Acute Myeloid Leukemia (AML)Phase 2NCT01772953
Treosulfan/Fludarabine/Low Dose TBI as a Preparative Regimen for Children With AML/MDS Undergoing Allo HCT
A Phase 2 study testing Treosulfan in people with Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS), run by Center for International Blood and Marrow Transplant Research at 23 sites in United States.
Trial summary
This is a prospective, open-label, nonrandomized, prospective clinical trial evaluating a fixed regimen of treosulfan, fludarabine and low-dose total body irradiation (TBI) in children with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic cell transplantation (HCT). Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS)
- What is being tested?
- Treosulfan
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 23 sitesUnited States
- Study dates
- Sep 2013to Aug 2015
- 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 Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age < 21 years.
- Disease and disease status:
- Acute myeloid leukemia (AML) in morphologic remission (defined as < 5% blasts in a bone marrow aspirate of adequate cellularity performed within 28 days from start of conditioning).
- Myelodysplastic syndrome (MDS): Any 2008 WHO classification subtype (Appendix I). RAEB-2 patients may proceed directly to transplant, but may also be considered for induction chemotherapy before transplant. Patients with ≥20% morphologic marrow blasts will require induction therapy to reduce morphologic marrow blasts below 5% before transplant.
- Karnofsky Index or Lansky Play-Performance Scale > 70 % on pre-transplant evaluation. Karnofsky scores must be used for patients > 16 years of age and Lansky scores for patients < 16 years of age.
- Able to give informed consent if > 18 years, or with a legal guardian capable of giving informed consent if < 18 years.
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- Negative pregnancy test (serum, urine β-HCG, or other test per institutional guidelines) for females of childbearing potential.
- A single previous autologous or allogeneic HCT is allowed as long as the time from first to second transplant hematopoietic cell infusion is no less than 6 months.
- With a suitable allogeneic hematopoietic cell donor including, as available:
- HLA-identical related donor matched for HLA-A, and -B at the serologic level at minimum and -DRB1 at high resolution by molecular typing. A single locus mismatched related donor (7/8 matched) is permitted only if there are no 8/8 matched unrelated donors available.
- Unrelated volunteer donor matched for HLA-A, -B, -C and -DRB1 defined by high resolution molecular typing. A single HLA antigen or allele mismatch (7/8 matched) is permitted.
- Unrelated cord blood (UCB) matched to the recipient at a minimum of 4 of 6 loci at HLA-A, and -B by intermediate resolution and -DRB1 by high resolution. Cord blood unit(s) will be selected using the following criteria:
- Unit selection is based on the cryopreserved total nucleated cell (TNC) dose and matching at HLA-A, -B intermediate resolution and -DRB1 high resolution typing. While HLA-C antigen/allele level typing is not considered in the matching criteria, if available, it may be used to optimize unit selection.
- Selection of two UCB units is required if a single UCB unit will not provide a sufficient cell dose (see Table 1 below). When two UCB units are not required per Table 1, two UCB units may be used with approval of the study chair or designee. When two units are selected, the following rules apply:
- The UCB unit with the least HLA disparity with the patient, followed by the larger cell dose for equivalently matched units, will be considered unit #1 (selection priority is 6/6 match >5/6 match>4/6 match).
- An additional UCB unit may be required to achieve the required cell dose, as outlined in the table below. The second unit will be the one that most closely HLA matches the patient and meets minimum size criteria as outlined below (i.e. a smaller and more closely matched unit will be selected over a larger less well matched unit as long as minimum cell dose criteria are met).
- Each UCB unit MUST contain at least 1.5 x 10^7 TNC per kilogram recipient weight.
- 3) Other comments about cord blood unit selection:
- Use of unlicensed cord blood units will adhere to current federal regulatory requirements for procurement.
- Units will be selected based on the TNC dose and HLA matching.
- A UCB unit that is 4/6 or 5/6 mismatched but homozygous at the locus of mismatch should be chosen over a 5/6 unit with bidirectional mismatch even if the latter unit provides a larger cell dose. This is only applicable to choosing units within a given match grade.
- Within the best HLA match grade, the unit containing the greatest number of cells will be chosen. If there are two units of equivalent cell dose within a match level, choose the unit with best match by higher resolution molecular typing, if known.
- Other factors to be considered:
- Within the same HLA match grade, matching at both DR loci is preferable. ii. UCB units sourced from cord blood banks located in the United States are preferred.
- Younger units are preferred over older units, all other factors being equal.
- Adequate organ function, defined as:
- Pulmonary: FEV1, FVC, and corrected DLCO must all be ≥ 50% of predicted by pulmonary function tests (PFTs). For children who are unable to perform for PFTs due to age, the criteria are: no evidence of dyspnea at rest and there is no need for supplemental oxygen.
- Renal: GFR estimated by the updated Schwartz formula ≥ 90/min/1.73 m2, i.e. height (cm)/serum creatinine (mg/dl) > 220. If the estimated creatinine clearance is < 90 ml/min/1.73 m2, then renal function must be measured by 24-hour creatinine clearance or nuclear GFR, and must be > 70 mL/minute/1.73 m2
- Cardiac: Shortening fraction of ≥ 27% by echocardiogram or radionuclide scan (MUGA) or ejection fraction of ≥ 50% by echocardiogram or radionuclide scan (MUGA)
- Hepatic: SGOT (AST) or SGPT (ALT) < 5 x upper limit of normal (ULN) for age; Conjugated bilirubin < 2.5 mg/dL, unless attributable to Gilbert's Syndrome.
- Co- enrollment in PBMTC ONC 1001 (CIBMTR 09-MRD) protocol and/or CIBMTR 10-CBA protocol (NMDP cord blood IND) is allowed. Co-enrollment on any other studies where experimental therapy is being administered will be handled on a case-by-case basis and must be discussed with the study chair or designee prior to enrollment.
Who cannot join
- Pregnant or lactating females are ineligible as many of the medications used in this protocol could be harmful to unborn children and infants.
- Patients with HIV or uncontrolled fungal, bacterial or viral infections are excluded. Patients with history of fungal disease during induction therapy may proceed if they have a significant response to antifungal therapy with no or minimal evidence of disease remaining by CT evaluation.
- Patients with active CNS leukemia or any other active site of extramedullary disease at the time of enrollment are not permitted. Note: Those with prior history of CNS or extramedullary disease, but with no active disease at the time of pre-transplant workup, are eligible.
- Patients undergoing a course of chemotherapy using another investigational drug.
- Patients diagnosed with Fanconi Anemia.
Trial locations and contacts
23 sites in 1 country.
23 sites in 1 country
United States23 sites
University of Michigan
Ann Arbor, Michigan, 48109
Children's Hospital of Colorado
Aurora, Colorado, 80045
Children's Hospital of Alabama/UAB
Birmingham, Alabama, 35233
Medical University of South Carolina
Charleston, South Carolina, 29425
University Hospitals Case Medical Center
Cleveland, Ohio, 44106
Cleveland Clinic
Cleveland, Ohio, 44195
Nationwide Children's Hospital
Columbus, Ohio, 43205
City of Hope
Duarte, California, 91010
Riley Hospital for Children/Indiana University
Indianapolis, Indiana, 46202
Children's Mercy Hospital
Kansas City, Missouri, 64108
Children's Hospital Los Angeles
Los Angeles, California, 99027
University of Wisconsin - Madison
Madison, Wisconsin, 53705
St. Jude's Children's Research Hospital
Memphis, Tennessee, 38105
Medical College of Wisconsin/Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226
University of Minnesota
Minneapolis, Minnesota, 55455
Phoenix Children's Hospital
Phoenix, Arizona, 85016
OHSU/Doernbecher Children's Hospital
Portland, Oregon, 97239
Primary Children's Medical Center - University of Utah
Salt Lake City, Utah, 84111
Methodist Children's Hospital of South Texas
San Antonio, Texas, 78229
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109
Washington University, St. Louis Children's
St Louis, Missouri, 63130
All Children's Hospital
St. Petersburg, Florida, 33701
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
Official title: A Phase II Study of Treosulfan/Fludarabine/Low Dose Total Body Irradiation as a Preparative Regimen for Children With AML/MDS Undergoing Allogeneic Hematopoietic Cell Transplantation
This is a prospective, open-label, nonrandomized, prospective clinical trial evaluating a fixed regimen of treosulfan, fludarabine and low-dose total body irradiation (TBI) in children with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic cell transplantation (HCT). The primary hypothesis is that HCT with a preparative regimen consisting of treosulfan, fludarabine and low-dose TBI will result in overall survival (OS) comparable to historical rates observed with conventional myeloablative regimens in the pediatric population. The preparative regimen will result in adequate incidence of neutrophil and platelet engraftment, and acceptable rates of graft-versus-host disease (GVHD), relapse and survival. The pharmacokinetic (PK) profile of treosulfan in children will be comparable to that of adults previously studied.
Read the detailed description
The proposed study will evaluate a regimen using treosulfan, fludarabine and low-dose TBI in children and adolescents with AML or MDS undergoing allogeneic HCT. We expect this regimen to yield lower toxicity and at least equivalent rates of disease control and overall survival, compared to current standard myeloablative regimens. The primary objective of this study is to determine the safety and preliminary efficacy of a transplant preparative regimen consisting of treosulfan, fludarabine and low-dose TBI for children with AML and MDS. The primary endpoint will be overall survival (OS) at one year. Secondary objectives to be studied include: pharmacokinetic (PK) profile of treosulfan in children < 40 kg, non-relapse mortality, disease-free survival, incidences of neutrophil and platelet engraftment, donor chimerism, acute and chronic graft-versus-host disease (GVHD), and relapse.
- Being tested
- Treosulfan
- Keywords
- AML, MDS, Treosulfan
- Medical subject headings
- Leukemia, Myeloid, Acute, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TreosulfanDrug
This is a phase II, open-label, nonrandomized, prospective study of a preparative regimen consisting of treosulfan, fludarabine and low-dose total body irradiation (TBI) for children with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic cell transplantation (HCT).
| Study group | Role | What they receive |
|---|---|---|
| treosulfan, fludarabine and low-dose TBI prep regimen | Experimental | Treosulfan: 10-14 g/m2/day IV over 120 minutes on days -6, -5 and -4. Treosulfan will be administered prior to fludarabine on days -6 to -4 to facilitate PK testing.
Fludarabine: 30 mg/m2 IV for patients > 10 kg (or 1 mg/kg IV for patients < 10 kg) once daily per institutional infusion standards on days -6 through -2 for a total dose of 150 mg/m2 (or 5 mg/kg).
A single fraction of 200 cGy TBI will be administered on day -1. Stem cell infusion on day 0 Receives: Drug: Treosulfan |
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary endpoint will be overall survival (OS) at one year
The primary objective of this study is to determine the safety and preliminary efficacy of a transplant preparative regimen consisting of treosulfan, fludarabine and low-dose TBI for children with AML and MDS. The primary endpoint will be overall survival (OS) at one year.
Measured: 1 year
Secondary outcomes (12)
Pharmacokinetic (PK) profile of Treosulfan in children < 40 kg
Drug plasma concentrations will be determined by: Cmax; half lives (t1/2); area under the curve (AUC); volumes of distribution (V); clearances (CL); mean residence times (MRT)
Measured: 1 year
Non-Relapse Mortality
The event is death in continuous remission treating relapse as the competing risk. Patients alive and in remission at the time of last observation will be censored.
Measured: 1 year
Disease-Free Survival
Disease-free survival is defined as the minimum time interval from transplant to relapse/recurrence of disease, to death or to last follow-up.
Measured: 1 year
Neutrophil Engraftment
Neutrophil engraftment is defined as achieving a donor derived absolute neutrophil count (ANC) ≥ 500/μL for three consecutive measurements on different days. The first of the three days will be designated as the time to neutrophil engraftment.
Measured: 1 year
Donor Chimerism
Peripheral blood chimerism (% of donor chimerism) in whole blood or fractions sorted for T-cell and myeloid subsets (CD3 and CD33) will be described on days 28, 42, 100, 180 and 365.
Measured: 1 year
Acute graft-versus-host disease (GVHD)
Incidences of grade II - IV and III - IV acute GVHD at days 42, 100, 180 and 365 will be graded according to the BMT CTN Manual of Procedures
Measured: 1 year
Relapse
Testing for recurrent malignancy in the blood, marrow or other sites will be used to assess relapse after transplantation. For the purpose of this study, relapse is defined by either morphological or cytogenetic evidence of AML or MDS consistent with pre-transplant features. The event for this endpoint is the time interval from transplant to relapse/recurrence of disease or to last follow-up. Death in remission is considered a competing risk.
Measured: 1 year
Primary graft failure
This endpoint will be evaluated separately for bone marrow/peripheral blood and cord blood.
Primary graft failure is defined as lack of donor-derived neutrophil engraftment by 56 days. This time point was chosen to adjust for potential differences in time to engraftment that may be observed in cord blood vs. marrow/PBSC recipients. This outcome will be evaluated separately for bone marrow/peripheral blood and cord blood based on neutrophil count and peripheral blood chimerism obtained on day 42 ± 14. Relapse and death prior to neutrophil engraftment are considered competing risks for the endpoint of primary graft failure.
Measured: 1 Year
Platelet engraftment of > 20,000/μL and >50,000/μL
Time to platelet engraftment is defined as the first day of a minimum of three consecutive measurements on different days such that the patient has achieved a platelet count > 20,000/μL and > 50,000/μL with no platelet transfusions in the preceding seven days. The first day of the three measurements will be designated as the day of platelet engraftment.
Measured: 1 Year
Chronic graft-versus-host disease (GVHD)
Incidence of chronic GVHD on days 100, 180 and 365 will be scored according to the BMT CTN MOP
Measured: 1 Year
Secondary graft failure
Secondary graft failure is defined as initial donor-derived neutrophil engraftment followed by subsequent decline in ANC to < 500/μL for three consecutive measurements on different days, and unresponsive to growth factor therapy, with loss of donor chimerism to < 50% donor CD3 in peripheral blood.
Measured: 1 year
Primary cause of death
Primary cause of death will be classified as: Relapse/Primary disease; GVHD; Infection; Organ Toxicity; Other
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jan 2013 (estimated)
- Study startSep 2013
- Primary completionAug 2015
- Study completionAug 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid Leukemia (AML)Studies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Center for International Blood and Marrow Transplant Research
- Sponsor type
- Research network
- Collaborators
- National Marrow Donor Program, Resource for Clinical Investigation in Blood and Marrow Transplantation (RCI BMT), Pediatric Blood and Marrow Transplant Consortium, St. Baldrick's Foundation, medac GmbH
- Responsible party
- Sponsor
- Investigators
- Eneida Nemecek, MD, Study chair, Doernbecher Children's Hospital, Oregon Health & Science UniversityColleen Delaney, MD, Study chair, Seattle Children's Hospital, Fred Hutchinson Cancer Research Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01772953
- Sponsor's protocol ID
- 11-TREO
- Status last verified
- Oct 2016
- First submitted
- 6 Dec 2012
- Last updated
- 12 Oct 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 12 Oct 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of Treosulfan/Fludarabine/Low Dose Total Body Irradiation as a Preparative Regimen for Children With AML/MDS Undergoing Allogeneic Hematopoietic Cell Transplantation
- ClinicalTrials.gov ID
- NCT01772953
- Acronym / sponsor ID
- 11-TREO
- Keywords
- AML, MDS, Treosulfan
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Center for International Blood and Marrow Transplant ResearchResearch network
- Collaborators
- National Marrow Donor Program, Resource for Clinical Investigation in Blood and Marrow Transplantation (RCI BMT), Pediatric Blood and Marrow Transplant Consortium, St. Baldrick's Foundation, medac GmbH
- Study start
- Sep 2013
- Primary completion
- Aug 2015 (actual)
- Study completion
- Aug 2015 (actual)
- First posted
- 21 Jan 2013 (estimated)
- 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.
This is a prospective, open-label, nonrandomized, prospective clinical trial evaluating a fixed regimen of treosulfan, fludarabine and low-dose total body irradiation (TBI) in children with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic cell transplantation (HCT). The primary hypothesis is that HCT with a preparative regimen consisting of treosulfan, fludarabine and low-dose TBI will result in overall survival (OS) comparable to historical rates observed with conventional myeloablative regimens in the pediatric population. The preparative regimen will result in adequate incidence of neutrophil and platelet engraftment, and acceptable rates of graft-versus-host disease (GVHD), relapse and survival. The pharmacokinetic (PK) profile of treosulfan in children will be comparable to that of adults previously studied.
Detailed description
The proposed study will evaluate a regimen using treosulfan, fludarabine and low-dose TBI in children and adolescents with AML or MDS undergoing allogeneic HCT. We expect this regimen to yield lower toxicity and at least equivalent rates of disease control and overall survival, compared to current standard myeloablative regimens. The primary objective of this study is to determine the safety and preliminary efficacy of a transplant preparative regimen consisting of treosulfan, fludarabine and low-dose TBI for children with AML and MDS. The primary endpoint will be overall survival (OS) at one year. Secondary objectives to be studied include: pharmacokinetic (PK) profile of treosulfan in children < 40 kg, non-relapse mortality, disease-free survival, incidences of neutrophil and platelet engraftment, donor chimerism, acute and chronic graft-versus-host disease (GVHD), and relapse.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| treosulfan, fludarabine and low-dose TBI prep regimen | Experimental | Drug: TreosulfanTreosulfan: 10-14 g/m2/day IV over 120 minutes on days -6, -5 and -4. Treosulfan will be administered prior to fludarabine on days -6 to -4 to facilitate PK testing. Fludarabine: 30 mg/m2 IV for patients > 10 kg (or 1 mg/kg IV for patients < 10 kg) once daily per institutional infusion standards on days -6 through -2 for a total dose of 150 mg/m2 (or 5 mg/kg). A single fraction of 200 cGy TBI will be administered on day -1. Stem cell infusion on day 0 |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (31 listed)
- Age < 21 years.
- Disease and disease status:
- Karnofsky Index or Lansky Play-Performance Scale > 70 % on pre-transplant evaluation. Karnofsky scores must be used for patients > 16 years of age and Lansky scores for patients < 16 years of age.
- Able to give informed consent if > 18 years, or with a legal guardian capable of giving informed consent if < 18 years.
- Negative pregnancy test (serum, urine β-HCG, or other test per institutional guidelines) for females of childbearing potential.
Key exclusion criteria (5 listed)
- Pregnant or lactating females are ineligible as many of the medications used in this protocol could be harmful to unborn children and infants.
- Patients with HIV or uncontrolled fungal, bacterial or viral infections are excluded. Patients with history of fungal disease during induction therapy may proceed if they have a significant response to antifungal therapy with no or minimal evidence of disease remaining by CT evaluation.
- Patients with active CNS leukemia or any other active site of extramedullary disease at the time of enrollment are not permitted. Note: Those with prior history of CNS or extramedullary disease, but with no active disease at the time of pre-transplant workup, are eligible.
- Patients undergoing a course of chemotherapy using another investigational drug.
- Patients diagnosed with Fanconi Anemia.
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 |
|---|---|---|
| TreosulfanThis is a phase II, open-label, nonrandomized, prospective study of a preparative regimen consisting of treosulfan, fludarabine and low-dose total body irradiation (TBI) for children with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic cell transplantation (HCT). | Drug | treosulfan, fludarabine and low-dose TBI prep regimen |
Primary outcome
The primary endpoint will be overall survival (OS) at one year
Time frame: 1 year
The primary objective of this study is to determine the safety and preliminary efficacy of a transplant preparative regimen consisting of treosulfan, fludarabine and low-dose TBI for children with AML and MDS. The primary endpoint will be overall survival (OS) at one year.
12 secondary outcomes
Pharmacokinetic (PK) profile of Treosulfan in children < 40 kg
Time frame: 1 year
Non-Relapse Mortality
Time frame: 1 year
Disease-Free Survival
Time frame: 1 year
Neutrophil Engraftment
Time frame: 1 year
Donor Chimerism
Time frame: 1 year
Acute graft-versus-host disease (GVHD)
Time frame: 1 year
Relapse
Time frame: 1 year
Primary graft failure
Time frame: 1 Year
Platelet engraftment of > 20,000/μL and >50,000/μL
Time frame: 1 Year
Chronic graft-versus-host disease (GVHD)
Time frame: 1 Year
Secondary graft failure
Time frame: 1 year
Primary cause of death
Time frame: 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Oct 2016.
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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.
Eneida Nemecek, MD study chair
Doernbecher Children's Hospital, Oregon Health & Science University
Colleen Delaney, MD study chair
Seattle Children's Hospital, Fred Hutchinson Cancer Research Center
23 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Treosulfan in people with Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS), run by Center for International Blood and Marrow Transplant Research at 23 sites 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 31 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Oct 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
The primary endpoint will be overall survival (OS) at one year (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Oct 2016.