MDS (Myelodysplastic Syndrome)Phase 1/2NCT01866839
Preventing Stem Cell Transplant Complications With a Blood Separator Machine
A Phase 1/2 study testing Graft Manipulation (CD34+ Selection) in people with MDS (Myelodysplastic Syndrome) and Myeloproliferative Disorder, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
Trial summary
Background: Researchers are working to make stem cell transplant procedures safer and more effective. One complication of transplants is graft-versus-host disease (GVHD). This complication happens when certain white blood cells from the donor attack the recipient's own body. Read more about this study
- Who is it for?
- Ages 2 to 80 years
- Conditions the study is for
- MDS (Myelodysplastic Syndrome), Myeloproliferative Disorder, Lymphoma, Non-Hodgkin
- What is being tested?
- Graft Manipulation (CD34+ Selection)
- Type of treatment
- Injection or infusion, Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 41 participants
- Where
- 1 siteUnited States
- Study dates
- 29 May 2013to 28 Jun 2018
- Phase
- Phase 1/2
- 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 2 to 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 5.1.1 Ages 10-80 years inclusive
- 5.1.2 Any one of the following hematologic conditions, confirmed by pathology, meeting a standard indication for allogeneic stem cell transplant:
- 5.1.2.1 Chronic myelogenous leukemia (CML): Subjects under the age of 21 in chronic phase OR Subjects ages 10-80 in chronic phase who have failed or are intolerant to treatment with second generation tyrosine inhibitors OR Subjects ages 10-80 in accelerated phase or blast transformation. OR
- 5.1.2.2 Acute lymphoblastic leukemia (ALL): any of these categories: Adult ALL including standard risk; Pediatric ALL in first remission with high-risk features (presenting leukocyte count >100,000/cu mm, karyotypes t(9; 22), t4, t19, t11, biphenotypic leukemia). All second or subsequent remissions, primary induction failure, partially responding or untreated relapse. OR
- 5.1.2.3 Acute myelogenous leukemia (AML): AML in first remission - except AML with good risk karyotypes: AML M3 (t15; 17), AML M4Eo (inv 16), c-kit unmutated AML t (8; 21). All AML in second or subsequent remission, primary induction failure and resistant relapse. OR
- 5.1.2.4 Myelodysplastic syndromes(MDS): any of these categories - refractory anemia with transfusion dependence, refractory anemia with ANC<500/microL, refractory anemia with excess of blasts, transformation to acute leukemia, chronic myelomonocytic leukemia, atypical MDS/myeloproliferative syndromes. OR
Show all 29Show fewer
- 5.1.2.5 Myeloproliferative disorders including atypical (Ph-negative) chronic myeloid and neutrophilic leukemias, progressing myelofibrosis, and polycythemia vera, essential thrombocythemia either in transformation to acute leukemia or with progressive transfusion requirements or pancytopenia. OR
- 5.1.2.6 Chronic lymphocytic leukemia refractory to fludarabine treatment and with bulky progressive disease or with thrombocytopenia (less than or equal to 100,000 / microl) or anemia (less than or equal to 10g/dl) not due to recent chemotherapy. OR
- 5.1.2.7 Non-Hodgkin s lymphoma including Mantle cell lymphoma relapsing or refractory to standard of care treatments. OR
- 5.1.2.8 Multiple myeloma, Waldenstroms macroglobulinemia, unresponsive or relapsed following standard of care treatments. OR
- 5.1.2.9 Hodgkin's Lymphoma relapsing following an autologous transplant. OR
- 5.1.2.10 Other rare hematologic malignancies for which hematopoietic stem cell transplantation has been performed and offers a durable remission or as the only option for potential for cure.
- Chemotherapy-resistant multisystem Langerhans cell histiocytosis (MSLCH) especially involving organs like the bone marrow, liver, spleen, and lungs
- Aggressive systemic mastocytosis, and mast cell leukemia (MCL) in first CR (CR1)
- Hypereosinophilic syndrome who have failed imatinib therapy or FIP1L1-PDGFRa-negative patients who develop end-organ dysfunction
- Adult T-cell leukemia/lymphoma at first diagnosis
- Refractory or disseminated nasal-type extranodal NK/T-lymphoma or aggressive Natural killer cell leukemia/lymphoma
- Mycosis fungoides and S(SqrRoot)(Copyright)zary syndrome after failure of two or three initial therapies
- Primary or relapsed refractory Angioimmunoblastic T-cell lymphoma at first diagnosis
- Hepatosplenic T-cell lymphoma (gamma/delta T-cell lymphoma) at first diagnosis
- T-cell prolymphocytic leukemia at first diagnosis
- Subcutaneous panniculitic T-cell lymphoma at first diagnosis
- Hematodermic neoplasm (blastic natural killer cell lymphoma or Blastic plasmacytoid dendritic cell neoplasm) at first diagnosis
- 5.1.3 HLA identical (6/6) related donor.
- 5.1.4 For adults: ability to comprehend the investigational nature of the study and provide informed consent. For minors: written informed consent from one parent or guardian. Informed oral assent from minors: the process will be explained to the minor on a level of complexity appropriate for their age and ability to comprehend.
- 5.3.1 Related donor, HLA identical (6/6) with recipient
- 5.3.2 Weight greater than or equal to 18 kg
- 5.3.3 Age greater than or equal to 2 or less than or equal to 80 years old
- 5.3.4 For adults: ability to comprehend the investigational nature of the study and provide informed consent. For minors: written informed consent from one parent or guardian and informed assent: The process will be explained to the minor on a level of complexity appropriate for their age and ability to comprehend.
Who cannot join
- 5.2.1 Major anticipated illness or organ failure incompatible with survival from transplant
- 5.2.2 Severe psychiatric illness or mental deficiency sufficiently severe as to make compliance with the transplant treatment unlikely and making informed consent impossible.
- 5.2.3 Positive pregnancy test for women of childbearing age
- 5.2.4 DLCO adjusted for Hb and ventilation< 50% predicted
- 5.2.5 Left ventricular ejection fraction < 40% (evaluated by ECHO) or < 30% (evaluated by MUGA)
- 5.2.6 AST/SGOT > 10 times ULN
Show all 14Show fewer
- 5.2.7 Total bilirubin > 5 times ULN
- 5.2.8 Estimated GFR < 15 mL/min
- 5.2.9 Recipients who have active infections with HIV or active hepatitis C (HCV)
- 5.4.1 Pregnant or breast-feeding. Lactating donors are permitted provided breast milk is discarded during the days filgrastim (G-CSF) is given
- 5.4.2 Unfit to receive G-CSF and undergo apheresis (abnormal blood counts, history of stroke, uncontrolled hypertension)
- 5.4.3 Sickling hemoglobinopathy including HbSS, HbSC
- 5.4.4 Donors who are positive for HIV, active hepatitis B (HBV), hepatitis C (HCV) or human Tcell lymphotropic virus (HTLV-I/II)
- 5.4.5 Severe psychiatric illness or mental deficiency sufficiently severe as to make compliance with the donation process unlikely, and making informed consent impossible.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
Official title: Peripheral Blood Stem Cell Allotransplantation For Hematological Malignancies Using Ex Vivo CD34 Selection - a Platform For Adoptive Cellular Therapies
Background:
- Researchers are working to make stem cell transplant procedures safer and more effective. One complication of transplants is graft-versus-host disease (GVHD). This complication happens when certain white blood cells from the donor attack the recipient's own body. Researchers want to test a blood separator machine that may help remove more of the donor's white blood cells before transplant. They will study donors and recipients during stem cell transplant to see how well this process can prevent GVHD and other complications.
Read more
Objectives:
- To see if a new blood separator machine can improve outcomes of stem cell transplants.
Eligibility:
- Individuals between 10 and 75 years of age who are having a stem cell transplant for leukemia or other blood-related cancers.
- Donors for the stem cell transplant.
Design:
- Recipients and donors will be screened with a physical exam and medical history.
- Donors will have two blood collection procedures. The first will collect only white blood cells, and return the rest of the blood. After the first collection, participants will have filgrastim injections to help their stem cells enter their blood. Then, they will have a second blood collection for the stem cells.
- Recipients will have radiation and chemotherapy to prepare for the stem cell transplant. They will then have the stem cell transplant with the donor cells that have been treated with the blood separator machine.
- Recipients will be monitored closely after the procedure. They may receive some of their donor's white blood cells if needed to fight serious infections.
- Recipients will have the regular standard of care after their transplant. Blood samples will be taken and any side effects will be monitored and treated.
Read the detailed description
Peripheral blood stem cell transplant research carried out by the NHLBI BMT Unit focus on transplant techniques designed to decrease graft versus host disease (GVHD), increase the graft-versus-leukemia (GVL) effect and reduce the risk of post-transplant graft rejection.
Through incremental transplant clinical trials we have shown that by controlling the stem cell (CD34+ cell) and T lymphocyte (CD3+ cell) dose, severe GVHD can be reduced whilst beneficial GVL effects can be preserved. We found that T cell depleted transplants using the Nexell/Baxter Isolex 300i system and subsequently, the Miltenyi CliniMACS[registered] CD34+ system to obtain high CD34+ doses depleted of lymphocytes were safe to administer and associated with less severe acute GVHD and promising response rates and overall survival. Our previous trials have helped us to create the transplant environment (significant lymphodepletion and minimal post transplant immunosuppression) that make for an ideal platform for adoptive cellular immunotherapy. Adoptive cell transfer is the passive transfer of immune cells, into a new recipient host with the goal of transferring the immunologic functionality and characteristics into the new host.
This protocol is designed to evaluate the safety and efficacy of the Miltenyi CliniMACS[registered] CD 34 selection system in HLA-matched sibling allogeneic peripheral blood stem cell transplant. The manipulation of the graft is the primary research intervention, subject to IDE# 15632, and all other aspects of clinical management on this protocol are standard care. The target CD34+ dose range will be >3 x 10(6)/kg and the target CD3+ dose range will be 5 x 10(4)/kg to 1 x 10(6)/kg. Once we demonstrate adequacy of this platform for engraftment and absence of significant GVHD in ten consecutive recipients, we will seek IRB permission to proceed with planned adoptive cellular therapies.
The protocol will accrue up to 96 transplant recipients aged 10-80 with a hematological malignancy and their HLA-matched sibling donors, in whom allogeneic stem cell transplantation from an HLA-matched sibling would be routinely indicated. Diagnostic categories will include acute and chronic leukemia, myelodysplastic syndromes, lymphomas, multiple myeloma and myeloproliferative syndromes.
Subjects will receive a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m(2) total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS[registered] system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
The overall objective is to assess the feasibility of using this system as a platform for cellular immunotherapy initiatives. The primary study endpoint will be overall survival at day +200. Stopping criteria for safety will monitor non-relapse mortality at day +200 and late disease free survival at 2 years. Secondary endpoints will be standard transplant outcome variables such as non-hematologic toxicity, incidence and severity of acute and chronic GVHD and relapse of disease.
- Conditions
- MDS (Myelodysplastic Syndrome), Myeloproliferative Disorder, Lymphoma, Non-Hodgkin, ALL (Acute B-Lymphoblastic Leukemia), AML (Acute Myelogenous Leukemia
- Being tested
- Graft Manipulation (CD34+ Selection)
- Keywords
- Acute Lymphoblastic Leukemia (ALL), Acute Myelogenous Leukemia (AML), Chronic Lymphocytic Leukemia (CLL), Chronic Myelogenous Leukemia (CML), Myelodysplastic Syndrome (MDS)
- Medical subject headings
- Anemia, Refractory, with Excess of Blasts, Myeloproliferative Disorders, Lymphoma, Non-Hodgkin, Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Lymphocytic, Chronic, B-Cell, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Graft Manipulation (CD34+ Selection)Device
| Study group | Role | What they receive |
|---|---|---|
| CD34+ cell positively selected graft stem cell recipient | Experimental | Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity. Receives: Device: Graft Manipulation (CD34+ Selection) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Survival
Determine the rate of overall survival at 200 day
Measured: 200 days
Secondary outcomes (12)
Disease Free Survival at 2 Years
Disease Free Survival at 2 years
Measured: 2 years
Disease Progression
Incidence of relapse / disease progression
Measured: 3 years
Incidence of Acute GVHD
Participants who develop acute GVHD
Measured: 100 days
Incidence of Chronic GVHD
Participants who develop chronic GVHD
Measured: 3 years
Median Time to ANC>500/mm3
Time in days
Measured: 3 years
Median Time to Platelets>20K/mm3
Time in days
Measured: 3 years
Severity of Acute GVHD
Grade I
Measured: 100 days
Severity of Acute GVHD
Grade II
Measured: 100 days
Severity of Acute GVHD
Grade III
Measured: 100 days
Severity of Acute GVHD
Grade IV
Measured: 100 days
Severity of Chronic GVHD
Extensive
Measured: 3 years
Severity of Chronic GVHD
Limited
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jun 2013 (estimated)
- Study start29 May 2013
- Primary completion23 May 2018
- Study completion28 Jun 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 (3)Papers the sponsor has linked to this study.
- Other trials for MDS (Myelodysplastic Syndrome)Studies of the same condition, open and under way.
- More trials run by National Heart, Lung, and Blood Institute (NHLBI)The 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 41 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Collaborators
- Children's National Research Institute
- Responsible party
- Sponsor
- Investigators
- Sawa Ito, M.D., Principal investigator, National Heart, Lung, and Blood Institute (NHLBI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01866839
- Sponsor's protocol ID
- 130144
- Other IDs
- 13-H-0144
- Status last verified
- 28 Jun 2018
- First submitted
- 29 May 2013
- Last updated
- 21 Jul 2020
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
Publications
Armitage JO. Bone marrow transplantation. N Engl J Med. 1994 Mar 24;330(12):827-38. doi: 10.1056/NEJM199403243301206. No abstract available.
PubMed 8114836Barrett AJ. Graft-versus-host disease: basic considerations. Recent Results Cancer Res. 1993;132:185-95. doi: 10.1007/978-3-642-84899-5_19. No abstract available.
PubMed 8265860Appelbaum FR. Haematopoietic cell transplantation as immunotherapy. Nature. 2001 May 17;411(6835):385-9. doi: 10.1038/35077251.
PubMed 11357147
Study at a glance
From the registry record, last updated by the sponsor on 21 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Peripheral Blood Stem Cell Allotransplantation For Hematological Malignancies Using Ex Vivo CD34 Selection - a Platform For Adoptive Cellular Therapies
- ClinicalTrials.gov ID
- NCT01866839
- Acronym / sponsor ID
- 130144
- Conditions
- MDS (Myelodysplastic Syndrome), Myeloproliferative Disorder, Lymphoma, Non-Hodgkin, ALL (Acute B-Lymphoblastic Leukemia), AML (Acute Myelogenous Leukemia
- Keywords
- Acute Lymphoblastic Leukemia (ALL), Acute Myelogenous Leukemia (AML), Chronic Lymphocytic Leukemia (CLL), Chronic Myelogenous Leukemia (CML), Myelodysplastic Syndrome (MDS)
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 41 participants (actual)
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)US National Institutes of Health
- Collaborators
- Children's National Research Institute
- Study start
- 29 May 2013
- Primary completion
- 23 May 2018 (actual)
- Study completion
- 28 Jun 2018 (actual)
- First posted
- 3 Jun 2013 (estimated)
- Results
- Posted 26 May 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Background:
- Researchers are working to make stem cell transplant procedures safer and more effective. One complication of transplants is graft-versus-host disease (GVHD). This complication happens when certain white blood cells from the donor attack the recipient's own body. Researchers want to test a blood separator machine that may help remove more of the donor's white blood cells before transplant. They will study donors and recipients during stem cell transplant to see how well this process can prevent GVHD and other complications.
Objectives:
- To see if a new blood separator machine can improve outcomes of stem cell transplants.
Eligibility:
- Individuals between 10 and 75 years of age who are having a stem cell transplant for leukemia or other blood-related cancers.
- Donors for the stem cell transplant.
Design:
- Recipients and donors will be screened with a physical exam and medical history.
- Donors will have two blood collection procedures. The first will collect only white blood cells, and return the rest of the blood. After the first collection, participants will have filgrastim injections to help their stem cells enter their blood. Then, they will have a second blood collection for the stem cells.
- Recipients will have radiation and chemotherapy to prepare for the stem cell transplant. They will then have the stem cell transplant with the donor cells that have been treated with the blood separator machine.
- Recipients will be monitored closely after the procedure. They may receive some of their donor's white blood cells if needed to fight serious infections.
- Recipients will have the regular standard of care after their transplant. Blood samples will be taken and any side effects will be monitored and treated.
Detailed description
Peripheral blood stem cell transplant research carried out by the NHLBI BMT Unit focus on transplant techniques designed to decrease graft versus host disease (GVHD), increase the graft-versus-leukemia (GVL) effect and reduce the risk of post-transplant graft rejection.
Through incremental transplant clinical trials we have shown that by controlling the stem cell (CD34+ cell) and T lymphocyte (CD3+ cell) dose, severe GVHD can be reduced whilst beneficial GVL effects can be preserved. We found that T cell depleted transplants using the Nexell/Baxter Isolex 300i system and subsequently, the Miltenyi CliniMACS[registered] CD34+ system to obtain high CD34+ doses depleted of lymphocytes were safe to administer and associated with less severe acute GVHD and promising response rates and overall survival. Our previous trials have helped us to create the transplant environment (significant lymphodepletion and minimal post transplant immunosuppression) that make for an ideal platform for adoptive cellular immunotherapy. Adoptive cell transfer is the passive transfer of immune cells, into a new recipient host with the goal of transferring the immunologic functionality and characteristics into the new host.
This protocol is designed to evaluate the safety and efficacy of the Miltenyi CliniMACS[registered] CD 34 selection system in HLA-matched sibling allogeneic peripheral blood stem cell transplant. The manipulation of the graft is the primary research intervention, subject to IDE# 15632, and all other aspects of clinical management on this protocol are standard care. The target CD34+ dose range will be >3 x 10(6)/kg and the target CD3+ dose range will be 5 x 10(4)/kg to 1 x 10(6)/kg. Once we demonstrate adequacy of this platform for engraftment and absence of significant GVHD in ten consecutive recipients, we will seek IRB permission to proceed with planned adoptive cellular therapies.
The protocol will accrue up to 96 transplant recipients aged 10-80 with a hematological malignancy and their HLA-matched sibling donors, in whom allogeneic stem cell transplantation from an HLA-matched sibling would be routinely indicated. Diagnostic categories will include acute and chronic leukemia, myelodysplastic syndromes, lymphomas, multiple myeloma and myeloproliferative syndromes.
Subjects will receive a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m(2) total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS[registered] system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
The overall objective is to assess the feasibility of using this system as a platform for cellular immunotherapy initiatives. The primary study endpoint will be overall survival at day +200. Stopping criteria for safety will monitor non-relapse mortality at day +200 and late disease free survival at 2 years. Secondary endpoints will be standard transplant outcome variables such as non-hematologic toxicity, incidence and severity of acute and chronic GVHD and relapse of disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CD34+ cell positively selected graft stem cell recipient | Experimental | Device: Graft Manipulation (CD34+ Selection)Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity. |
Who may qualify
- Ages
- 2 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (29 listed)
- 5.1.1 Ages 10-80 years inclusive
- 5.1.2 Any one of the following hematologic conditions, confirmed by pathology, meeting a standard indication for allogeneic stem cell transplant:
- 5.1.2.1 Chronic myelogenous leukemia (CML): Subjects under the age of 21 in chronic phase OR Subjects ages 10-80 in chronic phase who have failed or are intolerant to treatment with second generation tyrosine inhibitors OR Subjects ages 10-80 in accelerated phase or blast transformation. OR
- 5.1.2.2 Acute lymphoblastic leukemia (ALL): any of these categories: Adult ALL including standard risk; Pediatric ALL in first remission with high-risk features (presenting leukocyte count >100,000/cu mm, karyotypes t(9; 22), t4, t19, t11, biphenotypic leukemia). All second or subsequent remissions, primary induction failure, partially responding or untreated relapse. OR
- 5.1.2.3 Acute myelogenous leukemia (AML): AML in first remission - except AML with good risk karyotypes: AML M3 (t15; 17), AML M4Eo (inv 16), c-kit unmutated AML t (8; 21). All AML in second or subsequent remission, primary induction failure and resistant relapse. OR
Key exclusion criteria (14 listed)
- 5.2.1 Major anticipated illness or organ failure incompatible with survival from transplant
- 5.2.2 Severe psychiatric illness or mental deficiency sufficiently severe as to make compliance with the transplant treatment unlikely and making informed consent impossible.
- 5.2.3 Positive pregnancy test for women of childbearing age
- 5.2.4 DLCO adjusted for Hb and ventilation< 50% predicted
- 5.2.5 Left ventricular ejection fraction < 40% (evaluated by ECHO) or < 30% (evaluated by MUGA)
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 |
|---|---|---|
| Graft Manipulation (CD34+ Selection) | Device | CD34+ cell positively selected graft stem cell recipient |
Primary outcome
Overall Survival
Time frame: 200 days
Determine the rate of overall survival at 200 day
12 secondary outcomes
Disease Free Survival at 2 Years
Time frame: 2 years
Disease Progression
Time frame: 3 years
Incidence of Acute GVHD
Time frame: 100 days
Incidence of Chronic GVHD
Time frame: 3 years
Median Time to ANC>500/mm3
Time frame: 3 years
Median Time to Platelets>20K/mm3
Time frame: 3 years
Severity of Acute GVHD
Time frame: 100 days
Severity of Acute GVHD
Time frame: 100 days
Severity of Acute GVHD
Time frame: 100 days
Severity of Acute GVHD
Time frame: 100 days
Severity of Chronic GVHD
Time frame: 3 years
Severity of Chronic GVHD
Time frame: 3 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Jul 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 8114836Armitage JO. Bone marrow transplantation. N Engl J Med. 1994 Mar 24;330(12):827-38. doi: 10.1056/NEJM199403243301206. No abstract available.
- Background: PubMed 8265860Barrett AJ. Graft-versus-host disease: basic considerations. Recent Results Cancer Res. 1993;132:185-95. doi: 10.1007/978-3-642-84899-5_19. No abstract available.
- Background: PubMed 11357147Appelbaum FR. Haematopoietic cell transplantation as immunotherapy. Nature. 2001 May 17;411(6835):385-9. doi: 10.1038/35077251.
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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.
Sawa Ito, M.D. principal investigator
National Heart, Lung, and Blood Institute (NHLBI)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Graft Manipulation (CD34+ Selection) in people with MDS (Myelodysplastic Syndrome) and Myeloproliferative Disorder, run by National Heart, Lung, and Blood Institute (NHLBI) 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 29 inclusion and 14 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 21 Jul 2020. 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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 26 May 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?
Overall Survival (time frame: 200 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 NCT01866839, which also lists 3 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 21 Jul 2020.