112 completed, 27 recruiting, 18 active not recruiting, 12 terminated. 189 Phase 1, 58 Phase 2.
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 |
|---|---|---|---|---|---|---|
| A Safety Study of ST316 in Participants With Familial Adenomatous Polyposis (FAP). ST316 IV - Familial Adenomatous Polyposis (FAP) | Phase 1 | Not yet recruiting | Sapience Therapeutics | 40 | 2027-01-04 | 2026-08-12 |
| 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 |
| 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 |
| 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 |
| Study of Cabozantinib With Selumetinib for Plexiform Neurofibromas Cabozantinib Oral Tablet, Selumetinib Oral Capsule - Neurofibromatosis 1, Plexiform Neurofibroma | Phase 1 | Recruiting | Girish Dhall, MD | 30 | 2026-06-09 | 2026-10-02 |
| 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 |
| Phase I Study of Becotatug Vedotin for Safety and Efficacy in EGFR-Positive Pediatric Relapsed/Refractory or Metastatic Solid Tumors Becotatug Vedotin - Head and Neck Squamous Cell Carcinoma (HNSCC) - Recurrent/Metastatic (R/M), Nasopharyngeal Carcinoma | Phase 1 | Recruiting | Sun Yat-sen University | 51 | 2026-05-30 | 2026-05-13 |
| 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 |
| Feasibility and Tolerability of IMLYGIC for the Treatment of Cutaneous Neurofibromas in Adults With NF1 Imlygic - NF1, Neurofibromatosis | Phase 1 | Recruiting | Johns Hopkins University | 10 | 2026-02-01 | 2026-05-08 |
| Selective Antigen Specific T Cells and CAR T Cells in Subjects With Relapsed/Refractory Embryonal Tumors (SABRE) Selective Antigen Specific dTβRII-expressing T cells combined with B7-H3 CAR T cells - Rhabdomyosarcoma, Ewing Sarcoma | Phase 1 | Recruiting | Children's National Research Institute | 18 | 2026-01-27 | 2026-08-13 |
| Immunotherapy for Solid Tumor Malignancies in Pediatrics Using Interleukin-15 and -21 Armored Glypican-3-specific Chimeric Antigen Receptor T Cells SC-CAR.GPC3xIL15.21 CAR T cells - Solid Tumor (Excluding CNS), Liver Cell Carcinoma | Phase 1 | Recruiting | Seattle Children's Hospital | 21 | 2025-12-22 | 2026-02-17 |
| 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 |
| Testing for Safety and Colorectal Cancer Preventive Effects of ONC201 Biopsy Procedure, Biospecimen Collection, Colonoscopy - Colorectal Adenomatous Polyp, Colorectal Carcinoma | Phase 1 | Recruiting | National Cancer Institute (NCI) | 36 | 2025-10-06 | 2026-10-15 |
| First in Human Pilot Study to Assess the Safety and Efficacy of Dendritic Cells Loaded With Frameshift Derived Neopeptides for the Prevention of Cancer in of Lynch Syndrome Carriers Autologous Tolerogenic Dendritic cells - Lynch Syndrome | Phase 1 | Recruiting | Fundacion Clinic per a la Recerca Biomédica | 20 | 2025-09-04 | 2026-04-13 |
| Follow-up Study to Evaluate the Safety and Efficacy of FCN-159 in Pediatric Participants With Neurofibromatosis Type 1 Luvometinib Tablets - Neurofibromatosis 1, Plexiform Neurofibroma | Phase 1 / Phase 2 | Not yet recruiting | Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. | 65 | 2025-06-12 | 2025-06-17 |
| Immunotherapy For Adults With GPC3-Positive Solid Tumors Using IL-15 and IL-21 Armored GPC3-CAR T Cells 21.15.GPC3-CAR T cells - Hepatoblastoma, Hepatocellular Carcinoma | Phase 1 | Recruiting | Baylor College of Medicine | 21 | 2025-06-10 | 2025-11-03 |
| PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas PAS-004 Tablets, PAS-004 Capsule - NF1 Mutation, Neurofibroma Plexiform | Phase 1 | Recruiting | Pasithea Therapeutics Corp. | 56 | 2025-05-30 | 2026-10-28 |
| 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 |
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Wilms Tumor (72) · Neuroblastoma (50) · Sarcoma (37) · Rhabdomyosarcoma (31) · Neurofibromatosis 1 (30) · Osteosarcoma (30) · Sarcoma, Ewing (28) · Neoplasms (25) · Multiple Endocrine Neoplasia Type 1 (24) · Medulloblastoma (21) · Rhabdoid Tumor (19) · Astrocytoma (18) · Neuroectodermal Tumors, Primitive, Peripheral (17) · Ovarian Neoplasms (16) · Testicular Neoplasms (16)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (189) · Genetic Diseases, Inborn (187) · Neoplasms (164) · Neoplasms by Histologic Type (141) · Neoplasms by Site (130) · Neoplasms, Nerve Tissue (105) · Neoplasms, Glandular and Epithelial (90) · Nervous System Diseases (90) · Urogenital Diseases (78) · Urogenital Neoplasms (78) · Female Urogenital Diseases (77) · Female Urogenital Diseases and Pregnancy Complications (77) · Male Urogenital Diseases (77) · Urologic Diseases (74)
AZD 6244 (9) · Sirolimus (9) · Carboplatin (7) · Everolimus (7) · Filgrastim (7) · Specimen Handling (7) · Cyclophosphamide (6) · Temozolomide (6) · Granulocyte Colony-Stimulating Factor (5) · Biopsy (4) · Busulfan (4) · Deoxycholic Acid (4) · Etoposide (4) · Ifosfamide (4) · Melphalan (4)
National Cancer Institute (NCI) (26) · Bayer (13) · AstraZeneca (5) · Children's Hospital Medical Center, Cincinnati (5) · Massachusetts General Hospital (5) · Baylor College of Medicine (4) · Children's Oncology Group (4) · GlaxoSmithKline (4) · National Institutes of Health Clinical Center (CC) (4) · AeRang Kim (3) · City of Hope Medical Center (3) · Emory University (3) · GRIN Therapeutics, Inc. (3) · M.D. Anderson Cancer Center (3) · Masonic Cancer Center, University of Minnesota (3)
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Brigitte C Widemann, M.D. (6) · AeRang Kim, MD, PhD (3) · David Steffin, MD (3) · Richard R Anderson, MD (3) · Eduardo Vilar-Sanchez (2) · Elizabeth Fox, MD (2) · Holly Meany, MD (2) · Jason R Fangusaro (2) · Kenneth G. Lucas, MD (2)
PMID 31401903, PMID 15814645, PMID 16234526, PMID 16914576, PMID 18281544, PMID 18391430, PMID 19075272, PMID 19430790, PMID 20044751, PMID 20234350, PMID 20460632, PMID 21047224, PMID 21482669, PMID 21764821, PMID 22262746, PMID 22934754, PMID 23325902, PMID 23766359, PMID 23798472, PMID 24617713, PMID 25578927, PMID 25972901, PMID 26154941, PMID 26381530, PMID 26486517, PMID 27282116, PMID 27869650, PMID 28029918, PMID 29023494, PMID 29187393, PMID 30277655, PMID 30424962, PMID 31151904, PMID 31838007, PMID 31876107, PMID 32187457, PMID 32641470, PMID 33305087, PMID 33470897, PMID 33606922, PMID 33641042, PMID 33939067, PMID 34118197, PMID 34118198, PMID 34157616, PMID 34785698, PMID 35170228, PMID 35395680, PMID 35410754, PMID 35467749
2023 (3), UM1CA081457 (3), P30CA016672 (2), P30CA033572 (2), P30CA047904 (2), U01CA097452 (2), U01CA099168 (2), 2016 (1), 2018 (1), 2019 (1), 2025 (1), K08MH094441 (1), N01CM00038 (1), N01CN00034 (1), P30CA015704 (1)