21 completed, 5 recruiting, 4 unknown, 3 active not recruiting. 39 Phase 2, 12 Phase 1, 4 Phase 3.
Follow Malformations of Cortical Development: new trials by email Literature Review of Malformations of Cortical Development trials Research Report on Malformations of Cortical Development
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 and Efficacy of AV078 in Participants With Tuberous Sclerosis Complex (TSC) Refractory Epilepsy (RESTOR-1) AV078, Placebo - Tuberous Sclerosis Complex, Epilepsy | Phase 2 | Not yet recruiting | Aeovian Pharmaceuticals, Inc. | 42 | 2026-09-01 | 2026-09-29 |
| Fluorescence Guided Focal Cortical Dysplasia Surgery 5-ALA (Gliolan) - Drug Resistant Epilepsy | Phase 2 | Not yet recruiting | Hospices Civils de Lyon | 5 | 2026-09-01 | 2026-06-18 |
| 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 |
| 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 |
| Astroscape: A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II Radiprodil - Tuberous Sclerosis Complex, Focal Cortical Dysplasia | Phase 1 / Phase 2 | Active not recruiting | GRIN Therapeutics, Inc. | 30 | 2024-07-10 | 2026-04-01 |
| AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy AAV9-hSyn1-miGRIK2 - Mesial Temporal Lobe Epilepsy | Phase 1 / Phase 2 | Recruiting | UniQure Biopharma B.V. | 12 | 2024-06-12 | 2026-08-24 |
| 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 |
| Open-label, Long-term Safety Study of LP352 in Subjects With Developmental and Epileptic Encephalopathy LP352, bexicaserin - Developmental and Epileptic Encephalopathy, Dravet Syndrome | Phase 2 | Completed | Longboard Pharmaceuticals | 41 | 2022-11-08 | 2025-12-03 |
| Study to Investigate LP352 in Subjects With Developmental and Epileptic Encephalopathies LP352, Placebo - Developmental and Epileptic Encephalopathy, Dravet Syndrome | Phase 1 / Phase 2 | Completed | Longboard Pharmaceuticals | 52 | 2022-03-03 | 2024-11-08 |
| Basimglurant (NOE-101) in Children, Adolescents, and Young Adults With TSC Basimglurant with crossover to Placebo, Placebo with crossover to Basimglurant - Tuberous Sclerosis Complex | Phase 2 | Completed | Noema Pharma AG | 61 | 2022-03-03 | 2025-06-04 |
| Safety and Modulation of ABCC9 Pathways by Nicorandil for the Treatment of Hippocampal Sclerosis of Aging Nicorandil, Placebo - Dementia, Mild Cognitive Impairment | Phase 2 | Completed | Gregory Jicha, MD, PhD | 64 | 2021-12-01 | 2026-11-10 |
| Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study Sirolimus, Placebo - Tuberous Sclerosis Complex, Epilepsy | Phase 2 | Active not recruiting | Darcy Krueger | 64 | 2021-10-13 | 2026-10-29 |
| Feasibility of [11C]Acetate-PET in LAM and TSC [11C]acetate - Lymphangioleiomyomatosis, Tuberous Sclerosis Complex | Phase 1 / Phase 2 | Active not recruiting | Brigham and Women's Hospital | 7 | 2021-08-30 | 2026-01-21 |
| Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants Vigabatrin, Rapamycin, Placebo - Tuberous Sclerosis Complex | Phase 2 / Phase 3 | Recruiting | Katarzyna Kotulska | 60 | 2021-05-07 | 2026-10-22 |
| Topical Everolimus in Patients With Tuberous Sclerosis Complex Everolimus, Placebo - Facial Angiofibromas | Phase 2 / Phase 3 | Withdrawn | Hospices Civils de Lyon | 2020-12-01 | 2021-05-03 | |
| Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants TAVT-18 (sirolimus) - Tuberous Sclerosis Complex, Epilepsy | Phase 1 / Phase 2 | Completed | Children's Hospital Medical Center, Cincinnati | 5 | 2020-09-08 | 2024-05-16 |
| Adjunctive Ganaxolone Treatment (Part A) in TSC Followed by Long-term Treatment (Part B) Ganaxolone - Tuberous Sclerosis | Phase 2 | Completed | Marinus Pharmaceuticals | 23 | 2020-04-08 | 2023-04-04 |
| Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex rapamycin, placebo - Facial Angiofibroma, Tuberous Sclerosis | Phase 2 / Phase 3 | Completed | Dermatology Specialties Limited Partnership | 107 | 2019-07-28 | 2023-09-08 |
| A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic Encephalopathies TAK-935, Placebo - Epilepsy, Dravet Syndrome | Phase 2 | Completed | Takeda | 141 | 2018-08-08 | 2026-09-14 |
| Aspirin as an add-on Treatment of Refractory Epilepsy in Tuberous Sclerosis Complex Aspirin, AED, Placebo - Tuberous Sclerosis Complex, Aspirin | Phase 2 | Unknown | Peking Union Medical College Hospital | 98 | 2017-11-20 | 2020-06-09 |
Showing 20 of 39. Sign in to see all of them.
Tuberous Sclerosis (35) · Epilepsy (12) · Lymphangioleiomyomatosis (5) · Autistic Disorder (4) · Focal Cortical Dysplasia (4) · Seizures (4) · CDKL5 deficiency disorder (3) · Epilepsies, Myoclonic (3) · Hereditary Sensory and Autonomic Neuropathies (3) · Lennox Gastaut Syndrome (3) · Angiomyolipoma (2) · Cognitive Dysfunction (2) · Drug Resistant Epilepsy (2) · Hippocampal Sclerosis (2) · Neoplasms (2)
Congenital Abnormalities (39) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (39) · Malformations of Cortical Development, Group I (39) · Nervous System Malformations (39) · Nervous System Diseases (38) · Genetic Diseases, Inborn (35) · Hamartoma (35) · Heredodegenerative Disorders, Nervous System (35) · Neoplasms, Multiple Primary (35) · Neoplastic Syndromes, Hereditary (35) · Neurocutaneous Syndromes (35) · Neurodegenerative Diseases (35) · Neoplasms (33) · Brain Diseases (13)
Everolimus (12) · Sirolimus (11) · Vigabatrin (2) · 2-chloro-4-(1-(4-fluorophenyl)-2,5-dimethyl-1H-imidazol-4-ylethynyl)pyridine (1) · Anticonvulsants (1) · Aspirin (1) · Fluid Therapy (1) · Nicorandil (1) · Propranolol (1) · Selective Serotonin Reuptake Inhibitors (1) · Sertraline (1) · Simvastatin (1) · carbon-11 acetate (1) · dactolisib (1) · ganaxolone (1)
Children's Hospital Medical Center, Cincinnati (6) · Cardiff University (2) · Dana-Farber Cancer Institute (2) · Hospices Civils de Lyon (2) · Longboard Pharmaceuticals (2) · Aeovian Pharmaceuticals, Inc. (1) · Aucta Pharmaceuticals, Inc (1) · Brigham and Women's Hospital (1) · Darcy Krueger (1) · Dermatology Specialties Limited Partnership (1) · Elpida Therapeutics SPC (1) · Erasmus Medical Center (1) · GRIN Therapeutics, Inc. (1) · Gregory Jicha, MD, PhD (1) · Holland Bloorview Kids Rehabilitation Hospital (1)
United States (26) · Australia (7) · Spain (5) · Canada (4) · Poland (4) · United Kingdom (4) · China (3) · France (3) · Israel (2) · Italy (2) · Netherlands (2) · Belgium (1) · Czechia (1) · Hungary (1) · India (1)
Dennis J Dlugos, MD (2) · John Bissler, MD (2) · Martina Bebin, MD, MPA (2) · Carmen P Priolo, MD PhD (1) · Darcy A Krueger, MD, PhD (1) · Darcy Krueger, M.D. Ph.D (1) · Darcy Krueger, MD, PhD (1) · David Kwiatkowski, MD, PhD (1) · David N Franz, M.D. (1) · Dr. Evdokia Anagnostou (1) · Frank McCormack, MD (1)
PMID 18184959, PMID 21047224, PMID 21482669, PMID 21525172, PMID 21915260, PMID 22262746, PMID 23325902, PMID 23437092, PMID 23798472, PMID 26381530, PMID 29023494, PMID 29800048, PMID 30424962, PMID 31217257, PMID 31912454, PMID 35587487, PMID 35841234, PMID 37463422, PMID 37638552, PMID 39331837, PMID 41491634, PMID 42562625
2018 (2), 2025 (2), 2023 (1), P30CA006516 (1), R01AG061111 (1), R01CA107164 (1), R21CA103486 (1), U01NS092595 (1)