20 completed, 3 terminated, 1 not yet recruiting, 1 recruiting. 26 Phase 1, 7 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 26 | Completed 20, Terminated 3, Not yet recruiting 1, Recruiting 1, Unknown 1 |
| Phase 2 | 7 | Completed 4, Terminated 2, Not yet 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 |
|---|---|---|---|---|---|---|
| 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 |
| A Clinical Study of GenSci134 in Healthy Adults and Adult Growth Hormone Deficiency(AGHD) GenSci134, Placebo, Recombinant Human Growth Hormone Injection (Norditropin®) - Safety, Tolerability | Phase 1 | Recruiting | Changchun GeneScience Pharmaceutical Co., Ltd. | 64 | 2025-06-09 | 2026-04-14 |
| Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants BMN 111 administration via Injector Pen, BMN 111 administration via vial and syringe - Achondroplasia | Phase 1 | Terminated | BioMarin Pharmaceutical | 36 | 2023-03-15 | 2023-10-12 |
| A Study to Compare the Uptake Into the Blood of Two Strengths of Somapacitan After Injection Under the Skin in Healthy Subjects somapacitan - Healthy Volunteers, Growth Hormone Deficiency | Phase 1 | Completed | Novo Nordisk A/S | 33 | 2019-03-29 | 2019-08-06 |
| 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 |
| Investigation of Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Multiple Doses of Somapacitan in Subjects With Mild and Moderate Degrees of Hepatic Impairment Compared to Subjects With Normal Hepatic Function. Somapacitan - Growth Hormone Disorder, Adult Growth Hormone Deficiency | Phase 1 | Completed | Novo Nordisk A/S | 34 | 2017-08-16 | 2019-05-24 |
| Investigation of Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Multiple Doses of Somapacitan in Subjects With Various Degrees of Impaired Renal Function Compared to Subjects With Normal Renal Function Somapacitan - Growth Hormone Disorder, Adult Growth Hormone Deficiency | Phase 1 | Completed | Novo Nordisk A/S | 44 | 2017-06-20 | 2020-04-17 |
| 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 |
| Pappalysin 2 (PAPP-A2) Enzyme Replacement Fresh Frozen Plasma - Short Stature, Idiopathic | Phase 1 | Completed | Children's Hospital Medical Center, Cincinnati | 1 | 2015-04-01 | 2015-05-28 |
| Pharmacokinetics and Pharmacodynamics of Long Acting Human Growth Hormone (hGH) Product (MOD-4023) in Healthy Caucasian and Japanese Volunteers MOD-4023, Placebo - Growth Hormone Deficiency | Phase 1 | Completed | OPKO Health, Inc. | 42 | 2015-01-01 | 2019-09-30 |
| Evaluation of Pharmacokinetic and Pharmacodynamic Parameters of Somatropin Produced by Laboratory Blausiegel Indústria e Comércio Ltda., Compared to Saizen® From Laboratory Merck Serono S.A. Somatropin - GH Deficiency (GHD), Growth Retardation | Phase 1 | Unknown | Azidus Brasil | 32 | 2013-03-01 | 2012-09-25 |
| Phase 1 Safety Study of ALRN-5281 in Healthy Subjects ALRN-5281 0.015 mg/kg, ALRN-5281 0.05 mg/kg, ALRN-5281 0.15 mg/kg - Growth Hormone Deficiency | Phase 1 | Completed | Aileron Therapeutics, Inc. | 33 | 2013-01-01 | 2013-05-30 |
| A Trial Investigating the Safety, Tolerability, Availability and Distribution in the Body of Once-weekly Long-acting Growth Hormone (Somapacitan) Compared to Once Daily Norditropin NordiFlex® in Adults With Growth Hormone Deficiency somapacitan, Norditropin NordiFlex® - Growth Hormone Disorder, Adult Growth Hormone Deficiency | Phase 1 | Completed | Novo Nordisk A/S | 35 | 2012-10-12 | 2020-12-24 |
| 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 |
| A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers BMN 111, Normal Saline - Achondroplasia | Phase 1 | Completed | BioMarin Pharmaceutical | 74 | 2012-02-01 | 2012-06-11 |
| First Human Dose Trial of NNC0195-0092 (Somapacitan) in Healthy Subjects somapacitan, placebo (somapacitan) - Growth Hormone Disorder, Adult Growth Hormone Deficiency | Phase 1 | Completed | Novo Nordisk A/S | 105 | 2012-01-16 | 2020-12-24 |
| Safety, Pharmacokinetics and Pharmacodynamics Study of HL-032 in Healthy Male Volunteers Somatropin, Placebo - Growth Hormone Deficiency | Phase 1 | Completed | HanAll BioPharma Co., Ltd. | 40 | 2011-09-01 | 2012-10-16 |
| Free Fatty Acids, Body Weight, and Growth Hormones Secretion in Children Niacin First, Placebo First, Dose-Establishing Study 1 Niacin 250mg - Obesity, Short Stature | Phase 1 / Phase 2 | Terminated | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 37 | 2011-07-01 | 2018-12-17 |
| VRS-317 in Adult Subjects With Growth Hormone Deficiency VRS-317 - Growth Hormone Deficiency | Phase 1 | Completed | Versartis Inc. | 50 | 2011-03-01 | 2012-07-23 |
| Pharmacokinetics and Safety Study of Single and Multiple Oral Doses Prodarsan™ in Patients With Cockayne Syndrome Prodarsan - Cockayne Syndrome | Phase 1 / Phase 2 | Completed | DNage B.V. | 5 | 2010-06-01 | 2011-06-23 |
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Dwarfism, Pituitary (19) · Achondroplasia (3) · Bone Diseases (1) · Bone Diseases, Developmental (1) · Bone Diseases, Endocrine (1) · Brain Diseases (1) · Cockayne Syndrome (1) · DNA Repair-Deficiency Disorders (1) · Failure to Thrive (1) · Genetic Diseases, Inborn (1) · Hypopituitarism (1) · Musculoskeletal Diseases (1) · Obesity (1) · Overweight (1)
Bone Diseases (25) · Bone Diseases, Developmental (25) · Musculoskeletal Diseases (25) · Endocrine System Diseases (23) · Nervous System Diseases (20) · Central Nervous System Diseases (19) · Hypothalamic Diseases (19) · Bone Diseases, Endocrine (18) · Brain Diseases (18) · Hypopituitarism (18) · Pituitary Diseases (18) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (9) · Genetic Diseases, Inborn (8) · Osteochondrodysplasias (3)
Human Growth Hormone (8) · somapacitan (5) · Growth Hormone (3) · NNC0195-0092 (2) · Growth Hormone-Releasing Hormone (1) · Growth and Development (1) · Hormone Replacement Therapy (1) · Hormones (1) · Hormones, Hormone Substitutes, and Hormone Antagonists (1) · Insulin-Like Growth Factor Binding Protein 3 (1) · Insulin-Like Growth Factor I (1) · MOD-4023 (1) · National Institute of Arthritis and Musculoskeletal and Skin Diseases (U.S.) (1) · Pharmacokinetics (1) · Saline Solution (1)
Novo Nordisk A/S (6) · Children's Hospital Medical Center, Cincinnati (3) · BioMarin Pharmaceutical (2) · Changchun GeneScience Pharmaceutical Co., Ltd. (2) · Versartis Inc. (2) · Abbisko Therapeutics Co, Ltd (1) · Aileron Therapeutics, Inc. (1) · Azidus Brasil (1) · Columbia University (1) · DNage B.V. (1) · EMD Serono (1) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (1) · HanAll BioPharma Co., Ltd. (1) · OPKO Health, Inc. (1) · Phage Pharmaceuticals, Inc. (1)
United States (13) · Germany (4) · China (2) · Brazil (1) · Denmark (1) · Slovakia (1) · South Korea (1) · Sweden (1)
Philippe Backeljauw, MD (2) · Andrew Dauber, MD (1) · Bradley D Vince, D.O. (1) · Edward Neilan, MD (1) · Gajanathan Muthuvel, MD (1) · Hubert C Chen, M.D. (1) · Jack A Yanovski, M.D. (1) · Kyung-Sang Yu, Medicine (1) · Luo Xiaoping, Doctor (1) · Mark Kipnes, MD (1) · Michael Lissy, MD (1)
PMID 29671202, PMID 33754315, PMID 20961422, PMID 23585663, PMID 25013997, PMID 26727076, PMID 29029190, PMID 33864240, PMID 34922359, PMID 35001504, PMID 36646053, PMID 38969919
2016 (2), 2018 (1)