A Phase 2 interventional study of Bone marrow transplantation and Mesenchymal Stromal Cells in Sickle Cell Disease, Thalassemia and Diamond-Blackfan Anemia, sponsored by Stanford University. Completed at 2 sites in United States. Open to participants aged 1 Year to 25 Years. Per ClinicalTrials.gov, last updated 2018-08-06.
Sponsored by Stanford University · Phase 2, Interventional, and Treatment
The main purpose of this project is to cure patients with high risk Sickle cell disease and other red cell disorders including thalassemia and diamond-blackfan anemia by bone marrow transplantation. The patients enrolled in this study will be those who lack matched sibling donors and therefore have no other option but to undergo bone marrow transplantation using matched but unrelated bone marrow or umbilical cord blood from the national marrow donor program registry. Since bone marrow transplantation for these disorders using matched unrelated donors has two major problems i.e. engraftment, or , the process of new marrow being accepted and allowed to grow in the the patient; and graft-versus-host disease, or the process where the new marrow "rejects" the host or the patient, this study has been devised with methods to overcome these two problems and thus make transplantation from unrelated donors both successful in terms of engraftment and safe in terms of side effects, both acute and long term.
In order to accomplish these two goals, two important things will be done. Firstly, patients will get three medicines which are considered reduced intensity because they are not known to cause the serious organ damage seen with conventional chemotherapy. These medicines, however, do cause intense immune suppression so these can cause increased infections. Secondly, in addition to transplantation of bone marrow from unrelated donors, patients will also transplanted with mesenchymal stromal cells derived from the bone marrow of their parents. Mesenchymal stromal cells are adult stem cells that are normally found in the bone marrow and are thought to create the right background for the blood cells to grow. They have been shown in many animal and human studies to improve engraftment. In addition, they have a special property by which they prevent and are now even considered to treat graft versus host disease. Therefore, by using a reduced intensity chemotherapy regimen before transplant and transplanting mesenchymal stromal cells, we hope to improve engraftment while at the same time decrease the potential for severe side effects associated with a conventional transplant which uses extremely high doses of chemotherapy.
1,103 studies on the registry are indexed under Anemia, Sickle Cell; 235 are open to participants now.
This study's enrollment of 6 is below the median of 40 across 750 interventional studies indexed under Anemia, Sickle Cell.
Browse Anemia, Sickle Cell studies →Stanford University is the lead sponsor of 2,117 studies on the registry; 425 are open to participants now.
Of its 259 completed or terminated interventional studies of FDA-regulated products, 197 (76%) have results posted.
Counted across the registry records on this site, refreshed daily.
Patients with sickle cell disease (SCD) 1-25 years of age with an HLA-identical, but unrelated, donor or 1 human leukocyte antigen (HLA) allele mismatched bone marrow or up to 2 HLA antigen mismatched umbilical cord blood (UCB) donor with one or more of the following:
Exclusion Criteria:
Patients with one or more of the following:
Procedure: Bone marrow transplantation · Biological: Mesenchymal Stromal Cells
Bone marrow transplantation using matched unrelated donors, reduced intensity conditioning regimen, and co-transplanting mesenchymal stromal cells derived from parental bone marrow.
Count of Participants With Stable Engraftment Post Hematopoietic Cell Transplantation (HCT)
Stable engraftment was defined as absolute neutrophil count (ANC) \>500 cells /µL for 3 consecutive days and platelet count \>50,000 for one week without transfusion; subsequently stable engraftment was measured by percentage of donor cells.
Time frame: Up to 1 year
Overall Survival 6 Months Following HCT
Overall survival is reported at the count of participants alive 6 months following HCT.
Time frame: 6 months
Overall Survival 1 Year Following HCT
Overall survival is reported at the count of participants alive 1 year following HCT.
Time frame: 1 year
Count of Participants With Disease-free Survival 6 Months Following HCT
Disease-free survival is defined as alive without underlying disease.
Time frame: 6 months
Count of Participants With Disease-free Survival 1 Year Following HCT
Disease-free survival is defined as alive without underlying disease.
Time frame: 1 year
| Milestone | Mesenchymal Stromal Cells |
|---|---|
| Started | 6 |
| Completed | 2 |
| Not completed | 4 |
| Withdrew: Death | 4 |
Stable engraftment was defined as absolute neutrophil count (ANC) \>500 cells /µL for 3 consecutive days and platelet count \>50,000 for one week without transfusion; subsequently stable engraftment was measured by percentage of donor cells.
| Participants | Mesenchymal Stromal Cells |
|---|---|
| Count of Participants With Stable Engraftment Post Hematopoietic Cell Transplantation (HCT) | 3 |
Overall survival is reported at the count of participants alive 6 months following HCT.
| Participants | Mesenchymal Stromal Cells |
|---|---|
| Overall Survival 6 Months Following HCT | 2 |
Overall survival is reported at the count of participants alive 1 year following HCT.
| Participants | Mesenchymal Stromal Cells |
|---|---|
| Overall Survival 1 Year Following HCT | 2 |
Disease-free survival is defined as alive without underlying disease.
| Participants | Mesenchymal Stromal Cells |
|---|---|
| Count of Participants With Disease-free Survival 6 Months Following HCT | 0 |
Disease-free survival is defined as alive without underlying disease.
| Participants | Mesenchymal Stromal Cells |
|---|---|
| Count of Participants With Disease-free Survival 1 Year Following HCT | 0 |
Collected over 1 year. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Mesenchymal Stromal Cells | 4/6 (66.7%) | 6/6 (100%) | 0/6 (0%) |
| Event | Mesenchymal Stromal Cells |
|---|---|
| Graft failure with autologous recoveryBlood and lymphatic system disorders | 2/6 |
| CMV viremiaInfections and infestations | 2/6 |
| SOSHepatobiliary disorders | 1/6 |
| EBV-PTLDImmune system disorders | 1/6 |
| Grade II GVHDImmune system disorders | 1/6 |
| Grade III acute GVHDImmune system disorders | 1/6 |
| Adenovirus reactivationInfections and infestations | 1/6 |
| BK virus reactivationInfections and infestations | 1/6 |
| CMV pneumonitisInfections and infestations | 1/6 |
| CMV reactivationInfections and infestations | 1/6 |
| Age, Continuous(years) | Mesenchymal Stromal Cells |
|---|---|
| Median | 10 (8 to 18) |
| Sex: Female, Male(Participants) | Mesenchymal Stromal Cells |
|---|---|
| Female | 1 |
| Male | 5 |
| Number of known transfusions prior to HSCT(transfusions) | Mesenchymal Stromal Cells |
|---|---|
| Median | 47.5 (4 to 220) |
| Diagnosis(Participants) | Mesenchymal Stromal Cells |
|---|---|
| Sickle cell disease | 4 |
| Thalassemia major | 2 |
This study is completed, as verified in Jul 2018. You cannot join it, but the record below documents what was studied.
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