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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new method to make stem cell transplants safer and more effective for individuals with severe bone marrow diseases like aplastic anemia and myelodysplastic syndrome. Researchers are testing a combination of stem cells from a family member and umbilical cord blood from a donor (known as CordIn or Omidubicel) to evaluate its efficacy. The trial suits individuals aged 4-60 with these conditions who have not responded well to standard treatments. Participants will undergo chemotherapy and a short radiation dose before receiving the donor cells. The process includes a hospital stay and follow-up visits for several months to ensure the treatment's success and safety. As a Phase 1/Phase 2 trial, this study focuses on understanding the treatment's function in people and measuring its effectiveness in an initial, smaller group.

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.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Earlier studies have shown promising safety results for omidubicel. Specifically, research indicates that out of eight patients treated with omidubicel, seven experienced a quick and lasting recovery of their blood counts, suggesting the treatment is generally well-tolerated. Additionally, early results show a high rate of patients remaining disease-free, with 92.3% of those with severe aplastic anemia not experiencing a return of the disease. While these findings are encouraging, the treatment remains in early testing stages, so monitoring for any side effects or adverse reactions is important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about CordIn and Omidubicel for treating aplastic anemia and myelodysplastic syndrome because these treatments use a unique approach involving cryopreserved stem/progenitor cells from umbilical cord blood. Unlike traditional bone marrow transplants, which rely on finding a suitable donor match, CordIn and Omidubicel utilize ex vivo expanded allogeneic umbilical cord blood cells, offering a potentially faster and more accessible treatment option. Additionally, these treatments focus on purified CD133+ cells, which may enhance the effectiveness and recovery time compared to standard care options. This innovative method could lead to better outcomes for patients with these challenging conditions.

What evidence suggests that this trial's treatments could be effective for aplastic anemia and myelodysplastic syndrome?

Research has shown that Omidubicel, also known as CordIn, may effectively treat severe aplastic anemia (SAA) and myelodysplastic syndrome (MDS). In early studies, 7 out of 8 patients who received Omidubicel experienced a quick and lasting recovery of their blood cell counts. The disease-free survival rate after treatment was 92.3%, and the overall survival rate was also 92.3%. This trial will evaluate Omidubicel as a potential option for those needing a stem cell transplant for SAA or MDS. ^{1 3 5 6 7}

Who Is on the Research Team?

Richard W Childs, M.D.

Principal Investigator

National Heart, Lung, and Blood Institute (NHLBI)

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

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]

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]

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]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT03173937

Study Details | NCT03173937 | Unrelated Umbilical Cord ... This research protocol is therefore designed to evaluate the safety and effectiveness of transplantation with ex vivo expanded UCB (CordIn(TM)) to overcome the ...

2. clinicalstudies.info.nih.gov clinicalstudies.info.nih.gov/ProtocolDetails.aspx?id=17-H-0091

Unrelated Umbilical Cord Blood Transplantation for Severe ... Severe aplastic anemia (SAA) and myelodysplastic syndrome (MDS) are bone marrow diseases. People with these diseases usually need a bone marrow transplant.

3. hematologyadvisor.com hematologyadvisor.com/news/omidubicel-gets-priority-review-for-severe-aplastic-anemia/

Omidubicel Gets Priority Review for Severe Aplastic Anemia Initial results showed among the 8 patients (cohort 1: n=3; cohort 2: n=5) treated with omidubicel, 7 patients achieved early and sustained cord ...

4. astctjournal.org astctjournal.org/article/S2666-6367(25)00095-8/abstract

Interim Results of a Phase II Trial of Omidubicel, Ex-Vivo ... The disease-free and overall survival was 92.3%. CMV reactivation occurred in six (43%), with one CMV disease and one PTLD occurring which resolved following ...

5. thecstreet.com thecstreet.com/client-news/gamida-cell-presents-positive-initial-results-on-treating-severe-aplastic-anemia-saa-with-omidubicel/

Gamida Cell Presents Positive Initial Results on Treating ... The disease-free survival rate, as well as overall survival, was 92.3%, comparing favorably with outcomes from patients who receive transplants ...

6. clinicalstudies.info.nih.gov clinicalstudies.info.nih.gov/protocoldetails.aspx?id=17-H-0091&&query=I

Unrelated Umbilical Cord Blood Transplantation for Severe ... Severe aplastic anemia (SAA) and myelodysplastic syndrome (MDS) are bone marrow diseases. People with these diseases usually need a bone marrow transplant.

7. hcplive.com hcplive.com/view/fda-accepts-priority-review-application-of-omidubicel-for-severe-aplastic-anemia

FDA Accepts Priority Review Application of Omidubicel for ... Genetic testing in severe aplastic anemia is required for optimal hematopoietic cell transplant outcomes. Blood. 2022;140(8):909-921. doi ...