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Trial record 1 of 1 for:    NCT01147653
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A Randomized Study of Autologous Umbilical Cord Blood Reinfusion in Children With Cerebral Palsy

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ClinicalTrials.gov Identifier: NCT01147653
Recruitment Status : Completed
First Posted : June 22, 2010
Results First Posted : June 12, 2017
Last Update Posted : November 30, 2017
Sponsor:
Collaborator:
Information provided by (Responsible Party):

Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions: Cerebral Palsy
CP
Spastic Cerebral Palsy
Interventions: Biological: Autologous UCB Reinfusion
Other: Placebo

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Participants were enrolled and randomized on the same day.

Reporting Groups
  Description
Autologous UCB Reinfusion First, Then Placebo Subjects receive autologous umbilical cord blood at Baseline, then Placebo at Year 1.
Placebo First, Then Autologous UCB Reinfusion Subjects receive Placebo at Baseline, then autologous umbilical cord blood at Year 1.

Participant Flow for 2 periods

Period 1:   Year 1
    Autologous UCB Reinfusion First, Then Placebo   Placebo First, Then Autologous UCB Reinfusion
STARTED   32   31 
COMPLETED   32   31 
NOT COMPLETED   0   0 

Period 2:   Year 2
    Autologous UCB Reinfusion First, Then Placebo   Placebo First, Then Autologous UCB Reinfusion
STARTED   32   31 
COMPLETED   30   28 
NOT COMPLETED   2   3 
Withdrawal by Subject                2                3 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Autologous UCB First, Then Placebo Subjects receive autologous umbilical cord blood at Baseline, then Placebo at Year 1.
Placebo First, Then Autologous UCB Reinfusion Subjects receive Placebo at Baseline, then autologous umbilical cord blood at Year 1.
Total Total of all reporting groups

Baseline Measures
   Autologous UCB First, Then Placebo   Placebo First, Then Autologous UCB Reinfusion   Total 
Overall Participants Analyzed 
[Units: Participants]
 32   31   63 
Age 
[Units: Years]
Median (Full Range)
 2.1 
 (1.1 to 6.2) 
 2.3 
 (1.1 to 7.0) 
 2.3 
 (1.1 to 7.0) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Female      12  37.5%      9  29.0%      21  33.3% 
Male      20  62.5%      22  71.0%      42  66.7% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
     
Hispanic or Latino      3   9.4%      4  12.9%      7  11.1% 
Not Hispanic or Latino      29  90.6%      27  87.1%      56  88.9% 
Unknown or Not Reported      0   0.0%      0   0.0%      0   0.0% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
     
American Indian or Alaska Native      0   0.0%      0   0.0%      0   0.0% 
Asian      4  12.5%      3   9.7%      7  11.1% 
Native Hawaiian or Other Pacific Islander      0   0.0%      0   0.0%      0   0.0% 
Black or African American      1   3.1%      0   0.0%      1   1.6% 
White      27  84.4%      28  90.3%      55  87.3% 
More than one race      0   0.0%      0   0.0%      0   0.0% 
Unknown or Not Reported      0   0.0%      0   0.0%      0   0.0% 
GMFCS Level [1] 
[Units: Participants]
Count of Participants
     
Level I      11  34.4%      6  19.4%      17  27.0% 
Level II      9  28.1%      15  48.4%      24  38.1% 
Level III      4  12.5%      4  12.9%      8  12.7% 
Level IV      8  25.0%      6  19.4%      14  22.2% 
[1] The Gross Motor Function Classification System (GMFCS) classifies gross motor function of children with cerebral palsy into Levels I (least disabled) to V (most disabled). For example, children at Level I are able to walk at home, in school, and in the community and can climb stairs without a railing. At Level II children may walk with assistance and can climb stairs using a railing. At Level III children walk with handheld assistive devices for short distances but use a wheelchair for long distance mobility. Mobility at Levels IV and V require physical assistance or powered devices.
Type of Cerebral Palsy 
[Units: Participants]
Count of Participants
     
Spastic Diplegia      6  18.8%      6  19.4%      12  19.0% 
Spastic Hemiplegia      15  46.9%      15  48.4%      30  47.6% 
Spastic Quadriplegia      10  31.3%      7  22.6%      17  27.0% 
Hypotonic Quadriplegia      1   3.1%      3   9.7%      4   6.3% 


  Outcome Measures

1.  Primary:   Change in Gross Motor Function Measure 66 (GMFM-66) Score   [ Time Frame: Baseline to Year 1 ]

2.  Secondary:   Change in Peabody Gross Motor Quotient From Baseline to Year 1   [ Time Frame: Baseline to Year 1 ]

3.  Secondary:   Change in IT-QOL Questionnaire Score   [ Time Frame: Baseline to Year 1 ]

4.  Secondary:   Change in CP-QOL Score   [ Time Frame: Baseline, Year 1 ]

5.  Secondary:   Change in Whole Brain Connectivity Measured by Diffusion Tensor Magnetic Resonance Imaging (MRI)   [ Time Frame: Baseline to Year 1 ]

6.  Secondary:   Change in Loes Score of Functional MRI From Baseline to Year 1 and From Year 1 to Year 2   [ Time Frame: Baseline, Year 1, Year 2 ]

7.  Secondary:   Change in Bayley Scales of Infant and Toddler Development-III, Cognitive Composite From Baseline to Year 1   [ Time Frame: Baseline to Year 1 ]

8.  Secondary:   Change in Wechsler Preschool and Primary Scale of Intelligence (WPPSI) III Full Scale Intelligence Quotient (IQ) for Younger Children (Ages 2 Years & 6 Months to 3 Years & 11 Months) From Baseline to Year 1.   [ Time Frame: Baseline to Year 1 ]

9.  Secondary:   Change in the Wechsler Intelligence Scale for Children (WISC-IV) From Baseline to Year 1.   [ Time Frame: Baseline to Year 1 ]

10.  Secondary:   Change in Cognitive Z-Score From Baseline to Year 1   [ Time Frame: Baseline to Year 1 ]

11.  Secondary:   Change in Assisting Hand Assessment (AHA) Score From Baseline to Year 1   [ Time Frame: Baseline to Year 1 ]

12.  Secondary:   Change in Pediatric Evaluation of Disability (PEDI) Self Care Score   [ Time Frame: Baseline to Year 1 ]

13.  Secondary:   Change in Pediatric Evaluation of Disability (PEDI) Mobility Score   [ Time Frame: Baseline to Year 1 ]

14.  Secondary:   Change in Pediatric Evaluation of Disability (PEDI) Social Function Score   [ Time Frame: Baseline to Year 1 ]

15.  Secondary:   Change in Child Behavior Checklist (CBCL) Z-score Internalizing Problems From Baseline to Year 1   [ Time Frame: Baseline to Year 1 ]

16.  Secondary:   Change in Child Behavior Checklist (CBCL) Z-score Externalizing Problems From Baseline to Year 1   [ Time Frame: Baseline to Year 1 ]

17.  Secondary:   Change in Child Behavior Checklist (CBCL) Z-score Total Problems From Baseline to Year 1   [ Time Frame: Baseline to Year 1 ]

18.  Secondary:   Change in Parental Distress From Baseline to Year 1   [ Time Frame: Baseline to Year 1 ]

19.  Secondary:   Change in Parent-Child Dysfunctional Interaction From Baseline to Year 1   [ Time Frame: Baseline to Year 1 ]

20.  Secondary:   Change in Difficult Child Score From Baseline to Year 1   [ Time Frame: Baseline to Year 1 ]

21.  Secondary:   Modified Ashworth Scale at Baseline   [ Time Frame: Baseline ]

22.  Secondary:   Modified Ashworth Scale at Year 1   [ Time Frame: Year 1 ]

23.  Secondary:   Change in Bruininks-Oseretsky-2 Total Motor Composite From Baseline to Year 1   [ Time Frame: Baseline to Year 1 ]

24.  Secondary:   Parent Experience of Child Illness   [ Time Frame: Baseline to Year 1 ]

25.  Other Pre-specified:   Change in Barry-Albright Dystonia Total Score From Baseline to Year 1   [ Time Frame: Baseline to Year 1 ]

26.  Post-Hoc:   Gross Motor Function Measure 66 (GMFM-66) Change Score at 1 Year by Infused Dose   [ Time Frame: 1 Year ]

27.  Post-Hoc:   Observed Minus Expected GMFM-66 Change Score of All Subjects Receiving Autologous UCB and Who Are Greater Than or Equal to 2 Years of Age   [ Time Frame: 1 year post-infusion with autologous umbilical cord blood ]

28.  Post-Hoc:   Peabody Gross Motor Quotient Change Score 1 Year After Receiving Autologous UCB.   [ Time Frame: 1 year post-infusion with autologous umbilical cord blood ]

29.  Post-Hoc:   Change in Whole Brain Connectivity Measured by Diffusion Tensor Magnetic Resonance Imaging (MRI) of All Subjects 1 Year Post-infusion With Autologous UCB   [ Time Frame: 1 year post-infusion with autologous umbilical cord blood ]

30.  Secondary:   Correlation Between Clinical Response and RNA Expression of Neural, Endothelial and Inflammatory Cytokines Measured by RNA Arrays in Cord Blood Cells Given to These Patients.   [ Time Frame: 2 years ]
Results not yet reported.   Anticipated Reporting Date:   01/2022  


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information

Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Jesse Troy, PhD
Organization: Duke University School of Medicine, Division of Pediatric Blood and Marrow Transplantation
e-mail: cordbloodtherapyinfo@dm.duke.edu



Responsible Party: Joanne Kurtzberg, MD, Duke University Medical Center
ClinicalTrials.gov Identifier: NCT01147653     History of Changes
Other Study ID Numbers: Pro00017801
First Submitted: June 17, 2010
First Posted: June 22, 2010
Results First Submitted: March 15, 2017
Results First Posted: June 12, 2017
Last Update Posted: November 30, 2017