Trial · NCT02105766
Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure
NCT02105766, Phase 2, Active not recruiting, National Heart, Lung, and Blood Institute (NHLBI); results first posted 2024-11-29
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure. Trials, record NCT02105766. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02105766
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure
- NCT number
- NCT02105766
- Status
- Active not recruiting
- Phases
- Phase 2
- Enrollment
- 56 participants, actual
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- Conditions
- Sickle Cell Disease, Thalassemia, Stem Cell Transplantation, Graft vs Host Disease
- Interventions
- Alemtuzumab, Sirolimus, Cyclophosphamide, Pentostatin, Radiotherapy, Filgrastim
- First posted
- 2014-04-07
- Last updated
- 2026-08-18
- Results first posted
- 2024-11-29
Source ClinicalTrials.gov
Registered outcomes15
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Patients Who Have Sustained Donor Type Hemoglobin at One Year Post Transplant | 1 year | 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 |
| Primary | Number of Participants With Donor Red Cells at 2 Years Post Stem Cell Transplant | 2 years | 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. |
| Secondary | Mean CD34+ Cell Dose | Day 0 up to Day 1 | Mean CD34+ cell dose, filgrastim mobilized peripheral blood hematopoietic cells, unselected |
| Secondary | Mean CD3+ Cell Dose | Day 0 up to Day 1 | Mean CD3+ cell dose, filgrastim mobilized peripheral blood hematopoietic cells, unselected |
| Secondary | Median Percent of Donor T-cells and Myeloid Chimerism | day 30, day 60 , day 100, 1 year and 2 year | 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. |
| Secondary | Number of Participants Who Developed Acute Graft vs Host Disease (GVHD) Grades I, II, III, IV | Up to Day 100 | 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. |
| Secondary | Number of Participants Who Developed Moderate or Severe Chronic Graft vs Host Disease (GVHD) | Day 100 up to 2 years | 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. |
| Secondary | Number of Participants That Experienced Graft Failure or Graft Rejection, or Red Cell Aplasia at 2 Years After Transplant | Up to 2 years | 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. |
| Secondary | Number of Participants That Experienced Regimen Failure | Up to 2 years | 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. |
| Secondary | Number of Participants That Experienced Transplant-related Mortality | Up to 2 years | 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). |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 registered outcomesOutcomes15
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Patients Who Have Sustained Donor Type Hemoglobin at One Year Post Transplant | 1 year | Analysis is intended per protocol for cohort 1 participants that received a stem cell transplant (female participants with male donors) | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Donor Red Cells at 2 Years Post Stem Cell Transplant | 2 years | Analysis is intended per protocol to include those participants from cohort 2 (patients with pre-existing antibody to donor red cells) | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Mean CD34+ Cell Dose | Day 0 up to Day 1 | x10^6 cells/kg | MEAN | |
| SECONDARY | Mean CD3+ Cell Dose | Day 0 up to Day 1 | x10^8 cells/kg | MEAN | |
| SECONDARY | Median Percent of Donor T-cells and Myeloid Chimerism | day 30, day 60 , day 100, 1 year and 2 year | Analysis includes those participants who have sustained engraftment or did not experience graft failure. | percentage of donor cells | MEDIAN |
| SECONDARY | Number of Participants Who Developed Acute Graft vs Host Disease (GVHD) Grades I, II, III, IV | Up to Day 100 | Analysis includes those participants who have sustained engraftment or did not experience graft failure. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Who Developed Moderate or Severe Chronic Graft vs Host Disease (GVHD) | Day 100 up to 2 years | Analysis includes those participants who have sustained engraftment or did not experience graft failure. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants That Experienced Graft Failure or Graft Rejection, or Red Cell Aplasia at 2 Years After Transplant | Up to 2 years | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Number of Participants That Experienced Regimen Failure | Up to 2 years | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Number of Participants That Experienced Transplant-related Mortality | Up to 2 years | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 outcomesResults by group56
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Patients Who Have Sustained Donor Type Hemoglobin at One Year Post Transplant | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | 10 | Participants | ||
| Number of Participants With Donor Red Cells at 2 Years Post Stem Cell Transplant | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant | 16 | Participants | ||
| Mean CD34+ Cell Dose | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | 19.08 | Standard Deviation 8.38 | x10^6 cells/kg | |
| Mean CD34+ Cell Dose | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant | 14.98 | Standard Deviation 5.95 | x10^6 cells/kg | |
| Mean CD3+ Cell Dose | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | 3.12 | Standard Deviation 1.37 | x10^8 cells/kg | |
| Mean CD3+ Cell Dose | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant | 3.29 | Standard Deviation 1.57 | x10^8 cells/kg | |
| Median Percent of Donor T-cells and Myeloid Chimerism | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | myeloid chimerism D30 | 100 | Full Range 80 to 100 | percentage of donor cells |
| Median Percent of Donor T-cells and Myeloid Chimerism | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant | myeloid chimerism D30 | 99.5 | Full Range 68 to 100 | percentage of donor cells |
| Median Percent of Donor T-cells and Myeloid Chimerism | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | myeloid chimerism D60 | 100 | Full Range 54 to 100 | percentage of donor cells |
| Median Percent of Donor T-cells and Myeloid Chimerism | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant | myeloid chimerism D60 | 100 | Full Range 29 to 100 | percentage of donor cells |
1 to 10 of 56Source ClinicalTrials.gov
See all 56 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | 11 | |
| Overall Study | STARTED | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant | 18 | |
| Overall Study | STARTED | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | 27 | |
| Overall Study | COMPLETED | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | 7 | |
| Overall Study | COMPLETED | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant | 16 | |
| Overall Study | COMPLETED | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | 27 | |
| Overall Study | NOT COMPLETED | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | 4 | |
| Overall Study | NOT COMPLETED | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant | 2 | |
| Overall Study | NOT COMPLETED | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | 0 |
Source ClinicalTrials.gov
Adverse events381
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Acute GVHD | Blood and lymphatic system disorders | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | 1 | 11 | 1 |
| other | Acute GVHD | Blood and lymphatic system disorders | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant | 0 | 18 | 0 |
| other | Acute GVHD | Blood and lymphatic system disorders | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | 0 | 27 | 0 |
| other | Anemia | Blood and lymphatic system disorders | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | 0 | 11 | 0 |
| other | Anemia | Blood and lymphatic system disorders | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant | 5 | 18 | 8 |
| other | Anemia | Blood and lymphatic system disorders | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | 0 | 27 | 0 |
| other | CD4 lymphocytes decreased | Blood and lymphatic system disorders | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | 1 | 11 | 1 |
| other | CD4 lymphocytes decreased | Blood and lymphatic system disorders | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant | 0 | 18 | 0 |
| other | CD4 lymphocytes decreased | Blood and lymphatic system disorders | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | 0 | 27 | 0 |
| other | Febrile neutropenia | Blood and lymphatic system disorders | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | 1 | 11 | 1 |
1 to 10 of 381Source ClinicalTrials.gov
See all 381 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | 1 | 11 |
| Total, all-cause mortality | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant | 1 | 18 |
| Total, all-cause mortality | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | 0 | 27 |
| Total, other adverse events | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | 11 | 11 |
| Total, other adverse events | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant | 18 | 18 |
| Total, other adverse events | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | 0 | 27 |
| Total, serious adverse events | Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor | 9 | 11 |
| Total, serious adverse events | Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant | 13 | 18 |
| Total, serious adverse events | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | 2 | 27 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons4
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor vs Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant: Number of Participants That Experienced Transplant-related Mortality | Trial comparisons |
Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor vs Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant: Percentage of Participant Overall Survival Following Stem Cell Transplant | Trial comparisons |
Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor vs 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 | Trial comparisons |
Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor vs Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant: Number of Participants That Experienced Regimen Failure | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02105766
GET https://conductscience.org/api/trials/v1/NCT02105766/results?page=2