ThalassemiaTHALLONCT00578292
Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO
A study testing Busulfan, Fludarabine and Campath 1H in people with Thalassemia, run by Baylor College of Medicine at 1 site in United States.
Trial summary
Patients have severe beta-thalassemia or one of the thalassemia variants. Thalassemia is a hereditary disease in which the bone marrow produces abnormal red blood cells that have a shorter life span than normal red blood cells. Read more about this study
- Who is it for?
- Ages Up to 64 years
- Conditions the study is for
- Thalassemia
- What is being tested?
- Busulfan, Fludarabine and Campath 1H
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2004to May 2016
- Phase
- Interventional
- 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 64 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients with documented diagnosis of severe (transfusion-dependent) homozygous b0/+-thalassemia or severe variants of b0/+-thalassemia requiring chronic transfusion therapy and iron chelating agents, who fulfill the following conditions:
- Patient does not have an HLA genotype-identical donor available and has a 5/6 or 6/6 matched unrelated donor, or a 5/6 matched related donor available.
- Must be between 1 and 16 yrs of age (all Pesaro risk groups).
- Patients older than 17 yrs of age must be in Pesaro Risk Class 2 or lower (see Appendix B).
- Women of childbearing potential must have a negative pregnancy test.
- Documentation of compliance with iron chelation, absence or presence of hepatomegaly, and presence or absence of hepatic fibrosis prior to transplant (criteria for the Pesaro Risk Classification). This information will be obtained by history, physical exam and interpretation of liver biopsy results.
- Documentation of awareness of alternative treatment options.
Who cannot join
- Biopsy-proven chronic active hepatitis or fibrosis with portal bridging.
- Has previous history of malignancies.
- Creatinine clearance < 35 mL/min/1.73 M2.
- Severe cardiac dysfunction defined as shortening fraction < 25%.
- HIV infection.
- Inadequate intellectual capacity to give informed consent (in the case of minors, this criteria must be fulfilled by the legal guardian).
- Be pregnant, lactating or unwilling to use appropriate birth control.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Pilot Study of Allogeneic Stem Cell Transplantation From Unrelated Donors for Patients With Severe Homozygous Beta 0/+ Thalassemia or Severe Variants of Beta 0/+ Thalassemia
Patients have severe beta-thalassemia or one of the thalassemia variants. Thalassemia is a hereditary disease in which the bone marrow produces abnormal red blood cells that have a shorter life span than normal red blood cells. Because of that, the patient has chronically low red blood cell numbers (anemia) and need regular blood transfusions to help the patient feel better and to help prevent damage to important organs such as the heart. The following treatments are currently available to patients: lifelong blood transfusions and drugs that help remove iron from the body, and long-term antibiotics to prevent infections. These treatments are difficult for patients to take, and do not stop the effects of the disease.
Currently, the only treatment that may cure thalassemia is bone marrow or blood stem cell transplantation. Special blood or bone marrow cells from a healthy person might allow the bone marrow to create healthy cells, which will replace the abnormal red blood cells of thalassemia. There is a lot of experience using special blood or bone marrow cells from a healthy brother or sister who is the same HLA (immune) type. For patients who do not have such a donor in the family, an unrelated volunteer donor can be used. It is important for the patient to realize that this kind of transplant can have more problems than a transplant from a brother or sister.
Because we do not know the long-term effects of this treatment and because this type of transplant has not been used often for people with thalassemia, this is a research study. We hope, but cannot promise, that the transplanted marrow/stem cells will produce healthy cells and the patient will no longer have severe thalassemia.
Read the detailed description
To be treated on this study, we will test the blood to check for viruses, including HIV (the virus that causes AIDS). If the HIV test is positive, a transplant cannot be done because it would be too dangerous for the patient. Secondly, we will do a liver biopsy to determine if the liver has been damaged (which can happen from iron overload that develops after many transfusions). Too much liver damage could mean that the patient will have a higher risk to develop problems with the transplant.
To participate in this study, the patients also need to have a central line (a thin plastic catheter or tube that is placed during surgery into one of the large veins in the neck or chest). Central lines are used to give intravenous medications (go directly into the vein) or to take blood samples without the patient having to endure frequent needle sticks. Before the treatment starts, we will remove a small amount of the bone marrow (back-up bone marrow) and store it. The reason for this is that if the donors bone marrow or blood stem cells do not grow properly after the transplant and the patients blood counts stay low, we can put the patients own bone marrow cells back into their body. This will help the blood counts to recover, but this means that the patient will also have thalassemia again.
To prepare the body for the transplant, the patients own blood forming system has to be destroyed and their immune system has to be weakened. To do this, they will be given high dose chemotherapy and medications that weaken their immune system (also called a conditioning treatment) for 9 days before the transplant. The main chemotherapy drugs used in the conditioning treatment are: cyclophosphamide, fludarabine and busulfan. The chemotherapy treatment will last 9 days. The patient will be admitted 10 days before the transplant to start a medicine to prevent seizures before they receive the first dose of busulfan since one of the side effects of busulfan is risk of seizures. First the patient will be given a drug called busulfan through the central line every 6 hours starting 9 days before transplant (called Day -9) until 6 days before transplant (called Day -6). Starting one day after receiving the last busulfan dose (Day -5), they will receive cyclophosphamide, fludarabine and Campath IH, which will all be given through the central line once a day for the next four days. Campath IH is a special type of protein called an antibody that works against certain types of blood cells. Also on Day -5, we will add a drug called MESNA. MESNA is used to decrease the side-effects caused by cyclophosphamide.
One day after the chemotherapy treatment is finished (Say -1) the patient will have a day to rest. On Say 0, the patient will receive the bone marrow/stem cells from the donor. Once in the bloodstream, the cells will go to the bone marrow and should begin to grow. To help prevent a problem call graft-versus-host disease (GVHD), the patient will receive a small dose of methotrexate on four different days after transplant. Another drug to help prevent GVHD, tacrolimus, will be started 2 days (Day -2) before the transplant and continued for approximately one year after the transplant. To tell whether the transplant has "taken" or "engrafted", we will take samples of blood two to three weeks after the transplant.
The patient will need to be in the hospital for at least 4 weeks after the transplant to make sure the transplant has engrafted. To find out how much the treatment has helped them and how much it might help other patients, we will do several routine lung, kidney, and liver tests, including liver biopsies, after the bone marrow/stem cell transplant. Additionally, we will be looking at the immune function. To do this, we will take 30 mL (2 tablespoonfuls) of blood every three months for the first year after transplant and then every 6 months during the second year after transplant. When possible, the blood that is taken will be taken through an existing IV line. However, at times drawing the blood will require another stick with a needle. The total amount of blood to be taken will not exceed 12 tablespoonfuls.
Because bone marrow/stem cell transplant from an unrelated volunteer donor is a new therapy for severe thalassemia and because problems may happen months afterward, the patient will need to have exams and blood tests done every few months during the first and second year following transplantation.
The patient may still need to use iron removing agents for some time after transplant or undergo blood-letting to get rid of the excess iron in the body. During that time, we will monitor the amount of iron in the body. Looking at the iron stored in the liver can most accurately tell us how much excess iron the patient has in the body. We will do liver biopsies once or twice per year if the patient is receiving iron chelation treatment after the transplant.
- Conditions
- Thalassemia
- Being tested
- Busulfan, Fludarabine, Campath 1H, Cyclophosphamide, MESNA
- Keywords
- transfusion-dependent, homozygous b0/+-thalassemia, severe variants of b0/+-thalassemia, transfusion, iron chelating agents, severe, transfusion-dependent homozygous
- Medical subject headings
- Thalassemia, Lymphoma, Follicular
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BusulfanDrug
Also called Myleran
4.0 mg/kg/day divided into four doses daily for four days; total dose = 16 mg/kg
Days -9 through -6
FludarabineDrug
Also called Fludara
30mg/m2 Day -5 through Day -2
Campath 1HDrug
Also called Alemtuzumab
Per institutional guidelines Days -5 through -2
CyclophosphamideDrug
Also called Cytoxan
50 mg/kg Days -5 through -2
MESNADrug
Also called Mesnex
10 mg/kg x 5 Days -5 through -2
| Study group | Role | What they receive |
|---|---|---|
| Bone Marrow or Stem Cell Infusion | Experimental | Mesna, Cyclophosphamide, Busulfan, Fludarabine, Campath 1H
Bone Marrow or Stem Cell infusion with pre-meds to take place on Day 0.
Bone marrow dose/stem cell dose: To ensure the probability for bone marrow engraftment, 4 x 10e8 nucleated cells/kg patient weight or 5 x 10e6/kg of CD34+ cells/kg patient weight if the product is mobilized peripheral blood, will be the target to be obtained from the unrelated donor. Receives: Drug: Busulfan; Drug: Fludarabine; Drug: Campath 1H; Drug: Cyclophosphamide; Drug: MESNA |
What is being measured
The primary outcome is the main question the study is designed to answer.
Engraftment Rate After Transplant
Engraftment is defined as an absolute neutrophil count (ANC) >500/microL x 3 days.
Measured: up to 30 days
Number of Participants With Stable Mixed Hematopoietic Chimerism (HC)
Stable hematopoietic chimerism is defined as having 50-99 percent of donor cells.
Measured: 1 year post-transplant
Number of Participants With Transient Mixed Hematopoietic Chimerism (HC)
Transient mixed hematopoietic chimerism is defined as any mixed chimerism return to 100 percent donor.
Measured: 1 year post-transplant
Number of Participants With Infectious Complications
All AEs and SAEs (including infections) will be collected for evaluation of infectious complications.
Measured: up to day 100
Hematopoietic Reconstitution
Hematopoietic: defined as transfusion independence.
Measured: 1 year post-transplant
Immune Reconstitution
Immune reconstitution: defined as absolute lymphocyte count (ALC) >1000x10e3/microL
Measured: 1 year post-transplant
Number of Participants With ACUTE GVHD
Acute GVHD is graded by the method of Przepiorka D. et al, which evaluates skin involvement, lower and upper GI, and liver function (bilirubin), each being graded in stages from 0 to 4, where 0 means no acute GVHD, and 4 is the highest stage of acute GVHD
Measured: Assessed weekly from Day 0 to day 100
Number of Participants With CHRONIC GVHD
Chronic GVHD is graded by NIH guidelines for chronic GVHD, which evaluates skin, joints, oral, ocular, hepatic, esophagus, GI, respiratory, platelet, and musculoskeletal involvement, in stages from 0 to 3 where 0 means no chronic GVHD, and 3 is the highest stage of chronic GVHD.
Measured: Assessed monthly from month 3 to month 12
Event-free Survival
Event-free survival is calculated from the date of transplant to the date of graft failure, disease recurrence or death from any cause.
Measured: up to 2 years post transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Dec 2007 (estimated)
- Study startFeb 2004
- Primary completionMay 2016
- Study completionMay 2016
Why it stopped: Stem cell transplant was determined SOC for this disease (study is not relevant)
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 ThalassemiaStudies of the same condition, open and under way.
- More trials run by Baylor College of MedicineThe 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baylor College of Medicine
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Tami D. John, Baylor College of Medicine
- Investigators
- Tami John, MD, Principal investigator, Baylor College of MedicineTami John, MD, Study director, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00578292
- Sponsor's protocol ID
- THALLO (H-14539)
- Other IDs
- H-14539
- Acronym
- THALLO
- Status last verified
- Apr 2020
- First submitted
- 19 Dec 2007
- Last updated
- 1 May 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 1 May 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of Allogeneic Stem Cell Transplantation From Unrelated Donors for Patients With Severe Homozygous Beta 0/+ Thalassemia or Severe Variants of Beta 0/+ Thalassemia
- ClinicalTrials.gov ID
- NCT00578292
- Acronym / sponsor ID
- THALLO / THALLO (H-14539)
- Conditions
- Thalassemia
- Keywords
- transfusion-dependent, homozygous b0/+-thalassemia, severe variants of b0/+-thalassemia, transfusion, iron chelating agents, severe, transfusion-dependent homozygous
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Stem cell transplant was determined SOC for this disease (study is not relevant)
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Baylor College of MedicineUniversity, hospital or other
- Study start
- Feb 2004
- Primary completion
- May 2016 (actual)
- Study completion
- May 2016 (actual)
- First posted
- 21 Dec 2007 (estimated)
- Results
- Posted 1 May 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Patients have severe beta-thalassemia or one of the thalassemia variants. Thalassemia is a hereditary disease in which the bone marrow produces abnormal red blood cells that have a shorter life span than normal red blood cells. Because of that, the patient has chronically low red blood cell numbers (anemia) and need regular blood transfusions to help the patient feel better and to help prevent damage to important organs such as the heart. The following treatments are currently available to patients: lifelong blood transfusions and drugs that help remove iron from the body, and long-term antibiotics to prevent infections. These treatments are difficult for patients to take, and do not stop the effects of the disease.
Currently, the only treatment that may cure thalassemia is bone marrow or blood stem cell transplantation. Special blood or bone marrow cells from a healthy person might allow the bone marrow to create healthy cells, which will replace the abnormal red blood cells of thalassemia. There is a lot of experience using special blood or bone marrow cells from a healthy brother or sister who is the same HLA (immune) type. For patients who do not have such a donor in the family, an unrelated volunteer donor can be used. It is important for the patient to realize that this kind of transplant can have more problems than a transplant from a brother or sister.
Because we do not know the long-term effects of this treatment and because this type of transplant has not been used often for people with thalassemia, this is a research study. We hope, but cannot promise, that the transplanted marrow/stem cells will produce healthy cells and the patient will no longer have severe thalassemia.
Detailed description
To be treated on this study, we will test the blood to check for viruses, including HIV (the virus that causes AIDS). If the HIV test is positive, a transplant cannot be done because it would be too dangerous for the patient. Secondly, we will do a liver biopsy to determine if the liver has been damaged (which can happen from iron overload that develops after many transfusions). Too much liver damage could mean that the patient will have a higher risk to develop problems with the transplant.
To participate in this study, the patients also need to have a central line (a thin plastic catheter or tube that is placed during surgery into one of the large veins in the neck or chest). Central lines are used to give intravenous medications (go directly into the vein) or to take blood samples without the patient having to endure frequent needle sticks. Before the treatment starts, we will remove a small amount of the bone marrow (back-up bone marrow) and store it. The reason for this is that if the donors bone marrow or blood stem cells do not grow properly after the transplant and the patients blood counts stay low, we can put the patients own bone marrow cells back into their body. This will help the blood counts to recover, but this means that the patient will also have thalassemia again.
To prepare the body for the transplant, the patients own blood forming system has to be destroyed and their immune system has to be weakened. To do this, they will be given high dose chemotherapy and medications that weaken their immune system (also called a conditioning treatment) for 9 days before the transplant. The main chemotherapy drugs used in the conditioning treatment are: cyclophosphamide, fludarabine and busulfan. The chemotherapy treatment will last 9 days. The patient will be admitted 10 days before the transplant to start a medicine to prevent seizures before they receive the first dose of busulfan since one of the side effects of busulfan is risk of seizures. First the patient will be given a drug called busulfan through the central line every 6 hours starting 9 days before transplant (called Day -9) until 6 days before transplant (called Day -6). Starting one day after receiving the last busulfan dose (Day -5), they will receive cyclophosphamide, fludarabine and Campath IH, which will all be given through the central line once a day for the next four days. Campath IH is a special type of protein called an antibody that works against certain types of blood cells. Also on Day -5, we will add a drug called MESNA. MESNA is used to decrease the side-effects caused by cyclophosphamide.
One day after the chemotherapy treatment is finished (Say -1) the patient will have a day to rest. On Say 0, the patient will receive the bone marrow/stem cells from the donor. Once in the bloodstream, the cells will go to the bone marrow and should begin to grow. To help prevent a problem call graft-versus-host disease (GVHD), the patient will receive a small dose of methotrexate on four different days after transplant. Another drug to help prevent GVHD, tacrolimus, will be started 2 days (Day -2) before the transplant and continued for approximately one year after the transplant. To tell whether the transplant has "taken" or "engrafted", we will take samples of blood two to three weeks after the transplant.
The patient will need to be in the hospital for at least 4 weeks after the transplant to make sure the transplant has engrafted. To find out how much the treatment has helped them and how much it might help other patients, we will do several routine lung, kidney, and liver tests, including liver biopsies, after the bone marrow/stem cell transplant. Additionally, we will be looking at the immune function. To do this, we will take 30 mL (2 tablespoonfuls) of blood every three months for the first year after transplant and then every 6 months during the second year after transplant. When possible, the blood that is taken will be taken through an existing IV line. However, at times drawing the blood will require another stick with a needle. The total amount of blood to be taken will not exceed 12 tablespoonfuls.
Because bone marrow/stem cell transplant from an unrelated volunteer donor is a new therapy for severe thalassemia and because problems may happen months afterward, the patient will need to have exams and blood tests done every few months during the first and second year following transplantation.
The patient may still need to use iron removing agents for some time after transplant or undergo blood-letting to get rid of the excess iron in the body. During that time, we will monitor the amount of iron in the body. Looking at the iron stored in the liver can most accurately tell us how much excess iron the patient has in the body. We will do liver biopsies once or twice per year if the patient is receiving iron chelation treatment after the transplant.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Bone Marrow or Stem Cell Infusion | Experimental | Drug: Busulfan, Drug: Fludarabine, Drug: Campath 1H, Drug: Cyclophosphamide, Drug: MESNAMesna, Cyclophosphamide, Busulfan, Fludarabine, Campath 1H Bone Marrow or Stem Cell infusion with pre-meds to take place on Day 0. Bone marrow dose/stem cell dose: To ensure the probability for bone marrow engraftment, 4 x 10e8 nucleated cells/kg patient weight or 5 x 10e6/kg of CD34+ cells/kg patient weight if the product is mobilized peripheral blood, will be the target to be obtained from the unrelated donor. |
Who may qualify
- Ages
- Up to 64 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Patients with documented diagnosis of severe (transfusion-dependent) homozygous b0/+-thalassemia or severe variants of b0/+-thalassemia requiring chronic transfusion therapy and iron chelating agents, who fulfill the following conditions:
- Patient does not have an HLA genotype-identical donor available and has a 5/6 or 6/6 matched unrelated donor, or a 5/6 matched related donor available.
- Must be between 1 and 16 yrs of age (all Pesaro risk groups).
- Patients older than 17 yrs of age must be in Pesaro Risk Class 2 or lower (see Appendix B).
- Women of childbearing potential must have a negative pregnancy test.
Key exclusion criteria (7 listed)
- Biopsy-proven chronic active hepatitis or fibrosis with portal bridging.
- Has previous history of malignancies.
- Creatinine clearance < 35 mL/min/1.73 M2.
- Severe cardiac dysfunction defined as shortening fraction < 25%.
- HIV infection.
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 |
|---|---|---|
| BusulfanAlso: Myleran4.0 mg/kg/day divided into four doses daily for four days; total dose = 16 mg/kg Days -9 through -6 | Drug | Bone Marrow or Stem Cell Infusion |
| FludarabineAlso: Fludara30mg/m2 Day -5 through Day -2 | Drug | Bone Marrow or Stem Cell Infusion |
| Campath 1HAlso: AlemtuzumabPer institutional guidelines Days -5 through -2 | Drug | Bone Marrow or Stem Cell Infusion |
| CyclophosphamideAlso: Cytoxan50 mg/kg Days -5 through -2 | Drug | Bone Marrow or Stem Cell Infusion |
| MESNAAlso: Mesnex10 mg/kg x 5 Days -5 through -2 | Drug | Bone Marrow or Stem Cell Infusion |
Primary outcome
Engraftment Rate After Transplant
Time frame: up to 30 days
Engraftment is defined as an absolute neutrophil count (ANC) >500/microL x 3 days.
Number of Participants With Stable Mixed Hematopoietic Chimerism (HC)
Time frame: 1 year post-transplant
Stable hematopoietic chimerism is defined as having 50-99 percent of donor cells.
Number of Participants With Transient Mixed Hematopoietic Chimerism (HC)
Time frame: 1 year post-transplant
Transient mixed hematopoietic chimerism is defined as any mixed chimerism return to 100 percent donor.
Number of Participants With Infectious Complications
Time frame: up to day 100
All AEs and SAEs (including infections) will be collected for evaluation of infectious complications.
Hematopoietic Reconstitution
Time frame: 1 year post-transplant
Hematopoietic: defined as transfusion independence.
Immune Reconstitution
Time frame: 1 year post-transplant
Immune reconstitution: defined as absolute lymphocyte count (ALC) >1000x10e3/microL
Number of Participants With ACUTE GVHD
Time frame: Assessed weekly from Day 0 to day 100
Acute GVHD is graded by the method of Przepiorka D. et al, which evaluates skin involvement, lower and upper GI, and liver function (bilirubin), each being graded in stages from 0 to 4, where 0 means no acute GVHD, and 4 is the highest stage of acute GVHD
Number of Participants With CHRONIC GVHD
Time frame: Assessed monthly from month 3 to month 12
Chronic GVHD is graded by NIH guidelines for chronic GVHD, which evaluates skin, joints, oral, ocular, hepatic, esophagus, GI, respiratory, platelet, and musculoskeletal involvement, in stages from 0 to 3 where 0 means no chronic GVHD, and 3 is the highest stage of chronic GVHD.
Event-free Survival
Time frame: up to 2 years post transplant
Event-free survival is calculated from the date of transplant to the date of graft failure, disease recurrence or death from any cause.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 May 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.
Tami John, MD principal investigator
Baylor College of Medicine
Tami John, MD study director
Baylor College of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Busulfan, Fludarabine and Campath 1H in people with Thalassemia, run by Baylor College of Medicine 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 7 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: stopped early, as of the sponsor's last update on 1 May 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.
Are results available for this study?
Yes. Results are posted in the registry (first posted 1 May 2020). 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?
Engraftment Rate After Transplant (time frame: up to 30 days). The study lists 9 primary outcomes in all. 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 1 May 2020.