Sickle Cell DiseasePhase 2/3NCT00176852
Stem Cell Transplant for Hemoglobinopathy
A Phase 2/3 study testing Busulfan, Fludarabine, ATG, TLI, Busulfan, Cyclophosphamide, ATG, GCSF and Campath, Fludarabine, Cyclophosphamide in people with Sickle Cell Disease and Thalassemia, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial summary
This study tests the clinical outcomes of one of two preparative regimens (determined by available donor source) in patients with non-malignant hemoglobinopathies. The researchers hypothesize that these regimens will have a positive effect on post transplant engraftment and the incidence of graft-versus-host-disease. Read more about this study
- Who is it for?
- Ages Up to 50 years
- Conditions the study is for
- Sickle Cell Disease, Thalassemia, Severe Congenital Neutropenia
- What is being tested?
- Busulfan, Fludarabine, ATG, TLI, Busulfan, Cyclophosphamide, ATG, GCSF and Campath, Fludarabine, Cyclophosphamide
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2002to Jan 2020
- Phase
- Phase 2/3
- 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 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients with Sickle Cell Disease/Thalassemia (SCD/THAL) 0-50 years of age with an acceptable stem cell donor and disease characteristic defined by the following:
- Stroke, central nervous system (CNS) hemorrhage or a neurologic event lasting longer than 24 hours, or abnormal cerebral magnetic resonance imaging (MRI) or cerebral arteriogram or MRI angiographic study and impaired neuropsychological testing
- Acute chest syndrome with a history of recurrent hospitalizations or exchange transfusions
- Recurrent vaso-occlusive pain 3 or more episodes per year for 3 years or more years or recurrent priapism,
- Impaired neuropsychological function and abnormal cerebral MRI scan
- Stage I or II sickle lung disease,
Show all 20Show fewer
- Sickle nephropathy (moderate or severe proteinuria or a glomerular filtration rate [GFR] 30-50% of the predicted normal value)
- Bilateral proliferative retinopathy and major visual impairment in at least one eye
- Osteonecrosis of multiple joints with documented destructive changes
- Requirement for chronic transfusions but with red blood cell (RBC) alloimmunization >2 antibodies during long term transfusion therapy
- Patients with transfusion dependent alpha- or beta-thalassemia 0-35 years of age with an acceptable stem cell donor as defined in the criteria in section above.
- Patients with other non-malignant hematologic disorders that are transfusion-dependent or involve other potentially life-threatening cytopenias (including but not limited to Severe Congenital Neutropenia, Diamond-Blackfan Anemia and Shwachman-Diamond Syndrome) who are 0-35 years of age with an acceptable stem cell donor
- Second Transplants
- Patients with sickle cell disease or thalassemia who have failed to engraft or have autologous recovery after a myeloablative SCT regimen or non-myeloablative regimen are eligible for this protocol.
- Regimen A2 will be utilized for patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or for any patient who has pre-existing organ dysfunction making them ineligible for a myeloablative preparative regimen.
- Regimen B will be utilized for patients with sickle cell disease or thalassemia who have an HLA-identical sibling donor.
- Patients must meet above criteria.
- If the patient has received prior radiation therapy, eligibility to receive additional radiation therapy must be determined by Dr. Dusenbery
- If first transplant was a non-myeloablative regimen, the second transplant can occur at any time
- If the first transplant was a myeloablative regimen, then the second transplant must be > 6 months from the first transplant
Who cannot join
- Patients with one or more of the following:
- Karnofsky or Lansky performance score <70
- Acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy
- Stage III-IV lung disease
- GFR<30% predicted
- Pregnant or lactating females
Show all 9Show fewer
- Active serious infection whereby patient has been on intravenous antibiotics for one week prior to study entry. Any patient with AIDS or ARC or HIV seropositivity
- Psychologically incapable of undergoing bone marrow transplant (BMT) with associated strict isolation or documented history of medical non-compliance
- Patients not able to receive total lymphocytic irradiation (TLI) due to prior radiation therapy
Trial locations and contacts
1 site in 1 country.
United States1 site
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, 55455
About this study
Official title: Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathy Using a Preparative Regimen to Achieve Stable Mixed Chimerism
This study tests the clinical outcomes of one of two preparative regimens (determined by available donor source) in patients with non-malignant hemoglobinopathies. The researchers hypothesize that these regimens will have a positive effect on post transplant engraftment and the incidence of graft-versus-host-disease.
Regimen A2 has replaced Regimen A in this study. Two patients were treated on Regimen A but did not have evidence of initial engraftment thus triggering the stopping rule for that arm of this study.
Read the detailed description
Prior to transplantation, subjects will receive either:
Cyclophosphamide, Fludarabine, Campath, Total body irradiation (TBI)
Or
Busulfan, Cyclophosphamide, antithymocyte globulin (ATG), granulocyte colony-stimulating factor (GSCF)
These drugs (and the radiation) are being given to help the new stem cells take and grow. On the day of transplantation, subjects will receive stem cells transfused via intravenous (IV) catheter.
After stem cell transplantation, subjects will be given cyclosporine-A and mycophenolate (MMF)/or Methylprednisone/or Methotrexate to reduce the risk of graft-versus-host disease, the complication that occurs when the donor's stem cells react against the patient.
- Conditions
- Sickle Cell Disease, Thalassemia, Severe Congenital Neutropenia, Diamond-Blackfan Anemia, Shwachman-Diamond Syndrome
- Being tested
- Busulfan, Fludarabine, ATG, TLI, Busulfan, Cyclophosphamide, ATG, GCSF, Campath, Fludarabine, Cyclophosphamide, Total Body Irradiation, Stem cell infusion
- Keywords
- high risk hemoglobinopathy, stem cell transplant, donor lymphocyte infusion, transfusion dependent, stem cell donor, cord blood, marrow, transfusion dependent non-malignant hematologic disorders
- Medical subject headings
- Anemia, Sickle Cell, Thalassemia, Neutropenia, Severe Congenital, Autosomal Recessive 3, Anemia, Diamond-Blackfan, Shwachman-Diamond Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Busulfan, Fludarabine, ATG, TLIDrug
Also called Busulfex, Fludara, ATG, Total lymphoid Irradiation
Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy
Busulfan, Cyclophosphamide, ATG, GCSFDrug
Also called Busulfex, Cytoxan, antithymocyte globulin, granulocyte colony-stimulating factor
Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC >2500 x 2 days.
Campath, Fludarabine, CyclophosphamideDrug
Also called Fludara, alemtuzumab, Cytoxan
Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
Total Body IrradiationRadiation
Also called TBI
300 cGY Day -1
Stem cell infusionProcedure or surgery
Also called bone marrow transplant
Given Day 0
| Study group | Role | What they receive |
|---|---|---|
| RIC Bu/Flu (A) (discontinued) | Other | Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0. Receives: Drug: Busulfan, Fludarabine, ATG, TLI |
| MA Bu/Cy (B) | Experimental | Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days. Receives: Drug: Busulfan, Cyclophosphamide, ATG, GCSF; Radiation: Total Body Irradiation; Procedure: Stem cell infusion |
| RIC Cy/Flu/TBI (A2) | Experimental | Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0. Receives: Drug: Campath, Fludarabine, Cyclophosphamide; Radiation: Total Body Irradiation; Procedure: Stem cell infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Patients Who Experienced Grade 3-5 Treatment Related Toxicity
In general, grade 3 equates to moderate, grade 4 to severe and grade 5 to death.
Measured: 1 year
Secondary outcomes (16)
The Incidence of Chimerism at 100 Days
The number of patients whose blood and/or bone marrow contains > 10% donor cells.
Measured: 100 days
The Incidence of Chimerism at 6 Months
The number of patients whose blood and/or bone marrow contains > 10% donor cells.
Measured: 6 months
The Incidence of Chimerism at 1 Year
The number of patients whose blood and/or bone marrow contains > 10% donor cells.
Measured: 1 year
The Incidence of Grade 2-4 Acute Graft Versus Host Disease (Acute GVHD)
The number of patients who experienced grades 2-4 Acute GVHD. Acute GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. Grades 2-4 equate to mild to severe disease. Symptoms typically appear within weeks after transplant.
Measured: 100 days
The Incidence of Grade 3-4 Acute Graft Versus Host Disease (Acute GVHD)
The number of patients who experienced grades 3-4 Acute GVHD. Acute GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. IGrades 3-4 equate to moderate to severe disease. Symptoms typically appear within weeks after transplant.
Measured: 100 days
The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD)
The number of patients who experienced Chronic GVHD. Chronic GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. Chronic GVHD can appear at any time after allogeneic transplant or several years after transplant.
Measured: 6 months
The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD)
The number of patients who experienced Chronic GVHD. Chronic GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. Chronic GVHD can appear at any time after allogeneic transplant or several years after transplant.
Measured: 1 year
Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment
The measure for quality of life used in this study is the Karnofsky Performance Score. The Karnofsky Performance Score runs from 100 to 0, where 100 is "perfect" health and 0 is death.
Measured: pre-transplant
Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment
The measure for quality of life used in this study is the Karnofsky Performance Score. The Karnofsky Performance Score runs from 100 to 0, where 100 is "perfect" health and 0 is death.
Measured: 1 year
Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment
The measure for quality of life used in this study is the Karnofsky Performance Score. The Karnofsky Performance Score runs from 100 to 0, where 100 is "perfect" health and 0 is death.
Measured: 2 years
Determine Physical Characteristics and Biologic Effects of Mixed Populations of Donor and Host Red Blood Cells
Measured: During study
Determine the Concentration of Campath in the Serum
Measured: Day 0
Overall Survival
Number of patients alive 100 days after transplant.
Measured: 100 days
Overall Survival
Number of patients alive 1 year after transplant.
Measured: 1 year
Disease Free Survival
Number of patients alive without disease 100 days after transplant.
Measured: 100 days
Disease Free Survival
Number of patients alive without disease 1 year after transplant.
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 posted15 Sep 2005 (estimated)
- Study startJun 2002
- Primary completionMar 2014
- Study completionJan 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 Sickle Cell DiseaseStudies 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 2/3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Collaborators
- National Marrow Donor Program
- Responsible party
- Sponsor
- Investigators
- Angela Smith, MD, Principal investigator, Masonic Cancer Center, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00176852
- Sponsor's protocol ID
- MT2002-07
- Other IDs
- 0206M26241
- Status last verified
- Feb 2020
- First submitted
- 12 Sep 2005
- Last updated
- 27 Feb 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 27 Feb 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathy Using a Preparative Regimen to Achieve Stable Mixed Chimerism
- ClinicalTrials.gov ID
- NCT00176852
- Acronym / sponsor ID
- MT2002-07
- Conditions
- Sickle Cell Disease, Thalassemia, Severe Congenital Neutropenia, Diamond-Blackfan Anemia, Shwachman-Diamond Syndrome
- Keywords
- high risk hemoglobinopathy, stem cell transplant, donor lymphocyte infusion, transfusion dependent, stem cell donor, cord blood, marrow, transfusion dependent non-malignant hematologic disorders
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Collaborators
- National Marrow Donor Program
- Study start
- Jun 2002
- Primary completion
- Mar 2014 (actual)
- Study completion
- Jan 2020 (actual)
- First posted
- 15 Sep 2005 (estimated)
- Results
- Posted 9 May 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study tests the clinical outcomes of one of two preparative regimens (determined by available donor source) in patients with non-malignant hemoglobinopathies. The researchers hypothesize that these regimens will have a positive effect on post transplant engraftment and the incidence of graft-versus-host-disease.
Regimen A2 has replaced Regimen A in this study. Two patients were treated on Regimen A but did not have evidence of initial engraftment thus triggering the stopping rule for that arm of this study.
Detailed description
Prior to transplantation, subjects will receive either:
Cyclophosphamide, Fludarabine, Campath, Total body irradiation (TBI)
Or
Busulfan, Cyclophosphamide, antithymocyte globulin (ATG), granulocyte colony-stimulating factor (GSCF)
These drugs (and the radiation) are being given to help the new stem cells take and grow. On the day of transplantation, subjects will receive stem cells transfused via intravenous (IV) catheter.
After stem cell transplantation, subjects will be given cyclosporine-A and mycophenolate (MMF)/or Methylprednisone/or Methotrexate to reduce the risk of graft-versus-host disease, the complication that occurs when the donor's stem cells react against the patient.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| RIC Bu/Flu (A) (discontinued) | Other | Drug: Busulfan, Fludarabine, ATG, TLIFull Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0. |
| MA Bu/Cy (B) | Experimental | Drug: Busulfan, Cyclophosphamide, ATG, GCSF, Radiation: Total Body Irradiation, Procedure: Stem cell infusionMyeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days. |
| RIC Cy/Flu/TBI (A2) | Experimental | Drug: Campath, Fludarabine, Cyclophosphamide, Radiation: Total Body Irradiation, Procedure: Stem cell infusionPatients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0. |
Who may qualify
- Ages
- Up to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (20 listed)
- Patients with Sickle Cell Disease/Thalassemia (SCD/THAL) 0-50 years of age with an acceptable stem cell donor and disease characteristic defined by the following:
- Patients with transfusion dependent alpha- or beta-thalassemia 0-35 years of age with an acceptable stem cell donor as defined in the criteria in section above.
- Patients with other non-malignant hematologic disorders that are transfusion-dependent or involve other potentially life-threatening cytopenias (including but not limited to Severe Congenital Neutropenia, Diamond-Blackfan Anemia and Shwachman-Diamond Syndrome) who are 0-35 years of age with an acceptable stem cell donor
- Second Transplants
Key exclusion criteria (9 listed)
- Patients with one or more of the following:
- Karnofsky or Lansky performance score <70
- Acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy
- Stage III-IV lung disease
- GFR<30% predicted
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 |
|---|---|---|
| Busulfan, Fludarabine, ATG, TLIAlso: Busulfex, Fludara, ATG, Total lymphoid IrradiationBusulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy | Drug | RIC Bu/Flu (A) (discontinued) |
| Busulfan, Cyclophosphamide, ATG, GCSFAlso: Busulfex, Cytoxan, antithymocyte globulin, granulocyte colony-stimulating factorBusulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC >2500 x 2 days. | Drug | MA Bu/Cy (B) |
| Campath, Fludarabine, CyclophosphamideAlso: Fludara, alemtuzumab, CytoxanReceives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1. | Drug | RIC Cy/Flu/TBI (A2) |
| Total Body IrradiationAlso: TBI300 cGY Day -1 | Radiation | MA Bu/Cy (B), RIC Cy/Flu/TBI (A2) |
| Stem cell infusionAlso: bone marrow transplantGiven Day 0 | Procedure or surgery | MA Bu/Cy (B), RIC Cy/Flu/TBI (A2) |
Primary outcome
Number of Patients Who Experienced Grade 3-5 Treatment Related Toxicity
Time frame: 1 year
In general, grade 3 equates to moderate, grade 4 to severe and grade 5 to death.
16 secondary outcomes
The Incidence of Chimerism at 100 Days
Time frame: 100 days
The Incidence of Chimerism at 6 Months
Time frame: 6 months
The Incidence of Chimerism at 1 Year
Time frame: 1 year
The Incidence of Grade 2-4 Acute Graft Versus Host Disease (Acute GVHD)
Time frame: 100 days
The Incidence of Grade 3-4 Acute Graft Versus Host Disease (Acute GVHD)
Time frame: 100 days
The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD)
Time frame: 6 months
The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD)
Time frame: 1 year
Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment
Time frame: pre-transplant
Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment
Time frame: 1 year
Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment
Time frame: 2 years
Determine Physical Characteristics and Biologic Effects of Mixed Populations of Donor and Host Red Blood Cells
Time frame: During study
Determine the Concentration of Campath in the Serum
Time frame: Day 0
Overall Survival
Time frame: 100 days
Overall Survival
Time frame: 1 year
Disease Free Survival
Time frame: 100 days
Disease Free Survival
Time frame: 1 year
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Feb 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Angela Smith, MD principal investigator
Masonic Cancer Center, University of Minnesota
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Busulfan, Fludarabine, ATG, TLI, Busulfan, Cyclophosphamide, ATG, GCSF and Campath, Fludarabine, Cyclophosphamide in people with Sickle Cell Disease and Thalassemia, run by Masonic Cancer Center, University of Minnesota 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 20 inclusion and 9 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 27 Feb 2020. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 9 May 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?
Number of Patients Who Experienced Grade 3-5 Treatment Related Toxicity (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 27 Feb 2020.