What is the mechanism of action of this treatment?

Aplastic anemia treatments often involve immunosuppressive therapy and hematopoietic stem cell transplantation (HSCT). Immunosuppressive therapy, such as antithymocyte globulin (ATG) with cyclosporine, suppresses the immune system to prevent it from attacking the bone marrow. HSCT, including umbilical cord blood stem cell transplantation, introduces healthy stem cells to regenerate and repair the damaged bone marrow. This is vital for aplastic anemia patients as it restores normal blood cell production, addressing the root cause of the disease and improving patient outcomes.

What side effects are associated with this type of intervention?

The most common treatments for Aplastic Anemia include immunosuppressive therapy and hematopoietic stem cell transplantation. Immunosuppressive therapy, such as antithymocyte globulin (ATG) combined with cyclosporine, can lead to side effects like serum sickness, increased risk of infections, and renal dysfunction. Hematopoietic stem cell transplantation, including umbilical cord blood transplantation, can cause graft-versus-host disease (GVHD), infections, and organ toxicity. Both treatments require careful monitoring to manage these potential side effects.

What prior FDA approvals exist?

The FDA has approved several treatments for Aplastic Anemia, including immunosuppressive therapies such as antithymocyte globulin (ATG) and cyclosporine. These treatments help to suppress the immune system, allowing bone marrow to recover and produce blood cells. Additionally, hematopoietic stem cell transplantation (HSCT) is a key treatment for severe cases, involving the transplantation of stem cells to regenerate and repair damaged bone marrow. Drugs like eltrombopag, a thrombopoietin receptor agonist, have also been approved to stimulate platelet production in patients with Aplastic Anemia.

What other types of research exist?

Promising novel alternatives to umbilical cord blood stem cell transplantation for aplastic anemia patients include: 1) Induced pluripotent stem cells (iPSCs) which can be generated from the patient's own cells and differentiated into hematopoietic stem cells, reducing the risk of graft-versus-host disease. 2) Gene therapy approaches that correct genetic defects in hematopoietic stem cells, allowing for the regeneration of healthy blood cells. 3) Mesenchymal stem cell (MSC) therapy, which has immunomodulatory properties and can support hematopoiesis. These alternatives are currently being explored in clinical trials and have shown potential in preclinical studies.

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Alkylating agents

Cord Blood Transplant for Blood Diseases

Phase 1

Recruiting

Led By Omar Aljitawi, MD

Research Sponsored by University of Rochester

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Performance Status Karnofsky or Lansky score ≥ 70%

Patients must have a disease or syndrome amenable to therapy with hematopoietic stem cell transplantation

Must not have

Autologous HSCT < 6 months prior to proposed UCB transplant

Current uncontrolled infection

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at 30 days, 100 days, 6 months and yearly from the date of transplant until the date of documented graft failure or the subject's death up to 120 months.

Awards & highlights

No Placebo-Only Group

Summary

This trial uses stem cells from a baby's umbilical cord to treat patients who need new healthy stem cells. Patients first get strong medicine to clear out unhealthy cells, then receive the new stem cells, and take medications to prevent complications. Umbilical cord blood has been used in the treatment of various diseases, including leukemias, lymphomas, and immune system disorders.

Who is the study for?

This trial is for patients with various blood diseases, immune disorders, and cancers like leukemia, lymphoma, and solid tumors. Participants need to have a certain level of physical fitness (Karnofsky or Lansky score ≥ 70%) and good heart, lung, kidney, and liver function. They must not be pregnant or breastfeeding, HIV positive, or have had a recent autologous HSCT. A suitable HLA-matched donor should not be available.

What is being tested?

The study tests umbilical cord blood stem cell transplantation using one of four preparative regimens that include Melphalan, Mesna, Fludarabine among others. It aims to validate the transplantation process at the institution conducting the research.

What are the potential side effects?

Potential side effects may include reactions from chemotherapy drugs like nausea and hair loss; organ inflammation due to radiation; increased risk of infections post-transplantation; possible graft-versus-host disease where transplanted cells attack the body.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am mostly able to care for myself but may not be able to do active work.

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My condition can be treated with a stem cell transplant.

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My treatment includes confirmed cord blood products before starting conditioning therapy.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I had a stem cell transplant using my own cells less than 6 months ago.

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I do not have any infections that aren't responding to treatment.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at 30 days, 100 days, 6 months and yearly from the date of transplant until the date of documented graft failure or the subject's death up to 120 months.

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at 30 days, 100 days, 6 months and yearly from the date of transplant until the date of documented graft failure or the subject's death up to 120 months.

This trial's timeline: 3 weeks for screening, Varies for treatment, and at 30 days, 100 days, 6 months and yearly from the date of transplant until the date of documented graft failure or the subject's death up to 120 months. for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Engraftment of ANC and Platelets

Secondary study objectives

Disease-free survival

Incidence of acute graft-versus-host disease

Incidence of chronic graft-versus-host disease

+1 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

4Treatment groups

Experimental Treatment

Group I: Reduced Intensity ChemotherapyExperimental Treatment3 Interventions

Reduced Intensity Chemotherapy Fludarabine 30 mg/m2/day x 5 doses days -6 to -2 Melphalan 140 mg/m2/day x 1 dose day -2 Cord Blood Infusion Other names: Flu/Mel

Group II: Non-Myeloablative ConditioningExperimental Treatment4 Interventions

Fludarabine 40 mg/m2/day x 5 doses days -6 to -2 Cyclophosphamide 50 mg/kg/day x 1 dose day -6 Mesna 50 mg/kg/day with 20% loading dose with Cyclophosphamide dose followed by continuous infusion over 24 hours x 1 dose \[to be completed 24 hours after Cyclophosphamide dose\] Total Body Irradiation 200 cGy in a single fraction day -1 Cord Blood Infusion Other names: Flu/Cy/TBI

Group III: Full Intensity, TBI-based ConditioningExperimental Treatment4 Interventions

Full Intensity TBI-based Conditioning Total Body Irradiation 1200 cGy in fractions of 150 cGy days -8 or -7 to -4 Cyclophosphamide 60 mg/kg/day x 2 doses days -3 and -2 Mesna 60 mg/kg/day with 20% loading dose with first Cyclophosphamide followed by continuous infusion over 24 hours x 2 doses \[to be completed 24 hours after final Cyclophosphamide dose\] followed by Cord Blood Infusion Other names: TBI/Cy

Group IV: Full Intensity, Chemo-based ConditioningExperimental Treatment4 Interventions

Full Intensity, Chemotherapy Conditioning Busulfan days -7 to -4 Recipients \<5 years - 1 mg/kg/dose x 16 doses every 6 hours Recipients \>/= 5 years - 0.8 mg/kg/dose x 16 doses every 6 hours Cyclophosphamide 60 mg/kg/day x 2 doses days -3 and -2 Mesna 60 mg/kg/day with 20% loading dose with first Cyclophosphamide followed by continuous infusion over 24 hours x 2 doses \[to be completed 24 hours after final Cyclophosphamide dose\] followed by Cord Blood Infusion Other names: Bu/Cy

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Mesna

2003

Completed Phase 2

~1380

Cord Blood Infusion

2012

Completed Phase 2

~320

Busulfan

2008

Completed Phase 4

~1710

Fludarabine

2012

Completed Phase 4

~1860

Melphalan

2008

Completed Phase 3

~1500

Total Body Irradiation 1200 cGy

2015

Completed Phase 1

~80

Cyclophosphamide

2010

Completed Phase 4

~2310

0 / 5Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Aplastic anemia treatments often involve immunosuppressive therapy and hematopoietic stem cell transplantation (HSCT). Immunosuppressive therapy, such as antithymocyte globulin (ATG) with cyclosporine, suppresses the immune system to prevent it from attacking the bone marrow. HSCT, including umbilical cord blood stem cell transplantation, introduces healthy stem cells to regenerate and repair the damaged bone marrow. This is vital for aplastic anemia patients as it restores normal blood cell production, addressing the root cause of the disease and improving patient outcomes.

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