Blood Disorders & Hematology Clinical Trials Update: Week 40, 2026

Published October 2, 2026 — 5 trials covered

By Victor Lafforgue, Founder of TrialsAlert. Blog posts are AI-drafted from ClinicalTrials.gov source data and reviewed by the editorial team. See our editorial policy for details.

Revumenib in Combination With Azacitidine + Venetoclax in Patients NPM1-mutated or KMT2A-rearranged AML

This Phase 3 trial is testing a new drug called revumenib combined with standard treatments for adults with newly diagnosed acute myeloid leukemia (AML) who cannot receive intensive chemotherapy. By targeting specific genetic changes in leukemia cells, this treatment may improve survival and offer a better option for people with this type of AML. The study is recruiting about 448 patients worldwide and includes long-term follow-up to better understand outcomes.

CTX001 in Subjects With Severe Sickle Cell Disease

This Phase 2 trial is studying a gene therapy called CTX001 for people with severe sickle cell disease. It uses a patient’s own stem cells, modified to reduce painful crises and improve overall blood health. After completing enrollment in multiple countries, the trial has shared results that could help shape future treatments for sickle cell disease beyond current options.

Chimeric Antigen Receptor Treatment Targeting CD70 (SEVENTY)

This Phase 1 trial tests a new CAR T-cell therapy targeting CD70 for patients with certain types of leukemia that have come back or not responded to previous treatments. This approach uses specially engineered immune cells to attack leukemia more effectively, potentially offering a new treatment option. The study is actively recruiting in the United States and focuses on finding a safe dose and understanding possible side effects.

SC-PEG Asparaginase vs. Oncaspar in Pediatric Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma

This Phase 2 study compares a new intravenous form of asparaginase called SC-PEG to the standard drug Oncaspar in children and adolescents with acute lymphoblastic leukemia or lymphoblastic lymphoma. The goal is to see if SC-PEG, given every three weeks, is as safe and effective as Oncaspar, given every two weeks, potentially providing a more convenient treatment schedule. The trial also looks into ways to prevent infections and support bone health during treatment.

A Study to Assess the Anti-Tumor Activity and Safety of Odronextamab in Adult Patients With B-cell Non-Hodgkin Lymphoma

This Phase 2 trial is evaluating odronextamab, an antibody treatment, in adults with B-cell non-Hodgkin lymphoma who have already received other cancer therapies. It aims to find a new option that can better destroy cancer cells with manageable side effects. The study is active but not recruiting, with over 500 patients enrolled across multiple countries.

This week’s research highlights show ongoing efforts to develop treatments that are more targeted, less intensive, and tailored to individual patient needs. From gene therapy for sickle cell disease to new drug combinations for leukemia, these trials offer hope for improved care in blood disorders. TrialsAlert subscribers received the full impact-classified briefing and doctor-ready report this week. Get weekly briefings at trialsalert.com.