Sickle Cell DiseaseSCALLOPNCT00578344
Bone Marrow Transplantation, Hemoglobinopathies, SCALLOP
A study testing Busulfan, Campath 1H and Cyclophosphamide and MESNA in people with Sickle Cell Disease and Hemoglobin SC, run by Tami D. John at 2 sites in United States.
Trial summary
Patients are being asked to participate in this study because they have severe sickle cell anemia (SCD) with or without the beta thalassemia trait. Sickle cell anemia is an illness where the red blood cells change shape and can clog up blood vessels. This keeps the body from getting the oxygen it needs. Read more about this study
- Who is it for?
- Ages Up to 40 years
- Conditions the study is for
- Sickle Cell Disease, Hemoglobin SC
- What is being tested?
- Busulfan, Campath 1H and Cyclophosphamide and MESNA
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 2 sitesUnited States
- Study dates
- Jul 2005to Jul 2012
- 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 40 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients with a related HLA genotype identical donor and hemoglobin SS, hemoglobin SC, or hemoglobin Sb0/+ and at least one of the following conditions:
- Previous central nervous system vaso-occlusive episode with or without residual neurologic findings, or has an abnormal transcranial doppler exam without neurologic findings, or abnormal MRI/MRA of the brain with or without neurologic findings;
- Frequent painful vaso-occlusive episodes which significantly interfere with normal life activities and which necessitate chronic transfusion therapy;
- Recurrent SCD chest syndrome events, which necessitate chronic transfusion therapy;
- Severe anemia which prevents acceptable quality of life and necessitates chronic transfusion therapy;
- Any of the above symptoms in which the patient is not undergoing chronic transfusion therapy;
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- The patient is undergoing chronic transfusion therapy for symptoms other than those listed and which significantly interferes with normal life activities;
- Failed hydroxyurea therapy;
- Indication of pulmonary hypertension on 2 separate echocardiogram examinations;
- Patients who plan to return to resource poor areas/countries.
- Between the ages of birth and 40 years.
- Women of childbearing potential must have a negative pregnancy test.
Who cannot join
- Patient with biopsy proven chronic active hepatitis or fibrosis with portal bridging.
- Patient with SCD chronic lung disease > stage 3 (see Appendix 1).
- Patient with severe renal dysfunction defined as creatinine clearance < 40 mL/min/1.73 M^2.
- Patient with severe cardiac dysfunction defined as echocardiogram shortening fraction < 25% or NYHA class III or IV.
- Patient with HIV infection.
- Patient with unspecified chronic toxicity serious enough to detrimentally affect the patient's capacity to tolerate bone marrow transplantation.
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- Patient or patient's guardian(s) unable to understand the nature and risks inherent in the BMT process.
- Pregnant/lactating women and those unwilling to use acceptable contraception will be excluded.
- Patient or patient's guardian who have not signed an informed consent.
- NOTE: Patients who would be excluded from the protocol strictly for laboratory abnormalities can be included at the investigator's discretion after approval by the CAGT Protocol Review Committee and the FDA reviewer.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Methodist Hospital
Houston, Texas, 77030
Texas Children's Hospital
Houston, Texas, 77030
About this study
Official title: Allogeneic Bone Marrow Transplantation From HLA Identical Related Donors for Patients With Hemoglobinopathies: Hemoglobin SS, Hemoglobin SC, or Hemoglobin SB0/+ Thalassemia
Patients are being asked to participate in this study because they have severe sickle cell anemia (SCD) with or without the beta thalassemia trait. Sickle cell anemia is an illness where the red blood cells change shape and can clog up blood vessels. This keeps the body from getting the oxygen it needs. Thalassemia is when the body does not make enough hemoglobin, something that helps the oxygen get to the places it needs to go in the body. The patient may or may not need to get regular blood transfusions (getting more blood) to improve their quality of life (feel better) and prevent organ damage (problems with the brain, heart, lung, kidney, and gonad, for example.). The transfusions can also cause problems, including iron overload (too much iron in the blood), which can be fatal (patients can die) without regular deferoxamine shots. Even with the best usual treatments, people with thalassemia or SCD die sooner. There is no proven cure.
We would like to treat patients using bone marrow transplantation, a treatment that has been used for people with SCD. The transplant uses healthy "matched" bone marrow. This comes from a brother or sister who does not have sickle cell disease or severe thalassemia. If the treatment works, the sickle cell disease or thalassemia may be cured. This treatment has been used to treat patients with sickle cell disease or thalassemia. It has worked in most cases. We hope, but cannot promise, that the transplanted marrow will make healthy cells, and patients will not have sickle cell disease or severe thalassemia anymore.
We do not know what effect this treatment will have on the damage that has already been done by the disease. Finding that out is the main reason for this study. Currently, very little has been reported about organ function after bone marrow transplants in patients with sickle cell anemia.
Read the detailed description
Prior to any bone marrow transplant, we will need patients to:
- Answer questions about their medical history;
- Undergo a physical examination;
- Have tests done to see how well the lungs are working;
- Have a chest x-ray;
- Have an EKG to look at the heart;
- Have an MRI \& MRA (Magnetic Resonance Angiography, which looks at the blood vessels and the blood flowing through them). These 2 tests will look to see if the patient has had any strokes;
- Have a PET scan to look at the head and body;
- Have a liver biopsy to determine if the liver has been damaged (which can happen from iron overload that happens after many transfusions). Too much liver damage would mean a transplant cannot be done.
For the liver biopsy, the skin is numbed with medicine, and a special needle goes into the liver. The needle removes a very small piece of the liver (tissue). The tissue is taken and examined.
Also, about 30 cc (2 tablespoons) of blood will be drawn to test the blood for viruses, including HIV (the virus that causes AIDS). If the HIV test is positive, a transplant will not be done because it would be too dangerous for the patient.
At least 2 weeks before the bone marrow infusion, the patient will be immunized with Prevnar 7. Prevnar 7 is a vaccine that is used in children to protect against some types of bacteria called Streptococcus pneumoniae, which can cause the lung infection called pneumonia. People with Sickle Cell anemia are at a higher risk of dying from an infection from this type of bacteria. Although children are regularly given the Prevnar 7 vaccine, it is not common to test a child's immune response to the vaccine. In this study, we will check the immune system's response to the vaccine by drawing an extra 3 mL (less than 1 teaspoon) of blood 3 weeks after the patient gets the Prevnar 7 vaccine.
Just before the bone marrow transplant, we must kill the cells in the bone marrow that make the abnormal red blood cells found in patients with severe thalassemia or sickle cell disease. We will do this by using 3 drugs: busulfan, cyclophosphamide, and campath-1H. Campath-1H is used to prevent the body from rejecting or refusing to let the donor blood cells grow in the body. MESNA is given with the cyclophosphamide to prevent kidney damage. Methotrexate and cyclosporin are also given to prevent graft-versus-host disease (GVHD); methylprednisolone will be given after the bone marrow infusion if the patient develops GVHD. A drug will also be given to prevent seizures (either dilantin or lorazepam).
Graft-versus-host disease (GVHD) is a possible side-effect of the transplant. In GVHD, some cells in the donor marrow attack cells in the patient's body. This causes skin, liver, and bowel problems, and may damage other parts of the body. Often, these problems are fairly mild, but they can be severe or even cause death. Severe GVHD is likely to occur in about 10% of patients.
After the patient gets the drug treatment, they will be given bone marrow from a brother or sister who has healthy bone marrow that matches the patients. The healthy bone marrow will be put into a vein (given IV) in the same way that blood transfusions are given. The marrow cells then travel to the right places in the body, where they should grow and make new normal blood cells.
This is the treatment schedule:
Protocol Day \& Treatment:
14 or more days before the bone marrow infusion -- Prevnar 7 vaccine
10 days before the bone marrow infusion -- Begin Dilantin or Lorazepam (to prevent seizures)
9 days before the bone marrow infusion -- Busulfan
8 days before the bone marrow infusion -- Busulfan
7 days before the bone marrow infusion -- Busulfan
6 days before the bone marrow infusion -- Busulfan
5 days before the bone marrow infusion -- Campath 1H, Cyclophosphamide, and MESNA
4 days before the bone marrow infusion -- Campath 1H, Cyclophosphamide, and MESNA
3 days before the bone marrow infusion -- Campath 1H, Cyclophosphamide, and MESNA
2 days before the bone marrow infusion -- Campath 1H, Cyclophosphamide, MESNA, and cyclosporin
1 day before the bone marrow infusion -- DAY OF REST
Day "0" -- bone marrow infusion given
1 day after the bone marrow infusion -- Methotrexate
3 days after the bone marrow infusion -- Methotrexate
6 days after the bone marrow infusion -- Methotrexate
11 days after the bone marrow infusion -- Methotrexate
After transplant, Filgrastrim, a growth hormone for the bone marrow, may be given intravenously (through a vein), if medically necessary.
Sometimes, the donor's bone marrow does not grow. Then, the patient would be without working bone marrow cells. So that we will be ready to treat this problem if it happens, we will take bone marrow from the hip bone before the patient gets the drug treatment. The patient will be asleep (sedated) when the marrow is taken, and they will be given medicine for any pain they have afterwards. This bone marrow will be frozen and stored or preserved. If the donor bone marrow does not grow, we will thaw the stored marrow and put it back into the body. This stored bone marrow should grow and produce working blood cells. If the stored bone marrow must be given back, the disease will not be cured.
To tell whether the transplant has worked or "engrafted", we will take samples of bone marrow (bone marrow aspirate). We will do this 3 weeks after the transplant to make sure the new cells are beginning to grow. We will take another marrow sample at 3 months after the transplant. We want to make sure the new cells are still growing. This test will take about 30 to 45 minutes. Because this test is painful, the patient will be given pain medicine before, during, and after the test.
The patient will need to be in the hospital for at least 3 weeks after the transplant to make sure the transplant has engrafted. We will do several tests (of the lung, kidney, and liver) before and after the bone marrow transplant. We want to find out how much the treatment has helped the patient and how much it might help other patients. Most tests will be done every week for 4 months and at each visit to the hospital. Also, we will be looking at the immune function. To do this, we will take about 8 ml (less than 2 teaspoonful) at 6 months, 1 year, and 2 years after the transplant. When possible, we will take the blood from an IV line that the patient already has. However, at times we will have to draw the blood with another needle stick. A total amount of 200 mL (about 13 tablespoons) of blood will be collected from the patient during the entire study.
After 4 months, if the patient's health is good, they will not need to come to the hospital so often. The visits to the doctor should be more like they were before the bone marrow transplant. Since problems may happen months after the transplant and this is a new way to treat sickle cell disease and thalassemia the patient will need to have exams and blood tests done every few months during the first and second years after their transplant. After that, if all is well, the patient will need to be examined and have blood tests 4 times a year.
- Conditions
- Sickle Cell Disease, Hemoglobin SC
- Being tested
- Busulfan, Campath 1H, Cyclophosphamide and MESNA
- Keywords
- Sickle Cell Disease, SCD, Hemoglobin SS, Hemoglobin SC, Hemoglobin Sb0/+, HLA genotype, Severe anemia, Transfusion therapy
- Medical subject headings
- Anemia, Sickle Cell, Hemoglobin SC Disease, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BusulfanDrug
Also called Myleran
Starting Day -9 / Busulfan 4.0 mg/kg/day IV divided into four doses daily for four days; total dose = 16 mg/kg.
Campath 1HBiological
Day -5 through Day -2; Campath-1H dosed as per institutional guidelines.
Cyclophosphamide and MESNADrug
Also called CTX
Day -5 through Day -2; Cyclophosphamide 50 mg/kg + MESNA.
| Study group | Role | What they receive |
|---|---|---|
| Allogeneic BMT/SCT Transplant | Experimental | Busulfan, Campath 1H, Cyclophosphamide and MESNA:
Bone marrow infusion with pre-meds as per SOPs to take place on Day 0.
Bone marrow dose: To ensure the probability for bone marrow engraftment, 4 x 10^8 nucleated cells/kg patient weight will be the target at donor bone marrow harvest. Receives: Drug: Busulfan; Biological: Campath 1H; Drug: Cyclophosphamide and MESNA |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Evaluated for Evidence of Recovery of Organ Function Measured Via MRI or PET Scan.
Assess Number of participants that recovered organ function diagnosed with sickle cell disease (SCD) or sickle hemoglobin variants after undergoing allogeneic SCT/BMT from HLA genotype identical donors.
Measured: One year
Number of Participants With Pre and Post Transplant PET Scan to Assess Organ Recovery Based on Rate of Acquisition.
Number of participants that did or did not have pre- and post- PET scans.
Measured: One year
Number of Participants With Immune Response to Immunization After BMT in Participants With SCD, Hemoglobin SC, or Hemoglobin Sb0/+.
Vaccine response will be measured via a humoral immunity panel evaluating streptococcus pneumonia IgG antibodies (microgram/mL). Panels are obtained pre and 1+ month post vaccination. Standard recommendations define a normal responder as having >4 fold increase in antibody level in 50-70% of the serotypes found in the vaccine.
Measured: up to 12 months
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 startJul 2005
- Primary completionJul 2012
- Study completionJul 2012
Why it stopped: Terminated due to no new subject enrollment during the last 3 year period.
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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Tami D. John
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Tami D. John, Baylor College of Medicine
- Investigators
- Tami John, MD, Study director, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00578344
- Sponsor's protocol ID
- H-16447-SCALLOP
- Other IDs
- SCALLOP
- Acronym
- SCALLOP
- Status last verified
- Jul 2020
- First submitted
- 19 Dec 2007
- Last updated
- 31 Jul 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 31 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allogeneic Bone Marrow Transplantation From HLA Identical Related Donors for Patients With Hemoglobinopathies: Hemoglobin SS, Hemoglobin SC, or Hemoglobin SB0/+ Thalassemia
- ClinicalTrials.gov ID
- NCT00578344
- Acronym / sponsor ID
- SCALLOP / H-16447-SCALLOP
- Conditions
- Sickle Cell Disease, Hemoglobin SC
- Keywords
- Sickle Cell Disease, SCD, Hemoglobin SS, Hemoglobin SC, Hemoglobin Sb0/+, HLA genotype, Severe anemia, Transfusion therapy
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Terminated due to no new subject enrollment during the last 3 year period.
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Tami D. JohnUniversity, hospital or other
- Collaborators
- Baylor College of Medicine, The Methodist Hospital Research Institute
- Study start
- Jul 2005
- Primary completion
- Jul 2012 (actual)
- Study completion
- Jul 2012 (actual)
- First posted
- 21 Dec 2007 (estimated)
- Results
- Posted 31 Jul 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Patients are being asked to participate in this study because they have severe sickle cell anemia (SCD) with or without the beta thalassemia trait. Sickle cell anemia is an illness where the red blood cells change shape and can clog up blood vessels. This keeps the body from getting the oxygen it needs. Thalassemia is when the body does not make enough hemoglobin, something that helps the oxygen get to the places it needs to go in the body. The patient may or may not need to get regular blood transfusions (getting more blood) to improve their quality of life (feel better) and prevent organ damage (problems with the brain, heart, lung, kidney, and gonad, for example.). The transfusions can also cause problems, including iron overload (too much iron in the blood), which can be fatal (patients can die) without regular deferoxamine shots. Even with the best usual treatments, people with thalassemia or SCD die sooner. There is no proven cure.
We would like to treat patients using bone marrow transplantation, a treatment that has been used for people with SCD. The transplant uses healthy "matched" bone marrow. This comes from a brother or sister who does not have sickle cell disease or severe thalassemia. If the treatment works, the sickle cell disease or thalassemia may be cured. This treatment has been used to treat patients with sickle cell disease or thalassemia. It has worked in most cases. We hope, but cannot promise, that the transplanted marrow will make healthy cells, and patients will not have sickle cell disease or severe thalassemia anymore.
We do not know what effect this treatment will have on the damage that has already been done by the disease. Finding that out is the main reason for this study. Currently, very little has been reported about organ function after bone marrow transplants in patients with sickle cell anemia.
Detailed description
Prior to any bone marrow transplant, we will need patients to:
- Answer questions about their medical history;
- Undergo a physical examination;
- Have tests done to see how well the lungs are working;
- Have a chest x-ray;
- Have an EKG to look at the heart;
- Have an MRI \& MRA (Magnetic Resonance Angiography, which looks at the blood vessels and the blood flowing through them). These 2 tests will look to see if the patient has had any strokes;
- Have a PET scan to look at the head and body;
- Have a liver biopsy to determine if the liver has been damaged (which can happen from iron overload that happens after many transfusions). Too much liver damage would mean a transplant cannot be done.
For the liver biopsy, the skin is numbed with medicine, and a special needle goes into the liver. The needle removes a very small piece of the liver (tissue). The tissue is taken and examined.
Also, about 30 cc (2 tablespoons) of blood will be drawn to test the blood for viruses, including HIV (the virus that causes AIDS). If the HIV test is positive, a transplant will not be done because it would be too dangerous for the patient.
At least 2 weeks before the bone marrow infusion, the patient will be immunized with Prevnar 7. Prevnar 7 is a vaccine that is used in children to protect against some types of bacteria called Streptococcus pneumoniae, which can cause the lung infection called pneumonia. People with Sickle Cell anemia are at a higher risk of dying from an infection from this type of bacteria. Although children are regularly given the Prevnar 7 vaccine, it is not common to test a child's immune response to the vaccine. In this study, we will check the immune system's response to the vaccine by drawing an extra 3 mL (less than 1 teaspoon) of blood 3 weeks after the patient gets the Prevnar 7 vaccine.
Just before the bone marrow transplant, we must kill the cells in the bone marrow that make the abnormal red blood cells found in patients with severe thalassemia or sickle cell disease. We will do this by using 3 drugs: busulfan, cyclophosphamide, and campath-1H. Campath-1H is used to prevent the body from rejecting or refusing to let the donor blood cells grow in the body. MESNA is given with the cyclophosphamide to prevent kidney damage. Methotrexate and cyclosporin are also given to prevent graft-versus-host disease (GVHD); methylprednisolone will be given after the bone marrow infusion if the patient develops GVHD. A drug will also be given to prevent seizures (either dilantin or lorazepam).
Graft-versus-host disease (GVHD) is a possible side-effect of the transplant. In GVHD, some cells in the donor marrow attack cells in the patient's body. This causes skin, liver, and bowel problems, and may damage other parts of the body. Often, these problems are fairly mild, but they can be severe or even cause death. Severe GVHD is likely to occur in about 10% of patients.
After the patient gets the drug treatment, they will be given bone marrow from a brother or sister who has healthy bone marrow that matches the patients. The healthy bone marrow will be put into a vein (given IV) in the same way that blood transfusions are given. The marrow cells then travel to the right places in the body, where they should grow and make new normal blood cells.
This is the treatment schedule:
Protocol Day \& Treatment:
14 or more days before the bone marrow infusion -- Prevnar 7 vaccine
10 days before the bone marrow infusion -- Begin Dilantin or Lorazepam (to prevent seizures)
9 days before the bone marrow infusion -- Busulfan
8 days before the bone marrow infusion -- Busulfan
7 days before the bone marrow infusion -- Busulfan
6 days before the bone marrow infusion -- Busulfan
5 days before the bone marrow infusion -- Campath 1H, Cyclophosphamide, and MESNA
4 days before the bone marrow infusion -- Campath 1H, Cyclophosphamide, and MESNA
3 days before the bone marrow infusion -- Campath 1H, Cyclophosphamide, and MESNA
2 days before the bone marrow infusion -- Campath 1H, Cyclophosphamide, MESNA, and cyclosporin
1 day before the bone marrow infusion -- DAY OF REST
Day "0" -- bone marrow infusion given
1 day after the bone marrow infusion -- Methotrexate
3 days after the bone marrow infusion -- Methotrexate
6 days after the bone marrow infusion -- Methotrexate
11 days after the bone marrow infusion -- Methotrexate
After transplant, Filgrastrim, a growth hormone for the bone marrow, may be given intravenously (through a vein), if medically necessary.
Sometimes, the donor's bone marrow does not grow. Then, the patient would be without working bone marrow cells. So that we will be ready to treat this problem if it happens, we will take bone marrow from the hip bone before the patient gets the drug treatment. The patient will be asleep (sedated) when the marrow is taken, and they will be given medicine for any pain they have afterwards. This bone marrow will be frozen and stored or preserved. If the donor bone marrow does not grow, we will thaw the stored marrow and put it back into the body. This stored bone marrow should grow and produce working blood cells. If the stored bone marrow must be given back, the disease will not be cured.
To tell whether the transplant has worked or "engrafted", we will take samples of bone marrow (bone marrow aspirate). We will do this 3 weeks after the transplant to make sure the new cells are beginning to grow. We will take another marrow sample at 3 months after the transplant. We want to make sure the new cells are still growing. This test will take about 30 to 45 minutes. Because this test is painful, the patient will be given pain medicine before, during, and after the test.
The patient will need to be in the hospital for at least 3 weeks after the transplant to make sure the transplant has engrafted. We will do several tests (of the lung, kidney, and liver) before and after the bone marrow transplant. We want to find out how much the treatment has helped the patient and how much it might help other patients. Most tests will be done every week for 4 months and at each visit to the hospital. Also, we will be looking at the immune function. To do this, we will take about 8 ml (less than 2 teaspoonful) at 6 months, 1 year, and 2 years after the transplant. When possible, we will take the blood from an IV line that the patient already has. However, at times we will have to draw the blood with another needle stick. A total amount of 200 mL (about 13 tablespoons) of blood will be collected from the patient during the entire study.
After 4 months, if the patient's health is good, they will not need to come to the hospital so often. The visits to the doctor should be more like they were before the bone marrow transplant. Since problems may happen months after the transplant and this is a new way to treat sickle cell disease and thalassemia the patient will need to have exams and blood tests done every few months during the first and second years after their transplant. After that, if all is well, the patient will need to be examined and have blood tests 4 times a year.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Allogeneic BMT/SCT Transplant | Experimental | Drug: Busulfan, Biological: Campath 1H, Drug: Cyclophosphamide and MESNABusulfan, Campath 1H, Cyclophosphamide and MESNA: Bone marrow infusion with pre-meds as per SOPs to take place on Day 0. Bone marrow dose: To ensure the probability for bone marrow engraftment, 4 x 10^8 nucleated cells/kg patient weight will be the target at donor bone marrow harvest. |
Who may qualify
- Ages
- Up to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (12 listed)
- Patients with a related HLA genotype identical donor and hemoglobin SS, hemoglobin SC, or hemoglobin Sb0/+ and at least one of the following conditions:
- Between the ages of birth and 40 years.
- Women of childbearing potential must have a negative pregnancy test.
Key exclusion criteria (10 listed)
- Patient with biopsy proven chronic active hepatitis or fibrosis with portal bridging.
- Patient with SCD chronic lung disease > stage 3 (see Appendix 1).
- Patient with severe renal dysfunction defined as creatinine clearance < 40 mL/min/1.73 M^2.
- Patient with severe cardiac dysfunction defined as echocardiogram shortening fraction < 25% or NYHA class III or IV.
- Patient with 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: MyleranStarting Day -9 / Busulfan 4.0 mg/kg/day IV divided into four doses daily for four days; total dose = 16 mg/kg. | Drug | Allogeneic BMT/SCT Transplant |
| Campath 1HDay -5 through Day -2; Campath-1H dosed as per institutional guidelines. | Biological | Allogeneic BMT/SCT Transplant |
| Cyclophosphamide and MESNAAlso: CTXDay -5 through Day -2; Cyclophosphamide 50 mg/kg + MESNA. | Drug | Allogeneic BMT/SCT Transplant |
Primary outcome
Number of Participants Evaluated for Evidence of Recovery of Organ Function Measured Via MRI or PET Scan.
Time frame: One year
Assess Number of participants that recovered organ function diagnosed with sickle cell disease (SCD) or sickle hemoglobin variants after undergoing allogeneic SCT/BMT from HLA genotype identical donors.
Number of Participants With Pre and Post Transplant PET Scan to Assess Organ Recovery Based on Rate of Acquisition.
Time frame: One year
Number of participants that did or did not have pre- and post- PET scans.
Number of Participants With Immune Response to Immunization After BMT in Participants With SCD, Hemoglobin SC, or Hemoglobin Sb0/+.
Time frame: up to 12 months
Vaccine response will be measured via a humoral immunity panel evaluating streptococcus pneumonia IgG antibodies (microgram/mL). Panels are obtained pre and 1+ month post vaccination. Standard recommendations define a normal responder as having >4 fold increase in antibody level in 50-70% of the serotypes found in the vaccine.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jul 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 study director
Baylor College of Medicine
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Busulfan, Campath 1H and Cyclophosphamide and MESNA in people with Sickle Cell Disease and Hemoglobin SC, run by Tami D. John at 2 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 12 inclusion and 10 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 31 Jul 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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.
Are results available for this study?
Yes. Results are posted in the registry (first posted 31 Jul 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?
Number of Participants Evaluated for Evidence of Recovery of Organ Function Measured Via MRI or PET Scan. (time frame: One year). The study lists 3 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 31 Jul 2020.