Hematologic MalignanciesPhase 2NCT01810588
Targeting the IPA and Matching for the Non-Inherited Maternal Antigen for Haplo-Cord Transplantation
A Phase 2 study testing CliniMACS® CD34 Reagent System, Fludarabine and Melphalan in people with Hematologic Malignancies, run by Weill Medical College of Cornell University at 2 sites in United States.
Trial summary
In this trial, we aim to improve the outcomes of haplo cord transplant. Haplo cord transplant is a novel and promising way to improve transplant outcomes. We hypothesize that identification of a graft that is at least 5/6 matched and inherited paternal antigen (IPA) targeted (i.e., cord blood grafts share one or more… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hematologic Malignancies
- What is being tested?
- CliniMACS® CD34 Reagent System, Fludarabine and Melphalan
- Type of treatment
- Pill or oral medicine, Injection or infusion, Medical device, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 270 participants
- Where
- 2 sitesUnited States
- Study dates
- 16 Oct 2012to 24 Mar 2026
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subject must have a confirmed diagnosis of:
- Previously Relapsed or refractory acute leukemia (myeloid or lymphoid)
- Acute leukemia in first remission at high-risk for recurrence
- Chronic myelogenous leukemia in chronic, accelerated phase or blast-crisis
- Recurrent or refractory malignant lymphoma or Hodgkin lymphoma
- Chronic lymphocytic leukemia, relapsed or with poor prognostic features
Show all 18Show fewer
- Multiple myeloma
- Myelodysplastic syndrome
- Chronic myeloproliferative disease
- Hemoglobinopathies
- Aplastic anemia
- Other hematological disorder in need of allogeneic transplant (e.g. blastoid dendritic cell neoplasm)
- Age ≥ 18 years
- Likely to benefit from allogeneic transplant in the opinion of the transplant physician
- An HLA-identical related or unrelated donor cannot be identified within an appropriate time frame.
- Karnofsky (KPS) Performance status of >= 70%
- Acceptable organ function as defined below: Serum bilirubin: < 2.0mg/dL ALT(SGPT): < 3 X upper limit of normal Creatinine Clearance: > 50 mL/min/1.73m2 (as estimated by the modified MDRD equation)
- Ability to understand and the willingness to sign a written informed consent document
Who cannot join
- Life expectancy is severely limited by concomitant illness or uncontrolled infection
- Severely decreased Left Ventricular Ejection Fraction (LVEF) or impaired pulmonary function tests (PFT's)
- Evidence of chronic active hepatitis or cirrhosis
- Uncontrolled HIV disease
- Pregnant or lactating
Trial locations and contacts
2 sites in 1 country.
United States2 sites
University of Chicago Medical Center
Chicago, Illinois, 60637
Weill Cornell Medical College
New York, New York, 10065
About this study
Official title: A Prospective Study of Optimal Cord Selection for Haplo-Cord Transplantation: Targeting the Inherited Paternal Antigen (IPA) and Matching for the Non-Inherited Maternal Antigen (NIMA)
In this trial, we aim to improve the outcomes of haplo cord transplant. Haplo cord transplant is a novel and promising way to improve transplant outcomes. We hypothesize that identification of a graft that is at least 5/6 matched and inherited paternal antigen (IPA) targeted (i.e., cord blood grafts share one or more IPA antigens with the prospective recipient) is more important to the outcome of haplo cord transplant than the nucleated cell dose. The identification of such a graft for a large proportion of the subjects may necessitate accepting a lower umbilical cord graft dose.
In addition to a umbilical cord blood transplant, recipients will receive stem cells from a family member ( a haplo-identical donor) . After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. The subject will undergo a chemotherapy conditioning regimen prior to transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past.
Read the detailed description
This is a clinical trial for subjects with hematologic malignancies ( acute leukemia, myeloproliferative disorders, lymphoma, myeloma) who are in need of a donor stem cell transplant, and for whom an umbilical cord blood transplant is thought to be the best option. As donors for allogeneic transplant, we typically try to use related family members, such as brothers or sisters, or volunteer donors who are 'HLA matched', i.e. share similar proteins on their cells. This study is for subjects for whom such a matched sibling donor or a matched unrelated donor is not available.
For such subjects a commonly used transplant procedure is to use stem cells from one or two umbilical cords (UCB) from a newborn. These umbilical cord blood grafts, despite not completely matching the recipient, cause few problems with graft vs host disease (a common complication of transplant). But they tend to grow very slowly and subjects often have very prolonged hospital stays and are at high risk for complications due to low blood counts. Umbilical cord blood transplant will be the standard arm for this protocol.
This study uses a new method of bone marrow transplantation called combined haplo-identical cord (haplo-cord) transplantation. In this procedure, cells from a related donor who shares half of the HLA proteins ( haplo-identical) are collected from the blood, as well as cells from an umbilical cord, and then both are transplanted. It is hoped that by using cells from a haplo-identical relative, subjects will have a faster recovery and require fewer transfusions. Over time the haplo-identical cells from the relative are replaced by the cells from the cord blood. The combined transplantation of haplo-identical stem cells and cord blood has previously been used in approximately 60 subjects with very encouraging results.
Traditionally it has been felt that the most important determinant of outcome of an UCB stem cell transplant is the cord blood cell dose. The second determinant is the degree of matching between donor and recipient. Many times, we have difficulty identifying UCB units of sufficient cell dose that are well matched. Of interest,in our prior study of haplo-cord SCT indicated outcomes seemed independent of the UCB cell dose. If this preliminary observation is correct, we may be able to improve the outcomes of haplo cord transplant further by accepting lower threshold UCB doses and rather focusing on optimal matching (including matching for HLA and another characteristic called IPA). This is the primary objective of this study.
- Conditions
- Hematologic Malignancies
- Being tested
- CliniMACS® CD34 Reagent System, Fludarabine, Melphalan, anti-thymocyte globulin (rabbit), Rituximab
- Keywords
- Allogeneic transplant, Hematologic Malignancies, Acute Leukemia, Myeloproliferative disorders, Lymphoma, Myeloma
- Medical subject headings
- Hematologic Neoplasms, Myeloproliferative Disorders, Lymphoma, Neoplasms, Plasma Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CliniMACS® CD34 Reagent SystemDevice
The stem cells from the haplo-identical donor will be purified by a procedure called CD34 selection before they are given to the subject. A special device called the CliniMACS® CD34 Reagent System, which is not FDA approved, will be used for this purpose. The manufacturer of the device, Miltenyi Biotec, is providing the researchers access to the device for use in this research study. Because the stem cells from the haplo-identical donor are treated using the CliniMACS CD34 selection device, they cells are considered investigational.
FludarabineDrug
Also called Fludara, Fludarabine phosphate
Administer 30 mg/m2 /day intravenously x 5 days (Day -7 to Day -3) of a total dose of 150 mg/m2. Fludarabine will be dosed according to actual body weight.
MelphalanDrug
Administer 70mg/m2/day intravenously x 2 days. Melphalan will be dosed according to actual body weight. Cryotherapy with ice chips will be administered to prevent mucositis
anti-thymocyte globulin (rabbit)Drug
Also called rATG, Rabbit ATG
Administer 1.5 mg/kg/day intravenously x 3 days, total 4.5 mg/kg. ATG will be dosed according to actual body weight. The first dose will be infused over at least six hours, and subsequent doses over at least 4 hours. Pre-medications include acetaminophen 650 mg by mouth, diphenhydramine 25-50 mg by mouth or intravenously, and methylprednisolone 2 mg/kg (1 mg/ kg at the initiation and 1 mg/kg half-way through anti-thymocyte globulin administration).
RituximabDrug
Also called Rituxan
Administer one dose of 375 mg/m2 prior to or upon admission for all patients not previously exposed to rituximab or who have not received rituximab in the six months prior to transplant.
Total Body IrradiationRadiation
Also called TBI
Patients at high risk of CNS relapse (e.g. ALL or Burkitt's lymphoma) or patients at high risk for graft rejection (i.e., donor-specific HLA antibodies, patients with severe aplastic anemia, or hemoglobinopathies) may receive 2 doses of TBI as part of the conditioning.
Also called Cellcept, MMF
Will be started on Day -2 and given at a dose of 1000 mg every 8 hours until Day 28. Mycophenolae Mofetil can be given orally or intravenously. Infection, toxicity, very low patient weight (<50 kilograms) may prompt earlier discontinuation or adjustment of doses.
TacrolimusDrug
Also called Prograf
Administered 0.03/mg/kg/day intravenous continious infusion (CI) over 24 hours from 4pm Day -2 until engraftment or when subject is able to take orally, then tacrolimus approximately 0.09 mg/kg orally in 2 divided doses. Tacrolimus should be given at full dose to maintain levels of 5-15 ng/mL through Day 180, tapered by 20% every week thereafter. Infection, toxicity or other clinical circumstances may prompt earlier discontinuation or adjustment of doses. In the presence of Graft versus Host Disease, a clinical decision by the attending physician will determine if tacrolimus can be tapered or should be continued. Oral tacrolimus can be used when intravenous access for CI tacrolimus is unavailable.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 - Minimum Cell Dose 2 x 10^7 TNC/kg | Experimental | All subjects in this cohort will receive a minimal cell dose of 2 x 10^7 total nucleated cells (TNC)/kilogram (kg) for the umbilical cord blood unit.
Haplo-cord transplantation:
All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past.
For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products.
In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. Receives: Device: CliniMACS® CD34 Reagent System; Drug: Fludarabine; Drug: Melphalan; Drug: anti-thymocyte globulin (rabbit); Drug: Rituximab; Radiation: Total Body Irradiation; Drug: Mycophenolate Mofetil; Drug: Tacrolimus |
| Cohort 2 - Minimum Cell Dose 1 x 10^7 TNC/kg | Experimental | All subjects in this cohort will receive a minimal cell dose of 1 x 10^7 total nucleated cells (TNC)/kilogram (kg) for the umbilical cord blood unit.
Haplo-cord transplantation:
All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past.
For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products.
In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. Receives: Device: CliniMACS® CD34 Reagent System; Drug: Fludarabine; Drug: Melphalan; Drug: anti-thymocyte globulin (rabbit); Drug: Rituximab; Radiation: Total Body Irradiation; Drug: Mycophenolate Mofetil; Drug: Tacrolimus |
| Cohort 3 - Minimum Cell Dose 0.5 x 10^7 TNC/kg | Experimental | All subjects in this cohort will receive a minimal cell dose of 0.5 x 10^7 total nucleated cells (TNC)/kilogram (kg) for the umbilical cord blood unit.
Haplo-cord transplantation:
All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past.
For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products.
In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. Receives: Device: CliniMACS® CD34 Reagent System; Drug: Fludarabine; Drug: Melphalan; Drug: anti-thymocyte globulin (rabbit); Drug: Rituximab; Radiation: Total Body Irradiation; Drug: Mycophenolate Mofetil; Drug: Tacrolimus |
| Cohort 4 | Experimental | All subjects in this cohort will receive the minimum required cell dose that is determined following the dose de-escalation portion of the study (cohorts 1 through 3)
Haplo-cord transplantation:
All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past.
For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products.
In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. Receives: Device: CliniMACS® CD34 Reagent System; Drug: Fludarabine; Drug: Melphalan; Drug: anti-thymocyte globulin (rabbit); Drug: Rituximab; Radiation: Total Body Irradiation; Drug: Mycophenolate Mofetil; Drug: Tacrolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Subjects Who Achieved Engraftment With De-Escalating Umbilical Cord Total Nucleated Cell (TNC) Dose
We aim to identify the lowest threshold of umbilical cord total nucleated cell (TNC) dose that can be utilized assure durable umbilical cord blood engraftment in the haplo-cord transplants. The threshold will be defined as the lowest dose which assures cord blood engraftment occurs in at least 80% of subjects.
Measured: 100 days
Secondary outcomes (3)
Long-term Survival of Subjects Undergoing Haplo-cord Transplants
Evaluate the long term outcome of subjects undergoing haplo cord transplants using an optimally matched umbilical cord blood (UCB) graft (i.e. Survival, profession-free survival (PFS), Relapse, transplant-related mortality (TRM), toxicities, infections and GVHD)
Measured: 5 years after transplant
Impact of IPA Targeting on Transplant Outcome
As much as possible, UCB units will be chosen to be IPA targeted. This is not always possible. We will therefore also retrospectively analyze transplant outcomes and correlate with IPA status.
Measured: 5 years from transplantation
Impact of NIMA Matching on Transplant Outcome
As much as possible, UCB units will be chosen to be NIMA matched. This is not always possible. We will therefore also retrospectively analyze transplant outcomes and correlate with NIMA status.
Measured: 5 years from transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Mar 2013 (estimated)
- Study start16 Oct 2012
- Primary completion9 Aug 2023
- Study completion24 Mar 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Hematologic MalignanciesStudies of the same condition, open and under way.
- More trials run by Weill Medical College of Cornell UniversityThe sponsor's other registered studies.
- 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 270 participants (actual)
Sponsor and investigators
- Lead sponsor
- Weill Medical College of Cornell University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Alexandra Gomez Arteaga, MD, Principal investigator, Weill Medical College of Cornell University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01810588
- Sponsor's protocol ID
- 1205012383
- Status last verified
- Jul 2026
- First submitted
- 7 Jan 2013
- Last updated
- 19 Aug 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
van Besien K, Artz A, Champlin RE, Guarneri D, Bishop MR, Chen J, Gergis U, Shore T, Liu H, Rondon G, Mayer SA, Srour SA, Stock W, Ciurea SO. Haploidentical vs haplo-cord transplant in adults under 60 years receiving fludarabine and melphalan conditioning. Blood Adv. 2019 Jun 25;3(12):1858-1867. doi: 10.1182/bloodadvances.2019000200.
PubMed 31217161van Besien K, Hari P, Zhang MJ, Liu HT, Stock W, Godley L, Odenike O, Larson R, Bishop M, Wickrema A, Gergis U, Mayer S, Shore T, Tsai S, Rhodes J, Cushing MM, Korman S, Artz A. Reduced intensity haplo plus single cord transplant compared to double cord transplant: improved engraftment and graft-versus-host disease-free, relapse-free survival. Haematologica. 2016 May;101(5):634-43. doi: 10.3324/haematol.2015.138594. Epub 2016 Feb 11.
PubMed 26869630
Study at a glance
From the registry record, last updated by the sponsor on 19 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Study of Optimal Cord Selection for Haplo-Cord Transplantation: Targeting the Inherited Paternal Antigen (IPA) and Matching for the Non-Inherited Maternal Antigen (NIMA)
- ClinicalTrials.gov ID
- NCT01810588
- Acronym / sponsor ID
- 1205012383
- Conditions
- Hematologic Malignancies
- Keywords
- Allogeneic transplant, Hematologic Malignancies, Acute Leukemia, Myeloproliferative disorders, Lymphoma, Myeloma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 270 participants (actual)
- Lead sponsor
- Weill Medical College of Cornell UniversityUniversity, hospital or other
- Study start
- 16 Oct 2012 (actual)
- Primary completion
- 9 Aug 2023 (actual)
- Study completion
- 24 Mar 2026 (actual)
- First posted
- 13 Mar 2013 (estimated)
- Results
- Posted 9 Oct 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
In this trial, we aim to improve the outcomes of haplo cord transplant. Haplo cord transplant is a novel and promising way to improve transplant outcomes. We hypothesize that identification of a graft that is at least 5/6 matched and inherited paternal antigen (IPA) targeted (i.e., cord blood grafts share one or more IPA antigens with the prospective recipient) is more important to the outcome of haplo cord transplant than the nucleated cell dose. The identification of such a graft for a large proportion of the subjects may necessitate accepting a lower umbilical cord graft dose.
In addition to a umbilical cord blood transplant, recipients will receive stem cells from a family member ( a haplo-identical donor) . After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. The subject will undergo a chemotherapy conditioning regimen prior to transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past.
Detailed description
This is a clinical trial for subjects with hematologic malignancies ( acute leukemia, myeloproliferative disorders, lymphoma, myeloma) who are in need of a donor stem cell transplant, and for whom an umbilical cord blood transplant is thought to be the best option. As donors for allogeneic transplant, we typically try to use related family members, such as brothers or sisters, or volunteer donors who are 'HLA matched', i.e. share similar proteins on their cells. This study is for subjects for whom such a matched sibling donor or a matched unrelated donor is not available.
For such subjects a commonly used transplant procedure is to use stem cells from one or two umbilical cords (UCB) from a newborn. These umbilical cord blood grafts, despite not completely matching the recipient, cause few problems with graft vs host disease (a common complication of transplant). But they tend to grow very slowly and subjects often have very prolonged hospital stays and are at high risk for complications due to low blood counts. Umbilical cord blood transplant will be the standard arm for this protocol.
This study uses a new method of bone marrow transplantation called combined haplo-identical cord (haplo-cord) transplantation. In this procedure, cells from a related donor who shares half of the HLA proteins ( haplo-identical) are collected from the blood, as well as cells from an umbilical cord, and then both are transplanted. It is hoped that by using cells from a haplo-identical relative, subjects will have a faster recovery and require fewer transfusions. Over time the haplo-identical cells from the relative are replaced by the cells from the cord blood. The combined transplantation of haplo-identical stem cells and cord blood has previously been used in approximately 60 subjects with very encouraging results.
Traditionally it has been felt that the most important determinant of outcome of an UCB stem cell transplant is the cord blood cell dose. The second determinant is the degree of matching between donor and recipient. Many times, we have difficulty identifying UCB units of sufficient cell dose that are well matched. Of interest,in our prior study of haplo-cord SCT indicated outcomes seemed independent of the UCB cell dose. If this preliminary observation is correct, we may be able to improve the outcomes of haplo cord transplant further by accepting lower threshold UCB doses and rather focusing on optimal matching (including matching for HLA and another characteristic called IPA). This is the primary objective of this study.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 - Minimum Cell Dose 2 x 10^7 TNC/kg | Experimental | Device: CliniMACS® CD34 Reagent System, Drug: Fludarabine, Drug: Melphalan, Drug: anti-thymocyte globulin (rabbit), Drug: Rituximab, Radiation: Total Body Irradiation, Drug: Mycophenolate Mofetil, Drug: TacrolimusAll subjects in this cohort will receive a minimal cell dose of 2 x 10^7 total nucleated cells (TNC)/kilogram (kg) for the umbilical cord blood unit. Haplo-cord transplantation: All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past. For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products. In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. |
| Cohort 2 - Minimum Cell Dose 1 x 10^7 TNC/kg | Experimental | Device: CliniMACS® CD34 Reagent System, Drug: Fludarabine, Drug: Melphalan, Drug: anti-thymocyte globulin (rabbit), Drug: Rituximab, Radiation: Total Body Irradiation, Drug: Mycophenolate Mofetil, Drug: TacrolimusAll subjects in this cohort will receive a minimal cell dose of 1 x 10^7 total nucleated cells (TNC)/kilogram (kg) for the umbilical cord blood unit. Haplo-cord transplantation: All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past. For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products. In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. |
| Cohort 3 - Minimum Cell Dose 0.5 x 10^7 TNC/kg | Experimental | Device: CliniMACS® CD34 Reagent System, Drug: Fludarabine, Drug: Melphalan, Drug: anti-thymocyte globulin (rabbit), Drug: Rituximab, Radiation: Total Body Irradiation, Drug: Mycophenolate Mofetil, Drug: TacrolimusAll subjects in this cohort will receive a minimal cell dose of 0.5 x 10^7 total nucleated cells (TNC)/kilogram (kg) for the umbilical cord blood unit. Haplo-cord transplantation: All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past. For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products. In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. |
| Cohort 4 | Experimental | Device: CliniMACS® CD34 Reagent System, Drug: Fludarabine, Drug: Melphalan, Drug: anti-thymocyte globulin (rabbit), Drug: Rituximab, Radiation: Total Body Irradiation, Drug: Mycophenolate Mofetil, Drug: TacrolimusAll subjects in this cohort will receive the minimum required cell dose that is determined following the dose de-escalation portion of the study (cohorts 1 through 3) Haplo-cord transplantation: All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past. For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products. In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- Subject must have a confirmed diagnosis of:
- Age ≥ 18 years
- Likely to benefit from allogeneic transplant in the opinion of the transplant physician
- An HLA-identical related or unrelated donor cannot be identified within an appropriate time frame.
- Karnofsky (KPS) Performance status of >= 70%
Key exclusion criteria (5 listed)
- Life expectancy is severely limited by concomitant illness or uncontrolled infection
- Severely decreased Left Ventricular Ejection Fraction (LVEF) or impaired pulmonary function tests (PFT's)
- Evidence of chronic active hepatitis or cirrhosis
- Uncontrolled HIV disease
- Pregnant or lactating
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 stem cells from the haplo-identical donor will be purified by a procedure called CD34 selection before they are given to the subject. A special device called the CliniMACS® CD34 Reagent System, which is not FDA approved, will be used for this purpose. The manufacturer of the device, Miltenyi Biotec, is providing the researchers access to the device for use in this research study. Because the stem cells from the haplo-identical donor are treated using the CliniMACS CD34 selection device, they cells are considered investigational. | Device | Cohort 1 - Minimum Cell Dose 2 x 10^7 TNC/kg, Cohort 2 - Minimum Cell Dose 1 x 10^7 TNC/kg, Cohort 3 - Minimum Cell Dose 0.5 x 10^7 TNC/kg, Cohort 4 |
| FludarabineAlso: Fludara, Fludarabine phosphateAdminister 30 mg/m2 /day intravenously x 5 days (Day -7 to Day -3) of a total dose of 150 mg/m2. Fludarabine will be dosed according to actual body weight. | Drug | Cohort 1 - Minimum Cell Dose 2 x 10^7 TNC/kg, Cohort 2 - Minimum Cell Dose 1 x 10^7 TNC/kg, Cohort 3 - Minimum Cell Dose 0.5 x 10^7 TNC/kg, Cohort 4 |
| MelphalanAdminister 70mg/m2/day intravenously x 2 days. Melphalan will be dosed according to actual body weight. Cryotherapy with ice chips will be administered to prevent mucositis | Drug | Cohort 1 - Minimum Cell Dose 2 x 10^7 TNC/kg, Cohort 2 - Minimum Cell Dose 1 x 10^7 TNC/kg, Cohort 3 - Minimum Cell Dose 0.5 x 10^7 TNC/kg, Cohort 4 |
| anti-thymocyte globulin (rabbit)Also: rATG, Rabbit ATGAdminister 1.5 mg/kg/day intravenously x 3 days, total 4.5 mg/kg. ATG will be dosed according to actual body weight. The first dose will be infused over at least six hours, and subsequent doses over at least 4 hours. Pre-medications include acetaminophen 650 mg by mouth, diphenhydramine 25-50 mg by mouth or intravenously, and methylprednisolone 2 mg/kg (1 mg/ kg at the initiation and 1 mg/kg half-way through anti-thymocyte globulin administration). | Drug | Cohort 1 - Minimum Cell Dose 2 x 10^7 TNC/kg, Cohort 2 - Minimum Cell Dose 1 x 10^7 TNC/kg, Cohort 3 - Minimum Cell Dose 0.5 x 10^7 TNC/kg, Cohort 4 |
| RituximabAlso: RituxanAdminister one dose of 375 mg/m2 prior to or upon admission for all patients not previously exposed to rituximab or who have not received rituximab in the six months prior to transplant. | Drug | Cohort 1 - Minimum Cell Dose 2 x 10^7 TNC/kg, Cohort 2 - Minimum Cell Dose 1 x 10^7 TNC/kg, Cohort 3 - Minimum Cell Dose 0.5 x 10^7 TNC/kg, Cohort 4 |
| Total Body IrradiationAlso: TBIPatients at high risk of CNS relapse (e.g. ALL or Burkitt's lymphoma) or patients at high risk for graft rejection (i.e., donor-specific HLA antibodies, patients with severe aplastic anemia, or hemoglobinopathies) may receive 2 doses of TBI as part of the conditioning. | Radiation | Cohort 1 - Minimum Cell Dose 2 x 10^7 TNC/kg, Cohort 2 - Minimum Cell Dose 1 x 10^7 TNC/kg, Cohort 3 - Minimum Cell Dose 0.5 x 10^7 TNC/kg, Cohort 4 |
| Mycophenolate MofetilAlso: Cellcept, MMFWill be started on Day -2 and given at a dose of 1000 mg every 8 hours until Day 28. Mycophenolae Mofetil can be given orally or intravenously. Infection, toxicity, very low patient weight (<50 kilograms) may prompt earlier discontinuation or adjustment of doses. | Drug | Cohort 1 - Minimum Cell Dose 2 x 10^7 TNC/kg, Cohort 2 - Minimum Cell Dose 1 x 10^7 TNC/kg, Cohort 3 - Minimum Cell Dose 0.5 x 10^7 TNC/kg, Cohort 4 |
| TacrolimusAlso: PrografAdministered 0.03/mg/kg/day intravenous continious infusion (CI) over 24 hours from 4pm Day -2 until engraftment or when subject is able to take orally, then tacrolimus approximately 0.09 mg/kg orally in 2 divided doses. Tacrolimus should be given at full dose to maintain levels of 5-15 ng/mL through Day 180, tapered by 20% every week thereafter. Infection, toxicity or other clinical circumstances may prompt earlier discontinuation or adjustment of doses. In the presence of Graft versus Host Disease, a clinical decision by the attending physician will determine if tacrolimus can be tapered or should be continued. Oral tacrolimus can be used when intravenous access for CI tacrolimus is unavailable. | Drug | Cohort 1 - Minimum Cell Dose 2 x 10^7 TNC/kg, Cohort 2 - Minimum Cell Dose 1 x 10^7 TNC/kg, Cohort 3 - Minimum Cell Dose 0.5 x 10^7 TNC/kg, Cohort 4 |
Primary outcome
Number of Subjects Who Achieved Engraftment With De-Escalating Umbilical Cord Total Nucleated Cell (TNC) Dose
Time frame: 100 days
We aim to identify the lowest threshold of umbilical cord total nucleated cell (TNC) dose that can be utilized assure durable umbilical cord blood engraftment in the haplo-cord transplants. The threshold will be defined as the lowest dose which assures cord blood engraftment occurs in at least 80% of subjects.
3 secondary outcomes
Long-term Survival of Subjects Undergoing Haplo-cord Transplants
Time frame: 5 years after transplant
Impact of IPA Targeting on Transplant Outcome
Time frame: 5 years from transplantation
Impact of NIMA Matching on Transplant Outcome
Time frame: 5 years from transplantation
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Aug 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 31217161van Besien K, Artz A, Champlin RE, Guarneri D, Bishop MR, Chen J, Gergis U, Shore T, Liu H, Rondon G, Mayer SA, Srour SA, Stock W, Ciurea SO. Haploidentical vs haplo-cord transplant in adults under 60 years receiving fludarabine and melphalan conditioning. Blood Adv. 2019 Jun 25;3(12):1858-1867. doi: 10.1182/bloodadvances.2019000200.
- Derived from this study: PubMed 26869630van Besien K, Hari P, Zhang MJ, Liu HT, Stock W, Godley L, Odenike O, Larson R, Bishop M, Wickrema A, Gergis U, Mayer S, Shore T, Tsai S, Rhodes J, Cushing MM, Korman S, Artz A. Reduced intensity haplo plus single cord transplant compared to double cord transplant: improved engraftment and graft-versus-host disease-free, relapse-free survival. Haematologica. 2016 May;101(5):634-43. doi: 10.3324/haematol.2015.138594. Epub 2016 Feb 11.
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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.
Alexandra Gomez Arteaga, MD principal investigator
Weill Medical College of Cornell University
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing CliniMACS® CD34 Reagent System, Fludarabine and Melphalan in people with Hematologic Malignancies, run by Weill Medical College of Cornell University at 2 sites 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 18 inclusion and 5 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: finished, as of the sponsor's last update on 19 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 9 Oct 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Subjects Who Achieved Engraftment With De-Escalating Umbilical Cord Total Nucleated Cell (TNC) Dose (time frame: 100 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01810588, which also lists 2 linked publications.
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 19 Aug 2026.