29 completed, 9 recruiting, 9 unknown, 7 terminated. 67 Early Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Early Phase 1 | 67 | Completed 29, Recruiting 9, Unknown 9, Terminated 7, Not yet recruiting 5, Withdrawn 4, Active not recruiting 2, Enrolling by invitation 2 |
Follow Congenital Abnormalities: new trials by email Literature Review of Congenital Abnormalities trials Research Report on Congenital Abnormalities
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 |
|---|---|---|---|---|---|---|
| GBP-213 in Patients With Autosomal Dominant Polycystic Kidney Disease GBP-213 - Autosomal Dominant Polycystic Kidney | Early Phase 1 | Not yet recruiting | RenJi Hospital | 24 | 2026-11-01 | 2026-10-23 |
| A Pilot Study to Examine the Efficacy and Tolerability of Sirolimus on Epidermal Nevi Lesions in Patients 6 Years Old and Above. sirolimus topical gel 0.2% - Epidermal Nevi | Early Phase 1 | Not yet recruiting | Northwestern University | 14 | 2026-10-01 | 2026-09-10 |
| Coupled Gentamicin-Lactobacillus Rhamnosus in NLUTD Gentamicin, Lactobacillus Rhamnosus GG - Spinal Cord Injury, Neurogenic Bladder (NB) | Early Phase 1 | Not yet recruiting | Medstar Health Research Institute | 48 | 2026-08-01 | 2026-08-21 |
| Role of (Saline- Glycerin) Rectal Enema in Treatment of Fecal Incontinence in Children Rectal enema using saline and glycerin mixture once daily - Anorectal Malformation | Early Phase 1 | Not yet recruiting | Sohag University | 30 | 2026-02-15 | 2026-02-06 |
| The Use of Near-Infrared Fluorescence Cholangiography With Indocyanine Green (ICG) in the Work Up of Neonatal Cholestasis Indocyanine Green - Biliary Atresia, Kasai | Early Phase 1 | Recruiting | University of Alabama at Birmingham | 15 | 2026-01-23 | 2026-02-18 |
| Thera-Clean® Microbubbles System in Patients With Skin Diseases TheraClean - Epidermolysis Bullosa, Ichthyosis | Early Phase 1 | Enrolling by invitation | Northwestern University | 20 | 2026-01-13 | 2026-02-03 |
| Daratumumab for Familial Cerebral Cavernous Malformations: A Single-Arm Safety and Efficacy Study Daratumumab - Cavernous Malformation, Cerebral | Early Phase 1 | Not yet recruiting | Beijing Tiantan Hospital | 10 | 2025-07-01 | 2025-06-18 |
| Calcium Folinate Treatment of Spastic Paraplegia 56 calcium folinate - Hereditary Spastic Paraplegia | Early Phase 1 | Recruiting | Shanghai 6th People's Hospital | 10 | 2024-07-01 | 2024-06-27 |
| The Role of Thiamine After Transcatheter Closure in Children With Left-to-Right Shunt Congenital Heart Disease Thiamine - Congenital Heart Disease, Thiamine Deficiency | Early Phase 1 | Completed | Universitas Sumatera Utara | 86 | 2024-05-01 | 2026-03-24 |
| Hyaluronidase Via LADD Scleroderma-induced Microstomia Hyaluronidase, CO2 laser - Microstomia, Scleroderma | Early Phase 1 | Completed | University of Nebraska | 3 | 2024-03-01 | 2026-04-15 |
| Assessing Combined Bevacizumab-Mitomycin C Use During Ahmed Valve Revision in Recurrent Pediatric Glaucoma AGV plate, bevacizumab injection - Pediatric Glaucoma | Early Phase 1 | Completed | Benha University | 50 | 2024-02-09 | 2025-05-21 |
| Development of Methods for Effective Treatment and Improvement of Common Somatic Diseases in Children The use of a combination of microelements and vitamins against the background of physiotherapy - Respiratory Diseases, Congenital Heart Defects | Early Phase 1 | Enrolling by invitation | Tashkent State Medical University (Tashkent Pediatric Medical Institute), Uzbekistan | 600 | 2023-11-08 | 2026-09-03 |
| Effect of Antibiotics on Urinary Microbiome Bactrim DS 800Mg-160Mg Tablet, Placebo oral tablet - Microtia, UTI | Early Phase 1 | Withdrawn | Johns Hopkins University | 2023-10-01 | 2021-11-03 | |
| Repurposing Dupilumab for Management of Pruritic Genetic Inflammatory Skin Disorders Dupilumab - Skin Diseases | Early Phase 1 | Recruiting | Northwestern University | 30 | 2023-06-12 | 2026-05-01 |
| Interstage Monitoring Using an Implantable Loop Recorder: A Pilot Study LUX-Dx™ - Univentricular Heart | Early Phase 1 | Completed | Boston Children's Hospital | 10 | 2023-03-15 | 2026-05-19 |
| Characterization of the Microbiome in Colonized Dystrophic and Junctional Epidermolysis Bullosa Wounds Before and After Use of APR-TD011 ® Spray Solution APR-TD011 - Epidermolysis Bullosa | Early Phase 1 | Completed | Northwestern University | 15 | 2023-02-06 | 2025-04-27 |
| The Effect of GLP-1 Agonist in Patients With Hypothalamic Obesity: Prospective, Pilot Study Saxenda - Panhypopituitarism, Craniopharyngioma | Early Phase 1 | Unknown | Seoul National University Hospital | 50 | 2022-11-28 | 2024-01-23 |
| Effect of Parenteral Alpha-Tocopherol in the Definitive Surgery of Tetralogy of Fallot alpha-Tocopherol, Sterile Water for Injection - Tetralogy of Fallot (TOF), Alpha-tocopherol | Early Phase 1 | Completed | Dr Cipto Mangunkusumo General Hospital | 60 | 2022-05-01 | 2025-09-26 |
| Does Spironolactone Normalize Sleep-wake Luteinizing Hormone Pulse Frequency in Pubertal Girls With Hyperandrogenism? Spironolactone, Placebo - Hyperandrogenism, Polycystic Ovary Syndrome | Early Phase 1 | Recruiting | University of Virginia | 32 | 2021-11-12 | 2025-08-05 |
| A Clinical Study of CD19/BCMA CAR-T Cells in the Treatment of Refractory POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, and Vasculitis CD19/BCMA CAR T-cells - POEMS Syndrome, Amyloidosis | Early Phase 1 | Unknown | Zhejiang University | 75 | 2021-10-08 | 2022-03-03 |
Showing 20 of 67. Sign in to see all of them.
Heart Defects, Congenital (6) · Epidermolysis Bullosa (4) · Hyperandrogenism (4) · Polycystic Ovary Syndrome (4) · Epidermolysis Bullosa Dystrophica (3) · Ichthyosis (3) · POEMS Syndrome (3) · Biliary Atresia (2) · Brain Neoplasms (2) · Congenital Microtia (2) · Hemangioma, Cavernous, Central Nervous System (2) · Hydrophthalmos (2) · Leukemia, Myeloid, Acute (2) · Microstomia (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (67) · Cardiovascular Diseases (21) · Nervous System Diseases (21) · Neoplasms (17) · Cardiovascular Abnormalities (16) · Genetic Diseases, Inborn (16) · Female Urogenital Diseases (14) · Female Urogenital Diseases and Pregnancy Complications (14) · Male Urogenital Diseases (14) · Pathological Conditions, Signs and Symptoms (14) · Urogenital Diseases (14) · Abnormalities, Multiple (13) · Skin and Connective Tissue Diseases (12) · Skin Diseases (11)
Bevacizumab (3) · Spironolactone (3) · Magnetic Resonance Spectroscopy (2) · Progesterone (2) · fludarabine (2) · Acamprosate (1) · Albuterol (1) · Alemtuzumab (1) · Baclofen (1) · Blood Specimen Collection (1) · Busulfan (1) · Calcium Dobesilate (1) · Chondroitinases and Chondroitin Lyases (1) · Coconut Oil (1) · Continuous Positive Airway Pressure (1)
Northwestern University (7) · University of Virginia (4) · Mayo Clinic (3) · Brigham and Women's Hospital (2) · Children's Hospital Medical Center, Cincinnati (2) · Tongji Hospital (2) · Alta Bates Summit Medical Center (1) · Beijing Tiantan Hospital (1) · Benha University (1) · Biogen (1) · Boston Children's Hospital (1) · CHU de Quebec-Universite Laval (1) · Cairo University (1) · Carelon Research (1) · Children's Hospital of Philadelphia (1)
United States (43) · China (6) · Egypt (3) · Indonesia (2) · Canada (1) · Iran (1) · Israel (1) · Norway (1) · South Korea (1) · Taiwan (1) · Uzbekistan (1) · Vietnam (1)
Amy Paller, MD (3) · Christine Burt Solorzano, MD (2) · Alan L. Ho, MD, PhD (1) · Alicia Y Christy, M.D. (1) · Amanda Garver, DO (1) · Amy Paller (1) · Andrew J Cohen, MD (1) · Andrew Lovering, PhD (1) · Angela Christiano, PhD (1) · Angela Dispenzieri, M.D. (1) · Blaine Hart, MD (1) · Bohdan Pomahac, MD (1) · Casandra Fink (1) · Chia-Che Wu, MD (1) · Christine Burt Solorzano, M.D. (1)
PMID 17635375, PMID 21946867, PMID 23481118, PMID 29300925, PMID 30256977, PMID 35391541, PMID 36167501, PMID 37658205, PMID 40425008, PMID 41101309
P50HD028934 (1), R01HD102060 (1), R01HL117836 (1), U54HD082008 (1), U54NS065705 (1), UL1TR002366 (1)