44 completed. 44 Phase 2, 8 Phase 3, 4 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 44 | Completed 44 |
| Phase 3 | 8 | Completed 8 |
| Phase 1 | 4 | Completed 4 |
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 |
|---|---|---|---|---|---|---|
| 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 |
| A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Multiple Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children With Achondroplasia TransCon CNP, Placebo for TransCon CNP - Achondroplasia | Phase 2 | Completed | Visen Pharmaceuticals (Shanghai) Co., Ltd. | 24 | 2022-01-05 | 2026-08-10 |
| Phase 2 Study of LUM-201 in Children With Growth Hormone Deficiency (OraGrowtH210 Trial) LUM-201, rhGH Norditropin® pen (34 µg/kg) - Growth Hormone Deficiency | Phase 2 | Completed | Lumos Pharma | 104 | 2020-12-31 | 2026-06-17 |
| A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia TransCon CNP, Placebo for TransCon CNP - Achondroplasia | Phase 2 | Completed | Ascendis Pharma A/S | 57 | 2020-06-24 | 2026-07-02 |
| Study of Infigratinib in Children With Achondroplasia Infigratinib 0.016 mg/kg, Infigratinib 0.032 mg/kg, Infigratinib 0.064 mg/kg - Achondroplasia | Phase 2 | Completed | QED Therapeutics, a BridgeBio company | 84 | 2020-03-10 | 2025-10-22 |
| Safety and Efficacy of Y-shape Pegylated Somatropin in Growth Hormone Deficiency Children Y-shape pegylated somatropin, Norditropin®, Norditropin - Growth Hormone Deficiency | Phase 2 / Phase 3 | Completed | Xiamen Amoytop Biotech Co., Ltd. | 434 | 2018-12-26 | 2024-01-24 |
| A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia BMN 111, Placebo - Achondroplasia | Phase 2 | Completed | BioMarin Pharmaceutical | 75 | 2018-06-13 | 2024-06-13 |
| Growth Hormone Treatment in Patients With Aggrecan (ACAN) Deficiency Norditropin - Short Stature | Phase 1 / Phase 2 | Completed | Children's Hospital Medical Center, Cincinnati | 22 | 2018-02-01 | 2024-07-30 |
| Dose Finding Study of GX-H9 in Paeditaric Patients With Growth Hormone Deficiency GX-H9, Genotropin - Growth Hormone Deficiency | Phase 2 | Completed | Genexine, Inc. | 56 | 2016-01-18 | 2020-04-20 |
| IGF-1 Treatment for Individuals With Short Stature Due to PAPP-A2 Deficiency Increlex - Short Stature | Phase 1 / Phase 2 | Completed | Children's Hospital Medical Center, Cincinnati | 7 | 2015-12-21 | 2024-02-28 |
| Versartis International Trial in Adults With Long-Acting Growth Hormone somavaratan - Adult Growth Hormone Deficiency | Phase 2 | Completed | Versartis Inc. | 36 | 2015-07-01 | 2022-07-26 |
| A Clinical Study in AGHD to Assess Safety, Tolerability and Efficacy of GX-H9 GX-H9, Genotropin - Adult Growth Hormone Deficiency | Phase 2 | Completed | Genexine, Inc. | 45 | 2015-01-01 | 2017-09-07 |
| Long Term Follow up Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children MOD-4023 - Growth Hormone Deficiency (GHD) | Phase 2 | Completed | OPKO Health, Inc. | 48 | 2014-02-01 | 2025-04-09 |
| A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia BMN 111 - Achondroplasia | Phase 2 | Completed | BioMarin Pharmaceutical | 35 | 2014-01-13 | 2021-01-15 |
| Safety, PK/PD (Pharmacokinetics/Pharmacodynamics) and Efficacy of ACP-001 Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD) ACP-001, Human Growth Hormone - Growth Hormone Deficiency (GHD) | Phase 2 | Completed | Ascendis Pharma A/S | 53 | 2013-07-01 | 2026-07-08 |
| Safety, Tolerability and Efficacy of Weekly TV-1106 in Adults With Growth Hormone Deficiency TV-1106, Recombinant human growth hormone - Growth Hormone Deficiency | Phase 2 | Completed | Teva Branded Pharmaceutical Products R&D, Inc. | 52 | 2013-03-31 | 2021-12-10 |
| Versartis Trial in Children to Assess Long-Acting Growth Hormone Experimental: VRS-317 - Pediatric Growth Hormone Deficiency | Phase 1 / Phase 2 | Completed | Versartis Inc. | 64 | 2012-10-01 | 2022-07-26 |
| Safety and Efficacy Study of Recombinant Human Growth Hormone in Adult Growth Hormone Deficiency Patients HM10560A, Genotropin - Adult Growth Hormone Deficiency | Phase 2 | Completed | Hanmi Pharmaceutical Company Limited | 72 | 2011-10-01 | 2016-02-19 |
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Dwarfism, Pituitary (26) · Achondroplasia (6) · Bone Diseases (3) · Bone Diseases, Developmental (3) · Genetic Diseases, Inborn (3) · Growth Disorders (2) · Hypopituitarism (2) · Laron Syndrome (2) · Musculoskeletal Diseases (2) · Osteochondrodysplasias (2) · Turner Syndrome (2) · Cockayne Syndrome (1) · DNA Repair-Deficiency Disorders (1) · Fibromyalgia (1)
Endocrine System Diseases (43) · Musculoskeletal Diseases (42) · Bone Diseases (41) · Bone Diseases, Developmental (41) · Nervous System Diseases (27) · Bone Diseases, Endocrine (26) · Brain Diseases (26) · Central Nervous System Diseases (26) · Hypothalamic Diseases (26) · Pituitary Diseases (26) · Hypopituitarism (24) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (20) · Genetic Diseases, Inborn (17) · Gonadal Disorders (4)
Human Growth Hormone (13) · Growth Hormone (5) · GX-H9 (2) · MOD-4023 (2) · mecasermin (2) · vosoritide (2) · Amitriptyline (1) · Anastrozole (1) · Control Groups (1) · Fluoxetine (1) · Growth Hormone-Releasing Hormone (1) · Insulin-Like Growth Factor I (1) · NNC126-0083 (1) · Oxandrolone (1) · Testosterone (1)
Ascendis Pharma A/S (3) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (3) · BioMarin Pharmaceutical (2) · Children's Hospital Medical Center, Cincinnati (2) · Federico II University (2) · Genexine, Inc. (2) · Lumos Pharma (2) · Nemours Children's Clinic (2) · OPKO Health, Inc. (2) · Versartis Inc. (2) · Xiamen Amoytop Biotech Co., Ltd. (2) · Aarhus University Hospital (1) · Altus Pharmaceuticals (1) · Ambrx, Inc. (1) · Ascendis Pharma Growth Disorders A/S (1)
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PMID 38649657, PMID 17192294, PMID 18053120, PMID 19001519, PMID 20961566, PMID 21177789, PMID 23015655, PMID 24178787, PMID 24291224, PMID 24423356, PMID 28201598, PMID 28566441, PMID 29029190, PMID 30339244, PMID 31269546, PMID 34431071, PMID 34922359, PMID 35001504, PMID 35342457, PMID 36034448, PMID 36646053, PMID 36994923, PMID 37823031, PMID 37984383, PMID 39555818, PMID 39906034, PMID 41247754, PMID 41783511, PMID 42144862, PMID 42319555
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