Trial · NCT00923364
Pilot and Feasibility Study of Reduced-Intensity Hematopoietic Stem Cell Transplant for MonoMAC
NCT00923364, Phase 2, Completed, National Cancer Institute (NCI); results first posted 2017-11-30
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Pilot and Feasibility Study of Reduced-Intensity Hematopoietic Stem Cell Transplant for MonoMAC. Trials, record NCT00923364. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00923364
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Pilot and Feasibility Study of Reduced-Intensity Hematopoietic Stem Cell Transplant for MonoMAC
- NCT number
- NCT00923364
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 19 participants, actual
- Lead sponsor
- National Cancer Institute (NCI)
- Conditions
- MDS, Immunodeficiency, GATA2
- Interventions
- Cyclophosphamide (CTX, Cytoxan), Fludarabine(Fludara,Berlex Laboratories), Total Body Irradiation (TBI), Allogeneic Hematopoietic Stem Cell (HSC), Equine Anti-Thymocyte Globulin
- First posted
- 2009-06-18
- Last updated
- 2020-03-12
- Results first posted
- 2017-11-30
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Days to Neutrophil Engraftment | 30 days | Neutrophil engraftment is defined as a neutrophil count of \>0.5 x 10(9) cells/L for 3 consecutive days. |
| Primary | Days to Platelet Engraftment | 30 days | Platelet engraftment is defined as a platelet count of \>20 x 10(9) cells/L for 7 consecutive days without requiring a platelet transfusion. |
| Secondary | Percentage of Donor Cells at Last Follow Up in Patients With Mutations GATA Binding Protein 2 (GATA2) | 2 years | Engraftment of donor cells was assessed using polymorphisms in regions known to contain short tandem repeats. Peripheral blood cluster of differentiation 14 (CD14+), cluster of differentiation 3 (CD3-)/cluster of differentiation 56 (CD56+), cluster of differentiation 19 (CD19+), and CD3+ subsets were isolated by flow cytometry and chimerism was assessed. |
| Secondary | Incidence of Acute and Chronic Graft-versus-host Disease (GVHD) | 2 years | Acute GVHD is assessed according to the 1994 Consensus Conference Grading Criteria. Chronic GVHD is assessed by the 2005 Chronic GVHD Consensus Project. GVHD can affect performance status and attack multiple organ systems such as the skin, liver, gut, mouth, eyes, joints, lung, etc. that can lead to a rash, diarrhea, metabolic changes, infection and/or death. The clinical grading of acute GVHD is grade 0 (none) to 4 (severe). Chronic GVHD is a delayed form of GVHD that may occur after day 100 post transplant. |
| Secondary | Overall Survival | 2 years | Overall survival is defined as date of on-study to date of death from any cause or last follow up. |
| Secondary | Number of Participants With Disease Free Survival | 2 years | Number of participants with documented evidence of disease progression following start of treatment. |
| Secondary | Number of Participants With Serious and Non-serious Adverse Events | Date treatment consent signed to date off study, approximately, 91 months | Here is the number of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events v3.0. A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Days to Neutrophil Engraftment | 30 days | 10/19 analyzed:5 pts were healthy related donors, 1 pt taken off study prior to transplant \& 3 pts had early deaths (before outcome measurements could be evaluated) | Days | MEDIAN |
| PRIMARY | Days to Platelet Engraftment | 30 days | 10/19 analyzed:5 pts were healthy related donors, 1 pt taken off study prior to transplant \& 3 pts had early deaths (before outcome measurements could be evaluated) | Days | MEDIAN |
| SECONDARY | Percentage of Donor Cells at Last Follow Up in Patients With Mutations GATA Binding Protein 2 (GATA2) | 2 years | 10/19 analyzed:5 pts were healthy related donors, 1 pt taken off study prior to transplant \& 3 pts had early deaths (before outcome measurements could be evaluated) | percentage of donor cells | MEAN |
| SECONDARY | Incidence of Acute and Chronic Graft-versus-host Disease (GVHD) | 2 years | 10/19 analyzed:5 pts were healthy related donors, 1 pt taken off study prior to transplant \& 3 pts had early deaths (before outcome measurements could be evaluated) | participants | NUMBER |
| SECONDARY | Overall Survival | 2 years | 10/19 analyzed:5 pts were healthy related donors, 1 pt taken off study prior to transplant \& 3 pts had early deaths (before outcome measurements could be evaluated) | months | MEDIAN |
| SECONDARY | Number of Participants With Disease Free Survival | 2 years | 10/19 analyzed:5pts were healthy related donors, 1 pt taken off study prior to transplant \& 3 pts had early deaths (before outcome measurements could be evaluated) | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Serious and Non-serious Adverse Events | Date treatment consent signed to date off study, approximately, 91 months | Data were not collected separately for donors and recipients. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group11
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Days to Neutrophil Engraftment | Recipients Only | 12 | Full Range 12 to 80 | Days | |
| Days to Platelet Engraftment | Recipients Only | 30 | Full Range 0 to 30 | Days | |
| Percentage of Donor Cells at Last Follow Up in Patients With Mutations GATA Binding Protein 2 (GATA2) | Recipients Only | Donor CD14+ cells, % | 99.8 | Full Range 99 to 100 | percentage of donor cells |
| Percentage of Donor Cells at Last Follow Up in Patients With Mutations GATA Binding Protein 2 (GATA2) | Recipients Only | Donor CD19+ cells, % | 100 | Full Range 100 to 100 | percentage of donor cells |
| Percentage of Donor Cells at Last Follow Up in Patients With Mutations GATA Binding Protein 2 (GATA2) | Recipients Only | Donor CD3-/CD56+ cells, % | 99.8 | Full Range 99 to 100 | percentage of donor cells |
| Percentage of Donor Cells at Last Follow Up in Patients With Mutations GATA Binding Protein 2 (GATA2) | Recipients Only | Donor CD3+ cells, % | 97.6 | Full Range 91 to 100 | percentage of donor cells |
| Incidence of Acute and Chronic Graft-versus-host Disease (GVHD) | Recipients Only | Acute | 2 | participants | |
| Incidence of Acute and Chronic Graft-versus-host Disease (GVHD) | Recipients Only | Chronic | 3 | participants | |
| Overall Survival | Recipients Only | 76 | Full Range 18 to 95 | months | |
| Number of Participants With Disease Free Survival | Recipients Only | 8 | Participants |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Recipients | 14 | |
| Overall Study | STARTED | Healthy Related Donors | 5 | |
| Overall Study | COMPLETED | Recipients | 10 | |
| Overall Study | COMPLETED | Healthy Related Donors | 5 | |
| Overall Study | NOT COMPLETED | Recipients | 4 | |
| Overall Study | NOT COMPLETED | Healthy Related Donors | 0 |
Source ClinicalTrials.gov
Adverse events187
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 | Pain::Bone | Musculoskeletal and connective tissue disorders | Recipients and Healthy Related Donors | 1 | 19 | 1 |
| other | Pain::Head/headache | Nervous system disorders | Recipients and Healthy Related Donors | 2 | 19 | 2 |
| other | ALT, SGPT (serum glutamic pyruvic transaminase) | Metabolism and nutrition disorders | Recipients and Healthy Related Donors | 11 | 19 | 37 |
| other | AST, SGOT(serum glutamic oxaloacetic transaminase) | Metabolism and nutrition disorders | Recipients and Healthy Related Donors | 10 | 19 | 38 |
| other | Acidosis (metabolic or respiratory) | Metabolism and nutrition disorders | Recipients and Healthy Related Donors | 1 | 19 | 3 |
| other | Albumin, serum-low (hypoalbuminemia) | Metabolism and nutrition disorders | Recipients and Healthy Related Donors | 5 | 19 | 20 |
| other | Alkaline phosphatase | Metabolism and nutrition disorders | Recipients and Healthy Related Donors | 6 | 19 | 23 |
| other | Apnea | Respiratory, thoracic and mediastinal disorders | Recipients and Healthy Related Donors | 1 | 19 | 1 |
| other | Ascites (non-malignant) | Gastrointestinal disorders | Recipients and Healthy Related Donors | 2 | 19 | 2 |
| other | Bilirubin (hyperbilirubinemia) | Metabolism and nutrition disorders | Recipients and Healthy Related Donors | 5 | 19 | 12 |
1 to 10 of 187Source ClinicalTrials.gov
See all 187 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Recipients and Healthy Related Donors | 4 | 19 |
| Total, other adverse events | Recipients and Healthy Related Donors | 16 | 19 |
| Total, serious adverse events | Recipients and Healthy Related Donors | 12 | 19 |
Source ClinicalTrials.gov
Papers7
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/NCT00923364
GET https://conductscience.org/api/trials/v1/NCT00923364/events?page=2