NCT02553941

Ibrutinib and Azacitidine for Treatment of Higher Risk Myelodysplastic Syndrome

Completed Phase 1 Last updated 6 October 2022

What this trial tests

Phase 1 trial testing Azacitidine in Chronic Myelomonocytic Leukemia in 21 participants. Completed in 7 November 2019.

Timeline

17 May 2016

Primary endpoint
25 May 2019

7 November 2019

Quick facts

Lead sponsor	Brian Jonas
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	21
Start date	17 May 2016
Primary completion	25 May 2019
Estimated completion	7 November 2019
Sites	5 locations across United States

Drugs / interventions tested

• Azacitidine (azacitidine) — full drug profile →
• Ibrutinib (ibrutinib) — full drug profile →

Conditions studied

• Chronic Myelomonocytic Leukemia — all drugs for Chronic Myelomonocytic Leukemia →
• de Novo Myelodysplastic Syndrome — all drugs for de Novo Myelodysplastic Syndrome →
• Previously Treated Myelodysplastic Syndrome — all drugs for Previously Treated Myelodysplastic Syndrome →
• Refractory Anemia With Excess Blasts in Transformation — all drugs for Refractory Anemia With Excess Blasts in Transformation →

Sponsor

Brian Jonas

Who can join

18 and older, any sex, with Chronic Myelomonocytic Leukemia or de Novo Myelodysplastic Syndrome. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This phase Ib trial studies the side effects and best dose of ibrutinib when given together with azacitidine in treating patients with myelodysplastic syndrome that is likely to occur or spread (higher risk) and who were previously treated or untreated and unfit for or refused intense therapy. Ibrutinib and azacitidine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

Publications & conference data

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

1. The central role of inflammatory signaling in the pathogenesis of myelodysplastic syndromes.
   Sallman DA, List A. · · 2019 · cited 202× · PMID 30670444 · DOI 10.1182/blood-2018-10-844654
2. Therapeutic targeting of the inflammasome in myeloid malignancies.
   Chakraborty S, Shapiro LC, de Oliveira S, Rivera-Pena B, et al · · 2021 · cited 29× · PMID 34521810 · DOI 10.1038/s41408-021-00547-8
3. Role of innate immunological/inflammatory pathways in myelodysplastic syndromes and AML: a narrative review.
   Vegivinti CTR, Keesari PR, Veeraballi S, Martins Maia CMP, et al · · 2023 · cited 27× · PMID 37422676 · DOI 10.1186/s40164-023-00422-1
4. Clinical Trials of the BTK Inhibitors Ibrutinib and Acalabrutinib in Human Diseases Beyond B Cell Malignancies.
   Zhu S, Jung J, Victor E, Arceo J, et al · · 2021 · cited 23× · PMID 34778053 · DOI 10.3389/fonc.2021.737943
5. Immune dysregulation and potential targeted therapy in myelodysplastic syndrome.
   Zhang X, Yang X, Ma L, Zhang Y, et al · · 2023 · cited 11× · PMID 37547364 · DOI 10.1177/20406207231183330
6. From Immune Dysregulations to Therapeutic Perspectives in Myelodysplastic Syndromes: A Review.
   Comont T, Treiner E, Vergez F. · · 2021 · cited 8× · PMID 34829329 · DOI 10.3390/diagnostics11111982
7. Incorporating novel approaches in the management of MDS beyond conventional hypomethylating agents.
   Odenike O. · · 2017 · cited 5× · PMID 29222294 · DOI 10.1182/asheducation-2017.1.460
8. Bone marrow microenvironment in myelodysplastic neoplasms: insights into pathogenesis, biomarkers, and therapeutic targets.
   Bahmani F, Shayanmanesh M, Safari M, Alaei A, et al · · 2025 · cited 3× · PMID 40349084 · DOI 10.1186/s12935-025-03793-z