Myelodysplastic Syndrome (MDS)Phase 1/2NCT01517035
Improving Blood Stem Cell Collection and Transplant Procedures
A Phase 1/2 study testing Stem Cell Transplantation in people with Myelodysplastic Syndrome (MDS) and Chronic Myelogenous Leukemia, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
Trial summary
Background: People who have some kinds of cancer can benefit from donated bone marrow stem cells. These stem cells help produce healthy bone marrow and slow or stop the spread of abnormal cells. However, stem cells transplants do not always work. Read more about this study
- Who is it for?
- Ages 2 to 80 years
- Conditions the study is for
- Myelodysplastic Syndrome (MDS), Chronic Myelogenous Leukemia, Acute Lymphoblastic Leukemia
- What is being tested?
- Stem Cell Transplantation
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 43 participants
- Where
- 1 siteUnited States
- Study dates
- 13 Jan 2012to 29 Mar 2017
- Phase
- Phase 1/2
- 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 2 to 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients in remission as well as patients with primary induction failure or refractory disease will be considered for inclusion. At the discretion of the PI, patients may continue standard of care treatment options to control their baseline disease burden up to the start of the protocol.
- RECIPIENT
- Ages 10-75 years inclusive
- Chronic myelogenous leukemia (CML):
- Subjects under the age of 21 in chronic phase
- Subjects ages 10-75 in chronic phase who have failed treatment with imatinib, have intolerance to imatinib, or who did not receive imatinib at therapeutic doses within the first 12 months from diagnosis.
Show all 21Show fewer
- Subjects ages 10-75 in accelerated phase or blast transformation.
- Acute lymphoblastic leukemia (ALL): any of these categories: ALL in first remission with high-risk features (presenting leukocyte count >100,000/cu mm, Karyotypes t9; 22, t4, t19, t11, biphenotypic leukemia) All second or subsequent remissions, primary induction failure, partially responding or untreated relapse.
- Acute myelogenous leukemia (AML): AML in first remission - except AML with good risk karyotypes: AML M3 (t15; 17), AML M4Eo (inv 16), AML t (8; 21). All AML in second or subsequent remission, primary induction failure and resistant relapse.
- Myelodysplastic syndromes(MDS): any of these categories - refractory anemia with transfusion dependence, refractory anemia with excess of blasts, transformation to acute leukemia, chronic myelomonocytic leukemia, atypical MDS/myeloproliferative syndromes.
- Myeloproliferative disorders including atypical (Ph negative) chronic myeloid and neutrophilic leukemias, progressing myelofibrosis, and polycythemia vera, essential thrombocythemia in transformation to acute leukemia or with progressive transfusion requirements or pancytopenia.
- Chronic lymphocytic leukemia refractory to fludarabine treatment and with bulky progressive disease or with thrombocytopenia (less than or equal to 100,000/ micro-l) or anemia (less than or equal to 10g/dl) not due to recent chemotherapy.
- Non-Hodgkin s lymphoma including Mantle cell lymphoma relapsing or refractory to standard of care treatments.
- Multiple myeloma, Waldenstroms macroglobulinemia, unresponsive or relapsed following standard of care treatments.
- HLA identical (6/6) related donor.
- 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 consent from minors: the process will be explained to the minor on a level of complexity appropriate for their age and ability to comprehend.
- DONOR
- Related donor, HLA identical (6/6) with recipient
- Weight greater than or equal to 18 kg
- Age greater than or equal to 2 or less than or equal to 80 years old
- 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
- RECIPIENT (any of the following)
- Estimated probability of surviving less than three months
- Major anticipated illness or organ failure incompatible with survival from transplant
- Severe psychiatric illness. Mental deficiency sufficiently severe as to make compliance with the transplant treatment unlikely and making informed consent impossible.
- Positive pregnancy test for women of childbearing age.
- HIV positive
Show all 20Show fewer
- DLCO adjusted for Hb and ventilation < 50% predicted
- Left ventricular ejection fraction < 40% (evaluated by ECHO) or < 30% (evaluated by MUGA)
- AST/SGOT > 10 times ULN (>grade 3, CTCAE)
- Bilirubin > 5 times ULN (>grade 3, CTCAE)
- Creatinine > 4.5 times ULN (>grade 3, CTCAE)
- Prior allogeneic stem cell transplantation
- DONOR (any of the following)
- Pregnant or breast-feeding. Lactating donors are permitted provided breastmilk is discarded during the days filgatrim (G-CSF) is given.
- Unfit to receive G-CSF and undergo apheresis (abnormal blood counts, history of stroke, uncontrolled hypertension)
- Sickling hemaglobinopathy including HbSS, HbAS, HbSC
- Donors who are positive for HIV active hepatitis B (HBV), hepatitis C (HCV) or human T-cell lymphotropic virus (HTLV-I/II)
- Severe psychiatric illness. Mental deficiency sufficiently severe as to make compliance with the BMT treatment unlikely, and making informed consent impossible.
- Age greater than or equal to 80 years old
- Children who weigh less than or equal to 18 kg and are < 2 years of age.
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 CD3, CD19 Depletion and CD34 Selection
Background:
- People who have some kinds of cancer can benefit from donated bone marrow stem cells. These stem cells help produce healthy bone marrow and slow or stop the spread of abnormal cells. However, stem cells transplants do not always work. Also, they may have serious side effects that can cause illness or death. The Bone Marrow Stem Cell Transplant Program is studying methods to make stem cell transplant procedures safer and more effective.
Read more
Objectives:
- To test a new procedure that may improve the success and decrease the side effects of stem cell transplants.
Eligibility:
- Individuals 10 to 75 years of age who have a life-threatening illness that may require a stem cell transplant.
- Healthy siblings who are able to provide stem cells for transplant.
Design:
- Participants will be screened with a medical history, physical exam, and blood and urine tests.
- Donor procedures:
- Stem cell donors will start by having apheresis to donate white blood cells.
- Donors will receive filgrastim shots for 5 days to help move stem cells into the blood for collection.
- Donors will have another round of apheresis to donate the stem cells for transplant.
- Recipient procedures:
- Before the transplant, recipients will have radiation twice a day for 3 days and chemotherapy for 7 days.
- After the radiation and chemotherapy, recipients will receive the stem cells provided by the donor.
- After the transplant, recipients will receive the white blood cells provided by the donor.
- Recipients will be monitored closely for 4 months to study the success of the transplant. They will have more followup visits at least yearly thereafter.
- Recipients will have a research apheresis prior to transplant and at 3 months.
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 CD34+ CliniMACs 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
This protocol is designed to evaluate the safety and efficacy of an improved ex vivo graft manipulation procedure using the Miltenyi CliniMACs CD 34, 3 and 19 selection system in HLA-matched sibling allogeneic peripheral blood stem cell transplant. The manipulation of the graft is the primary research intervention and all other aspects of clinical management on this protocol are the standard of care. The target CD34+ dose range will be 3 times 10(6)/kg to 10 times 10(6)/kg and the target CD3+ dose range will be 5 x 10(4)/kg to 1 times 10(6)/kg. The technique will preserve greater numbers of NK cells. Once we demonstrate adequacy of engraftment in the first ten recipients, we plan an amendment to permit further use of this protocol to serve as a platform for several planned adoptive cellular therapy initiatives.
The protocol will accrue up to 44 subjects aged 10-75 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 prepared using the Miltenyi CliniMACS system for CD34, 3 \& 19 selection. Older subjects will receive a lower dose of irradiation (600 cGy) without lung shielding 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 plus 200. Non-relapse mortality at day plus 200 will be monitored for safety. 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
- Myelodysplastic Syndrome (MDS), Chronic Myelogenous Leukemia, Acute Lymphoblastic Leukemia, CML (Chronic Myelogenous Leukemia, CLL (Chronic Lymphocytic Leukemia)
- Being tested
- Stem Cell Transplantation
- Keywords
- Peripheral Blood Stem Cell Transplantation, Stem Cell Transplant, Transplant, Myelodyplastic Syndrome, MDS, Leukemia
- Medical subject headings
- Myelodysplastic Syndromes, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, B-Cell, Leukemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Stem Cell TransplantationProcedure or surgery
Myeloablative conditioning (Flu/Cy/TBI) followed by CD3/19/34 selected stem cell graft.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Myeloablative conditioning (Flu/Cy/TBI) followed by CD3/19/34 selected stem cell graft. Receives: Procedure: Stem Cell Transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary endpoint is all cause mortality at day 200, and the proportion of subjects who have survived at day 200 will be used to determine the sample size.
Measured: 200 days
Secondary outcomes (5)
Cumulative incidence of relapse
Measured: 3 years
Secondary Outcome Measure: Standard transplant outcome variables such as relapse, non-relapse mortality, neutrophil and platelet recovery kinetics, incidence and severity of acute GVHD and chronic GVHD. Technical success rates and the use of DLI...
Non Relapse Mortality
Measured: 3 years
Days till ANC greater than 500/uL
Measured: variable
Days till Platelet greater than 20 k/ul
Measured: variable
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jan 2012 (estimated)
- Study start13 Jan 2012
- Primary completion29 Mar 2017
- Study completion29 Mar 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Myelodysplastic Syndrome (MDS)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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 43 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Minocher M Battiwalla, M.D., Principal investigator, National Heart, Lung, and Blood Institute (NHLBI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01517035
- Sponsor's protocol ID
- 120028
- Other IDs
- 12-H-0028
- Status last verified
- 29 Mar 2017
- First submitted
- 24 Jan 2012
- Last updated
- 5 Jul 2018
- Results posted
- No
Publications
Muller AM, Linderman JA, Florek M, Miklos D, Shizuru JA. Allogeneic T cells impair engraftment and hematopoiesis after stem cell transplantation. Proc Natl Acad Sci U S A. 2010 Aug 17;107(33):14721-6. doi: 10.1073/pnas.1009220107. Epub 2010 Aug 2.
PubMed 20679222Maury S, Lemoine FM, Hicheri Y, Rosenzwajg M, Badoual C, Cherai M, Beaumont JL, Azar N, Dhedin N, Sirvent A, Buzyn A, Rubio MT, Vigouroux S, Montagne O, Bories D, Roudot-Thoraval F, Vernant JP, Cordonnier C, Klatzmann D, Cohen JL. CD4+CD25+ regulatory T cell depletion improves the graft-versus-tumor effect of donor lymphocytes after allogeneic hematopoietic stem cell transplantation. Sci Transl Med. 2010 Jul 21;2(41):41ra52. doi: 10.1126/scitranslmed.3001302.
PubMed 20650872Thomson KJ, Morris EC, Milligan D, Parker AN, Hunter AE, Cook G, Bloor AJ, Clark F, Kazmi M, Linch DC, Chakraverty R, Peggs KS, Mackinnon S. T-cell-depleted reduced-intensity transplantation followed by donor leukocyte infusions to promote graft-versus-lymphoma activity results in excellent long-term survival in patients with multiply relapsed follicular lymphoma. J Clin Oncol. 2010 Aug 10;28(23):3695-700. doi: 10.1200/JCO.2009.26.9100. Epub 2010 Jul 6.
PubMed 20606089
Study at a glance
From the registry record, last updated by the sponsor on 5 Jul 2018. 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 CD3, CD19 Depletion and CD34 Selection
- ClinicalTrials.gov ID
- NCT01517035
- Acronym / sponsor ID
- 120028
- Conditions
- Myelodysplastic Syndrome (MDS), Chronic Myelogenous Leukemia, Acute Lymphoblastic Leukemia, CML (Chronic Myelogenous Leukemia, CLL (Chronic Lymphocytic Leukemia)
- Keywords
- Peripheral Blood Stem Cell Transplantation, Stem Cell Transplant, Transplant, Myelodyplastic Syndrome, MDS, Leukemia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 43 participants (actual)
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)US National Institutes of Health
- Study start
- 13 Jan 2012
- Primary completion
- 29 Mar 2017 (actual)
- Study completion
- 29 Mar 2017 (actual)
- First posted
- 25 Jan 2012 (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.
Background:
- People who have some kinds of cancer can benefit from donated bone marrow stem cells. These stem cells help produce healthy bone marrow and slow or stop the spread of abnormal cells. However, stem cells transplants do not always work. Also, they may have serious side effects that can cause illness or death. The Bone Marrow Stem Cell Transplant Program is studying methods to make stem cell transplant procedures safer and more effective.
Objectives:
- To test a new procedure that may improve the success and decrease the side effects of stem cell transplants.
Eligibility:
- Individuals 10 to 75 years of age who have a life-threatening illness that may require a stem cell transplant.
- Healthy siblings who are able to provide stem cells for transplant.
Design:
- Participants will be screened with a medical history, physical exam, and blood and urine tests.
- Donor procedures:
- Stem cell donors will start by having apheresis to donate white blood cells.
- Donors will receive filgrastim shots for 5 days to help move stem cells into the blood for collection.
- Donors will have another round of apheresis to donate the stem cells for transplant.
- Recipient procedures:
- Before the transplant, recipients will have radiation twice a day for 3 days and chemotherapy for 7 days.
- After the radiation and chemotherapy, recipients will receive the stem cells provided by the donor.
- After the transplant, recipients will receive the white blood cells provided by the donor.
- Recipients will be monitored closely for 4 months to study the success of the transplant. They will have more followup visits at least yearly thereafter.
- Recipients will have a research apheresis prior to transplant and at 3 months.
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 CD34+ CliniMACs 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
This protocol is designed to evaluate the safety and efficacy of an improved ex vivo graft manipulation procedure using the Miltenyi CliniMACs CD 34, 3 and 19 selection system in HLA-matched sibling allogeneic peripheral blood stem cell transplant. The manipulation of the graft is the primary research intervention and all other aspects of clinical management on this protocol are the standard of care. The target CD34+ dose range will be 3 times 10(6)/kg to 10 times 10(6)/kg and the target CD3+ dose range will be 5 x 10(4)/kg to 1 times 10(6)/kg. The technique will preserve greater numbers of NK cells. Once we demonstrate adequacy of engraftment in the first ten recipients, we plan an amendment to permit further use of this protocol to serve as a platform for several planned adoptive cellular therapy initiatives.
The protocol will accrue up to 44 subjects aged 10-75 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 prepared using the Miltenyi CliniMACS system for CD34, 3 \& 19 selection. Older subjects will receive a lower dose of irradiation (600 cGy) without lung shielding 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 plus 200. Non-relapse mortality at day plus 200 will be monitored for safety. 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Procedure: Stem Cell TransplantationMyeloablative conditioning (Flu/Cy/TBI) followed by CD3/19/34 selected stem cell graft. |
Who may qualify
- Ages
- 2 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (21 listed)
- Patients in remission as well as patients with primary induction failure or refractory disease will be considered for inclusion. At the discretion of the PI, patients may continue standard of care treatment options to control their baseline disease burden up to the start of the protocol.
- RECIPIENT
- Ages 10-75 years inclusive
- Subjects under the age of 21 in chronic phase
- Subjects ages 10-75 in chronic phase who have failed treatment with imatinib, have intolerance to imatinib, or who did not receive imatinib at therapeutic doses within the first 12 months from diagnosis.
Key exclusion criteria (20 listed)
- RECIPIENT (any of the following)
- Estimated probability of surviving less than three months
- Major anticipated illness or organ failure incompatible with survival from transplant
- Severe psychiatric illness. Mental deficiency sufficiently severe as to make compliance with the transplant treatment unlikely and making informed consent impossible.
- Positive pregnancy test for women of childbearing age.
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 |
|---|---|---|
| Stem Cell TransplantationMyeloablative conditioning (Flu/Cy/TBI) followed by CD3/19/34 selected stem cell graft. | Procedure or surgery | 1 |
Primary outcome
The primary endpoint is all cause mortality at day 200, and the proportion of subjects who have survived at day 200 will be used to determine the sample size.
Time frame: 200 days
5 secondary outcomes
Cumulative incidence of relapse
Time frame: 3 years
Secondary Outcome Measure: Standard transplant outcome variables such as relapse, non-relapse mortality, neutrophil and platelet recovery kinetics, incidence and severity of acute GVHD and chronic GVHD. Technical success rates and the use of DLI...
Non Relapse Mortality
Time frame: 3 years
Days till ANC greater than 500/uL
Time frame: variable
Days till Platelet greater than 20 k/ul
Time frame: variable
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jul 2018.
- Background: PubMed 20679222Muller AM, Linderman JA, Florek M, Miklos D, Shizuru JA. Allogeneic T cells impair engraftment and hematopoiesis after stem cell transplantation. Proc Natl Acad Sci U S A. 2010 Aug 17;107(33):14721-6. doi: 10.1073/pnas.1009220107. Epub 2010 Aug 2.
- Background: PubMed 20650872Maury S, Lemoine FM, Hicheri Y, Rosenzwajg M, Badoual C, Cherai M, Beaumont JL, Azar N, Dhedin N, Sirvent A, Buzyn A, Rubio MT, Vigouroux S, Montagne O, Bories D, Roudot-Thoraval F, Vernant JP, Cordonnier C, Klatzmann D, Cohen JL. CD4+CD25+ regulatory T cell depletion improves the graft-versus-tumor effect of donor lymphocytes after allogeneic hematopoietic stem cell transplantation. Sci Transl Med. 2010 Jul 21;2(41):41ra52. doi: 10.1126/scitranslmed.3001302.
- Background: PubMed 20606089Thomson KJ, Morris EC, Milligan D, Parker AN, Hunter AE, Cook G, Bloor AJ, Clark F, Kazmi M, Linch DC, Chakraverty R, Peggs KS, Mackinnon S. T-cell-depleted reduced-intensity transplantation followed by donor leukocyte infusions to promote graft-versus-lymphoma activity results in excellent long-term survival in patients with multiply relapsed follicular lymphoma. J Clin Oncol. 2010 Aug 10;28(23):3695-700. doi: 10.1200/JCO.2009.26.9100. Epub 2010 Jul 6.
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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.
Minocher M Battiwalla, 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 Stem Cell Transplantation in people with Myelodysplastic Syndrome (MDS) and Chronic Myelogenous Leukemia, 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 21 inclusion and 20 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 5 Jul 2018. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
The primary endpoint is all cause mortality at day 200, and the proportion of subjects who have survived at day 200 will be used to determine the sample size. (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 NCT01517035, 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 5 Jul 2018.