NCT03326310

Selumetinib and Azacitidine in High Risk Chronic Blood Cancers

Recruiting now Phase 1 Last updated 29 December 2025

What this trial tests

Phase 1 trial testing Azacitidine in Chronic Myeloid Leukemia in 18 participants. Currently enrolling.

Timeline

4 September 2018

Primary endpoint
4 September 2027

4 September 2027

Quick facts

Lead sponsor	University of Chicago
Phase	Phase 1
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	18
Start date	4 September 2018
Primary completion	4 September 2027
Estimated completion	4 September 2027
Sites	1 location across United States

Drugs / interventions tested

• Azacitidine (azacitidine) — full drug profile →
• Selumetinib — full drug profile →

Conditions studied

• Chronic Myeloid Leukemia — all drugs for Chronic Myeloid Leukemia →
• Myelofibroses — all drugs for Myelofibroses →

Sponsor

University of Chicago

Who can join

18 and older, any sex, with Chronic Myeloid Leukemia or Myelofibroses. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This is a phase I, open-label, dose-escalation study to determine the MTD of selumetinib when combined with the standard dose of azacitidine. Treatment will begin within 28 days of screening procedures. Treatment will continue indefinitely, provided that the patient continues to derive benefit. A patient will be taken off study for reasons described in detail in section 3.12 including disease progression, unacceptable toxicity, inter-current illness, withdrawal of consent, or at the discretion of the investigator. Patients will be followed for 12 weeks after the last dose of study drug, until any study treatment related toxicities have stabilized, or until death. The total duration of the study is expected to be approximately 24 months.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Resistance to Tyrosine Kinase Inhibitors in Chronic Myeloid Leukemia-From Molecular Mechanisms to Clinical Relevance.
   Alves R, Gonçalves AC, Rutella S, Almeida AM, et al · · 2021 · cited 112× · PMID 34638304 · DOI 10.3390/cancers13194820
2. Progression of Myeloproliferative Neoplasms (MPN): Diagnostic and Therapeutic Perspectives.
   Baumeister J, Chatain N, Sofias AM, Lammers T, et al · · 2021 · cited 50× · PMID 34944059 · DOI 10.3390/cells10123551
3. Phase II Trial of MEK Inhibitor Binimetinib (MEK162) in RAS-mutant Acute Myeloid Leukemia.
   Maiti A, Naqvi K, Kadia TM, Borthakur G, et al · · 2019 · cited 41× · PMID 30635233 · DOI 10.1016/j.clml.2018.12.009
4. RAS mutations in myeloid malignancies: revisiting old questions with novel insights and therapeutic perspectives.
   Alawieh D, Cysique-Foinlan L, Willekens C, Renneville A. · · 2024 · cited 37× · PMID 38658558 · DOI 10.1038/s41408-024-01054-2
5. Inhibition of ERK1/2 signaling prevents bone marrow fibrosis by reducing osteopontin plasma levels in a myelofibrosis mouse model.
   Bianchi E, Rontauroli S, Tavernari L, Mirabile M, et al · · 2023 · cited 18× · PMID 36928007 · DOI 10.1038/s41375-023-01867-3
6. Genomics of MPN progression.
   Patel AA, Odenike O. · · 2020 · cited 14× · PMID 33275731 · DOI 10.1182/hematology.2020000129
7. Mcl-1 and Bcl-xL levels predict responsiveness to dual MEK/Bcl-2 inhibition in B-cell malignancies.
   Melvold K, Giliberto M, Karlsen L, Ayuda-Durán P, et al · · 2022 · cited 12× · PMID 34861096 · DOI 10.1002/1878-0261.13153
8. Historical expectations with DNA methyltransferase inhibitor monotherapy in MDS: when is combination therapy truly "promising"?
   Brunner AM, Fell G, Steensma DP. · · 2022 · cited 3× · PMID 35143613 · DOI 10.1182/bloodadvances.2021006357