Hematologic MalignanciesNCT01881334
Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants
A study testing CliniMACS CD34 Reagent System in people with Hematologic Malignancies and Inborn Errors of Metabolism Disorders, run by Joanne Kurtzberg, MD at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages Up to 65 years
- Conditions the study is for
- Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies
- What is being tested?
- CliniMACS CD34 Reagent System
- How many people
- Not given
- Where
- 1 siteUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 65 years
- All sexes
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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 locations and contacts
1 site in 1 country.
United States1 site
- Available
Duke University Medical Center
Durham, North Carolina, 27710
Erin Arbuckle, erin.arbuckle@duke.edu
Joanne Kurtzberg, MD
About this study
Official title: A Compassionate Release Protocol: Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Allogeneic Transplantation Using a Related Haplo-Identical Donor and Unrelated, Umbilical Cord Blood Donor(s) for the Treatment of High Risk Malignancies or Non-Malignant Disorders Requiring Allogeneic Transplantation
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 to give the bone marrow a "jump start" towards recovery. Ultimately, the cord blood cells will grow and permanently rescue the bone marrow.
Read the detailed description
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.
- Being tested
- CliniMACS CD34 Reagent System
- Keywords
- Haploidentical Donor, T-cell depleted Stem Cells, Allogeneic Transplant, Umbilical Cord Blood Donor, High Risk Malignancies, Metabolic Disorders, Immune Deficiency, Acute Lymphoblastic Leukemia, Acute Myelogenous Leukemia, Myelodysplastic Syndrome, ALL, AML, MDS, CGD, SCID, Adrenoleukodystrophy, Metachromaticleukodystrophy, Krabbe, PMD, Hunter's
- Medical subject headings
- Hematologic Neoplasms, Metabolic Diseases, Immunologic Deficiency Syndromes, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Adrenoleukodystrophy, Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CliniMACS CD34 Reagent SystemBiological
The CliniMACS CD34 Reagent System is a medical device that is used in vitro to select and enrich CD34+ cells from heterogeneous hematologic cell populations for transplantation in cases where this is clinically indicated.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jun 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematologic MalignanciesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Joanne Kurtzberg, MD
- Sponsor type
- University, hospital or other
- Collaborators
- Duke University, Miltenyi Biotec, Inc.
- Responsible party
- Sponsor-investigator, Joanne Kurtzberg, MD, Duke University
- Investigators
- Joanne Kurtzberg, MD, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01881334
- Sponsor's protocol ID
- Pro00045700
- Status last verified
- Nov 2025
- First submitted
- 17 Jun 2013
- Last updated
- 20 Nov 2025
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Compassionate Release Protocol: Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Allogeneic Transplantation Using a Related Haplo-Identical Donor and Unrelated, Umbilical Cord Blood Donor(s) for the Treatment of High Risk Malignancies or Non-Malignant Disorders Requiring Allogeneic Transplantation
- ClinicalTrials.gov ID
- NCT01881334
- Acronym / sponsor ID
- Pro00045700
- Keywords
- Haploidentical Donor, T-cell depleted Stem Cells, Allogeneic Transplant, Umbilical Cord Blood Donor, High Risk Malignancies, Metabolic Disorders, Immune Deficiency, Acute Lymphoblastic Leukemia
- Study type
- Expanded access
- Recruitment status
- Available
- Lead sponsor
- Joanne Kurtzberg, MDUniversity, hospital or other
- Collaborators
- Duke University, Miltenyi Biotec, Inc.
- First posted
- 19 Jun 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 to give the bone marrow a "jump start" towards recovery. Ultimately, the cord blood cells will grow and permanently rescue the bone marrow.
Detailed description
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.
Study design
Who may qualify
- Ages
- Up to 65 years
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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).
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CliniMACS CD34 Reagent SystemThe CliniMACS CD34 Reagent System is a medical device that is used in vitro to select and enrich CD34+ cells from heterogeneous hematologic cell populations for transplantation in cases where this is clinically indicated. | Biological | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Nov 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Joanne Kurtzberg, MD principal investigator
Duke University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing CliniMACS CD34 Reagent System in people with Hematologic Malignancies and Inborn Errors of Metabolism Disorders, run by Joanne Kurtzberg, MD at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 20 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Erin Arbuckle (919-684-3293, erin.arbuckle@duke.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Nov 2025.