144 completed, 35 recruiting, 32 terminated, 28 active not recruiting. 285 Phase 2, 58 Phase 1, 17 Phase 3.
Follow Neoplastic Syndromes, Hereditary: new trials by email Literature Review of Neoplastic Syndromes, Hereditary trials Research Report on Neoplastic Syndromes, Hereditary
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 |
|---|---|---|---|---|---|---|
| Efficacy of Mirdametinib Alone or Combination With Radiotherapy for Germline and Sporadic NF1-Altered High-Grade Glioma mirdametinib with radiation, mirdametinib - Neurofibromatosis 1 (NF1) | Phase 2 | Not yet recruiting | University of Alabama at Birmingham | 55 | 2027-01-01 | 2026-10-05 |
| Combination of Avutometinib and Defactinib for Treatment of Relapsed, Refractory, Metastatic, or Unresectable Malignant Peripheral Nerve Sheath Tumor avutometinib capsule + defactinib tablet - MPNST (Malignant Peripheral Nerve Sheath Tumor) | Phase 2 | Not yet recruiting | Girish Dhall, MD | 25 | 2027-01-01 | 2026-10-28 |
| Selumetinib for NF2-Related Schwannomatosis Selumetinib - Neurofibromatosis Type 2, NF2 | Phase 2 | Not yet recruiting | Beijing Tiantan Hospital | 20 | 2026-12-01 | 2026-10-28 |
| Preventive Dendritic Cell Vaccination for Lynch Syndrome DC vaccination - Lynch Syndrome | Phase 1 / Phase 2 | Not yet recruiting | Radboud University Medical Center | 16 | 2026-10-01 | 2026-08-19 |
| A Study to Evaluate the Efficacy of VVD-133214 in Combination With Pembrolizumab Compared With Pembrolizumab Alone in Participants With Advanced Cancer of the Colon or Rectum VVD-133214, Pembrolizumab - Colorectal Adenocarcinoma | Phase 2 | Recruiting | Vividion Therapeutics, Inc. | 140 | 2026-09-11 | 2026-10-21 |
| Safety Study for the Use of Rapamycin in Children With Familial Adenomatous Polyposis Rapamycin - Familial Adenomatous Polyposis | Phase 2 | Not yet recruiting | University Hospital, Toulouse | 25 | 2026-09-01 | 2026-05-08 |
| 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 |
| Trial of Selumetinib and Bromodomain Inhibitor With Durvalumab for Sarcomas Selumetinib + ZEN-3694 ± Durvalumab - MPNST, NF1 | Phase 2 | Recruiting | University of Alabama at Birmingham | 41 | 2026-08-04 | 2026-08-24 |
| Luvometinib in Combination With Serplulimab for NF2-Related Tumors Luvometinib, Serplulimab - NF2-related Schwannomatosis, NF2 | Phase 1 / Phase 2 | Not yet recruiting | Beijing Tiantan Hospital | 30 | 2026-07-31 | 2026-07-16 |
| Platform Research for Innovative Medicines in NF2-SWN (PRIME-NF2) Selumetinib 25mg/m2, Luvometinib, Serplulimab - Neurofibromatosis Type 2, Vestibular Schwannoma | Phase 2 | Not yet recruiting | Beijing Tiantan Hospital | 200 | 2026-07-18 | 2026-07-20 |
| 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 |
| Study to Determine Optimal Dose, Evaluate the Efficacy and Safety of PRG-N-01 in Patients With Neurofibromatosis Type II Trineumin - Neurofibromatosis Type II, NF2 | Phase 1 / Phase 2 | Not yet recruiting | PRG Science & Technology Co., Ltd. | 25 | 2026-07-01 | 2026-01-23 |
| Early Phase Study Evaluating MEK and MDM2 Inhibition in Patients With NF1 and MPNST APG-115, Selumetinib - Malignant Peripheral Nerve Sheath Tumor (MPNST), Neurofibromatosis 1 (NF1) | Phase 1 / Phase 2 | Recruiting | AeRang Kim | 45 | 2026-06-01 | 2026-07-27 |
| 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 |
| An Investigational Drug (TPST-1495) in Patients With Familial Adenomatous Polyposis Biopsy Procedure, Biospecimen Collection, EP2/EP4 Antagonist TPST-1495 - Colorectal Carcinoma, Familial Adenomatous Polyposis | Phase 2 | Recruiting | National Cancer Institute (NCI) | 38 | 2026-03-31 | 2026-09-14 |
| Safety and Efficacy of Tegavivint in Patients With Metastatic Colorectal Carcinoma Tegavivint - Metastatic Colorectal Carcinoma (mCRC), Colorectal Cancer (CRC) | Phase 1 / Phase 2 | Recruiting | HonorHealth Research Institute | 126 | 2026-02-17 | 2026-03-11 |
| Alpha/Beta T and B Cell Depletion With Zoledronic Acid for Solid Tumors Miltenyi CliniMACS Prodigy ® system, Zoledronic acid - Neuroblastoma, Rhabdomyosarcoma | Phase 1 / Phase 2 | Recruiting | University of Florida | 27 | 2026-02-11 | 2026-06-30 |
| 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 |
| Mirdametinib in Patients With Advanced NF1-mutant Melanoma Mirdametinib - Advanced Unresectable Melanoma, Metastatic Melanoma | Phase 2 | Recruiting | Kevin Kim, MD | 10 | 2025-10-13 | 2025-11-19 |
Showing 20 of 285. Sign in to see all of them.
Wilms Tumor (67) · Neurofibromatosis 1 (55) · Neuroblastoma (41) · Sarcoma (41) · Tuberous Sclerosis (35) · Osteosarcoma (28) · Rhabdomyosarcoma (25) · Neurofibroma, Plexiform (24) · Neurofibromatosis 2 (24) · Lymphoma, Non-Hodgkin (23) · Adenomatous Polyposis Coli (22) · Neuroectodermal Tumors, Primitive, Peripheral (22) · Sarcoma, Ewing (22) · Ependymoma (20) · Hepatoblastoma (20)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (285) · Genetic Diseases, Inborn (283) · Neoplasms (269) · Neoplasms by Histologic Type (217) · Neoplasms by Site (164) · Nervous System Diseases (146) · Neoplasms, Nerve Tissue (143) · Neurodegenerative Diseases (122) · Neurocutaneous Syndromes (121) · Heredodegenerative Disorders, Nervous System (120) · Neoplasms, Glandular and Epithelial (117) · Neoplasms, Germ Cell and Embryonal (90) · Neuroectodermal Tumors (89) · Nerve Sheath Neoplasms (85)
Everolimus (23) · Sirolimus (19) · AZD 6244 (17) · Specimen Handling (14) · Biopsy (11) · Magnetic Resonance Spectroscopy (9) · Phantoms, Imaging (8) · Vincristine (8) · X-Rays (8) · Carboplatin (7) · Cyclophosphamide (7) · Etoposide (7) · Radiotherapy (7) · Doxorubicin (6) · Filgrastim (6)
National Cancer Institute (NCI) (48) · University of Alabama at Birmingham (10) · Children's Hospital Medical Center, Cincinnati (9) · Novartis Pharmaceuticals (8) · Children's Oncology Group (6) · Fred Hutchinson Cancer Center (6) · Memorial Sloan Kettering Cancer Center (6) · M.D. Anderson Cancer Center (5) · Assistance Publique - Hôpitaux de Paris (4) · Beijing Tiantan Hospital (4) · NYU Langone Health (4) · UCSF Benioff Children's Hospital Oakland (4) · Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) (3) · Ascend Biopharmaceuticals Ltd (3) · Dana-Farber Cancer Institute (3)
United States (203) · Australia (23) · Canada (23) · Puerto Rico (21) · China (19) · United Kingdom (18) · Spain (16) · France (14) · Netherlands (14) · Germany (11) · Italy (11) · Belgium (9) · Israel (7) · Poland (6) · New Zealand (5)
Brigitte C Widemann, M.D. (6) · Ervin Epstein, MD (5) · AeRang Kim, MD, PhD (4) · Brian H. Kushner, MD (3) · Girish Dhall, MD (3) · Matthias A Karajannis, MD, MS (3) · Chie-Schin Shih, MD (2) · D. Bradley Welling, MD, PhD (2) · David R. Bickers, MD (2) · Dennis J Dlugos, MD (2) · Eduardo Vilar-Sanchez (2) · Fu Zhao (2) · Gregory Siller (2)
PMID 31401903, PMID 28029918, PMID 32187457, PMID 15683544, PMID 17105749, PMID 21996156, PMID 22353803, PMID 26851632, PMID 28230061, PMID 28409229, PMID 31010905, PMID 31582003, PMID 33354735, PMID 33863389, PMID 34541874, PMID 37119510, PMID 18184959, PMID 20348368, PMID 21047224, PMID 21482669, PMID 21525172, PMID 21899713, PMID 21915260, PMID 22032650, PMID 22262746, PMID 22670904, PMID 23099009, PMID 23325902, PMID 23344409, PMID 23437092, PMID 23766359, PMID 23798472, PMID 23919274, PMID 24245549, PMID 24441673, PMID 24500418, PMID 24617713, PMID 24623282, PMID 24753394, PMID 25578927, PMID 25792707, PMID 25956795, PMID 25972901, PMID 26381530, PMID 26976425, PMID 27002448, PMID 27838224, PMID 27956565, PMID 29023494, PMID 29175429
U10CA180886 (19), U10CA098543 (7), P30CA016672 (5), 2018 (3), UG1CA242609 (3), 2016 (2), 2023 (2), P30CA006973 (2), P30CA015083 (2), R01CA109584 (2), UG1CA242596 (2), UG1CA242635 (2), UM1CA081457 (2), UM1CA228823 (2), 2009 (1)