Cerebral PalsyPhase 2ACT for CPNCT01988584
Safety and Effectiveness of Banked Cord Blood or Bone Marrow Stem Cells in Children With Cerebral Palsy (CP).
A Phase 2 study testing umbilical cord blood (hUCB) cells and bone marrow derived mononuclear cells (BMMNCs) in people with Cerebral Palsy, run by The University of Texas Health Science Center, Houston at 1 site in United States.
Trial summary
The purpose of this study is to compare the safety and effectiveness of two types of stem cells,(either banked cord blood or bone marrow), in children between the ages of 2 to 10 years with CP. 15 children with banked cord blood at CBR and 15 children without banked cord blood will be enrolled into the study. Read more about this study
- Who is it for?
- Ages 2 to 10 years
- Conditions the study is for
- Cerebral Palsy
- What is being tested?
- umbilical cord blood (hUCB) cells and bone marrow derived mononuclear cells (BMMNCs)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2013to 21 Feb 2018
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Children with diagnosis of Cerebral Palsy (spastic CP due to periventricular white matter damage or neonatal brain injury from perinatal stroke or intra-ventricular hemorrhage)
- Gross Motor Function Classification Score level II-V
- Ages 24 months to 10 years
- English speaking, if verbal
- Ability to travel to Houston for treatment and follow-up -
Who cannot join
- Known history of:
- Intractable seizures
- Traumatic brain injury
- Genetic disorder (as demonstrated by newborn screening or genetic diagnostic testing)
- Recently treated or current infection
- Renal insufficiency or altered renal function (as defined by serum creatinine > 1.5 mg/dl at screening)
Show all 25Show fewer
- Hepatic disease or altered liver function (as defined by SGPT > 150 U/L [non-contusion related], and/or T. Bilirubin >1.3 mg/dL at screening)
- HIV+ (as demonstrated by positive blood test)
- Immunosuppression (as defined by WBC <3,000 cells/ml at screening)
- Infectious related neurological injury
- Sensitivity to Ethylene Oxide (EtO) [found in fumigants and disinfectants]
- If Athetoid CP diagnosis, other etiologies such as degenerative, mitochondrial, and metabolic disorders must be excluded, and the outcome assessments must be able to be conducted to assess for potential treatment effects
- Normal brain MRI
- Evidence of acute illness at the time of infusion, such as, but not limited to, fever (temperature > 37.5 C), vomiting, diarrhea, wheezing or crackles
- Progressing neurological disease (as defined by Batten Disease, Leukodystrophies, Metabolic disorders, Mitochondrial disorders, Neurotransmitter disorders)
- Microcephaly, macrocephaly, cortical malformations, genetic disorders of dysgenesis brain malformations due to infection or metabolic disorders
- Pulmonary disease requiring ventilator support
- If hUCB candidate, banked cord cells totaling <10 million/kg
- If hUCB candidate, any positive maternal infectious disease test (Hepatitis A, Hepatitis B, HIV 1, HIV 2, HTLV 1, HTLV 2, and Syphilis)
- If hUCB candidate, cord blood sample contamination
- Participation in a concurrent intervention study
- Unwillingness to return for follow-up visits
- Contraindications to MRI
- Any patient that the investigators feel in their opinion the study intervention is unlikely to benefit the patient will be a screen failure.
- Any patients who are currently or has previously been enrolled in a clinical stem cell study.
Trial locations and contacts
1 site in 1 country.
United States1 site
UTHealth, Medical School, Dept. of Pediatric Surgery
Houston, Texas, 77030
About this study
Official title: Autologous Cell Therapies for Cerebral Palsy-Chronic (ACT for CP)
The purpose of this study is to compare the safety and effectiveness of two types of stem cells,(either banked cord blood or bone marrow), in children between the ages of 2 to 10 years with CP. 15 children with banked cord blood at CBR and 15 children without banked cord blood will be enrolled into the study. The study involves one baseline/treatment visit and 3 follow-up visits at 6 months, 12 months, and 2 years. Five children in each group will be randomized to a placebo control group at the baseline/treatment visit. Parents will not be told if their child received stem cells or a placebo until the 12 month follow-up visit. At that time parents may elect to have their child receive the stem cell treatment; either bone marrow harvest or umbilical cord blood if banked with CBR. All study visits will be conducted at the UTHealth Medical School and Children's Memorial Hermann Hospital in Houston, Texas.
As of 1/21/2014 we have met our enrollment limit for children without banked cord blood undergoing bone marrow harvest for stem cells.
- Conditions
- Cerebral Palsy
- Being tested
- umbilical cord blood (hUCB) cells, Saline Infusion (Placebo), bone marrow derived mononuclear cells (BMMNCs)
- Keywords
- Cerebral Palsy, Brain Injury, Stem Cells, Mononuclear Cells, Bone Marrow, Umbilical Cord Blood
- Medical subject headings
- Cerebral Palsy, Brain Injuries
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
umbilical cord blood (hUCB) cellsBiological
Also called Autolgous Stem Cells
Autologous umbilical cord blood banked with the Cord Blood Registry.
Saline Infusion (Placebo)Drug
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)Biological
Also called Autologous Stem Cells
Autologous stem cells from bone marrow harvest.
| Study group | Role | What they receive |
|---|---|---|
| umbilical cord blood (UCB) cells | Experimental | Children who have banked UCB with CBR will receive an umbilical cord blood stem cell infusion at the baseline/treatment visit. Receives: Biological: umbilical cord blood (hUCB) cells |
| bone marrow-derived mononuclear cells (BMMNCs) | Experimental | Children in the BMMNC group will undergo bone marrow harvest and stem cell infusion at the baseline/treatment visit. Receives: Biological: bone marrow derived mononuclear cells (BMMNCs) |
| saline infusion (placebo), then umbilical cord blood (UCB) cells | Experimental | 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. Receives: Biological: umbilical cord blood (hUCB) cells; Drug: Saline Infusion (Placebo) |
| saline infusion (placebo), then bone marrow-derived mononuclear cells (BMMNCs) | Experimental | Not described Receives: Drug: Saline Infusion (Placebo); Biological: bone marrow derived mononuclear cells (BMMNCs) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety as Assessed by Number of Participants With In-hospital Infusion Toxicity
In-hospital infusion toxicity includes hemodynamic, pulmonary, hepatic, renal, or neurologic complications.
Measured: 24 hours after infusion
Long-term Safety
Long-term safety as assessed by number of participants who developed new mass lesions or other pathological structural changes or had worsening neurological status
Measured: from the time of infusion to 1 year after infusion
Secondary outcomes (41)
Number of Participants With an Improvement in White Matter Integrity.
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.
Measured: from baseline to 1 year after infusion
Gross Motor Function Classification Score (GMFM-66)
Interval age adjusted scaled scores from 0 to 100 with 0 reflecting a child with low motor ability and 100 indicating high motor ability.
Measured: baseline before infusion
Gross Motor Function Classification Score (GMFM-66)
Interval age adjusted scaled scores from 0 to 100 with 0 reflecting a child with low motor ability and 100 indicating high motor ability.
Measured: 1 year after infusion
Gross Motor Function Classification Score (GMFM-88)
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.
Measured: baseline before infusion
Gross Motor Function Classification Score (GMFM-88)
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.
Measured: 1 year after infusion
Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication
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.
Measured: baseline before infusion
Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication
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.
Measured: 1 year after infusion
Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living
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.
Measured: baseline before infusion
Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living
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.
Measured: 1 year after infusion
Score on Vineland Adaptive Behavior Scales (VABS-2) - Social
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.
Measured: baseline before infusion
Score on Vineland Adaptive Behavior Scales (VABS-2) - Social
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.
Measured: 1 year after infusion
Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor
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.
Measured: baseline before infusion
Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor
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.
Measured: 1 year after infusion
Score on Pediatric Evaluation of Disability Inventory - Self-Care
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.
Measured: baseline before infusion
Score on Pediatric Evaluation of Disability Inventory - Self-Care
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.
Measured: 1 year after infusion
Score on Pediatric Evaluation of Disability Inventory - Mobility
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.
Measured: baseline before infusion
Score on Pediatric Evaluation of Disability Inventory - Mobility
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.
Measured: 1 year after infusion
Score on Pediatric Evaluation of Disability Inventory - Social
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.
Measured: baseline before infusion
Score on Pediatric Evaluation of Disability Inventory - Social
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.
Measured: 1 year after infusion
Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall
Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.
Measured: baseline before infusion
Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall
Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.
Measured: 1 year after infusion
Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued
Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.
Measured: baseline before infusion
Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued
Scales scores from 1 to 19 with higher scores indicate better outcomes and lower scores indicate poorer outcomes.
Measured: 1 year after infusion
Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4)
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.
Measured: baseline before infusion
Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4)
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.
Measured: 1 year after infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family
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.
Measured: baseline before infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family
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.
Measured: 1 year after infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social
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.
Measured: baseline before infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social
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.
Measured: 1 year after infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings
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.
Measured: baseline before infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings
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.
Measured: 1 year after infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation
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.
Measured: baseline before infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation
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.
Measured: 1 year after infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional
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.
Measured: baseline before infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional
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.
Measured: 1 year after infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access
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.
Measured: baseline before infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access
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.
Measured: 1 year after infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain
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.
Measured: baseline before infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain
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.
Measured: 1 year after infusion
Visual-Spatial Processing as Assessed by Score on the Motor-Free Visual Perception Test (MVPT-3)
Standard scores ranging from 55 to 145 with higher scores indicating better outcomes and lower scores poorer outcomes.
Measured: baseline before infusion
Visual-Spatial Processing as Assessed by Score on the Motor-Free Visual Perception Test (MVPT-3)
Standard scores ranging from 55 to 145 with higher scores indicating better outcomes and lower scores poorer outcomes.
Measured: 1 year after infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Nov 2013 (estimated)
- Study startNov 2013
- Primary completion21 Feb 2018
- Study completion21 Feb 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cerebral PalsyStudies of the same condition, open and under way.
- More trials run by The University of Texas Health Science Center, HoustonThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Outcomes assessor
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Cord Blood Registry, Inc., Let's Cure CP Foundation, Mission Connect, a program of TIRR Foundation
- Responsible party
- Principal investigator, Charles Cox, The University of Texas Health Science Center, Houston
- Investigators
- Charles S Cox, MD, Principal investigator, UTHealth, Medical School, Dept. of Pediatric Surgery
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01988584
- Sponsor's protocol ID
- HSC-MS-12-0876
- Acronym
- ACT for CP
- Status last verified
- Oct 2022
- First submitted
- 4 Nov 2013
- Last updated
- 21 Nov 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 21 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Autologous Cell Therapies for Cerebral Palsy-Chronic (ACT for CP)
- ClinicalTrials.gov ID
- NCT01988584
- Acronym / sponsor ID
- ACT for CP / HSC-MS-12-0876
- Conditions
- Cerebral Palsy
- Keywords
- Cerebral Palsy, Brain Injury, Stem Cells, Mononuclear Cells, Bone Marrow, Umbilical Cord Blood
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- The University of Texas Health Science Center, HoustonUniversity, hospital or other
- Collaborators
- Cord Blood Registry, Inc., Let's Cure CP Foundation, Mission Connect, a program of TIRR Foundation
- Study start
- Nov 2013 (actual)
- Primary completion
- 21 Feb 2018 (actual)
- Study completion
- 21 Feb 2018 (actual)
- First posted
- 20 Nov 2013 (estimated)
- Results
- Posted 21 Nov 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to compare the safety and effectiveness of two types of stem cells,(either banked cord blood or bone marrow), in children between the ages of 2 to 10 years with CP. 15 children with banked cord blood at CBR and 15 children without banked cord blood will be enrolled into the study. The study involves one baseline/treatment visit and 3 follow-up visits at 6 months, 12 months, and 2 years. Five children in each group will be randomized to a placebo control group at the baseline/treatment visit. Parents will not be told if their child received stem cells or a placebo until the 12 month follow-up visit. At that time parents may elect to have their child receive the stem cell treatment; either bone marrow harvest or umbilical cord blood if banked with CBR. All study visits will be conducted at the UTHealth Medical School and Children's Memorial Hermann Hospital in Houston, Texas.
As of 1/21/2014 we have met our enrollment limit for children without banked cord blood undergoing bone marrow harvest for stem cells.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| umbilical cord blood (UCB) cells | Experimental | Biological: umbilical cord blood (hUCB) cellsChildren who have banked UCB with CBR will receive an umbilical cord blood stem cell infusion at the baseline/treatment visit. |
| bone marrow-derived mononuclear cells (BMMNCs) | Experimental | Biological: 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. |
| saline infusion (placebo), then umbilical cord blood (UCB) cells | Experimental | Biological: umbilical cord blood (hUCB) cells, Drug: Saline Infusion (Placebo)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. |
| saline infusion (placebo), then bone marrow-derived mononuclear cells (BMMNCs) | Experimental | Drug: Saline Infusion (Placebo), Biological: bone marrow derived mononuclear cells (BMMNCs) |
Who may qualify
- Ages
- 2 to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Children with diagnosis of Cerebral Palsy (spastic CP due to periventricular white matter damage or neonatal brain injury from perinatal stroke or intra-ventricular hemorrhage)
- Gross Motor Function Classification Score level II-V
- Ages 24 months to 10 years
- English speaking, if verbal
- Ability to travel to Houston for treatment and follow-up -
Key exclusion criteria (25 listed)
- Known history of:
- If Athetoid CP diagnosis, other etiologies such as degenerative, mitochondrial, and metabolic disorders must be excluded, and the outcome assessments must be able to be conducted to assess for potential treatment effects
- Normal brain MRI
- Evidence of acute illness at the time of infusion, such as, but not limited to, fever (temperature > 37.5 C), vomiting, diarrhea, wheezing or crackles
- Progressing neurological disease (as defined by Batten Disease, Leukodystrophies, Metabolic disorders, Mitochondrial disorders, Neurotransmitter disorders)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| umbilical cord blood (hUCB) cellsAlso: Autolgous Stem CellsAutologous umbilical cord blood banked with the Cord Blood Registry. | Biological | saline infusion (placebo), then umbilical cord blood (UCB) cells, umbilical cord blood (UCB) cells |
| 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. | Drug | saline infusion (placebo), then bone marrow-derived mononuclear cells (BMMNCs), saline infusion (placebo), then umbilical cord blood (UCB) cells |
| bone marrow derived mononuclear cells (BMMNCs)Also: Autologous Stem CellsAutologous stem cells from bone marrow harvest. | Biological | bone marrow-derived mononuclear cells (BMMNCs), saline infusion (placebo), then bone marrow-derived mononuclear cells (BMMNCs) |
Primary outcome
Safety as Assessed by Number of Participants With In-hospital Infusion Toxicity
Time frame: 24 hours after infusion
In-hospital infusion toxicity includes hemodynamic, pulmonary, hepatic, renal, or neurologic complications.
Long-term Safety
Time frame: from the time of infusion to 1 year after infusion
Long-term safety as assessed by number of participants who developed new mass lesions or other pathological structural changes or had worsening neurological status
41 secondary outcomes
Number of Participants With an Improvement in White Matter Integrity.
Time frame: from baseline to 1 year after infusion
Gross Motor Function Classification Score (GMFM-66)
Time frame: baseline before infusion
Gross Motor Function Classification Score (GMFM-66)
Time frame: 1 year after infusion
Gross Motor Function Classification Score (GMFM-88)
Time frame: baseline before infusion
Gross Motor Function Classification Score (GMFM-88)
Time frame: 1 year after infusion
Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication
Time frame: baseline before infusion
Score on Vineland Adaptive Behavior Scales (VABS-2) - Communication
Time frame: 1 year after infusion
Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living
Time frame: baseline before infusion
Score on Vineland Adaptive Behavior Scales (VABS-2) - Daily Living
Time frame: 1 year after infusion
Score on Vineland Adaptive Behavior Scales (VABS-2) - Social
Time frame: baseline before infusion
Score on Vineland Adaptive Behavior Scales (VABS-2) - Social
Time frame: 1 year after infusion
Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor
Time frame: baseline before infusion
Score on Vineland Adaptive Behavior Scales (VABS-2) - Motor
Time frame: 1 year after infusion
Score on Pediatric Evaluation of Disability Inventory - Self-Care
Time frame: baseline before infusion
Score on Pediatric Evaluation of Disability Inventory - Self-Care
Time frame: 1 year after infusion
Score on Pediatric Evaluation of Disability Inventory - Mobility
Time frame: baseline before infusion
Score on Pediatric Evaluation of Disability Inventory - Mobility
Time frame: 1 year after infusion
Score on Pediatric Evaluation of Disability Inventory - Social
Time frame: baseline before infusion
Score on Pediatric Evaluation of Disability Inventory - Social
Time frame: 1 year after infusion
Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall
Time frame: baseline before infusion
Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Recall
Time frame: 1 year after infusion
Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued
Time frame: baseline before infusion
Score on A Developmental NEuroPSYchological Assessment (NEPSY-II) - Learning and Memory Memory Subtest - Free/Cued
Time frame: 1 year after infusion
Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4)
Time frame: baseline before infusion
Expressive and Receptive Vocabulary as Assessed by Score on the Peabody Picture Vocabulary Test (PPVT-4)
Time frame: 1 year after infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family
Time frame: baseline before infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Family
Time frame: 1 year after infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social
Time frame: baseline before infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Social
Time frame: 1 year after infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings
Time frame: baseline before infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Feelings
Time frame: 1 year after infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation
Time frame: baseline before infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Participation
Time frame: 1 year after infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional
Time frame: baseline before infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Emotional
Time frame: 1 year after infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access
Time frame: baseline before infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Access
Time frame: 1 year after infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain
Time frame: baseline before infusion
Score on Cerebral Palsy Quality of Life Questionnaire (CPQOL) - Pain
Time frame: 1 year after infusion
Visual-Spatial Processing as Assessed by Score on the Motor-Free Visual Perception Test (MVPT-3)
Time frame: baseline before infusion
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Nov 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Charles S Cox, MD principal investigator
UTHealth, Medical School, Dept. of Pediatric Surgery
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing umbilical cord blood (hUCB) cells and bone marrow derived mononuclear cells (BMMNCs) in people with Cerebral Palsy, run by The University of Texas Health Science Center, Houston at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 25 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 21 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 21 Nov 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety as Assessed by Number of Participants With In-hospital Infusion Toxicity (time frame: 24 hours after infusion). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Nov 2022.