Acute Biphenotypic LeukemiaPhase 2NCT01690520

Donor Umbilical Cord Blood Transplant With or Without Ex-vivo Expanded Cord Blood Progenitor Cells in Treating Patients With Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Chronic Myelogenous Leukemia, or Myelodysplastic Syndromes

A Phase 2 study testing Cyclophosphamide, Cyclosporine and Ex Vivo-Expanded Cord Blood Progenitor Cell Infusion in people with Acute Biphenotypic Leukemia and Acute Lymphoblastic Leukemia in Remission, run by Nohla Therapeutics, Inc. at 8 sites 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 6 months to 65 years
Conditions the study is for: Acute Biphenotypic Leukemia, Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission
What is being tested?: Cyclophosphamide, Cyclosporine and Ex Vivo-Expanded Cord Blood Progenitor Cell Infusion
Type of treatment: Injection or infusion, Surgery or procedure
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 163 participants
Where: 8 sitesUnited States
Study dates: 11 Dec 2012to 29 May 2020
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 6 months to 65 years
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Age criteria:
High dose TBI regimen: 6 months to =< 45 years
Middle intensity TBI regimen: 6 months to =< 65 years
Conditioning regimen selection should be based on the underlying disease, presence of minimal residual disease (MRD), age, co-morbidities, attending physician, and site preference; conditioning regimen will not require stratification of the randomization due to heterogeneity in the cohort of eligible patients.
Acute myeloid leukemia, including biphenotypic acute leukemia or mixed-lineage leukemia
All patients must have acute myeloid leukemia (AML) that is considered best treated by stem cell transplant by the referring physician and the attending transplant physician

Show all 26Show fewer

All patients must be in complete remission (CR) as defined by < 5% blasts by morphology/flow cytometry in a representative bone marrow sample with cellularity >= 15% for age
Patients in which adequate marrow/biopsy specimens cannot be obtained to determine remission status by morphologic assessment, but have fulfilled criteria of remission by flow cytometry, recovery of peripheral blood counts with no circulating blasts, and/or normal cytogenetics (if applicable) may still be eligible; reasonable attempts must be made to obtain an adequate specimen for morphologic assessment, including possible repeat procedures; these patients must be discussed with the principal investigator prior to enrollment
Acute lymphoblastic leukemia, including biphenotypic acute leukemia or mixed-lineage leukemia
High risk first complete remission (CR1) (for example, but not limited to: t(9;22), t(1;19), t(4;11) or other mixed-lineage leukemia [MLL] rearrangements, hypodiploid); or high risk (HR) as defined by referring institution treatment protocol greater than 1 cycle to obtain CR; second complete remission (CR2) or greater
All patients must be in CR as defined by < 5% blasts by morphology/flow cytometry in a representative bone marrow sample with cellularity >= 15% for age
Patients in which adequate marrow/biopsy specimens cannot be obtained to determine remission status by morphologic assessment, but have fulfilled criteria of remission by flow cytometry, recovery of peripheral blood counts with no circulating blasts, and/or normal cytogenetics (if applicable) may still be eligible; reasonable attempts must be made to obtain an adequate specimen for morphologic assessment, including possible repeat procedures; these patients must be discussed with the principal investigator prior to enrollment
Chronic myelogenous leukemia excluding refractory blast crisis; to be eligible in first chronic phase (CP1) patient must have failed or be intolerant to tyrosine kinase inhibitor therapy
Myelodysplasia (MDS) International Prognostic Scoring System (IPSS) intermediate (Int)-2 or high risk (i.e., refractory anemia with excess blasts [RAEB], refractory anemia with excess blasts in transformation [RAEBt]) or refractory anemia with severe pancytopenia or high risk cytogenetics; blasts must be < 10% by a representative bone marrow aspirate morphology
Karnofsky (>= 16 years old) >= 70 or Eastern Cooperative Oncology Group (ECOG) 0-1
Lansky (< 16 years old) >= 60
Adults: calculated creatinine clearance must be > 60 mL and serum creatinine =< 2 mg/dL
Children (< 18 years old): calculated creatinine clearance must be > 60 mL/min
Total serum bilirubin must be < 3 mg/dL unless the elevation is thought to be due to Gilbert's disease or hemolysis
Transaminases must be < 3 x the upper limit of normal per reference values of referring institution
Diffusing capacity of the lung for carbon monoxide (DLCO) corrected > 60% normal
For pediatric patients unable to perform pulmonary function tests, oxygen (O2) saturation > 92% on room air
May not be on supplemental oxygen
Left ventricular ejection fraction > 45% OR
Shortening fraction > 26%
Ability to understand and the willingness to sign a written informed consent document

Who cannot join

Uncontrolled viral or bacterial infection at the time of study enrollment
Active or recent (prior 6 month) invasive fungal infection without infectious disease (ID) consult and approval
History of human immunodeficiency virus (HIV) infection
Pregnant or breastfeeding
Prior myeloablative transplant containing full dose TBI (greater than 8 Gy)
Central nervous system (CNS) leukemic involvement not clearing with intrathecal chemotherapy and/or cranial radiation prior to initiation of conditioning; diagnostic lumbar puncture is to be performed per protocol
Patients >= 45 years: comorbidity score of 5 or higher

Trial locations and contacts

8 sites in 1 country.

City, postcode or hospital

8 sites in 1 country

United States8 sites

Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Cleveland Clinic Foundation
Cleveland, Ohio, 44195
University of Colorado
Denver, Colorado, 80217-3364
City of Hope Comprehensive Cancer Center
Duarte, California, 91010
Duke University Medical Center
Durham, North Carolina, 27710
Mount Sinai Hospital
New York, New York, 10029
Stanford Cancer Institute Palo Alto
Palo Alto, California, 94304
Fred Hutch/University of Washington Cancer Consortium
Seattle, Washington, 98109

Other trials in United States

About this study

Official title: Multi-Center, Open-Label Randomized Study of Single or Double Myeloablative Cord Blood Transplantation With or Without Infusion of Off-The-Shelf Ex Vivo Expanded Cryopreserved Cord Blood Progenitor Cells in Patients With Hematologic Malignancies

This randomized phase II trial studies how well donor umbilical cord blood transplant with or without ex-vivo expanded cord blood progenitor cells works in treating patients with acute myeloid leukemia, acute lymphoblastic leukemia, chronic myelogenous leukemia, or myelodysplastic syndromes. Giving chemotherapy and total-body irradiation before a donor umbilical cord blood transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's cells. When the healthy stem cells and ex-vivo expanded cord blood progenitor cells are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. It is not yet known whether giving donor umbilical cord blood transplant plus ex-vivo expanded cord blood progenitor cells is more effective than giving a donor umbilical cord blood transplant alone.

Read the detailed description

PRIMARY OBJECTIVES:

Compare the time to neutrophil engraftment (absolute neutrophil count [ANC] >= 500) in patients receiving a standard of care myeloablative cord blood transplant (CBT) augmented with an off-the-shelf pre-expanded and cryopreserved cord blood product to those who do not receive the product.

SECONDARY OBJECTIVES:

Provide initial data on clinical and economic benefit, such as time to platelet engraftment, duration of initial hospitalization, transplant-related mortality (TRM), death without engraftment, and incidence of severe infections in the first 100 days post-transplant.

II. The kinetics of immune system recovery will also be evaluated in both arms.

OUTLINE: Patients are randomized to 1 of 2 treatment arms.

Standard of Care Arm:

CONDITIONING REGIMEN: One of two possible conditioning regimens is chosen by the attending physician: Patients receive fludarabine phosphate intravenously (IV) over 30 minutes on days -8 to -6, cyclophosphamide IV on days -7 to -6., and undergo high dose total-body irradiation (TBI) twice daily (BID) on days -4 to -1 OR, patients receive fludarabine phosphate IV over 30-60 minutes on days -6 to -2, cyclophosphamide IV on day -6, thiotepa IV over 4 hours on days -5 to -4, and undergo middle intensity TBI once daily (QD) on days -2 to -1.

TRANSPLANT: Patients undergo single-unit or double-unit unmanipulated umbilical cord blood (UCB) transplant on day 0.

GRAFT-VERSUS-HOST DISEASE (GVHD) PROPHYLAXIS: Patients receive cyclosporine IV over 1 hour BID (adults) or thrice daily (TID) (children) or orally (PO) on days -3 to 100 with taper beginning on day 101. Patients also receive mycophenolate mofetil (MMF) IV TID on days 0-7 then may receive MMF PO TID. Patients remain on MMF TID for a minimum of 30 days, and then may begin taper if there is no evidence of GVHD and are well-engrafted from one donor unit.

Experimental Arm:

CONDITIONING REGIMEN: Patients receive the conditioning regimen chosen by the attending physician as in Standard of Care Arm.

TRANSPLANT: Patients undergo single-unit or double-unit unmanipulated UCB transplant on day 0. Patients also receive an infusion of ex vivo-expanded cord blood progenitors at least 4 hours after completion of UCB transplant.

GVHD PROPHYLAXIS: Patients receive cyclosporine IV or PO and mycophenolate mofetil IV or PO as in Standard of Care Arm.

After completion of study treatment, patients are followed up periodically for 2 years.

Conditions: Acute Biphenotypic Leukemia, Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission, Chronic Myelogenous Leukemia, Myelodysplastic Syndrome
Being tested: Cyclophosphamide, Cyclosporine, Ex Vivo-Expanded Cord Blood Progenitor Cell Infusion, Fludarabine Phosphate, Mycophenolate Mofetil
Medical subject headings: Leukemia, Biphenotypic, Acute, 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.

CyclophosphamideDrug
Given IV
CyclosporineDrug
Given IV or PO
Ex Vivo-Expanded Cord Blood Progenitor Cell InfusionBiological
Also called NLA101, Dilanubicel
Given IV
Fludarabine PhosphateDrug
Given IV
Mycophenolate MofetilDrug
Given IV or PO
ThiotepaDrug
Given IV
Total-Body IrradiationRadiation
Undergo high dose or middle intensity TBI
Umbilical Cord Blood TransplantationProcedure or surgery
Also called Cord Blood Transplantation, UCB transplantation
Undergo single-unit or double-unit unmanipulated umbilical cord blood transplant

Study groups in NCT01690520
Study group	Role	What they receive
Arm I (standard of care)	Active comparator	CONDITIONING REGIMEN: One of two possible conditioning regimens is chosen by the attending physician: Patients receive fludarabine phosphate IV over 30 minutes on days -8 to -6, cyclophosphamide IV on days -7 to -6., and undergo high dose TBI BID on days -4 to -1 OR Patients receive fludarabine phosphate IV over 30-60 minutes on days -6 to -2, cyclophosphamide IV on day -6, thiotepa IV over 4 hours on days -5 to -4, and undergo middle intensity TBI QD on days -2 to -1. TRANSPLANT: Patients undergo single-unit or double-unit unmanipulated UCB transplant on day 0. GVHD PROPHYLAXIS: Patients receive cyclosporine IV over 1 hour BID (adults) or TID (children) or PO on days -3 to 100 with taper beginning on day 101. Patients also receive MMF IV TID a day on days 0-7 then may receive MMF PO TID. Patients remain on MMF TID for a minimum of 30 days, and then may begin a taper if there is no evidence of GVHD and are well-engrafted from one donor unit. Receives: Drug: Cyclophosphamide; Drug: Cyclosporine; Drug: Fludarabine Phosphate; Drug: Mycophenolate Mofetil; Drug: Thiotepa; Radiation: Total-Body Irradiation; Procedure: Umbilical Cord Blood Transplantation
Arm II (experimental)	Experimental	CONDITIONING REGIMEN: Patients receive the conditioning regimen chosen by the attending physician as in Standard of Care Arm. TRANSPLANT: Patients undergo single-unit or double-unit unmanipulated UCB transplant on day 0. Patients also undergo infusion of ex vivo-expanded cord blood progenitor cell infusion at least 4 hours after completion of UCB transplant. GVHD PROPHYLAXIS: Patients receive cyclosporine IV or PO and mycophenolate mofetil IV or PO as in Standard of Care Arm. Receives: Drug: Cyclophosphamide; Drug: Cyclosporine; Biological: Ex Vivo-Expanded Cord Blood Progenitor Cell Infusion; Drug: Fludarabine Phosphate; Drug: Mycophenolate Mofetil; Drug: Thiotepa; Radiation: Total-Body Irradiation; Procedure: Umbilical Cord Blood Transplantation

What is being measured

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

Time to Neutrophil Engraftment
First of two consecutive days with absolute neutrophil count (ANC) equal to or greater than 500 cell/microliter measured from day of transplant.
Measured: Up to 55 days post-transplant

Secondary outcomes (5)

Time to Platelet Engraftment (20k)
First day of seven consecutive days with platelet count equal to or greater than 20,000 cells/microliter without platelet transfusions measured from day of transplant.
Measured: Up to 100 days post-transplant
Overall Survival
Measured: Up to 2 years
Non-relapse Mortality
Measured: Up to 2 years
Proportion of Patients With Severe Acute Graft Versus Host Disease
Measured: Up to 100 days post-transplant
Proportion of Participants With Chronic Graft Versus Host Disease
Measured: Up to 2 years

Timeline

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

11 Dec 201229 May 2020

First posted21 Sep 2012 (estimated)
Study start11 Dec 2012
Primary completion18 Sep 2018
Study completion29 May 2020

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 Acute Biphenotypic 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: 163 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01690520
Sponsor's protocol ID: 2603.00
Other IDs: NCI-2012-01572, 2603, 2603.00, P30CA015704, P50HL110787
Status last verified: Jun 2021
First submitted: 19 Sep 2012
Last updated: 6 Jul 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
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

RecruitingPhase 1/2
Venetoclax and CLAG-M for the Treatment of Acute Myeloid Leukemia and High-Grade Myeloid Neoplasms
Acute Biphenotypic Leukemia, Acute Myeloid Leukemia
Not yet recruitingPhase 1
A Study to Assess the Pharmacokinetics and Safety of Revumenib in Participants With Advanced Cancer and Hepatic or Renal Impairment
Relapsed/Refractory (R/R) Hematologic Malignancies, Acute Leukemia
RecruitingPhase 2
Personalized NK Cell Therapy in CBT
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Biphenotypic Leukemia
RecruitingPhase 1/2
Phase I/II Trial: Engineered Donor Graft (Orca Q) for Pediatric Hematopoietic Cell Transplant (HCT)
Acute Myeloid Leukemia, Acute Lymphoid Leukemia
RecruitingPhase 1
Prophylactic DLI-X for Leukemia Relapse After HCT
Acute Lymphoid Leukemia, Acute Myeloid Leukemia

All Acute Biphenotypic Leukemia trials

Study at a glance

From the registry record, last updated by the sponsor on 6 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Multi-Center, Open-Label Randomized Study of Single or Double Myeloablative Cord Blood Transplantation With or Without Infusion of Off-The-Shelf Ex Vivo Expanded Cryopreserved Cord Blood Progenitor Cells in Patients With Hematologic Malignancies
ClinicalTrials.gov ID: NCT01690520
Acronym / sponsor ID: 2603.00
Conditions: Acute Biphenotypic Leukemia, Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission, Chronic Myelogenous Leukemia, Myelodysplastic Syndrome
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 163 participants (actual)
Lead sponsor: Nohla Therapeutics, Inc.Industry (pharma, biotech, devices)
Collaborators: National Cancer Institute (NCI), National Heart, Lung, and Blood Institute (NHLBI)
Study start: 11 Dec 2012 (actual)
Primary completion: 18 Sep 2018 (actual)
Study completion: 29 May 2020 (actual)
First posted: 21 Sep 2012 (estimated)
Results: Posted 6 Jul 2021

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:

Compare the time to neutrophil engraftment (absolute neutrophil count [ANC] >= 500) in patients receiving a standard of care myeloablative cord blood transplant (CBT) augmented with an off-the-shelf pre-expanded and cryopreserved cord blood product to those who do not receive the product.

SECONDARY OBJECTIVES:

Provide initial data on clinical and economic benefit, such as time to platelet engraftment, duration of initial hospitalization, transplant-related mortality (TRM), death without engraftment, and incidence of severe infections in the first 100 days post-transplant.

II. The kinetics of immune system recovery will also be evaluated in both arms.

OUTLINE: Patients are randomized to 1 of 2 treatment arms.

Standard of Care Arm:

TRANSPLANT: Patients undergo single-unit or double-unit unmanipulated umbilical cord blood (UCB) transplant on day 0.

Experimental Arm:

CONDITIONING REGIMEN: Patients receive the conditioning regimen chosen by the attending physician as in Standard of Care Arm.

GVHD PROPHYLAXIS: Patients receive cyclosporine IV or PO and mycophenolate mofetil IV or PO as in Standard of Care Arm.

After completion of study treatment, patients are followed up periodically for 2 years.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Arm I (standard of care)	Active comparator	Drug: Cyclophosphamide, Drug: Cyclosporine, Drug: Fludarabine Phosphate, Drug: Mycophenolate Mofetil, Drug: Thiotepa, Radiation: Total-Body Irradiation, Procedure: Umbilical Cord Blood TransplantationCONDITIONING REGIMEN: One of two possible conditioning regimens is chosen by the attending physician: Patients receive fludarabine phosphate IV over 30 minutes on days -8 to -6, cyclophosphamide IV on days -7 to -6., and undergo high dose TBI BID on days -4 to -1 OR Patients receive fludarabine phosphate IV over 30-60 minutes on days -6 to -2, cyclophosphamide IV on day -6, thiotepa IV over 4 hours on days -5 to -4, and undergo middle intensity TBI QD on days -2 to -1. TRANSPLANT: Patients undergo single-unit or double-unit unmanipulated UCB transplant on day 0. GVHD PROPHYLAXIS: Patients receive cyclosporine IV over 1 hour BID (adults) or TID (children) or PO on days -3 to 100 with taper beginning on day 101. Patients also receive MMF IV TID a day on days 0-7 then may receive MMF PO TID. Patients remain on MMF TID for a minimum of 30 days, and then may begin a taper if there is no evidence of GVHD and are well-engrafted from one donor unit.
Arm II (experimental)	Experimental	Drug: Cyclophosphamide, Drug: Cyclosporine, Biological: Ex Vivo-Expanded Cord Blood Progenitor Cell Infusion, Drug: Fludarabine Phosphate, Drug: Mycophenolate Mofetil, Drug: Thiotepa, Radiation: Total-Body Irradiation, Procedure: Umbilical Cord Blood TransplantationCONDITIONING REGIMEN: Patients receive the conditioning regimen chosen by the attending physician as in Standard of Care Arm. TRANSPLANT: Patients undergo single-unit or double-unit unmanipulated UCB transplant on day 0. Patients also undergo infusion of ex vivo-expanded cord blood progenitor cell infusion at least 4 hours after completion of UCB transplant. GVHD PROPHYLAXIS: Patients receive cyclosporine IV or PO and mycophenolate mofetil IV or PO as in Standard of Care Arm.

Who may qualify

Ages: 6 months to 65 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (26 listed)

Age criteria:
Acute myeloid leukemia, including biphenotypic acute leukemia or mixed-lineage leukemia
Acute lymphoblastic leukemia, including biphenotypic acute leukemia or mixed-lineage leukemia
Chronic myelogenous leukemia excluding refractory blast crisis; to be eligible in first chronic phase (CP1) patient must have failed or be intolerant to tyrosine kinase inhibitor therapy
Myelodysplasia (MDS) International Prognostic Scoring System (IPSS) intermediate (Int)-2 or high risk (i.e., refractory anemia with excess blasts [RAEB], refractory anemia with excess blasts in transformation [RAEBt]) or refractory anemia with severe pancytopenia or high risk cytogenetics; blasts must be < 10% by a representative bone marrow aspirate morphology

Key exclusion criteria (7 listed)

Uncontrolled viral or bacterial infection at the time of study enrollment
Active or recent (prior 6 month) invasive fungal infection without infectious disease (ID) consult and approval
History of human immunodeficiency virus (HIV) infection
Pregnant or breastfeeding
Prior myeloablative transplant containing full dose TBI (greater than 8 Gy)

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
CyclophosphamideGiven IV	Drug	Arm I (standard of care), Arm II (experimental)
CyclosporineGiven IV or PO	Drug	Arm I (standard of care), Arm II (experimental)
Ex Vivo-Expanded Cord Blood Progenitor Cell InfusionAlso: NLA101, DilanubicelGiven IV	Biological	Arm II (experimental)
Fludarabine PhosphateGiven IV	Drug	Arm I (standard of care), Arm II (experimental)
Mycophenolate MofetilGiven IV or PO	Drug	Arm I (standard of care), Arm II (experimental)
ThiotepaGiven IV	Drug	Arm I (standard of care), Arm II (experimental)
Total-Body IrradiationUndergo high dose or middle intensity TBI	Radiation	Arm I (standard of care), Arm II (experimental)
Umbilical Cord Blood TransplantationAlso: Cord Blood Transplantation, UCB transplantationUndergo single-unit or double-unit unmanipulated umbilical cord blood transplant	Procedure or surgery	Arm I (standard of care), Arm II (experimental)

Primary outcome

Time to Neutrophil Engraftment
Time frame: Up to 55 days post-transplant
First of two consecutive days with absolute neutrophil count (ANC) equal to or greater than 500 cell/microliter measured from day of transplant.

5 secondary outcomes

Time to Platelet Engraftment (20k)
Time frame: Up to 100 days post-transplant
Overall Survival
Time frame: Up to 2 years
Non-relapse Mortality
Time frame: Up to 2 years
Proportion of Patients With Severe Acute Graft Versus Host Disease
Time frame: Up to 100 days post-transplant
Proportion of Participants With Chronic Graft Versus Host Disease
Time frame: Up to 2 years

Results and safety

Results posted to the registry on 6 Jul 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Arm I (Standard of Care)	78	27
Arm II (Experimental)	82	35

Primary outcome: Time to Neutrophil Engraftment

Time frame: Up to 55 days post-transplant. Median (95% Confidence Interval). Unit: days

Measure	Arm I (Standard of Care) n = 72	Arm II (Experimental) n = 75
Value	20.0 (19.0 to 22.0)	22.0 (20.0 to 24.0)

Population analysed: The primary analysis set for the analysis of Neutrophil Enrgraftment and Platelet Engraftment was the modified intent to treat (mITT) analysis set. The difference between the full analysis set (FAS) and the mITT is the requirement to undergo hematopoietic cell transplant (HCT) and receive study drug. Two subjects in Arm II withdrew prior to receiving study drug, and are therefore not included in the mITT analysis set, nor the primary efficacy analyses.

5 secondary outcomes are reported in the full results.

Adverse events

Time frame: 84 days. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Arm I (Standard of Care)	48 of 78 (61.5%)	71 of 78 (91%)	25 of 78
Arm II (Experimental)	55 of 82 (67.1%)	73 of 82 (89%)	21 of 82

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 6 Jul 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Acute Biphenotypic Leukemia

RecruitingPhase 1/2
Venetoclax and CLAG-M for the Treatment of Acute Myeloid Leukemia and High-Grade Myeloid Neoplasms
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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.

Filippo Milano principal investigator

Fred Hutch/University of Washington Cancer Consortium

8 sites 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 26 inclusion and 7 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 6 Jul 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 8 sites 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 Jul 2021). 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?

Time to Neutrophil Engraftment (time frame: Up to 55 days post-transplant). 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 NCT01690520.

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 6 Jul 2021.