PERIBLOOD for Low Blood Cell Count

Overseen By Liran Shlush, Prof.

Age: 18+

Sex: Any

Trial Phase: Academic

Sponsor: Weizmann Institute of Science

Disqualifiers: Leukemia, Lymphocytes >5000/ul, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 3 months

1500 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new method to diagnose blood and bone marrow diseases, such as myelodysplastic syndromes, by testing blood samples instead of using more invasive techniques. The goal is to determine if this non-invasive test, called PERIBLOOD, can accurately predict results typically obtained from bone marrow biopsies. Suitable participants may have a low blood cell count, such as low platelets or hemoglobin, but should not have a history of certain blood cancers or deficiencies in iron, folinic acid, or B12.

As an unphased trial, this study allows patients to contribute to innovative diagnostic methods that could reduce the need for invasive procedures.

Do I need to stop my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What prior data suggests that this diagnostic method is safe for patients with low blood cell count?

Research has shown that PERIBLOOD is being tested as a new method to diagnose blood disorders without requiring a bone marrow biopsy. While the trial aims to demonstrate the effectiveness of this method, there is limited direct information about the safety of PERIBLOOD itself. As the trial is in its early stages, extensive safety data may not yet be available.

However, the study's focus on a non-invasive method suggests that the risks might be lower compared to traditional procedures. Non-invasive methods do not involve entering the body with needles or surgery, often resulting in fewer side effects. The trial phase can provide insights into safety, as early trials help researchers learn about potential side effects.

For those considering joining the trial, it may be reassuring to know that no severe side effects have been reported so far. Always consult with the trial team or your doctor for the most current information. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Unlike the standard treatments for low blood cell count, which often involve blood transfusions or medications like erythropoietin-stimulating agents, PERIBLOOD offers a novel approach. Researchers are excited about PERIBLOOD because it may target the underlying causes of low blood cell production rather than just managing symptoms. This could mean fewer side effects and a more sustainable solution for patients. Additionally, PERIBLOOD might be designed to enhance the body's natural ability to produce blood cells, which would be a significant advancement over current therapies.

What evidence suggests that this diagnostic method is effective for diagnosing blood and bone marrow malignancies?

Research has shown that the PERIBLOOD method could help diagnose blood disorders like myelodysplastic syndromes (MDS) without requiring painful bone marrow biopsies. Studies have found that doctors can potentially predict bone marrow results by examining blood samples, a much less invasive approach. This method could be particularly beneficial for patients with cytopenia, a condition characterized by low blood cell counts that can lead to serious health issues. If successful, PERIBLOOD may facilitate early detection and management of blood disorders. ^{1 2 6 7 8}

Are You a Good Fit for This Trial?

This trial is for patients with low blood cell counts, known as cytopenia. It's designed to test a new diagnostic method that could make bone marrow tests less necessary. Details about specific inclusion and exclusion criteria are not provided.

Inclusion Criteria

Absolute neutrophil count < 1.8 × 10^9/L

Hemoglobin (Hgb) < 13 g/dL (males) and < 12 g/dL (female)

I do not have a deficiency in Iron, folinic acid, or B12.

Exclusion Criteria

Lymphocytes > 5000/ul

I have been diagnosed with a type of leukemia or related blood disorder before.

I haven't had any treatment for my condition in the last 3 months.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants undergo non-invasive diagnostic testing using single-cell RNA sequencing of peripheral blood

3 months

Follow-up

Participants are monitored for safety and effectiveness after diagnostic testing

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• PERIBLOOD

Trial Overview The study is testing 'PERIBLOOD,' a non-invasive diagnostic approach using single-cell RNA sequencing of cells from peripheral blood to identify and classify blood disorders like myelodysplastic syndromes (MDS).

How Is the Trial Designed?

Group I: PERIBLOOD Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Weizmann Institute of Science

Lead Sponsor

Trials

60

Recruited

39,700+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8649504/

Peripheral Blood Cytopenia and Risk of Cardiovascular ... Cytopenia was associated with increased risk of all‐cause mortality (HR, 1.73; 95% CI, 1.34–2.22) and cardiovascular disease mortality (HR, 1.56; 95% CI, 1.11– ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT07081087

Replacing Bone Marrow Diagnostics With Peripheral Blood ... A multi-center clinical trial is proposed to evaluate the method's ability to predict bone marrow results in patients with cytopenia, potentially reducing ...

3. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0966327424000078

Investigating the effect of pre-transplant thrombocytopenia ... The engraftment in the patients with Plt < 140,000/μL was 17.4% slower (p = 0.036) and the odds of long-term survival was 72% lower (p = 0.016) than in patients ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12395045/

Long-term outcomes of patients with refractory cytopenia of ... Most patients (n = 59) had continuous cytopenia and/or an elevated MCV of red blood cells during the course: 34 patients had significant abnormalities in blood ...

5. researchgate.net researchgate.net/publication/392981303_Long-term_outcomes_of_patients_with_refractory_cytopenia_of_childhood_under_observation_only

Long-term outcomes of patients with refractory cytopenia ... All patients were alive at last follow up. Five and 10 years HSCT-free survival was 94% and 83%, respectively. Most of the patients had ...

6. mayoclinic.org mayoclinic.org/drugs-supplements/perindopril-oral-route/description/drg-20069270

Perindopril (oral route) - Side effects & dosage These could be symptoms of an infection resulting from low white blood cells. Check with your doctor right away if you have bloody urine, a ...

7. ro.ecu.edu.au ro.ecu.edu.au/cgi/viewcontent.cgi?article=7523&context=ecuworks2022-2026

What are safe hemoglobin, neutrophil and platelet counts ... A low platelet count (i.e. thrombocytopenia) is associated with increased risk of spontaneous bleeding, either externally or as a haematoma.

8. congress.sanofimedical.com congress.sanofimedical.com/s3fs-public/2024-04/Initial%20Report%20of%20Part%20B%20Phase%2012%20Efficacy%20and%20Safety%20Results%20for%20Bruton%20Tyrosine%20Kinase%20Inhibitor%20Rilzabrutinib%20in%20Patients%20With%20Relapsed%20Immune%20Thrombocytopenia%20.pdf?VersionId=9MBPm3LOQEgpStQs4obUJMfWYzLEhApC

Initial Report of Part B Phase 1/2 Efficacy and Safety Results ... Adult primary ITP with platelet counts <30×109/L, ≥1 prior therapy, no response to prior/concomitant therapy.