Dwarfism clinical trials
The registry lists 125 studies for Dwarfism across more than 10 countries. 19 are recruiting or about to start. Per ClinicalTrials.gov, last updated 2026-09-23.
Status
- Completed
- 72
- Status unknown
- 14
- Recruiting
- 13
- Active, not recruiting
- 11
- Terminated
- 6
- Enrolling by invitation
- 3
Phase
- Phase 2
- 28
- Phase 3
- 24
- Phase 4
- 14
- Phase 1
- 5
Where
- United States
- 52
- France
- 23
- Australia
- 16
- United Kingdom
- 16
- Spain
- 14
- Germany
- 12
Who runs them
Novo Nordisk A/S (8) · QED Therapeutics, a BridgeBio company (7) · BioMarin Pharmaceutical (7) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (7) · Changchun GeneScience Pharmaceutical Co., Ltd. (6) · Rabin Medical Center (6) · Pfizer (6) · Merck KGaA, Darmstadt, Germany (5)
Studies
Recruiting first, then most recently updated. Showing 50 of 125 — search for more.
- 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 DisordersRecruitingNCT07221851Phase 37 countries
- Open-Label, Long-Term, Extension Study of Infigratinib in Children With HypochondroplasiaEnrolling by invitationNCT07393373Phase 210 countries
- A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With AchondroplasiaEnrolling by invitationNCT05929807Phase 2/312 countries
- Study of Skeletal DisordersRecruitingNCT050315071 country
- An Interventional Study of Infigratinib in Children With HypochondroplasiaEnrolling by invitationNCT06873035Phase 2/310 countries
- Prospective Clinical Assessment Study in Children With HypochondroplasiaRecruitingNCT0641097610 countries
- Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)RecruitingNCT07169279Phase 27 countries
- Non-invasive Functional Assessment and Pathogenesis of Morquio ARecruitingNCT052840061 country
- Oral Manifestations in Egyptian Children With Short StatureNot yet recruitingNCT06372366
- A Multicenter Multinational Observational Study of Children With HypochondroplasiaRecruitingNCT0621294711 countries
- The Dosage Exploration Study of PEG-rhGH for Treating Short Stature in Prepubertal and Pubertal ChildrenNot yet recruitingNCT06768411Not applicable
- Long-term Efficacy and Safety Evaluation of Growth Hormone in Children in China(CGLS)RecruitingNCT061109101 country
- Study of the Pathophysiology of RNU4ATAC and RTTN Associated SyndromesRecruitingNCT06111950Not applicable1 country
- Vosoritide for Short Stature in Turner SyndromeRecruitingNCT05849389Phase 21 country
- The Effect of Nutritional Formula Supplementation on Linear Growth of Growth Hormone (GH) Treated Prepubertal Children With Idiopathic Short Stature (ISS) After 2 Years From the Beginning of GH-therapyRecruitingNCT04962360Not applicable1 country
- Celiac Disease Among Egyptian Children With Unexplained Short StatureNot yet recruitingNCT06164548
- A Natural History Study in Children With a Type II Collagen Disorder With Short StatureRecruitingNCT054087152 countries
- Registry for Patients With Achondroplasia / Hypochondroplasia (OMPR-Ach/Hy)RecruitingNCT053280501 country
- Long-term Safety and Effectiveness of Growth Hormone With GHD, TS, CRF, SGA , ISS and PWS in ChildrenRecruitingNCT016043951 country
- A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone TreatmentActive, not recruitingNCT06668805Phase 27 countries
- Natural History of the Collagen-Related Disorder Osteogenesis Imperfecta and Genotype Phenotype CorrelationCompletedNCT035752211 country
- A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Multiple Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children With AchondroplasiaCompletedNCT05246033Phase 21 country
- A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With AchondroplasiaCompletedNCT04085523Phase 28 countries
- Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)CompletedNCT0403581111 countries
- Vosoritide for Selected Genetic Causes of Short StatureActive, not recruitingNCT04219007Phase 21 country
- A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With AchondroplasiaCompletedNCT06164951Phase 310 countries
- A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With AchondroplasiaActive, not recruitingNCT04554940Phase 22 countries
- A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)Active, not recruitingNCT02724228Phase 24 countries
- An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With AchondroplasiaActive, not recruitingNCT03989947Phase 24 countries
- An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With AchondroplasiaActive, not recruitingNCT03424018Phase 37 countries
- Interventional Study of Vosoritide for the Treatment of Children With HypochondroplasiaActive, not recruitingNCT06455059Phase 39 countries
- Skeletal Muscle Effects of GH in BoysCompletedNCT040209131 country
- Study of Infigratinib in Children With AchondroplasiaCompletedNCT04265651Phase 26 countries
- Biological Age in Children With GH Deficiency Undergoing Hormone Replacement TherapyCompletedNCT062948601 country
- A Study Extension Period of PEG-somatropin (Pegylated-somatropin) in the Treatment of Children With Idiopathic Short StatureActive, not recruitingNCT03255694Phase 21 country
- Clinical Study of Pegylated Somatropin (PEG Somatropin) to Treat SGA Children With Short StatureActive, not recruitingNCT02375620Phase 21 country
- Short Stature and Psychological Well-beingCompletedNCT062953411 country
- Testing the Feasibility of a Novel Growth Monitoring Smartphone AppCompletedNCT058292521 country
- Nutritional Stimulation of Growth in Children With Short StatureCompletedNCT04226586Not applicable1 country
- Growth Hormone Treatment in Patients With Aggrecan (ACAN) DeficiencyCompletedNCT03288103Phase 1/21 country
- PREPL in Health and DiseaseActive, not recruitingNCT02263781Not applicable1 country
- Mauriac Syndrome: Isotopic Techniques and Genetic AnalysisCompletedNCT04275141Not applicable1 country
- IGF-1 Treatment for Individuals With Short Stature Due to PAPP-A2 DeficiencyCompletedNCT02636270Phase 1/2
- A Trial of YPEG-rhGH in Children With Short StatureCompletedNCT05838885Phase 21 country
- A 4 Year Combination Therapy of Growth Hormone and (GnRH) Agonist in Children With a Short Predicted HeightActive, not recruitingNCT00840944Phase 41 country
- Web-based Adherence Information Integrated Nurse-led Monitoring ClinicCompletedNCT042441231 country
- Genetic Causes of Growth DisordersTerminatedNCT023113221 country
- Post Marketing Surveillance on Long-term Use With Norditropin® (Short Stature Due to Noonan Syndrome)CompletedNCT034356271 country
- Adaption and Testing of the Quality of Life in Short Stature Youth (QoLISSY) Questionnaire for Parents With Children From 0-4Status unknownNCT056039361 country
- The Impact of the Use of Recombinant Human Growth Hormone on ADHD Characteristics in Children and AdolescentsStatus unknownNCT029730611 country