5 recruiting, 3 enrolling by invitation, 2 active not recruiting, 2 completed. 5 Phase 2, 3 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 5 | Enrolling by invitation 2, Active not recruiting 1, Completed 1, Recruiting 1 |
| Phase 3 | 3 | Enrolling by invitation 2, Active not recruiting 1 |
Follow Hypochondroplasia: new trials by email Literature Review of Hypochondroplasia trials Research Report on Hypochondroplasia
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 |
|---|---|---|---|---|---|---|
| Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia Infigratinib - Hypochondroplasia | Phase 2 | Enrolling by invitation | QED Therapeutics, a BridgeBio company | 135 | 2026-04-23 | 2026-08-26 |
| 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 Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months Vosoritide, Placebo - Hypochondroplasia | Phase 2 | Recruiting | BioMarin Pharmaceutical | 60 | 2025-07-30 | 2026-03-31 |
| Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia Vosoritide - Hypochondroplasia | Phase 3 | Enrolling by invitation | BioMarin Pharmaceutical | 140 | 2025-06-20 | 2026-09-09 |
| An Interventional Study of Infigratinib in Children With Hypochondroplasia infigratinib 0.128 mg/kg/day, infigratinib 0.25 mg/kg/day - Hypochondroplasia | Phase 2 / Phase 3 | Enrolling by invitation | QED Therapeutics, a BridgeBio company | 24 | 2025-04-22 | 2026-07-16 |
| Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia Vosoritide, Placebo - Hypochondroplasia | Phase 3 | Active not recruiting | BioMarin Pharmaceutical | 80 | 2024-06-17 | 2026-01-20 |
| Prospective Clinical Assessment Study in Children With Hypochondroplasia Hypochondroplasia | Recruiting | QED Therapeutics, a BridgeBio company | 150 | 2024-06-05 | 2026-07-16 | |
| A Multicenter Multinational Observational Study of Children With Hypochondroplasia Hypochondroplasia | Recruiting | BioMarin Pharmaceutical | 400 | 2023-11-27 | 2025-04-03 | |
| Registry for Patients With Achondroplasia / Hypochondroplasia (OMPR-Ach/Hy) Registry - Achondroplasia, Hypochondroplasia | Recruiting | Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico | 200 | 2021-09-01 | 2022-04-28 | |
| Vosoritide for Selected Genetic Causes of Short Stature Vosoritide - Short Stature | Phase 2 | Active not recruiting | Andrew Dauber | 56 | 2020-08-04 | 2026-03-18 |
| C-Type Natriuretic Peptide and Achondroplasia Achondroplasia, Hypochondroplasia | Completed | Nemours Children's Clinic | 91 | 2012-02-01 | 2015-04-14 | |
| Efficacy and Safety Evaluation of Recombinant Human Growth Hormone (r-hGH), Saizen®, on a Population of Children With Hypochondroplasia, Treated at Least 3 Years or Until Near Final Height, When Applicable, in Comparison With a Historic Cohort of Non-treated Children Recombinant human growth hormone (r-hGH) - Hypochondroplasia | Phase 2 | Completed | Merck KGaA, Darmstadt, Germany | 19 | 2006-03-21 | 2019-02-15 |
Dwarfism (6) · Bone Diseases (4) · Genetic Diseases, Inborn (4) · Mucopolysaccharidosis IV (4) · Musculoskeletal Diseases (4) · Osteochondrodysplasias (4) · Achondroplasia (3) · Bone Diseases, Developmental (1) · Noonan Syndrome (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (9) · Bone Diseases, Developmental (7) · Endocrine System Diseases (6) · Connective Tissue Diseases (5) · Genetic Diseases, Inborn (5) · Skin and Connective Tissue Diseases (5) · Bone Diseases (4) · Carbohydrate Metabolism, Inborn Errors (4) · Lysosomal Storage Diseases (4) · Metabolic Diseases (4) · Metabolism, Inborn Errors (4) · Mucinoses (4) · Mucopolysaccharidoses (4) · Musculoskeletal Diseases (4) · Nutritional and Metabolic Diseases (4)
vosoritide (4) · infigratinib (2) · Growth Hormone (1) · Human Growth Hormone (1) · Registries (1)
BioMarin Pharmaceutical (4) · QED Therapeutics, a BridgeBio company (3) · Andrew Dauber (1) · Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico (1) · Merck KGaA, Darmstadt, Germany (1) · Nemours Children's Clinic (1) · SYSNAV (1)
France (9) · United States (9) · United Kingdom (8) · Australia (7) · Canada (6) · Spain (6) · Italy (5) · Germany (4) · Japan (4) · Norway (3) · Portugal (3) · Singapore (3) · Sweden (3) · Argentina (1) · Belgium (1)
Andrew Dauber, MS MMSc (1) · Genevieve Baujat (1) · Michael Bober, MD, PhD (1) · Robert Olney, MD (1)