Cord Blood Transplant for Blood Cancers and Diseases
Recruiting in Palo Alto (17 mi)
Overseen byRichard W Childs, M.D.
Age: Any Age
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 1 & 2
Recruiting
Sponsor: National Heart, Lung, and Blood Institute (NHLBI)
Disqualifiers: Licensed CBUs, International centers, Other IND protocol, others
No Placebo Group
Approved in 1 Jurisdiction
Trial Summary
What is the purpose of this trial?Background:
- Cord blood transplants can treat cancers and other diseases in children and adults. The U.S. Food and Drug Administration (FDA) requires cord blood to be collected and stored under certain safety standards. However, most available cord blood units were collected before the FDA set these standards. These units may not meet FDA standards, but they do meet similar standards set and followed by the National Marrow Donor Program (NMDP). Cord blood units that do not meet the new FDA standards may be used for transplants only as part of a research study. Doctors want to allow people who need transplants to receive cord blood that meets NMDP standards but may not meet FDA standards.
Objectives:
- To allow selected cord blood units that do not meet current FDA standards to be used for transplant.
Eligibility:
- Individuals who need cord blood units for transplant, and who best match cord blood units that are not FDA-licensed.
Design:
Participants will provide consent to receive cord blood that meets NMDP standards but may not meet FDA standards.
Participants will remain on the study for observation for up to 1 year after transplant, or until they withdraw from the study for personal or medical reasons....
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
What data supports the effectiveness of the treatment Cord Blood Transplant for Blood Cancers and Diseases?
Research shows that omidubicel, a treatment derived from umbilical cord blood, leads to faster recovery of blood cells, fewer infections, and shorter hospital stays compared to standard cord blood transplants. It has also been associated with better long-term survival rates and improved quality of life for patients with blood cancers.
12345Is cord blood transplant generally safe for humans?
Cord blood transplant using omidubicel has shown to be generally safe in humans, with faster recovery and fewer infections compared to standard cord blood transplants. Long-term follow-up studies indicate stable blood cell production and immune function, with low rates of complications like secondary graft failure.
13456How is the cord blood transplant treatment Omidubicel different from other treatments for blood cancers and diseases?
Omidubicel is unique because it is a modified stem cell product derived from umbilical cord blood that has been expanded outside the body to increase the number of stem cells. This results in faster recovery of blood cells and fewer infections compared to traditional cord blood transplants, making it a promising option for patients without a matched donor.
13478Eligibility Criteria
This trial is for pediatric and adult patients of any age with blood cancers or diseases affecting the bone marrow, like Myelodysplastic Syndrome or Aplastic Anemia. Participants must need a cord blood transplant and match with unlicensed cord blood units that meet NMDP standards but not current FDA standards.Inclusion Criteria
I have a blood disorder that is inherited, acquired, or caused by treatment.
I am of any age.
Signed informed consent (and signed assent, if applicable) obtained prior to study enrollment
Exclusion Criteria
I am only receiving treatments approved by health authorities.
Patients whose selected unlicensed CBU(s) will be more than minimally manipulated
Cord blood transplant recipients at international transplant centers
+1 more
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Pre-transplant Conditioning
Pre-transplant conditioning and GVHD prophylaxis as per each transplant center's specification
2-3 weeks
Transplantation
Participants receive cord blood transplant with unlicensed CBU
1 day
Post-transplant Monitoring
Monitoring for neutrophil recovery, graft rejection, infection transmission, and GVHD
100 days to 1 year
Follow-up
Participants are monitored for safety and effectiveness after treatment
1 year
Participant Groups
The study is testing the use of unlicensed preserved cord blood units for transplants in individuals who require them. These cord blood units comply with National Marrow Donor Program standards but may fall short of newer FDA requirements.
1Treatment groups
Experimental Treatment
Group I: 1Experimental Treatment1 Intervention
cord blood transplant with unlicensed CBU
Cord Blood Transplant is already approved in United States for the following indications:
🇺🇸 Approved in United States as Omisirge for:
- Hematologic malignancies scheduled for umbilical cord transplantation following myeloablative conditioning
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
National Institutes of Health Clinical Center, 9000 Rockville PikeBethesda, MD
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Who Is Running the Clinical Trial?
National Heart, Lung, and Blood Institute (NHLBI)Lead Sponsor
References
Omidubicel: First Approval. [2023]Omidubicel (omidubicel-onlv; Omisirge®) is a nicotinamide-modified stem cell graft derived from cord blood that is being developed by Gamida Cell for the treatment of haematological malignancies and haemoglobinopathies. In April 2023, omidubicel received its first approval in the USA for use in adults and children aged ≥ 12 years with haematological malignancies who are planned for cord blood transplantation following myeloablative conditioning to reduce the incidence of infection and the time to neutrophil recovery. This article summarizes the milestones in the development of omidubicel leading to this first approval.
Hospitalization and Healthcare Resource Utilization of Omidubicel-Onlv versus Umbilical Cord Blood Transplantation for Hematologic Malignancies: Secondary Analysis from a Pivotal Phase 3 Clinical Trial. [2023]A phase 3 trial (ClincialTrials.gov identifier NCT02730299) of omidubicel-onlv, a nicotinamide-modified allogeneic hematopoietic progenitor cell therapy manufactured from a single umbilical cord blood (UCB) unit, showed faster hematopoietic recovery, reduced rate of infections, and shorter hospital stay compared with patients randomized to standard UCB. This prospective secondary analysis of the phase 3 trial characterized resource utilization in the first 100 days post-transplantation with omidubicel-onlv compared with UCB. This analysis examined resource utilization, including hospital length of stay, hospital care setting, visits by provider type, rate of transfusions, and readmissions, among the 108 treated patients (omidubicel-onlv, n = 52; UCB, n = 56) from day 0 to day 100 post-transplantation. Demographics were generally balanced between the 2 arms, except a higher proportion of females (52% versus 37%) and older median age (40 years versus 36 years) were noted in the omidubicel-onlv arm. Compared with patients receiving UCB transplantation, patients receiving omidubicel-onlv had a shorter average total hospital length of stay (mean, 41.2 days versus 50.8 days; P = .027) in the first 100 days post-transplantation and more days alive and out of the hospital (mean, 55.8 days versus 43.7 days; P = .023). Fewer patients died in the omidubicel-onlv arm compared with the UCB arm (12% vs 16%) before day 100 post-transplantation. During primary hospitalization (ie, time from transplantation to discharge), fewer patients receiving omidubicel-onlv required intensive care unit (ICU) admission (10% versus 23%) and spent fewer days in the ICU (mean, .4 day versus 4.7 days; P = .028) and transplant unit (mean, 25.3 days versus 32.9 days; P = .022) compared with those receiving UCB. Patients receiving omidubicel-onlv required fewer outpatient consultant and nonconsultant visits and fewer platelet or other transfusions within 100 days from transplantation. Our findings suggest that faster hematopoietic recovery in omidubicel-onlv patients is associated with significantly shorter hospital stay and reduced healthcare resource use compared with UCB.
A new beginning: can omidubicel emerge as the next, viable alternative donor source? [2023]Umbilical cord blood (UCB) transplantation (CBT) has been an important alternative donor option for patients lacking matched related donor (MRD) or unrelated donor (URD) grafts. Only 30% of patients with high-risk hematologic malignancies have a human leukocyte antigen (HLA)-identical sibling; subjects without a MRD option are referred for HLA-matched URD selection, or utilize alternative donor sources such as HLA-mismatched URD, UCB, or haploidentical donor grafts. While CBT demonstrates an excellent graft-versus-leukemia (GVL) effect, use of UCB as a graft source is limited due to a lower cell dose that can result in delayed engraftment and an immature immune system with increased infectious risk as a consequence. Together, increased transplant related mortality (TRM) has been associated with UCB allografts. Omidubicel is an ex vivo expanded single cord blood product that has demonstrated rapid engraftment, improved immune reconstitution, and reduced infectious complications in clinical trials. Omidubicel has now been granted U.S. Food & Drug Administration approval to enhance neutrophil recovery and decrease infectious risk. This review will focus on CBT, benefits and barriers to using this alternative donor source, and finally the potential advancements with incorporation of omidubicel in the transplant setting for malignant and non-malignant diseases.
Multicenter Long-Term Follow-Up of Allogeneic Hematopoietic Cell Transplantation with Omidubicel: A Pooled Analysis of Five Prospective Clinical Trials. [2023]Omidubicel is an umbilical cord blood (UCB)-derived ex vivo-expanded cellular therapy product that has demonstrated faster engraftment and fewer infections compared with unmanipulated UCB in allogeneic hematopoietic cell transplantation. Although the early benefits of omidubicel have been established, long-term outcomes remain unknown. We report on a planned pooled analysis of 5 multicenter clinical trials including 105 patients with hematologic malignancies or sickle cell hemoglobinopathy who underwent omidubicel transplantation at 26 academic transplantation centers worldwide. With a median follow-up of 22 months (range, .3 to 122 months), the 3-year estimated overall survival and disease-free survival were 62.5% and 54.0%, respectively. With up to 10 years of follow-up, omidubicel showed durable trilineage hematopoiesis. Serial quantitative assessments of CD3+, CD4+, CD8+, CD19+, CD116+CD56+, and CD123+ immune subsets revealed median counts remaining within normal ranges through up to 8 years of follow-up. Secondary graft failure occurred in 5 patients (5%) in the first year, with no late cases reported. One case of donor-derived myeloid neoplasm was reported at 40 months post-transplantation. This was also observed in a control arm patient who received only unmanipulated UCB. Overall, omidubicel demonstrated stable trilineage hematopoiesis, immune competence, and graft durability in extended follow-up.
Health-Related Quality of Life Following Allogeneic Hematopoietic Cell Transplantation with Omidubicel versus Umbilical Cord Blood. [2023]Omidubicel is an advanced cell therapy derived from umbilical cord blood (UCB) for use in allogeneic hematopoietic cell transplantation (HCT). A recent randomized phase 3 clinical trial demonstrated faster engraftment, shorter length of hospital stays, and lower rates of infection with omidubicel compared with standard UCB transplantation in patients with high-risk hematologic malignancies. Despite the proven clinical benefits of omidubicel, its impact on health-related quality of life (HRQL) from the patient's perspective has not been described. This study analyzed patient-reported HRQL measures collected prospectively in the randomized phase 3 trial comparing omidubicel to standard UCB transplantation. A total of 108 patients at 33 international stem cell transplantation centers underwent myeloablative allogeneic HCT with either omidubicel or standard UCB. Patients completed serial HRQL questionnaires at screening and on days 42, 100, 180, and 365 post-transplantation. The HRQL surveys included the Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT), a 50-item cancer-specific questionnaire assessing physical, functional, emotional, social/family, and HCT-specific well-being, and the EuroQol 5-Dimension 3-Level, a 5-item generic HRQL survey. A mixed model with repeated measures was used to compare changes in HRQL from baseline in the 2 treatment arms. The average change in HRQL scores over time was compared by estimating the difference in the area under the curve (AUC) in each treatment group. Seventy-five patients (omidubicel arm, n = 37; standard UCB arm, n = 38) who completed the FACT-BMT at baseline and on 1 or more follow-up visits were included in this study. Baseline characteristics were similar in the 2 treatment arms. Over the first year post-transplantation, the AUCs of mean changes in physical, functional, and total FACT-BMT scores indicated significantly better HRQL with omidubicel (P
Omidubicel vs standard myeloablative umbilical cord blood transplantation: results of a phase 3 randomized study. [2023]Omidubicel is an ex vivo expanded hematopoietic progenitor cell and nonexpanded myeloid and lymphoid cell product derived from a single umbilical cord blood unit. We report results of a phase 3 trial to evaluate the efficacy of omidubicel compared with standard umbilical cord blood transplantation (UCBT). Between January 2017 and January 2020, 125 patients age 13 to 65 years with hematologic malignancies were randomly assigned to omidubicel vs standard UCBT. Patients received myeloablative conditioning and prophylaxis with a calcineurin inhibitor and mycophenolate mofetil for graft-versus-host disease (GVHD). The primary end point was time to neutrophil engraftment. The treatment arms were well balanced and racially diverse. Median time to neutrophil engraftment was 12 days (95% confidence interval [CI], 10-14 days) for the omidubicel arm and 22 days (95% CI, 19-25 days) for the control arm (P
Banking and transplantation of umbilical cord blood in Guangzhou, China. [2008]Umbilical cord blood (UCB) is an alternative source of hematopoietic stem cells (HSC) for transplantation of patients with hematologic malignancies or hereditary diseases.
Past, present, and future efforts to enhance the efficacy of cord blood hematopoietic cell transplantation. [2020]Cord blood (CB) has been used as a viable source of hematopoietic stem cells (HSCs) and hematopoietic progenitor cells (HPCs) in over 35,000 clinical hematopoietic cell transplantation (HCT) efforts to treat the same variety of malignant and non-malignant disorders treated by bone marrow (BM) and mobilized peripheral blood (mPB) using HLA-matched or partially HLA-disparate related or unrelated donor cells for adult and children recipients. This review documents the beginning of this clinical effort that started in the 1980's, the pros and cons of CB HCT compared to BM and mPB HCT, and recent experimental and clinical efforts to enhance the efficacy of CB HCT. These efforts include means for increasing HSC numbers in single CB collections, expanding functional HSCs ex vivo, and improving CB HSC homing and engraftment, all with the goal of clinical translation. Concluding remarks highlight the need for phase I/II clinical trials to test the experimental procedures that are described, either alone or in combination.