79 completed, 35 recruiting, 18 unknown, 12 active not recruiting. 39 Phase 2, 25 Not applicable, 20 Phase 3, 18 Phase 1, 6 Phase 4.
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 |
|---|---|---|---|---|---|---|
| Neurocutaneous Syndromes in Children: Prevalence and Clinical Characteristics Sturge-Weber Syndrome, Neurocutaneous Syndromes | Not yet recruiting | Assiut University | 100 | 2026-09-20 | 2026-09-28 | |
| 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 |
| Periodontal Disease in Rare Renal Disorders (PERIO-RA-RE) Observational assessment - Periodontal Disease, Periodontitis | Recruiting | Stefan Lujinschi | 100 | 2026-05-04 | 2026-06-26 | |
| A Smart Phone Application to Improve Adoption of the 2024 Kidney Disease Improving Global Outcomes (KDIGO) Chronic Kidney Disease (CKD) Guidelines Use of an electronic digital Application - Chronic Kidney Disease, Proteinuria | Not applicable | Recruiting | St. James's Hospital, Ireland | 80 | 2026-01-14 | 2026-05-01 |
| Sapu003 in Advanced mTOR-sensitive Solid Tumors Sapu003, Exemestane 25 MG - Breast Cancer Metastatic, Renal Cell Carcinoma (RCC) | Phase 1 | Recruiting | SAPU NANO (US) LLC | 27 | 2025-12-15 | 2026-01-27 |
| Quantitative Assessment of the Etiologies of Megalencephaly Associated With a Detectable Tumor Risk Megalocephaly | Not yet recruiting | Assistance Publique - Hôpitaux de Paris | 200 | 2025-09-01 | 2025-08-27 | |
| TANDem-2: Closing the Gap to Interventions for TAND Well-Beans for Caregivers Program - Adult Caregivers of Individuals With TSC, Tuberous Sclerosis Complex (TSC) | Not applicable | Enrolling by invitation | Vrije Universiteit Brussel | 30 | 2025-07-15 | 2025-09-22 |
| Phase 1, Open-label, Drug-drug Interaction Study to Assess Effect Between Radiprodil and the Co-administered Drugs. Radiprodil + co-administered drugs - Tuberous Sclerosis Complex (TSC), Focal Cortical Dysplasia | Phase 1 | Completed | GRIN Therapeutics, Inc. | 18 | 2025-05-20 | 2025-11-18 |
| Study to Evaluate the Pharmacokinetics and Safety Effects Following Coadministration of Carbamazepine or Itraconazole With Radiprodil in Healthy Adults Radiprodil with Carbamazepine, Radiprodil with Itraconazole - Tuberous Sclerosis Complex (TSC), Focal Cortical Dysplasia | Phase 1 | Terminated | GRIN Therapeutics, Inc. | 30 | 2025-05-08 | 2025-11-21 |
| Cartography of Social Cognition Network and Their Alterations in Patients With Epilepsy MRI 3T and MRI 7T - Epilepsy, Drug Resistant Epilepsy | Not applicable | Recruiting | Assistance Publique - Hôpitaux de Paris | 20 | 2025-03-19 | 2025-03-21 |
| Epidyolex® in Lennox Gastaut, Dravet Syndrome and Tuberous Sclerosis Complex: an Observational Study in ITALY Epidiolex 100 mg/mL Oral Solution - Lennox Gastaut Syndrome, Dravet Syndrome | Active not recruiting | Jazz Pharmaceuticals | 111 | 2025-02-24 | 2026-06-03 | |
| Normative Brain Volume Profiles From Multicenter Fetal MRI Questionnaire - Cortical Development Malformation, Multiparametric Magnetic Resonnance Imaging | Recruiting | Assistance Publique Hopitaux De Marseille | 1423 | 2024-12-02 | 2025-06-11 | |
| 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 |
| Exploration and Evaluation of Amygdalo-Hippocampectomy According to Prof. Coubes' Technique: An Anatomical, Clinical, and Educational Approach Amygdalo-Hippocampectomy - Hippocampal Sclerosis, Dysembryoplastic Neuroepithelial Tumor | Recruiting | University Hospital, Montpellier | 3504 | 2024-08-31 | 2025-04-08 | |
| 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 |
| Treatment With Full-spectrum Cannabis Extract of Refractory Epilepsy Associated With Tuberous Sclerosis Complex (TSC) YCJ-01 Full Spectrum cannbis Extract, Placebo - Tuberous Sclerosis Complex (TSC) | Phase 3 | Recruiting | Oils4Cure | 84 | 2024-06-01 | 2026-02-11 |
| First-in-human Experience Using Novel Ultraflexible Low-impedance Electrode Arrays: an IDEAL Stage 1 Study Insertion of electrode during planned neurosurgery for epilepsy - Epilepsy, Focal Cortical Dysplasia | Not applicable | Unknown | Great Ormond Street Hospital for Children NHS Foundation Trust | 6 | 2024-02-01 | 2023-11-03 |
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Tuberous Sclerosis (114) · Epilepsy (37) · Microcephaly (22) · Seizures (18) · Lymphangioleiomyomatosis (16) · Autistic Disorder (14) · Focal Cortical Dysplasia (12) · Intellectual Disability (12) · Lennox Gastaut Syndrome (9) · Epilepsies, Myoclonic (8) · Hippocampal Sclerosis (8) · Angiomyolipoma (7) · Drug Resistant Epilepsy (6) · Megalencephaly (6)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (167) · Nervous System Malformations (166) · Nervous System Diseases (165) · Congenital Abnormalities (162) · Malformations of Cortical Development, Group I (154) · Genetic Diseases, Inborn (127) · Heredodegenerative Disorders, Nervous System (118) · Neurodegenerative Diseases (118) · Hamartoma (116) · Neoplasms, Multiple Primary (116) · Neoplasms (115) · Neoplastic Syndromes, Hereditary (115) · Neurocutaneous Syndromes (111) · Central Nervous System Diseases (56)
Everolimus (18) · Sirolimus (15) · Cannabidiol (6) · Activator Appliances (3) · Vigabatrin (3) · ganaxolone (3) · radiprodil (3) · Anticonvulsants (2) · Melatonin (2) · Phlebotomy (2) · Surveys and Questionnaires (2) · Urination (2) · 2-chloro-4-(1-(4-fluorophenyl)-2,5-dimethyl-1H-imidazol-4-ylethynyl)pyridine (1) · Acceptance and Commitment Therapy (1) · Actigraphy (1)
Children's Hospital Medical Center, Cincinnati (7) · Jazz Pharmaceuticals (6) · Novartis Pharmaceuticals (6) · University of California, Los Angeles (6) · National Heart, Lung, and Blood Institute (NHLBI) (5) · Assistance Publique - Hôpitaux de Paris (4) · Beijing Children's Hospital (3) · Boston Children's Hospital (3) · GRIN Therapeutics, Inc. (3) · Institut National de la Santé Et de la Recherche Médicale, France (3) · Longboard Pharmaceuticals (3) · Marinus Pharmaceuticals (3) · Nobelpharma (3) · Cardiff University (2) · Dana-Farber Cancer Institute (2)
United States (84) · France (26) · United Kingdom (19) · Australia (18) · Canada (13) · Italy (13) · China (12) · Netherlands (12) · Spain (12) · Poland (11) · Belgium (7) · Germany (7) · Japan (6) · Brazil (5) · Israel (5)
Darcy Krueger, MD, PhD (3) · Joel Moss, M.D. (3) · Anna Jansen, MD, PhD (2) · Antoni Gostynski, MD, PhD (2) · Dennis J Dlugos, MD (2) · Hope Northrup, MD (2) · John Bissler, MD (2) · Martina Bebin, MD, MPA (2) · Mary Kay Koenig, MD (2) · Mustafa Sahin, MD, PhD (2) · Alessandra Pereira,, PhD, MD (1) · Alessandro Flavio Ferri (1)
PMID 29023494, PMID 30053159, PMID 30291925, PMID 31912454, PMID 34957550, PMID 38996393, PMID 16275846, PMID 17056927, PMID 17375041, PMID 17632789, PMID 17668379, PMID 18184959, PMID 19001538, PMID 19141364, PMID 19246528, PMID 20206699, PMID 20610117, PMID 20646909, PMID 20683985, PMID 21047224, PMID 21220724, PMID 21482669, PMID 21525172, PMID 21915260, PMID 22002941, PMID 22113127, PMID 22262746, PMID 22396514, PMID 22934754, PMID 23158522, PMID 23325902, PMID 23437092, PMID 23732715, PMID 23733802, PMID 23798472, PMID 24729041, PMID 25456370, PMID 25568299, PMID 25682485, PMID 26156073, PMID 26310427, PMID 26381530, PMID 26858193, PMID 27222591, PMID 27351628, PMID 27872178, PMID 28087826, PMID 28431241, PMID 28715527, PMID 28877754
2015 (2), 2018 (2), 2023 (2), 2025 (2), U54NS092090 (2), 2008 (1), P30CA006516 (1), R01AG061111 (1), R01CA107164 (1), R01HD090138 (1), R21CA103486 (1), U01HG006487 (1), U01NS082320 (1), U01NS092595 (1), U54NS053672 (1)