Umbilical Cord Blood Transplant for Aplastic Anemia and Myelodysplastic Syndrome

Overseen By Jennifer A Farren

Age: Any Age

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: National Heart, Lung, and Blood Institute (NHLBI)

Disqualifiers: HIV, Pregnancy, Fanconi anemia, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

37 Participants Needed

Trial Summary

What is the purpose of this trial?

Background: Severe aplastic anemia (SAA) and myelodysplastic syndrome (MDS) are bone marrow diseases. People with these diseases usually need a bone marrow transplant. Researchers are testing ways to make stem cell transplant safer and more effective. Objective: To test if treating people with SAA or MDS with a co-infusion of blood stem cells from a family member and cord blood stem cells from an unrelated donor is safe and effective. Eligibility: Recipients ages 4-60 with SAA or MDS Donors ages 4-75 Design: Recipients will be screened with: * Blood, lung, and heart tests * Bone marrow biopsy * CT scan Recipients will have an IV line placed into a vein in the neck. Starting 11 days before the transplant they will have several chemotherapy infusions and 1 30-minute radiation dose. Recipients will get the donor cells through the IV line. They will stay in the hospital 3-4 weeks. After discharge, they will have visits: * First 3-4 months: 1-2 times weekly * Then every 6 months for 5 years Donors will be screened with: * Physical exam * Medical history * Blood tests Donors veins will be checked for suitability for stem cell collection. They may need an IV line to be placed in a thigh vein. Donors will get Filgrastim or biosimilar (G-CSF) injections daily for 5-7 days. On the last day, they will have apheresis: Blood drawn from one arm or leg runs through a machine and into the other arm or leg. This may be repeated 2 days or 2-4 weeks later.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

How is the treatment Omidubicel unique for aplastic anemia and myelodysplastic syndrome?

Omidubicel is unique because it is an ex vivo expanded stem cell product derived from umbilical cord blood, which allows for faster engraftment and fewer infections compared to standard umbilical cord blood transplants. This makes it a promising alternative for patients who lack a matched donor, offering improved immune recovery and reduced infectious complications. ^{1 2 3 4 5}

Research Team

Richard W Childs, M.D.

Principal Investigator

National Heart, Lung, and Blood Institute (NHLBI)

Eligibility Criteria

This trial is for people aged 4-60 with severe aplastic anemia or myelodysplastic syndrome (MDS) who haven't responded to standard treatments. They need a matching cord blood unit and must understand the study's nature. Excluded are those with certain heart, kidney, liver conditions, active infections, other cancers within 5 years, specific allergies, HIV positive status or if they're pregnant.

Inclusion Criteria

My cord blood unit was processed and stored according to required standards.

My cord blood unit meets specific cell count criteria and has approval from Gamida Cell.

I understand this study is experimental and I (or my guardian) can consent.

Exclusion Criteria

HIV positive

Current pregnancy, or unwillingness to take oral contraceptives or use a barrier method of birth control or practice abstinence to refrain from pregnancy, if of childbearing potential for one year

My lung's ability to transfer gas is less than 40% of what's expected.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Multiple visits for blood, lung, heart tests, bone marrow biopsy, and CT scan

Conditioning

Participants receive chemotherapy infusions and a radiation dose to prepare for transplantation

11 days

Inpatient

Transplantation

Participants receive the donor cells through an IV line

1 day

Inpatient

Hospital Stay

Participants remain in the hospital for monitoring and recovery post-transplant

3-4 weeks

Inpatient

Follow-up

Participants are monitored for safety and effectiveness after treatment

5 years

1-2 times weekly for the first 3-4 months, then every 6 months

Treatment Details

Interventions

• CordIn
• Omidubicel (former CordIn)
• Omidubicel (formerly Nicord (Registered Trademark) and Cordin (TM))
• Unrelated Umbilical Cord Blood Transplantation

Trial Overview The trial tests a new treatment method using Omidubicel (formerly CordIn), which involves co-infusing stem cells from both a family member and unrelated donor cord blood to improve bone marrow transplant outcomes in patients with SAA or MDS.

Participant Groups

Group I: 1 Experimental Treatment 2 Interventions

CordIn is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells.

Who Is Running the Clinical Trial?

National Heart, Lung, and Blood Institute (NHLBI)

Lead Sponsor

Trials

3,987

Recruited

47,860,000+

Findings from Research

Omidubicel, an ex vivo-expanded umbilical cord blood therapy, shows promising long-term outcomes with a 3-year overall survival rate of 62.5% and disease-free survival rate of 54.0% in 105 patients with hematologic malignancies or sickle cell disease, based on a pooled analysis of 5 multicenter trials.

The therapy demonstrated stable blood cell production and immune function over a median follow-up of 22 months, with normal immune cell counts maintained for up to 8 years, indicating its potential for durable engraftment and safety in long-term use.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Multicenter Long-Term Follow-Up of Allogeneic Hematopoietic Cell Transplantation with Omidubicel: A Pooled Analysis of Five Prospective Clinical Trials. Lin, C., Schwarzbach, A., Sanz, J., et al.[2023]

Omidubicel, a modified stem cell graft from cord blood, was approved in April 2023 for treating adults and children aged 12 and older with hematological malignancies, aiming to improve infection rates and speed up neutrophil recovery after transplantation.

The approval of omidubicel marks a significant milestone in its development, highlighting its potential as a treatment option for patients undergoing cord blood transplantation following myeloablative conditioning.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Omidubicel: First Approval. Heo, YA.[2023]

In a phase 3 trial involving 125 patients aged 13 to 65 with hematologic malignancies, omidubicel led to significantly faster neutrophil engraftment (12 days) compared to standard umbilical cord blood transplantation (22 days), indicating improved efficacy.

Patients receiving omidubicel also experienced quicker platelet recovery, fewer severe infections, and spent more time out of the hospital in the first 100 days post-transplant, suggesting it may reduce early transplant-related complications.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Omidubicel vs standard myeloablative umbilical cord blood transplantation: results of a phase 3 randomized study. Horwitz, ME., Stiff, PJ., Cutler, C., et al.[2023]

Umbilical cord blood transplantation (CBT) is a crucial alternative for patients without matched donors, but traditional UCB use is limited by lower cell doses leading to delayed engraftment and higher infection risks.

Omidubicel, an ex vivo expanded cord blood product, has received FDA approval for its ability to enhance neutrophil recovery and reduce infection risks, showing promise for improving outcomes in CBT.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A new beginning: can omidubicel emerge as the next, viable alternative donor source? Gandhi, AP., Newell, LF., Maziarz, RT.[2023]

In a phase I/II trial involving 36 patients aged 13 to 63, omidubicel (nicotinamide-expanded cord blood) demonstrated a 90% success rate in achieving adequate CD4+ T-cell immune reconstitution within 100 days post-transplant, indicating its efficacy as an alternative source for hematopoietic cell transplantation.

The study found that omidubicel recipients experienced rapid and complete immune reconstitution, including T-cells, natural killer cells, B-cells, and monocytes, comparable to traditional graft sources, suggesting it could be a viable option for patients lacking HLA-identical donors.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Lymphoid and myeloid immune cell reconstitution after nicotinamide-expanded cord blood transplantation. de Koning, C., Tao, W., Lacna, A., et al.[2023]

References

1. United States pubmed.ncbi.nlm.nih.gov

Multicenter Long-Term Follow-Up of Allogeneic Hematopoietic Cell Transplantation with Omidubicel: A Pooled Analysis of Five Prospective Clinical Trials. [2023]

2. New Zealand pubmed.ncbi.nlm.nih.gov

Omidubicel: First Approval. [2023]

3. United States pubmed.ncbi.nlm.nih.gov

Omidubicel vs standard myeloablative umbilical cord blood transplantation: results of a phase 3 randomized study. [2023]

4. England pubmed.ncbi.nlm.nih.gov

A new beginning: can omidubicel emerge as the next, viable alternative donor source? [2023]

5. England pubmed.ncbi.nlm.nih.gov

Lymphoid and myeloid immune cell reconstitution after nicotinamide-expanded cord blood transplantation. [2023]