LeukemiaNCT00943800
Combined Haploidentical-Cord Blood Transplantation for Adults and Children
A study testing Fludarabine-Melphalan & Rabbit antithymocyte globulin (r-ATG), Stem Cell Transplant and Stem Cells Collections in people with Leukemia and Myelodysplastic Syndrome, run by University of Chicago at 1 site in United States.
Trial summary
The primary objective is to assess the rate of engraftment with combined haploidentical-cord blood transplantation. The secondary objective is to evaluate the incidence and severity of acute and chronic graft-versus-host disease (GVHD). Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Leukemia, Myelodysplastic Syndrome, Multiple Myeloma
- What is being tested?
- Fludarabine-Melphalan & Rabbit antithymocyte globulin (r-ATG), Stem Cell Transplant and Stem Cells Collections
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 87 participants
- Where
- 1 siteUnited States
- Study dates
- 9 Oct 2006to Sep 2018
- Phase
- Interventional
- 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 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients will be eligible for this study if they have any one of the diseases that are known to be cured after allogeneic stem cell transplantation.
- Relapsed or refractory acute leukemia (myeloid or lymphoid)
- Acute leukemia in first remission at high-risk for recurrence
- Chronic myelogenous leukemia in accelerated phase or blast-crisis
- Chronic myelogenous leukemia in chronic phase
- Recurrent or refractory malignant lymphoma or Hodgkin lymphoma
Show all 12Show fewer
- Chronic lymphocytic leukemia, relapsed or with poor prognostic features
- Multiple myeloma
- Myelodysplastic syndrome
- Chronic myeloproliferative disease
- Hemoglobinopathies
- Aplastic anemia
Who cannot join
- Zubrod performance status > 2
- Life expectancy is severely limited by concomitant illness
- Patients with severely decreased LVEF or impaired pulmonary function tests(PFT's)
- Estimated Creatinine Clearance <50 ml/min
- Serum bilirubin> 2.0 mg/dl or SGPT >3 x upper limit of normal
- Evidence of chronic active hepatitis or cirrhosis
Show all 9Show fewer
- HIV-positive
- Patient is pregnant
- Patient or guardian not able to sign informed consent
Trial locations and contacts
1 site in 1 country.
About this study
The primary objective is to assess the rate of engraftment with combined haploidentical-cord blood transplantation. The secondary objective is to evaluate the incidence and severity of acute and chronic graft-versus-host disease (GVHD).
- Conditions
- Leukemia, Myelodysplastic Syndrome, Multiple Myeloma, Lymphoma
- Being tested
- Fludarabine-Melphalan & Rabbit antithymocyte globulin (r-ATG), Stem Cell Transplant, Stem Cells Collections, Fludarabine, Thiotepa, Antithymocyte globulin (ATG), and Total Body Irradiation (TBI), Fludarabine, Busulfan, and ATG
- Keywords
- Appropriate candidate for transplantation, An HLA-identical related or unrelated donor cannot be identified within an appropriate time frame.
- Medical subject headings
- Leukemia, Myelodysplastic Syndromes, Multiple Myeloma, Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fludarabine-Melphalan & Rabbit antithymocyte globulin (r-ATG)Drug
Fludarabine is given through the vein daily for 5 days. Melphalan is given through the vein daily for 2 days. ATG is given every day in the vein for four days.
Stem Cell TransplantProcedure or surgery
Infusion of haploidentical donor, umbilical cord blood
Stem Cells CollectionsProcedure or surgery
Haploidentical cells will be T-cell depleted using the Miltenyi Clinimax device.
Fludarabine, Thiotepa, Antithymocyte globulin (ATG), and Total Body Irradiation (TBI)Drug
Fludarabine is given through the vein daily for 5 days. Thiotepa is given through the vein daily for 2 days. ATG is given through the vein every other day for 4 days. TBI is given twice a day for 3 days.
Fludarabine, Busulfan, and ATGDrug
Fludarabine is given through the vein daily for 5 days. Busulfan is given through the vein daily for 4 days. ATG is given through the vein every other day for 4 days.
| Study group | Role | What they receive |
|---|---|---|
| Good Risks patients | Experimental | For patients transplanted in remission. Receives: Drug: Fludarabine-Melphalan & Rabbit antithymocyte globulin (r-ATG); Procedure: Stem Cell Transplant; Procedure: Stem Cells Collections |
| High Risk Patients eligible for radiation | Experimental | Not described Receives: Procedure: Stem Cell Transplant; Procedure: Stem Cells Collections; Drug: Fludarabine, Thiotepa, Antithymocyte globulin (ATG), and Total Body Irradiation (TBI) |
| High Risk Patients not eligible for radiation | Experimental | Not described Receives: Procedure: Stem Cell Transplant; Procedure: Stem Cells Collections; Drug: Fludarabine, Busulfan, and ATG |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants With Neutrophil Engraftment
Cumulative incidence of graft failure (neutrophil) by day 28 was reported. Patients who did not have neutrophil engraftment before death was considered as a competing risk. Failure to engraft was defined as lack of evidence of hematopoietic recovery (ANC <500/mm3 and platelet count < 20,000/mm3) by day +35, confirmed by a biopsy revealing a marrow cellularity < 5%. Graft failure was also defined as initial myeloid engraftment by day +35, documented to be of donor origin, followed by a drop in the ANC to < 500/mm3 for more than three days, independent of any myelosuppressive drugs, severe GVHD, CMV, or other infection.
Measured: Transplant (Day 0) through Day +28
Secondary outcomes (2)
Percentage of Participants With Incidence of Acute (Grade II-IV) and Chronic Graft-vs-host Disease(GVHD)
Acute GVHD is defined by the Przepiorka criteria, which stages the degree of organ involvement in the skin, liver, and gastrointestinal (GI) tract, based on severity, with Stage 1+ being least severe and stage 4+ being the most severe. Grading of acute GVHD is as follows: Grade II (skin involvement stages 1+ to 3+, liver 1+, GI tract 1+), Grade III (skin involvement stages 2+ to 3+, liver 1+, GI tract 2+ to 4+), Grade IV (skin involvement stages 4+, Liver 4+).
Chronic GVHD is assessed by NIH consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. Diagnosis and staging working group report. Biol Blood Marrow Transplant. 2005; 11:945-56., for grading criteria. (See Citation: Filipovich AH et al)
The incidence of patients with acute GVHD (Grade II-IV) was determined at 180 days. The incidence of Chronic GVHD by 2 years was reported
Measured: Up to 2 years
Overall Survival- Percentage of Participants Who Survived at 2 Years and 5 Years
We reported overall survival at 2 years and 5 years after transplant
Measured: up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jul 2009 (estimated)
- Study start9 Oct 2006
- Primary completionSep 2018
- Study completionSep 2018
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 LeukemiaStudies of the same condition, open and under way.
- More trials run by University of ChicagoThe 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 N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 87 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Chicago
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Hongtao Liu, M.D., Principal investigator, University of Chicago
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00943800
- Sponsor's protocol ID
- 14736B
- Status last verified
- Jan 2021
- First submitted
- 20 Jul 2009
- Last updated
- 27 Jan 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Liu H, Rich ES, Godley L, Odenike O, Joseph L, Marino S, Kline J, Nguyen V, Cunningham J, Larson RA, del Cerro P, Schroeder L, Pape L, Stock W, Wickrema A, Artz AS, van Besien K. Reduced-intensity conditioning with combined haploidentical and cord blood transplantation results in rapid engraftment, low GVHD, and durable remissions. Blood. 2011 Dec 8;118(24):6438-45. doi: 10.1182/blood-2011-08-372508. Epub 2011 Oct 5.
PubMed 21976674van 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 27 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Combined Haploidentical-Cord Blood Transplantation for Adults and Children
- ClinicalTrials.gov ID
- NCT00943800
- Acronym / sponsor ID
- 14736B
- Conditions
- Leukemia, Myelodysplastic Syndrome, Multiple Myeloma, Lymphoma
- Keywords
- Appropriate candidate for transplantation, An HLA-identical related or unrelated donor cannot be identified within an appropriate time frame.
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 87 participants (actual)
- Lead sponsor
- University of ChicagoUniversity, hospital or other
- Study start
- 9 Oct 2006 (actual)
- Primary completion
- Sep 2018 (actual)
- Study completion
- Sep 2018 (actual)
- First posted
- 22 Jul 2009 (estimated)
- Results
- Posted 27 Oct 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective is to assess the rate of engraftment with combined haploidentical-cord blood transplantation. The secondary objective is to evaluate the incidence and severity of acute and chronic graft-versus-host disease (GVHD).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Good Risks patients | Experimental | Drug: Fludarabine-Melphalan & Rabbit antithymocyte globulin (r-ATG), Procedure: Stem Cell Transplant, Procedure: Stem Cells CollectionsFor patients transplanted in remission. |
| High Risk Patients eligible for radiation | Experimental | Procedure: Stem Cell Transplant, Procedure: Stem Cells Collections, Drug: Fludarabine, Thiotepa, Antithymocyte globulin (ATG), and Total Body Irradiation (TBI) |
| High Risk Patients not eligible for radiation | Experimental | Procedure: Stem Cell Transplant, Procedure: Stem Cells Collections, Drug: Fludarabine, Busulfan, and ATG |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (12 listed)
- Patients will be eligible for this study if they have any one of the diseases that are known to be cured after allogeneic stem cell transplantation.
- Relapsed or refractory acute leukemia (myeloid or lymphoid)
- Acute leukemia in first remission at high-risk for recurrence
- Chronic myelogenous leukemia in accelerated phase or blast-crisis
- Chronic myelogenous leukemia in chronic phase
Key exclusion criteria (9 listed)
- Zubrod performance status > 2
- Life expectancy is severely limited by concomitant illness
- Patients with severely decreased LVEF or impaired pulmonary function tests(PFT's)
- Estimated Creatinine Clearance <50 ml/min
- Serum bilirubin> 2.0 mg/dl or SGPT >3 x upper limit of normal
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 |
|---|---|---|
| Fludarabine-Melphalan & Rabbit antithymocyte globulin (r-ATG)Fludarabine is given through the vein daily for 5 days. Melphalan is given through the vein daily for 2 days. ATG is given every day in the vein for four days. | Drug | Good Risks patients |
| Stem Cell TransplantInfusion of haploidentical donor, umbilical cord blood | Procedure or surgery | Good Risks patients, High Risk Patients eligible for radiation, High Risk Patients not eligible for radiation |
| Stem Cells CollectionsHaploidentical cells will be T-cell depleted using the Miltenyi Clinimax device. | Procedure or surgery | Good Risks patients, High Risk Patients eligible for radiation, High Risk Patients not eligible for radiation |
| Fludarabine, Thiotepa, Antithymocyte globulin (ATG), and Total Body Irradiation (TBI)Fludarabine is given through the vein daily for 5 days. Thiotepa is given through the vein daily for 2 days. ATG is given through the vein every other day for 4 days. TBI is given twice a day for 3 days. | Drug | High Risk Patients eligible for radiation |
| Fludarabine, Busulfan, and ATGFludarabine is given through the vein daily for 5 days. Busulfan is given through the vein daily for 4 days. ATG is given through the vein every other day for 4 days. | Drug | High Risk Patients not eligible for radiation |
Primary outcome
Percentage of Participants With Neutrophil Engraftment
Time frame: Transplant (Day 0) through Day +28
Cumulative incidence of graft failure (neutrophil) by day 28 was reported. Patients who did not have neutrophil engraftment before death was considered as a competing risk. Failure to engraft was defined as lack of evidence of hematopoietic recovery (ANC <500/mm3 and platelet count < 20,000/mm3) by day +35, confirmed by a biopsy revealing a marrow cellularity < 5%. Graft failure was also defined as initial myeloid engraftment by day +35, documented to be of donor origin, followed by a drop in the ANC to < 500/mm3 for more than three days, independent of any myelosuppressive drugs, severe GVHD, CMV, or other infection.
2 secondary outcomes
Percentage of Participants With Incidence of Acute (Grade II-IV) and Chronic Graft-vs-host Disease(GVHD)
Time frame: Up to 2 years
Overall Survival- Percentage of Participants Who Survived at 2 Years and 5 Years
Time frame: up to 5 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jan 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 21976674Liu H, Rich ES, Godley L, Odenike O, Joseph L, Marino S, Kline J, Nguyen V, Cunningham J, Larson RA, del Cerro P, Schroeder L, Pape L, Stock W, Wickrema A, Artz AS, van Besien K. Reduced-intensity conditioning with combined haploidentical and cord blood transplantation results in rapid engraftment, low GVHD, and durable remissions. Blood. 2011 Dec 8;118(24):6438-45. doi: 10.1182/blood-2011-08-372508. Epub 2011 Oct 5.
- 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.
Hongtao Liu, M.D. principal investigator
University of Chicago
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Fludarabine-Melphalan & Rabbit antithymocyte globulin (r-ATG), Stem Cell Transplant and Stem Cells Collections in people with Leukemia and Myelodysplastic Syndrome, run by University of Chicago 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 12 inclusion and 9 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 27 Jan 2021. 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?
Yes. Results are posted in the registry (first posted 27 Oct 2020). 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?
Percentage of Participants With Neutrophil Engraftment (time frame: Transplant (Day 0) through Day +28). 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 NCT00943800, 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 27 Jan 2021.