Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants

Study Purpose

The objective of this study is to make T-cell depleted stem cells from a family member who is a half match (haplo-identical) available on an expanded access basis to patients receiving one or two unrelated cord blood transplants who are at a higher risk of not engrafting in a safe amount of time. The purpose of the related stem cells is the give the bone marrow a "jump start" towards recovery. Ultimately, the cord blood cells will grow and permanently rescue the bone marrow.

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Expanded Access
Eligible Ages	N/A - 65 Years
Gender	All

More Inclusion & Exclusion Criteria

Inclusion Criteria:

- Have a consenting related haplo-identical (3/6, 4/6, or 5/6 if DRB1 mismatch) stem cell donor.

- Have one or two available 4, 5, or 6/6 antigen matching unrelated UCB unit(s) that will deliver a total cell dose >3.0 x 10e7 cells/kg.

Patients who do not have a single UCB unit that will deliver the minimum required cell dose, two partially HLA-matched UCB units which together meet the minimum cell dose requirement, can be used for 1 transplant. These units must be HLA-matched minimally at 4 of 6 HLA-A and B (at intermediate resolution by molecular typing) and DRB1 (at high resolution by molecular typing) loci with the patient, and HLA-matched at 3 of 6 HLA- A, B, DRB1 loci with each other (using same resolution of HLA typing as indicated above). There is no limitation on maximum cell dose.

- Have a high risk or refractory malignancy, or non-malignant disorder amenable to stem cell transplantation therapy.

- Meet eligibility requirements for allogeneic transplant per institutional standard practices.

- Have given written informed consent according to FDA guidelines (or consent of parent/legal guardian as applicable).

- Be <65 years of age at the time of study enrollment.

Exclusion Criteria:

- Have a consenting 8/8 or 10/10 allele matched, consenting, related or unrelated hematopoietic stem cell transplant (HSCT) donor.

- Have a life expectancy of less than 3 months.

- Have uncontrolled infections at time of cytoreduction.

Trial Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT01881334
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	Joanne Kurtzberg, MD
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	Joanne Kurtzberg, MD
Principal Investigator Affiliation	Duke University
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Other, Industry
Overall Status	Available
Countries	United States
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies

Additional Details

The primary purpose of the study is to provide expanded access of T-cell depleted haplo-identical stem cells for patients receiving allogeneic transplantation from a related haplo-identical donor and an unrelated, umbilical cord blood (UUCB) unit(s) for the treatment of high risk malignancies and non-malignant disorders. The T-cell depleted haplo-identical stems cells are intended to facilitate early, short-term myeloid engraftment with the primary goal of minimizing early infections and other non-relapse mortality while the UUCB cells engraft as the durable and permanent graft. Patients with high risk or refractory malignancies, or non-malignant disorders amenable to stem cell transplantation therapy but lacking conventional related or unrelated donors will be eligible for this protocol.

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Duke University Medical Center, Durham 4464368, North Carolina 4482348

Status

Available

Address

Duke University Medical Center

Durham 4464368, North Carolina 4482348, 27710

Site Contact

Erin Arbuckle

[email protected]

919-684-3293

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