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A slowly progressing leukemia characterized by a clonal (malignant) proliferation of maturing and mature myeloid cells or mature lymphocytes. When the clonal cellular population is composed of myeloid cells, the process is called chronic myelogenous leukemia. When the clonal cellular population is composed of lymphocytes, it is classified as chronic lymphocytic leukemia, hairy cell leukemia, or T-cell large granular lymphocyte leukemia.
Biomarker and diagnostic research for chronic leukemia has been reported in the published literature.
No approved treatments are currently available for chronic leukemia. An additional 1 compound holds orphan drug designation.
While no drugs are FDA-approved specifically for chronic leukemia, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.
The following drugs have received orphan drug designation from the FDA for chronic leukemia. Orphan designation reflects regulatory interest and does not indicate approval for treatment.
Brand Name | Generic Name | Sponsor |
|---|
10 clinical trials registered, 7 recruiting. Interventions under study include other interventions, biologic therapy, drug therapy, and medical devices. Pipeline includes 4 NA. Research is sponsored by a mix of industry and academic institutions.
NCT ID | Title | Phase | Sponsor | Status |
|---|---|---|---|---|
[NCT07445438](https://clinicaltrials.gov/study/NCT07445438) |
Data assembled from 4 of 12 sources · Last updated Sep 18, 2026, 7:59 PM UTC
Common questions about chronic leukemia
Designated
Exclusivity End |
|---|
Designation Status |
|---|
Tretinoin | Tretinoin | Antigenics, Inc. | 1993 | — | Designated |
Gene therapy approaches for chronic leukemia have been reported in the published literature.
10 trials found
Feasibility of a Multi-omics Platform for Hematological Malignancies |
NA |
Azienda Ospedaliero-Universitaria di Parma |
RECRUITING |
[NCT05794880](https://clinicaltrials.gov/study/NCT05794880) | MCW Alpha/Beta T-Cell and B-Cell Depletion With Targeted ATG Dosing | NA | Medical College of Wisconsin | RECRUITING |
[NCT07403630](https://clinicaltrials.gov/study/NCT07403630) | INTEGRATIVE "MULTI-OMICS" AND FUNCTIONAL PLATFORM FOR THE COMPLETE DIAGNOSTIC CHARACTERIZATION OF TUMORS: THE ITALIAN TUMOR CHEMOGENOMIC PROFILER (IT-TCP) | NA | Azienda Ospedaliero-Universitaria di Parma | RECRUITING |
[NCT06297161](https://clinicaltrials.gov/study/NCT06297161) | A Study to Learn About the Study Medicine Bosulif in Adult Patients With Chronic Myeloid Leukemia(CML). | — | Pfizer | RECRUITING |
[NCT03016806](https://clinicaltrials.gov/study/NCT03016806) | Umbilical Cord Blood Transplantation From Unrelated Donors | — | University of Rochester | RECRUITING |
155 publications have been identified in PubMed for chronic leukemia. Kisho has analyzed 44 by research type. Research spans Review / Meta-Analysis (45%), Basic Science / Preclinical (16%), and Epidemiology / Natural History (14%).
Research Type | Count | % of Total |
|---|---|---|
Research summaries | 20 | 45% |
Laboratory research | 7 | 16% |
Disease patterns and progression | 6 | 14% |
Patient case studies | 5 | 11% |
Clinical study results | 2 | 5% |
New treatment approaches | 2 | 5% |
Other research | 1 | 2% |
Testing and diagnosis research | 1 | 2% |
Ye M (2026). [PMID: 41836260](https://pubmed.ncbi.nlm.nih.gov/41836260/). *Front Oncol*. [Review / Meta-Analysis]
Baral MR (2026). [PMID: 41058090](https://pubmed.ncbi.nlm.nih.gov/41058090/). *J Hosp Med*. [Review / Meta-Analysis]
Hajizadeh A (2026). [PMID: 41612765](https://pubmed.ncbi.nlm.nih.gov/41612765/). *Biotechnol Appl Biochem*. [Review / Meta-Analysis]
Yokoyama Y (2026). [PMID: 41726365](https://pubmed.ncbi.nlm.nih.gov/41726365/). *JCEM Case Rep*. [Case Report / Case Series]
Fleury M (2026). [PMID: 41976301](https://pubmed.ncbi.nlm.nih.gov/41976301/). *Cancers (Basel)*. [Clinical Trial Publication]
Alqahtani S (2025). [PMID: 41221040](https://pubmed.ncbi.nlm.nih.gov/41221040/). *Front Pharmacol*. [Review / Meta-Analysis]
Zhou Q (2025). [PMID: 40123764](https://pubmed.ncbi.nlm.nih.gov/40123764/). *Blood Sci*. [Review / Meta-Analysis]
Zaini RG (2025). [PMID: 40209798](https://pubmed.ncbi.nlm.nih.gov/40209798/). *Clin Lab*. [Epidemiology / Natural History]
Özay B (2025). [PMID: 41489186](https://pubmed.ncbi.nlm.nih.gov/41489186/). *Per Med*. [Review / Meta-Analysis]
Verma S (2025). [PMID: 40827484](https://pubmed.ncbi.nlm.nih.gov/40827484/). *Future Med Chem*. [Review / Meta-Analysis]