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Safety and Effectiveness of Banked Cord Blood or Bone Marrow Stem Cells in Children With Cerebral Palsy (CP). (ACT for CP)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01988584
Recruitment Status : Completed
First Posted : November 20, 2013
Results First Posted : November 21, 2022
Last Update Posted : November 21, 2022
Sponsor:
Collaborators:
Cord Blood Registry (CBR)
Let's Cure CP Foundation
Mission Connect, a program of TIRR Foundation
Information provided by (Responsible Party):
Charles Cox, The University of Texas Health Science Center, Houston

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Triple (Participant, Care Provider, Outcomes Assessor);   Primary Purpose: Treatment
Condition Cerebral Palsy
Interventions Biological: umbilical cord blood (hUCB) cells
Drug: Saline Infusion (Placebo)
Biological: bone marrow derived mononuclear cells (BMMNCs)
Enrollment 20
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo), Then Umbilical Cord Blood (UCB) Cells Saline Infusion (Placebo), Then Bone Marrow-derived Mononuclear Cells (BMMNCs)
Hide Arm/Group Description

Children who have banked UCB with CBR will receive an umbilical cord blood stem cell infusion at the baseline/treatment visit.

umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.

Children in the BMMNC group will undergo bone marrow harvest and stem cell infusion at the baseline/treatment visit.

bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.

Five children in each group will be randomly assigned to receive an inactive substance (placebo) at the baseline/treatment visit. Parents will be given the opportunity to cross-over to either the umbilical cord blood or bone marrow harvest group at the one year visit.

umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.

Saline Infusion (Placebo): A total of 10 children (5 from each cohort) will be randomized to a placebo infusion at the baseline visit and then have the opportunity to cross-over to stem cell treatment at the 1yr. visit.

Saline Infusion (Placebo): A total of 10 children (5 from each cohort) will be randomized to a placebo infusion at the baseline visit and then have the opportunity to cross-over to stem cell treatment at the 1yr. visit.

bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
Period Title: Blinded Phase
Started 3 10 2 5
12 Month Follow-up 3 9 2 5
Completed 3 9 2 4 [1]
Not Completed 0 1 0 1
[1]
One BMMNC Subject Lost To Follow-up After 1yr. Cross-Over Visit
Period Title: Unblinded Phase - Placebo Arm Crossover
Started [1] 0 0 2 5
Completed [2] 0 0 2 4
Not Completed 0 0 0 1
[1]
Placebo arms cross over to stem cell intervention 12 months after start of study.
[2]
Crossover arms complete follow-up 12 months after start of stem cell intervention (which is 24 months after start of study).
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Total
Hide Arm/Group Description

Children who have banked UCB with CBR will receive an umbilical cord blood stem cell infusion at the baseline/treatment visit.

umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.

Children in the BMMNC group will undergo bone marrow harvest and stem cell infusion at the baseline/treatment visit.

bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.

 Total of all reporting groups
Overall Number of Baseline Participants 5 15 20
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants 15 participants 20 participants
<=18 years
5
 100.0%
15
 100.0%
20
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 5 participants 15 participants 20 participants
4  (2.12) 6.13  (2.92) 5.6  (2.85)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants 15 participants 20 participants
Female
2
  40.0%
6
  40.0%
8
  40.0%
Male
3
  60.0%
9
  60.0%
12
  60.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants 15 participants 20 participants
Hispanic or Latino
1
  20.0%
0
   0.0%
1
   5.0%
Not Hispanic or Latino
4
  80.0%
15
 100.0%
19
  95.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants 15 participants 20 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
2
  13.3%
2
  10.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
4
  80.0%
13
  86.7%
17
  85.0%
More than one race
1
  20.0%
0
   0.0%
1
   5.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
 Number Analyzed 5 participants 15 participants 20 participants
United States
5
 100.0%
13
  86.7%
18
  90.0%
United Kingdom
0
   0.0%
1
   6.7%
1
   5.0%
Italy
0
   0.0%
1
   6.7%
1
   5.0%
1.Primary Outcome
Title Safety as Assessed by Number of Participants With In-hospital Infusion Toxicity
Hide Description In-hospital infusion toxicity includes hemodynamic, pulmonary, hepatic, renal, or neurologic complications.
Time Frame 24 hours after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 5 15 7
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
0
   0.0%
2.Primary Outcome
Title Long-term Safety
Hide Description Long-term safety as assessed by number of participants who developed new mass lesions or other pathological structural changes or had worsening neurological status
Time Frame from the time of infusion to 1 year after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Two in the BMMNC arm were lost to follow up.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 5 13 7
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
0
   0.0%
3.Secondary Outcome
Title Number of Participants With an Improvement in White Matter Integrity.
Hide Description

Number of participants with an improvement in white matter integrity as measured by diffusion tensor imaging (DTI) magnetic resonance imaging (MRI) reconstruction of corticospinal tract (CST) radial diffusivity (RD).

Improvement is defined as radial diffusivity (RD) decreased in at least one corticospinal tract (CST) reconstruction.
Time Frame from baseline to 1 year after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Two in the BMMNC arm were lost to follow up.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 5 13 0
Measure Type: Count of Participants
Unit of Measure: Participants
5
 100.0%
9
  69.2%
 0
4.Secondary Outcome
Title Gross Motor Function Classification Score (GMFM-66)
Hide Description Interval age adjusted scaled scores from 0 to 100 with 0 reflecting a child with low motor ability and 100 indicating high motor ability.
Time Frame baseline before infusion
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 3 10 7
Mean (Standard Deviation)
Unit of Measure: score on a scale
43.17  (19.8) 25.61  (12.83) 23.24  (15.15)
5.Secondary Outcome
Title Gross Motor Function Classification Score (GMFM-66)
Hide Description Interval age adjusted scaled scores from 0 to 100 with 0 reflecting a child with low motor ability and 100 indicating high motor ability.
Time Frame 1 year after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Two in the BMMNC arm were lost to follow up.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 5 13 0
Mean (Standard Deviation)
Unit of Measure: score on a scale
36.5  (24.1) 25.95  (13.88)
6.Secondary Outcome
Title Gross Motor Function Classification Score (GMFM-88)
Hide Description Ordinal age adjusted scaled raw and percent scores from 0 to 100 with 0 reflecting a child with low motor ability and 100 indicating high motor ability.
Time Frame baseline before infusion
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 3 10 7
Mean (Standard Deviation)
Unit of Measure: score on a scale
50.67  (37.5) 24.72  (20.06) 18.06  (21.56)
7.Secondary Outcome
Title Gross Motor Function Classification Score (GMFM-88)
Hide Description Ordinal age adjusted scaled raw and percent scores from 0 to 100 with 0 reflecting a child with low motor ability and 100 indicating high motor ability.
Time Frame 1 year after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Two in the BMMNC arm were lost to follow up.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 5 13 0
Mean (Standard Deviation)
Unit of Measure: score on a scale
34.77  (40.38) 25.92  (19.83)
8.Secondary Outcome
Title Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication
Hide Description Standard score ranging from 20 to 140 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame baseline before infusion
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 3 10 7
Mean (Standard Deviation)
Unit of Measure: score on a scale
79  (16.09) 67.56  (21.04) 52.86  (15.95)
9.Secondary Outcome
Title Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication
Hide Description Standard score ranging from 20 to 140 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame 1 year after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Two in the BMMNC arm were lost to follow up.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 5 13 0
Mean (Standard Deviation)
Unit of Measure: score on a scale
64.4  (18.96) 62.23  (20.79)
10.Secondary Outcome
Title Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living
Hide Description Standard score ranging from 20 to 140 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame baseline before infusion
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 3 10 7
Mean (Standard Deviation)
Unit of Measure: score on a scale
67  (16.09) 57.44  (9.58) 49.14  (8.95)
11.Secondary Outcome
Title Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living
Hide Description Standard score ranging from 20 to 140 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame 1 year after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Two in the BMMNC arm were lost to follow up.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 5 13 0
Mean (Standard Deviation)
Unit of Measure: score on a scale
63  (23.16) 52.54  (13.2)
12.Secondary Outcome
Title Score on Vineland Adaptive Behavior Scales (VABS-2) - Social
Hide Description Standard score ranging from 20 to 140 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame baseline before infusion
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 3 10 7
Mean (Standard Deviation)
Unit of Measure: score on a scale
83  (13) 68.89  (19.59) 57.71  (7.13)
13.Secondary Outcome
Title Score on Vineland Adaptive Behavior Scales (VABS-2) - Social
Hide Description Standard score ranging from 20 to 140 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame 1 year after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Two in the BMMNC arm were lost to follow up. One BMMNC subject was untestable at this time point.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 5 12 0
Mean (Standard Deviation)
Unit of Measure: score on a scale
65  (16) 60.75  (16.84)
14.Secondary Outcome
Title Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor
Hide Description Standard score ranging from 20 to 140 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame baseline before infusion
Hide Outcome Measure Data
Hide Analysis Population Description
9 subjects were untestable.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 3 4 4
Mean (Standard Deviation)
Unit of Measure: score on a scale
58.67  (15.5) 43  (11.75) 38.5  (14.62)
15.Secondary Outcome
Title Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor
Hide Description Standard score ranging from 20 to 140 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame 1 year after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Two in the BMMNC arm were lost to follow up. Nine subjects were untestable at this time point.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 3 6 0
Mean (Standard Deviation)
Unit of Measure: score on a scale
52.33  (16.8) 40.67  (11.24)
16.Secondary Outcome
Title Score on Pediatric Evaluation of Disability Inventory - Self-Care
Hide Description Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame baseline before infusion
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 3 10 5
Mean (Standard Deviation)
Unit of Measure: score on a scale
35.4  (12.29) 43.31  (13.04) 27.36  (9.35)
17.Secondary Outcome
Title Score on Pediatric Evaluation of Disability Inventory - Self-Care
Hide Description Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame 1 year after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Two in the BMMNC arm were lost to follow up.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 5 13 0
Mean (Standard Deviation)
Unit of Measure: score on a scale
36.28  (13.97) 37.13  (16.35)
18.Secondary Outcome
Title Score on Pediatric Evaluation of Disability Inventory - Mobility
Hide Description Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame baseline before infusion
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 3 10 5
Mean (Standard Deviation)
Unit of Measure: score on a scale
46.83  (27.62) 33.31  (11.91) 26.54  (12.88)
19.Secondary Outcome
Title Score on Pediatric Evaluation of Disability Inventory - Mobility
Hide Description Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame 1 year after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Two in the BMMNC arm were lost to follow up.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 5 13 0
Mean (Standard Deviation)
Unit of Measure: score on a scale
39.66  (36.36) 34.16  (14.8)
20.Secondary Outcome
Title Score on Pediatric Evaluation of Disability Inventory - Social
Hide Description Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame baseline before infusion
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 3 10 5
Mean (Standard Deviation)
Unit of Measure: score on a scale
46.9  (7.82) 52  (12.02) 34.12  (16.15)
21.Secondary Outcome
Title Score on Pediatric Evaluation of Disability Inventory - Social
Hide Description Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame 1 year after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Two in the BMMNC arm were lost to follow up.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 5 13 0
Mean (Standard Deviation)
Unit of Measure: score on a scale
44.54  (9.29) 47.32  (14.82)
22.Secondary Outcome
Title Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall
Hide Description Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.
Time Frame baseline before infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Data were not collected for the 5 in the UCB cell arm and 4 in the placebo arm. Untestable with this instrument.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 0 10 1
Mean (Standard Deviation)
Unit of Measure: score on a scale
8.33  (0.58) 6  (0)
23.Secondary Outcome
Title Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall
Hide Description Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.
Time Frame 1 year after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Only 4 subjects were testable at this time point.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 1 3 0
Mean (Standard Deviation)
Unit of Measure: score on a scale
6  (0) 5  (1)
24.Secondary Outcome
Title Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued
Hide Description Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.
Time Frame baseline before infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Only 4 subjects were testable at this time point.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 0 3 1
Mean (Standard Deviation)
Unit of Measure: score on a scale
5.67  (1.53) 6  (0)
25.Secondary Outcome
Title Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued
Hide Description Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.
Time Frame 1 year after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Only 3 subjects were testable at this time point.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 0 3 0
Mean (Standard Deviation)
Unit of Measure: score on a scale
4  (0)
26.Secondary Outcome
Title Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4)
Hide Description Standard score ranging from 20 to 160. Higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame baseline before infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Only 7 subjects were testable with this instrument.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 2 4 1
Mean (Standard Deviation)
Unit of Measure: score on a scale
80  (26.87) 85  (13.34) 89  (0)
27.Secondary Outcome
Title Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4)
Hide Description Standard score ranging from 20 to 160. Higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame 1 year after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Only 6 subjects were testable at this time point.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 2 4 0
Mean (Standard Deviation)
Unit of Measure: score on a scale
56.5  (21.92) 86  (11.58)
28.Secondary Outcome
Title Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family
Hide Description Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame baseline before infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Only 7 subjects were testable with this instrument.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 1 4 2
Mean (Standard Deviation)
Unit of Measure: score on a scale
87.5  (0) 75.78  (15.39) 75  (36.36)
29.Secondary Outcome
Title Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family
Hide Description Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame 1 year after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Two in the BMMNC arm were lost to follow up.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 4 10 0
Mean (Standard Deviation)
Unit of Measure: score on a scale
79.69  (22.89) 72.81  (17.74)
30.Secondary Outcome
Title Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social
Hide Description Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame baseline before infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Only 8 subjects were testable with this instrument
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 1 4 3
Mean (Standard Deviation)
Unit of Measure: score on a scale
88.64  (0) 75.57  (9.75) 83.71  (15.01)
31.Secondary Outcome
Title Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social
Hide Description Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame 1 year after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Two in the BMMNC arm were lost to follow up. Five subjects were untestable.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 4 10 0
Mean (Standard Deviation)
Unit of Measure: score on a scale
87.7  (8.67) 74.77  (9.21)
32.Secondary Outcome
Title Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings
Hide Description Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame baseline before infusion
Hide Outcome Measure Data
Hide Analysis Population Description
13 subjects were testable with this instrument.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 0 10 3
Mean (Standard Deviation)
Unit of Measure: score on a scale
60.16  (6.1) 48.96  (2.76)
33.Secondary Outcome
Title Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings
Hide Description Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame 1 year after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Two in the BMMNC arm were lost to follow up. Five subjects were untestable.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 3 10 0
Mean (Standard Deviation)
Unit of Measure: score on a scale
67.01  (26.85) 55.73  (9.68)
34.Secondary Outcome
Title Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation
Hide Description Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame baseline before infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Only 8 subjects were testable with this instrument.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 1 4 3
Mean (Standard Deviation)
Unit of Measure: score on a scale
72.73  (0) 56.82  (8.25) 56.82  (17.71)
35.Secondary Outcome
Title Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation
Hide Description Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame 1 year after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Two in the BMMNC arm were lost to follow up. Five subjects were untestable at this time point.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 4 9 0
Mean (Standard Deviation)
Unit of Measure: score on a scale
69.88  (18.57) 55.81  (8.74)
36.Secondary Outcome
Title Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional
Hide Description Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame baseline before infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Only 8 subjects were testable with this instrument.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 1 4 3
Mean (Standard Deviation)
Unit of Measure: score on a scale
85.42  (0) 75.52  (5.98) 69.44  (6.7)
37.Secondary Outcome
Title Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional
Hide Description Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame 1 year after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Two in the BMMNC arm were lost to follow up. Five other subjects were untestable at this time point.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 4 10 0
Mean (Standard Deviation)
Unit of Measure: score on a scale
78.12  (15.73) 70.83  (8.56)
38.Secondary Outcome
Title Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access
Hide Description Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame baseline before infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Only 8 subjects were testable with this instrument.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 1 4 3
Mean (Standard Deviation)
Unit of Measure: score on a scale
47.5  (0) 63.75  (10.31) 80.83  (17.02)
39.Secondary Outcome
Title Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access
Hide Description Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame 1 year after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Two in the BMMNC arm were lost to follow up. Five subjects were untestable at this time point.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 4 10 0
Mean (Standard Deviation)
Unit of Measure: score on a scale
72.5  (29.01) 65.5  (10.26)
40.Secondary Outcome
Title Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain
Hide Description Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame baseline before infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Only 7 subjects were testable with this instrument.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 1 4 2
Mean (Standard Deviation)
Unit of Measure: score on a scale
3.13  (0) 40.63  (20.53) 35.94  (19.88)
41.Secondary Outcome
Title Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain
Hide Description Standard scores ranging from 0 to 100 with higher scores indicate a child with a higher level of functioning and lower scores indicate a lower level of functioning.
Time Frame 1 year after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Two in the BMMNC arm were lost to follow up. Eight subjects were untestable at this time point.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 2 9 0
Mean (Standard Deviation)
Unit of Measure: score on a scale
22.39  (20.82) 34.38  (21.26)
42.Secondary Outcome
Title Visual-Spatial Processing as Assessed by Score on the Motor-Free Visual Perception Test (MVPT-3)
Hide Description Standard scores ranging from 55 to 145 with higher scores indicating better outcomes and lower scores poorer outcomes.
Time Frame baseline before infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Only three subjects were testable with this instrument.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 1 2 0
Mean (Standard Deviation)
Unit of Measure: score on a scale
55  (0) 55  (0)
43.Secondary Outcome
Title Visual-Spatial Processing as Assessed by Score on the Motor-Free Visual Perception Test (MVPT-3)
Hide Description Standard scores ranging from 55 to 145 with higher scores indicating better outcomes and lower scores poorer outcomes.
Time Frame 1 year after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Data were not collected for the 5 in the UCB cell arm.
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description:
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
saline infusion (placebo): inactive substance
Overall Number of Participants Analyzed 0 2 0
Mean (Standard Deviation)
Unit of Measure: score on a scale
63  (11.31)
Time Frame 1 year
Adverse Event Reporting Description Serious Adverse Events (SAEs) were defined using the the Common Terminology Criteria for Adverse Events (CTCAE) V5.0. All SAEs and AEs are reported, and all SAEs and AEs were deemed to be unrelated to the stem cell intervention.
 
Arm/Group Title Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Hide Arm/Group Description umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry. bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest. saline infusion (placebo): inactive substance
All-Cause Mortality
Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/5 (0.00%)      0/15 (0.00%)      0/7 (0.00%)    
Hide Serious Adverse Events
Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/5 (40.00%)      3/15 (20.00%)      0/7 (0.00%)    
Infections and infestations       
Upper Tract Respiratory Infection † 1  0/5 (0.00%)  0 2/15 (13.33%)  2 0/7 (0.00%)  0
Pneumonia † 1  0/5 (0.00%)  0 1/15 (6.67%)  1 0/7 (0.00%)  0
Nervous system disorders       
Febrile Convulsion (Febrile Seizure) † 1  1/5 (20.00%)  1 0/15 (0.00%)  0 0/7 (0.00%)  0
Surgical and medical procedures       
Osteotomy (Planned Hip Osteotomy) † 1  1/5 (20.00%)  1 0/15 (0.00%)  0 0/7 (0.00%)  0
1
Term from vocabulary, MedDra SOC
†
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Umbilical Cord Blood (UCB) Cells Bone Marrow-derived Mononuclear Cells (BMMNCs) Saline Infusion (Placebo)
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/5 (20.00%)      8/15 (53.33%)      3/7 (42.86%)    
Gastrointestinal disorders       
Haematochezia (Bloody Stools/Chrohn's Disease) † 1  0/5 (0.00%)  0 0/15 (0.00%)  0 1/7 (14.29%)  1
Infections and infestations       
Gastroenteritis (Stomach Flu) † 1  0/5 (0.00%)  0 2/15 (13.33%)  3 0/7 (0.00%)  0
Ear Infection † 1  0/5 (0.00%)  0 2/15 (13.33%)  2 0/7 (0.00%)  0
Pneumonia † 1  1/5 (20.00%)  1 0/15 (0.00%)  0 0/7 (0.00%)  0
Croup Infectious † 1  1/5 (20.00%)  1 0/15 (0.00%)  0 0/7 (0.00%)  0
Upper Respiratory Tract Infection † 1  1/5 (20.00%)  1 2/15 (13.33%)  2 0/7 (0.00%)  0
Respiratory Tract Infection † 1  0/5 (0.00%)  0 0/15 (0.00%)  0 1/7 (14.29%)  2
Nervous system disorders       
Seizures (More Frequent Seizures) † 1  1/5 (20.00%)  1 1/15 (6.67%)  1 1/7 (14.29%)  1
Respiratory, thoracic and mediastinal disorders       
Epistaxis (Nose Bleed) † 1  0/5 (0.00%)  0 0/15 (0.00%)  0 1/7 (14.29%)  1
Nasal Congestion † 1  1/5 (20.00%)  1 1/15 (6.67%)  1 0/7 (0.00%)  0
Skin and subcutaneous tissue disorders       
Vitiligo † 1  0/5 (0.00%)  0 1/15 (6.67%)  1 0/7 (0.00%)  0
1
Term from vocabulary, MedDra SOC
†
Indicates events were collected by systematic assessment
Limitations common to treating heterogeneous conditions and to cell based therapies. First, patients enrolled had wide range of clinical and imaging findings, which made pooling small data sample problematic. Imaging studies are problematic with hemispheric volume loss secondary to in utero stroke, etc. Second, variability in available cord blood doses. Due to a competing trial, few US families with banked autologous units and not already treated. Unable to reach target enrollment.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Charles S. Cox, MD, Professor
Organization: The University of Texas Health Science Center at Houston
Phone: 713-500-7300
EMail: charles.s.cox@uth.tmc.edu
Layout table for additonal information
Responsible Party: Charles Cox, The University of Texas Health Science Center, Houston
ClinicalTrials.gov Identifier: NCT01988584    
Other Study ID Numbers: HSC-MS-12-0876
First Submitted: November 4, 2013
First Posted: November 20, 2013
Results First Submitted: October 29, 2020
Results First Posted: November 21, 2022
Last Update Posted: November 21, 2022