11 completed, 4 recruiting, 2 active not recruiting, 1 terminated. 18 Phase 1, 12 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 18 | Completed 11, Recruiting 4, Active not recruiting 2, Terminated 1 |
| Phase 2 | 12 | Completed 7, Recruiting 3, Active not recruiting 2 |
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, 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| Safety of Simvastatin in LAM and TSC Simvastatin, Sirolimus Oral Product, Everolimus Oral Product - Lymphangioleiomyomatosis, Tuberous Sclerosis Complex | Phase 1 / Phase 2 | Completed | University of Pennsylvania | 10 | 2014-03-01 | 2020-08-25 |
| Tuberous Sclerosis Complex: Facial Angiofibroma Skin Cream Rapamune - Facial Angiofibroma | Phase 1 | Completed | Le Bonheur Children's Hospital | 11 | 2013-10-01 | 2016-08-19 |
| Assessment of the Pharmacokinetics of Circadin® in Children With Neurodevelopmental Disorders and Sleep Disturbances Circadin 2 mg, Circadin 10 mg - Sleep Problems | Phase 1 | Completed | Neurim Pharmaceuticals Ltd. | 16 | 2013-03-01 | 2014-04-04 |
| Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex (TSC) Everolimus - Epilepsy, Tuberous Sclerosis Complex | Phase 1 / Phase 2 | Completed | Children's Hospital Medical Center, Cincinnati | 20 | 2010-01-01 | 2017-03-13 |
| Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1) Skincerity, Skincerity plus sirolimus/rapamycin, Skinercity plus sirolimus/rapamycin - Tuberous Sclerosis, Neurofibromatoses | Phase 1 | Completed | The University of Texas Health Science Center, Houston | 52 | 2009-12-01 | 2012-02-27 |
| Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis (TSC) and Sporadic Lymphangioleiomyomatosis (LAM) everolimus (RAD001) - Tuberous Sclerosis, Angiolipoma | Phase 1 / Phase 2 | Completed | Children's Hospital Medical Center, Cincinnati | 20 | 2008-12-01 | 2013-09-18 |
| Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex Everolimus - Tuberous Sclerosis, Subependymal Giant Cell Astrocytoma | Phase 1 / Phase 2 | Completed | Children's Hospital Medical Center, Cincinnati | 28 | 2007-01-01 | 2014-10-20 |
| RAD001 Therapy of Angiomyolipomata in Patients With TS Complex and Sporadic LAM RAD001 - Tuberous Sclerosis, Lymphangioleiomyomatosis | Phase 1 / Phase 2 | Completed | Children's Hospital Medical Center, Cincinnati | 36 | 2005-08-01 | 2013-09-25 |
Tuberous Sclerosis (16) · Epilepsy (4) · Focal Cortical Dysplasia (4) · Hereditary Sensory and Autonomic Neuropathies (3) · Lymphangioleiomyomatosis (3) · Angiofibroma (2) · Angelman Syndrome (1) · Angiolipoma (1) · Astrocytoma (1) · Autism Spectrum Disorder (1) · CDKL5 deficiency disorder (1) · Carcinoma, Hepatocellular (1) · Carcinoma, Renal Cell (1) · Epilepsies, Myoclonic (1) · Epileptic Syndromes (1)
Congenital Abnormalities (18) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (18) · Malformations of Cortical Development, Group I (18) · Nervous System Diseases (18) · Nervous System Malformations (18) · Genetic Diseases, Inborn (16) · Hamartoma (16) · Heredodegenerative Disorders, Nervous System (16) · Neoplasms (16) · Neoplasms, Multiple Primary (16) · Neoplastic Syndromes, Hereditary (16) · Neurocutaneous Syndromes (16) · Neurodegenerative Diseases (16) · Neoplasms by Histologic Type (8)
Everolimus (6) · Sirolimus (4) · radiprodil (3) · Carbamazepine (1) · Injections (1) · Itraconazole (1) · Melatonin (1) · Simvastatin (1) · carbon-11 acetate (1) · exemestane (1)
Children's Hospital Medical Center, Cincinnati (5) · GRIN Therapeutics, Inc. (3) · Brigham and Women's Hospital (1) · Elpida Therapeutics SPC (1) · Le Bonheur Children's Hospital (1) · Longboard Pharmaceuticals (1) · Neurim Pharmaceuticals Ltd. (1) · SAPU NANO (US) LLC (1) · Sovargen (1) · The University of Texas Health Science Center, Houston (1) · UniQure Biopharma B.V. (1) · University of Pennsylvania (1)
United States (12) · Australia (6) · Netherlands (2) · Belgium (1) · Canada (1) · Italy (1) · Poland (1) · Spain (1) · United Kingdom (1)
Carmen P Priolo, MD PhD (1) · Darcy Krueger, M.D. Ph.D (1) · Darcy Krueger, MD, PhD (1) · David N Franz, M.D. (1) · Dennis J Dlugos, MD (1) · Frank McCormack, MD (1) · Hope Northrup, MD (1) · James W Wheless, MD (1) · John Bissler, MD (1) · John J. Bissler, M.D. (1) · Mary Kay Koenig, MD (1) · Maryl Kreider, MD, MSCE (1) · Russell Chin (1) · Susan T. Iannaccone, MD, FAAN (1)
PMID 21047224, PMID 21482669, PMID 22262746, PMID 22934754, PMID 23325902, PMID 23798472, PMID 26381530, PMID 29023494, PMID 30424962, PMID 39331837, PMID 41491634
2023 (1)