A Phase 2 interventional study of Alemtuzumab and Sirolimus in Sickle Cell Disease, Thalassemia and Stem Cell Transplantation, sponsored by National Heart, Lung, and Blood Institute (NHLBI). Active, not recruiting at 1 site in United States. Open to participants aged 4 Years to 80 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2026-08-18.
Sponsored by National Heart, Lung, and Blood Institute (NHLBI) · Phase 2, Interventional, and Treatment
Background:
- Some sickle cell disease or beta-thalassemia can be cured with transplant. Researchers want to test a variation of transplant that uses low dose radiation and a combination of immunosuppressive drugs. They want to know if it helps a body to better accept donor stem cells.
Objectives:
- To see if low dose radiation (300 rads), oral cyclophosphamide, pentostatin, and sirolimus help a body to better accept donor stem cells.
Eligibility:
- People 4 and older with beta-thalassemia or sickle cell disease that can be cured with transplant, and their donors.
Design:
Our ongoing nonmyeloablative allogeneic peripheral blood stem cell (PBSC) transplant protocol (03-H-0170) for patients with severe sickle cell disease (SCD) and B-thalassemia from HLA-matched family donors has excellent results thus far. Our long term leukocyte engraftment rate is 85-90% with the same disease-free survival. None of the engrafted patients had acute sickle-related events, significant toxicity associated with the conditioning regimen, or any evidence of graft versus host disease (GVHD).
While these results rival the transplant outcomes from low risk transplant patients with B-thalassemia, there are areas for improvement. The first is the 10-15% graft rejection rate, where a majority of these individuals were male donor and female recipient pairs. Another limitation is the significant delay in donor red cell engraftment in one recipient who had pre-existing allo-antibody to donor red cells from previous transfusions. Also we have excluded another group of individuals with preformed antibodies, recipients having major ABO incompatibility to the donors.
To overcome these limitations (and reduce the transplant failure rate) in this new protocol, we will continue our nonmyeloablative approach in the patients with SCD and B-thalassemia with HLA-matched family donors, but using an increased intensity regimen in a subset considered at high risk for transplant failure. This modified regimen consists of pentostatin and oral cyclophosphamide, which we hypothesize will reduce both the T cells that mediate leukocyte rejection and the B/plasma cells that produce anti-donor erythrocyte antibodies. The main transplant backbone will remain as alemtuzumab, low dose total body irradiation of 300 cGy, and sirolimus; the transplant graft will remain as unmanipulated G-CSF mobilized, T-cell replete, PBSC product for hematopoietic and lymphoid reconstitution.
The primary endpoint of this study is the percentage/number of patients who have sustained donor type hemoglobin at 1 year post transplant for male donors - female recipients. The primary endpoint for those with pre-existing antibodies is the presence of donor red cells with reticulocytes greater than or equal to 30 k/uL at 2 years post-transplant. Other endpoints include the toxicity of the pentostatin-cyclophosphamide regimen, the degree of donor-host chimerism necessary for long-term graft survival and disease amelioration, incidence of acute and chronic GVHD, incidence of graft rejection, transplant-related morbidity, as well as disease-free and overall survival. Since SCD and B-thalassemia are non-malignant disorders of red cells, severe GVHD, lack of donor erythrocyte (prolonged donor red cell aplasia), or graft rejection is collectively considered transplant failure.
1,103 studies on the registry are indexed under Anemia, Sickle Cell; 235 are open to participants now.
This study's enrollment of 56 is above the median of 40 across 750 interventional studies indexed under Anemia, Sickle Cell.
Browse Anemia, Sickle Cell studies →National Heart, Lung, and Blood Institute (NHLBI) is the lead sponsor of 1,117 studies on the registry; 71 are open to participants now.
Of its 57 completed or terminated interventional studies of FDA-regulated products, 49 (86%) have results posted.
Counted across the registry records on this site, refreshed daily.
-INCLUSION CRITERIA- recipients (must fulfill one disease category in 1 and all of 2)
Disease specific
Patients with severe sickle cell disease (not limited to Hb SS, SC, or S beta-thal) at high risk for disease-related morbidity or mortality, defined by having severe end-organ damage (A, B, C, D, or E) or potentially modifiable complication(s) not ameliorated by hydroxyurea or sickle specific therapy (F):
--A. Stroke defined as a clinically significant neurologic event that is accompanied by an infarct on cerebral MRI or cerebral arteriopathy requiring chronic transfusion therapy; OR
--B. Sickle cell-related renal insufficiency defined by a creatinine level greater than or equal to 1.5 times the upper limit of normal and kidney biopsy consistent with sickle cell nephropathy OR nephrotic syndrome OR creatinine clearance less than \< 50mL/min OR requiring peritoneal or hemodialysis; OR
--C. Tricuspid regurgitant jet velocity (TRV) of greater than or equal to 2.5 m/s 40, 41 at baseline; OR
--D. Recurrent priapism defined as at least 2 episodes of an erection lasting >4 hours involving the corpora cavernosa and corpus spongiosa; OR
--E. Sickle hepatopathy defined as EITHER ferritin >1000mcg/L OR direct bilirubin >0.4 mg/dL at baseline
--F. Any one of the below complications:
---Complication/ Eligible for hydroxyurea*/ Eligible for HSCT
----Vaso-occlusive crises/ At least 3 hospital admissions in the last year/ More than one hospital admission in the last year while on therapeutic dose of hydroxyurea or sickle cell therapy
Red cell alloimmunization/ Transfusion dependent/ Total hemoglobin increases less than 1g/dL while on hydroxurea
2. Patients with beta-thalassemia who have grade 2 or 3 iron overload, determined by the presence of 2 or more of the following:
-- portal fibrosis by liver biopsy
Non-disease specific:
-Age greater than or equal to 4 years
-6/6 HLA matched family donor available
EXCLUSION CRITERIA -recipient (any of the following would exclude the subject from participating)
-ECOG performance status of 3 or more
-Evidence of uncontrolled bacterial, viral, or fungal infections (currently taking medication and progression of clinical symptoms) within one month prior to starting the conditioning regimen. Patients with fever or suspected minor infection should await resolution of symptoms before starting the conditioning regimen.
-Major anticipated illness or organ failure incompatible with survival from PBSC transplant
-Pregnant or lactating
INCLUSION CRITERIA -donor
-6/6 HLA matched family donor deemed suitable and eligible, and willing to donate, per clinical evaluations who are additionally willing to donate blood for research. Matched related donors will be evaluated in accordance with existing Standard NIH Policies and Procedures for determination of eligibility and suitability for clinical donation. Note that participation in this study is offered to all matched related donors, but is not required for a donor to make a stem cell donation, so it is possible that not all related donors will enroll onto this study.
EXCLUSION CRITERIA -donor
-None
Female participants with Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant with male donor. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0.
Drug: Alemtuzumab · Drug: Sirolimus · Drug: Cyclophosphamide · Drug: Pentostatin · Procedure: Radiotherapy
Participants with pre-existing antibodies and Sickle Cell Disease (SCD) or Beta-thalassemia receiving stem cell transplant. Pentostatin given on days -21, -17, -13, -9 and oral cyclophosphamide from days -21 to -8, with the intention to be administered in the outpatient setting. Alemtuzumab on days 7 to 3, and 300 cGy TBI on day 2. Sirolimus started at a loading dose of 5mg PO every 4 hours for three doses on day -1 and adjusted to maintain trough levels between 10-15 ng/mL. The PBSC graft targeted to deliver .10 x 106 CD34+ cells/kg (minimum .5 x 106) and infused on day 0.
Drug: Alemtuzumab · Drug: Sirolimus · Drug: Cyclophosphamide · Drug: Pentostatin · Procedure: Radiotherapy
Participants received filgrastim to mobilize peripheral blood stem cells for apheresis collection. Collected stem cells of donor will then be infused to HLA matched sibling.
Drug: Filgrastim
Immunosuppressant
Also known as: Campath
Immunosuppressant
Also known as: Rapamune
Immunosuppressant
Also known as: Cytoxan
Immunosuppressant
Also known as: Deoxycoformycin
Immunosuppressant and myelosuppressant
mobilize peripheral blood stem cells for apheresis collection
Also known as: Neupogen
Number of Patients Who Have Sustained Donor Type Hemoglobin at One Year Post Transplant
Number of patients who have sustained donor type hemoglobin at one year post transplant. Sustained donor type hemoglobin is based on hemoglobin electrophoresis for patients with SCD and transfusion independence for patients with beta-thalassemia
Time frame: 1 year
Number of Participants With Donor Red Cells at 2 Years Post Stem Cell Transplant
Number of participants with donor red cells at 2 years post stem cell transplant. Number of participants with donor red cells is detected by hemoglobin electrophoresis or donor type red cell antigen, and reticulocyte count ≥30 k/uL at 2 years post-transplant.
Time frame: 2 years
Mean CD34+ Cell Dose
Mean CD34+ cell dose, filgrastim mobilized peripheral blood hematopoietic cells, unselected
Time frame: Day 0 up to Day 1
Mean CD3+ Cell Dose
Mean CD3+ cell dose, filgrastim mobilized peripheral blood hematopoietic cells, unselected
Time frame: Day 0 up to Day 1
Median Percent of Donor T-cells and Myeloid Chimerism
Median Percent of donor T-cells and myeloid chimerism. Leukocytes are selected by magnetic beads for CD3 (T-cells) and CD14/15 (myeloid cells), then microsatellite PCR analyses are performed to obtain donor chimerism percent.
Time frame: day 30, day 60 , day 100, 1 year and 2 year
Number of Participants Who Developed Acute Graft vs Host Disease (GVHD) Grades I, II, III, IV
Number of participants who developed Acute Graft vs Host Disease (GVHD) Grades I, II, III, IV as defined by CIMBTR criteria for Organ Stages of Acute GVHD. Grades are defined as: Grade I: Skin = Maculopapular rash\< 25% of body surface area (BSA); Liver = Total Bilirubin 2-3 mg/dL; Lower GI = stool output/day is 500-999 mL/day. Grade II: Skin = rash on 25-50 percent body surface area; Liver = Total Bilirubin 3.1-6.0 mg/dL; Lower GI = Diarrhea 1001-1500 mL/day. Grade III: Skin = Rash on \>50% of body surface; Liver = Total Bilirubin 6.1 - 15.0 mg/dL; Lower GI = Diarrhea \> 1500 mL/day. Grade IV: Skin = Generalized erythroderma plus bullous formation; Liver = Total Bilirubin \>15 mg/dL; Lower GI = Severe abdominal pain with or without ileus. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening.
Time frame: Up to Day 100
Number of Participants Who Developed Moderate or Severe Chronic Graft vs Host Disease (GVHD)
Number of Participants Who Developed Moderate or Severe Chronic Graft vs Host Disease (GVHD) up to 5 years. Moderate chronic GVHD involves EITHER 3 organs/sites with no clinically significant functional impairment OR a less than or equal to 1 organ/site with clinically significant functional impairment, but no major disability. Severe GVHD is associated with a major disability caused by chronic GVHD.
Time frame: Day 100 up to 2 years
Number of Participants That Experienced Graft Failure or Graft Rejection, or Red Cell Aplasia at 2 Years After Transplant
Number of participants that experienced graft failure or graft rejection, or red cell aplasia at 2 years after transplant. Graft failure or graft rejection is defined as \<5% donor cells in both CD3 and myeloid chimerism. Red cell aplasia is defined as reticulocyte \<30 k/uL and requiring red cell transfusions.
Time frame: Up to 2 years
Number of Participants That Experienced Regimen Failure
Number of Participants That Experienced Regimen Failure. Regimen failure is defined as those participants that experienced grade 3 or higher acute GVHD, moderate/severe chronic GVHD, graft failure/rejection, or red cell aplasia. Together any one of these count toward the combined endpoint of regimen failure.
Time frame: Up to 2 years
Number of Participants That Experienced Transplant-related Mortality
Number of participants that experienced transplant-related mortality. Transplant-related mortality is defined as death that is at least possibly related to the transplant (GVHD, toxicity, infection, other causes).
Time frame: Up to 2 years
Percentage of Participant With Disease-free Survival Following Stem Cell Transplant
Percentage of participant with disease-free survival following stem cell transplant. Disease-free survival is defined as alive and free acute complications related to sickle cell disease.
Time frame: Up to 2 years
Percentage of Participant Overall Survival Following Stem Cell Transplant
Percentage of Participant Overall Survival up to year 2 following stem cell transplant. Overall survival is defined as being alive following stem cell transplant.
Time frame: Up to 2 years
Median Day to Neutrophil Recovery
Median Day to Neutrophil recovery. Neutrophil recovery is defined as the first of three consecutive days of neutrophil count \>0.5 x 10\^9 cells/uL.
Time frame: Up to Day 100
Median Days to Platelet Recovery
Median Days to Platelet Recovery. Platelet recovery is defined as count \>50 cells/uL and 7 days from the last platelet transfusion.
Time frame: Up to Day 120
Median Days to Red Cell Recovery
Median Days to Red Cell Recovery. Red cell recovery defined as days to recovery of reticulocyte count .30 k/uL, detection of donor red cells, transfusion independence.
Time frame: Up to 2 years
| Milestone | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor |
|---|---|---|---|
| Started | 11 | 18 | 27 |
| Completed | 7 | 16 | 27 |
| Not completed | 4 | 2 | 0 |
| Withdrew: Physician decision | 1 | 1 | 0 |
| Withdrew: Lost to follow-up | 2 | 0 | 0 |
| Withdrew: Death | 1 | 1 | 0 |
Number of patients who have sustained donor type hemoglobin at one year post transplant. Sustained donor type hemoglobin is based on hemoglobin electrophoresis for patients with SCD and transfusion independence for patients with beta-thalassemia
| Participants | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor |
|---|---|
| Number of Patients Who Have Sustained Donor Type Hemoglobin at One Year Post Transplant | 10 |
Number of participants with donor red cells at 2 years post stem cell transplant. Number of participants with donor red cells is detected by hemoglobin electrophoresis or donor type red cell antigen, and reticulocyte count ≥30 k/uL at 2 years post-transplant.
| Participants | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant |
|---|---|
| Number of Participants With Donor Red Cells at 2 Years Post Stem Cell Transplant | 16 |
Mean CD34+ cell dose, filgrastim mobilized peripheral blood hematopoietic cells, unselected
| x10^6 cells/kg | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant |
|---|---|---|
| Mean CD34+ Cell Dose | 19.08 ± 8.38 | 14.98 ± 5.95 |
Mean CD3+ cell dose, filgrastim mobilized peripheral blood hematopoietic cells, unselected
| x10^8 cells/kg | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant |
|---|---|---|
| Mean CD3+ Cell Dose | 3.12 ± 1.37 | 3.29 ± 1.57 |
Median Percent of donor T-cells and myeloid chimerism. Leukocytes are selected by magnetic beads for CD3 (T-cells) and CD14/15 (myeloid cells), then microsatellite PCR analyses are performed to obtain donor chimerism percent.
| percentage of donor cells | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant |
|---|---|---|
| myeloid chimerism D30 | 100 (80 to 100) | 99.5 (68 to 100) |
| myeloid chimerism D60 | 100 (54 to 100) | 100 (29 to 100) |
| myeloid chimerism D100 | 100 (42 to 100) | 100 (16 to 100) |
| myeloid chimerism Year 1 | 100 (47 to 100) | 100 (54 to 100) |
| myeloid chimerism Year 2 | 100 (44 to 100) | 99 (45 to 100) |
| donor T-cells D30 | 65.5 (16 to 94) | 25 (1 to 85) |
| donor T-cells D60 | 39 (2 to 86) | 23 (1 to 100) |
| donor T-cells D100 | 34 (0 to 91) | 22.5 (2 to 96) |
| donor T-cells Year 1 | 70 (29 to 78) | 43 (24 to 100) |
| donor T-cells Year 2 | 74.5 (53 to 84) | 62 (27 to 94) |
Number of participants who developed Acute Graft vs Host Disease (GVHD) Grades I, II, III, IV as defined by CIMBTR criteria for Organ Stages of Acute GVHD. Grades are defined as: Grade I: Skin = Maculopapular rash\< 25% of body surface area (BSA); Liver = Total Bilirubin 2-3 mg/dL; Lower GI = stool output/day is 500-999 mL/day. Grade II: Skin = rash on 25-50 percent body surface area; Liver = Total Bilirubin 3.1-6.0 mg/dL; Lower GI = Diarrhea 1001-1500 mL/day. Grade III: Skin = Rash on \>50% of body surface; Liver = Total Bilirubin 6.1 - 15.0 mg/dL; Lower GI = Diarrhea \> 1500 mL/day. Grade IV: Skin = Generalized erythroderma plus bullous formation; Liver = Total Bilirubin \>15 mg/dL; Lower GI = Severe abdominal pain with or without ileus. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening.
| Participants | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant |
|---|---|---|
| Grade I | 0 | 0 |
| Grade II | 0 | 1 |
| Grade III | 1 | 0 |
| Grade IV | 0 | 0 |
Number of Participants Who Developed Moderate or Severe Chronic Graft vs Host Disease (GVHD) up to 5 years. Moderate chronic GVHD involves EITHER 3 organs/sites with no clinically significant functional impairment OR a less than or equal to 1 organ/site with clinically significant functional impairment, but no major disability. Severe GVHD is associated with a major disability caused by chronic GVHD.
| Participants | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant |
|---|---|---|
| Moderate | 0 | 0 |
| Severe | 0 | 0 |
Number of participants that experienced graft failure or graft rejection, or red cell aplasia at 2 years after transplant. Graft failure or graft rejection is defined as \<5% donor cells in both CD3 and myeloid chimerism. Red cell aplasia is defined as reticulocyte \<30 k/uL and requiring red cell transfusions.
| Participants | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant |
|---|---|---|
| graft failure | 1 | 0 |
| red cell aplasia | 0 | 2 |
Number of Participants That Experienced Regimen Failure. Regimen failure is defined as those participants that experienced grade 3 or higher acute GVHD, moderate/severe chronic GVHD, graft failure/rejection, or red cell aplasia. Together any one of these count toward the combined endpoint of regimen failure.
| Participants | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant |
|---|---|---|
| Number of Participants That Experienced Regimen Failure | 2 | 2 |
Number of participants that experienced transplant-related mortality. Transplant-related mortality is defined as death that is at least possibly related to the transplant (GVHD, toxicity, infection, other causes).
| Participants | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant |
|---|---|---|
| Number of Participants That Experienced Transplant-related Mortality | 0 | 0 |
Percentage of participant with disease-free survival following stem cell transplant. Disease-free survival is defined as alive and free acute complications related to sickle cell disease.
| Participants | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant |
|---|---|---|
| Percentage of Participant With Disease-free Survival Following Stem Cell Transplant | 9 | 16 |
Percentage of Participant Overall Survival up to year 2 following stem cell transplant. Overall survival is defined as being alive following stem cell transplant.
| Participants | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant |
|---|---|---|
| Percentage of Participant Overall Survival Following Stem Cell Transplant | 10 | 17 |
Median Day to Neutrophil recovery. Neutrophil recovery is defined as the first of three consecutive days of neutrophil count \>0.5 x 10\^9 cells/uL.
| days | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant |
|---|---|---|
| Median Day to Neutrophil Recovery | 22 (17 to 67) | 20.5 (8 to 25) |
Median Days to Platelet Recovery. Platelet recovery is defined as count \>50 cells/uL and 7 days from the last platelet transfusion.
| days | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant |
|---|---|---|
| Median Days to Platelet Recovery | 21.5 (16 to 69) | 22.5 (10 to 112) |
Median Days to Red Cell Recovery. Red cell recovery defined as days to recovery of reticulocyte count .30 k/uL, detection of donor red cells, transfusion independence.
| days | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant |
|---|---|---|
| Median Days to Red Cell Recovery | 22 (15 to 75) | 19 (8 to 195) |
Collected over 5 years. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | 1/11 (9.1%) | 9/11 (81.8%) | 11/11 (100%) |
| Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant | 1/18 (5.6%) | 13/18 (72.2%) | 18/18 (100%) |
| Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | 0/27 (0%) | 2/27 (7.4%) | 0/27 (0%) |
| Event | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor |
|---|---|---|---|
| PainMusculoskeletal and connective tissue disorders | 2/11 | 6/18 | 1/27 |
| BacteremiaInfections and infestations | 3/11 | 0/18 | 0/27 |
| Lung infectionInfections and infestations | 3/11 | 1/18 | 0/27 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 2/11 | 0/18 | 0/27 |
| Avascular necrosisMusculoskeletal and connective tissue disorders | 2/11 | 0/18 | 0/27 |
| Vaso-occlusive pain crisisMusculoskeletal and connective tissue disorders | 2/11 | 3/18 | 0/27 |
| Skin infectionInfections and infestations | 2/11 | 1/18 | 0/27 |
| Cytomegalovirus (CMV) ReactivationInfections and infestations | 0/11 | 3/18 | 0/27 |
| DiarrheaGastrointestinal disorders | 1/11 | 3/18 | 0/27 |
| DizzinessNervous system disorders | 0/11 | 2/18 | 0/27 |
| Event | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor |
|---|---|---|---|
| Lymphocyte count decreasedBlood and lymphatic system disorders | 9/11 | 10/18 | 0/27 |
| Neutrophil count decreasedBlood and lymphatic system disorders | 8/11 | 8/18 | 0/27 |
| Platelet count decreasedBlood and lymphatic system disorders | 6/11 | 5/18 | 0/27 |
| White blood cell decreasedBlood and lymphatic system disorders | 5/11 | 2/18 | 0/27 |
| PainMusculoskeletal and connective tissue disorders | 4/11 | 6/18 | 0/27 |
| AnemiaBlood and lymphatic system disorders | 0/11 | 5/18 | 0/27 |
| DiarrheaGastrointestinal disorders | 3/11 | 1/18 | 0/27 |
| Cytomegalovirus (CMV) ReactivationInfections and infestations | 3/11 | 4/18 | 0/27 |
| InsomniaPsychiatric disorders | 3/11 | 0/18 | 0/27 |
| HypertensionCardiac disorders | 2/11 | 4/18 | 0/27 |
| Age, Categorical(Participants) | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | Total |
|---|---|---|---|---|
| <=18 years | 0 | 3 | 5 | 8 |
| Between 18 and 65 years | 11 | 15 | 22 | 48 |
| >=65 years | 0 | 0 | 0 | 0 |
| Sex: Female, Male(Participants) | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | Total |
|---|---|---|---|---|
| Female | 11 | 5 | 8 | 24 |
| Male | 0 | 13 | 19 | 32 |
| Ethnicity (NIH/OMB)(Participants) | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | Total |
|---|---|---|---|---|
| Hispanic or Latino | 0 | 0 | 0 | 0 |
| Not Hispanic or Latino | 10 | 17 | 25 | 52 |
| Unknown or Not Reported | 1 | 1 | 2 | 4 |
| Race (NIH/OMB)(Participants) | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | Total |
|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 |
| Asian | 0 | 2 | 2 | 4 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 |
| Black or African American | 10 | 16 | 23 | 49 |
| White | 0 | 0 | 0 | 0 |
| More than one race | 1 | 0 | 0 | 1 |
| Unknown or Not Reported | 0 | 0 | 2 | 2 |
| Region of Enrollment(participants) | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | Total |
|---|---|---|---|---|
| United States | 11 | 18 | 27 | 56 |
Documents are hosted by the registry — open the source record to download them.
Plan to share: Yes — All IPD that underlie results in a publication
Supporting information: Study protocol, Icf
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