140 Participants Needed

Stem Cell Transplant for Blood Cancers

(AB-CliniMACs Trial)

MT
PH
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MR
MA
Overseen ByMegan Atkinson
Age: < 65
Sex: Any
Trial Phase: Phase 2
Sponsor: Children's Hospital of Philadelphia
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 2 JurisdictionsThis treatment is already approved in other countries

Trial Summary

Do I have to stop taking my current medications for the trial?

The trial protocol does not specify whether you need to stop taking your current medications. Please consult with the trial coordinators for more details.

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 Stem Cell Transplant for Blood Cancers is an effective treatment?

The available research shows that Stem Cell Transplant for Blood Cancers is an effective treatment because it has a strong anti-tumor effect, known as 'graft versus leukemia', which helps fight blood cancers. Studies indicate that this treatment is a proven option for patients with blood-related cancers, offering a curative potential. Although there are risks like graft versus host disease, the treatment is expanding due to its demonstrated effectiveness in both myeloid and lymphoid cancers. Compared to other treatments, it provides a unique immune response that targets cancer cells, making it a powerful option for patients with high-risk blood cancers.12345

What data supports the effectiveness of the treatment Allogeneic Stem Cell Transplant for blood cancers?

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a proven treatment for blood cancers, as it uses the immune system from a donor to fight cancer cells, known as the 'graft versus leukemia' effect. This treatment has shown benefits in both myeloid and lymphoid blood cancers, despite some risks like graft versus host disease, which occurs when the donor's immune cells attack the patient's body.12345

What safety data is available for stem cell transplants in blood cancer treatment?

Allogeneic hematopoietic stem cell transplantation (HSCT) is associated with significant risks, including morbidity and mortality. While the donation process is generally safe, with the main risk being related to anesthesia, the transplantation itself can lead to serious adverse events, including cardiac issues and late-onset pulmonary complications. Long-term survivors are at risk for cardiovascular disease, and there is limited data on the long-term effects on cardiac function. Despite these risks, HSCT remains a potential cure for various diseases, but careful monitoring and further studies are needed to better understand and mitigate these risks.678910

Is allogeneic stem cell transplantation safe for humans?

Allogeneic stem cell transplantation can be risky, with potential for serious side effects like heart problems and lung issues, especially in long-term survivors. However, the donation process itself is generally safe, with the main risks being related to anesthesia and some pain at the donation site.678910

Is the treatment Allogeneic Stem Cell Transplant a promising treatment for blood cancers?

Yes, Allogeneic Stem Cell Transplant is a promising treatment for blood cancers. It can cure patients by using the body's immune system to fight cancer cells, known as the 'graft versus leukemia' effect. This treatment has shown success in both myeloid and lymphoid cancers, and new methods have made it safer and more effective. It also offers hope for other types of cancers, like colorectal cancer, by using the body's immune response.14111213

How is the treatment Allogeneic Stem Cell Transplant different from other treatments for blood cancers?

Allogeneic Stem Cell Transplant is unique because it uses stem cells from a donor to help the patient's body fight cancer through a process called graft-versus-leukemia, where the donor's immune cells attack the cancer cells. This treatment can be curative and is often used when other treatments have failed, but it carries risks like graft-versus-host disease, where the donor cells attack the patient's healthy tissues.14111213

What is the purpose of this trial?

This is a single arm pilot study for patients using α/β T cell-depleted peripheral Stem Cell Transplantation (PSCT) in with alternative donor sources with hematologic malignancies receiving alternative donor (unrelated or partially matched related) mobilized peripheral stem cells (PSCs) using the CliniMACS system for T cell depletion plus CD19+ B cell depletion to determine efficacy as determined by engraftment and graft versus host disease (GVHD), and one year leukemia free survival.

Research Team

Timothy S. Olson, MD, PhD | Children's ...

Timothy S Olson, MD, PhD

Principal Investigator

Children's Hospital of Philadelphia

Eligibility Criteria

This trial is for patients with various blood cancers like leukemia and lymphoma who are in remission or have minimal disease presence. They must have acceptable organ function, no active untreated infections, and a negative pregnancy test if applicable. Those who've had previous allogeneic transplants or lack a suitable donor can't participate.

Inclusion Criteria

My leukemia/lymphoma is in remission or has less than 10% bone marrow blasts.
I am not pregnant.
My organs are functioning well.
See 2 more

Exclusion Criteria

No suitable donor
I have had a transplant from a donor who is not my sibling.
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

Treatment

Participants receive α/β T cell-depleted peripheral Stem Cell Transplantation with various conditioning regimens including TBI, Busulfan, Thiotepa, and Cyclophosphamide

11 days

Follow-up

Participants are monitored for engraftment, graft versus host disease (GVHD), and leukemia-free survival

1 year

Treatment Details

Interventions

  • Allogeneic Stem Cell Transplant
  • Alpha Beta T cell depletion
Trial Overview The study tests α/β T cell-depleted peripheral Stem Cell Transplantation using alternative donors to treat blood cancers. It aims to see how well patients accept the transplant without developing graft versus host disease (GVHD) and their survival rate without leukemia after one year.
Participant Groups
3Treatment groups
Experimental Treatment
Group I: Alpha Beta Total Body Irradiation - total body irradiation (TBI) firstExperimental Treatment1 Intervention
Alpha Beta Total Body Irradiation - TBI first Day Treatment * 11 Anti-thymocyte globulin (ATG) * 10 ATG * 9 ATG * 8 TBI * 7 TBI * 6 TBI * 5 Thiotepa * 4 Thiotepa * 3 Cyclophosphamide * 2 Cyclophosphamide * 1 Rest 0 Transplant with alpha beta T cell depleted stem cells
Group II: Alpha Beta Total Body Irradiation - TBI lastExperimental Treatment1 Intervention
Alpha Beta Total Body Irradiation - TBI last Day Treatment * 9 ATG * 8 ATG * 7 Thiotepa + ATG * 6 Thiotepa * 5 Cyclophosphamide * 4 Cyclophosphamide * 3 TBI * 2 TBI * 1 TBI 0 Transplant with alpha beta T cell depleted stem cells
Group III: Alpha Beta Non-irradiation regimenExperimental Treatment1 Intervention
Alpha Beta Non-irradiation regimen Day Treatment * 9 Busulfan + ATG * 8 Busulfan + ATG * 7 Busulfan +ATG * 6 Busulfan * 5 Thiotepa * 4 Thiotepa * 3 Cyclophosphamide * 2 Cyclophosphamide * 1 0 Transplant with alpha beta T cell depleted stem cells

Allogeneic Stem Cell Transplant is already approved in United States, European Union for the following indications:

🇺🇸
Approved in United States as Allogeneic Hematopoietic Stem Cell Transplantation for:
  • Acute Leukemia
  • Chronic Leukemia
  • Lymphoma
  • Multiple Myeloma
  • Other hematologic malignancies
🇪🇺
Approved in European Union as Allo-HSCT for:
  • Acute Leukemia
  • Chronic Leukemia
  • Lymphoma
  • Multiple Myeloma
  • Other hematologic malignancies

Find a Clinic Near You

Who Is Running the Clinical Trial?

Children's Hospital of Philadelphia

Lead Sponsor

Trials
749
Recruited
11,400,000+

Findings from Research

In a study of 206 patients who underwent unrelated haematopoietic stem cell transplantation (HSCT), those receiving HLA-identical grafts had similar overall survival rates (52%) compared to those with one or more mismatched antigens (48%) after a median follow-up of 49 months.
The findings suggest that using donors with one antigen mismatch is a viable option for patients with hematological malignancies when fully matched donors are not available, as outcomes in terms of survival and graft-versus-host disease were comparable.
Comparable outcome after single-antigen-mismatched versus matched unrelated donor haematopoietic cell transplantation.Rockstroh, A., Al-Ali, HK., Lange, T., et al.[2018]
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) has become a powerful curative option for patients with specific blood disorders, showing significant advancements in recent years.
Key improvements include the use of haploidentical HSCT and enhanced strategies for managing aplastic anemia, relapse, and graft versus host disease, particularly in the context of practices in China.
Allo-Hematopoietic Stem Cell Transplant in China: 2014 Update.Lv, M., Huang, XJ.[2015]
Hematopoietic stem cell transplantation (HSCT) has evolved significantly over the past 45 years, with autologous HSCT primarily used after high-dose chemotherapy and allogeneic HSCT utilized for treating marrow failures and enhancing anti-tumor effects.
In 2010, approximately 30,000 patients underwent HSCT in Europe, and recent advancements in patient and donor selection, stem cell sourcing, and supportive care have expanded the indications for allogeneic HSCT, improving treatment outcomes.
Hematopoietic stem cell transplantation: a review and recommendations for follow-up care for the general practitioner.Passweg, JR., Halter, J., Bucher, C., et al.[2018]

References

Comparable outcome after single-antigen-mismatched versus matched unrelated donor haematopoietic cell transplantation. [2018]
Allo-Hematopoietic Stem Cell Transplant in China: 2014 Update. [2015]
Hematopoietic stem cell transplantation: a review and recommendations for follow-up care for the general practitioner. [2018]
[Allogeneic hematopoietic stem cell transplantation]. [2009]
Chronic graft versus host disease but not the intensity of conditioning has impact on survival after allogeneic hematopoietic stem cell transplantation for advanced hematological diseases. [2014]
Safety of bone marrow stem cell donation: a review. [2010]
Allogeneic hematopoietic stem cell transplantation for severe autoimmune diseases. [2009]
Having a sibling as donor: patients' experiences immediately before allogeneic hematopoietic stem cell transplantation. [2022]
Late-Onset, Noninfectious Pulmonary Complications following Allogeneic Hematopoietic Stem Cell Transplantation: A Nationwide Cohort Study of Long-Term Survivors. [2022]
10.United Statespubmed.ncbi.nlm.nih.gov
Left Ventricular Systolic Function in Long-Term Survivors of Allogeneic Hematopoietic Stem Cell Transplantation. [2022]
Allogeneic haematopoietic stem cell transplantation: current status and future outlook. [2019]
12.United Statespubmed.ncbi.nlm.nih.gov
Reduced-intensity allogeneic hematopoietic stem-cell transplantation as an immunotherapy for metastatic colorectal cancer. [2019]
Reliable engraftment, low toxicity, and durable remissions following allogeneic blood stem cell transplantation with minimal conditioning. [2019]
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