7 recruiting. 7 Phase 2, 3 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 7 | Recruiting 7 |
| Phase 1 | 3 | Recruiting 3 |
Follow Craniofacial Abnormalities: new trials by email Literature Review of Craniofacial Abnormalities trials Research Report on Craniofacial 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 |
|---|---|---|---|---|---|---|
| Safety, Tolerability, and Pharmacokinetics of SVG103 (Paxalisib) in Focal Cortical Dysplasia Type II (FCD-II), Tuberous Sclerosis Complex (TSC) or Hemimegalencephaly (HME) SVG103 - Focal Cortical Dysplasia, Tuberous Sclerosis Complex (TSC) | Phase 1 / Phase 2 | Recruiting | Sovargen | 12 | 2026-07-14 | 2026-08-10 |
| Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD) Hematopoietic stem cell transplant (HSCT) - Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder | Phase 2 | Recruiting | Paul Szabolcs | 25 | 2026-05-04 | 2026-05-15 |
| A Phase 1b/2 Study of the Safety and Efficacy of the Monoclonal Antibody OM-RCA-01 in Patients With Metastatic Tumors Expressing Fibroblast Growth Factor Receptor 1 humanized monoclonal anti-FGFR1 antibody OM-RCA-01 - Renal Cell Carcinoma Metastatic, Prostate Cancer Metastatic | Phase 1 / Phase 2 | Recruiting | Kidney Cancer Research Bureau | 58 | 2025-12-02 | 2026-01-21 |
| Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental Disorders Sertraline, Placebo - Neurodevelopmental Disorders, Autism | Phase 2 | Recruiting | Holland Bloorview Kids Rehabilitation Hospital | 130 | 2024-09-16 | 2026-07-27 |
| MEK Inhibitors for the Treatment of Hypertrophic Cardiomyopathy in Patients With RASopathies Trametinib tablet, Standard therapy - Cardiomegaly, Noonan Syndrome | Phase 2 | Recruiting | Medical University of Warsaw | 40 | 2024-08-01 | 2024-08-15 |
| Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt) MELPIDA - Spasticity, Muscle, Microcephaly | Phase 1 / Phase 2 | Recruiting | Elpida Therapeutics SPC | 4 | 2023-02-15 | 2026-07-08 |
| NCCH2006/MK010 Trial (FORTUNE Trial) E7090 - Advanced or Recurrent Solid Tumors, FGFR Gene Alterations | Phase 2 | Recruiting | National Cancer Center, Japan | 75 | 2021-06-15 | 2025-10-03 |
Acrocephalosyndactylia (2) · DiGeorge Syndrome (2) · Tuberous Sclerosis (2) · 22q11 Deletion Syndrome (1) · Anxiety Disorders (1) · Attention Deficit Disorder with Hyperactivity (1) · Autism Spectrum Disorder (1) · Autistic Disorder (1) · Breast Neoplasms (1) · Cardiomegaly (1) · Cardiomyopathies (1) · Common Variable Immunodeficiency (1) · Focal Cortical Dysplasia (1) · Fragile X Syndrome (1) · Granulomatous Disease, Chronic (1)
Congenital Abnormalities (7) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (7) · Musculoskeletal Abnormalities (7) · Musculoskeletal Diseases (7) · Pathological Conditions, Signs and Symptoms (4) · Cardiovascular Abnormalities (3) · Cardiovascular Diseases (3) · Genetic Diseases, Inborn (3) · Heart Defects, Congenital (3) · Heart Diseases (3) · Malformations of Cortical Development (3) · Malformations of Cortical Development, Group I (3) · Neoplasms (3) · Nervous System Diseases (3)
Selective Serotonin Reuptake Inhibitors (1) · Sertraline (1) · Standard of Care (1) · Stem Cell Transplantation (1) · trametinib (1)
Elpida Therapeutics SPC (1) · Holland Bloorview Kids Rehabilitation Hospital (1) · Kidney Cancer Research Bureau (1) · Medical University of Warsaw (1) · National Cancer Center, Japan (1) · Paul Szabolcs (1) · Sovargen (1)
United States (2) · Australia (1) · Canada (1) · Japan (1) · Poland (1) · Russia (1)
Dr. Evdokia Anagnostou (1) · Paul Szabolcs, MD (1) · Susan T. Iannaccone, MD, FAAN (1)