Bone Marrow Transplantation, Hemoglobinopathies, SCALLOP (SCALLOP)
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|ClinicalTrials.gov Identifier: NCT00578344|
Recruitment Status : Terminated (Terminated due to no new subject enrollment during the last 3 year period.)
First Posted : December 21, 2007
Results First Posted : July 31, 2020
Last Update Posted : July 31, 2020
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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.
|Condition or disease||Intervention/treatment||Phase|
|Sickle Cell Disease Hemoglobin SC||Drug: Busulfan Biological: Campath 1H Drug: Cyclophosphamide and MESNA||Not Applicable|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||8 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Allogeneic Bone Marrow Transplantation From HLA Identical Related Donors for Patients With Hemoglobinopathies: Hemoglobin SS, Hemoglobin SC, or Hemoglobin SB0/+ Thalassemia|
|Study Start Date :||July 2005|
|Actual Primary Completion Date :||July 2012|
|Actual Study Completion Date :||July 2012|
Experimental: Allogeneic BMT/SCT Transplant
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.
Starting Day -9 / Busulfan 4.0 mg/kg/day IV divided into four doses daily for four days; total dose = 16 mg/kg.
Other Name: Myleran
Biological: Campath 1H
Day -5 through Day -2; Campath-1H dosed as per institutional guidelines.
Drug: Cyclophosphamide and MESNA
Day -5 through Day -2; Cyclophosphamide 50 mg/kg + MESNA.
Other Name: CTX
- 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.
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|Ages Eligible for Study:||up to 40 Years (Child, Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
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;
- 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.
- 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.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00578344
|United States, Texas|
|Houston, Texas, United States, 77030|
|Texas Children's Hospital|
|Houston, Texas, United States, 77030|
|Study Director:||Tami John, MD||Baylor College of Medicine - Texas Children's Hospital|
Documents provided by Tami D. John, Baylor College of Medicine:
|Responsible Party:||Tami D. John, Assistant Professor, Pediatrics-Hema & Oncology, Baylor College of Medicine|
|Other Study ID Numbers:||
SCALLOP ( Other Identifier: Baylor College of Medicine )
|First Posted:||December 21, 2007 Key Record Dates|
|Results First Posted:||July 31, 2020|
|Last Update Posted:||July 31, 2020|
|Last Verified:||July 2020|
Sickle Cell Disease
Anemia, Sickle Cell
Hemoglobin SC Disease
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Physiological Effects of Drugs
Antineoplastic Agents, Alkylating
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents, Immunological