9 active not recruiting. 9 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 9 | Active not recruiting 9 |
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 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 |
| PK and PD Study of LUM-201 in Children With Idiopathic Growth Hormone Deficiency: (OraGrowtH212) LUM-201 - Growth Hormone Deficiency | Phase 2 | Active not recruiting | Lumos Pharma | 24 | 2021-07-14 | 2023-10-04 |
| A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia vosoritide - Achondroplasia | Phase 2 | Active not recruiting | BioMarin Pharmaceutical | 20 | 2020-10-10 | 2026-03-13 |
| Vosoritide for Selected Genetic Causes of Short Stature Vosoritide - Short Stature | Phase 2 | Active not recruiting | Andrew Dauber | 56 | 2020-08-04 | 2026-03-18 |
| An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily. - Achondroplasia | Phase 2 | Active not recruiting | BioMarin Pharmaceutical | 73 | 2019-06-12 | 2026-03-13 |
| A Study Extension Period of PEG-somatropin (Pegylated-somatropin) in the Treatment of Children With Idiopathic Short Stature PEG-somatropin - Dwarfism | Phase 2 | Active not recruiting | Changchun GeneScience Pharmaceutical Co., Ltd. | 360 | 2017-05-12 | 2025-08-15 |
| A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) BMN 111 - Achondroplasia | Phase 2 | Active not recruiting | BioMarin Pharmaceutical | 30 | 2016-01-26 | 2026-03-13 |
Achondroplasia (5) · Bone Diseases (4) · Musculoskeletal Diseases (3) · Noonan Syndrome (2) · Bone Diseases, Developmental (1) · Dwarfism, Pituitary (1) · Hypochondroplasia (1)
Bone Diseases, Developmental (8) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (8) · Genetic Diseases, Inborn (8) · Endocrine System Diseases (7) · Musculoskeletal Diseases (6) · Bone Diseases (5) · Osteochondrodysplasias (5) · Cardiovascular Abnormalities (2) · Cardiovascular Diseases (2) · Congenital Abnormalities (2) · Connective Tissue Diseases (2) · Craniofacial Abnormalities (2) · Heart Defects, Congenital (2) · Heart Diseases (2)
vosoritide (5)
BioMarin Pharmaceutical (4) · Andrew Dauber (1) · Ascendis Pharma A/S (1) · Ascendis Pharma Growth Disorders A/S (1) · Changchun GeneScience Pharmaceutical Co., Ltd. (1) · Lumos Pharma (1)
United Kingdom (5) · Australia (4) · United States (4) · France (3) · Canada (2) · Denmark (2) · Ireland (2) · Chile (1) · China (1) · Germany (1) · Italy (1) · Japan (1) · Spain (1)
Andrew Dauber, MS MMSc (1) · Xiaoping Luo (1)
PMID 31269546, PMID 33761804, PMID 38649657, PMID 38813446, PMID 42144862
2015 (1)