24 completed. 24 Phase 2, 9 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 24 | Completed 24 |
| Phase 1 | 9 | Completed 9 |
| Phase 3 | 1 | Completed 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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| Sirolimus for Cowden Syndrome With Colon Polyposis Sirolimus - PTEN Gene Mutation, PTEN Hamartoma Tumor Syndrome | Phase 2 | Completed | Ohio State University | 5 | 2019-09-16 | 2025-06-10 |
| 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 |
| 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 |
| Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex Early Vigabatrin, Delayed Vigabatrin (Placebo) - Tuberous Sclerosis Complex | Phase 2 | Completed | Martina Bebin | 84 | 2016-12-01 | 2024-08-19 |
| Everolimus for Cancer With TSC1 or TSC2 Mutation Everolimus - TSC1, TSC2 | Phase 2 | Completed | Dana-Farber Cancer Institute | 30 | 2014-09-01 | 2020-09-16 |
| 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 |
| A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCD Everolimus - Epilepsy, Tuberous Sclerosis Complex | Phase 2 | Completed | NYU Langone Health | 15 | 2014-01-01 | 2021-09-01 |
| Rapalogues for Autism Phenotype in TSC: A Feasibility Study Sirolimus, Everolimus - Tuberous Sclerosis Complex, Self-injury | Phase 2 | Completed | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | 3 | 2013-07-01 | 2021-03-29 |
| Topical Rapamycin to Erase Angiofibromas in TSC Placebo, Rapamycin - Angiofibromas, Tuberous Sclerosis | Phase 2 | Completed | The University of Texas Health Science Center, Houston | 179 | 2012-05-01 | 2020-10-26 |
| Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC) RAD001, Placebo - Tuberous Sclerosis Complex | Phase 2 | Completed | Mustafa Sahin | 52 | 2011-01-01 | 2018-01-25 |
| 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 |
| 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 |
| Sirolimus to Treat Cowden Syndrome and Other PTEN Hamartomatous Tumor Syndromes fludeoxyglucose F 18, sirolimus, Clinical Videography - Cowden's Disease, Hamartoma Syndrome, Multiple | Phase 2 | Completed | National Cancer Institute (NCI) | 18 | 2008-07-01 | 2015-09-30 |
| 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 |
Showing 20 of 24. Sign in to see all of them.
Tuberous Sclerosis (20) · Epilepsy (6) · Hamartoma Syndrome, Multiple (4) · Hereditary Sensory and Autonomic Neuropathies (4) · Autistic Disorder (3) · CDKL5 deficiency disorder (3) · Epilepsies, Myoclonic (3) · Lennox Gastaut Syndrome (3) · Lymphangioleiomyomatosis (3) · Seizures (2) · Angiofibroma (1) · Angiolipoma (1) · Angiomyolipoma (1) · Astrocytoma (1) · Brain Diseases (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (24) · Genetic Diseases, Inborn (24) · Neoplasms, Multiple Primary (24) · Neoplastic Syndromes, Hereditary (24) · Neoplasms (23) · Congenital Abnormalities (21) · Heredodegenerative Disorders, Nervous System (21) · Nervous System Diseases (21) · Nervous System Malformations (21) · Neurodegenerative Diseases (21) · Malformations of Cortical Development, Group I (20) · Neurocutaneous Syndromes (20) · Malformations of Cortical Development (19) · Neoplasms by Histologic Type (7)
Everolimus (10) · Sirolimus (9) · 2-chloro-4-(1-(4-fluorophenyl)-2,5-dimethyl-1H-imidazol-4-ylethynyl)pyridine (1) · 8-(6-methoxypyridin-3-yl)-3-methyl-1-(4-piperazin-1-yl-3-trifluoromethylphenyl)-1,3-dihydroimidazo(4,5-c)quinolin-2-one (1) · Fluorodeoxyglucose F18 (1) · Simvastatin (1) · Vigabatrin (1) · ganaxolone (1)
Children's Hospital Medical Center, Cincinnati (6) · Dana-Farber Cancer Institute (2) · Longboard Pharmaceuticals (2) · Boston Children's Hospital (1) · Dermatology Specialties Limited Partnership (1) · Hugo W. Moser Research Institute at Kennedy Krieger, Inc. (1) · Marinus Pharmaceuticals (1) · Martina Bebin (1) · Mustafa Sahin (1) · NYU Langone Health (1) · National Cancer Institute (NCI) (1) · Noema Pharma AG (1) · Novartis Pharmaceuticals (1) · Ohio State University (1) · Takeda (1)
United States (24) · Australia (5) · Spain (4) · Canada (2) · Israel (2) · Poland (2) · China (1) · Czechia (1) · Hungary (1) · India (1) · Italy (1) · New Zealand (1) · Portugal (1) · Serbia (1) · Slovakia (1)
Dennis J Dlugos, MD (2) · John Bissler, MD (2) · Mustafa Sahin, MD, PhD (2) · Arun Rajan, M.D. (1) · Darcy Krueger, M.D. Ph.D (1) · Darcy Krueger, MD, PhD (1) · David Kwiatkowski, MD, PhD (1) · David N Franz, M.D. (1) · Frank McCormack, MD (1) · Hope Northrup, MD (1) · Ioana Stanescu (1) · John J. Bissler, M.D. (1) · Martina Bebin, MD, MPA (1) · Mary Kay Koenig, MD (1)
PMID 18184959, PMID 21047224, PMID 21482669, PMID 21915260, PMID 22262746, PMID 23325902, PMID 23344409, PMID 23437092, PMID 23798472, PMID 26381530, PMID 29023494, PMID 29800048, PMID 30424962, PMID 31350329, PMID 31912454, PMID 35587487, PMID 35594551, PMID 35841234, PMID 37463422, PMID 37638552, PMID 39331837, PMID 40478255
2018 (1), P30CA006516 (1), R01CA107164 (1), R21CA103486 (1), U01NS092595 (1), U54NS092090 (1)