Hereditary neoplastic syndrome
MONDO:0015356The inherited predisposition toward getting a tumor.
Also known as: cancer syndrome, hereditary, cancer syndromes, hereditary, familial neoplastic syndrome, familial tumor syndrome, familial tumour syndrome, hereditary cancer syndrome, hereditary cancer syndromes, hereditary neoplastic syndrome
734 clinical trials for this condition and its sub-types, 60 tagged with Hereditary neoplastic syndrome itself.
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Sub-types of Hereditary neoplastic syndrome
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Glioma susceptibility 0 trials · 133 incl. sub-types
10 sub-types
- Glioma susceptibility 1 133 trials
- Glioma susceptibility 2 0 trials
- Glioma susceptibility 3 0 trials
- Glioma susceptibility 4 0 trials
- Glioma susceptibility 5 0 trials
- Glioma susceptibility 6 0 trials
- Glioma susceptibility 7 0 trials
- Glioma susceptibility 8 0 trials
- Melanoma, cutaneous malignant, susceptibility to, 9 0 trials
- Tumor predisposition syndrome 3 0 trials
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Neurofibromatosis 19 trials · 94 incl. sub-types
5 sub-types
- Neurofibromatosis type 1 73 trials Sub-types →
- NF2-related schwannomatosis 21 trials
- Schwannomatosis 3 trials Sub-types →
- Neurofibromatosis-Noonan syndrome 2 trials Sub-types →
- Neurofibromatosis, type IV, of Riccardi 0 trials
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Hereditary nonpolyposis colon cancer 5 trials · 88 incl. sub-types
5 sub-types
- Lynch syndrome 81 trials · 83 incl. sub-types Sub-types →
- Muir-Torre syndrome 2 trials
- Colorectal cancer, hereditary nonpolyposis, type 6 1 trial
- Colorectal cancer, hereditary nonpolyposis, type 7 0 trials
- Familial colorectal cancer type X 0 trials
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Multiple endocrine neoplasia 7 trials · 62 incl. sub-types
3 sub-types
- Multiple endocrine neoplasia type 1 34 trials
- Multiple endocrine neoplasia type 2 4 trials · 30 incl. sub-types Sub-types →
- Multiple endocrine neoplasia type 4 0 trials
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Intestinal polyposis syndrome 3 trials · 53 incl. sub-types
8 sub-types
- Classic or attenuated familial adenomatous polyposis 0 trials · 35 incl. sub-types Sub-types →
- Peutz-Jeghers syndrome 9 trials
- Hereditary mixed polyposis syndrome 5 trials · 6 incl. sub-types Sub-types →
- Juvenile polyposis syndrome 5 trials Sub-types →
- Bannayan-Riley-Ruvalcaba syndrome 1 trial
- Cronkhite-Canada syndrome 1 trial
- Hyperplastic polyposis syndrome 1 trial Sub-types →
- Juvenile polyposis/hereditary hemorrhagic telangiectasia syndrome 0 trials
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Tuberous sclerosis 41 trials · 44 incl. sub-types
2 sub-types
- Tuberous sclerosis 1 33 trials
- Tuberous sclerosis 2 2 trials
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BRCA2-related cancer predisposition 36 trials · 38 incl. sub-types
3 sub-types
- Breast-ovarian cancer, familial, susceptibility to, 2 2 trials
- Glioma susceptibility 3 0 trials
- Pancreatic cancer, susceptibility to, 2 0 trials
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Hereditary breast ovarian cancer syndrome 35 trials · 38 incl. sub-types
2 sub-types
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Mismatch repair cancer syndrome 1 34 trials
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BRCA1-related cancer predisposition 23 trials · 27 incl. sub-types
2 sub-types
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Li-Fraumeni syndrome 16 trials
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PALB2-related cancer predisposition 14 trials
1 sub-type
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Dyskeratosis congenita 12 trials
16 sub-types
- DKC1-related disorder 0 trials · 3 incl. sub-types Sub-types →
- Revesz syndrome 2 trials
- Dyskeratosis congenita and related telomere biology disorder 1 trial Sub-types →
- Autosomal recessive dyskeratosis congenita 4 0 trials
- Dyskeratosis congenita, autosomal dominant 1 0 trials
- Dyskeratosis congenita, autosomal dominant 2 0 trials
- Dyskeratosis congenita, autosomal dominant 3 0 trials
- Dyskeratosis congenita, autosomal dominant 4 0 trials
- Dyskeratosis congenita, autosomal dominant 6 0 trials
- Dyskeratosis congenita, autosomal recessive 1 0 trials
- Dyskeratosis congenita, autosomal recessive 2 0 trials
- Dyskeratosis congenita, autosomal recessive 3 0 trials
- Dyskeratosis congenita, autosomal recessive 6 0 trials
- Dyskeratosis congenita, autosomal recessive 7 0 trials
- Dyskeratosis congenita, autosomal recessive 8 0 trials
- Dyskeratosis congenita, digenic 0 trials
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Wiskott-Aldrich syndrome 10 trials
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PTEN hamartoma tumor syndrome 6 trials · 8 incl. sub-types
4 sub-types
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CHEK2-related cancer predisposition 7 trials
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Susceptibility to familial cutaneous melanoma 0 trials · 7 incl. sub-types
10 sub-types
- Melanoma, cutaneous malignant, susceptibility to, 2 6 trials
- Melanoma, cutaneous malignant, susceptibility to, 1 1 trial
- Melanoma, cutaneous malignant, susceptibility to, 3 0 trials
- Melanoma, cutaneous malignant, susceptibility to, 4 0 trials
- Melanoma, cutaneous malignant, susceptibility to, 5 0 trials
- Melanoma, cutaneous malignant, susceptibility to, 6 0 trials
- Melanoma, cutaneous malignant, susceptibility to, 7 0 trials
- Melanoma, cutaneous malignant, susceptibility to, 8 0 trials
- Melanoma, cutaneous malignant, susceptibility to, 9 0 trials
- Tumor predisposition syndrome 3 0 trials
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Beckwith-Wiedemann syndrome 6 trials
8 sub-types
- Beckwith-Wiedemann syndrome due to 11p15 microdeletion 0 trials
- Beckwith-Wiedemann syndrome due to 11p15 microduplication 0 trials
- Beckwith-Wiedemann syndrome due to 11p15 translocation/inversion 0 trials
- Beckwith-Wiedemann syndrome due to CDKN1C mutation 0 trials
- Beckwith-Wiedemann syndrome due to NSD1 mutation 0 trials
- Beckwith-Wiedemann syndrome due to imprinting defect of 11p15 0 trials
- Beckwith-Wiedemann syndrome due to paternal uniparental disomy of chromosome 11 0 trials
- Franceschini Vardeu Guala syndrome 0 trials
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RAD51C-related cancer predisposition 6 trials
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9 sub-types
- MAX-related tumor predisposition 0 trials
- TMEM127-related tumor predisposition 0 trials
- Pheochromocytoma/paraganglioma syndrome 1 0 trials
- Pheochromocytoma/paraganglioma syndrome 2 0 trials
- Pheochromocytoma/paraganglioma syndrome 3 0 trials
- Pheochromocytoma/paraganglioma syndrome 4 0 trials
- Pheochromocytoma/paraganglioma syndrome 5 0 trials
- Pheochromocytoma/paraganglioma syndrome 6 0 trials
- Pheochromocytoma/paraganglioma syndrome 7 0 trials
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Nevoid basal cell carcinoma syndrome 5 trials
2 sub-types
- Basal cell nevus syndrome 1 0 trials
- Basal cell nevus syndrome 2 0 trials
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Hereditary thrombocytopenia and hematologic cancer predisposition syndrome 1 trial · 5 incl. sub-types
2 sub-types
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RAD51D-related cancer predisposition 4 trials
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Kostmann syndrome 3 trials
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Li-fraumeni-like syndrome 3 trials
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1 sub-type
- Melanoma-pancreatic cancer syndrome 0 trials
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Hereditary multiple osteochondromas 3 trials
3 sub-types
- Exostoses, multiple, type 1 2 trials
- Exostoses, multiple, type 2 0 trials
- Exostoses, multiple, type III 0 trials
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BARD1-related cancer predisposition 2 trials
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Maffucci syndrome 2 trials
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Blue rubber bleb nevus 2 trials
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Hereditary retinoblastoma 2 trials
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Neuroblastoma, susceptibility to, 3 2 trials
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Brooke-Spiegler syndrome 0 trials · 2 incl. sub-types
2 sub-types
- Familial cylindromatosis 2 trials
- Familial multiple trichoepithelioma 0 trials Sub-types →
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Rothmund-Thomson syndrome 1 trial
4 sub-types
- Rothmund-Thomson syndrome type 1 0 trials
- Rothmund-Thomson syndrome type 2 0 trials
- Rothmund-Thomson syndrome type 3 0 trials
- Rothmund-Thomson syndrome type 4 0 trials
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WAGR syndrome 1 trial
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Cherubism 1 trial
1 sub-type
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Familial rhabdoid tumor 0 trials · 1 incl. sub-types
2 sub-types
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ATM-related cancer predisposition 0 trials
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Carney-Stratakis syndrome 0 trials
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Cobb syndrome 0 trials
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HAVCR2-related cancer predisposition 0 trials
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Kaposi sarcoma, susceptibility to 0 trials
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N syndrome 0 trials
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3 sub-types
- Hyperparathyroidism 1 0 trials
- Hyperparathyroidism 3 0 trials
- Hyperparathyroidism 4 0 trials
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Familial multiple fibrofolliculoma 0 trials
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2 sub-types
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Lung cancer susceptibility 1 0 trials
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Lung cancer susceptibility 3 0 trials
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Lung cancer susceptibility 4 0 trials
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Lung cancer susceptibility 5 0 trials
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3 sub-types
- Mosaic NF2-related schwannomatosis 0 trials
- Mosaic neurofibromatosis type 1 0 trials
- Mosaic schwannomatosis 0 trials
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7 sub-types
- Atelis syndrome 1 0 trials
- Atelis syndrome 2 0 trials
- Mosaic variegated aneuploidy syndrome 1 0 trials
- Mosaic variegated aneuploidy syndrome 2 0 trials
- Mosaic variegated aneuploidy syndrome 3 0 trials
- Mosaic variegated aneuploidy syndrome 4 0 trials
- Mosaic variegated aneuploidy syndrome 7 with inflammation and tumor predisposition 0 trials
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Neuroblastoma, susceptibility to, 1 0 trials
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Neuroblastoma, susceptibility to, 2 0 trials
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Neuroblastoma, susceptibility to, 4 0 trials
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Neuroblastoma, susceptibility to, 5 0 trials
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Neuroblastoma, susceptibility to, 6 0 trials
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Neuroblastoma, susceptibility to, 7 0 trials
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Ovarian cancer, susceptibility to, 1 0 trials
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Susceptibility to uveal melanoma 0 trials
2 sub-types
- Melanoma, uveal, susceptibility to, 1 0 trials
- Melanoma, uveal, susceptibility to, 2 0 trials
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Tumor predisposition syndrome 2 0 trials
Most studied deeper sub-types
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Brain cancer vaccine trial halted: what we know
Vaccine Stopped earlyThis study tested a vaccine called VBI-1901 in 70 people with recurrent glioblastoma, an aggressive brain cancer. The goal was to find a safe dose and see if the vaccine could boost the immune system against the tumor. The trial was terminated early, so results are limited.
Phase 1/2 • Sponsor: VBI Vaccines Inc. • Aim: Vaccine
Last updated Jun 27, 2026 12:30 UTC
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Can a Chemo-Targeted drug combo outsmart Treatment-Resistant prostate cancer?
Disease control Stopped earlyThis phase 2 trial is testing whether giving two chemotherapy drugs (docetaxel and carboplatin) followed by a maintenance drug called rucaparib can help men with metastatic castration-resistant prostate cancer that has specific DNA repair defects. The idea is that the chemo shrin…
Phase 2 • Sponsor: University of Washington • Aim: Disease control
Last updated Aug 28, 2026 00:00 UTC
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Engineered immune cells take on deadly brain tumors
Disease control Stopped earlyThis early-phase study tests a new treatment for glioblastoma, an aggressive brain cancer. The approach uses a patient's own immune cells, modified in the lab to better recognize and attack the tumor, combined with chemotherapy. The main goal is to check the safety of this therap…
Phase 1 • Sponsor: Shenzhen Geno-Immune Medical Institute • Aim: Disease control
Last updated Aug 26, 2026 00:00 UTC
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Immune cells take on nerve tumors: new trial launches
Disease control Stopped earlyThis early-stage trial is testing whether specially engineered immune cells (called CAR-T and CTL cells) and a dendritic cell vaccine can safely treat people with neurofibromatosis or schwannomatosis, conditions that cause nerve tumors. The study will enroll 100 participants aged…
Phase 1/2 • Sponsor: Shenzhen Geno-Immune Medical Institute • Aim: Disease control
Last updated Aug 26, 2026 00:00 UTC
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Promising Dup15q drug trial halted early
Disease control Stopped earlyThis study tested a drug called basmisanil in children aged 2 to 14 with Dup15q syndrome, a rare genetic condition that affects development. The goal was to see if the drug could improve daily living skills like communication and movement. The trial was stopped early after enroll…
Phase 2 • Sponsor: Hoffmann-La Roche • Aim: Disease control
Last updated Aug 20, 2026 00:00 UTC
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Experimental drug REC-2282 targets NF2 brain tumors in early trial
Disease control Stopped earlyThis study tested a drug called REC-2282 in people aged 12 and older with progressive meningiomas (brain tumors) linked to NF2 gene mutations. Participants took the drug or a placebo three times a week in cycles. The goal was to see if the drug could slow tumor growth. The trial …
Phase 2/3 • Sponsor: Recursion Pharmaceuticals Inc. • Aim: Disease control
Last updated Aug 19, 2026 00:00 UTC
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Can a new nucleotide analogue boost cancer treatment?
Disease control Stopped earlyThis trial is testing an experimental drug called NUC-3373, a nucleotide analogue, in combination with standard cancer drugs for people with advanced solid tumors. The study has two parts: one combining NUC-3373 with pembrolizumab, and the other combining it with docetaxel. The g…
Phase 1/2 • Sponsor: NuCana plc • Aim: Disease control
Last updated Jul 25, 2026 00:00 UTC
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Could a gel cut down on skin cancer surgeries? new trial investigates
Disease control Stopped earlyThis trial tests a gel called patidegib applied to the face twice daily for 9 months in people who develop many basal cell carcinomas (a common skin cancer) but do not have Gorlin syndrome. The goal is to see if the gel reduces the number of new skin cancers that would normally r…
Phase 2 • Sponsor: Sol-Gel Technologies, Ltd. • Aim: Disease control
Last updated Jul 23, 2026 00:00 UTC
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Light-Activated cream could slow growth of NF1 skin tumors
Disease control Stopped earlyThis trial tests whether a light-activated cream (Levulan Kerastick) followed by red light therapy can slow the growth of benign skin tumors called neurofibromas in people with neurofibromatosis type 1 (NF1). About 30 participants aged 14 and older with superficial tumors on the …
Phase 2 • Sponsor: Donald Basel • Aim: Disease control
Last updated Jul 12, 2026 00:00 UTC
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Real-World study tracks Selumetinib's impact on NF1 tumors in children
Disease control Stopped earlyThis study observes children aged 3 to 18 with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas and are starting treatment with selumetinib. Researchers will track how well the drug shrinks or stabilizes tumors, how long patients stay on tre…
Sponsor: AstraZeneca • Aim: Disease control
Last updated Jul 10, 2026 00:00 UTC
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Engineered immune cells take aim at deadly brain tumors
Disease control Stopped earlyThis early-phase trial is testing a new treatment called CHM-1101 for people with a type of brain cancer (glioblastoma) that has come back or is getting worse. The treatment uses the patient's own immune cells, which are modified in a lab to recognize and attack tumor cells. The …
Phase 1 • Sponsor: Chimeric Therapeutics • Aim: Disease control
Last updated Jul 01, 2026 00:00 UTC
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Experimental vaccine aims to shrink colorectal tumors before surgery
Disease control Stopped earlyThis trial tested a vaccine called PD1-Vaxx given before surgery to people with a specific type of colorectal cancer (MSI-high). The goal was to see if the vaccine could shrink tumors, measured by how many cancer cells remained after treatment. Only 3 people took part, and the st…
Phase 2 • Sponsor: University of Southampton • Aim: Disease control
Last updated Jun 27, 2026 14:03 UTC
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Could a common blood pressure drug shrink kidney tumors?
Disease control Stopped earlyThis pilot study tested whether propranolol, a beta-blocker, could shrink or stabilize kidney angiomyolipomas in people with tuberous sclerosis. Only 2 participants were enrolled before the trial was terminated, so the results are very limited. The goal was to find a less invasiv…
Phase 2 • Sponsor: University Hospital, Bordeaux • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC
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Gene therapy takes on deadly brain tumor
Disease control Stopped earlyThis study tests a new treatment called Temferon for people with glioblastoma, a fast-growing brain cancer. The therapy uses a patient's own blood stem cells, modified to deliver interferon directly to the tumor. Up to 27 patients with a specific genetic marker (unmethylated MGMT…
Phase 1/2 • Sponsor: Genenta Science • Aim: Disease control
Last updated Jun 27, 2026 13:07 UTC
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Experimental cancer drug study halted early
Disease control Stopped earlyThis early-stage study tested a new drug called PF-07284892, alone or with other medicines, in people with advanced solid tumors that had specific genetic changes. The goal was to find the safest dose and check for side effects. The study was stopped early, so results are limited…
Phase 1 • Sponsor: Pfizer • Aim: Disease control
Last updated Jun 27, 2026 13:02 UTC
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New hope for tough leukemias? trial of vodobatinib begins, then stops
Disease control Stopped earlyThis study tested a new drug called Vodobatinib (K0706) in people with chronic myeloid leukemia or a related leukemia who had not responded to at least three other treatments. The trial was split into parts: first in healthy volunteers to check safety, then in patients to find th…
Phase 1/2 • Sponsor: Sun Pharma Advanced Research Company Limited • Aim: Disease control
Last updated Jun 27, 2026 12:34 UTC
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Could a liver drug tame polyps in rare gut condition?
Disease control Stopped earlyThis study tested whether obeticholic acid, a drug already used for liver disease, can safely reduce the number of polyps in the small bowel and colon in people with familial adenomatous polyposis (FAP). FAP is a rare genetic condition that greatly raises the risk of intestinal c…
Phase 2 • Sponsor: National Cancer Institute (NCI) • Aim: Disease control
Last updated Jun 27, 2026 12:33 UTC
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Cancer drug niraparib tested before prostate surgery – early trial stopped
Disease control Stopped earlyThis phase II trial tested the drug niraparib (Zejula) in 11 men with high-risk prostate cancer that had not spread. All men had specific DNA repair gene mutations. They took niraparib before having their prostate removed surgically. The goal was to see if the drug could eliminat…
Phase 2 • Sponsor: University of California, Davis • Aim: Disease control
Last updated Jun 27, 2026 12:33 UTC
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New cocktail of cancer drugs tested in desperate cases
Disease control Stopped earlyThis early-phase trial tested combinations of the drugs bevacizumab and temsirolimus, sometimes with valproic acid or cetuximab, in 154 people with advanced cancers or certain non-cancerous but progressive diseases. The main goal was to find safe doses and look for any signs that…
Phase 1 • Sponsor: M.D. Anderson Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 12:32 UTC
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Experimental triple therapy for advanced cancers shows early promise but trial halted
Disease control Stopped earlyThis early-phase trial tested the drug plinabulin combined with radiation and immunotherapy in 19 people with advanced cancers (like lung, bladder, or melanoma) that had stopped responding to standard immunotherapy. The goal was to see if the combination was safe and could shrink…
Phase 1/2 • Sponsor: M.D. Anderson Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 12:26 UTC
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Experimental kidney cancer drug trial halted early
Disease control Stopped earlyThis early-stage trial tested a new drug called DFF332, which targets a protein (HIF2α) that helps some cancers grow. The study included 40 adults with advanced clear cell renal cell carcinoma or other cancers with specific gene changes. DFF332 was tested alone and in combination…
Phase 1 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 09:09 UTC
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Experimental cancer drug tested in rare tumors, but trial stopped early
Disease control Stopped earlyThis phase 2 trial tested the drug guadecitabine (SGI-110) in people aged 12 and older with rare cancers like wild-type GIST, pheochromocytoma, paraganglioma, and HLRCC-related kidney cancer that had not responded to other treatments. Participants received daily injections for 5 …
Phase 2 • Sponsor: National Cancer Institute (NCI) • Aim: Disease control
Last updated Jun 27, 2026 09:05 UTC
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Old arthritis drug tested against rare tumor syndrome – but trial stalls
Disease control Stopped earlyThis study tested leflunomide, a drug used for rheumatoid arthritis, in 3 adults with MEN1 syndrome—a genetic condition that raises the risk of multiple tumors. The goal was to see if 6 months of daily leflunomide could shrink or stabilize tumors. However, the trial was terminate…
Sponsor: University Hospital, Basel, Switzerland • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC
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Colon cancer combo trial ends early – only one patient enrolled
Disease control Stopped earlyThis study tested a combination of two drugs (regorafenib and pembrolizumab) for people with a specific type of advanced colorectal cancer called MSI-H. The goal was to see if the combination worked better than pembrolizumab alone. The trial was stopped early after enrolling only…
Phase 2 • Sponsor: Anwaar Saeed • Aim: Disease control
Last updated Jun 27, 2026 08:13 UTC
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Experimental virus gets a second shot at brain tumors
Disease control Stopped earlyThis study tested the safety of giving a second dose of an experimental virus (C134) directly into the brain tumor of patients whose malignant glioma had returned. Only one patient was enrolled before the study was stopped. The goal was to see if repeat dosing was safe and tolera…
Phase 1 • Sponsor: University of Alabama at Birmingham • Aim: Disease control
Last updated Jun 27, 2026 08:13 UTC
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Experimental cancer drug TAK-500 tested in 61 patients – study halted early
Disease control Stopped earlyThis early-phase trial tested an experimental drug called TAK-500, alone or with the immunotherapy pembrolizumab, in 61 adults with advanced solid tumors like pancreatic, liver, and lung cancer. The main goals were to check safety and see if the drug could shrink tumors. The stud…
Phase 1/2 • Sponsor: Takeda • Aim: Disease control
Last updated Jun 27, 2026 08:13 UTC
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Experimental radioactive therapy targets Hard-to-Treat cancers
Disease control Stopped earlyThis early-stage study tested a radioactive drug called 177Lu-FF58 in 24 people with advanced pancreatic, gastroesophageal, or brain cancer. The drug seeks out and attaches to specific proteins on cancer cells to deliver radiation directly. The main goals were to check safety, fi…
Phase 1 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 08:06 UTC
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Experimental drug AR-42 tested on brain tumors – early results uncertain
Disease control Stopped earlyThis early-phase trial tested the drug AR-42 in 7 adults with vestibular schwannoma or meningioma brain tumors who were already scheduled for surgery. Participants took AR-42 pills for about 3 weeks before their operation. The main goal was to see if the drug could lower a protei…
Early phase 1 • Sponsor: Massachusetts Eye and Ear Infirmary • Aim: Disease control
Last updated Jun 27, 2026 08:01 UTC
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Promising cream for rare skin condition falls short in early trial
Disease control Stopped earlyThis study tested a sirolimus cream (0.2% and 0.4%) applied daily for 12 weeks to treat facial angiofibromas in children aged 2-21 with tuberous sclerosis complex. The goal was to see if the cream could safely reduce the size and redness of these skin growths. The trial was termi…
Phase 2 • Sponsor: Aucta Pharmaceuticals, Inc • Aim: Disease control
Last updated Jun 27, 2026 08:00 UTC
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Experimental cancer drug INCB062079 tested in early trial
Disease control Stopped earlyThis early-phase trial tested a new drug called INCB062079 in 25 people with advanced liver cancer and other solid tumors. The main goal was to check safety and find the best dose. The study was terminated early, so results are limited.
Phase 1 • Sponsor: Incyte Corporation • Aim: Disease control
Last updated Jun 27, 2026 07:55 UTC
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Experimental seizure drug tested in rare genetic disorder
Disease control Stopped earlyThis phase 3 study tested the drug ganaxolone as an add-on treatment for seizures in children and adults with tuberous sclerosis complex (TSC). The trial was open-label, meaning everyone received the drug, and it included 117 people who had previously been in related studies. The…
Phase 3 • Sponsor: Marinus Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 07:52 UTC
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New drug combo for kids with resistant leukemia shows early promise but study halted
Disease control Stopped earlyThis study tested a targeted cancer drug called ponatinib combined with standard chemotherapy in children and young adults up to age 21 with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) that had come back or stopped responding to other treatments. The g…
Phase 1/2 • Sponsor: Takeda • Aim: Disease control
Last updated Jun 27, 2026 07:52 UTC
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Brain cancer drug trial halted after just two patients
Disease control Stopped earlyThis study tested a new drug called VAL-083 in people with glioblastoma, an aggressive brain cancer that had returned after standard treatments. The trial aimed to see if VAL-083 could help patients live longer. However, the study was stopped early after only 2 people enrolled, s…
Phase 3 • Sponsor: DelMar Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 26, 2026 13:46 UTC
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New imaging agent aims to spot Hard-to-Find cancers
Diagnosis Stopped earlyThis early-phase trial tested a radioactive imaging agent called 68Ga-FF58 in 14 adults with certain advanced solid tumors (glioblastoma, brain metastases from breast cancer, gastroesophageal cancer, or pancreatic cancer). The goal was to see if the agent could help detect these …
Early phase 1 • Sponsor: Novartis Pharmaceuticals • Aim: Diagnosis
Last updated Jun 27, 2026 07:54 UTC
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CBD study for baby seizures halted early
Symptom relief Stopped earlyThis study tested a cannabidiol (CBD) oral solution in children under 2 years old with tuberous sclerosis complex, Dravet syndrome, or Lennox-Gastaut syndrome who had uncontrolled seizures. The goal was to see if CBD is safe and can reduce seizures. However, the study was termina…
Phase 3 • Sponsor: Jazz Pharmaceuticals • Aim: Symptom relief
Last updated Jun 27, 2026 07:56 UTC
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Study checks if stopping a drug made polyps grow faster
Knowledge-focused Stopped earlyThis study reviewed medical records of 42 people with familial adenomatous polyposis (FAP) who had previously taken the drug erlotinib. Researchers wanted to see how quickly the disease progressed after the drug was stopped. The goal was to better understand what happens when tre…
Sponsor: National Cancer Institute (NCI) • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:05 UTC
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Spinal tap could replace brain surgery for young tumor patients
Knowledge-focused Stopped earlyThis study looked at whether a liquid biopsy—testing the fluid around the brain and spinal cord—can find tumor DNA in children and young adults with brain tumors. The goal was to see if this safer, non-surgical test could replace risky tissue biopsies for diagnosis and monitoring…
Sponsor: Pediatric Brain Tumor Consortium • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:23 UTC
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New test may expand olaparib benefits to more ovarian cancer patients
Knowledge-focused Stopped earlyThis study tested a new lab method (RAD51 assay) to see if it could identify ovarian cancer patients whose tumors are vulnerable to the drug olaparib, even if they don't have BRCA gene mutations. The goal was to find a larger group of patients who might benefit from this treatmen…
Phase 2 • Sponsor: Leiden University Medical Center • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:06 UTC
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Could a common antifungal pill fight brain tumors?
Knowledge-focused Stopped earlyThis early-phase trial tested whether posaconazole, a drug used for fungal infections, can reach brain tumors in high enough amounts to stop cancer cells from dividing. Seven adults with glioblastoma took the drug before surgery, and researchers measured drug levels in their spin…
Early phase 1 • Sponsor: Milton S. Hershey Medical Center • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:13 UTC
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Antifungal drug tested against brain cancer — but trial stops early
Knowledge-focused Stopped earlyThis early-phase trial tested whether ketoconazole, a drug normally used for fungal infections, can enter brain tumors in high enough amounts to stop tumor cells from dividing. Only 1 person was enrolled before the study was terminated. The goal was to measure drug levels in the …
Early phase 1 • Sponsor: Milton S. Hershey Medical Center • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:08 UTC
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Drug interaction study for radiprodil halted early
Knowledge-focused Stopped earlyThis early-stage study looked at how two common drugs (carbamazepine and itraconazole) change the levels of radiprodil in the blood of 30 healthy adults. The goal was to understand safety and dosing for future use in neurological conditions like tuberous sclerosis. However, the s…
Phase 1 • Sponsor: GRIN Therapeutics, Inc. • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:59 UTC