Acute Myeloid LeukemiaPhase 2BigeminyNCT04166929
Haploidentical Stem Cell Transplant With or Without NK Cell Infusion in AML and MDS
A Phase 2 study testing Haplo SCT with NK cells and Haplo SCT in people with Acute Myeloid Leukemia and Myelodysplasia, run by Fondazione Policlinico Universitario Agostino Gemelli IRCCS at 1 site in Italy.
Trial summary
Relapse after an allogeneic hematopoietic stem cell transplantation (HSCT) is high in patients with advanced AML, in the 50% range. NK cells have been shown to possess significant anti-leukemic activity and may be used to reduce the incidence of relapse in patients with advanced AML. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Acute Myeloid Leukemia, Myelodysplasia
- What is being tested?
- Haplo SCT with NK cells and Haplo SCT
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteItaly
- Study dates
- 15 Jan 2020to 28 Sep 2023
- Phase
- Phase 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 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of AML or MDS
- Patients not in remission at the time of transplant
- Age 18-75
- ECOG performance status <2
- Availability of a family HLA haploidentical donor, eligible to donate both marrow cells and unstimulated lymphocytes (1 apheresis procedure at day +7) .
Who cannot join
- Positive serologic markers for human immunodeficiency virus (HIV)
- Acute hepatitis B virus (HBV) or acute hepatitis C virus (HCV) infection
Trial locations and contacts
1 site in 1 country.
Italy1 site
Fondazione Policlinico Universitario A. Gemelli IRCCS
Roma, RM, 00168
About this study
Official title: Haploidentical Stem Cell Transplantation With or Without NK Cell Infusion in Refractory/Relapsed Acute Myeloid Leukemia and Myelodysplastic Syndrome:The BigEMINy Study
Relapse after an allogeneic hematopoietic stem cell transplantation (HSCT) is high in patients with advanced AML, in the 50% range. NK cells have been shown to possess significant anti-leukemic activity and may be used to reduce the incidence of relapse in patients with advanced AML.
Investigators hypothesize that the administration of a purified boost of NK cells on day +7 post HSCT, will reduce the incidence of relapse from the current 50% to 25%. In a phase III multicenter clinical study, 116 patients will be randomized to receive or not a boost of donor NK cells on day +7 post-HSCT. The first 10 patients in the experimental arm will be analyzed for toxicity. The stopping rule will be a transplant related mortality of more than 50% in the first 20 patients who received NK cells.
- Conditions
- Acute Myeloid Leukemia, Myelodysplasia
- Being tested
- Haplo SCT with NK cells, Haplo SCT
- Medical subject headings
- Leukemia, Myeloid, Acute, Anemia, Refractory, with Excess of Blasts
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Haplo SCT with NK cellsBiological
Also called haplo SCT followed by NK cell infusion
Patients in this arm receive haplo-SCT followed by one boost of NK cells one/two weeks after the transplant. Lymphocyte are collected by apheresis from the donor one-two weeks after BM harvest. CD3 negative/CD56 positive cells are selected by immunomagnetic columns (other lymphocytes are stored to be used as DLI)
Haplo SCTBiological
Also called Standard haplo SCT without subsequent NK cell boost.
Patients in this arm receive a standard haplo-SCT. All administerd therapies are the same as in other arm, except for the NK cell boost..
| Study group | Role | What they receive |
|---|---|---|
| Haplo-stem cell transplant followed by NK infusion | Experimental | Haplo stem cell transplant (from bone marrow or apheresis) is followed by a haplo-NK cell infusion (target dose ≥1*106/kg b.w.) at day 7.
Unstimulated haplo-NK cells are collected through apheresis Mononuclear cells are then subjected to a preliminary negative selection of CD3+ cells and to a subsequent positive selection of CD56+ cells. CD3 negative/CD56 positive cells are infused Receives: Biological: Haplo SCT with NK cells |
| Haplo-stem cell transplant | Active comparator | Patients in this arm receive standard haplo stem cell transplant (from bone marrow or apheresis) transplant without subsequent NK cell infusion. Receives: Biological: Haplo SCT |
What is being measured
The primary outcome is the main question the study is designed to answer.
Leukemia relapse
Cumulative incidence of leukemia relapse.
Measured: 1 year after bone marrow transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2019
- Study start15 Jan 2020
- Primary completion28 Sep 2023
- Study completion28 Sep 2023
Why it stopped: low recruitment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Sponsor type
- University, hospital or other
- Collaborators
- Ministry of Health, Italy
- Responsible party
- Sponsor
- Investigators
- Andrea Bacigalupo, Prof, Principal investigator, Fondazione Policlinico Gemelli IRCCS
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04166929
- Sponsor's protocol ID
- 2588
- Acronym
- Bigeminy
- Status last verified
- May 2021
- First submitted
- 14 Nov 2019
- Last updated
- 4 Oct 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Haploidentical Stem Cell Transplantation With or Without NK Cell Infusion in Refractory/Relapsed Acute Myeloid Leukemia and Myelodysplastic Syndrome:The BigEMINy Study
- ClinicalTrials.gov ID
- NCT04166929
- Acronym / sponsor ID
- Bigeminy / 2588
- Conditions
- Acute Myeloid Leukemia, Myelodysplasia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: low recruitment
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCSUniversity, hospital or other
- Collaborators
- Ministry of Health, Italy
- Study start
- 15 Jan 2020 (actual)
- Primary completion
- 28 Sep 2023 (actual)
- Study completion
- 28 Sep 2023 (actual)
- First posted
- 18 Nov 2019 (actual)
- 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.
Relapse after an allogeneic hematopoietic stem cell transplantation (HSCT) is high in patients with advanced AML, in the 50% range. NK cells have been shown to possess significant anti-leukemic activity and may be used to reduce the incidence of relapse in patients with advanced AML.
Investigators hypothesize that the administration of a purified boost of NK cells on day +7 post HSCT, will reduce the incidence of relapse from the current 50% to 25%. In a phase III multicenter clinical study, 116 patients will be randomized to receive or not a boost of donor NK cells on day +7 post-HSCT. The first 10 patients in the experimental arm will be analyzed for toxicity. The stopping rule will be a transplant related mortality of more than 50% in the first 20 patients who received NK cells.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Haplo-stem cell transplant followed by NK infusion | Experimental | Biological: Haplo SCT with NK cellsHaplo stem cell transplant (from bone marrow or apheresis) is followed by a haplo-NK cell infusion (target dose ≥1*106/kg b.w.) at day 7. Unstimulated haplo-NK cells are collected through apheresis Mononuclear cells are then subjected to a preliminary negative selection of CD3+ cells and to a subsequent positive selection of CD56+ cells. CD3 negative/CD56 positive cells are infused |
| Haplo-stem cell transplant | Active comparator | Biological: Haplo SCTPatients in this arm receive standard haplo stem cell transplant (from bone marrow or apheresis) transplant without subsequent NK cell infusion. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Diagnosis of AML or MDS
- Patients not in remission at the time of transplant
- Age 18-75
- ECOG performance status <2
- Availability of a family HLA haploidentical donor, eligible to donate both marrow cells and unstimulated lymphocytes (1 apheresis procedure at day +7) .
Key exclusion criteria (2 listed)
- Positive serologic markers for human immunodeficiency virus (HIV)
- Acute hepatitis B virus (HBV) or acute hepatitis C virus (HCV) infection
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 |
|---|---|---|
| Haplo SCT with NK cellsAlso: haplo SCT followed by NK cell infusionPatients in this arm receive haplo-SCT followed by one boost of NK cells one/two weeks after the transplant. Lymphocyte are collected by apheresis from the donor one-two weeks after BM harvest. CD3 negative/CD56 positive cells are selected by immunomagnetic columns (other lymphocytes are stored to be used as DLI) | Biological | Haplo-stem cell transplant followed by NK infusion |
| Haplo SCTAlso: Standard haplo SCT without subsequent NK cell boost.Patients in this arm receive a standard haplo-SCT. All administerd therapies are the same as in other arm, except for the NK cell boost.. | Biological | Haplo-stem cell transplant |
Primary outcome
Leukemia relapse
Time frame: 1 year after bone marrow transplant
Cumulative incidence of leukemia relapse.
Results and safety
The study was stopped early (reason given: low recruitment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Oct 2023.
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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.
Andrea Bacigalupo, Prof principal investigator
Fondazione Policlinico Gemelli IRCCS
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 Haplo SCT with NK cells and Haplo SCT in people with Acute Myeloid Leukemia and Myelodysplasia, run by Fondazione Policlinico Universitario Agostino Gemelli IRCCS at 1 site in Italy.
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 5 inclusion and 2 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 4 Oct 2023. 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 Italy. 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?
The study was stopped early (reason given: low recruitment). Results are not available in the source record.
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?
Leukemia relapse (time frame: 1 year after bone marrow transplant). 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 4 Oct 2023.