Hematopoietic Stem Cell TransplantPhase 2NCT02350777
T-cell Depleted Hematopoietic Stem Cell Boosts Without Conditioning for Poor Marrow Graft Function Following Allogeneic Hematopoietic Stem Cell Transplantation
A Phase 2 study testing Donor peripheral blood progenitor cells: stimulation, harvesting, isolation and T-cell depletion and CliniMACS Cell Selection System in people with Hematopoietic Stem Cell Transplant and Poor Marrow Graft Function, run by Memorial Sloan Kettering Cancer Center at 1 site in United States.
Trial summary
The purpose of this study is to see if giving the patient stem cells their original donor (boost) after removing the T cells (T cell depleted- TCD boost) without further chemotherapy. The investigators want to see if this can improve bone marrow function. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hematopoietic Stem Cell Transplant, Poor Marrow Graft Function
- What is being tested?
- Donor peripheral blood progenitor cells: stimulation, harvesting, isolation and T-cell depletion and CliniMACS Cell Selection System
- Type of treatment
- Injection or infusion, Medical device, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- 21 Jan 2015to 25 Oct 2017
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients who are diagnosed with PGF are candidates for this trial.
- Patients who underwent transplant at another facility and suffer from PGF will be eligible as well as long as a donor is available. PGF can be primary (no counts recovery after the preparative regimen) or secondary (cytopenia after engraftment has occurred).
- Patients with auto-immune cytopenia with auto antibodies to neutrophils or platelets or positive Coombs test that did not respond to immunosuppressive agents within 3 months from initiation of therapy are eligible as well.
- Persistent cytopenia requiring growth factors and/or blood products AND evidence of hypocellular BM (<25%). Persistent cytopenia (at least 4 weeks period) is defined by presence of TWO of the following:
- ANC <1.0x10^9/L without filgrastim support or any ANC value that requires recurrent support by filgrastim (administered at least once a week).
- Plt<50x10^9/L
Show all 12Show fewer
- Hb<8 or PRBC transfusion dependent (once every 2 weeks or more) with reticulocyte count of < 40x10^9/L.
- This criteria for persistent cytopenia and hypocellular bone marrow does not apply to patients with auto-immune cytopenia, ONLY PGF patients
- Full donor myeloid chimerism. Patients after T cell depletion transplant can have a significant mixed T cell chimerism and this can affect the testing of marrow chimerism. In this case, the neutrophil chimerism will be used to determine eligibility for this trial. Patients will be excluded if neutrophils are less than 90% donor cells; a higher percentage of host cells could be due to relapse or impending relapse.
- Age: pediatrics and adults patients. No age exclusion.
- Each patient must be willing to participate as a research subject and must sign an informed consent form.
- For infections and end organs related criteria (at time of TCD boost administration) see table in protocol.
Who cannot join
- Patients will be excluded from the trial if at time of enrollment:
- Evidence of relapsed disease by morphologic, cytogenetic or molecular diagnostic tools.
- Hypersplenism documented by imaging study (US or CT)
- Pregnant women
- Patient who underwent TCD boost without counts recovery and are considered for another TCD boost will be treated off protocol.
Trial locations and contacts
1 site in 1 country.
United States1 site
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
About this study
Official title: Phase II Trial of T-cell Depleted Hematopoietic Stem Cell Boosts Without Conditioning for Poor Marrow Graft Function Following Allogeneic Hematopoietic Stem Cell Transplantation
The purpose of this study is to see if giving the patient stem cells their original donor (boost) after removing the T cells (T cell depleted- TCD boost) without further chemotherapy. The investigators want to see if this can improve bone marrow function. This would also improve the patients white blood counts, red blood counts and platelets. This may make the patients chances of improving and surviving better. The investigators will also be looking at the short term side effects and risks of the TCD boost.
- Being tested
- Donor peripheral blood progenitor cells: stimulation, harvesting, isolation and T-cell depletion, CliniMACS Cell Selection System
- Keywords
- T-cell Depleted Hematopoietic Stem Cell, Allogeneic Hematopoietic Stem Cell Transplantation, CliniMACS Cell Selection System, 14-228, HSCT
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Donor peripheral blood progenitor cells: stimulation, harvesting, isolation and T-cell depletionProcedure or surgery
For related donors, beginning 5-6 days before the day of HPC(A) or HPC (M) TCD boost infusion, the normal donor will receive GCSF per institutional guidelines. On the fifth and sixth days of this course of G-CSF, the donor will undergo daily leukapheresis designed to provide a minimum of 5x10^6 CD34+ cells/kg of the transplant recipient's weight. For unrelated donors, the G-CSF will be administered and the leukapheresis obtained according to the National Marrow Donor Program protocol IND, and institutional guidelines. Mononuclear cell fractions (i.e., CD34+ cells) collected on the fourth and fifth days will be pooled.
CliniMACS Cell Selection SystemDevice
| Study group | Role | What they receive |
|---|---|---|
| active infection and/or organ compromise or GVHD. | Experimental | This protocol includes two single-arm phase II trials to assess the efficacy and confirm the safety of administration of TCD stem cell boost or bone marrow (BM) HPC (M) boost from the original donor for patients with poor graft function after allogeneic hematopoietic stem cell transplantation. Receives: Procedure: Donor peripheral blood progenitor cells: stimulation, harvesting, isolation and T-cell depletion; Device: CliniMACS Cell Selection System |
| active infection, active or controlled GVHD &/or organ compro | Experimental | This protocol includes two single-arm phase II trials to assess the efficacy and confirm the safety of administration of TCD stem cell boost or bone marrow (BM) HPC (M) boost from the original donor for patients with poor graft function after allogeneic hematopoietic stem cell transplantation. Receives: Procedure: Donor peripheral blood progenitor cells: stimulation, harvesting, isolation and T-cell depletion; Device: CliniMACS Cell Selection System |
What is being measured
The primary outcome is the main question the study is designed to answer.
Peripheral Blood Counts Recovery (Response Will be Defined by Stable Blood Counts (ANC>1000, Hb>8 With Absolute Reticulocyte Count>20x10^9/L ,and Plt>50,000) Without Support of Blood Product Transfusions and/or Growth Factors.
will be documented by CBC checks every 2 weeks. Response will be defined by stable blood counts (ANC>1000, Hb>8 with absolute reticulocyte count>20x10^9/L ,and Plt>50,000) without support of blood product transfusions and/or growth factors.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jan 2015 (estimated)
- Study start21 Jan 2015
- Primary completion25 Oct 2017
- Study completion25 Oct 2017
Why it stopped: Lack of accrual
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.
- Other trials for Hematopoietic Stem Cell TransplantStudies of the same condition, open and under way.
- More trials run by Memorial Sloan Kettering Cancer CenterThe 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
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Roni Tamari, MD, Principal investigator, Memorial Sloan Kettering Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02350777
- Sponsor's protocol ID
- 14-228
- Status last verified
- Oct 2017
- First submitted
- 21 Jan 2015
- Last updated
- 9 Oct 2018
- Results posted
- Yes, on ClinicalTrials.gov
Study at a glance
From the registry record, last updated by the sponsor on 9 Oct 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Trial of T-cell Depleted Hematopoietic Stem Cell Boosts Without Conditioning for Poor Marrow Graft Function Following Allogeneic Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT02350777
- Acronym / sponsor ID
- 14-228
- Keywords
- T-cell Depleted Hematopoietic Stem Cell, Allogeneic Hematopoietic Stem Cell Transplantation, CliniMACS Cell Selection System, 14-228, HSCT
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Lack of accrual
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Memorial Sloan Kettering Cancer CenterUniversity, hospital or other
- Study start
- 21 Jan 2015 (actual)
- Primary completion
- 25 Oct 2017 (actual)
- Study completion
- 25 Oct 2017 (actual)
- First posted
- 30 Jan 2015 (estimated)
- Results
- Posted 6 Jun 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to see if giving the patient stem cells their original donor (boost) after removing the T cells (T cell depleted- TCD boost) without further chemotherapy. The investigators want to see if this can improve bone marrow function. This would also improve the patients white blood counts, red blood counts and platelets. This may make the patients chances of improving and surviving better. The investigators will also be looking at the short term side effects and risks of the TCD boost.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| active infection and/or organ compromise or GVHD. | Experimental | Procedure: Donor peripheral blood progenitor cells: stimulation, harvesting, isolation and T-cell depletion, Device: CliniMACS Cell Selection SystemThis protocol includes two single-arm phase II trials to assess the efficacy and confirm the safety of administration of TCD stem cell boost or bone marrow (BM) HPC (M) boost from the original donor for patients with poor graft function after allogeneic hematopoietic stem cell transplantation. |
| active infection, active or controlled GVHD &/or organ compro | Experimental | Procedure: Donor peripheral blood progenitor cells: stimulation, harvesting, isolation and T-cell depletion, Device: CliniMACS Cell Selection SystemThis protocol includes two single-arm phase II trials to assess the efficacy and confirm the safety of administration of TCD stem cell boost or bone marrow (BM) HPC (M) boost from the original donor for patients with poor graft function after allogeneic hematopoietic stem cell transplantation. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (12 listed)
- Patients who are diagnosed with PGF are candidates for this trial.
- Patients who underwent transplant at another facility and suffer from PGF will be eligible as well as long as a donor is available. PGF can be primary (no counts recovery after the preparative regimen) or secondary (cytopenia after engraftment has occurred).
- Patients with auto-immune cytopenia with auto antibodies to neutrophils or platelets or positive Coombs test that did not respond to immunosuppressive agents within 3 months from initiation of therapy are eligible as well.
- Persistent cytopenia requiring growth factors and/or blood products AND evidence of hypocellular BM (<25%). Persistent cytopenia (at least 4 weeks period) is defined by presence of TWO of the following:
- This criteria for persistent cytopenia and hypocellular bone marrow does not apply to patients with auto-immune cytopenia, ONLY PGF patients
Key exclusion criteria (5 listed)
- Evidence of relapsed disease by morphologic, cytogenetic or molecular diagnostic tools.
- Hypersplenism documented by imaging study (US or CT)
- Pregnant women
- Patient who underwent TCD boost without counts recovery and are considered for another TCD boost will be treated off protocol.
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 |
|---|---|---|
| Donor peripheral blood progenitor cells: stimulation, harvesting, isolation and T-cell depletionFor related donors, beginning 5-6 days before the day of HPC(A) or HPC (M) TCD boost infusion, the normal donor will receive GCSF per institutional guidelines. On the fifth and sixth days of this course of G-CSF, the donor will undergo daily leukapheresis designed to provide a minimum of 5x10^6 CD34+ cells/kg of the transplant recipient's weight. For unrelated donors, the G-CSF will be administered and the leukapheresis obtained according to the National Marrow Donor Program protocol IND, and institutional guidelines. Mononuclear cell fractions (i.e., CD34+ cells) collected on the fourth and fifth days will be pooled. | Procedure or surgery | active infection and/or organ compromise or GVHD., active infection, active or controlled GVHD &/or organ compro |
| CliniMACS Cell Selection System | Device | active infection and/or organ compromise or GVHD., active infection, active or controlled GVHD &/or organ compro |
Primary outcome
Peripheral Blood Counts Recovery (Response Will be Defined by Stable Blood Counts (ANC>1000, Hb>8 With Absolute Reticulocyte Count>20x10^9/L ,and Plt>50,000) Without Support of Blood Product Transfusions and/or Growth Factors.
Time frame: 1 year
will be documented by CBC checks every 2 weeks. Response will be defined by stable blood counts (ANC>1000, Hb>8 with absolute reticulocyte count>20x10^9/L ,and Plt>50,000) without support of blood product transfusions and/or growth factors.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Oct 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Memorial Sloan Kettering Cancer CenterLink provided by the sponsor.
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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.
Roni Tamari, MD principal investigator
Memorial Sloan Kettering Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Donor peripheral blood progenitor cells: stimulation, harvesting, isolation and T-cell depletion and CliniMACS Cell Selection System in people with Hematopoietic Stem Cell Transplant and Poor Marrow Graft Function, run by Memorial Sloan Kettering Cancer Center 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 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: stopped early, as of the sponsor's last update on 9 Oct 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 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 6 Jun 2018). 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?
Peripheral Blood Counts Recovery (Response Will be Defined by Stable Blood Counts (ANC>1000, Hb>8 With Absolute Reticulocyte Count>20x10^9/L ,and Plt>50,000) Without Support of Blood Product Transfusions and/or Growth Factors. (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Oct 2018.