Trial · NCT00176852
Stem Cell Transplant for Hemoglobinopathy
NCT00176852, Phase 2, Phase 3, Completed, Masonic Cancer Center, University of Minnesota; results first posted 2017-05-09
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Stem Cell Transplant for Hemoglobinopathy. Trials, record NCT00176852. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00176852
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Stem Cell Transplant for Hemoglobinopathy
- NCT number
- NCT00176852
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 22 participants, actual
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Conditions
- Sickle Cell Disease, Thalassemia, Severe Congenital Neutropenia, Diamond-Blackfan Anemia, Shwachman-Diamond Syndrome
- Interventions
- Busulfan, Fludarabine, ATG, TLI, Busulfan, Cyclophosphamide, ATG, GCSF, Campath, Fludarabine, Cyclophosphamide, Total Body Irradiation, Stem cell infusion
- First posted
- 2005-09-15
- Last updated
- 2020-02-27
- Results first posted
- 2017-05-09
Source ClinicalTrials.gov
Registered outcomes17
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Patients Who Experienced Grade 3-5 Treatment Related Toxicity | 1 year | In general, grade 3 equates to moderate, grade 4 to severe and grade 5 to death. |
| Secondary | The Incidence of Chimerism at 100 Days | 100 days | The number of patients whose blood and/or bone marrow contains \> 10% donor cells. |
| Secondary | The Incidence of Chimerism at 6 Months | 6 months | The number of patients whose blood and/or bone marrow contains \> 10% donor cells. |
| Secondary | The Incidence of Chimerism at 1 Year | 1 year | The number of patients whose blood and/or bone marrow contains \> 10% donor cells. |
| Secondary | The Incidence of Grade 2-4 Acute Graft Versus Host Disease (Acute GVHD) | 100 days | The number of patients who experienced grades 2-4 Acute GVHD. Acute GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. Grades 2-4 equate to mild to severe disease. Symptoms typically appear within weeks after transplant. |
| Secondary | The Incidence of Grade 3-4 Acute Graft Versus Host Disease (Acute GVHD) | 100 days | The number of patients who experienced grades 3-4 Acute GVHD. Acute GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. IGrades 3-4 equate to moderate to severe disease. Symptoms typically appear within weeks after transplant. |
| Secondary | The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD) | 6 months | The number of patients who experienced Chronic GVHD. Chronic GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. Chronic GVHD can appear at any time after allogeneic transplant or several years after transplant. |
| Secondary | The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD) | 1 year | The number of patients who experienced Chronic GVHD. Chronic GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. Chronic GVHD can appear at any time after allogeneic transplant or several years after transplant. |
| Secondary | Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment | pre-transplant | The measure for quality of life used in this study is the Karnofsky Performance Score. The Karnofsky Performance Score runs from 100 to 0, where 100 is "perfect" health and 0 is death. |
| Secondary | Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment | 1 year | The measure for quality of life used in this study is the Karnofsky Performance Score. The Karnofsky Performance Score runs from 100 to 0, where 100 is "perfect" health and 0 is death. |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 registered outcomesOutcomes17
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 Experienced Grade 3-5 Treatment Related Toxicity | 1 year | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | The Incidence of Chimerism at 100 Days | 100 days | One of the 3 patients treated on Arm A was retreated at Day 40 and was not evaluable. One of the 14 patients on Arm A2 died before 100 days. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | The Incidence of Chimerism at 6 Months | 6 months | One of the 3 patients treated on Arm A was retreated at Day 40 and was not evaluable. One of the 14 patients on Arm A2 died before 6 months. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | The Incidence of Chimerism at 1 Year | 1 year | One of the 3 patients treated on Arm A was retreated at Day 40 and was not evaluable. One of the 14 patients on Arm A2 died before 1 year. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | The Incidence of Grade 2-4 Acute Graft Versus Host Disease (Acute GVHD) | 100 days | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | The Incidence of Grade 3-4 Acute Graft Versus Host Disease (Acute GVHD) | 100 days | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD) | 6 months | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD) | 1 year | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment | pre-transplant | units on a scale | MEDIAN | |
| SECONDARY | Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment | 1 year | Two of the 14 patients treated on Arm A2 died before 1 year. | units on a scale | MEDIAN |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 outcomesResults by group45
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 Experienced Grade 3-5 Treatment Related Toxicity | RIC Bu/Flu (A) (Discontinued) | 0 | Participants | ||
| Number of Patients Who Experienced Grade 3-5 Treatment Related Toxicity | MA Bu/Cy (B) | 0 | Participants | ||
| Number of Patients Who Experienced Grade 3-5 Treatment Related Toxicity | RIC Cy/Flu/TBI (A2) | 2 | Participants | ||
| The Incidence of Chimerism at 100 Days | RIC Bu/Flu (A) (Discontinued) | 1 | Participants | ||
| The Incidence of Chimerism at 100 Days | MA Bu/Cy (B) | 5 | Participants | ||
| The Incidence of Chimerism at 100 Days | RIC Cy/Flu/TBI (A2) | 11 | Participants | ||
| The Incidence of Chimerism at 6 Months | RIC Bu/Flu (A) (Discontinued) | 1 | Participants | ||
| The Incidence of Chimerism at 6 Months | MA Bu/Cy (B) | 5 | Participants | ||
| The Incidence of Chimerism at 6 Months | RIC Cy/Flu/TBI (A2) | 8 | Participants | ||
| The Incidence of Chimerism at 1 Year | RIC Bu/Flu (A) (Discontinued) | 1 | Participants |
1 to 10 of 45Source ClinicalTrials.gov
See all 45 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Full Conditioning (Discontinued / A) | 3 | |
| Overall Study | STARTED | Busulfan Conditioning (B) | 5 | |
| Overall Study | STARTED | Campath and TBI Conditioning (A2) | 14 | |
| Overall Study | COMPLETED | Full Conditioning (Discontinued / A) | 3 | |
| Overall Study | COMPLETED | Busulfan Conditioning (B) | 5 | |
| Overall Study | COMPLETED | Campath and TBI Conditioning (A2) | 14 | |
| Overall Study | NOT COMPLETED | Full Conditioning (Discontinued / A) | 0 | |
| Overall Study | NOT COMPLETED | Busulfan Conditioning (B) | 0 | |
| Overall Study | NOT COMPLETED | Campath and TBI Conditioning (A2) | 0 |
Source ClinicalTrials.gov
Adverse events123
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 | Bacterial Infection, Blood | Infections and infestations | Full Conditioning (Discontinued / A) | 2 | 3 | |
| other | Bacterial Infection, Blood | Infections and infestations | Busulfan Conditioning (B) | 2 | 5 | |
| other | Bacterial Infection, Blood | Infections and infestations | Campath and TBI Conditioning (A2) | 5 | 14 | |
| other | Bacterial Infection, Pulmonary | Infections and infestations | Full Conditioning (Discontinued / A) | 0 | 3 | |
| other | Bacterial Infection, Pulmonary | Infections and infestations | Busulfan Conditioning (B) | 1 | 5 | |
| other | Bacterial Infection, Pulmonary | Infections and infestations | Campath and TBI Conditioning (A2) | 2 | 14 | |
| other | Conjunctival Hemorrhage | Eye disorders | Full Conditioning (Discontinued / A) | 0 | 3 | |
| other | Conjunctival Hemorrhage | Eye disorders | Busulfan Conditioning (B) | 0 | 5 | |
| other | Conjunctival Hemorrhage | Eye disorders | Campath and TBI Conditioning (A2) | 2 | 14 | |
| other | Delayed Growth/Stature | Musculoskeletal and connective tissue disorders | Full Conditioning (Discontinued / A) | 3 | 3 |
1 to 10 of 123Source ClinicalTrials.gov
See all 123 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Full Conditioning (Discontinued / A) | ||
| Total, all-cause mortality | Busulfan Conditioning (B) | ||
| Total, all-cause mortality | Campath and TBI Conditioning (A2) | ||
| Total, other adverse events | Full Conditioning (Discontinued / A) | 3 | 3 |
| Total, other adverse events | Busulfan Conditioning (B) | 5 | 5 |
| Total, other adverse events | Campath and TBI Conditioning (A2) | 13 | 14 |
| Total, serious adverse events | Full Conditioning (Discontinued / A) | 2 | 3 |
| Total, serious adverse events | Busulfan Conditioning (B) | 0 | 5 |
| Total, serious adverse events | Campath and TBI Conditioning (A2) | 0 | 14 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT00176852
GET https://conductscience.org/api/trials/v1/NCT00176852/results?page=2