CytopeniaNCT07081087
Replacing Bone Marrow Diagnostics With Peripheral Blood Analysis in Cytopenia Patients
An observational study of Cytopenia and MDS, run by Weizmann Institute of Science at 2 sites in United States.
Trial summary
This observational, multi-center study aims to collect data in order to develop a novel, minimally invasive diagnostic tool for MDS based on peripheral blood profiling of circulating hematopoietic stem and progenitor cells (cHSPCs) using single-cell RNA sequencing and DNA sequencing. Read more about this study
- Who is it for?
- Ages 18 years and older
- What is being tested?
- PERIBLOOD-MDS
- How many people
- 1,500 participants
- Where
- 2 sitesUnited States
- Study dates
- 1 Jun 2025to Dec 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients with cytopenia, who are referred to BM from the primary care clinic will be recruited to the study
Who may be able to join
- Patients aged 18 and up with suspected/confirmed MDS cases referred to BM evaluation either for diagnosis or risk assessment due to cytopenia
- Platelets < 150 × 10E9/L or
- Absolute neutrophil count < 1.8 × 10E9/L or
- Hemoglobin (Hgb) < 13 g/dL (males) and < 12 g/dL (female) and
- For both sexes, no evidence of Iron, folinic acid, or B12 deficiency
Who cannot join
- Women who are pregnant
- Previous diagnosis of leukemia; AML, MPN, ALL, CLL, MGUS/MM or any other gammopathy
- Lymphocytes > 5000/ul
- Patients who are on disease-related therapy are excluded, unless they are treated with Erythropoietin or Prednisone. See Appendix 2 for the list of excluded treatments.
- Patients who have undergone a bone marrow transplant.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites, 2 recruiting
- Recruiting
University of Miami
Miami, Florida, 33146
Helen Kattoura, Dr., 305-243-6438
Stephen Nimer
- Recruiting
University of California San Diego
San Diego, California, 92093
Tan Tuyet
Rafael Bejar
About this study
Official title: Development of a Peripheral Blood Assay to Replace BM Evaluation in Cytopenia - a Multi-Center Observational Study
This observational, multi-center study aims to collect data in order to develop a novel, minimally invasive diagnostic tool for MDS based on peripheral blood profiling of circulating hematopoietic stem and progenitor cells (cHSPCs) using single-cell RNA sequencing and DNA sequencing.
Read the detailed description
This observational, multi-center study aims to collect data in order to develop a novel, minimally invasive diagnostic tool for MDS based on peripheral blood profiling of circulating hematopoietic stem and progenitor cells (cHSPCs) using single-cell RNA sequencing and DNA sequencing. Current MDS diagnosis relies on bone marrow (BM) aspiration and biopsy, which is invasive, technically challenging, and sometimes inconclusive, particularly in early or low-risk disease. Our prior research established a healthy reference map of cHSPC subtypes and developed a computational pipeline that distinguishes MDS from non-clonal cytopenias based on deviations from this reference. In this study, patients referred, as part of standard care, for BM evaluation due to unexplained cytopenias or abnormal blood counts and undergo PB collection for genomic profiling. The study's primary objective is to develop a PB-based test by comparing the developed test diagnoses to the conventional BM-based one. Secondary objectives include evaluating its potential for MDS risk stratification and assessing its ability to reduce the need for BM evaluations.
- Being tested
- PERIBLOOD-MDS
- Medical subject headings
- Cytopenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PERIBLOOD-MDSDiagnostic test
In the study, peripheral blood will be taken from all patients, shipped and analyzed at the Weizmann Institute of Science to produce a report based on our technology. The report will contain data on the diagnosis, blast counts and karyotype. This information will be compared with the results from the bone marrow scan, taken separately to each patient, to validate the accuracy of our technology
| Study group | Role | What they receive |
|---|---|---|
| PERIBLOOD-MDS | No intervention is given Receives: Diagnostic Test: PERIBLOOD-MDS |
What is being measured
The primary outcome is the main question the study is designed to answer.
To develop PERIBLOOD-MDS diagnostic test of PB cHPCs for the diagnosis of MDS
Comparison between PERIBLOOD-MDS diagnosis and bone marrow evaluation of MDS
Measured: Three months following the peripheral blood sample
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jul 2025
- Study start1 Jun 2025
- Primary completionDec 2028 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CytopeniaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 1,500 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Weizmann Institute of Science
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Liran Shlush, Principal investigator, The Weizmann Institute of Science
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07081087
- Sponsor's protocol ID
- PERIBLODD-MDS
- Status last verified
- Sep 2025
- First submitted
- 15 Jul 2025
- Last updated
- 25 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Development of a Peripheral Blood Assay to Replace BM Evaluation in Cytopenia - a Multi-Center Observational Study
- ClinicalTrials.gov ID
- NCT07081087
- Acronym / sponsor ID
- PERIBLODD-MDS
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 1,500 participants (estimated)
- Lead sponsor
- Weizmann Institute of ScienceUniversity, hospital or other
- Study start
- 1 Jun 2025 (actual)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 23 Jul 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This observational, multi-center study aims to collect data in order to develop a novel, minimally invasive diagnostic tool for MDS based on peripheral blood profiling of circulating hematopoietic stem and progenitor cells (cHSPCs) using single-cell RNA sequencing and DNA sequencing.
Detailed description
This observational, multi-center study aims to collect data in order to develop a novel, minimally invasive diagnostic tool for MDS based on peripheral blood profiling of circulating hematopoietic stem and progenitor cells (cHSPCs) using single-cell RNA sequencing and DNA sequencing. Current MDS diagnosis relies on bone marrow (BM) aspiration and biopsy, which is invasive, technically challenging, and sometimes inconclusive, particularly in early or low-risk disease. Our prior research established a healthy reference map of cHSPC subtypes and developed a computational pipeline that distinguishes MDS from non-clonal cytopenias based on deviations from this reference. In this study, patients referred, as part of standard care, for BM evaluation due to unexplained cytopenias or abnormal blood counts and undergo PB collection for genomic profiling. The study's primary objective is to develop a PB-based test by comparing the developed test diagnoses to the conventional BM-based one. Secondary objectives include evaluating its potential for MDS risk stratification and assessing its ability to reduce the need for BM evaluations.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PERIBLOOD-MDS | – | Diagnostic Test: PERIBLOOD-MDSNo intervention is given |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with cytopenia, who are referred to BM from the primary care clinic will be recruited to the study
Key inclusion criteria (5 listed)
- Patients aged 18 and up with suspected/confirmed MDS cases referred to BM evaluation either for diagnosis or risk assessment due to cytopenia
- Platelets < 150 × 10E9/L or
- Absolute neutrophil count < 1.8 × 10E9/L or
- Hemoglobin (Hgb) < 13 g/dL (males) and < 12 g/dL (female) and
- For both sexes, no evidence of Iron, folinic acid, or B12 deficiency
Key exclusion criteria (5 listed)
- Women who are pregnant
- Previous diagnosis of leukemia; AML, MPN, ALL, CLL, MGUS/MM or any other gammopathy
- Lymphocytes > 5000/ul
- Patients who are on disease-related therapy are excluded, unless they are treated with Erythropoietin or Prednisone. See Appendix 2 for the list of excluded treatments.
- Patients who have undergone a bone marrow transplant.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PERIBLOOD-MDSIn the study, peripheral blood will be taken from all patients, shipped and analyzed at the Weizmann Institute of Science to produce a report based on our technology. The report will contain data on the diagnosis, blast counts and karyotype. This information will be compared with the results from the bone marrow scan, taken separately to each patient, to validate the accuracy of our technology | Diagnostic test | PERIBLOOD-MDS |
Primary outcome
To develop PERIBLOOD-MDS diagnostic test of PB cHPCs for the diagnosis of MDS
Time frame: Three months following the peripheral blood sample
Comparison between PERIBLOOD-MDS diagnosis and bone marrow evaluation of MDS
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Liran Shlush principal investigator
The Weizmann Institute of Science
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cytopenia and MDS, run by Weizmann Institute of Science at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 25 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Trial Manage (+972522666230, gil.gonen-yaacovi@weizmann.ac.il), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To develop PERIBLOOD-MDS diagnostic test of PB cHPCs for the diagnosis of MDS (time frame: Three months following the peripheral blood sample). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Mar 2026.