Trial · NCT00987480

Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine

NCT00987480, Phase 2, Completed, Memorial Sloan Kettering Cancer Center; results first posted 2018-07-10

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine. Trials, record NCT00987480. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00987480

On this page

Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine
NCT number: NCT00987480
Status: Completed
Phases: Phase 2
Enrollment: 45 participants, actual
Lead sponsor: Memorial Sloan Kettering Cancer Center
Conditions: Aplastic Anemia, Leukemia, Myelodysplastic Syndrome
Interventions: Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine., CliniMACS device
First posted: 2009-10-01
Last updated: 2018-07-10
Results first posted: 2018-07-10

Source ClinicalTrials.gov

Registered outcomes6

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Successful Neutrophil Engraftment	2 years
Primary	The Incidence of Early Transplant Related Mortality	2 years
Primary	The Incidence of Acute GvHD	100 days
Primary	The Incidence of Chronic GvHD	2 years
Secondary	Overall Survival at 3 Years	3 years	Overall Survival is defined as time from date of transplant to event (death from any cause) or last follow-up.
Secondary	Disease-free Survival at 3 Years	3 years	Defined as time from date of transplant to relapse, graft rejection or graft failure, or death. Primary non-engraftment is diagnosed when the participants fails to achieve an ANC \>/= 500/ul at any time in the first 28 days post-transplant. For participants with MDS or AML, relapse will be analyzed as to type and genetic origin of the MDS/leukemic cells. These will be defined by an increasing number of blasts in the marrow over 5% by the presence of circulating peripheral blasts, or by the presence of blasts in any extramedullary site. Cytogenetic analysis of the marrow and/or peripheral blood will also be obtained for the diagnosis of relapse.

Source ClinicalTrials.gov

Outcomes6

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Units	Measured as
PRIMARY	Successful Neutrophil Engraftment	2 years	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	The Incidence of Early Transplant Related Mortality	2 years	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	The Incidence of Acute GvHD	100 days	percentage of participants	NUMBER
PRIMARY	The Incidence of Chronic GvHD	2 years	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Overall Survival at 3 Years	3 years	percentage of participants	NUMBER
SECONDARY	Disease-free Survival at 3 Years	3 years	percentage of participants	NUMBER

Source ClinicalTrials.gov

Results by group8

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Units
Successful Neutrophil Engraftment	Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected	Achieved neutrophil engraftment	44	Participants
Successful Neutrophil Engraftment	Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected	Did not achieve neutrophil engraftment	1	Participants
The Incidence of Early Transplant Related Mortality	Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected		0	Participants
The Incidence of Acute GvHD	Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected		6.7	percentage of participants
The Incidence of Chronic GvHD	Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected	Developed limited chronic GVHD	3	Participants
The Incidence of Chronic GvHD	Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected	Did not develop limited chronic GVHD	42	Participants
Overall Survival at 3 Years	Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected		80	percentage of participants
Disease-free Survival at 3 Years	Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected		77.8	percentage of participants

Source ClinicalTrials.gov

Participant flow3

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected	45
Overall Study	COMPLETED	Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected	45
Overall Study	NOT COMPLETED	Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected	0

Source ClinicalTrials.gov

Adverse events57

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
other	Aspartate aminotransferase increased	Investigations	Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected	3	45
other	Bicarbonate, serum-low	Investigations	Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected	3	45
other	Blood bilirubin increased	Investigations	Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected	3	45
other	Sodium, high (hypernatremia)	Metabolism and nutrition disorders	Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected	3	45
other	Glucose, low (hypoglycemia)	Metabolism and nutrition disorders	Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected	4	45
other	Hypermagnesemia	Metabolism and nutrition disorders	Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected	4	45
other	Mucositis (Clin exam)- Pharynx	Gastrointestinal disorders	Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected	4	45
other	Phosphate, low (hypophosphatemia)	Metabolism and nutrition disorders	Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected	4	45
other	Alanine aminotransferase increased	Investigations	Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected	5	45
other	Infection, other	Infections and infestations	Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected	5	45

1 to 10 of 57Source ClinicalTrials.gov

See all 57 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected	9	45
Total, other adverse events	Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected	31	45
Total, serious adverse events	Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected	11	45

Source ClinicalTrials.gov

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
28179273	DERIVED	PubMed
31851762	DERIVED	PubMed

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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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/NCT00987480

GET https://conductscience.org/api/trials/v1/NCT00987480/events?page=2

API reference For AI assistants