44 completed, 14 terminated, 9 active not recruiting, 6 recruiting. 85 Phase 2, 15 Phase 3, 7 Phase 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 |
| 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 |
| A Study of GS3-007a for Oral Suspension in the Diagnosis of Adult Growth Hormone Deficiency (AGHD) GS3-007a dry suspension, Insulin - Adult Growth Hormone Deficiency (AGHD) | Phase 2 | Terminated | Changchun GeneScience Pharmaceutical Co., Ltd. | 120 | 2025-12-19 | 2026-05-22 |
| 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 |
| Evaluating the Efficacy and Safety of GB08 Injection in Pediatric Patients With Growth Hormone Deficiency GB08, Norditropin NordiFlex - Growth Hormone Deficiency in Children, Growth Hormone Deficiency (GHD) | Phase 2 / Phase 3 | Not yet recruiting | Shenzhen Kexing Pharmaceutical Co., Ltd. | 268 | 2025-08-31 | 2025-08-22 |
| 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 |
| 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 |
| 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 |
| 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 |
| Vosoritide for Short Stature in Turner Syndrome Vosoritide - Turner Syndrome, Short Stature | Phase 2 | Unknown | Roopa Kanakatti Shankar, MBBS, MS | 20 | 2024-04-12 | 2024-06-24 |
| 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 |
| A Long-Term Safety Trial of LUM-201 in Children With Idiopathic Growth Hormone Deficiency Who Have Previously Completed a LUM-201 Clinical Trial (OraGrowtH211) LUM-201 - Growth Hormone Deficiency | Phase 2 | Enrolling by invitation | Lumos Pharma | 60 | 2023-08-01 | 2024-02-15 |
| 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 |
| 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 |
| Phase 2 Study of LUM-201 in Children Who Have Previously Completed the LUM-201-01 Trial (OraGrowtH213) LUM-201 - Growth Hormone Deficiency | Phase 2 | Completed | Lumos Pharma | 15 | 2022-02-18 | 2026-05-15 |
| A Trial of YPEG-rhGH in Children With Short Stature YPEG-rhGH, rhGH - Idiopathic Short Stature, Small for Gestational Age | Phase 2 | Completed | Xiamen Amoytop Biotech Co., Ltd. | 78 | 2022-02-15 | 2024-01-24 |
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Dwarfism, Pituitary (44) · Achondroplasia (20) · Bone Diseases (13) · Musculoskeletal Diseases (11) · Genetic Diseases, Inborn (8) · Osteochondrodysplasias (8) · Mucopolysaccharidosis IV (7) · Bone Diseases, Developmental (4) · Growth Disorders (4) · Hypochondroplasia (3) · Hypopituitarism (3) · Turner Syndrome (3) · Laron Syndrome (2) · Noonan Syndrome (2)
Bone Diseases, Developmental (81) · Endocrine System Diseases (76) · Musculoskeletal Diseases (74) · Bone Diseases (72) · Nervous System Diseases (46) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (45) · Bone Diseases, Endocrine (44) · Brain Diseases (44) · Central Nervous System Diseases (44) · Hypothalamic Diseases (44) · Pituitary Diseases (44) · Hypopituitarism (41) · Genetic Diseases, Inborn (37) · Osteochondrodysplasias (14)
Human Growth Hormone (20) · Growth Hormone (11) · vosoritide (9) · infigratinib (5) · Growth Hormone-Releasing Hormone (3) · GX-H9 (2) · MOD-4023 (2) · albusomatropin (2) · mecasermin (2) · Administration, Oral (1) · Amitriptyline (1) · Anastrozole (1) · Control Groups (1) · Fluoxetine (1) · Injections (1)
BioMarin Pharmaceutical (7) · Ascendis Pharma A/S (5) · Changchun GeneScience Pharmaceutical Co., Ltd. (5) · QED Therapeutics, a BridgeBio company (5) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (4) · Lumos Pharma (4) · Pfizer (4) · Versartis Inc. (4) · Ascendis Pharma Growth Disorders A/S (3) · Xiamen Amoytop Biotech Co., Ltd. (3) · Altus Pharmaceuticals (2) · Children's Hospital Medical Center, Cincinnati (2) · Federico II University (2) · Genexine, Inc. (2) · Nemours Children's Clinic (2)
United States (48) · Australia (23) · United Kingdom (20) · France (14) · Spain (14) · Canada (12) · Denmark (12) · Germany (12) · China (11) · Italy (10) · Hungary (7) · Portugal (7) · Ireland (6) · Israel (6) · Norway (6)
Dariush Elahi, PhD (2) · Nelly Mauras, MD (2) · Philippe Backeljauw, MD (2) · Will Charlton, MD (2) · Xiaoping Luo (2) · Albert Nadal, MD (1) · Andrew Dauber, MS MMSc (1) · Antonio Cittadini, MD (1) · Cecilia Lansang, MD (1) · Dana S Hardin, MD (1) · Daniela Rogoff, MD, PhD (1)
PMID 38649657, PMID 31269546, PMID 42144862, PMID 17192294, PMID 18053120, PMID 19001519, PMID 19654233, PMID 20961566, PMID 21177789, PMID 23015655, PMID 24178787, PMID 24291224, PMID 24423356, PMID 28201598, PMID 28566441, PMID 29029190, PMID 30339244, PMID 33761804, PMID 34431071, PMID 34922359, PMID 35001504, PMID 35342457, PMID 36034448, PMID 36646053, PMID 36994923, PMID 37823031, PMID 37984383, PMID 38685585, PMID 38813446, PMID 39555818, PMID 39906034, PMID 41247754, PMID 41783511, PMID 42319555
2008 (1), 2015 (1), 2016 (1), P01AG000599 (1), R01AR050618 (1), R01HL085268 (1)