Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 PositivePhase 1/2NCT01823198

Donor Natural Killer Cells and Donor Stem Cell Transplant in Treating Patients With High Risk Myeloid Malignancies

A Phase 1/2 study testing Aldesleukin, Allogeneic CD56-positive CD3-negative Natural Killer Cells and Allogeneic Hematopoietic Stem Cell Transplantation in people with Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive and Acute Erythroid Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages 7 to 65 years
Conditions the study is for: Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Erythroid Leukemia, Acute Megakaryoblastic Leukemia
What is being tested?: Aldesleukin, Allogeneic CD56-positive CD3-negative Natural Killer Cells and Allogeneic Hematopoietic Stem Cell Transplantation
Type of treatment: Injection or infusion, Surgery or procedure
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 63 participants
Where: 1 siteUnited States
Study dates: 11 Jun 2013to 10 May 2022
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 7 to 65 years
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Acute myeloid leukemia who fail to achieve complete remission with one course of induction chemotherapy or after relapse; patients must have less than 20% bone marrow or peripheral blood blasts
Acute myeloid leukemia in first remission with any of the following high risk features defined as:
Adverse cytogenetics: -5, deletion (del) 5q, -7, del7q, abnormalities involving 3q, 9q, 11q, 20q, 21q, 17, +8 or complex karyotype (> 3 abnormalities)
Preceding myelodysplastic or myeloproliferative syndrome
Presence of high risk molecular abnormalities including FLT3 mutations, DNMT3A, TET2; ras; kit
French-American-British (FAB) monosomy (M)6 or M7 classification

Show all 27Show fewer

Treatment related acute myeloid leukemia (AML)
Residual cytogenetic or molecular abnormalities
Myelodysplastic syndromes with intermediate, high or very high risk Revised International Prognostic Scoring System (R-IPSS) score, chronic myelomonocytic leukemia (CMML) or therapy related myelodysplastic syndromes (MDS)
Chronic myeloid leukemia (CML) which:
Failed to achieve a cytogenetic remission to tyrosine kinase inhibitor treatment or has a cytogenetic relapse
Has ever been in accelerated phase or blast crisis
Patient must have an identified HLA (A,B,C,DR) compatible related or unrelated donor who is age 16 years of age or older and weighs at least 110 pounds for the stem cell donation
Zubrod performance status 0 to 2 or Karnofsky of at least 60
Left ventricular ejection fraction >= 45%; no uncontrolled arrhythmias or uncontrolled symptomatic cardiac disease
Forced expiratory volume in one second (FEV1) >= 50% of expected, corrected for hemoglobin
Forced vital capacity (FVC) >= 50% of expected, corrected for hemoglobin
Diffusing capacity of the lung for carbon monoxide (DLCO) >= 50% of expected, corrected for hemoglobin
Bilirubin =< 1.5 mg/dl (unless Gilbert's syndrome)
Serum glutamate pyruvate transaminase (SGPT) =< 200 IU/ml unless related to patient malignancy
Hepatitis B surface antigen negative and hepatitis C antibody negative
No evidence of chronic active hepatitis or cirrhosis
Patients with a history of hepatitis C, but have a negative viral load, are eligible
The protocol chairman will determine the eligibility of patients related to hepatic abnormalities
Serum creatinine < 1.5 mg%
Patient or patient's legal representative, parent(s) or guardian able to sign informed consent; patients aged 7 to < 18 to provide assent
Pediatric patients (age 7-18 years) will be entered only after 3 adult patients have been entered without dose limiting toxicity

Who cannot join

Uncontrolled infection, not responding to appropriate antimicrobial agents after seven days of therapy; the protocol principal investigator (PI) is the final arbiter of eligibility
Pleural/pericardial effusion or ascites > 1 L
Patients who are known to be human immunodeficiency virus (HIV)-seropositive
Pregnancy: positive pregnancy test in a woman with child bearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization
Women of child bearing potential not willing to use an effective contraceptive measure while on study
Patients who are known to have allergy to mouse proteins

Trial locations and contacts

1 site in 1 country.

United States1 site

M D Anderson Cancer Center
Houston, Texas, 77030

Other trials in United States

About this study

Official title: NK Cells With HLA Compatible Hematopoietic Transplantation for High Risk Myeloid Malignancies

This phase I/II trial studies the side effects and best dose of donor natural killer cells when given together with donor stem cell transplant and to see how well they work in treating patients with myeloid malignancies that are likely to come back or spread. Giving chemotherapy, such as busulfan and fludarabine phosphate, before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells and natural killer cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets.

Read the detailed description

PRIMARY OBJECTIVES:

Assess the safety of infusing ex vivo expanded natural killer (NK) cells in patients receiving busulfan-fludarabine phosphate (fludarabine) with an allogeneic human leukocyte antigen (HLA) matched hematopoietic transplantation for myeloid malignancies. Two sources of NK cells could be studied, depending on what donor source is available: cells from the HLA matched related donor or cells from an unrelated cord blood unit.

II. For each source of NK cells: the maximum tolerated cell dose; the phenotype and function of the ex vivo expanded NK cells and their survival in vivo; the rate of engraftment, graft-vs.-host disease (GVHD), immune reconstitution, relapse rates and survival for patients receiving this regimen will be determined.

OUTLINE: This is a phase I, dose-escalation study of NK cells followed by a phase II study.

Patients receive fludarabine phosphate intravenously (IV) over 1 hour and busulfan IV over 3 hours on days -13 to -10. Patients then receive allogeneic CD56-positive CD3-negative natural killer cells IV over 1 hour on day -8. Patients also receive aldesleukin subcutaneously (SC) once daily (QD) on days -8 to -4. Patients then undergo allogeneic peripheral blood stem cell (PBSC) transplant on day 0.

Conditions: Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Erythroid Leukemia, Acute Megakaryoblastic Leukemia, Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome, Acute Myeloid Leukemia in Remission
Being tested: Aldesleukin, Allogeneic CD56-positive CD3-negative Natural Killer Cells, Allogeneic Hematopoietic Stem Cell Transplantation, Busulfan, Fludarabine Phosphate
Medical subject headings: Leukemia, Myeloid, Accelerated Phase, Leukemia, Erythroblastic, Acute, Leukemia, Megakaryoblastic, Acute, Leukemia, Myeloid, Acute, Blast Crisis, Leukemia, Myelomonocytic, Chronic, Myelodysplastic Syndromes, Leukemia, Myelogenous, Chronic, BCR-ABL Positive

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

AldesleukinBiological
Also called 125-L-Serine-2-133-interleukin 2, Proleukin, r-serHuIL-2, Recombinant Human IL-2
Given SC
Allogeneic CD56-positive CD3-negative Natural Killer CellsBiological
Given IV
Allogeneic Hematopoietic Stem Cell TransplantationProcedure or surgery
Also called Allogeneic Hematopoietic Cell Transplantation, Allogeneic Stem Cell Transplantation, HSC, HSCT
Undergo allogeneic PBSC transplant
BusulfanDrug
Also called 1, 4-Bis[methanesulfonoxy]butane, BUS, Bussulfam, Busulfanum
Given IV
Fludarabine PhosphateDrug
Also called 2-F-ara-AMP, 9H-Purin-6-amine, 2-fluoro-9-(5-O-phosphono-.beta.-D-arabinofuranosyl)-, Beneflur, Fludara
Given IV
Laboratory Biomarker AnalysisOther
Correlative studies
Peripheral Blood Stem Cell TransplantationProcedure or surgery
Also called PBPC transplantation, PBSCT, Peripheral Blood Progenitor Cell Transplantation, Peripheral Stem Cell Support
Undergo allogeneic PBSC transplant
Pharmacological StudyOther
Correlative studies

Study groups in NCT01823198
Study group	Role	What they receive
Treatment (NK cells, PBSC transplant)	Experimental	Patients receive fludarabine phosphate IV over 1 hour and busulfan IV over 3 hours on days -13 to -10. Patients then receive allogeneic CD56-positive CD3-negative natural killer cells IV over 1 hour on day -8. Patients also receive aldesleukin SC QD on days -8 to -4. Patients then undergo allogeneic PBSC transplant on day 0. Receives: Biological: Aldesleukin; Biological: Allogeneic CD56-positive CD3-negative Natural Killer Cells; Procedure: Allogeneic Hematopoietic Stem Cell Transplantation; Drug: Busulfan; Drug: Fludarabine Phosphate; Other: Laboratory Biomarker Analysis; Procedure: Peripheral Blood Stem Cell Transplantation; Other: Pharmacological Study

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Participants Who Experienced Dose-limiting Toxicities (DLT)
Participants that experienced DLT related to the NK Cells post transplant at different dose levels.
Measured: Up to 42 days

Secondary outcomes (2)

Overall Survival
Participants that survived between day of transplant and day of death on different dose levels.
Measured: Up to 2 years
Number of Participants With Grade 3 Toxicities
Number of participants that had grade 3 toxicities up to day 42.
Measured: Up to day 42

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

11 Jun 201310 May 2022

First posted4 Apr 2013 (estimated)
Study start11 Jun 2013
Primary completion10 May 2022
Study completion10 May 2022

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 Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 PositiveStudies of the same condition, open and under way.
More trials run by M.D. Anderson 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 1/2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 63 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01823198
Sponsor's protocol ID: 2012-0819
Other IDs: NCI-2013-00993, RP110553-P3, 2012-0819
Status last verified: Nov 2023
First submitted: 28 Mar 2013
Last updated: 7 Nov 2023
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Study at a glance

From the registry record, last updated by the sponsor on 7 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: NK Cells With HLA Compatible Hematopoietic Transplantation for High Risk Myeloid Malignancies
ClinicalTrials.gov ID: NCT01823198
Acronym / sponsor ID: 2012-0819
Conditions: Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Erythroid Leukemia, Acute Megakaryoblastic Leukemia, Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome, Acute Myeloid Leukemia in Remission
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 63 participants (actual)
Lead sponsor: M.D. Anderson Cancer CenterUniversity, hospital or other
Collaborators: National Cancer Institute (NCI)
Study start: 11 Jun 2013 (actual)
Primary completion: 10 May 2022 (actual)
Study completion: 10 May 2022 (actual)
First posted: 4 Apr 2013 (estimated)
Results: Posted 7 Nov 2023

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

PRIMARY OBJECTIVES:

Assess the safety of infusing ex vivo expanded natural killer (NK) cells in patients receiving busulfan-fludarabine phosphate (fludarabine) with an allogeneic human leukocyte antigen (HLA) matched hematopoietic transplantation for myeloid malignancies. Two sources of NK cells could be studied, depending on what donor source is available: cells from the HLA matched related donor or cells from an unrelated cord blood unit.

OUTLINE: This is a phase I, dose-escalation study of NK cells followed by a phase II study.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Treatment (NK cells, PBSC transplant)	Experimental	Biological: Aldesleukin, Biological: Allogeneic CD56-positive CD3-negative Natural Killer Cells, Procedure: Allogeneic Hematopoietic Stem Cell Transplantation, Drug: Busulfan, Drug: Fludarabine Phosphate, Other: Laboratory Biomarker Analysis, Procedure: Peripheral Blood Stem Cell Transplantation, Other: Pharmacological StudyPatients receive fludarabine phosphate IV over 1 hour and busulfan IV over 3 hours on days -13 to -10. Patients then receive allogeneic CD56-positive CD3-negative natural killer cells IV over 1 hour on day -8. Patients also receive aldesleukin SC QD on days -8 to -4. Patients then undergo allogeneic PBSC transplant on day 0.

Who may qualify

Ages: 7 to 65 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (27 listed)

Acute myeloid leukemia who fail to achieve complete remission with one course of induction chemotherapy or after relapse; patients must have less than 20% bone marrow or peripheral blood blasts
Acute myeloid leukemia in first remission with any of the following high risk features defined as:
Myelodysplastic syndromes with intermediate, high or very high risk Revised International Prognostic Scoring System (R-IPSS) score, chronic myelomonocytic leukemia (CMML) or therapy related myelodysplastic syndromes (MDS)
Chronic myeloid leukemia (CML) which:
Patient must have an identified HLA (A,B,C,DR) compatible related or unrelated donor who is age 16 years of age or older and weighs at least 110 pounds for the stem cell donation

Key exclusion criteria (6 listed)

Uncontrolled infection, not responding to appropriate antimicrobial agents after seven days of therapy; the protocol principal investigator (PI) is the final arbiter of eligibility
Pleural/pericardial effusion or ascites > 1 L
Patients who are known to be human immunodeficiency virus (HIV)-seropositive
Pregnancy: positive pregnancy test in a woman with child bearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization
Women of child bearing potential not willing to use an effective contraceptive measure while on study

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
AldesleukinAlso: 125-L-Serine-2-133-interleukin 2, Proleukin, r-serHuIL-2, Recombinant Human IL-2Given SC	Biological	Treatment (NK cells, PBSC transplant)
Allogeneic CD56-positive CD3-negative Natural Killer CellsGiven IV	Biological	Treatment (NK cells, PBSC transplant)
Allogeneic Hematopoietic Stem Cell TransplantationAlso: Allogeneic Hematopoietic Cell Transplantation, Allogeneic Stem Cell Transplantation, HSC, HSCTUndergo allogeneic PBSC transplant	Procedure or surgery	Treatment (NK cells, PBSC transplant)
BusulfanAlso: 1, 4-Bis[methanesulfonoxy]butane, BUS, Bussulfam, BusulfanumGiven IV	Drug	Treatment (NK cells, PBSC transplant)
Fludarabine PhosphateAlso: 2-F-ara-AMP, 9H-Purin-6-amine, 2-fluoro-9-(5-O-phosphono-.beta.-D-arabinofuranosyl)-, Beneflur, FludaraGiven IV	Drug	Treatment (NK cells, PBSC transplant)
Laboratory Biomarker AnalysisCorrelative studies	Other	Treatment (NK cells, PBSC transplant)
Peripheral Blood Stem Cell TransplantationAlso: PBPC transplantation, PBSCT, Peripheral Blood Progenitor Cell Transplantation, Peripheral Stem Cell SupportUndergo allogeneic PBSC transplant	Procedure or surgery	Treatment (NK cells, PBSC transplant)
Pharmacological StudyCorrelative studies	Other	Treatment (NK cells, PBSC transplant)

Primary outcome

Number of Participants Who Experienced Dose-limiting Toxicities (DLT)
Time frame: Up to 42 days
Participants that experienced DLT related to the NK Cells post transplant at different dose levels.

2 secondary outcomes

Overall Survival
Time frame: Up to 2 years
Number of Participants With Grade 3 Toxicities
Time frame: Up to day 42

Results and safety

Results posted to the registry on 7 Nov 2023, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Phase I: NK Cell Dose Level 1_10^6	6	6
Phase I: NK Cell Dose Level 2_10^7	6	5
Phase I: NK Cell Dose Level 3_ 3x10^7	4	4
Phase I: NK Cell Dose Level 4_ 10^8	4	4
Phase II: NK Cell Dose Level 3_3x10^7	1	1
Phase II: NK Cell Dose Level 4_ 10^8	21	18
Donors	21	18

Primary outcome: Number of Participants Who Experienced Dose-limiting Toxicities (DLT)

Time frame: Up to 42 days. Count of participants. Unit: Participants

Measure	Phase I: NK Cell Dose Level 1_10^6 n = 6	Phase I: NK Cell Dose Level 2_10^7 n = 6	Phase I: NK Cell Dose Level 3_ 3x10^7 n = 4	Phase I: NK Cell Dose Level 4_ 10^8 n = 4	Phase II: NK Cell Dose Level 3_3x10^7 n = 1	Phase II: NK Cell Dose Level 4_ 10^8 n = 21
Group A: NK cells from KIR mismatched haplo donors	2	1	0	0	0	0
Group B: NK cells from KIR mismatched cord blood donors	2	2	2	2	1	12
Group C: NK cells from matched related donors	2	3	2	2	0	9

Population analysed: Twenty one donor participants were not included in outcome analysis.

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: Up to 42 days. All-Cause Mortality was monitored/assessed for up to 2 years).. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Phase I: NK Cell Dose Level 1_10^6	1 of 6 (16.7%)	6 of 6 (100%)	1 of 6
Phase I: NK Cell Dose Level 2_10^7	2 of 6 (33.3%)	5 of 6 (83.3%)	4 of 6
Phase I: NK Cell Dose Level 3_ 3x10^7	1 of 4 (25%)	4 of 4 (100%)	2 of 4
Phase I: NK Cell Dose Level 4_ 10^8	0 of 4 (0%)	4 of 4 (100%)	1 of 4
Phase II: NK Cell Dose Level 3_3x10^7	1 of 1 (100%)	1 of 1 (100%)	0 of 1
Phase II: NK Cell Dose Level 4_ 10^8	6 of 21 (28.6%)	17 of 21 (81%)	9 of 21
Donors	0 of 0	0 of 0	0

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 7 Nov 2023.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
University of Texas MD Anderson Cancer Center WebsiteLink 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.

Richard E Champlin principal investigator

M.D. Anderson Cancer Center

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 27 inclusion and 6 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 7 Nov 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 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 7 Nov 2023). 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?

Number of Participants Who Experienced Dose-limiting Toxicities (DLT) (time frame: Up to 42 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 NCT01823198.

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 7 Nov 2023.