Clinical Trials

International phase 2 randomised controlled trial comparing venetoclax-low dose cytarabine with standard intensive chemotherapy (daunorubicin, cytarabine and gemtuzumab ozogamicin) in newly diagnosed AML. Adaptive design allowing staged expansion to younger patients. CRUK/19/013.

International phase 3 randomised controlled trial comparing intensive treatment strategies for newly diagnosed FLT3-mutated AML. ISRCTN34016918.

International phase 3 randomised trial of newly diagnosed patients ineligible for intensive chemotherapy with IDH1m AML using ivosidenib plus azacitidine ± venetoclax.

International phase 3 randomised trial of newly diagnosed patients ineligible for intensive chemotherapy with either NPM1m or KMT2Ar AML 1:1 to venetoclax plus-azacitidine ± revumenib (menin inhibitor).

Multi-phase study of ASTX030 (oral azacitidine + cedazuridine) in combination with venetoclax in newly diagnosed AML patients ineligible for intensive chemotherapy. NCT04256317.

Phase 3 randomised, double-blind, placebo-controlled studies assessing ziftomenib in combination with either Venetoclax+Azacitidine or intensive (7+3) therapy in patients with untreated NPM1 mutated or KMT2A-rearranged AML

Phase 3 randomised, double-blind, placebo-controlled study of bleximenib, venetoclax and azacitidine for untreated NPM1 mutated or KMT2A-rearranged AML ineligible for intensive chemotherapy

Phase 3, Double-Blind, Randomised, Placebo-Controlled Trial Of Quizartinib or Placebo Plus Chemotherapy in Newly Diagnosed Patients With FLT3-ITD Negative AML

Phase 1/2 study of the menin inhibitor bleximenib in relapsed/refractory (R/R) NPM1 mutated or KMT2A rearranged AML

Phase 1/2 study of the menin inhibitor enzomenib in relapsed/refractory (R/R) AML, ALL, MDS, and MM with KMT2A, NPM1, or other qualifying alterations

Phase 1 study to investigate the safety and efficacy of APL-4098 alone, and in combination with azacitidine or azacitidine and venetoclax in AML and MDS/AML

Phase 1/2 study of a CD33xVδ2 T-Cell Engager for Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Neoplasms

Phase 1/2 study of AZD3632 monotherapy or in combination with anticancer agents in participants with advanced haematologic malignancies with KMT2Ar, NPM1m, or other genotypes associated with HOX overexpression

BioDrive seeks to identify the most effective way to prevent and detect fungal infection in patients with acute leukaemia (acute myeloid leukaemia, acute lymphoblastic leukaemia, high-risk myelodysplastic syndromes and transformed myeloproliferative neoplasms); assessing if monitoring the risk of fungal infection by regular blood tests is safe and reduces the need to use antifungal drugs.

PROPEL is a multicentre randomised controlled trial comparing best practice usual care (BPUC) with a personalised prehabilitation care package (PPCP) incorporating a 12-month internal pilot, parallel process evaluation and economic evaluation. NIHR134257. Chief Investigators: Prof Janet Dunn, Prof Simon Stanworth.

MO-TRANS is a randomised multi-centre phase 3 trial of mocravimod maintenance treatment post allogeneic stem cell transplant

Phase 1/2 study assessing granulocyte augmentation to single-cord blood transplantation for high risk AML

Phase 1/2a adaptive open-label study of CCS1477 (inobrodib), a first-in-class p300/CBP bromodomain inhibitor, as monotherapy and in combination with azacitidine ± venetoclax in relapsed/refractory AML and higher-risk MDS. NCT04068597. CRUK 17062.

Phase 1/2a modular study of EP0042, a dual FLT3 and Aurora kinase inhibitor, as monotherapy and in combination with venetoclax ± azacitidine in relapsed/refractory FLT3-wildtype AML, CMML, and MDS. NCT04581512.

Phase 1/2 open-label dose escalation and expansion study of revumenib (SNDX-5613), a menin inhibitor, in relapsed/refractory acute leukemias harbouring KMT2A rearrangements, NPM1 mutations, or NUP98 rearrangements. 447 patients across 57+ sites. Led to FDA approval of revumenib. NCT04065399.

Phase 1 study of ziftomenib (menin inhibitor, FDA-approved for R/R NPM1m AML) in combination with venetoclax/azacitidine, venetoclax alone, or 7+3 chemotherapy ± quizartinib in newly diagnosed and R/R AML with NPM1 mutations or KMT2A rearrangements. 420 patients. NCT05735184.

Phase 3, open-label, randomised, non-comparative study of ivosidenib monotherapy versus azacitidine monotherapy in HMA-naive adults with IDH1-mutated MDS. Prior Phase 2 showed 72% overall response rate for ivosidenib. 48 patients. NCT06465953.

Phase 3, randomised, double-blind, placebo-controlled study of elritercept (KER-050/TAK-226) for transfusion-dependent anaemia in very low-, low-, or intermediate-risk MDS. 225 patients globally. NCT06499285.

Phase 3, randomised, open-label, multicentre international trial comparing ruxolitinib with either hydroxycarbamide or interferon-alpha as first-line therapy for high-risk polycythaemia vera (PV). 586 patients. NCT04116502.

Phase 3 study of rusfertide (PTG-300), a hepcidin mimetic, versus placebo as add-on to existing PV therapy for haematocrit control in phlebotomy-dependent polycythaemia vera. 293 patients. NCT05210790. Active, not currently recruiting.

Phase 3, randomised, open-label study comparing givinostat (HDAC inhibitor) versus hydroxyurea in JAK2V617F-positive high-risk polycythaemia vera patients. 220 patients over 48-week core phase. NCT06093672.

Phase 2a, randomised, open-label study of sapablursen (TMPRSS6 antisense inhibitor) for reducing phlebotomy frequency and improving quality of life in phlebotomy-dependent polycythaemia vera. 50 patients. NCT05143957. Active, not currently recruiting.

Phase 3, randomised, open-label study of bomedemstat (LSD1 inhibitor) versus best available therapy in essential thrombocythaemia with inadequate response to or intolerance of hydroxyurea. 340 patients. NCT06079879.

Phase 1b, open-label study of STP938 (CTPS1 inhibitor) in adults with high-risk essential thrombocythaemia who cannot tolerate or have failed hydroxycarbamide. 20 patients. NCT06786234.

Phase 3, randomised, controlled study of pacritinib versus physician's choice in myelofibrosis with severe thrombocytopenia (platelets <50,000/µL). 399 patients. NCT03165734.

Phase 3, randomised, open-label study of imetelstat (telomerase inhibitor) versus best available therapy in intermediate-2 or high-risk myelofibrosis relapsed/refractory to JAK inhibitor. 327 patients. NCT04576156. Active, not currently recruiting.

Phase 3, randomised, double-blind, add-on study of navtemadlin (MDM2 inhibitor) plus ruxolitinib versus placebo plus ruxolitinib in JAK inhibitor-naive myelofibrosis patients with suboptimal response to ruxolitinib. Requires TP53 wild-type. 600 patients. NCT06479135.

Phase 1, first-in-human study of JNJ-88549968, a T-cell redirecting bispecific antibody targeting CD3 and mutant calreticulin (CALR) on MPN cancer cells, in CALR-mutated essential thrombocythaemia and myelofibrosis. 220 patients. NCT06150157.

Phase 1/2 study of TERN-701, a highly selective allosteric BCR-ABL1 inhibitor, in previously treated chronic-phase CML. Includes mutation cohort targeting T315I. 75% MMR rate reported at RP2D. 180 patients. NCT06163430.

Phase 2, randomised, double-blind, placebo-controlled dose-finding study of pacritinib (IRAK1/JAK2 inhibitor) in VEXAS syndrome. 24-week double-blind period followed by 48-week open-label period. Requires UBA1 mutation. 78 patients. NCT06782373.

Phase 3, multicentre, randomised, open-label, pragmatic trial comparing high-intensity reinduction chemotherapy with low-intensity therapies (VenAza, Ven+LDAC, gilteritinib, etc.) in 1st or 2nd relapse AML. EU-funded. 339 patients. NCT06713837.

Phase 2, open-label study of fedratinib (JAK2 inhibitor) combined with ropeginterferon alfa-2b in treatment-naive, JAK2 V617F-mutated myelofibrosis. Met its primary tolerability endpoint. 30 patients across 13 UK TAP centres. ISRCTN88102629.

Phase 2, randomised UK trial of oral decitabine/cedazuridine (ASTX727) versus hydroxycarbamide/best supportive care in CMML and MDS/MPN overlap syndromes. Showed superior ORR (53% vs 30%) and PFS (23.5 vs 13.9 months). 13 UK hospitals.