Phase 2 Study of Venetoclax-containing Therapy in Combination With HLA-mismatched Mobilized Peripheral Blood Mononuclear Cell Infusion for Intermediate-risk and Higher Myelodysplastic Syndromes

Status: Recruiting
Location: See location...
Intervention Type: Biological, Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY

This study aims to evaluate the safety and efficacy of a Venetoclax and hypomethylating agent-based regimen combined with infusion of HLA-mismatched donor G-CSF mobilized peripheral blood mononuclear cells (GPBMC) in patients with intermediate-risk and higher myelodysplastic syndromes who are ineligible for allogeneic hematopoietic stem cell transplantation.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• Age \>=18 years, male or female, non-limited by race or ethnicity.

• Confirmed diagnosis of MDS according to the World Health Organization (WHO) 5th edition classification, based on histopathology and cytogenetics.

• Risk stratification according to the Revised International Prognostic Scoring System (IPSS-R) must place the patient in the intermediate-, high-, or very high-risk category.

• Not candidates for or refuse allogeneic hematopoietic stem cell transplantation.

• Adequate hepatic function including alanine transaminase (ALT) and aspartate aminotransferase (AST )\<= 3 × upper limit of normal(ULN), and total bilirubin \<= 1.5 × ULN.

• Adequate renal function including serum creatinine \<= 2 × ULN or CrCl\>= 40mL/min.

• LVEF measured by echocardiogram is within the normal range (LVEF \> 50%).

• The subject must have one donor who is \>= 18 years old and HLA matched at 0-7/10 loci (i.e., at least 3 HLA loci must be mismatched). In addition, the donor voluntarily donates hematopoietic stem cells and signs the consent form.

• Each subject (or his/her legal representatives) must sign the Informed Consent Form (ICF), indicating that he/she understands the purpose and procedures of research, and is willing to participate in research.

• Donor inclusion criteria: The donor meets the institution's criteria for related peripheral blood hematopoietic stem cell donors. The donor must be able to tolerate the cell separation and collection process, and sign the Informed Consent Form.

Locations
Other Locations
China
Department of Hematology, the Fifth Medical Center of Chinese PLA General Hospital
RECRUITING
Beijing
Contact Information
Primary
Bo Cai, MD
caibo2008@163.com
+861066947168
Backup
Fei Peng, MD
pengfei0118@foxmail.com
+861066947180
Time Frame
Start Date: 2025-06-20
Estimated Completion Date: 2031-06
Participants
Target number of participants: 40
Treatments
Experimental: Cohort 1
This cohort includes patients with intermediate-risk and higher MDS who are ineligible for or refuse allogeneic HSCT. Patients receive Venetoclax in combination with hypomethylating agent and HLA-mismatched donor GPBMC infusion. Treatment cycles are 28 days, repeated until disease progression or unacceptable toxicity.
Related Therapeutic Areas
Sponsors
Leads: Beijing 302 Hospital

This content was sourced from clinicaltrials.gov