Biologically Focused Therapy of Treatment-Refractory MDS Patients

Biologically Focused Therapy of Treatment Refractory MDS Patients

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.

Study Overview

• Status: Completed
• Conditions: Myelodysplastic Syndromes

Detailed 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

• Study Type: Observational
• Enrollment (Actual): 33

Contacts and Locations

Study Locations

• United States
  • California
    • Stanford, California, United States, 94304
      • Stanford MDS Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: Yes

• Sampling Method: Probability Sample

Study Population

The study will be conducted at a single site, namely Stanford's MDS Center.

Description

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

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Feasibility testing; 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of patients with successful ex vivo results	Proportion of patients with successful ex vivo results within a 30 day period from the time marrow sample is sent to Notable Labs for ex vivo testing	30 days
Proportion of patients with successful treatment decisions	Proportion of patients with successful treatment decisions made by the molecular oncology board within a 30 day period from the time marrow sample is sent to Notable Labs for ex vivo testing	30 days

Collaborators and Investigators

• Sponsor: Stanford University
• Collaborators: Notable Labs Inc.
• Investigators: Principal Investigator: Peter L Greerberg, Stanford Universiy

Study record dates

Study Major Dates

• Study Start (Actual): February 18, 2020
• Primary Completion (Actual): December 1, 2022
• Study Completion (Actual): December 1, 2022

Study Registration Dates

• First Submitted: February 9, 2022
• First Submitted That Met QC Criteria: February 9, 2022
• First Posted (Actual): February 17, 2022

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: March 11, 2025
• Last Verified: March 1, 2025

More Information

Terms related to this study

• Keywords: MDS
• Additional Relevant MeSH Terms: Hematologic Diseases; Bone Marrow Diseases; Myelodysplastic Syndromes
• Other Study ID Numbers: IRB-45236 (Other Identifier: Stanford IRB); HEMMDS0035 (Other Identifier: OnCore)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No