Congenital, Hereditary, and Neonatal Diseases and Abnormalities clinical trials
The registry lists 110 studies for Congenital, Hereditary, and Neonatal Diseases and Abnormalities across more than 10 countries. 20 are recruiting or about to start. 67 of them are mainly about Congenital, Hereditary, and Neonatal Diseases and Abnormalities (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-13. Counting its 323 narrower conditions too, such as Cystic Fibrosis: 26,582 studies, 5,481 open.
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A study of drug A after surgery for early breast cancer
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Changed
· Sites: 4 added, including Seoul and Busan
· Enrollment: 300 → 450 participants
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Who runs them
Children's Healthcare of Atlanta (49) · Emory University (12) · G. d'Annunzio University (2) · Taysha Gene Therapies, Inc. (2) · Ain Shams University (2) · University of Arizona (1) · Erasmus Medical Center (1) · Fresenius Medical Care Deutschland GmbH (1)
What is being tested here
Drugs under test in Congenital, Hereditary, and Neonatal Diseases and Abnormalities studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| Senicapoc | 1 | 0 | 1 |
| Zodasiran | 1 | 1 | 1 |
| Ampicillin | 1 | 0 | 1 |
| Amoxicillin | 1 | 0 | 1 |
| Melatonin | 0 | 1 | 1 |
| Hydrocortisone | 0 | 0 | 1 |
| Etanercept | 0 | 0 | 1 |
| Spironolactone | 0 | 0 | 1 |
| Epinephrine | 0 | 0 | 1 |
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 3, phase 3 first
- Dec 2026Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Microvillus Inclusion Disease (MVID) · Phase 3 · Napo Pharmaceuticals, Inc.
- Dec 2026Vision Baby : The Measurement of Vital Signs in Neonates by Lifelight® Software in Comparison to the Standard of Care · Xim Limited
- Mar 2027Multimodal Biocollection Linked to the French Register of Intracranial Aneurysms · Nantes University Hospital
Who these studies take
Of the 15 Congenital, Hereditary, and Neonatal Diseases and Abnormalities studies recruiting now, 6 (40%) set no upper age limit and 8 (53%) stop at 75 or younger. 11 (73%) take children under 18. 3 (20%) are for women only and 1 (7%) for men only; 2 (13%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 4 (27%) | 4 (27%) |
| 18 | 10 (67%) | 8 (53%) |
| 30 | 10 (67%) | 7 (47%) |
| 40 | 10 (67%) | 7 (47%) |
| 50 | 10 (67%) | 7 (47%) |
| 65 | 8 (53%) | 6 (40%) |
| 75 | 7 (47%) | 5 (33%) |
| 85 | 7 (47%) | 5 (33%) |
Across all 110 studies, recruiting or not: 70% stop at 75 or younger and 68% at 65; 81% take children; 10% are for women only and 4% for men only; 14% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Oct 16, 2026.
Newly registered3
- A Phase 3 Evaluation of Sutacimig in Glanzmann ThrombastheniaNov 13, 2026NCT07935356
- Fetal Embolization for Severe Vein of Galen Malformation - A Feasibility and Safety TrialNov 10, 2026NCT07927725
- EDIT-401 in Adults Who Require Additional Lowering of Low-Density Lipoprotein Cholesterol (LDL-C)Oct 26, 2026NCT07894887
Studies
Recruiting first, then most recently updated. Showing 50 of 110. Browse all 110 → or search them.
- A Phase 3 Evaluation of Sutacimig in Glanzmann ThrombastheniaRecruitingNCT07935356Phase 31 country
- Genome Medical Sequencing for Gene DiscoveryRecruitingNCT010873201 country
- Fetal Embolization for Severe Vein of Galen Malformation - A Feasibility and Safety TrialNot yet recruitingNCT07927725Not applicable1 country
- Efficacy and Safety of SIL-8301 for Control of Hemolysis in a Uniform Sickle Cell Disease EndotypeRecruitingNCT07282210Phase 21 country
- EDIT-401 in Adults Who Require Additional Lowering of Low-Density Lipoprotein Cholesterol (LDL-C)Not yet recruitingNCT07894887Phase 1/21 country
- Study of Zodasiran in Adolescent Participants With Homozygous Familial HypercholesterolemiaRecruitingNCT07473843Phase 33 countries
- A Long-Term Observational Study of Patients With FucosidosisRecruitingNCT076154006 countries
- Single Injection of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIBNot yet recruitingNCT07818759Phase 1/21 country
- Multimodal Biocollection Linked to the French Register of Intracranial AneurysmsRecruitingNCT06689410Not applicable1 country
- Vision Baby : The Measurement of Vital Signs in Neonates by Lifelight® Software in Comparison to the Standard of CareRecruitingNCT077998701 country
- Measuring Adverse Pregnancy and Newborn Congenital OutcomesRecruitingNCT044057001 country
- Prenatal Transplantation for Fetuses With Fanconi AnemiaNot yet recruitingNCT07408583Phase 1/21 country
- Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)RecruitingNCT07608432Phase 31 country
- International Study of Cerebral Oxygenation and Electrical Activity During Major Neonatal SurgeryRecruitingNCT056734997 countries
- Prospective Registry for Long-term Outcomes Following FETO in Severe Left and Right CDHRecruitingNCT07166172Not applicable1 country
- Azithromycin and Ampicillin for Late PPROMRecruitingNCT07183462Phase 41 country
- Clinical Application of Artificial Intelligence in New Borns With Cleft Lip and PalateRecruitingNCT06970158Not applicable1 country
- Vaccinations and People With DisabilitiesRecruitingNCT057494191 country
- Study of Late-foetal Human Organ DevelopmentRecruitingNCT046610591 country
- Vocal Pattern Assessment as a New Key to Identifying Rare SyndromesNot yet recruitingNCT05955794Not applicable1 country
- Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett SyndromeActive, not recruitingNCT07480564Phase 31 country
- A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)Active, not recruitingNCT05606614Phase 32 countries
- Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Microvillus Inclusion Disease (MVID)Active, not recruitingNCT06721871Phase 2/33 countries
- Evaluation of Bili-ruler, a Low-cost, Plastic Icterometer for Community-based Screening of Jaundice in NewbornsCompletedNCT066877464 countries
- Prescreening Study to Identify Potential Wilson Disease Participants for Gene-Editing Clinical TrialActive, not recruitingNCT072266221 country
- TAAI Erasmus Research Initiative to Fight CF: Monitoring Inflammation in CF Lung Disease Into a New EraCompletedNCT057520191 country
- Melatonin for Pulmonary Hypertension in Full Term NeonatesCompletedNCT06685679Phase 31 country
- Subcutaneous Hydrocortisone Children With Congenital Adrenal HyperplasiaCompletedNCT03718234Phase 11 country
- Congenital Disability and Rehabilitation: the TOPS ProgramCompletedNCT04293705Not applicable1 country
- OPTI-Prem: Optimising Neonatal Service Provision for Preterm Babies Born Between 27 and 31 Weeks of Gestation in EnglandCompletedNCT029948491 country
- Nasal High Frequency in Preterm NeonatesCompletedNCT06687083Not applicable1 country
- The Effect of Feng Shui Delivery Room Design on Maternal and Infant HealthCompletedNCT05468658Not applicable1 country
- Investigating How Childhood Tumours and Congenital Disease DevelopStatus unknownNCT065848771 country
- Learning Skills in Shoulder Dystocia Delivery Using Simulator ModelsCompletedNCT059210451 country
- Turkish Affordances in the Home Environment for Motor Development-Infant Scale (AHEMD-IS)CompletedNCT045564871 country
- The Effect of Birth Ball Exercise on Labor Pain, Delivery Duration, Birth Comfort, and Birth SatisfactionCompletedNCT05711472Not applicable1 country
- Learning Skills in Young Specialists and Residents in Operative Vaginal Delivery Using Simulator ModelsCompletedNCT056056261 country
- Flexible URS in Management of Renal Stones in Anomalous KidneyStatus unknownNCT05299996Not applicable1 country
- Kids Intestinal Dysfunction in Congenital Heart Disease: the Kind Heart StudyCompletedNCT028247051 country
- Tracheal Occlusion To Accelerate Lung Growth (TOTAL) Trial for Severe Pulmonary HypoplasiaCompletedNCT01240057Phase 2/311 countries
- HaemoDialysis Interventions to REduce Multi-Organ Dysfunction and Effect on Quality of Life Assessed by MRI ScanningCompletedNCT03280901Not applicable1 country
- Pilot Study of Etanercept (Enbrel) in Children With Fanconi AnemiaCompletedNCT00965666Early Phase 11 country
- A Review of Surgical Management of Congenital Pulmonary Airway Malformations (CPAM): A Decade of ExperienceCompletedNCT044496141 country
- Parametric Mapping in Paediatric Magnetic Resonance ImagingStatus unknownNCT040689871 country
- Supported Exercise Programme for Adults With Congenital Heart DiseaseCompletedNCT03262168Not applicable1 country
- Adult Congenital Heart Disease SurgeryCompletedNCT004461601 country
- Research Network for Neonatal Diseases Induced by Tissular Fetomaternal AlloimmunizationCompletedNCT001996281 country
- Low-dose Epinephrine Infusion Tests in Adolescent and Pediatric PatientsTerminatedNCT00548886Not applicable1 country
- Spironolactone in Patients With Single Ventricle HeartCompletedNCT00211081Not applicable1 country
- D-tecting Disease - From Exposure to Vitamin D During Critical Periods of LifeCompletedNCT033303011 country