150 Participants Needed

Haploidentical Transplant for Blood Disorders

DS
Overseen ByDerek Schatz
Age: 18+
Sex: Any
Trial Phase: Academic
Sponsor: University of Colorado, Denver
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Approved in 1 JurisdictionThis treatment is already approved in other countries

Trial Summary

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

The trial protocol does not specify whether you need to stop taking your current medications.

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 idea that Haploidentical Transplant for Blood Disorders is an effective treatment?

The available research shows that Haploidentical Transplant for Blood Disorders, when combined with umbilical cord blood, can lead to successful outcomes. In one study, 83% of patients experienced successful engraftment, which means the transplanted cells started to grow and produce healthy blood cells. Additionally, the study reported a 54% overall survival rate at one year, indicating that more than half of the patients were still alive after a year. This suggests that the treatment can be effective for patients who do not have a fully matched donor. Compared to other methods, such as using two mismatched cord blood units, this approach provides a viable alternative with rapid recovery of blood cells and a lower risk of complications like graft-versus-host disease, where the new immune cells attack the patient's body.12345

What data supports the effectiveness of the treatment Haploidentical CD34+ Selected Cells Combined With Single Unit Umbilical Cord Blood Transplant?

Research shows that using a combination of haploidentical CD34+ selected cells and umbilical cord blood can lead to rapid recovery of blood cells and a good chance of successful engraftment, with 83% of patients achieving this by day 60. This approach also has a relatively low incidence of severe graft-versus-host disease, a common complication in transplants.12345

What safety data is available for haploidentical transplants for blood disorders?

The safety data for haploidentical transplants, including those using CD34+ selected cells combined with umbilical cord blood, indicates that this approach can result in rapid engraftment with no increased rate of graft failure or graft-versus-host disease (GVHD). In a study of 26 patients, the incidence of grade 2-4 acute GVHD was 27% at day +100, and chronic GVHD was 4% at 1 year. Overall survival at 1 year was 54%. Recent advances have reduced historical issues of graft rejection and severe GVHD, making haploidentical stem cell transplantation a viable option for patients without a fully matched donor. However, post-transplant infectious complications and relapse remain challenges.16789

Is haploidentical transplant generally safe for humans?

Haploidentical transplants, which use a partially matched family donor, have shown promising safety results. Studies indicate that recent advances have reduced issues like graft rejection and severe graft-versus-host disease (a condition where the donor's immune cells attack the recipient's body), making it a viable option for those without fully matched donors.136710

Is the treatment Haploidentical CD34+ Selected Cells Combined With Single Unit Umbilical Cord Blood Transplant a promising treatment for blood disorders?

Yes, this treatment is promising because it provides an alternative for patients who don't have a fully matched donor. It allows for nearly immediate donor availability and has shown improved outcomes over the past decade. This approach helps meet the need for stem cell transplants, especially for those with diverse backgrounds or urgent needs.1011121314

How is the treatment Haploidentical CD34+ Selected Cells Combined With Single Unit Umbilical Cord Blood Transplant different from other treatments for blood disorders?

This treatment is unique because it combines haploidentical stem cells, which are partially matched from a family member, with umbilical cord blood cells, allowing for a broader range of donor options and potentially faster availability. It addresses the challenge of finding fully matched donors and aims to improve outcomes by using both sources to enhance engraftment and immune recovery.1011121314

What is the purpose of this trial?

This is a study to evaluate the safety and efficacy of Miltenyi CliniMACS® CD34 Reagent System to promote engraftment of haploidentical CD34+ selected cells combined with single unit umbilical cord blood transplant for treatment of high-risk hematologic disorders.

Research Team

Dr. Jonathan A. Gutman, MD | Aurora, CO ...

Jonathan Gutman, MD

Principal Investigator

University of Colorado, Denver

Eligibility Criteria

This trial is for adults aged 18-80 with high-risk blood disorders who lack an exact HLA-matched donor but have a partially matched (5/10 to 8/10) relative and a suitable umbilical cord blood unit. It's not open to those with a fully matched (6/6) related donor or who don't meet standard transplant guidelines.

Inclusion Criteria

I have a cord blood match with at least 4/6 HLA compatibility and enough cells for my body weight.
I have a family donor who is a partial match for a stem cell transplant.
I understand the treatment I will be receiving.
See 2 more

Exclusion Criteria

I have a family donor who is a perfect match for my transplant.
Any patient not meeting institutional standard guidelines for transplant eligibility

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Conditioning Regimen

Recipients receive a standard conditioning regimen before transplantation

1 week

Transplantation

Participants receive an allograft containing donor CD34+ cells and a single UCB unit

Day 0

Engraftment Monitoring

Participants are monitored for successful engraftment and treatment-related serious adverse events

6 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • Haploidentical CD34+ Selected Cells Combined With Single Unit Umbilical Cord Blood Transplant
  • Miltenyi CliniMACS® CD34 Reagent System
Trial Overview The study tests the Miltenyi CliniMACS® CD34 Reagent System, aiming to improve how well patients accept stem cells from partly matched relatives combined with umbilical cord blood transplants in treating serious blood diseases.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: Miltenyi CliniMACS® CD34 Reagent SystemExperimental Treatment1 Intervention
The haploidentical donor will be mobilized by G-CSF and undergo one apheresis to collect CD34+ selected stem cell product after Miltenyi CliniMACS® CD34+ selection. The products will be cryopreserved until the time of transplantation. Recipients will receive a standard conditioning regimen. After the conditioning regimen, the subjects will receive an allograft on day 0 containing donor CD34+ cells that have been positively selected and T-cell depleted following G-CSF mobilization combined with a single UCB unit. UCB unit will not be manipulated, and will be prepared and infused separately following standard of care procedure.

Haploidentical CD34+ Selected Cells Combined With Single Unit Umbilical Cord Blood Transplant is already approved in United States for the following indications:

🇺🇸
Approved in United States as CliniMACS CD34 Reagent System for:
  • Processing hematopoietic progenitor cells collected by apheresis (HPC, Apheresis) from an allogeneic, HLA-identical, sibling donor to obtain a CD34+ cell-enriched population for hematopoietic reconstitution following a myeloablative preparative regimen without the need for additional graft versus host disease (GVHD) prophylaxis in patients with acute myeloid leukemia (AML) in first morphologic complete remission.

Find a Clinic Near You

Who Is Running the Clinical Trial?

University of Colorado, Denver

Lead Sponsor

Trials
1,842
Recruited
3,028,000+

Findings from Research

Haplo-cord transplantation using CD34+-selected partially matched unrelated donor grafts allows for rapid engraftment in patients lacking a suitable haploidentical donor, with 83% achieving cord blood unit (CBU) engraftment by day +60.
The procedure demonstrated a manageable safety profile, with a 27% incidence of grade 2-4 acute graft-versus-host disease (GVHD) and a 4% incidence of chronic GVHD at one year, while achieving an overall survival rate of 54% at one year.
Cord blood transplants supported by unrelated donor CD34+ progenitor cells.Gomez-Arteaga, A., Orfali, N., Guarneri, D., et al.[2021]
Transplanting two units of umbilical cord blood (UCB) simultaneously can enhance hematopoietic reconstitution in adults, as demonstrated in a patient with advanced acute lymphocytic leukemia who achieved complete remission without graft-versus-host disease.
Both UCB units contributed to blood cell production, showing double chimerism, which suggests that this method could improve outcomes for adult patients who do not have a compatible donor.
Double-chimaerism after transplantation of two human leucocyte antigen mismatched, unrelated cord blood units.De Lima, M., St John, LS., Wieder, ED., et al.[2019]
The study successfully created a novel Human Multi-Chimeric Cell (HMCC) line by fusing umbilical cord blood cells from three unrelated donors, which may offer a new therapeutic approach for inducing transplant tolerance without the need for lifelong immunosuppression.
HMCC demonstrated safety with no genotoxicity, maintained viability, and showed clonogenic properties, indicating its potential effectiveness for bone marrow and solid organ transplants.
Human Multi-Chimeric Cell (HMCC) Therapy as a Novel Approach for Tolerance Induction in Transplantation.Siemionow, M., Cwykiel, J., Brodowska, S., et al.[2023]

References

Cord blood transplants supported by unrelated donor CD34+ progenitor cells. [2021]
Double-chimaerism after transplantation of two human leucocyte antigen mismatched, unrelated cord blood units. [2019]
Human Multi-Chimeric Cell (HMCC) Therapy as a Novel Approach for Tolerance Induction in Transplantation. [2023]
Refining eligibility criteria of unit selection for myeloablative cord blood transplantation in acute leukemia: Real-world experience of a referral center. [2023]
Double umbilical cord blood transplantation: relevance of persistent mixed-unit chimerism. [2015]
Factors associated with outcomes of unrelated cord blood transplant: guidelines for donor choice. [2019]
The role of haploidentical stem cell transplantation in the management of children with haematological disorders. [2019]
[Treatment of two case childhood acute lymphoblastic leukemia by HLA-mismatched unrelated umbilical cord blood transplantation]. [2016]
Should double cord blood transplants be the preferred choice when a sibling donor is unavailable? [2021]
Alternate Donor Transplantation. [2023]
11.United Statespubmed.ncbi.nlm.nih.gov
Haploidentical bone marrow transplantation in Mexico. [2012]
Strategies in haploidentical stem cell transplantation in adults. [2021]
The great debate: haploidentical or cord blood transplant. [2011]
14.United Statespubmed.ncbi.nlm.nih.gov
Overview of the progress on haploidentical hematopoietic transplantation. [2020]
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