6 recruiting. 6 Phase 2, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 6 | Recruiting 6 |
| Phase 3 | 1 | Recruiting 1 |
Follow Dwarfism: new trials by email Literature Review of Dwarfism trials Research Report on Dwarfism
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 |
|---|---|---|---|---|---|---|
| Safety, Pharmacokinetic (PK) Profile and Efficacy of Inpegsomatropin Injection in Adults With Growth Hormone Deficiency Inpegsomatropin Injection, Placebo for Inpegsomatropin Injection - Adult Growth Hormone Deficiency | Phase 2 | Recruiting | Xiamen Amoytop Biotech Co., Ltd. | 36 | 2026-06-10 | 2026-07-22 |
| 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 |
| 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 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 |
| Growth Hormone Replacement in Veterans With GWI and AGHD (GWIT) Recombinant human growth hormone - Gulf War Syndrome, Adult Growth Hormone Deficiency | Phase 2 | Recruiting | Baylor College of Medicine | 20 | 2024-03-11 | 2026-05-08 |
| 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 |
Achondroplasia (4) · Bone Diseases (2) · Dwarfism, Pituitary (2) · Musculoskeletal Diseases (2) · Osteochondrodysplasias (2) · Genetic Diseases, Inborn (1) · Growth Disorders (1) · Mucopolysaccharidosis IV (1) · Persian Gulf Syndrome (1)
Bone Diseases, Developmental (6) · Bone Diseases (4) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Musculoskeletal Diseases (4) · Endocrine System Diseases (3) · Genetic Diseases, Inborn (3) · Bone Diseases, Endocrine (2) · Brain Diseases (2) · Central Nervous System Diseases (2) · Hypopituitarism (2) · Hypothalamic Diseases (2) · Nervous System Diseases (2) · Osteochondrodysplasias (2) · Pituitary Diseases (2)
Administration, Oral (1) · Growth Hormone (1) · Human Growth Hormone (1) · infigratinib (1) · vosoritide (1)
Ascendis Pharma A/S (1) · Baylor College of Medicine (1) · BioMarin Pharmaceutical (1) · QED Therapeutics, a BridgeBio company (1) · Tyra Biosciences, Inc (1) · Xiamen Amoytop Biotech Co., Ltd. (1)
United States (5) · Australia (4) · Canada (4) · United Kingdom (4) · France (2) · Italy (2) · Norway (2) · Spain (2) · Sweden (2) · Austria (1) · China (1) · Denmark (1) · Finland (1) · Germany (1) · Ireland (1)
Doug Warner, MD (1) · Ricardo Jorge, MD (1) · Yiming Li (1)