A Phase 1/1b Study Evaluating the Safety, Pharmacokinetics, and Preliminary Efficacy of Mutant Calreticulin Targeted Antibodies, Administered Alone or in Combination with Ruxolitinib, in Participants with CALR-mutant Myeloproliferative Neoplasms (NCT07849231)

This study is designed to characterise the safety, tolerability, and preliminary efficacy of mutant calreticulin targeted antibodies in CALR-mutant myeloproliferative neoplasms.

Trial phase

IA/IB

Trial participation type

This trial has been designed for the Clinical Research of a Drug.

Principal investigator

Prof Andrew Perkins

Key inclusion data

Adults (≥18 years) with documented CALR exon 9-mutated ET, PMF (including pre-fibrotic MF), or post-ET MF. ET cohort: High-risk ET with platelet count >450 × 10⁹/L and resistant, refractory, intolerant, or unsuitable for ≥1 prior cytoreductive therapy. MF cohort: Intermediate-1 symptomatic or intermediate-2/high-risk MF with measurable disease burden and resistant/refractory/intolerant to JAK inhibitor therapy (or suboptimal response to ruxolitinib in Part 3). Key exclusions: Active malignancy or uncontrolled infection, recent major thrombosis/bleeding or significant cardiovascular disease, recent stem cell transplant/major surgery, and clinically significant organ dysfunction or laboratory abnormalities.