Trial Outcomes & Findings for Safety and Effectiveness of Banked Cord Blood or Bone Marrow Stem Cells in Children With Cerebral Palsy (CP). (NCT NCT01988584)
NCT ID: NCT01988584
Last Updated: 2022-11-21
Results Overview
In-hospital infusion toxicity includes hemodynamic, pulmonary, hepatic, renal, or neurologic complications.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
20 participants
Primary outcome timeframe
24 hours after infusion
Results posted on
2022-11-21
Participant Flow
Participant milestones
| Measure |
Umbilical Cord Blood (UCB) Cells
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.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
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.
|
Saline Infusion (Placebo), Then Umbilical Cord Blood (UCB) Cells
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), Then Bone Marrow-derived Mononuclear Cells (BMMNCs)
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.
|
|---|---|---|---|---|
|
Blinded Phase
COMPLETED
|
3
|
9
|
2
|
4
|
|
Blinded Phase
NOT COMPLETED
|
0
|
1
|
0
|
1
|
|
Blinded Phase
STARTED
|
3
|
10
|
2
|
5
|
|
Blinded Phase
12 Month Follow-up
|
3
|
9
|
2
|
5
|
|
Unblinded Phase - Placebo Arm Crossover
STARTED
|
0
|
0
|
2
|
5
|
|
Unblinded Phase - Placebo Arm Crossover
COMPLETED
|
0
|
0
|
2
|
4
|
|
Unblinded Phase - Placebo Arm Crossover
NOT COMPLETED
|
0
|
0
|
0
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety and Effectiveness of Banked Cord Blood or Bone Marrow Stem Cells in Children With Cerebral Palsy (CP).
Baseline characteristics by cohort
| Measure |
Umbilical Cord Blood (UCB) Cells
n=5 Participants
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.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=15 Participants
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
n=20 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
5 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
4 years
STANDARD_DEVIATION 2.12 • n=5 Participants
|
6.13 years
STANDARD_DEVIATION 2.92 • n=7 Participants
|
5.6 years
STANDARD_DEVIATION 2.85 • n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
4 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
19 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
4 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
5 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
Italy
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 24 hours after infusionIn-hospital infusion toxicity includes hemodynamic, pulmonary, hepatic, renal, or neurologic complications.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=5 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=15 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
n=7 Participants
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Safety as Assessed by Number of Participants With In-hospital Infusion Toxicity
|
0 Participants
|
0 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: from the time of infusion to 1 year after infusionPopulation: Two in the BMMNC arm were lost to follow up.
Long-term safety as assessed by number of participants who developed new mass lesions or other pathological structural changes or had worsening neurological status
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=5 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=13 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
n=7 Participants
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Long-term Safety
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: from baseline to 1 year after infusionPopulation: Two in the BMMNC arm were lost to follow up.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=5 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=13 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Number of Participants With an Improvement in White Matter Integrity.
|
5 Participants
|
9 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: baseline before infusionInterval age adjusted scaled scores from 0 to 100 with 0 reflecting a child with low motor ability and 100 indicating high motor ability.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=3 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=10 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
n=7 Participants
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Gross Motor Function Classification Score (GMFM-66)
|
43.17 score on a scale
Standard Deviation 19.8
|
25.61 score on a scale
Standard Deviation 12.83
|
23.24 score on a scale
Standard Deviation 15.15
|
SECONDARY outcome
Timeframe: 1 year after infusionPopulation: Two in the BMMNC arm were lost to follow up.
Interval age adjusted scaled scores from 0 to 100 with 0 reflecting a child with low motor ability and 100 indicating high motor ability.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=5 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=13 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Gross Motor Function Classification Score (GMFM-66)
|
36.5 score on a scale
Standard Deviation 24.1
|
25.95 score on a scale
Standard Deviation 13.88
|
—
|
SECONDARY outcome
Timeframe: baseline before infusionOrdinal 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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=3 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=10 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
n=7 Participants
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Gross Motor Function Classification Score (GMFM-88)
|
50.67 score on a scale
Standard Deviation 37.5
|
24.72 score on a scale
Standard Deviation 20.06
|
18.06 score on a scale
Standard Deviation 21.56
|
SECONDARY outcome
Timeframe: 1 year after infusionPopulation: Two in the BMMNC arm were lost to follow up.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=5 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=13 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Gross Motor Function Classification Score (GMFM-88)
|
34.77 score on a scale
Standard Deviation 40.38
|
25.92 score on a scale
Standard Deviation 19.83
|
—
|
SECONDARY outcome
Timeframe: baseline before infusionStandard 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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=3 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=10 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
n=7 Participants
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication
|
79 score on a scale
Standard Deviation 16.09
|
67.56 score on a scale
Standard Deviation 21.04
|
52.86 score on a scale
Standard Deviation 15.95
|
SECONDARY outcome
Timeframe: 1 year after infusionPopulation: Two in the BMMNC arm were lost to follow up.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=5 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=13 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication
|
64.4 score on a scale
Standard Deviation 18.96
|
62.23 score on a scale
Standard Deviation 20.79
|
—
|
SECONDARY outcome
Timeframe: baseline before infusionStandard 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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=3 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=10 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
n=7 Participants
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living
|
67 score on a scale
Standard Deviation 16.09
|
57.44 score on a scale
Standard Deviation 9.58
|
49.14 score on a scale
Standard Deviation 8.95
|
SECONDARY outcome
Timeframe: 1 year after infusionPopulation: Two in the BMMNC arm were lost to follow up.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=5 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=13 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living
|
63 score on a scale
Standard Deviation 23.16
|
52.54 score on a scale
Standard Deviation 13.2
|
—
|
SECONDARY outcome
Timeframe: baseline before infusionStandard 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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=3 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=10 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
n=7 Participants
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Vineland Adaptive Behavior Scales (VABS-2) - Social
|
83 score on a scale
Standard Deviation 13
|
68.89 score on a scale
Standard Deviation 19.59
|
57.71 score on a scale
Standard Deviation 7.13
|
SECONDARY outcome
Timeframe: 1 year after infusionPopulation: Two in the BMMNC arm were lost to follow up. One BMMNC subject was untestable at this time point.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=5 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=12 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Vineland Adaptive Behavior Scales (VABS-2) - Social
|
65 score on a scale
Standard Deviation 16
|
60.75 score on a scale
Standard Deviation 16.84
|
—
|
SECONDARY outcome
Timeframe: baseline before infusionPopulation: 9 subjects were untestable.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=3 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=4 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
n=4 Participants
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor
|
58.67 score on a scale
Standard Deviation 15.5
|
43 score on a scale
Standard Deviation 11.75
|
38.5 score on a scale
Standard Deviation 14.62
|
SECONDARY outcome
Timeframe: 1 year after infusionPopulation: Two in the BMMNC arm were lost to follow up. Nine subjects were untestable at this time point.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=3 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=6 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor
|
52.33 score on a scale
Standard Deviation 16.8
|
40.67 score on a scale
Standard Deviation 11.24
|
—
|
SECONDARY outcome
Timeframe: baseline before infusionStandard 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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=3 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=10 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
n=5 Participants
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Pediatric Evaluation of Disability Inventory - Self-Care
|
35.4 score on a scale
Standard Deviation 12.29
|
43.31 score on a scale
Standard Deviation 13.04
|
27.36 score on a scale
Standard Deviation 9.35
|
SECONDARY outcome
Timeframe: 1 year after infusionPopulation: Two in the BMMNC arm were lost to follow up.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=5 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=13 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Pediatric Evaluation of Disability Inventory - Self-Care
|
36.28 score on a scale
Standard Deviation 13.97
|
37.13 score on a scale
Standard Deviation 16.35
|
—
|
SECONDARY outcome
Timeframe: baseline before infusionStandard 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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=3 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=10 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
n=5 Participants
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Pediatric Evaluation of Disability Inventory - Mobility
|
46.83 score on a scale
Standard Deviation 27.62
|
33.31 score on a scale
Standard Deviation 11.91
|
26.54 score on a scale
Standard Deviation 12.88
|
SECONDARY outcome
Timeframe: 1 year after infusionPopulation: Two in the BMMNC arm were lost to follow up.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=5 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=13 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Pediatric Evaluation of Disability Inventory - Mobility
|
39.66 score on a scale
Standard Deviation 36.36
|
34.16 score on a scale
Standard Deviation 14.8
|
—
|
SECONDARY outcome
Timeframe: baseline before infusionStandard 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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=3 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=10 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
n=5 Participants
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Pediatric Evaluation of Disability Inventory - Social
|
46.9 score on a scale
Standard Deviation 7.82
|
52 score on a scale
Standard Deviation 12.02
|
34.12 score on a scale
Standard Deviation 16.15
|
SECONDARY outcome
Timeframe: 1 year after infusionPopulation: Two in the BMMNC arm were lost to follow up.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=5 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=13 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Pediatric Evaluation of Disability Inventory - Social
|
44.54 score on a scale
Standard Deviation 9.29
|
47.32 score on a scale
Standard Deviation 14.82
|
—
|
SECONDARY outcome
Timeframe: baseline before infusionPopulation: Data were not collected for the 5 in the UCB cell arm and 4 in the placebo arm. Untestable with this instrument.
Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=10 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
n=1 Participants
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall
|
—
|
8.33 score on a scale
Standard Deviation 0.58
|
6 score on a scale
Standard Deviation 0
|
SECONDARY outcome
Timeframe: 1 year after infusionPopulation: Only 4 subjects were testable at this time point.
Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=1 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=3 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall
|
6 score on a scale
Standard Deviation 0
|
5 score on a scale
Standard Deviation 1
|
—
|
SECONDARY outcome
Timeframe: baseline before infusionPopulation: Only 4 subjects were testable at this time point.
Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=3 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
n=1 Participants
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued
|
—
|
5.67 score on a scale
Standard Deviation 1.53
|
6 score on a scale
Standard Deviation 0
|
SECONDARY outcome
Timeframe: 1 year after infusionPopulation: Only 3 subjects were testable at this time point.
Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=3 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued
|
—
|
4 score on a scale
Standard Deviation 0
|
—
|
SECONDARY outcome
Timeframe: baseline before infusionPopulation: Only 7 subjects were testable with this instrument.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=2 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=4 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
n=1 Participants
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4)
|
80 score on a scale
Standard Deviation 26.87
|
85 score on a scale
Standard Deviation 13.34
|
89 score on a scale
Standard Deviation 0
|
SECONDARY outcome
Timeframe: 1 year after infusionPopulation: Only 6 subjects were testable at this time point.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=2 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=4 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4)
|
56.5 score on a scale
Standard Deviation 21.92
|
86 score on a scale
Standard Deviation 11.58
|
—
|
SECONDARY outcome
Timeframe: baseline before infusionPopulation: Only 7 subjects were testable with this instrument.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=1 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=4 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
n=2 Participants
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family
|
87.5 score on a scale
Standard Deviation 0
|
75.78 score on a scale
Standard Deviation 15.39
|
75 score on a scale
Standard Deviation 36.36
|
SECONDARY outcome
Timeframe: 1 year after infusionPopulation: Two in the BMMNC arm were lost to follow up.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=4 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=10 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family
|
79.69 score on a scale
Standard Deviation 22.89
|
72.81 score on a scale
Standard Deviation 17.74
|
—
|
SECONDARY outcome
Timeframe: baseline before infusionPopulation: Only 8 subjects were testable with this instrument
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=1 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=4 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
n=3 Participants
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social
|
88.64 score on a scale
Standard Deviation 0
|
75.57 score on a scale
Standard Deviation 9.75
|
83.71 score on a scale
Standard Deviation 15.01
|
SECONDARY outcome
Timeframe: 1 year after infusionPopulation: Two in the BMMNC arm were lost to follow up. Five subjects were untestable.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=4 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=10 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social
|
87.7 score on a scale
Standard Deviation 8.67
|
74.77 score on a scale
Standard Deviation 9.21
|
—
|
SECONDARY outcome
Timeframe: baseline before infusionPopulation: 13 subjects were testable with this instrument.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=10 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
n=3 Participants
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings
|
—
|
60.16 score on a scale
Standard Deviation 6.1
|
48.96 score on a scale
Standard Deviation 2.76
|
SECONDARY outcome
Timeframe: 1 year after infusionPopulation: Two in the BMMNC arm were lost to follow up. Five subjects were untestable.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=3 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=10 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings
|
67.01 score on a scale
Standard Deviation 26.85
|
55.73 score on a scale
Standard Deviation 9.68
|
—
|
SECONDARY outcome
Timeframe: baseline before infusionPopulation: Only 8 subjects were testable with this instrument.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=1 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=4 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
n=3 Participants
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation
|
72.73 score on a scale
Standard Deviation 0
|
56.82 score on a scale
Standard Deviation 8.25
|
56.82 score on a scale
Standard Deviation 17.71
|
SECONDARY outcome
Timeframe: 1 year after infusionPopulation: Two in the BMMNC arm were lost to follow up. Five subjects were untestable at this time point.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=4 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=9 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation
|
69.88 score on a scale
Standard Deviation 18.57
|
55.81 score on a scale
Standard Deviation 8.74
|
—
|
SECONDARY outcome
Timeframe: baseline before infusionPopulation: Only 8 subjects were testable with this instrument.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=1 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=4 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
n=3 Participants
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional
|
85.42 score on a scale
Standard Deviation 0
|
75.52 score on a scale
Standard Deviation 5.98
|
69.44 score on a scale
Standard Deviation 6.7
|
SECONDARY outcome
Timeframe: 1 year after infusionPopulation: Two in the BMMNC arm were lost to follow up. Five other subjects were untestable at this time point.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=4 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=10 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional
|
78.12 score on a scale
Standard Deviation 15.73
|
70.83 score on a scale
Standard Deviation 8.56
|
—
|
SECONDARY outcome
Timeframe: baseline before infusionPopulation: Only 8 subjects were testable with this instrument.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=1 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=4 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
n=3 Participants
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access
|
47.5 score on a scale
Standard Deviation 0
|
63.75 score on a scale
Standard Deviation 10.31
|
80.83 score on a scale
Standard Deviation 17.02
|
SECONDARY outcome
Timeframe: 1 year after infusionPopulation: Two in the BMMNC arm were lost to follow up. Five subjects were untestable at this time point.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=4 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=10 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access
|
72.5 score on a scale
Standard Deviation 29.01
|
65.5 score on a scale
Standard Deviation 10.26
|
—
|
SECONDARY outcome
Timeframe: baseline before infusionPopulation: Only 7 subjects were testable with this instrument.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=1 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=4 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
n=2 Participants
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain
|
3.13 score on a scale
Standard Deviation 0
|
40.63 score on a scale
Standard Deviation 20.53
|
35.94 score on a scale
Standard Deviation 19.88
|
SECONDARY outcome
Timeframe: 1 year after infusionPopulation: Two in the BMMNC arm were lost to follow up. Eight subjects were untestable at this time point.
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.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=2 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=9 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain
|
22.39 score on a scale
Standard Deviation 20.82
|
34.38 score on a scale
Standard Deviation 21.26
|
—
|
SECONDARY outcome
Timeframe: baseline before infusionPopulation: Only three subjects were testable with this instrument.
Standard scores ranging from 55 to 145 with higher scores indicating better outcomes and lower scores poorer outcomes.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
n=1 Participants
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=2 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Visual-Spatial Processing as Assessed by Score on the Motor-Free Visual Perception Test (MVPT-3)
|
55 score on a scale
Standard Deviation 0
|
55 score on a scale
Standard Deviation 0
|
—
|
SECONDARY outcome
Timeframe: 1 year after infusionPopulation: Data were not collected for the 5 in the UCB cell arm.
Standard scores ranging from 55 to 145 with higher scores indicating better outcomes and lower scores poorer outcomes.
Outcome measures
| Measure |
Umbilical Cord Blood (UCB) Cells
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=2 Participants
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Visual-Spatial Processing as Assessed by Score on the Motor-Free Visual Perception Test (MVPT-3)
|
—
|
63 score on a scale
Standard Deviation 11.31
|
—
|
Adverse Events
Umbilical Cord Blood (UCB) Cells
Serious events: 2 serious events
Other events: 1 other events
Deaths: 0 deaths
Bone Marrow-derived Mononuclear Cells (BMMNCs)
Serious events: 3 serious events
Other events: 8 other events
Deaths: 0 deaths
Saline Infusion (Placebo)
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Umbilical Cord Blood (UCB) Cells
n=5 participants at risk
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=15 participants at risk
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
n=7 participants at risk
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Infections and infestations
Upper Tract Respiratory Infection
|
0.00%
0/5 • 1 year
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.
|
13.3%
2/15 • Number of events 2 • 1 year
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.
|
0.00%
0/7 • 1 year
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.
|
|
Nervous system disorders
Febrile Convulsion (Febrile Seizure)
|
20.0%
1/5 • Number of events 1 • 1 year
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.
|
0.00%
0/15 • 1 year
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.
|
0.00%
0/7 • 1 year
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.
|
|
Surgical and medical procedures
Osteotomy (Planned Hip Osteotomy)
|
20.0%
1/5 • Number of events 1 • 1 year
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.
|
0.00%
0/15 • 1 year
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.
|
0.00%
0/7 • 1 year
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.
|
|
Infections and infestations
Pneumonia
|
0.00%
0/5 • 1 year
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.
|
6.7%
1/15 • Number of events 1 • 1 year
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.
|
0.00%
0/7 • 1 year
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.
|
Other adverse events
| Measure |
Umbilical Cord Blood (UCB) Cells
n=5 participants at risk
umbilical cord blood (hUCB) cells: Autologous umbilical cord blood banked with the Cord Blood Registry.
|
Bone Marrow-derived Mononuclear Cells (BMMNCs)
n=15 participants at risk
bone marrow derived mononuclear cells (BMMNCs): Autologous stem cells from bone marrow harvest.
|
Saline Infusion (Placebo)
n=7 participants at risk
saline infusion (placebo): inactive substance
|
|---|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis (Nose Bleed)
|
0.00%
0/5 • 1 year
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.
|
0.00%
0/15 • 1 year
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.
|
14.3%
1/7 • Number of events 1 • 1 year
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.
|
|
Infections and infestations
Gastroenteritis (Stomach Flu)
|
0.00%
0/5 • 1 year
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.
|
13.3%
2/15 • Number of events 3 • 1 year
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.
|
0.00%
0/7 • 1 year
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.
|
|
Infections and infestations
Ear Infection
|
0.00%
0/5 • 1 year
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.
|
13.3%
2/15 • Number of events 2 • 1 year
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.
|
0.00%
0/7 • 1 year
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.
|
|
Nervous system disorders
Seizures (More Frequent Seizures)
|
20.0%
1/5 • Number of events 1 • 1 year
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.
|
6.7%
1/15 • Number of events 1 • 1 year
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.
|
14.3%
1/7 • Number of events 1 • 1 year
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.
|
|
Skin and subcutaneous tissue disorders
Vitiligo
|
0.00%
0/5 • 1 year
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.
|
6.7%
1/15 • Number of events 1 • 1 year
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.
|
0.00%
0/7 • 1 year
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.
|
|
Gastrointestinal disorders
Haematochezia (Bloody Stools/Chrohn's Disease)
|
0.00%
0/5 • 1 year
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.
|
0.00%
0/15 • 1 year
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.
|
14.3%
1/7 • Number of events 1 • 1 year
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.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal Congestion
|
20.0%
1/5 • Number of events 1 • 1 year
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.
|
6.7%
1/15 • Number of events 1 • 1 year
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.
|
0.00%
0/7 • 1 year
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.
|
|
Infections and infestations
Pneumonia
|
20.0%
1/5 • Number of events 1 • 1 year
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.
|
0.00%
0/15 • 1 year
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.
|
0.00%
0/7 • 1 year
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.
|
|
Infections and infestations
Croup Infectious
|
20.0%
1/5 • Number of events 1 • 1 year
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.
|
0.00%
0/15 • 1 year
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.
|
0.00%
0/7 • 1 year
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.
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
20.0%
1/5 • Number of events 1 • 1 year
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.
|
13.3%
2/15 • Number of events 2 • 1 year
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.
|
0.00%
0/7 • 1 year
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.
|
|
Infections and infestations
Respiratory Tract Infection
|
0.00%
0/5 • 1 year
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.
|
0.00%
0/15 • 1 year
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.
|
14.3%
1/7 • Number of events 2 • 1 year
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.
|
Additional Information
Charles S. Cox, MD, Professor
The University of Texas Health Science Center at Houston
Phone: 713-500-7300
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place