What side effects are associated with this type of intervention?

Common treatments for Bone Marrow Failure Syndrome, such as allogeneic stem cell transplantation with CD34+ selection, aim to reduce graft versus host disease (GVHD) and improve engraftment. Known side effects include acute and chronic GVHD, which can affect multiple organs and systems, leading to symptoms like skin rashes, liver dysfunction, and gastrointestinal issues. Other potential side effects are primary and secondary graft rejection, increased risk of infections (bacterial, viral, fungal), and delayed immune reconstitution. Additionally, conditioning regimens used before transplantation can cause hematologic, gastrointestinal, hepatic, and pulmonary toxicities.

What prior FDA approvals exist?

FDA-approved treatments for Bone Marrow Failure Syndrome include various forms of hematopoietic stem cell transplantation (HSCT), which aim to restore bone marrow function. One notable approach is the use of CD34+ stem cell selection to enrich stem cells and reduce the risk of graft-versus-host disease (GVHD) while improving engraftment. Additionally, supportive therapies such as granulocyte-colony stimulating factor (G-CSF) and thrombopoietin receptor agonists like romiplostim have been approved to manage complications such as neutropenia and thrombocytopenia, respectively.

What other types of research exist?

Promising novel alternatives to CD34+ selection for Bone Marrow Failure Syndrome patients include: 1) Thrombopoietin mimetics to enhance platelet production and support engraftment. 2) Reduced-intensity conditioning regimens to minimize toxicity while maintaining efficacy. 3) Use of alternative donor sources such as haploidentical donors or umbilical cord blood, which have shown comparable outcomes to matched sibling donors. 4) Immunotherapeutic approaches like CAR-T cell therapy to target specific immune pathways and reduce GVHD. 5) Genetic modifications of donor cells to enhance engraftment and reduce complications.

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Stem Cell Therapy

CD34+ Stem Cell Selection for Bone Marrow Failure Syndromes

Phase 1 & 2

Waitlist Available

Led By Diane George, MD

Research Sponsored by Diane George

Eligibility Criteria Checklist

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

Must have

Patient must be ≤ 40 years of age. Patients with sickle cell anemia must be at least 2 years of age.

Requirement for CD34+ stem cell selection for a second infusion of stem cells following an allogeneic stem cell transplant from a related or unrelated adult donor.

Must not have

Patients with sickle cell anemia: Patients with bridging fibrosis or cirrhosis of the liver, uncontrolled bacterial, viral or fungal infection in the past month, seropositivity for HIV, and patients who have received prior hematocrit (HCT) within three months of enrollment for reduced intensity regimen and within six months for myeloablative regimen/reduced toxicity regimens.

Patients with documented uncontrolled infection at the time of study entry are not eligible.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial uses a special machine to filter out harmful cells from donor blood to make stem cell transplants safer for young patients with non-cancerous diseases. By removing specific cells, it aims to prevent a serious immune reaction.

Who is the study for?

This trial is for children, adolescents, and young adults with various non-malignant diseases like sickle cell anemia or immune deficiencies. Participants must be under 40 years old, have a matched family or unrelated adult donor for stem cells, and good organ function. Pregnant women and those with uncontrolled infections or certain complications from sickle cell anemia are excluded.

What is being tested?

The study tests CD34 Stem Cell Selection Therapy in patients receiving allogeneic peripheral blood stem cell transplants. It aims to assess the treatment's impact on graft versus host disease, graft rejection, survival rates, immune system recovery timeframes, and infection incidences post-transplant.

What are the potential side effects?

While specific side effects aren't listed here, similar procedures may include risks of infection due to weakened immunity post-transplantation; reactions at the infusion site; potential organ inflammation; delayed marrow engraftment leading to prolonged hospitalization; and possible development of new autoimmune disorders.

Eligibility Criteria

Inclusion Criteria

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

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I am 40 years old or younger. If I have sickle cell anemia, I am at least 2 years old.

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I need a second stem cell transplant from a donor.

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I have a matched unrelated donor for my treatment.

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My family donor matches me at least 50% for the transplant.

Exclusion Criteria

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

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I have sickle cell anemia but no recent severe infections, HIV, or recent HCT.

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I do not have any untreated infections.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 5 years for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Incidence of acute graft versus host disease (GVHD)

Secondary study objectives

Incidence of infectious complications

Incidence of primary graft failure

Incidence of secondary graft failure

+3 more

Awards & Highlights

No Placebo-Only Group

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

Trial Design

1Treatment groups

Experimental Treatment

Group I: CliniMACS PLUS followed by chemotherapyExperimental Treatment1 Intervention

Patients will receive a pre-transplant conditioning regimen of Busulfan Fludarabine and Alemtuzumab. For patients with pre-transplant hepatic dysfunction, Melphalan will be substituted for the Busulfan. For patients receiving a second transplant or a "boost", pre-transplant conditioning based on the clinical condition of the patient will be determined by the Principal Investigator and the patient's bone marrow transplantation (BMT) physician. The donor peripheral blood stem cells will undergo CD34+ selection (Biological/Vaccine: CD34 Stem Cell Selection Therapy). The CliniMACS (PLUS) Reagent System will be used to remove T-cells from the peripheral blood stem cell transplant in order to decrease the risk of acute and chronic graft versus host disease (GVHD).

0 / 6Eligibility 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

The most common treatments for Bone Marrow Failure Syndrome (BMFS) include hematopoietic stem cell transplantation (HSCT), immunosuppressive therapy, and growth factor administration. HSCT, particularly with CD34+ stem cell selection, works by enriching the graft with stem cells that can repopulate the bone marrow, thereby reducing the risk of GVHD and improving engraftment. Immunosuppressive therapy helps to control the immune system's attack on bone marrow cells, while growth factors stimulate the production of blood cells. These mechanisms are crucial for BMFS patients as they aim to restore normal bone marrow function, reduce complications, and improve overall survival and quality of life.

Bone-marrow-derived cells for enhancing collateral development: mechanisms, animal data, and initial clinical experiences.