Acute Lymphoblastic LeukemiaPhase 2NCT01621477
T-Cell Replete Haploidentical Donor Hematopoietic Stem Cell Plus Natural Killer (NK) Cell Transplantation in Patients With Hematologic Malignancies Relapsed or Refractory Despite Previous Allogeneic Transplant
A Phase 2 study testing clofarabine, cytarabine and busulfan in people with Acute Lymphoblastic Leukemia and Acute Myelocytic Leukemia, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial summary
The primary aim of this protocol is to evaluate if the one-year survival is significantly improved in the group of patients who receive a T-cell replete haploidentical donor hematopoietic cell transplant (HCT) with a novel reduced intensity conditioning regimen. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Acute Lymphoblastic Leukemia, Acute Myelocytic Leukemia, Chronic Myelocytic Leukemia
- What is being tested?
- clofarabine, cytarabine and busulfan
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 34 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2012to Dec 2015
- 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 Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age less than 21 years.
- One of the following hematologic malignancies that has relapsed or remains refractory after prior allogeneic HCT:
- Acute lymphoblastic leukemia (ALL)
- Acute myeloid leukemia (AML) (including myeloid sarcoma)
- Chronic myelogenous leukemia (CML), juvenile myelomonocytic leukemia (JMML), myelodysplastic syndrome (MDS), Hodgkin or non-Hodgkin lymphoma (NHL)
- Has a suitable single haplotype matched (≥ 3 of 6) family member donor.
Show all 27Show fewer
- Does not have any other active malignancy other than the one for which this transplant is indicated.
- If prior central nervous system (CNS) leukemia, it must be treated and have no evidence of CNS disease
- Does not have current uncontrolled bacterial, fungal, or viral infection per the judgment of the principal investigator.
- Patient must fulfill pre-transplant evaluation:
- Left ventricular ejection fraction greater than 40%, or shortening fraction greater than or equal to 25%.
- Creatinine clearance or Glomerular Filtration Rate of ≥70 ml/min/1.73m^2.
- Forced vital capacity (FVC) ≥ 40% of predicted value; or pulse oximetry ≥ 92% on room air if patient is unable to perform pulmonary function testing.
- Karnofsky or Lansky (age-dependent) performance score ≥ 50.
- Total bilirubin ≤ 1.5 times the upper limit of normal for age.
- Alanine aminotransferase (ALT) ≤ 3 times the upper limit of normal for age.
- Not pregnant. If female with child bearing potential, must be confirmed by negative serum or urine pregnancy test within 14 days prior to enrollment.
- Not breast feeding.
- Does not have active acute bronchiolitis obliterans or bronchiolitis obliterans organizing pneumonia.
- At least single haplotype matched (≥ 3 of 6) family member,
- At least 18 years of age.
- Human immunodeficiency virus (HIV) negative.
- Not pregnant as confirmed by negative serum or urine pregnancy test within 14 days prior to enrollment (if female).
- Not breast feeding.
- A suitable donor is identified as either:
- Has completed the process of donor eligibility determination as outlined in 21 CFR 1271 and agency guidance; OR
- Does not meet 21 CFR 1271 eligibility requirements, but has a declaration of urgent medical need completed by the principal investigator or physician sub-investigator per 21 CFR 1271.
Who cannot join
- Does not meet above inclusion criteria.
Trial locations and contacts
1 site in 1 country.
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
The primary aim of this protocol is to evaluate if the one-year survival is significantly improved in the group of patients who receive a T-cell replete haploidentical donor hematopoietic cell transplant (HCT) with a novel reduced intensity conditioning regimen. Study population will consist of patients (21 years or under) with hematologic malignancies that have relapsed or are refractory after prior allogeneic transplant. Toxicity will be evaluated by the rate of transplant related mortality and the rates of moderate and severe graft-versus-host disease (GvHD) at day 100. The investigators will describe event-free, and disease-free survival at one year, as well as the rates of hematopoietic recovery and donor engraftment and study comprehensively immune reconstitution following T-cell replete haploidentical transplantation.
Read the detailed description
Patients with refractory hematologic malignancies, including those who develop recurrent disease after allogeneic hematopoietic cell transplantation, have a dismal prognosis. Historically, both regimen-related mortality and disease recurrence have been significant causes of treatment failure in this heavily pre-treated patient population. Our institution has utilized mismatched family member (haploidentical) donors for these patients for a number of years for the following reasons: (1) Only 30% of patients have matched related donors available; (2) transplantation can be performed more rapidly since the time to unrelated donor transplantation averages 3 to 4 months; (3) no other curative treatment options are available. These therapeutic interventions have been largely successful given the dismal prognosis in this patient group; however disease recurrence remains the most significant cause of treatment failure. To provide maximum benefit for this challenging population, the goals of a therapeutic transplant protocol should include: (1) a conditioning regimen that is well tolerated, even in a heavily pre-treated population; but it should also provide substantial antileukemia effects, and (2) should establish rapid immune recovery such that the patient may benefit from graft versus leukemia effect and early protection from life threatening infections while also limiting dangerous and counter-productive graft versus host disease.
The primary aim of this protocol will be to evaluate if the one-year survival is significantly improved in the group of patients receiving T-cell replete haploidentical donor HCT with a novel clofarabine, cytarabine, busulfan, plerixafor, cyclophosphamide, and ATG based reduced intensity conditioning regimen whose hematologic malignancy has relapsed or is refractory after prior allogeneic transplant. Toxicity will be evaluated by the rate of transplant related mortality and the rates of moderate and severe graft versus host disease at day 100. The investigators will also describe event-free, and disease-free survival at one year, as well as the rates of hematopoietic recovery and donor engraftment. Additionally, the investigators will study comprehensively immune reconstitution following T-cell replete haploidentical transplantation.
PRIMARY OBJECTIVE:
- Evaluate if the one-year survival is significantly improved in a group of children receiving a therapeutic regimen for high-risk hematologic malignancy that is relapsed or refractory despite previous allogeneic hematopoietic cell transplantation (HCT) using a novel reduced intensity conditioning and T-cell replete haploidentical donor hematopoietic stem cell plus NK cell transplantation.
SECONDARY OBJECTIVES:
- Estimate the incidence of malignant relapse, event-free survival, and disease free survival (DFS) at one-year post-transplantation.
- Estimate incidence and severity of acute and chronic (GVHD).
- Estimate the rate of transplant related mortality (TRM) in the first 100 days after transplantation.
- Conditions
- Acute Lymphoblastic Leukemia, Acute Myelocytic Leukemia, Chronic Myelocytic Leukemia, Juvenile Myelomonocytic Leukemia, Myelodysplastic Syndrome, Hodgkin or Non-Hodgkin Lymphoma
- Being tested
- clofarabine, cytarabine, busulfan, Plerixafor, cyclophosphamide
- Keywords
- Hematologic malignancy, Hematopoietic cell transplant
- Medical subject headings
- Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myeloid, Acute, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Leukemia, Myelomonocytic, Juvenile, Myelodysplastic Syndromes, Lymphoma, Non-Hodgkin, Sarcoma, Myeloid, Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
clofarabineDrug
Also called Cl-F-Ara-A, CAFdA, Clofarex, Clolar(TM)
Given on Day -9 and Day -8 (Day 0 is first stem cell infusion). Drug class: antineoplastic agent
cytarabineDrug
Also called Ara-C
Given on Day -9 and Day -8 (Day 0 is first stem cell infusion). Drug class: antineoplastic agent
busulfanDrug
Also called Myleran(R), Busulfex
Given on Day -7 and Day -6 (Day 0 is first stem cell infusion). Drug class: antineoplastic agent
PlerixaforDrug
Also called AMD3100, Mozobil(R)
Given on Day -7 and Day -6 (Day 0 is first stem cell infusion). Drug class: Hematopoietic Stem Cell Mobilizer
cyclophosphamideDrug
Also called Cytoxan
Given on Day -5 and Day +4 (Day 0 is first stem cell infusion). Drug class: antineoplastic agent; immunosuppressive agent.
antithymocyte globulin (rabbit)Drug
Also called Rabbit ATG, Rabbit Thymoglobulin(R)
Given on Day -4, Day -3, Day -2, and Day -1 (Day 0 is first stem cell infusion). Drug class: immunosuppressive agent.
stem cellsBiological
Also called HSC infusion
Patients undergo T cell replete Hematopoietic stem cell infusion on Day 0 and Day +1. Patients undergo natural killer (NK) cell transplantation on day +6 (Day 0 is first stem cell infusion).
TacrolimusDrug
Also called FK506, Prograf(R), Protonic(R)
Given on Day +11 (Day 0 is first stem cell infusion). Drug class: immunosuppressive agent.
Also called MMF, CellCept(R)
Given on Day +11 (Day 0 is first stem cell infusion). Drug class: immunosuppressive agent.
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | All study participants.
Interventions: clofarabine, cytarabine, busulfan, plerixafor, cyclophosphamide, antithymocyte globulin (rabbit), stem cells, tacrolimus, mycophenolate mofetil Receives: Drug: clofarabine; Drug: cytarabine; Drug: busulfan; Drug: Plerixafor; Drug: cyclophosphamide; Drug: antithymocyte globulin (rabbit); Biological: stem cells; Drug: Tacrolimus; Drug: mycophenolate mofetil |
What is being measured
The primary outcome is the main question the study is designed to answer.
One-year Survival (OS)
Evaluate the number of participants alive at 1 year. The number of participants surviving to one-year post-transplantation is given.
Measured: One year post transplant
Secondary outcomes (6)
Incidence of Malignant Relapse
Estimate the incidence of malignant relapse at one year post-transplant. The number of participants with malignant relapse or progressive disease is given. Relapse was evaluated using standard WHO criteria for each disease.
Measured: One year post transplantation.
Event-Free Survival (EFS)
Estimate the EFS at one-year post-transplantation. The event is defined as relapse or death due to any cause. The number of participants who were alive without relapse at one year post-transplant is reported.
Measured: one year post transplant
Disease-Free Survival (DFS)
Estimate the DFS at one-year post-transplantation. The event is defined as relapse or death due to relapse. The number of participants who did not relapse up to one year post transplant is reported.
Measured: one year post transplant
Incidence and Severity of Acute Graft Versus Host Disease (GVHD)
The number of participants with acute GVHD is given, organized by grade. Participants are graded on a scale from 1 to 4, with 1 being mild and 4 being severe.
Measured: 100 days post transplant
Incidence and Severity of Chronic Graft Versus Host Disease (GVHD)
The severity of chronic GVHD will be described. Chronic GVHD was evaluated using NIH Consensus Global Severity Scoring. The number of participants with chronic GVHD is given, organized by severity.
Measured: 100 days post transplant
Number of Participants With Transplant Related Mortality (TRM)
The number of participants who died due to TRM in the first 100 days post-transplant is given.
Measured: 100 days post transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jun 2012 (estimated)
- Study startAug 2012
- Primary completionDec 2015
- Study completionDec 2015
Why it stopped: Study was terminated in August 2016 due to replacement by a new study.
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 Lymphoblastic LeukemiaStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research HospitalThe 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 34 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Assisi Foundation
- Responsible party
- Sponsor
- Investigators
- Brandon M. Triplett, MD, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01621477
- Sponsor's protocol ID
- HAP3R
- Other IDs
- NCI-2012-00554
- Status last verified
- Dec 2016
- First submitted
- 8 Jun 2012
- Last updated
- 20 Feb 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Feb 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- T-Cell Replete Haploidentical Donor Hematopoietic Stem Cell Plus Natural Killer (NK) Cell Transplantation in Patients With Hematologic Malignancies Relapsed or Refractory Despite Previous Allogeneic Transplant
- ClinicalTrials.gov ID
- NCT01621477
- Acronym / sponsor ID
- HAP3R
- Conditions
- Acute Lymphoblastic Leukemia, Acute Myelocytic Leukemia, Chronic Myelocytic Leukemia, Juvenile Myelomonocytic Leukemia, Myelodysplastic Syndrome, Hodgkin or Non-Hodgkin Lymphoma
- Keywords
- Hematologic malignancy, Hematopoietic cell transplant
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study was terminated in August 2016 due to replacement by a new study.
- Enrolment
- 34 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Collaborators
- Assisi Foundation
- Study start
- Aug 2012
- Primary completion
- Dec 2015 (actual)
- Study completion
- Dec 2015 (actual)
- First posted
- 18 Jun 2012 (estimated)
- Results
- Posted 20 Feb 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary aim of this protocol is to evaluate if the one-year survival is significantly improved in the group of patients who receive a T-cell replete haploidentical donor hematopoietic cell transplant (HCT) with a novel reduced intensity conditioning regimen. Study population will consist of patients (21 years or under) with hematologic malignancies that have relapsed or are refractory after prior allogeneic transplant. Toxicity will be evaluated by the rate of transplant related mortality and the rates of moderate and severe graft-versus-host disease (GvHD) at day 100. The investigators will describe event-free, and disease-free survival at one year, as well as the rates of hematopoietic recovery and donor engraftment and study comprehensively immune reconstitution following T-cell replete haploidentical transplantation.
Detailed description
Patients with refractory hematologic malignancies, including those who develop recurrent disease after allogeneic hematopoietic cell transplantation, have a dismal prognosis. Historically, both regimen-related mortality and disease recurrence have been significant causes of treatment failure in this heavily pre-treated patient population. Our institution has utilized mismatched family member (haploidentical) donors for these patients for a number of years for the following reasons: (1) Only 30% of patients have matched related donors available; (2) transplantation can be performed more rapidly since the time to unrelated donor transplantation averages 3 to 4 months; (3) no other curative treatment options are available. These therapeutic interventions have been largely successful given the dismal prognosis in this patient group; however disease recurrence remains the most significant cause of treatment failure. To provide maximum benefit for this challenging population, the goals of a therapeutic transplant protocol should include: (1) a conditioning regimen that is well tolerated, even in a heavily pre-treated population; but it should also provide substantial antileukemia effects, and (2) should establish rapid immune recovery such that the patient may benefit from graft versus leukemia effect and early protection from life threatening infections while also limiting dangerous and counter-productive graft versus host disease.
The primary aim of this protocol will be to evaluate if the one-year survival is significantly improved in the group of patients receiving T-cell replete haploidentical donor HCT with a novel clofarabine, cytarabine, busulfan, plerixafor, cyclophosphamide, and ATG based reduced intensity conditioning regimen whose hematologic malignancy has relapsed or is refractory after prior allogeneic transplant. Toxicity will be evaluated by the rate of transplant related mortality and the rates of moderate and severe graft versus host disease at day 100. The investigators will also describe event-free, and disease-free survival at one year, as well as the rates of hematopoietic recovery and donor engraftment. Additionally, the investigators will study comprehensively immune reconstitution following T-cell replete haploidentical transplantation.
PRIMARY OBJECTIVE:
- Evaluate if the one-year survival is significantly improved in a group of children receiving a therapeutic regimen for high-risk hematologic malignancy that is relapsed or refractory despite previous allogeneic hematopoietic cell transplantation (HCT) using a novel reduced intensity conditioning and T-cell replete haploidentical donor hematopoietic stem cell plus NK cell transplantation.
SECONDARY OBJECTIVES:
- Estimate the incidence of malignant relapse, event-free survival, and disease free survival (DFS) at one-year post-transplantation.
- Estimate incidence and severity of acute and chronic (GVHD).
- Estimate the rate of transplant related mortality (TRM) in the first 100 days after transplantation.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Drug: clofarabine, Drug: cytarabine, Drug: busulfan, Drug: Plerixafor, Drug: cyclophosphamide, Drug: antithymocyte globulin (rabbit), Biological: stem cells, Drug: Tacrolimus, Drug: mycophenolate mofetilAll study participants. Interventions: clofarabine, cytarabine, busulfan, plerixafor, cyclophosphamide, antithymocyte globulin (rabbit), stem cells, tacrolimus, mycophenolate mofetil |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (27 listed)
- Age less than 21 years.
- One of the following hematologic malignancies that has relapsed or remains refractory after prior allogeneic HCT:
- Has a suitable single haplotype matched (≥ 3 of 6) family member donor.
- Does not have any other active malignancy other than the one for which this transplant is indicated.
- If prior central nervous system (CNS) leukemia, it must be treated and have no evidence of CNS disease
Key exclusion criteria (1 listed)
- Does not meet above inclusion criteria.
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 |
|---|---|---|
| clofarabineAlso: Cl-F-Ara-A, CAFdA, Clofarex, Clolar(TM)Given on Day -9 and Day -8 (Day 0 is first stem cell infusion). Drug class: antineoplastic agent | Drug | Treatment |
| cytarabineAlso: Ara-CGiven on Day -9 and Day -8 (Day 0 is first stem cell infusion). Drug class: antineoplastic agent | Drug | Treatment |
| busulfanAlso: Myleran(R), BusulfexGiven on Day -7 and Day -6 (Day 0 is first stem cell infusion). Drug class: antineoplastic agent | Drug | Treatment |
| PlerixaforAlso: AMD3100, Mozobil(R)Given on Day -7 and Day -6 (Day 0 is first stem cell infusion). Drug class: Hematopoietic Stem Cell Mobilizer | Drug | Treatment |
| cyclophosphamideAlso: CytoxanGiven on Day -5 and Day +4 (Day 0 is first stem cell infusion). Drug class: antineoplastic agent; immunosuppressive agent. | Drug | Treatment |
| antithymocyte globulin (rabbit)Also: Rabbit ATG, Rabbit Thymoglobulin(R)Given on Day -4, Day -3, Day -2, and Day -1 (Day 0 is first stem cell infusion). Drug class: immunosuppressive agent. | Drug | Treatment |
| stem cellsAlso: HSC infusionPatients undergo T cell replete Hematopoietic stem cell infusion on Day 0 and Day +1. Patients undergo natural killer (NK) cell transplantation on day +6 (Day 0 is first stem cell infusion). | Biological | Treatment |
| TacrolimusAlso: FK506, Prograf(R), Protonic(R)Given on Day +11 (Day 0 is first stem cell infusion). Drug class: immunosuppressive agent. | Drug | Treatment |
| mycophenolate mofetilAlso: MMF, CellCept(R)Given on Day +11 (Day 0 is first stem cell infusion). Drug class: immunosuppressive agent. | Drug | Treatment |
Primary outcome
One-year Survival (OS)
Time frame: One year post transplant
Evaluate the number of participants alive at 1 year. The number of participants surviving to one-year post-transplantation is given.
6 secondary outcomes
Incidence of Malignant Relapse
Time frame: One year post transplantation.
Event-Free Survival (EFS)
Time frame: one year post transplant
Disease-Free Survival (DFS)
Time frame: one year post transplant
Incidence and Severity of Acute Graft Versus Host Disease (GVHD)
Time frame: 100 days post transplant
Incidence and Severity of Chronic Graft Versus Host Disease (GVHD)
Time frame: 100 days post transplant
Number of Participants With Transplant Related Mortality (TRM)
Time frame: 100 days post transplant
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Feb 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- St. Jude Children's Research HospitalLink provided by the sponsor.
- Clinical Trials Open at St. JudeLink provided by the sponsor.
Related trials for Acute Lymphoblastic Leukemia
Recruiting
A Multicenter Study to Evaluate Next-Generation Sequencing (NGS) Testing and Monitoring of B-Cell Recovery to Guide Management Following Chimeric Antigen Receptor T-cell (CART) Induced Remission in Children and Young Adults With B Lineage Acute Lymph...
B-All, Acute Lymphoblastic Leukemia
RecruitingPhase 1
Anti-CRLF2-R/TSLPR Chimeric Antigen Receptor T Cells (TSLPR-CART) in Participants With Recurrent or Refractory CRLF2-R/TSLPR-Overexpressing B-Cell Acute Lymphoblastic Leukemia (B-ALL)
B-All, Acute Lymphoblastic Leukemia
RecruitingObservational study
Collecting Blood Samples From Patients With and Without Cancer to Evaluate Tests for Early Cancer Detection
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
RecruitingPhase 1
In VIVO CAR-T Therapy for Relapsed/Refractory Hematological Malignancies
B-Acute Lymphoblastic Leukemia, B Cell Non-Hodgkin's Lymphoma
RecruitingPhase 2
Auto Stem Cell Transplant for Lymphoma Patients
Non-Hodgkin Lymphoma, Hodgkin Lymphoma
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.
Brandon M. Triplett, MD principal investigator
St. Jude Children's Research Hospital
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 clofarabine, cytarabine and busulfan in people with Acute Lymphoblastic Leukemia and Acute Myelocytic Leukemia, run by St. Jude Children's Research Hospital 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 27 inclusion and 1 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 20 Feb 2017. 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 20 Feb 2017). 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?
One-year Survival (OS) (time frame: One year post 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 20 Feb 2017.