Biologically Focused Therapy of Treatment-Refractory MDS Patients

Stanford University

Status: Completed

Conditions: Myelodysplastic Syndromes

Study type: Observational

Funder types: Other

Identifiers

NCT05245032

HEMMDS0035 (Other Identifier)

IRB-45236 (Other Identifier)

Details and patient eligibility

About

This non interventional study examines the feasibility of using patient specific therapeutic screening method, ex vivo to enhance current treatment recommendations in a clinically feasible time frame of 30 days.

Full description

This study utilizes the Notable Labs personalized testing service using an ex vivo assay, but does not involve actual treatment or treatment determinations. The results of these screenings will be made available to patients and their physicians for their use off study.

The primary objective of the study is:

• To determine the feasibility of selecting patient specific treatment regimens based on ex vivo within 30 days

The exploratory objectives of the study are:

• To identify biomarkers that explain ex vivo drug sensitivity results

Enrollment: 33 patients

Sex: All

Ages: 18+ years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

• Provide informed consent;
• Myelodysplastic syndrome as defined by WHO classification (see Appendix 2), or MDS/MPN with >= 5% blasts in the bone marrow or peripheral blood, including chronic myelomonocytic leukemia (CMML) 1 and 2 by WHO classification
• Relapsed/refractory disease, defined as failed or are ineligible for hypomethylating agent therapy
• Cytogenetics reported

Exclusion criteria

• Hypoplastic MDS
• Patients without adequate marrow samples for ex vivo analysis

Trial design

33 participants in 1 patient group

Feasibility testing

Description:

During the screening visit, blood and/or a bone marrow sample will be obtained for the patient's standard clinical evaluation. An aliquot of the blood and/or marrow sample will be obtained for ex vivo drug sensitivity assay