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Dorocubicel

Dorocubicel, an innovative drug being studied in clinical trials, offers hope for patients with high-risk acute leukemia and myelodysplasia. This article explores the ongoing research on Dorocubicel, which uses expanded cord blood transplantation to potentially improve outcomes for patients requiring allogeneic hematopoietic stem cell transplantation.

Table of Contents

What is DOROCUBICEL?

DOROCUBICEL, also known as ECT-001-CB, is an innovative medical treatment being studied for patients with high-risk blood cancers[1]. It is a type of cell therapy that uses specially processed stem cells from umbilical cord blood. The technical name for this treatment is “UM171 dihydrobromide dihydrate expanded umbilical cord blood”[1].

Medical Conditions Treated

DOROCUBICEL is being investigated for the treatment of high-risk and very high-risk acute leukemia and myelodysplasia[1]. These are serious blood disorders that affect the production of healthy blood cells in the bone marrow. Patients with these conditions often require a stem cell transplant, also known as an allogeneic hematopoietic stem cell transplantation (HSCT), as part of their treatment[1].

How DOROCUBICEL Works

DOROCUBICEL is a combination of two components[1]:

  1. Expanded cord blood cells (DOROCUBICEL): These are stem cells from umbilical cord blood that have been treated with a special molecule called UM171. This process helps increase the number of stem cells available for transplant.
  2. Non-expanded cord blood cells: These are additional stem cells from cord blood that haven’t been expanded.
Together, these components are designed to provide a more effective stem cell transplant for patients who need it.

Clinical Trial Details

DOROCUBICEL is currently being studied in a Phase II clinical trial[1]. This means that while it has shown promise in earlier studies, it is still considered experimental and is not yet approved for widespread use. The main goals of this trial are to:

  • Examine the safety and feasibility of using DOROCUBICEL in patients with high-risk blood cancers[1]
  • Evaluate how well patients do after receiving the treatment, particularly looking at relapse-free survival at 1 and 2 years after the transplant[1]

Eligibility Criteria

Not everyone with leukemia or myelodysplasia is eligible for this trial. Some key eligibility criteria include[1]:

  • Having a high or very high-risk blood cancer that requires a stem cell transplant
  • Meeting certain health requirements related to heart, lung, liver, and kidney function
  • Not having had certain other treatments recently, such as another stem cell transplant within the last 6 months
  • Not having active infections or other serious medical conditions

Potential Benefits

While the effectiveness of DOROCUBICEL is still being studied, researchers hope it may offer several potential benefits[1]:

  • Improved survival rates for patients with high-risk blood cancers
  • Faster recovery of blood cell counts after transplant
  • Reduced risk of transplant-related complications
  • Potentially lower risk of graft-versus-host disease (GVHD), a common complication of stem cell transplants

Safety Considerations

As with any experimental treatment, there are potential risks and side effects associated with DOROCUBICEL. The clinical trial is designed to carefully monitor patients for[1]:

  • Graft failure (when the transplanted cells don’t work properly)
  • Infections
  • Graft-versus-host disease (GVHD)
  • Other transplant-related complications

It’s important to note that participating in a clinical trial involves close monitoring by healthcare professionals to ensure patient safety and to gather important data about the treatment’s effectiveness and potential side effects[1].

Aspect Details
Drug Name Dorocubicel (ECT-001-CB)
Trial Phase Phase II
Target Condition High and very high-risk acute leukemia/myelodysplasia
Main Objectives 1. Examine safety and feasibility of infusion
2. Evaluate relapse-free survival at 1 and 2 years post-transplant
Key Eligibility Criteria – High/very high-risk hematologic malignancy
– Availability of suitable cord blood units
– Specific health requirements for heart, lung, liver, and kidney function
Administration Intravenous infusion
Maximum Dose 7,500,000 cells per kg of body weight
Secondary Endpoints – Hematopoietic engraftment kinetics
– Transplant-related mortality
– Incidence of GVHD
– Incidence of infectious complications
– Incidence of pre-engraftment/engraftment syndrome

FAQ

  • What is Dorocubicel? Dorocubicel is an investigational drug that uses expanded cord blood cells for transplantation in patients with high-risk acute leukemia and myelodysplasia. It combines UM171-expanded cord blood cells with non-expanded allogeneic umbilical cord-derived CD34- cells.
  • Who might benefit from Dorocubicel treatment? Patients with high and very high-risk acute leukemia or myelodysplasia who require an allogeneic hematopoietic stem cell transplantation may potentially benefit from Dorocubicel treatment.
  • What is the main goal of the clinical trial studying Dorocubicel? The main objectives of the trial are to examine the safety and feasibility of infusing Dorocubicel in patients with high-risk acute leukemia or myelodysplasia, and to evaluate relapse-free survival at 1 and 2 years post-transplant.
  • How is Dorocubicel administered? Dorocubicel is administered as an intravenous infusion. The maximum daily dose amount is 7,500,000 cells per kilogram of body weight.
  • What are some of the key eligibility criteria for participating in the Dorocubicel clinical trial? Key eligibility criteria include having a high or very high-risk hematologic malignancy, availability of suitable cord blood units for transplantation, and meeting specific health requirements related to heart, lung, liver, and kidney function. Patients must also have a hematopoietic cell transplantation specific comorbidity index (HCT-CI) score within certain limits based on their age and disease status.

Ongoing Clinical Trials on Dorocubicel

  • Study of UM171-expanded cord blood transplantation for patients with high-risk and very high-risk acute leukemia or myelodysplasia

    Not yet recruiting

    2 1 1
    Investigated diseases:
    Investigated drugs:
    The Netherlands

Glossary

  • Acute Leukemia: A fast-growing cancer of the blood and bone marrow that affects the production of normal blood cells.
  • Myelodysplasia: A group of disorders in which the bone marrow does not produce enough healthy blood cells.
  • Allogeneic Hematopoietic Stem Cell Transplantation: A procedure where a patient receives blood-forming stem cells from a genetically similar, but not identical, donor.
  • Cord Blood: Blood collected from the umbilical cord and placenta after birth, which is rich in stem cells.
  • CD34- Cells: A type of cell found in cord blood that can develop into different types of blood cells.
  • Graft Failure: A complication where the transplanted stem cells fail to grow and make new blood cells in the recipient's body.
  • Relapse-Free Survival: The length of time after treatment during which no signs of cancer are found.
  • GVHD (Graft-versus-Host Disease): A condition that can occur after a stem cell transplant when the donor's immune cells attack the recipient's tissues.
  • Hematopoietic Engraftment: The process by which transplanted stem cells start to produce new blood cells in the recipient's body.
  • HLA Match: The degree of similarity between the human leukocyte antigens (HLA) of the donor and recipient, which is important for transplant success.

References

  1. http://clinicaltrials.eu/trial/study-on-um171-expanded-cord-blood-transplant-for-high-risk-acute-leukemia-myelodysplasia-patients-using-dorocubicel-and-allogeneic-umbilical-cord-derived-cd34-cells/