15 completed, 2 active not recruiting, 2 recruiting, 1 terminated. 21 Phase 1, 13 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 21 | Completed 15, Active not recruiting 2, Recruiting 2, Terminated 1, Withdrawn 1 |
| Phase 2 | 13 | Completed 9, Active not recruiting 2, Recruiting 1, Withdrawn 1 |
Follow Hamartoma: new trials by email Literature Review of Hamartoma trials Research Report on Hamartoma
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 |
| Sirolimus for Improving Social Abilities in People With PTEN Germline Mutations Sirolimus, Placebo - PTEN Gene Mutation, PTEN Hamartoma Tumor Syndrome | Phase 1 / Phase 2 | Withdrawn | Stanford University | 2024-07-01 | 2024-07-10 | |
| 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 |
| RAD001 and Neurocognition in PTEN Hamartoma Tumor Syndrome RAD001, Placebo - PTEN Gene Mutation, PTEN Hamartoma Tumor Syndrome | Phase 1 / Phase 2 | Completed | Boston Children's Hospital | 46 | 2017-06-12 | 2025-02-17 |
| Dose Finding Trial of MK-7075 in Children and Adults With Proteus Syndrome MK-7075 (miransertib) - Proteus Syndrome | Phase 1 | Completed | National Human Genome Research Institute (NHGRI) | 6 | 2015-11-16 | 2022-06-30 |
| 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 |
| A Phase I/II Study of BGT226 in Adult Patients With Advanced Solid Malignancies Including Patients With Advanced Breast Cancer BGT226 - Solid Tumors, Breast Cancer | Phase 1 / Phase 2 | Completed | Novartis Pharmaceuticals | 57 | 2007-12-01 | 2020-12-17 |
| 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 |
| A Phase I/II Study of BEZ235 in Patients With Advanced Solid Malignancies Enriched by Patients With Advanced Breast Cancer BEZ235 - Breast Cancer, Advanced Solid Tumors | Phase 1 | Completed | Novartis Pharmaceuticals | 183 | 2006-12-21 | 2020-12-09 |
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Tuberous Sclerosis (16) · Focal Cortical Dysplasia (4) · Hamartoma Syndrome, Multiple (4) · Hereditary Sensory and Autonomic Neuropathies (4) · Epilepsy (3) · Lymphangioleiomyomatosis (3) · Angiofibroma (2) · Breast Neoplasms (2) · Angelman Syndrome (1) · Angiolipoma (1) · Astrocytoma (1) · Autism Spectrum Disorder (1) · Autistic Disorder (1) · Breast Diseases (1) · CDKL5 deficiency disorder (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (21) · Neoplasms, Multiple Primary (21) · Genetic Diseases, Inborn (20) · Neoplasms (20) · Neoplastic Syndromes, Hereditary (20) · Congenital Abnormalities (18) · Nervous System Diseases (18) · Heredodegenerative Disorders, Nervous System (17) · Nervous System Malformations (17) · Neurodegenerative Diseases (17) · Malformations of Cortical Development (16) · Malformations of Cortical Development, Group I (16) · Neurocutaneous Syndromes (16) · Neoplasms by Histologic Type (8)
Everolimus (7) · Sirolimus (5) · radiprodil (3) · 8-(6-methoxypyridin-3-yl)-3-methyl-1-(4-piperazin-1-yl-3-trifluoromethylphenyl)-1,3-dihydroimidazo(4,5-c)quinolin-2-one (1) · Carbamazepine (1) · Injections (1) · Itraconazole (1) · Melatonin (1) · Miransertib (1) · Simvastatin (1) · carbon-11 acetate (1) · dactolisib (1) · exemestane (1)
Children's Hospital Medical Center, Cincinnati (5) · GRIN Therapeutics, Inc. (3) · Novartis Pharmaceuticals (2) · Boston Children's Hospital (1) · Brigham and Women's Hospital (1) · Le Bonheur Children's Hospital (1) · Longboard Pharmaceuticals (1) · National Human Genome Research Institute (NHGRI) (1) · Neurim Pharmaceuticals Ltd. (1) · SAPU NANO (US) LLC (1) · Sovargen (1) · Stanford University (1) · The University of Texas Health Science Center, Houston (1) · University of Pennsylvania (1)
United States (15) · Australia (6) · Netherlands (3) · Spain (3) · Canada (2) · United Kingdom (2) · Belgium (1) · Germany (1) · Italy (1) · Poland (1)
Antonio Hardan, MD (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) · Leslie G Biesecker, M.D. (1) · Mary Kay Koenig, MD (1) · Maryl Kreider, MD, MSCE (1)
PMID 21047224, PMID 21482669, PMID 22262746, PMID 22934754, PMID 23325902, PMID 23798472, PMID 26381530, PMID 29023494, PMID 30424962, PMID 30803705, PMID 34649967, PMID 35594551, PMID 39331837
2023 (1), U54NS092090 (1)