52 completed, 10 terminated, 7 unknown, 5 recruiting. 81 Phase 3, 15 Phase 2.
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| Growth Hormone Replacement Therapy in Veterans With Mild Traumatic Brain Injury (mTBI) and Adult Growth Hormone Deficiency (AGHD) Somatropin, Placebo - Adult Growth Hormone Deficiency, Mild Traumatic Brain Injury | Phase 3 | Recruiting | VA Office of Research and Development | 172 | 2025-01-13 | 2026-10-13 |
| Evaluate the Efficacy and Safety of KK8398 in Patients With Achondroplasia（AOBA Study） KK8398 - Achondroplasia | Phase 3 | Active not recruiting | Kyowa Kirin Co., Ltd. | 13 | 2024-12-01 | 2026-10-08 |
| Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia Vosoritide, Placebo - Hypochondroplasia | Phase 3 | Completed | BioMarin Pharmaceutical | 81 | 2024-06-17 | 2026-11-12 |
| A Study to Investigate Efficacy and Safety of Weekly PEG-somatropin (GenSci004) in Treatment Naive Children With Growth Hormone Deficiency GenSci004, Genotropin - GHD | Phase 3 | Unknown | Changchun GeneScience Pharmaceutical Co., Ltd. | 162 | 2023-12-01 | 2023-09-11 |
| A Study to Investigate the Safety and Efficacy of Once-weekly PEG-somatropin (GenSci004) in Children With Growth Hormone Deficiency PEG-somatropin - GHD | Phase 3 | Unknown | Changchun GeneScience Pharmaceutical Co., Ltd. | 180 | 2023-12-01 | 2023-09-11 |
| A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia Infigratinib 0.25 mg/kg/day, Placebo Comparator 0.25 mg/kg/day - Achondroplasia | Phase 3 | Completed | QED Therapeutics, a BridgeBio company | 114 | 2023-11-10 | 2026-03-17 |
| 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 |
| A Trial to Investigate Long Term Efficacy and Safety of Lonapegsomatropin in Adults With Growth Hormone Deficiency Lonapegsomatropin - Adult Growth Hormone Deficiency, Endocrine System Diseases | Phase 3 | Completed | Ascendis Pharma Endocrinology Division A/S | 220 | 2021-12-16 | 2026-07-07 |
| A Research Study of How Well Macimorelin Works to Find Out if Children Have a Lack of Growth Hormone and How Safe it is Macimorelin, Arginine, Clonidine - Growth Hormone Deficiency | Phase 3 | Completed | AEterna Zentaris | 101 | 2021-11-16 | 2024-08-12 |
| A Trial to Compare the Efficacy and Safety of Once-weekly Lonapegsomatropin With Placebo and a Daily Somatropin Product in Adults With Growth Hormone Deficiency Lonapegsomatropin, Placebo, Somatropin - Growth Hormone Deficiency, Endocrine System Diseases | Phase 3 | Completed | Ascendis Pharma Endocrinology Division A/S | 264 | 2020-12-03 | 2026-07-07 |
| Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Chinese Pediatric Growth Hormone Deficiency TransCon hGH, daily hGH - Growth Hormone Deficiency, Endocrine System Diseases | Phase 3 | Unknown | Visen Pharmaceuticals (Shanghai) Co., Ltd. | 150 | 2019-12-30 | 2020-03-30 |
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Dwarfism, Pituitary (57) · Achondroplasia (8) · Failure to Thrive (8) · Endocrine System Diseases (7) · Pituitary Diseases (7) · Bone Diseases (6) · Genetic Diseases, Inborn (6) · Musculoskeletal Diseases (6) · Osteochondrodysplasias (6) · Turner Syndrome (4) · Bone Diseases, Developmental (3) · Growth Disorders (3) · Hypochondroplasia (2) · Laron Syndrome (2)
Bone Diseases, Developmental (78) · Bone Diseases (75) · Musculoskeletal Diseases (75) · Endocrine System Diseases (70) · Nervous System Diseases (59) · Brain Diseases (58) · Central Nervous System Diseases (58) · Hypothalamic Diseases (58) · Bone Diseases, Endocrine (57) · Hypopituitarism (56) · Pituitary Diseases (51) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (26) · Genetic Diseases, Inborn (20) · Pathological Conditions, Signs and Symptoms (14)
Human Growth Hormone (34) · Growth Hormone (13) · lonapegsomatropin (5) · vosoritide (4) · mecasermin (3) · somapacitan (3) · Insulin-Like Growth Factor I (2) · MOD-4023 (2) · WW Domain-Containing Oxidoreductase (2) · albusomatropin (2) · infigratinib (2) · macimorelin (2) · somatrogon (2) · Anastrozole (1) · Arginine (1)
Novo Nordisk A/S (8) · Pfizer (7) · Changchun GeneScience Pharmaceutical Co., Ltd. (5) · Ascendis Pharma Endocrinology Division A/S (4) · BioMarin Pharmaceutical (4) · Eli Lilly and Company (4) · Ascendis Pharma A/S (3) · Ipsen (3) · LG Life Sciences (3) · Versartis Inc. (3) · AEterna Zentaris (2) · Ascendis Pharma Growth Disorders A/S (2) · EMD Serono (2) · Federico II University (2) · Lumos Pharma (2)
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