Trial · NCT00061568
Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias
NCT00061568, Phase 1, Phase 2, Active not recruiting, National Heart, Lung, and Blood Institute (NHLBI); results first posted 2024-02-29
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias. Trials, record NCT00061568. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00061568
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias
- NCT number
- NCT00061568
- Status
- Active not recruiting
- Phases
- Phase 1, Phase 2
- Enrollment
- 130 participants, actual
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- Conditions
- Congenital Hemolytic Anemia, Sickle Cell Disease
- Interventions
- Peripheral blood hematopoietic progenitor cell (PBPC) transplant, Alemtuzumab, Peripheral blood hematopoietic progenitor cell Apheresis, Sirolimus
- First posted
- 2003-05-29
- Last updated
- 2026-09-09
- Results first posted
- 2024-02-29
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants That Experience Treatment Success Following Stem Cell Transplant | Up to 1 year | Number of participants that experience treatment success at one year following stem cell transplant. Treatment success is defined as full donor type hemoglobin on hemoglobin electrophoresis for patients with sickle cell disease and transfusion-independence for patients with beta-thalassemia. |
| Secondary | Mean Myeloid Chimerism Level | up to 2 years | Mean Myeloid Chimerism Level in participants following stem cell transplant. |
| Secondary | 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. | Up to 1 year | 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 Limited or Extensive Chronic GVHD | Day 100 up to 3 Years | Number of participants who developed Limited or Extensive Chronic Graft vs Host Disease (GVHD). Limited disease is characterized by localized skin involvement and/or evidence of hepatic dysfunction. Limited disease is associated with a favorable outcome without systemic therapy, while extensive disease patients have an unfavorable outcome. Extensive chronic GVHD is defined as GVHD occurring after day 100 that did not meet the definition of limited chronic GVHD. Extensive disease presents either with generalized skin involvement, or with localized skin involvement or hepatic dysfunction plus at least one of the following: Liver histology showing chronic progressive hepatitis, bridging necrosis, or cirrhosis Involvement of the eye (Schirmer's test with less than 5 mm wetting) (see "Diagnosis and classification of Sjögren's syndrome") Involvement of minor salivary glands or oral mucosa (as demonstrated on labial or mucosal biopsy specimen) Involvement of any other target organ |
| Secondary | Number of Participants With Regimen Failure | Up to 1 year | Number of participants with regimen failure. Regimen failure is defined as those participants that experienced graft verses host disease or relapse of sickle cell disease or beta-thalassemia. |
| Secondary | Number of Participants With Disease-free Survival | Up to 2 year | Number of participants with disease-free survival, as defined by: alive and free of acute complications related to sickle cell disease or beta-thalassemia. |
| Secondary | Number of Participants Overall Survival | 1 year and 2 year | Number of participants overall survival at year 1 and year 2. Overall survival is defined as participants alive at 1 and 2 years following stem cell transplant. |
| Secondary | Number of Participants That Experienced a Transplant-related Mortality | Up to 2 year | Number of participants that experienced a transplant related mortality, as defined as death from causes other than relapse (such as: GVHD, toxicity, infection, other and unknown causes). |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants That Experience Treatment Success Following Stem Cell Transplant | Up to 1 year | Pre-specified in the protocol to only assess this Outcome Measure in the Participants with Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants arm. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Mean Myeloid Chimerism Level | up to 2 years | Pre-specified in the protocol to only assess this Outcome Measure in the Participants with Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants arm. | % of Donor Chimerism | MEAN |
| SECONDARY | 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. | Up to 1 year | Pre-specified in the protocol to only assess this Outcome Measure in the Participants with Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants arm. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Who Developed Limited or Extensive Chronic GVHD | Day 100 up to 3 Years | Pre-specified in the protocol to only assess this Outcome Measure in the Participants with Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants arm. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Regimen Failure | Up to 1 year | Pre-specified in the protocol to only assess this Outcome Measure in the Participants with Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants arm. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Disease-free Survival | Up to 2 year | Pre-specified in the protocol to only assess this Outcome Measure in the Participants with Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants arm. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Overall Survival | 1 year and 2 year | Pre-specified in the protocol to only assess this Outcome Measure in the Participants with Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants arm. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants That Experienced a Transplant-related Mortality | Up to 2 year | Pre-specified in the protocol to only assess this Outcome Measure in the Participants with Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants arm. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group14
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants That Experience Treatment Success Following Stem Cell Transplant | Participants With Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants | 53 | Participants | ||
| Mean Myeloid Chimerism Level | Participants With Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants | Myeloid Chimerism | 90.1 | Standard Error 2.8 | % of Donor Chimerism |
| Mean Myeloid Chimerism Level | Participants With Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants | CD3+ Chimerism | 55.0 | Standard Error 3.5 | % of Donor Chimerism |
| 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. | Participants With Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants | Grade I Acute Graft Verses Host Disease | 0 | Participants | |
| 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. | Participants With Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants | Grade II Acute Graft Verses Host Disease | 0 | Participants | |
| 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. | Participants With Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants | Grade III Acute Graft Verses Host Disease | 0 | Participants | |
| 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. | Participants With Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants | Grade IV Acute Graft Verses Host Disease | 0 | Participants | |
| Number of Participants Who Developed Limited or Extensive Chronic GVHD | Participants With Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants | Limited Chronic Graft vs Host Disease | 0 | Participants | |
| Number of Participants Who Developed Limited or Extensive Chronic GVHD | Participants With Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants | Extensive Chronic Graft vs Host Disease | 0 | Participants | |
| Number of Participants With Regimen Failure | Participants With Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants | 9 | Participants |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Participants With Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants | 62 | |
| Overall Study | STARTED | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | 68 | |
| Overall Study | COMPLETED | Participants With Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants | 43 | |
| Overall Study | COMPLETED | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | 68 | |
| Overall Study | NOT COMPLETED | Participants With Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants | 19 | |
| Overall Study | NOT COMPLETED | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | 0 |
Source ClinicalTrials.gov
Adverse events182
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 | Anemia | Blood and lymphatic system disorders | Participants With Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants | 4 | 62 | 4 |
| other | Anemia | Blood and lymphatic system disorders | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | 0 | 68 | 0 |
| other | Weight gain | Investigations | Participants With Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants | 5 | 62 | 5 |
| other | Weight gain | Investigations | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | 0 | 68 | 0 |
| other | Hypothyroid | Endocrine disorders | Participants With Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants | 3 | 62 | 3 |
| other | Hypothyroid | Endocrine disorders | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | 0 | 68 | 0 |
| other | Hyperthyroid | Endocrine disorders | Participants With Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants | 3 | 62 | 3 |
| other | Hyperthyroid | Endocrine disorders | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | 0 | 68 | 0 |
| other | Rhabdomyolysis | Musculoskeletal and connective tissue disorders | Participants With Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants | 13 | 62 | 13 |
| other | Rhabdomyolysis | Musculoskeletal and connective tissue disorders | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | 0 | 68 | 0 |
1 to 10 of 182Source ClinicalTrials.gov
See all 182 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Participants With Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants | 6 | 62 |
| Total, all-cause mortality | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | 0 | 68 |
| Total, other adverse events | Participants With Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants | 13 | 62 |
| Total, other adverse events | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | 0 | 68 |
| Total, serious adverse events | Participants With Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants | 44 | 62 |
| Total, serious adverse events | Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor | 6 | 68 |
Source ClinicalTrials.gov
Papers8
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Participants With Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants vs Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor: deaths from any cause | Trial comparisons |
| Participants With Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants vs Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor: serious adverse events | 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/NCT00061568
GET https://conductscience.org/api/trials/v1/NCT00061568/events?page=2