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

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ClinicalTrials.gov Identifier: NCT00744692
Recruitment Status : Completed
First Posted : September 1, 2008
Results First Posted : August 13, 2014
Last Update Posted : August 13, 2014
Sponsor:
Information provided by (Responsible Party):
Duke University

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Non Malignant Disorders
Immunodeficiencies
Congenital Marrow Failures
Hemoglobinopathies
Inborn Errors of Metabolism
Sickle Cell
Thalassemia
Lysosomal Storage Disease
Interventions Biological: Unrelated Umbilical Cord Blood Transplant
Drug: Reduced Intensity Conditioning
Enrollment 22

Recruitment Details  
Pre-assignment Details  
Arm/Group Title RIC Cord Blood Transplant
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Reduced Intensity Conditioning for Umbilical Cord Blood Transplant

Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant

Reduced Intensity Conditioning

Period Title: Overall Study
Started 22
Completed 22
Not Completed 0
Arm/Group Title RIC Cord Blood Transplant
Hide Arm/Group Description

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant

Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant

Reduced Intensity Conditioning

Overall Number of Baseline Participants 22
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22 subjects undergoing umbilical cord blood transplant
Age, Continuous   [1] 
Median (Full Range)
Unit of measure:  Years
Number Analyzed 22 participants
2.83
(0.3 to 8)
[1]
Measure Description: Age (in years) on the day of transplant
Age, Categorical   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 22 participants
<=18 years
22
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
[1]
Measure Description: Age (years) on the day of transplant
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 22 participants
Female
9
  40.9%
Male
13
  59.1%
1.Primary Outcome
Title 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.
Hide Description 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.
Time Frame 180 days post transplant
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Of the 22 patients enrolled, 18 patients were alive at 180 days, the time-point for primary end point
Arm/Group Title RIC Cord Blood Transplant
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Reduced Intensity Conditioning for Umbilical Cord Blood Transplant

Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant

Reduced Intensity Conditioning

Overall Number of Participants Analyzed 18
Measure Type: Number
Unit of Measure: % of participants
88
2.Secondary Outcome
Title To Describe the Pace of Neutrophil Recovery
Hide Description 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
Time Frame 42 days post transplant
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Hide Analysis Population Description
[Not Specified]
Arm/Group Title RIC Cord Blood Transplant
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Reduced Intensity Conditioning for Umbilical Cord Blood Transplant

Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant

Reduced Intensity Conditioning

Overall Number of Participants Analyzed 22
Median (Full Range)
Unit of Measure: days
20
(12 to 28)
3.Secondary Outcome
Title To Evaluate the Pace of Immune Reconstitution.
Hide Description 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.
Time Frame 1 year post transplant
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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.
Arm/Group Title RIC Cord Blood Transplant
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Reduced Intensity Conditioning for Umbilical Cord Blood Transplant

Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant

Reduced Intensity Conditioning

Overall Number of Participants Analyzed 15
Median (Full Range)
Unit of Measure: cells/uL
805
(6 to 2013)
4.Secondary Outcome
Title To Determine the Overall Survival at day180 Post-transplant
Hide Description To determine the overall survival at day180 post-transplant: determined by Kaplan Meier survival analysis
Time Frame 180 days
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Hide Analysis Population Description
[Not Specified]
Arm/Group Title RIC Cord Blood Transplant
Hide Arm/Group Description:

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant

Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant

Reduced Intensity Conditioning

Overall Number of Participants Analyzed 22
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
81.8
(58.5 to 92.8)
5.Secondary Outcome
Title To Describe Incidence of Acute Graft Versus Host Disease (GVHD) (II - IV)
Hide Description To describe incidence of acute Graft Versus Host Disease (GVHD) (II - IV) : measured by cumulative incidence analysis
Time Frame 100 days post transplant
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Hide Analysis Population Description
[Not Specified]
Arm/Group Title RIC Cord Blood Transplant
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Reduced Intensity Conditioning for Umbilical Cord Blood Transplant

Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant

Reduced Intensity Conditioning

Overall Number of Participants Analyzed 22
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
22.7
(5.3 to 40.1)
6.Secondary Outcome
Title To Describe the Incidence of Grade 3-4 Organ Toxicity
Hide Description [Not Specified]
Time Frame 2 years post transplant
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Hide Analysis Population Description
[Not Specified]
Arm/Group Title RIC Cord Blood Transplant
Hide Arm/Group Description:

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant

Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant

Reduced Intensity Conditioning

Overall Number of Participants Analyzed 22
Measure Type: Number
Unit of Measure: participants
0
7.Secondary Outcome
Title To Evaluate Long-term Complications, Such as Sterility, Endocrinopathy, and Growth Failure
Hide Description [Not Specified]
Time Frame at least 2 years post transplant
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Hide Analysis Population Description
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.
Arm/Group Title RIC Cord Blood Transplant
Hide Arm/Group Description:

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant

Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant

Reduced Intensity Conditioning

Overall Number of Participants Analyzed 15
Measure Type: Number
Unit of Measure: percentage of patients
0
8.Secondary Outcome
Title To Evaluate the Incidence of Late Graft Failures at 2 Years Post-transplant
Hide Description [Not Specified]
Time Frame 2 years post transplant
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Hide Analysis Population Description
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.
Arm/Group Title RIC Cord Blood Transplant
Hide Arm/Group Description:

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant

Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant

Reduced Intensity Conditioning

Overall Number of Participants Analyzed 15
Measure Type: Number
Unit of Measure: participants
0
9.Secondary Outcome
Title To Describe the Pace of Platelet Recovery
Hide Description Platelet engraftment was defined as the first day of platelet counts more than 50,000/uL for 7 consecutive days without transfusions
Time Frame 180 days post transplant
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Hide Analysis Population Description
[Not Specified]
Arm/Group Title RIC Cord Blood Transplant
Hide Arm/Group Description:

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant

Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant

Reduced Intensity Conditioning

Overall Number of Participants Analyzed 22
Median (Full Range)
Unit of Measure: days
48
(33 to 144)
Time Frame [Not Specified]
Adverse Event Reporting Description Adverse events that were expected and non-serious were not collected in this study.
 
Arm/Group Title RIC Cord Blood Transplant
Hide Arm/Group Description

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant

Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant

Reduced Intensity Conditioning

All-Cause Mortality
RIC Cord Blood Transplant
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
RIC Cord Blood Transplant
Affected / at Risk (%)
Total   18/22 (81.82%) 
Blood and lymphatic system disorders   
Auto Immune Hemolytic Anemia  2/22 (9.09%) 
Hemolytic Transfusion Reaction  1/22 (4.55%) 
Graft Failure Post Transplant  2/22 (9.09%) 
Deep Vein Thrombosis  1/22 (4.55%) 
Endocrine disorders   
Hyperglycemia  1/22 (4.55%) 
Gastrointestinal disorders   
Abdominal Pain  1/22 (4.55%) 
GI Distress  3/22 (13.64%) 
Pnematosis  1/22 (4.55%) 
General disorders   
Multi System Organ Failure  2/22 (9.09%) 
Graft Versus Host Disease  4/22 (18.18%) 
Infections and infestations   
Infection  12/22 (54.55%) 
Sepsis  3/22 (13.64%) 
Investigations   
Fever  5/22 (22.73%) 
Metabolism and nutrition disorders   
Dehydration  2/22 (9.09%) 
Nervous system disorders   
VP Shunt Malfunction  1/22 (4.55%) 
Seizure  1/22 (4.55%) 
Posterior Reversible Encephalopathy Syndrome  1/22 (4.55%) 
Renal and urinary disorders   
Nephrotic Syndrome  1/22 (4.55%) 
Respiratory, thoracic and mediastinal disorders   
Respiratory Distress  2/22 (9.09%) 
Respiratory Failure  1/22 (4.55%) 
Skin and subcutaneous tissue disorders   
Necrotic Ulcer (skin)  1/22 (4.55%) 
Surgical and medical procedures   
Central Line Removal  1/22 (4.55%) 
Dental Rehabilitation  1/22 (4.55%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
RIC Cord Blood Transplant
Affected / at Risk (%)
Total   0/22 (0.00%) 
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Dr. Suhag Parikh
Organization: Duke University Medical Center
Phone: 919-668-1100
Responsible Party: Duke University
ClinicalTrials.gov Identifier: NCT00744692     History of Changes
Other Study ID Numbers: Pro00008753
First Submitted: August 28, 2008
First Posted: September 1, 2008
Results First Submitted: July 23, 2014
Results First Posted: August 13, 2014
Last Update Posted: August 13, 2014