · Hospices Civils de Lyon (1) · Johns Hopkins University (1) · Kyowa Kirin Co., Ltd. (1) · National Human Genome Research Institute (NHGRI) (1) · Nemours Children's Clinic (1) · SYSNAV (1)
United States (29) · Australia (23) · United Kingdom (22) · Spain (16) · France (14) · Canada (13) · Denmark (11) · Italy (11) · Japan (11) · Germany (10) · Ireland (7) · Portugal (7) · China (6) · Norway (6) · Austria (5)
Doug Warner, MD (1) · Feihong Luo (1) · Genevieve Baujat (1) · Guimei Li (1) · Jin Wu (1) · Julia Quitmann (1) · Julie Hoover-Fong, MD,PhD (1) · Massimiliano Rossi, MD (1)
PMID 38649657, PMID 31269546, PMID 34431071, PMID 39740666, PMID 35342457, PMID 38685585, PMID 39305160, PMID 42144862, PMID 25387261, PMID 33204020, PMID 33761804, PMID 33827611, PMID 34736503, PMID 36107167, PMID 36922864, PMID 37823031, PMID 37984383, PMID 39555818, PMID 41247754, PMID 41783511, PMID 42319555, PMID 42370681
2015 (2), 2017 (2), 2016 (1)