24 completed, 4 active not recruiting, 4 unknown, 3 recruiting. 42 Phase 2, 13 Phase 1, 5 Phase 3.
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 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 |
| Double-Blind Trial of Everolimus for Improving Social Abilities in PTEN Germline Mutations Everolimus blinded oral capsules, Placebo - Cowden's Disease, Cowden's Syndrome | Phase 2 / Phase 3 | Not yet recruiting | Boston Children's Hospital | 60 | 2026-09-01 | 2026-06-04 |
| 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 |
| 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PTEN Germline Mutations Sirolimus, Placebo - PTEN Gene Mutation, PTEN Hamartoma Tumor Syndrome | Phase 1 / Phase 2 | Withdrawn | Stanford University | 2024-07-01 | 2024-07-10 | |
| 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 |
| MK-7075 (Miransertib) in Proteus Syndrome MK-7075 (miransertib) - Proteus Syndrome | Phase 2 | Active not recruiting | National Human Genome Research Institute (NHGRI) | 38 | 2022-05-20 | 2026-07-17 |
| 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 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 |
| 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 |
| 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 42. Sign in to see all of them.
Tuberous Sclerosis (35) · Epilepsy (10) · Autistic Disorder (6) · Hamartoma Syndrome, Multiple (6) · Lymphangioleiomyomatosis (5) · Hereditary Sensory and Autonomic Neuropathies (4) · Seizures (4) · CDKL5 deficiency disorder (3) · Epilepsies, Myoclonic (3) · Focal Cortical Dysplasia (3) · Lennox Gastaut Syndrome (3) · Angiomyolipoma (2) · Intellectual Disability (2) · Neoplasms (2) · 22q11 Deletion Syndrome (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (42) · Neoplasms, Multiple Primary (42) · Genetic Diseases, Inborn (41) · Neoplastic Syndromes, Hereditary (41) · Neoplasms (40) · Congenital Abnormalities (37) · Heredodegenerative Disorders, Nervous System (36) · Nervous System Diseases (36) · Nervous System Malformations (36) · Neurodegenerative Diseases (36) · Malformations of Cortical Development, Group I (35) · Neurocutaneous Syndromes (35) · Malformations of Cortical Development (34) · Neoplasms by Histologic Type (11)
Everolimus (14) · Sirolimus (14) · Vigabatrin (2) · 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) · Anticonvulsants (1) · Aspirin (1) · Fluid Therapy (1) · Fluorodeoxyglucose F18 (1) · Miransertib (1) · Propranolol (1) · Selective Serotonin Reuptake Inhibitors (1) · Sertraline (1) · Simvastatin (1) · carbon-11 acetate (1)
Children's Hospital Medical Center, Cincinnati (6) · Boston Children's Hospital (2) · Cardiff University (2) · Dana-Farber Cancer Institute (2) · Longboard Pharmaceuticals (2) · Novartis Pharmaceuticals (2) · Aeovian Pharmaceuticals, Inc. (1) · Aucta Pharmaceuticals, Inc (1) · Brigham and Women's Hospital (1) · Darcy Krueger (1) · Dermatology Specialties Limited Partnership (1) · Erasmus Medical Center (1) · GRIN Therapeutics, Inc. (1) · Holland Bloorview Kids Rehabilitation Hospital (1) · Hospices Civils de Lyon (1)
United States (30) · Australia (7) · Spain (6) · Canada (5) · Poland (4) · United Kingdom (4) · China (3) · France (2) · 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) · Mustafa Sahin, MD, PhD (2) · Antonio Hardan, MD (1) · Arun Rajan, M.D. (1) · 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)
PMID 18184959, PMID 21047224, PMID 21482669, PMID 21525172, PMID 21915260, PMID 22262746, PMID 23325902, PMID 23344409, PMID 23437092, PMID 23798472, PMID 26381530, PMID 29023494, PMID 29800048, PMID 30424962, PMID 31217257, PMID 31350329, PMID 31912454, PMID 35587487, PMID 35594551, PMID 35841234, PMID 37463422, PMID 37638552, PMID 39331837, PMID 40478255, PMID 42224880, PMID 42562625
2018 (2), 2023 (1), 2025 (1), P30CA006516 (1), R01CA107164 (1), R21CA103486 (1), U01NS092595 (1), U54NS092090 (1)