Stem Cell Transplant for Blood Cancer

Age: < 65

Sex: Female

Trial Phase: Phase 2 & 3

Sponsor: Julie-An M. Talano

Disqualifiers: No suitable donor, others

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

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

60 Participants Needed

Trial Summary

What is the purpose of this trial?

This is a single arm pilot study for patients with hematologic malignancies with alternative donor sources receiving unrelated or partially matched related/Haploidentical mobilized peripheral stem cells (PSCs) using the CliniMACS system for Alpha Beta T cell depletion plus CD19+ B cell depletion to determine efficacy as determined by engraftment and GVHD, and one year leukemia free survival.

Will I have to stop taking my current medications?

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 data supports the effectiveness of the treatment CliniMACS, Peripheral Blood Stem Cell Transplantation for blood cancer?

Peripheral blood stem cell transplantation (PBSCT) is shown to improve outcomes for patients with blood cancers, as it is used as an alternative to bone marrow transplantation and has been effective in treating various hematologic diseases. ^{1 2 3 4 5}

Is peripheral blood stem cell transplantation generally safe for humans?

Peripheral blood stem cell transplantation (PBSC) is generally considered safe, with a lower risk of serious adverse events compared to bone marrow donation. In a study, only 0.56% of PBSC donors experienced serious issues, which is significantly lower than the 2.38% for bone marrow donors. ^{2 4 6 7 8}

How is the treatment CliniMACS, Peripheral Blood Stem Cell Transplantation different from other treatments for blood cancer?

This treatment is unique because it involves using a patient's own stem cells from their blood, which are collected and then transplanted back after high-dose chemotherapy. This method, known as autologous peripheral blood stem cell transplantation, is gradually replacing traditional bone marrow transplants due to its effectiveness and safety. ^{2 3 7 9 10}

Research Team

Julie-An Talano

Principal Investigator

Medical College of Wisconsin

Eligibility Criteria

This trial is for patients under 23 years old with various blood cancers, including lymphoma and leukemia, who are in remission or have less than 10% bone marrow blasts. It's open to all genders and races but excludes those without a suitable donor or who don't meet specific disease, organ function, or infection criteria.

Inclusion Criteria

I am younger than 23 years old.

My condition is acute myeloid leukemia.

My condition is in a specific phase of chronic illness.

Exclusion Criteria

There is no available person who can donate what is needed for the study.

My condition does not match the specific disease, organ, or infection criteria.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Conditioning

Participants undergo conditioning regimen prior to stem cell transplantation

1-2 weeks

Transplantation

Participants receive peripheral stem cell transplantation with alpha beta T cell and CD19+ B cell depletion

1 day

Engraftment

Monitoring for engraftment defined as ANC >500 for the first of 3 consecutive days

6 weeks

Follow-up

Participants are monitored for safety, effectiveness, and incidence of GVHD and TRM

1 year

Treatment Details

Interventions

• CliniMACS
• Peripheral Blood Stem Cell Transplantation

Trial Overview The study tests the CliniMACS device's ability to deplete Alpha Beta T cells plus CD19+ B cells from stem cell transplants in patients with blood cancers using alternative donors. The goal is to see how well it works by looking at engraftment success, avoidance of GVHD (graft-versus-host disease), and one-year survival without leukemia.

Participant Groups

Group I: Single Experimental Arm Experimental Treatment 1 Intervention

Alpha Beta T cell depletion is performed for all PBSC grafts using the CliniMACs device

Who Is Running the Clinical Trial?

Julie-An M. Talano

Lead Sponsor

Trials

1

Recruited

60+

Miltenyi Biotec, Inc.

Industry Sponsor

Trials

11

Recruited

280+

Findings from Research

Allogeneic peripheral blood stem cell transplantation (allo-PBSCT) showed a significantly lower relapse rate (5.7%) compared to autologous PBSCT (auto-PBSCT) which had a relapse rate of 38.9%, indicating better long-term efficacy in treating malignant hematologic diseases.

The study found that allo-PBSCT resulted in a higher disease-free survival rate (69.5%) compared to auto-PBSCT (57.9%), suggesting that allo-PBSCT may be a more effective treatment option for patients with these conditions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Peripheral blood stem cell transplantation for 53 patients with malignant hematologic diseases]. Xie, XS., Wan, DM., Sun, H., et al.[2016]

In a study of 215 patients receiving autologous peripheral blood progenitor cell (PBPC) infusions, 56.9% experienced infusion-related adverse reactions, highlighting the need for careful monitoring during treatment.

Factors such as female gender, multiple myeloma diagnosis, and the number of granulocytes infused per kg body weight were significant predictors of these adverse reactions, indicating that patient characteristics and graft composition play crucial roles in infusion safety.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Toxicity related to autologous peripheral blood haematopoietic progenitor cell infusion is associated with number of granulocytes in graft, gender and diagnosis of multiple myeloma. Bojanic, I., Cepulic, BG., Mazic, S., et al.[2011]

Peripheral blood stem cell transplantation can lead to severe complications, such as graft versus host disease (GVH) and subsequent organ failure, particularly in immunocompromised patients.

Adenoviral infections pose a significant risk for these patients, potentially leading to fatal outcomes, highlighting the need for improved diagnostics and early treatment strategies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Multiple organ failure and disseminated adenoviral infection]. Bretonnière, C., Touzeau, C., Guillaume, T., et al.[2010]

References

1. United States pubmed.ncbi.nlm.nih.gov

Overview of bone marrow and peripheral blood stem cell transplantation. [2005]

2. United States pubmed.ncbi.nlm.nih.gov

The comparison of effectiveness and safety between different biosimilars of G-CSF in the mobilization of peripheral blood stem cells (PBSCs) for autologous transplantation (autologous peripheral blood stem cell transplantation, auto-PBSCT). [2021]

3. China pubmed.ncbi.nlm.nih.gov

[Peripheral blood stem cell transplantation for 53 patients with malignant hematologic diseases]. [2016]

4. Germany pubmed.ncbi.nlm.nih.gov

Changes of immunological markers after autologous peripheral blood stem cell transplantation. [2019]

5. United States pubmed.ncbi.nlm.nih.gov

Analysis of laboratory parameters for optimal autologous peripheral blood stem cell collection from lymphoma and myeloma patients. [2021]

6. United States pubmed.ncbi.nlm.nih.gov

Lower risk for serious adverse events and no increased risk for cancer after PBSC vs BM donation. [2022]

7. China pubmed.ncbi.nlm.nih.gov

[Unrelated peripheral blood stem cell transplantation for a patient with acute myeloid leukemia]. [2004]

8. England pubmed.ncbi.nlm.nih.gov

Toxicity related to autologous peripheral blood haematopoietic progenitor cell infusion is associated with number of granulocytes in graft, gender and diagnosis of multiple myeloma. [2011]

9. France pubmed.ncbi.nlm.nih.gov

[Multiple organ failure and disseminated adenoviral infection]. [2010]

10. Iceland pubmed.ncbi.nlm.nih.gov

[Hematopoietic stem cell grafts.]. [2009]