4 active not recruiting. 4 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Active not recruiting 4 |
Follow Musculoskeletal Abnormalities: new trials by email Literature Review of Musculoskeletal Abnormalities trials Research Report on Musculoskeletal Abnormalities
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 |
|---|---|---|---|---|---|---|
| A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment Vosoritide Injection - Noonan Syndrome | Phase 2 | Active not recruiting | BioMarin Pharmaceutical | 30 | 2024-11-22 | 2026-09-23 |
| MK-7075 (Miransertib) in Proteus Syndrome MK-7075 (miransertib) - Proteus Syndrome | Phase 2 | Active not recruiting | National Human Genome Research Institute (NHGRI) | 38 | 2022-05-20 | 2026-07-17 |
| 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 |
| Efficacy of Amicar for Children Having Craniofacial Surgery Amicar (ε-aminocaproic acid), normal saline - Craniosynostosis | Phase 2 | Active not recruiting | Children's National Research Institute | 22 | 2014-10-01 | 2026-04-07 |
Dwarfism (2) · Noonan Syndrome (2) · Bone Diseases (1) · Craniosynostoses (1) · Hypochondroplasia (1) · Musculoskeletal Diseases (1) · Proteus Syndrome (1)
Bone Diseases, Developmental (4) · Congenital Abnormalities (4) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Bone Diseases (3) · Craniofacial Abnormalities (3) · Musculoskeletal Diseases (3) · Cardiovascular Abnormalities (2) · Cardiovascular Diseases (2) · Connective Tissue Diseases (2) · Endocrine System Diseases (2) · Genetic Diseases, Inborn (2) · Heart Defects, Congenital (2) · Heart Diseases (2) · Skin and Connective Tissue Diseases (2)
vosoritide (2) · Aminocaproic Acid (1) · Miransertib (1) · Saline Solution (1)
Andrew Dauber (1) · BioMarin Pharmaceutical (1) · Children's National Research Institute (1) · National Human Genome Research Institute (NHGRI) (1)
United States (4) · Australia (1) · Canada (1) · France (1) · Germany (1) · Italy (1) · Spain (1)
Andrew Dauber, MS MMSc (1) · Leslie G Biesecker, M.D. (1) · Srijaya K Reddy, MD, MBA (1)