232 completed, 47 unknown, 41 terminated, 40 recruiting. 85 Phase 2, 80 Phase 3, 52 Not applicable, 37 Phase 4, 26 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 |
|---|---|---|---|---|---|---|
| The AgRP and GH/IGF-1 Axis in Children Growth Hormone Deficiency | Not yet recruiting | Columbia University | 156 | 2027-06-10 | 2026-09-25 | |
| Evaluation of an Intensified Systematic Screening for Congenital Hypothyroidism in Premature Newborns Blood test - Congenital Hypothyroidism | Not yet recruiting | University Hospital, Lille | 1600 | 2027-03-01 | 2026-05-22 | |
| Phase 3 Long Term Safety Extension Study of LUM-201 in Children With Growth Hormone Deficiency LUM-201 - Growth Hormone Deficiency (GHD) | Phase 3 | Not yet recruiting | Lumos Pharma | 150 | 2027-02-01 | 2026-07-08 |
| Evaluation of (Cardio)Metabolic and Auxological Outcomes of GHD Patients Under rhGH or LAGH With Stratification According to IGF-1 Levels metabolic and auxological outcomes - Growth Hormone Deficiency | Not applicable | Not yet recruiting | Cliniques universitaires Saint-Luc- Université Catholique de Louvain | 300 | 2026-07-01 | 2026-05-11 |
| 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 |
| GB06 Phase III Trial for Pediatric Growth Hormone Deficiency GB06, Norditropin®FlexProTM - Growth Hormone Deficiency (GHD), Growth Hormone Deficiency in Children | Phase 3 | Recruiting | Kexing Biopharm Co., Ltd. | 192 | 2026-06-06 | 2026-06-10 |
| Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency LUM-201, Matched Placebo (Capsules) - Growth Hormone Deficiency (GHD) | Phase 3 | Recruiting | Lumos Pharma | 150 | 2026-05-20 | 2026-10-19 |
| Ultrasound Measurement of Thyroid Volume in Term Newborns Thyroid ultrasound - Thyroid Volume, Thyroid Ultrasound | Not yet recruiting | IRCCS Ospedale San Raffaele | 400 | 2026-05-01 | 2026-05-12 | |
| 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 |
| 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 | |
| Infants With Primary Congenital Hypothyroidism and Development neurological status, sensory processing functions, Motor development - Congenital Hypothyroidism | Recruiting | Nigde Omer Halisdemir University | 40 | 2025-12-20 | 2026-02-12 | |
| 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 |
| Oral Manifestations in Egyptian Children With Short Stature Oral Manifestations, Short Stature | Not yet recruiting | Cairo University | 2 | 2025-12-15 | 2025-12-02 | |
| Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders Lonapegsomatropin [SKYTROFA®], Somatropin Pen Injector - Turner Syndrome, Short Stature Homeobox Gene Mutation | Phase 3 | Recruiting | Ascendis Pharma A/S | 186 | 2025-12-12 | 2026-11-05 |
| 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 |
| REMS25: Study on the Use of REMS Technology in Diseases Commonly Associated With Reduced Bone Mineral Density (BMD) REMS - Osteogenesis Imperfecta, Osteoporosis | Not applicable | Recruiting | Meyer Children's Hospital IRCCS | 100 | 2025-12-04 | 2026-02-17 |
| 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 |
| Levothyroxine Dosing in Neonatal Congenital Hypothyroidism Levothyroxine - Congenital Hypothyroidism | Phase 4 | Completed | Menoufia University | 101 | 2025-11-02 | 2026-08-10 |
| Palliative Care Needs of Children With Rare Diseases and Their Families Family Centered pediatric palliative care for family caregivers of children with rare diseases. - Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia | Not applicable | Enrolling by invitation | Children's National Research Institute | 480 | 2025-10-02 | 2026-01-28 |
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Dwarfism, Pituitary (252) · Achondroplasia (46) · Congenital Hypothyroidism (27) · Bone Diseases (21) · Turner Syndrome (21) · Genetic Diseases, Inborn (18) · Musculoskeletal Diseases (18) · Mucopolysaccharidosis IV (16) · Osteochondrodysplasias (14) · Failure to Thrive (12) · Cockayne Syndrome (11) · Pituitary Diseases (11) · Endocrine System Diseases (10) · Growth Disorders (10)
Bone Diseases, Developmental (393) · Musculoskeletal Diseases (389) · Bone Diseases (382) · Endocrine System Diseases (360) · Bone Diseases, Endocrine (278) · Nervous System Diseases (272) · Central Nervous System Diseases (259) · Brain Diseases (257) · Hypothalamic Diseases (253) · Hypopituitarism (243) · Pituitary Diseases (242) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (188) · Genetic Diseases, Inborn (170) · Nutritional and Metabolic Diseases (65)
Human Growth Hormone (94) · Growth Hormone (56) · vosoritide (13) · somapacitan (11) · Blood Specimen Collection (7) · Growth Hormone-Releasing Hormone (6) · Injections (6) · Thyroxine (6) · infigratinib (6) · MOD-4023 (5) · mecasermin (5) · albusomatropin (4) · lonapegsomatropin (4) · somatrogon (4) · Anastrozole (3)
Novo Nordisk A/S (29) · Pfizer (22) · Changchun GeneScience Pharmaceutical Co., Ltd. (18) · BioMarin Pharmaceutical (16) · Federico II University (10) · Merck KGaA, Darmstadt, Germany (9) · Ascendis Pharma A/S (8) · Columbia University (8) · QED Therapeutics, a BridgeBio company (8) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (7) · Massachusetts General Hospital (7) · Versartis Inc. (7) · Ascendis Pharma Endocrinology Division A/S (6) · Lumos Pharma (6) · Nemours Children's Clinic (6)
United States (161) · France (62) · United Kingdom (56) · Italy (51) · Germany (45) · Australia (42) · Spain (41) · China (28) · Canada (27) · Japan (27) · Denmark (25) · Israel (24) · Sweden (21) · Norway (15) · Poland (15)
Will Charlton, MD (4) · Nelly Mauras, MD (3) · Pamela U. Freda, MD (3) · Philippe Lysy, MD, PhD (3) · Xiaoping Luo (3) · Adeline Vanderver, MD (2) · Antonio Cittadini, MD (2)
PMID 38649657, PMID 31269546, PMID 34431071, PMID 36380045, PMID 39740666, PMID 18395480, PMID 22508317, PMID 22788856, PMID 23658497, PMID 24291224, PMID 28566441, PMID 29671202, PMID 31305924, PMID 31972476, PMID 33571362, PMID 33709288, PMID 33754315, PMID 35342457, PMID 36947589, PMID 38685585, PMID 39305160, PMID 40919719, PMID 42144862, PMID 10022400, PMID 10199767, PMID 10199774, PMID 10372687, PMID 10405442, PMID 10453734, PMID 10598807, PMID 11134092, PMID 11203878, PMID 11207626, PMID 11273062, PMID 11344175, PMID 11531927, PMID 11580992, PMID 11839953, PMID 11889170, PMID 12390993, PMID 12794705, PMID 12919160, PMID 12924350, PMID 15126541, PMID 15309449, PMID 15355453, PMID 15540176, PMID 15579750, PMID 15585563, PMID 15671089
2016 (5), 2015 (4), 2018 (4), 2008 (3), 2017 (3), 2004 (2), 2014 (2), R01DK110771 (2), R01HL085268 (2), 2019 (1), 2025 (1), M01RR001346 (1), P01AG000599 (1), R01AR050618 (1), R01HD102545 (1)