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Evaluating the Safety and Effectiveness of Bone Marrow Transplants in Children With Sickle Cell Disease (BMT CTN 0601) (SCURT)

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ClinicalTrials.gov Identifier: NCT00745420
Recruitment Status : Completed
First Posted : September 3, 2008
Results First Posted : December 26, 2017
Last Update Posted : May 10, 2018
Sponsor:
Collaborators:
National Heart, Lung, and Blood Institute (NHLBI)
Blood and Marrow Transplant Clinical Trials Network
National Cancer Institute (NCI)
National Marrow Donor Program
Sickle Cell Disease Clinical Research Network
Information provided by (Responsible Party):
Medical College of Wisconsin

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Sickle Cell Disease
Intervention Biological: Hematopoietic Stem Cell Transplantation
Enrollment 30

Recruitment Details Participants were enrolled between August 2008 and April 2014 from 19 different transplant centers.
Pre-assignment Details  
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description Bone Marrow Transplant with GVHD Prophylaxis Regimen
Period Title: Overall Study
Started 30
Completed 29
Not Completed 1
Reason Not Completed
Ineligible             1
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description Bone Marrow Transplant with GVHD Prophylaxis Regimen
Overall Number of Baseline Participants 29
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 29 participants
14
(6 to 19)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 29 participants
Female
13
  44.8%
Male
16
  55.2%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 29 participants
Black or African American
26
  89.7%
Hispanic or Latino
3
  10.3%
Indications for Transplant   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 29 participants
Stroke
12
  41.4%
Trans cranial doppler velocity > 200cm/s
2
   6.9%
Acute chest syndrome
4
  13.8%
Vaso-occlusive pain crisis
12
  41.4%
[1]
Measure Description: Some patients had more than 1 indication
Chronic Blood Transfusion Prior to Transplant  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 29 participants
14
  48.3%
Karnofsky Performance Score   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 29 participants
100
17
  58.6%
90
9
  31.0%
80
2
   6.9%
70
1
   3.4%
[1]
Measure Description: Assesses patient self-perceived global quality of life and functioning on a scale of 0 - 100; where 100 equals normal quality of life with no evidence of disease, 90 equals ability to carry on normal activity with minor signs/symptoms of disease, 80 equals normal activity with effort and some signs/symptoms of disease, and 70 equals ability to care for oneself but unable to carry on normal activity or to do active work.
Cytomegalovirus (CMV) Status  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 29 participants
CMV - donor and recipient
8
  27.6%
CMV + donor and recipient
9
  31.0%
CMV - donor and CMV + recipient
3
  10.3%
CMV + donor and CMV - recipient
9
  31.0%
Donor Blood Type Match  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 29 participants
ABO Blood group matched
15
  51.7%
ABO Blood group major mismatch
9
  31.0%
ABO Blood group minor mismatch
5
  17.2%
Donor Gender Match  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 29 participants
Gender matched transplants
15
  51.7%
Female donor, male recipient
7
  24.1%
Male donor, female recipient
7
  24.1%
Donor Age  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 29 participants
35
(21 to 55)
Donor Race/Ethnicity  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 29 participants
Caucasian
11
  37.9%
Black or African American
11
  37.9%
Multiracial
5
  17.2%
Not Reported
2
   6.9%
1.Primary Outcome
Title Percentage of Participants With Event-Free Survival (EFS)
Hide Description EFS is defined as percentage of participants that have not had an event. Primary or secondary graft rejection, disease recurrence, or death will count as events for this endpoint.
Time Frame 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description:
Bone Marrow Transplant with GVHD Prophylaxis Regimen
Overall Number of Participants Analyzed 29
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
1 year
76
(56 to 88)
2 years
69
(48 to 82)
2.Secondary Outcome
Title Percentage of Participants With Overall Survival (OS)
Hide Description OS is defined as the percentage of participants that have not died.
Time Frame 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description:
Bone Marrow Transplant with GVHD Prophylaxis Regimen
Overall Number of Participants Analyzed 29
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
1 year
86
(67 to 95)
2 years
79
(59 to 90)
3.Secondary Outcome
Title Neutrophil and Platelet Recovery
Hide Description Time to neutrophil recovery is defined as the time of the first of three measurements on consecutive days where the patient has an absolute neutrophil count of >= 500/uL following conditioning regimen induced nadir. Time to platelet recovery is defined as the time of the first of three measurements on consecutive days where the patient has achieved a platelet count > 50,000/uL and is platelet transfusion independent for a minimum of seven days following conditioning regimen induced nadir.
Time Frame Up to 100 days
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description:
Bone Marrow Transplant with GVHD Prophylaxis Regimen
Overall Number of Participants Analyzed 29
Median (Full Range)
Unit of Measure: days
Neutrophil Recovery
12
(6 to 16)
Platelet Recovery
24
(7 to 90)
4.Secondary Outcome
Title Graft Rejection
Hide Description Primary graft rejection is defined as the presence of less than 20% donor cells as assessed by peripheral blood or bone marrow chimerism assays on or after Day 42. Secondary graft rejection is defined as the presence of less than 20% donor derived hematopoietic cells in peripheral blood or bone marrow that occurs after prior evidence of 20% or greater donor cells.
Time Frame 1 year
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description:
Bone Marrow Transplant with GVHD Prophylaxis Regimen
Overall Number of Participants Analyzed 29
Measure Type: Count of Participants
Unit of Measure: Participants
Primary Graft Rejection
2
   6.9%
Secondary Graft Rejection
1
   3.4%
5.Secondary Outcome
Title Percentage of Participants With Acute Graft-vs-Host-Disease (GVHD)
Hide Description

Acute GVHD is graded according to the scoring system proposed by Przepiorka et al.1995:

Skin stage:

0: No rash

  1. Rash <25% of body surface area
  2. Rash on 25-50% of body surface area
  3. Rash on > 50% of body surface area
  4. Generalized erythroderma with bullous formation

Liver stage (based on bilirubin level)*:

0: <2 mg/dL

  1. 2-3 mg/dL
  2. 3.01-6 mg/dL
  3. 6.01-15.0 mg/dL
  4. >15 mg/dL

GI stage*:

0: No diarrhea or diarrhea <500 mL/day

  1. Diarrhea 500-999 mL/day or persistent nausea with histologic evidence of GVHD
  2. Diarrhea 1000-1499 mL/day
  3. Diarrhea >1500 mL/day
  4. Severe abdominal pain with or without ileus * If multiple etiologies are listed for liver or GI, the organ system is downstaged by 1.

GVHD grade:

0: All organ stages 0 or GVHD not listed as an etiology I: Skin stage 1-2 and liver and GI stage 0 II: Skin stage 3 or liver or GI stage 1 III: Liver stage 2-3 or GI stage 2-4 IV: Skin or liver stage 4

Time Frame 100 days
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description:
Bone Marrow Transplant with GVHD Prophylaxis Regimen
Overall Number of Participants Analyzed 29
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
Grade II-IV Acute GVHD
28
(13 to 45)
Grade III-IV Acute GVHD
17
(6 to 33)
6.Secondary Outcome
Title Percentage of Participants With Chronic GVHD
Hide Description Chronic GVHD is defined per NIH 2005 Consensus Criteria.
Time Frame 1 year post-transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description:
Bone Marrow Transplant with GVHD Prophylaxis Regimen
Overall Number of Participants Analyzed 29
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
62
(41 to 77)
7.Secondary Outcome
Title Number of Participants With Chronic GVHD by Severity
Hide Description Chronic GVHD severity is defined per NIH 2005 Consensus Criteria.
Time Frame 1 year post-transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description:
Bone Marrow Transplant with GVHD Prophylaxis Regimen
Overall Number of Participants Analyzed 29
Measure Type: Count of Participants
Unit of Measure: Participants
None
10
  34.5%
Mild
6
  20.7%
Moderate
8
  27.6%
Severe
5
  17.2%
8.Secondary Outcome
Title Percentage of Participants With Posterior Reversible Encephalopathy Syndrome (PRES)
Hide Description [Not Specified]
Time Frame 1 year
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description:
Bone Marrow Transplant with GVHD Prophylaxis Regimen
Overall Number of Participants Analyzed 29
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
34
(18 to 52)
9.Secondary Outcome
Title Change From Baseline to Day 100 in Participant Reported Health-Related Quality of Life (HRQL)
Hide Description HRQL will be assessed using the Self-Esteem, General Health Perception, and Change in Health subscales of the Child Health Questionnaire (CHQ Child Form 87). The changes in participant reported scores on these HRQL subscales from a pre-transplant baseline assessment to day 100 post-transplant will be evaluated. Each subscale is scored is scored in the range 0-100, with higher scores indicating better health and well-being. Therefore, a negative mean change in score denotes worsening HRQL score and positive mean change in score denotes an improved HRQL score over time.
Time Frame 100 days post-transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants age 10 years or older that returned the HRQL surveys
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description:
Bone Marrow Transplant with GVHD Prophylaxis Regimen
Overall Number of Participants Analyzed 18
Mean (Standard Error)
Unit of Measure: units on a scale
Self esteem score -0.59  (4.51)
General health perception score -1.90  (4.29)
Change in health score 0.33  (0.45)
10.Secondary Outcome
Title Change From Baseline to Day 100 in Parent Proxy Reported Health-Related Quality of Life (HRQL)
Hide Description HRQL will be assessed using the Self-Esteem, General Health Perception, and Change in Health subscales of the Child Health Questionnaire (CHQ Child Form 87). The changes in parent proxy reported scores on these HRQL subscales from a pre-transplant baseline assessment to day 100 post-transplant will be evaluated. Each subscale is scored is scored in the range 0-100, with higher scores indicating better health and well-being. Therefore, a negative mean change in score denotes worsening HRQL score and positive mean change in score denotes an improved HRQL score over time.
Time Frame 100 days post-transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Parent proxies of participants that returned the HRQL surveys
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description:
Bone Marrow Transplant with GVHD Prophylaxis Regimen
Overall Number of Participants Analyzed 21
Mean (Standard Error)
Unit of Measure: units on a scale
Self esteem scale score -15.12  (4.89)
General health perception score 11.13  (2.51)
Change in health score 0.90  (0.38)
11.Secondary Outcome
Title Change From Baseline to Day 180 in Participant Reported Health-Related Quality of Life (HRQL)
Hide Description HRQL will be assessed using the Self-Esteem, General Health Perception, and Change in Health subscales of the Child Health Questionnaire (CHQ Child Form 87). The changes in participant reported scores on these HRQL subscales from a pre-transplant baseline assessment to day 180 post-transplant will be evaluated. Each subscale is scored is scored in the range 0-100, with higher scores indicating better health and well-being. Therefore, a negative mean change in score denotes worsening HRQL score and positive mean change in score denotes an improved HRQL score over time.
Time Frame 180 days post-transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants age 10 years or older that returned the HRQL surveys
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description:
Bone Marrow Transplant with GVHD Prophylaxis Regimen
Overall Number of Participants Analyzed 16
Mean (Standard Error)
Unit of Measure: units on a scale
Self esteem scale score -4.33  (5.87)
General health perception score -6.69  (4.99)
Change in health score 0.19  (0.52)
12.Secondary Outcome
Title Change From Baseline to Day 180 in Parent Proxy Reported Health-Related Quality of Life (HRQL)
Hide Description HRQL will be assessed using the Self-Esteem, General Health Perception, and Change in Health subscales of the Child Health Questionnaire (CHQ Child Form 87). The changes in parent proxy reported scores on these HRQL subscales from a pre-transplant baseline assessment to day 180 post-transplant will be evaluated. Each subscale is scored is scored in the range 0-100, with higher scores indicating better health and well-being. Therefore, a negative mean change in score denotes worsening HRQL score and positive mean change in score denotes an improved HRQL score over time.
Time Frame 180 days post-transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Parent proxies of participants that returned the HRQL surveys
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description:
Bone Marrow Transplant with GVHD Prophylaxis Regimen
Overall Number of Participants Analyzed 20
Mean (Standard Error)
Unit of Measure: units on a scale
Self esteem scale score -11.46  (6.28)
General health perception score 7.63  (3.31)
Change in health score 1.15  (0.36)
13.Secondary Outcome
Title Change From Baseline to 1 Year in Participant Reported Health-Related Quality of Life (HRQL)
Hide Description HRQL will be assessed using the Self-Esteem, General Health Perception, and Change in Health subscales of the Child Health Questionnaire (CHQ Child Form 87). The changes in participant reported scores on these HRQL subscales from a pre-transplant baseline assessment to 1 year post-transplant will be evaluated. Each subscale is scored is scored in the range 0-100, with higher scores indicating better health and well-being. Therefore, a negative mean change in score denotes worsening HRQL score and positive mean change in score denotes an improved HRQL score over time.
Time Frame 1 year post-transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants age 10 years or older that returned the HRQL surveys
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description:
Bone Marrow Transplant with GVHD Prophylaxis Regimen
Overall Number of Participants Analyzed 13
Mean (Standard Error)
Unit of Measure: units on a scale
Self esteem scale score 4.97  (5.33)
General health perception score 6.99  (6.35)
Change in health score 1.46  (0.35)
14.Secondary Outcome
Title Change From Baseline to 1 Year in Parent Proxy Reported Health-Related Quality of Life (HRQL)
Hide Description HRQL will be assessed using the Self-Esteem, General Health Perception, and Change in Health subscales of the Child Health Questionnaire (CHQ Child Form 87). The changes in parent proxy reported scores on these HRQL subscales from a pre-transplant baseline assessment to 1 year post-transplant will be evaluated. Each subscale is scored is scored in the range 0-100, with higher scores indicating better health and well-being. Therefore, a negative mean change in score denotes worsening HRQL score and positive mean change in score denotes an improved HRQL score over time.
Time Frame 1 year post-transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Parent proxies of participants that returned the HRQL surveys
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description:
Bone Marrow Transplant with GVHD Prophylaxis Regimen
Overall Number of Participants Analyzed 15
Mean (Standard Error)
Unit of Measure: units on a scale
Self esteem scale score -3.61  (6.66)
General health perception score 8.89  (5.27)
Change in health score 1.53  (0.48)
Time Frame 2 years post-transplant
Adverse Event Reporting Description Serious Adverse Events (AE) are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event. Only unexpected grades 3-5 adverse events were required to be reported through the AE system per protocol.
 
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description Bone Marrow Transplant with GVHD Prophylaxis Regimen
All-Cause Mortality
Hematopoietic Stem Cell Transplantation
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Hematopoietic Stem Cell Transplantation
Affected / at Risk (%) # Events
Total   21/29 (72.41%)    
Blood and lymphatic system disorders   
Autoimmune haemolytic anaemia * 1  1/29 (3.45%)  1
Cardiac disorders   
Cardiac arrest * 1  1/29 (3.45%)  1
Gastrointestinal disorders   
Pancreatitis * 1  1/29 (3.45%)  1
General disorders   
Death * 1  1/29 (3.45%)  1
Infections and infestations   
Sepsis * 1  1/29 (3.45%)  1
Septic shock * 1  1/29 (3.45%)  1
Staphylococcal sepsis * 1  1/29 (3.45%)  1
Investigations   
Oxygen saturation decreased * 1  1/29 (3.45%)  1
Nervous system disorders   
Cerebrovascular accident * 1  1/29 (3.45%)  1
Depressed level of consciousness * 1  1/29 (3.45%)  1
Posterior reversible encephalopathy syndrome * 1  2/29 (6.90%)  2
Seizure * 1  1/29 (3.45%)  1
Somnolence * 1  1/29 (3.45%)  1
Psychiatric disorders   
Mental status changes * 1  1/29 (3.45%)  1
Renal and urinary disorders   
Acute kidney injury * 1  1/29 (3.45%)  1
Respiratory, thoracic and mediastinal disorders   
Pulmonary embolism * 1  1/29 (3.45%)  1
Respiratory failure * 1  2/29 (6.90%)  2
Skin and subcutaneous tissue disorders   
Rash macular * 1  1/29 (3.45%)  1
Surgical and medical procedures   
Cholecystectomy * 1  1/29 (3.45%)  1
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA (12.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 2%
Hematopoietic Stem Cell Transplantation
Affected / at Risk (%) # Events
Total   2/29 (6.90%)    
Nervous system disorders   
Seizure * 1  1/29 (3.45%)  1
Respiratory, thoracic and mediastinal disorders   
Respiratory failure * 1  1/29 (3.45%)  1
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA (12.0)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Adam Mendizabal, PhD
Organization: The Emmes Corporation
Phone: 301-251-1161
Responsible Party: Medical College of Wisconsin
ClinicalTrials.gov Identifier: NCT00745420     History of Changes
Other Study ID Numbers: BMTCTN0601
U01HL069294 ( U.S. NIH Grant/Contract )
5U24CA076518 ( U.S. NIH Grant/Contract )
First Submitted: August 29, 2008
First Posted: September 3, 2008
Results First Submitted: August 23, 2017
Results First Posted: December 26, 2017
Last Update Posted: May 10, 2018