42 completed, 7 recruiting, 7 unknown, 5 active not recruiting. 70 Phase 2, 19 Phase 1, 8 Phase 3.
Follow Neoplasms, Multiple Primary: new trials by email Literature Review of Neoplasms, Multiple Primary trials Research Report on Neoplasms, Multiple Primary
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 |
| Accuracy of 18F-Fluorocholine PET/MR and NeuroEXPLORER PET/CT Imaging for Localization of Parathyroid Tumors 18F-FCH, NeuroEXPLORER - Primary Hyperparathyroidism, Parathyroid Cancer | Phase 2 / Phase 3 | Recruiting | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | 193 | 2026-04-16 | 2026-10-29 |
| Proof-of-Concept Trial to Assess the Efficacy and Safety of Fezolinetant in Improving Vasomotor Symptoms in Men With Prostate Cancer Undergoing Androgen Deprivation Therapy fezolinetant - reference formulation, Placebo - Prostate Cancer, Prostate Cancer (Adenocarcinoma) | Phase 2 | Recruiting | Shehzad Basaria, M.D. | 60 | 2026-01-14 | 2026-08-17 |
| 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 |
| Open Label, Dose Escalation, Repeat Dose Study Evaluating YCT-529 in Healthy Males YCT-529 - Male Contraception | Phase 1 / Phase 2 | Recruiting | YourChoice Therapeutics, Inc. | 88 | 2024-09-11 | 2026-02-13 |
| 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 | |
| Study to Evaluate the Effect of Metformin in the Prevention of HG in HR[+]/HER2[-] PIK3CA-mut Advanced BC Patients Alpelisib, Metformin, Fulvestrant - Breast Cancer | Phase 2 | Completed | MedSIR | 69 | 2020-10-23 | 2025-07-29 |
| 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 |
| Phase II Clinical Study of Leflunomide in the Treatment of MEN-1 Neuroendocrine Tumor Leflunomide Pill - Neuroendocrine Tumors | Phase 2 | Unknown | Cancer Institute and Hospital, Chinese Academy of Medical Sciences | 50 | 2020-07-01 | 2024-08-07 |
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Tuberous Sclerosis (35) · Multiple Endocrine Neoplasia Type 1 (20) · Epilepsy (11) · Autistic Disorder (6) · Hamartoma Syndrome, Multiple (6) · Neuroendocrine Tumors (6) · Lymphangioleiomyomatosis (5) · Multiple Endocrine Neoplasia (5) · Seizures (5) · Hereditary Sensory and Autonomic Neuropathies (4) · Breast Neoplasms (3) · CDKL5 deficiency disorder (3) · Carcinoma, Medullary (3) · Epilepsies, Myoclonic (3) · Focal Cortical Dysplasia (3)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (69) · Genetic Diseases, Inborn (68) · Neoplastic Syndromes, Hereditary (68) · Neoplasms (67) · Hamartoma (42) · Nervous System Diseases (40) · Congenital Abnormalities (38) · Neurodegenerative Diseases (37) · Heredodegenerative Disorders, Nervous System (36) · Nervous System Malformations (36) · Neurocutaneous Syndromes (36) · Malformations of Cortical Development, Group I (35) · Malformations of Cortical Development (34) · Endocrine System Diseases (27)
Everolimus (15) · Sirolimus (14) · Sodium Chloride (2) · Vigabatrin (2) · gallium Ga 68 dotatate (2) · (18F)GTP1 (1) · 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) · Alpelisib (1) · Anastrozole (1) · Anticonvulsants (1) · Arginine (1) · Aspirin (1) · Autogenic Training (1) · Cytidine Triphosphate (1)
Children's Hospital Medical Center, Cincinnati (6) · National Cancer Institute (NCI) (6) · Boston Children's Hospital (2) · Cardiff University (2) · Dana-Farber Cancer Institute (2) · Longboard Pharmaceuticals (2) · National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) (2) · Novartis Pharmaceuticals (2) · Premier Research (2) · Aeovian Pharmaceuticals, Inc. (1) · Astellas Pharma Inc (1) · Aucta Pharmaceuticals, Inc (1) · Beth Israel Deaconess Medical Center (1) · Brigham and Women's Hospital (1) · Cancer Institute and Hospital, Chinese Academy of Medical Sciences (1)
United States (51) · Spain (8) · Australia (7) · Canada (6) · China (6) · United Kingdom (6) · Italy (5) · Poland (4) · France (3) · Israel (3) · Netherlands (3) · Belgium (2) · New Zealand (2) · Taiwan (2) · Chile (1)
Dennis J Dlugos, MD (2) · John Bissler, MD (2) · Martina Bebin, MD, MPA (2) · Mustafa Sahin, MD, PhD (2) · Smita Jha, M.D. (2) · Andrew Herzog, M.D., M.Sc. (1) · Annamaria Colao, MD, PhD (1) · Antonio Hardan, MD (1) · Antonio Llombart (1) · Arun Rajan, M.D. (1) · Bhavana Konda (1) · Brigitte C Widemann, M.D. (1) · Carmen P Priolo, MD PhD (1)
PMID 18184959, PMID 21047224, PMID 21482669, PMID 21525172, PMID 21915260, PMID 22262746, PMID 23325902, PMID 23344409, PMID 23437092, PMID 23766359, PMID 23798472, PMID 24617713, PMID 25578927, PMID 25956795, PMID 25972901, PMID 26381530, PMID 29023494, PMID 29187393, PMID 29500160, PMID 29800048, PMID 30424962, PMID 31217257, PMID 31350329, PMID 31912454, PMID 34118197, PMID 34118198, PMID 35169281, PMID 35587487, PMID 35594551, PMID 35715405, PMID 35841234, PMID 35962206, PMID 36347310, PMID 37282666, PMID 37463422, PMID 37638552, PMID 37916501, PMID 37934000, PMID 38009200, PMID 39289825, PMID 39331837, PMID 40478255, PMID 40639400, PMID 41886723, PMID 42224880, PMID 42562625
2018 (2), P30CA016672 (2), 2016 (1), 2019 (1), 2023 (1), 2025 (1), K08MH094441 (1), N01CM00039 (1), N01CM00070 (1), N01CM62202 (1), N01CM62207 (1), P01DK068055 (1), P30CA006516 (1), P30CA016058 (1), R01CA107164 (1)