Trial · NCT00744692

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

NCT00744692, Phase 1, Completed, Duke University; results first posted 2014-08-13

3 sourcestrials-2026-11-12-0.5.0

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ConductScience Library. Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders. Trials, record NCT00744692. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00744692

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Trial

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

Title: Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
NCT number: NCT00744692
Status: Completed
Phases: Phase 1
Enrollment: 22 participants, actual
Lead sponsor: Duke University
Conditions: Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies, Inborn Errors of Metabolism, Sickle Cell, Thalassemia, Lysosomal Storage Disease
Interventions: Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning
First posted: 2008-09-01
Last updated: 2014-08-13
Results first posted: 2014-08-13

Source ClinicalTrials.gov

Registered outcomes9

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

List	Outcome	Time frame	Description
Primary	Determine the Feasibility of Attaining Acceptable Rates of Donor Cell Engraftment (>25% Donor Cells at 180 Days) Following RIC Regimens in Children < 21 Years Receiving UCBT for Non-malignant Disorders.	180 days post transplant	Determine the feasibility of attaining acceptable rates of donor cell engraftment (\>25% donor cells at 180 days) following reduced intensity conditioning regimens in children \< 21 years receiving cord blood transplant for non-malignant disorders.
Secondary	To Describe the Pace of Neutrophil Recovery	42 days post transplant	Neutrophil recovery was defined as the first day of an absolute neutrophil count (ANC) more than 500/uL for 3 consecutive days not secondary to granulocyte infusions
Secondary	To Evaluate the Pace of Immune Reconstitution.	1 year post transplant	Immune reconstitution after RIC in UCBT was described. CD4 count is a standard measure of immune reconstitution and is described here. Additional data is available upon request.
Secondary	To Determine the Overall Survival at day180 Post-transplant	180 days	To determine the overall survival at day180 post-transplant: determined by Kaplan Meier survival analysis
Secondary	To Describe Incidence of Acute Graft Versus Host Disease (GVHD) (II - IV)	100 days post transplant	To describe incidence of acute Graft Versus Host Disease (GVHD) (II - IV) : measured by cumulative incidence analysis
Secondary	To Describe the Incidence of Grade 3-4 Organ Toxicity	2 years post transplant
Secondary	To Evaluate Long-term Complications, Such as Sterility, Endocrinopathy, and Growth Failure	at least 2 years post transplant
Secondary	To Evaluate the Incidence of Late Graft Failures at 2 Years Post-transplant	2 years post transplant
Secondary	To Describe the Pace of Platelet Recovery	180 days post transplant	Platelet engraftment was defined as the first day of platelet counts more than 50,000/uL for 7 consecutive days without transfusions

Source ClinicalTrials.gov

Outcomes9

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Determine the Feasibility of Attaining Acceptable Rates of Donor Cell Engraftment (>25% Donor Cells at 180 Days) Following RIC Regimens in Children < 21 Years Receiving UCBT for Non-malignant Disorders.	180 days post transplant	Of the 22 patients enrolled, 18 patients were alive at 180 days, the time-point for primary end point	% of participants	NUMBER
SECONDARY	To Describe the Pace of Neutrophil Recovery	42 days post transplant		days	MEDIAN
SECONDARY	To Evaluate the Pace of Immune Reconstitution.	1 year post transplant	Of the 22 patients at baseline, 5 patients died early before a year; and 2 additional patients had early graft failure. Thus 7 patients were not available for analysis of Immune reconstitution endpoint. CD4 count is reported here.	cells/uL	MEDIAN
SECONDARY	To Determine the Overall Survival at day180 Post-transplant	180 days		percentage of participants	NUMBER
SECONDARY	To Describe Incidence of Acute Graft Versus Host Disease (GVHD) (II - IV)	100 days post transplant		percentage of participants	NUMBER
SECONDARY	To Describe the Incidence of Grade 3-4 Organ Toxicity	2 years post transplant		participants	NUMBER
SECONDARY	To Evaluate Long-term Complications, Such as Sterility, Endocrinopathy, and Growth Failure	at least 2 years post transplant	Of the 22 patients at baseline, 5 patients died early before a year; and 2 additional patients had early graft failure. Thus 7 patients were not available for analysis of this 2 year late effects endpoint.	percentage of patients	NUMBER
SECONDARY	To Evaluate the Incidence of Late Graft Failures at 2 Years Post-transplant	2 years post transplant	Of the 22 patients at baseline, 5 patients died early before a year; and 2 additional patients had early graft failure. Thus 7 patients were not available for analysis of this 2 year late graft failure endpoint.	participants	NUMBER
SECONDARY	To Describe the Pace of Platelet Recovery	180 days post transplant		days	MEDIAN

Source ClinicalTrials.gov

Results by group9

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

Outcome	Group	Value	Spread	Units
Determine the Feasibility of Attaining Acceptable Rates of Donor Cell Engraftment (>25% Donor Cells at 180 Days) Following RIC Regimens in Children < 21 Years Receiving UCBT for Non-malignant Disorders.	RIC Cord Blood Transplant	88		% of participants
To Describe the Pace of Neutrophil Recovery	RIC Cord Blood Transplant	20	Full Range 12 to 28	days
To Evaluate the Pace of Immune Reconstitution.	RIC Cord Blood Transplant	805	Full Range 6 to 2013	cells/uL
To Determine the Overall Survival at day180 Post-transplant	RIC Cord Blood Transplant	81.8	95% Confidence Interval 58.5 to 92.8	percentage of participants
To Describe Incidence of Acute Graft Versus Host Disease (GVHD) (II - IV)	RIC Cord Blood Transplant	22.7	95% Confidence Interval 5.3 to 40.1	percentage of participants
To Describe the Incidence of Grade 3-4 Organ Toxicity	RIC Cord Blood Transplant	0		participants
To Evaluate Long-term Complications, Such as Sterility, Endocrinopathy, and Growth Failure	RIC Cord Blood Transplant	0		percentage of patients
To Evaluate the Incidence of Late Graft Failures at 2 Years Post-transplant	RIC Cord Blood Transplant	0		participants
To Describe the Pace of Platelet Recovery	RIC Cord Blood Transplant	48	Full Range 33 to 144	days

Source ClinicalTrials.gov

Participant flow3

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

Period	Milestone	Group	Participants
Overall Study	STARTED	RIC Cord Blood Transplant	22
Overall Study	COMPLETED	RIC Cord Blood Transplant	22
Overall Study	NOT COMPLETED	RIC Cord Blood Transplant	0

Source ClinicalTrials.gov

Adverse events23

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
serious	Respiratory Distress	Respiratory, thoracic and mediastinal disorders	RIC Cord Blood Transplant	2	22
serious	Fever	Investigations	RIC Cord Blood Transplant	5	22
serious	Dehydration	Metabolism and nutrition disorders	RIC Cord Blood Transplant	2	22
serious	Auto Immune Hemolytic Anemia	Blood and lymphatic system disorders	RIC Cord Blood Transplant	2	22
serious	Infection	Infections and infestations	RIC Cord Blood Transplant	12	22
serious	Hemolytic Transfusion Reaction	Blood and lymphatic system disorders	RIC Cord Blood Transplant	1	22
serious	Graft Failure Post Transplant	Blood and lymphatic system disorders	RIC Cord Blood Transplant	2	22
serious	Sepsis	Infections and infestations	RIC Cord Blood Transplant	3	22
serious	Abdominal Pain	Gastrointestinal disorders	RIC Cord Blood Transplant	1	22
serious	Nephrotic Syndrome	Renal and urinary disorders	RIC Cord Blood Transplant	1	22

1 to 10 of 23Source ClinicalTrials.gov

See all 23 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	RIC Cord Blood Transplant
Total, other adverse events	RIC Cord Blood Transplant	0	22
Total, serious adverse events	RIC Cord Blood Transplant	18	22

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
24296492	DERIVED

Source 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/NCT00744692

GET https://conductscience.org/api/trials/v1/NCT00744692/events?page=2

API reference For AI assistants