Trial · NCT00426517
Donor Stem Cell Transplantation for Congenital Immunodeficiencies
NCT00426517, Early phase 1, Completed, National Institute of Allergy and Infectious Diseases (NIAID); results first posted 2021-05-11
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Donor Stem Cell Transplantation for Congenital Immunodeficiencies. Trials, record NCT00426517. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00426517
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Donor Stem Cell Transplantation for Congenital Immunodeficiencies
- NCT number
- NCT00426517
- Status
- Completed
- Phases
- Early phase 1
- Enrollment
- 48 participants, actual
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- Conditions
- Inherited Immune Deficiencies
- Interventions
- Campath 1H, Busulfan, Horse Anti-human Thymocyte Globulin (h-ATG), Total Body Irradiation (TBI), Sirolimus or equivalent based on response
- First posted
- 2007-01-24
- Last updated
- 2021-05-11
- Results first posted
- 2021-05-11
- Limitations and caveats
- No limitations. Treatment of a rare disease.
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Stem Cell Transplant Engraftment | 1 year | Engraftment of allogeneic or matched unrelated (including cord blood) hematopoietic progenitor cells using moderate-dose busulfan and Campath-1H with or without whole body irradiation so as to attain phenotypic correction of congenital immunodeficiencies. |
| Secondary | Engraftment Without Development of GVHD | 1 year | Participants who achieved engraftment without development of graft versus host disease (GVHD). |
| Secondary | Participants With Established Stable Mixed Chimerism | 1 year | Number of participants with myeloid chimerism of greater than 10% of donor cells at 1 year post transplant |
| Secondary | Days to CD3 Count Greater Than 100 u/L | 1 year | Rapidity of immune reconstitution based on number of days to CD3 count greater than 100 u/L. |
| Secondary | Days to Absolute Neutrophil Recovery (ANC) | 1 year | Recovery is defined as an absolute neutrophil count (ANC) of ≥ 0.5 x 109 /L (500/mm3 ) for three consecutive laboratory values obtained on different days. Date of ANC recovery is the date of the first of three consecutive laboratory values where the ANC is ≥ 0.5 x 109 /L. |
| Secondary | Number of RBC Transfusions Per Subject | 1 year | Average number of red blood cell (RBC) transfusion per subject |
| Secondary | Days to Platelet Recovery | 1 year | Platelet recovery is defined as platelet value ≥ 20 × 109/L for three consecutive days and no platelet transfusions administered for previous seven consecutive days. The date of platelet recovery is the date of the first of three consecutive laboratory values ≥ 20 × 109/L. |
| Secondary | Incidence of Cytomegalovirus (CMV) Disease | 1 year | Number of events of Cytomegalovirus disease based on clinical sequelae that requires treatment (not reactivation) |
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 | Stem Cell Transplant Engraftment | 1 year | The analyses include only those who underwent transplant | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Engraftment Without Development of GVHD | 1 year | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Participants With Established Stable Mixed Chimerism | 1 year | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Days to CD3 Count Greater Than 100 u/L | 1 year | The analyses include only those who underwent transplant | Days | MEAN |
| SECONDARY | Days to Absolute Neutrophil Recovery (ANC) | 1 year | The analyses include only those who underwent transplant | Days | MEAN |
| SECONDARY | Number of RBC Transfusions Per Subject | 1 year | The analyses include only those who underwent transplant | transfusions per person | MEAN |
| SECONDARY | Days to Platelet Recovery | 1 year | The analyses include only those who underwent transplant | Days | MEAN |
| SECONDARY | Incidence of Cytomegalovirus (CMV) Disease | 1 year | The analyses include only those who underwent transplant | Number of events | MEAN |
Source ClinicalTrials.gov
Results by group32
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Stem Cell Transplant Engraftment | Matched Related Donor Stem Cell Transplant | 7 | Participants | ||
| Stem Cell Transplant Engraftment | Matched Unrelated Donor Stem Cell Transplant | 31 | Participants | ||
| Stem Cell Transplant Engraftment | Matched Unrelated Donor Stem Cell Transplant (MUD-non CGD) | 3 | Participants | ||
| Stem Cell Transplant Engraftment | Matched Unrelated Donor Transplant (MUD-CGD) Cord Blood | 0 | Participants | ||
| Engraftment Without Development of GVHD | Matched Related Donor Stem Cell Transplant | 6 | Participants | ||
| Engraftment Without Development of GVHD | Matched Unrelated Donor Stem Cell Transplant | 30 | Participants | ||
| Engraftment Without Development of GVHD | Matched Unrelated Donor Stem Cell Transplant (MUD-non CGD) | 3 | Participants | ||
| Engraftment Without Development of GVHD | Matched Unrelated Donor Transplant (MUD-CGD) Cord Blood | NA | Participants | ||
| Participants With Established Stable Mixed Chimerism | Matched Related Donor Stem Cell Transplant | 7 | Participants | ||
| Participants With Established Stable Mixed Chimerism | Matched Unrelated Donor Stem Cell Transplant | 30 | Participants |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Matched Related Donor Stem Cell Transplant | 7 | |
| Overall Study | STARTED | Matched Unrelated Donor Stem Cell Transplant | 36 | |
| Overall Study | STARTED | Matched Unrelated Donor Stem Cell Transplant (MUD-non CGD) | 3 | |
| Overall Study | STARTED | Matched Unrelated Donor Transplant (MUD-CGD) Cord Blood | 2 | |
| Overall Study | COMPLETED | Matched Related Donor Stem Cell Transplant | 7 | |
| Overall Study | COMPLETED | Matched Unrelated Donor Stem Cell Transplant | 36 | |
| Overall Study | COMPLETED | Matched Unrelated Donor Stem Cell Transplant (MUD-non CGD) | 3 | |
| Overall Study | COMPLETED | Matched Unrelated Donor Transplant (MUD-CGD) Cord Blood | 2 | |
| Overall Study | NOT COMPLETED | Matched Related Donor Stem Cell Transplant | 0 | |
| Overall Study | NOT COMPLETED | Matched Unrelated Donor Stem Cell Transplant | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events60
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 | Thyroiditis | Endocrine disorders | Matched Related Donor Stem Cell Transplant | 0 | 7 | |
| other | Thyroiditis | Endocrine disorders | Matched Unrelated Donor Stem Cell Transplant | 2 | 36 | |
| other | Thyroiditis | Endocrine disorders | Matched Unrelated Donor Stem Cell Transplant (MUD-non CGD) | 0 | 3 | |
| other | Thyroiditis | Endocrine disorders | Matched Unrelated Donor Transplant (MUD-CGD) Cord Blood | 0 | 2 | |
| other | Acute graft versus host disease in intestine | Gastrointestinal disorders | Matched Related Donor Stem Cell Transplant | 1 | 7 | |
| other | Acute graft versus host disease in intestine | Gastrointestinal disorders | Matched Unrelated Donor Stem Cell Transplant | 2 | 36 | |
| other | Acute graft versus host disease in intestine | Gastrointestinal disorders | Matched Unrelated Donor Stem Cell Transplant (MUD-non CGD) | 0 | 3 | |
| other | Acute graft versus host disease in intestine | Gastrointestinal disorders | Matched Unrelated Donor Transplant (MUD-CGD) Cord Blood | 0 | 2 | |
| other | Chronic graft versus host disease | Immune system disorders | Matched Related Donor Stem Cell Transplant | 0 | 7 | |
| other | Chronic graft versus host disease | Immune system disorders | Matched Unrelated Donor Stem Cell Transplant | 1 | 36 |
1 to 10 of 60Source ClinicalTrials.gov
See all 60 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Matched Related Donor Stem Cell Transplant | 0 | 7 |
| Total, all-cause mortality | Matched Unrelated Donor Stem Cell Transplant | 9 | 36 |
| Total, all-cause mortality | Matched Unrelated Donor Stem Cell Transplant (MUD-non CGD) | 1 | 3 |
| Total, all-cause mortality | Matched Unrelated Donor Transplant (MUD-CGD) Cord Blood | 0 | 2 |
| Total, other adverse events | Matched Related Donor Stem Cell Transplant | 3 | 7 |
| Total, other adverse events | Matched Unrelated Donor Stem Cell Transplant | 17 | 36 |
| Total, other adverse events | Matched Unrelated Donor Stem Cell Transplant (MUD-non CGD) | 1 | 3 |
| Total, other adverse events | Matched Unrelated Donor Transplant (MUD-CGD) Cord Blood | 2 | 2 |
| Total, serious adverse events | Matched Related Donor Stem Cell Transplant | 1 | 7 |
| Total, serious adverse events | Matched Unrelated Donor Stem Cell Transplant | 4 | 36 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers6
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 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/NCT00426517
GET https://conductscience.org/api/trials/v1/NCT00426517/results?page=2