Table 1 NUT midline carcinoma clinical trials available at Clinicaltrials.gov
From: The first report of molecular characterized BRD4-NUT carcinoma in Brazil: a case report
Trial | Type of study/ population | Regimen | Mechanism of action | Status |
|---|---|---|---|---|
NCT01587703 | Phase I/II – Diagnosis of NMC determined by IHC and/or detection of NUT gene translocation by FISH, treatment naïve or with prior therapy | GSK525762 | BET protein inhibitor | Active, not recruiting |
NCT03702036 | Compassionate use – Diagnosis of NMC determined by IHC and/or detection of NUT gene translocation by FISH, with no other satisfactory alternative treatment | GSK525762 (molibresib) | Inhibitor of the binding of BET proteins to acetylated histones | Available |
NCT02307240 | Phase I – Diagnosis of an advanced solid tumor such as breast cancer or NMC, that has progressed despite standard therapy, or for which no standard therapy exists | CUDC-907 | HDAC and PI3K Inhibitor | Active, not recruiting |
NCT02698176 | Phase IB – Diagnosis of one of the following advanced solid tumors for which standard therapy either does not exist or has proven ineffective, intolerable, or inacceptable for the participant: NMC;TNBC; NSCLC; or CRPC | MK-8628 | BET protein inhibitor | Terminated due to limited efficacy |
NCT02516553 | Phase I – Diagnosis of advanced unresectable and/or metastatic solid tumor, refractory to conventional treatment or for whom no therapy of proven efficacy exists, or who are not amenable to standard therapies. Part Ib includes patients with SCLC, CRPC, CRC or NMC | BI 894999 | BET protein inhibitor | Recruiting |
NCT02259114 | Phase I – Advanced or metastatic: TNBC, NSCLC, CRPC, pancreatic ductal adenocarcinoma, NMC, for which standard therapy either does not exist or has proven ineffective, intolerable or inacceptable for the patient | OTX015/MK-8628 (birabresib) | BET protein inhibitor | 3/10 patients with NMC with PR with duration of 1.4 to 8.4 months |
NCT02711137 | Phase 1/2 – Histologically or cytologically confirmed diagnosis of relapsed or refractory advanced or metastatic malignancies (solid or hematologic) | INCB057643 | BET protein inhibitor | Terminated due to safety issues |
NCT02431260 | Phase 1/2 – Any advanced solid tumor or lymphoma; acute leukemia, myelodysplastic syndrome, myelodysplastic /myeloproliferative neoplasms, myelofibrosis, and multiple myeloma | INCB054329 | BET protein inhibitor | Terminated by the sponsor due to pharmacokinetics variability [20] |
NCT02369029 | Phase 1 – Patients with advanced tumors refractory to any standard treatment, with no standard therapy available or in whom standard therapy is not a therapeutic option | BAY1238097 | BET protein inhibitor | Terminated because of dose-limiting toxicities at a dose below targeted drug exposure [21] |