Unlicensed Cord Blood Transplant for Blood/Immune System Disorders
What You Need to Know Before You Apply
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.
Is 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.12345
How 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.36789
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.27101112
Who Is on the Research Team?
Patricia Shi, MD
Principal Investigator
NY Blood Center
Are You a Good Fit for This Trial?
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
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive the investigational cord blood units and are monitored for infusion-related reactions
Follow-up
Participants are monitored for safety and effectiveness after treatment, including engraftment evaluation
What Are the Treatments Tested in This Trial?
Interventions
- Unlicensed CBU
Unlicensed CBU is already approved in United States for the following indications:
- Unrelated transplantation
Find a Clinic Near You
Who Is Running the Clinical Trial?
New York Blood Center
Lead Sponsor