142 completed, 55 recruiting, 39 terminated, 30 unknown. 83 Phase 2, 50 Not applicable, 37 Phase 1, 36 Phase 3, 22 Phase 4.
Follow Osteochondrodysplasias: new trials by email Literature Review of Osteochondrodysplasias trials Research Report on Osteochondrodysplasias
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 |
|---|---|---|---|---|---|---|
| Mesenchymal Cell Therapy in Osteogenesis Imperfecta (OI) Bone marrow-derived mesenchymal stromal cells (MSCs) - Osteogenesis Imperfecta, Osteogenesis Imperfecta Type III | Phase 1 / Phase 2 | Not yet recruiting | Emory University | 12 | 2027-01-01 | 2026-05-08 |
| Morbidity in Adults With Craniofacial Fibrous Dysplasia: The CRANIO-DF Study No Intervention: Observational Cohort - Craniofacial Fibrous Dysplasia | Not yet recruiting | Centre Hospitalier Universitaire de Nice | 500 | 2026-12-01 | 2026-10-13 | |
| 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 |
| A Study Evaluating Ficerafusp Alfa (BCA101) QW in Combination With Pembrolizumab vs Alternative Ficerafusp Alfa Dosing in 1L R or M HNSCC Ficerafusp alfa, Pembrolizumab (KEYTRUDA®) - Metastatic Head and Neck Squamous Cell Carcinoma, Recurrent Head and Neck Squamous Cell Carcinoma | Phase 2 | Recruiting | Bicara Therapeutics | 160 | 2026-09-01 | 2026-10-13 |
| Morphology in Oral Rare Syndromes & Artificial Intelligence for Clinical Diagnosis intra-oral 3D optical impression - Osteogenesis Imperfecta, Rare Bone Disorders | Not applicable | Not yet recruiting | University Hospital, Bordeaux | 240 | 2026-09-01 | 2026-06-24 |
| GRACE II (General Retrospective Analysis of Commercial Experience With AGN1 LOEP) AGN1 LOEP - Osteoporosis | Enrolling by invitation | AgNovos Healthcare, LLC | 305 | 2026-08-25 | 2026-10-30 | |
| A Dose REgimen-Finding Study of AGA2115 in Chinese Patients With Osteogenesis ImpeRfecta (EIR) AGA2115 - Osteogenesis Imperfecta (OI) | Phase 2 | Recruiting | Angitia Biopharmaceuticals Guangzhou Limited | 48 | 2026-07-14 | 2026-09-22 |
| Gene-Modified Stem Cell Therapy for Children With Autosomal Recessive Osteopetrosis (ARO) FT024 - Osteopetrosis | Phase 1 / Phase 2 | Recruiting | Fondazione Telethon | 8 | 2026-06-20 | 2026-06-24 |
| The Role of Transforming Growth Factor- Beta (TGF- β) Expression in Renal Biopsy of Lupus Nephritis Patients renal biopsy - Lupus Nephritis (LN) | Not applicable | Active not recruiting | Ain Shams University | 30 | 2026-05-31 | 2026-09-16 |
| Clinical Evaluation of Patient-Specific 3D-Printed Titanium Implants for Facial Reconstruction. Patient-specific titanium implants for facial reconstruction - Hemifacial Microsomia, Mucormycosis | Not applicable | Recruiting | Latakia University | 30 | 2026-04-26 | 2026-06-17 |
| 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 |
| 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 |
| Physiotherapy and Rehabilitation in Melorheostosis: A Case Report Assigned Interventions - Melorheostosis | Not applicable | Active not recruiting | Yeditepe University | 1 | 2026-04-18 | 2026-06-26 |
| PAINDYS_Characterizing Pain in Fibrous Dysplasia of Bone/McCune-Albright Syndrome: an Exploratory Pilot Study Visit 1 at the center - Fibrous Dysplasia of Bone, McCune Albright Syndrome | Not applicable | Completed | University Hospital, Clermont-Ferrand | 39 | 2026-04-08 | 2026-08-21 |
| Pediatric Safety Follow-up Study of Prior Treatment With Romosozumab for Osteogenesis Imperfecta Vitamin D, Calcium - Osteogenesis Imperfecta | Phase 3 | Recruiting | Amgen | 71 | 2026-03-18 | 2026-06-22 |
| Effects of Non-Surgical Periodontal Treatment on Inflammatory and Bone Markers in Periodontitis Patients Scaling and Root Planing (SRP) - Periodontitis (Stage 3) | Not applicable | Active not recruiting | Saglik Bilimleri Universitesi | 56 | 2026-03-15 | 2026-04-27 |
| 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 | |
| MDR Vivacit-E Elevated Study Vivacit-E Elevated Liners - Osteoarthritis (OA) of the Hip, Avascular Necrosis of Bone of Hip | Enrolling by invitation | Zimmer Biomet | 52 | 2026-01-07 | 2026-04-01 | |
| Osteogenesis Imperfecta Trial of AGA2115 for ADUlts With COL1A1 and/or COL1A2 GeNetic Variations (IDUN) AGA2115, Placebo - Osteogenesis Imperfecta (OI) | Phase 2 | Recruiting | Angitia Incorporated Limited | 80 | 2025-12-12 | 2026-11-04 |
| 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 |
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Osteogenesis Imperfecta (84) · Camurati-Engelmann Syndrome (58) · Achondroplasia (47) · Bone Diseases (24) · Dwarfism (24) · Fibrous Dysplasia of Bone (24) · Fibrous Dysplasia, Polyostotic (23) · Osteopetrosis (22) · Genetic Diseases, Inborn (19) · Musculoskeletal Diseases (19) · Slipped Capital Femoral Epiphyses (19) · Osteoarthritis (16) · Mucopolysaccharidosis IV (15) · Collagen Diseases (14)
Bone Diseases, Developmental (305) · Musculoskeletal Diseases (294) · Bone Diseases (289) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (240) · Genetic Diseases, Inborn (205) · Skin and Connective Tissue Diseases (128) · Connective Tissue Diseases (117) · Collagen Diseases (83) · Pathological Conditions, Signs and Symptoms (53) · Pathologic Processes (43) · Metabolic Diseases (42) · Neoplasms (42) · Nutritional and Metabolic Diseases (42) · Endocrine System Diseases (40)
vosoritide (10) · setrusumab (7) · Blood Specimen Collection (6) · Denosumab (6) · Zoledronic Acid (6) · infigratinib (6) · Busulfan (5) · Cyclophosphamide (5) · Pamidronate (5) · Alemtuzumab (4) · Bone Marrow Transplantation (4) · Diphosphonates (4) · Radiotherapy (4) · Risedronic Acid (4) · Vitamin D (4)
BioMarin Pharmaceutical (15) · DePuy International (13) · Hospices Civils de Lyon (11) · Istituto Ortopedico Rizzoli (10) · National Institute of Dental and Craniofacial Research (NIDCR) (9) · Baylor College of Medicine (8) · QED Therapeutics, a BridgeBio company (8) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (7) · Ultragenyx Pharmaceutical Inc (7) · Assiut University (6) · Amgen (5) · Ascendis Pharma A/S (5) · Pfizer (5) · St. Jude Children's Research Hospital (4) · University of British Columbia (4)
United States (148) · France (58) · United Kingdom (49) · Italy (48) · Australia (41) · Canada (41) · Spain (35) · Germany (33) · Japan (22) · China (18) · Turkey (Türkiye) (17) · Belgium (16) · Denmark (16) · Egypt (14) · Poland (14)
Alison M Boyce, M.D. (4) · Christine Alvarez, MD (3) · Frank Rauch, M.D. (3) · Joshua J Zimmerberg, M.D. (3) · Hesaham M Elbaseet, MD (2) · Jeffrey Krischer, Ph.D. (2)
PMID 38649657, PMID 42470570, PMID 31269546, PMID 34431071, PMID 39740666, PMID 32461347, PMID 35342457, PMID 38685585, PMID 39305160, PMID 42144862, PMID 10535539, PMID 10589640, PMID 10831929, PMID 11288855, PMID 11381256, PMID 11415615, PMID 11415789, PMID 11479750, PMID 11676731, PMID 11834520, PMID 12119276, PMID 12525569, PMID 15579374, PMID 15955304, PMID 16088908, PMID 16183806, PMID 16601194, PMID 16603886, PMID 16622779, PMID 17099022, PMID 17188619, PMID 17218256, PMID 17229001, PMID 17911499, PMID 18547784, PMID 18804556, PMID 19083701, PMID 19430350, PMID 20117257, PMID 20157193, PMID 20200960, PMID 20458640, PMID 21244705, PMID 21765218, PMID 21904698, PMID 22210669, PMID 22258776, PMID 22530188, PMID 22640403, PMID 22640754
2016 (4), 2017 (4), 2023 (4), 2015 (3), 2018 (2), 2019 (2), U01TR001263 (2), 2005 (1), 2014 (1), P01CA094237 (1), UL1RR024140 (1)