Unlicensed Cord Blood Transplant for Blood/Immune System Disorders
Recruiting in Palo Alto (17 mi)
+63 other locations
Overseen byPatricia Shi, MD
Age: Any Age
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 2
Recruiting
Sponsor: New York Blood Center
Disqualifiers: Licensed cord blood, Non-US centers, others
No Placebo Group
Prior Safety Data
Approved in 1 Jurisdiction
Trial Summary
What is the purpose of this trial?This trial is testing the safety of giving patients special blood from umbilical cords. The study focuses on patients who receive these infusions to see if there are any serious side effects. The cord blood has special cells that can help make new blood cells in the body. Umbilical cord blood has been used in the treatment of various diseases for many years, including leukemia, lymphoma, and congenital immunodeficiency.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications.
What data supports the effectiveness of the treatment Unlicensed Cord Blood Units for blood or immune system disorders?
Research shows that umbilical cord blood (UCB) transplantation is a viable alternative for patients needing stem cell transplants, with over 6000 procedures performed worldwide. UCB is noted for its easy availability, low infection risk, and ability to be used even when donor matches are not perfect, making it a promising option for those without matched donors.
12345Is unlicensed cord blood transplantation generally safe for humans?
In Japan, cord blood has been used for many stem cell transplants, and while adverse events have been reported, efforts are being made to improve safety reporting and reduce risks. Additionally, over 1000 successful transplants have been performed worldwide, indicating a level of safety, though some patients experienced complications.
56789How is the Unlicensed Cord Blood Transplant treatment different from other treatments for blood or immune system disorders?
Unlicensed Cord Blood Transplant uses umbilical cord blood as a source of stem cells, which is unique because it allows for rapid availability and greater tolerance of genetic mismatches compared to traditional bone marrow transplants. This treatment is particularly beneficial for patients who cannot find a fully matched donor, especially those of mixed ethnicity, and it can be used safely and effectively under the FDA's Investigational New Drug program.
47101112Eligibility Criteria
This trial is open to people of any age and gender with disorders affecting the blood-making system, whether inherited, acquired or due to intensive treatment. Participants must receive at least one unlicensed cord blood unit (CBU) made by NCBP. It's not for those treated outside the US, receiving manipulated CBUs post-thaw, or exclusively licensed/unlicensed CBUs from other banks.Inclusion Criteria
I have a blood disorder that is inherited, acquired, or caused by treatment.
My age or gender does not limit my participation.
My cord blood transplant uses a product made by the NCBP.
Exclusion Criteria
I am currently receiving treatment with licensed cord blood products.
I am receiving a cord blood treatment that involves special processing.
Patients who are receiving unlicensed cord blood products from other banks
+1 more
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment
Participants receive the investigational cord blood units and are monitored for infusion-related reactions
1 week
1 visit (in-person)
Follow-up
Participants are monitored for safety and effectiveness after treatment, including engraftment evaluation
6 months
Participant Groups
The study is testing the safety of infusing unlicensed investigational cord blood units (CBUs) manufactured by NCBP. The focus is on documenting all problems related to the infusion process to assess potential risks.
1Treatment groups
Experimental Treatment
Group I: unlicensed CBUExperimental Treatment1 Intervention
The Principal Investigators will be the transplant physicians at participating US transplant centers
Unlicensed CBU is already approved in United States for the following indications:
🇺🇸 Approved in United States as Unlicensed IND Cord Blood Units for:
- Unrelated transplantation
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Nationwide Children's HospitalColumbus, OH
Abramson Cancer Center of the University of Pennsylvania Medical CenterPhiladelphia, PA
Texas Children's Cancer CenterHouston, TX
Children's Hospital of MichiganDetroit, MI
More Trial Locations
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Who Is Running the Clinical Trial?
New York Blood CenterLead Sponsor
References
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.
Umbilical cord blood transplantation: a new alternative option. [2016]Allogeneic hematopoietic stem cell transplantation is a life-saving procedure for hematopoietic malignancies, marrow failure syndromes, and hereditary immunodeficiency disorders. However, wide application of this procedure is limited by availability of suitably HLA-matched adult donors. Umbilical cord blood (UCB) has being increasingly used as an alternative hematopoietic stem cell source for these patients. To date, over 6000 UCB transplant procedures in children and adults have been performed worldwide using UCB donors. Broader use of UCB for adult patients is however limited by the available infused cell dose. This has prompted intensive research on ex vivo expansion of UCB stem cells and UCB graft-engineering including accessory cells able to improve UCB engraftment and reconstitution and for tissue regenerative potential. Recently, two large European and North American retrospective studies demonstrated that UCB is an acceptable alternative source of hematopoietic stem cells for adult recipients who lack HLA-matched adult donors. UCB is anticipated to address needs in both transplantation and regenerative medicine fields. It has advantages of easy procurement, no risk to donors, low risk of transmitting infections, immediate availability and immune tolerance allowing successful transplantation despite HLA disparity.
Use of cost-effectiveness analysis to determine inventory size for a national cord blood bank. [2018]Transplantation with stem cells from stored umbilical cord blood units is an alternative to living unrelated bone marrow transplantation. The larger the inventory of stored cord units, the greater the likelihood that transplant candidates will match to a unit, but storing units is costly. The authors present the results of a study, commissioned by the Institute of Medicine, as part of a report on the establishment of a national cord blood bank, examining the optimal inventory level. They emphasize the unique challenges of undertaking cost-effectiveness analysis in this field and the contribution of the analysis to policy.
Optimizing donor selection for public cord blood banking: influence of maternal, infant, and collection characteristics on cord blood unit quality. [2023]Banked unrelated donor umbilical cord blood (CB) has improved access to hematopoietic stem cell transplantation for patients without a suitably matched donor. In a resource-limited environment, ensuring that the public inventory is enriched with high-quality cord blood units (CBUs) addressing the needs of a diverse group of patients is a priority. Identification of donor characteristics correlating with higher CBU quality could guide operational strategies to increase the yield of banked high-quality CBUs.
Impact of cord blood banking technologies on clinical outcome: a Eurocord/Cord Blood Committee (CTIWP), European Society for Blood and Marrow Transplantation and NetCord retrospective analysis. [2022]Techniques for banking cord blood units (CBUs) as source for hematopoietic stem cell transplantation have been developed over the past 20 years, aimed to improve laboratory efficiency without altering the biologic properties of the graft. A large-scale, registry-based assessment of the impact of the banking variables on the clinical outcome is currently missing.
Adverse events caused by cord blood infusion in Japan during a 5-year period. [2023]In Japan, cord blood is used for more than half of all unrelated stem cell transplantations. The public cord blood banks (CBBs) have been collecting information on cord blood transplantation-related adverse events from physicians on a voluntary basis, without common definitions of the adverse reactions. The aims of this study were to compare two classification systems to improve the reporting system and to clarify the actual risk from cord blood infusion, which can then provide the impetus to take appropriate measures to reduce adverse events.
Cord blood unit access and selection: 2010 and beyond: best practices and emerging trends in cord blood unit selection. [2018]One-fifth, more than 1000, of all transplants facilitated in 2010 by the National Marrow Donor Program (NMDP) have employed 1 or 2 umbilical cord blood units as the graft source. As the use of umbilical cord blood for unrelated allogeneic hematopoietic cell transplantation increases, several issues emerge that require additional attention and refinement. The U.S. Food and Drug Administration is now far along in its implementation of regulatory controls for umbilical cord blood. After October 20, 2011, every unrelated-donor cord blood unit transplanted in the United States must be either licensed or covered under an FDA-accepted IND. It is incumbent upon transplant physicians to review and understand the implications of the FDA's new regulations. In addition, as more transplant programs adopt umbilical cord blood for transplantation, it is important to stay current with the best practices surrounding identification and selection of the best available units. Cell dose, HLA matching, location of mismatched loci, and the role of noninherited maternal alleles are all important considerations for unit selection. This complexity in selection of appropriate units raises issues about the desired inventory of umbilical cord blood units. How many units are needed to meet the needs of all patients who might benefit from cord blood transplantation? Newly developed simulation models are being utilized by NMDP to answer this question.
Worldwide survey on key indicators for public cord blood banking technologies: By the World Marrow Donor Association Cord Blood Working Group. [2023]The Cord Blood Working Group of the World Marrow Donor Association created a survey for cord blood banks (CBBs) aimed to identify and understand the main technical procedures currently used by public CBBs worldwide regarding cord blood units (CBUs) available for unrelated hematopoietic stem cell transplantation. These technical procedures include CBU collection, (pre-) processing, packaging, testing, storage, and transport. The survey was an online survey created with SurveyGizmo and was completed individually by each CBB at the end of 2017. The information is valuable to transplant centers, CBBs as well as the global industry of public cord blood banking. In general, we can conclude from this survey that the majority of CBBs are up to standard in terms of CBB technologies. Areas of improvement include accreditation, increase standardization in testing, and setting of total nucleated cells thresholds for acceptance of CBU for public use. Furthermore, there is a need for a consensus in the way CBBs operate in term of reservation and release to facilitate a more straightforward access to the therapy.
Haematopoietic transplant potential of unrelated and related cord blood: the first six years of the EUROCORD/NETCORD Bank Germany. [2023]To date, human umbilical cord blood (CB) has been employed successfully in well over 1000 allogeneic (unrelated and sibling) stem cell transplantations. Because of primary limitations in volume and cell numbers, over 90% of these transplantations were performed in children. Therefore requests for well standardised cord blood units of high quality are now increasing constantly. Examination and standardisation of unrelated and related cord blood stem cell preparations and banking as well as their biological characterisation was already initiated in Düsseldorf in 1992. Hitherto a total of 3236 CB samples with a mean volume of 89 +/- 25 ml, a mean total number of nucleated cells (NC) of 10 +/- 5 x 10(8) and a mean number of CFU-GM of 6 +/- 5 x 10(5) have also been validated by haematological, immunological and microbiological criteria. In addition to that, 97 directed CB donations of siblings with a clinical indication have been characterised and banked along the same lines. All CB units were collected from the umbilical cord vein immediately after vaginal full term delivery or caesarean section, then frozen and stored in liquid nitrogen. 1940 CB units were stored unseparated, the other 1296 were volume reduced using Hetastarch (HES) with a mean recovery of 85 +/- 13% of the nucleated cells, 86 +/- 12% and 84 +/- 13% for CFC and CD34+ cells, respectively. Only 5.0 ml of a CB sample is required for routine laboratory testing as there are HLA-class I typing, HLA-class II typing by sequence specific oligonucleotide probes (PCR-SOP), ABO typing, sterility control, assessment of progenitor and stem cells by colony forming assays, and CD34+ status as well as certain viral infections such as CMV, Hepatitis B, C, HIV, Parvo B19 by PCR technology before releasing the CB unit for transplantation. For apparent viral infections, maternal sera obtained at birth were tested for HBsAg, anti-HBc, anti-HCV, -HAV-(IgG, IgM), -HIV-1-2, -EBV- (IgG, IgM), -HTLVI-II, -CMV (IgM, IgG), toxoplasmosis and syphilis. Within the last three years a total of 4860 preliminary searches and 680 extended unit reports were submitted to the CB bank Düsseldorf by fax or World Wide Web. So far 68 unrelated and 3 related CB units were delivered. From these 70 have been transplanted in 30 different transplant centres world-wide. Until now the evaluation of the first 53 unrelated CB-transplantations was performed together with the EUROCORD transplant registry. Three patients were excluded from the analysis, since they received an unrelated CB-transplant for non-engraftment after previous allotransplants. The median patient age of these 50 patients was 5.0 years (range 0.3-44), the median weight 18 kg (range 4-70 kg). The majority of the patients transplanted for malignancies (66%) suffered from ALL (n = 19), AML (n = 7), CML (n = 4) and lymphoma (n = 2) with two third (75%) in an intermediate (2nd CR) or advanced stage of disease (> 2nd CR); 13 patients had metabolic diseases and immunodeficiencies and three had aplastic anaemia. All CB samples as well as the patients' blood samples were typed in Düsseldorf for HLA-class I by serology confirmed by PCR-SSP and by high resolution DNA typing for HLA-DRB1 and HLA-DQB1 alleles. 96% of the 50 patients receiving unrelated CB were mismatched at one or more HLA-antigens. 41 of the 50 patients transplanted with unrelated CB from Düsseldorf were evaluable for engraftment with an overall engraftment rate of 83%. According to the defined criteria of EUROCORD, 9 of the 50 patients were not evaluable for engraftment, since they died before day 60. The present median follow-up time is 14 months (1.4-38). The Kaplan-Meier estimate of survival at one year is 42 percent. The three paediatric patients after sibling CB transplantations (ALL, amegakaryocytic thrombocytopenia and CML) are alive with a follow-up time of 350, 379 days and 531 days. (ABSTRACT TRUNCATED)
HLA frequency in candidates to transplant without compatible cord blood at the National Center of Blood Transfusion (Mexico). [2018]Umbilical Cord Blood Units (UCBU) for transplantation, are a therapeutic possibility for patients with a wide range of oncohaematological diseases and other immunologic disorders. The search of compatible donors for bone marrow transplantation is increasingly difficult for patients of mixed ethnicity. The aim of this work was determine the HLA frequency of candidates for transplantation without compatible UCBU at the National Center of from Blood Transfusion (NCBT) - Mexico.
Double umbilical cord blood transplantation. [2007]Unrelated umbilical cord blood (UCB) is an alternative donor source for allogeneic haematopoietic cell transplantation and, compared with unrelated donor bone marrow, has the advantages of rapid availability, greater tolerance of HLA disparity and lower incidence of severe graft-versus-host disease. Graft cell dose is an important determinant of haematopoietic recovery and overall outcome following UCB transplantation, and the limited cell dose of single UCB units has been a major barrier to its more widespread use. Transplantation with two unrelated UCB units is feasible, safe and effective and can overcome the limitation of cell dose of single UCB units.
Unlicensed Umbilical Cord Blood Units Provide a Safe and Effective Graft Source for a Diverse Population: A Study of 2456 Umbilical Cord Blood Recipients. [2021]Umbilical cord blood (UCB) transplantation (UCBT) is a curative procedure for patients with hematologic malignancies and genetic disorders and expands access to transplantation for non-Caucasian patients unable to find a fully matched unrelated donor. In 2011, the US Food and Drug Administration required that unrelated UCBT be performed using either licensed UCB or unlicensed UCB under the Investigational New Drug (IND) program. The National Marrow Donor Program manages an IND under which 2456 patients (1499 adults and 957 children, 564 with malignant diseases and 393 with nonmalignant diseases) underwent single or double UCBT between October 2011 and December 2016. The median patient age was 31 years (range, <1 to 81 years), and 50% of children and 36% of adults were non-Caucasian. The median time to neutrophil engraftment (ie, absolute neutrophil count ≥500/mm3) was 22 days for adults, 20 days for pediatric patients with malignant diseases, and 19 days for pediatric patients with nonmalignant diseases, with corresponding rates of engraftment at 42 days of 89%, 88%, and 90%. In these 3 groups of patients, the incidence of acute graft-versus-host disease (GVHD) grade II-IV was 35%, 32%, and 24%; the incidence of chronic GVHD was 24%, 26%, and 24%; and 1-year overall survival (OS) was 57%, 71%, and 79%, respectively. In multivariate analysis, younger age, lower Hematopoietic Cell Transplantation-Specific Comorbidity Index, early-stage chemotherapy-sensitive disease, and higher performance score were predictive of improved OS for adults. In a subset analysis of children with malignancies undergoing single UCBT, the use of either licensed UCB (n = 48) or unlicensed UCB (n = 382) was associated with similar engraftment and survival. The use of unlicensed UCB units is safe and effective and provides an important graft source for a diverse population.