- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01072370
Safety and Effectiveness of Cord Blood Stem Cell Infusion for the Treatment of Cerebral Palsy in Children
October 6, 2022 updated by: James E. Carroll, Augusta University
A Placebo-Controlled, Observer-Blinded, Crossover Study to Evaluate the Safety and Effectiveness of a Single, Autologous, Cord Blood Stem Cell Infusion for the Treatment of Cerebral Palsy in Children
The purpose of this study is to test the safety and effectiveness of a cord blood infusion in children who have motor disability due to cerebral palsy (CP).
The subjects will be children whose parents have saved their infant's cord blood, who have non-progressive motor disability, and whose parents intend to have a cord blood infusion.
Study Overview
Status
Withdrawn
Conditions
Intervention / Treatment
Detailed Description
The purpose of this study is to conduct an observer-blinded crossover investigation of the safety and efficacy of autologous cord blood infusion in children who demonstrate non-progressive motor disability due to brain dysfunction (commonly called cerebral palsy) and who do not have an apparent disorder of brain development or obstructive hydrocephalus.
The degree of delay in motor development will be such that the children are unable to sit independently by 12 months of age or unable to walk independently by 18 months of age.
However, because the diagnosis is one of exclusion, we will enroll patients only after they have reached two years of age.
By this age, it is likely other conditions would be excluded.
As the Gross Motor Function Classification System (GMFCS) was developed for children up to 12 years of age, the maximum age of recruitment will be 12 years.
Any level of cerebral palsy severity will be allowed.
The subjects will be children whose parents have saved their infants cord blood, who have clinical evidence of a non-progressive motor disability, and whose parents intend to have a cord blood infusion.
Study Type
Interventional
Phase
- Phase 2
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
Georgia
-
Augusta, Georgia, United States, 30912
- Augusta University
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
1 year to 12 years (Child)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- Must be more than 1 year of age and less than 12 years of age at the time of screening for inclusion in the study.
- Clinical evidence of a non-progressive motor disability due to brain dysfunction. The subjects will not have the ability to sit independently by one year of age or the ability to walk by 18 months of age.
- Have stored umbilical cord blood with Cord Blood Registry (CBR) that meets all selection and testing criteria.
- Willing to comply with all study procedures.
- The nucleated cells available in the cord blood sample stored at CBR must exceed 1 X 107 cells per kg body weight. (Note: Because cord blood collection has been in process for about 16 years but widely for far less than that period, the age of most subjects likely will be considerably less than 12 years of age. Due to the amount due of cord blood available for most subjects, the body weight of subjects usually will not exceed 25 kg).
- The patients must be seizure-free or seizures adequately controlled. If there is a suspicion of seizures and EEG should be done prior to inclusion.
Exclusion Criteria:
- Have complicating medical issues that would interfere with blood drawing, such as venous access so limited that success is unlikely
- Presence of obstructive hydrocephalus.
- Presence of progressive neurological disease.
- Presence of significant defect of brain development, such as schizencephaly or agenesis of corpus callosum
- Presence of known chromosomal anomaly
- Presence of major congenital anomaly
- Severe intrauterine growth restriction (birth weight less than 1800 grams)
- Cord blood viability <60%
- Positive infectious disease markers from mother's blood or cord blood at the time of collection.
- Evidence of illness on planned infusion date (such as but not limited to fever >38.5, vomiting, diarrhea, wheezing, or crackles)
- Pregnancy
- Use of immunosuppressive drugs
- Evidence of known genetic disorder
- Impaired hepatic or renal function
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Crossover Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Active Comparator: Treatment Group 1
|
red-cell depleted, mononuclear cell enriched cord blood unit prepared for infusion
Other Names:
|
Sham Comparator: Treatment Group 2
|
intravenous infusion of 5% dextrose, ¼ normal saline solution
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Confirm the safety of autologous cord blood infusion in children with cerebral palsy by repeated follow-up over one year with clinical and laboratory evaluations.
Time Frame: 1 year
|
1 year
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Confirm the efficacy of autologous cord blood infusion in children with cerebral palsy using patient questionnaire and standardized Gross Motor Function Measure evaluation.
Time Frame: 3-4 months
|
3-4 months
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Chair: James E Carroll, M.D., Augusta University
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Helpful Links
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
January 1, 2010
Primary Completion (Actual)
February 28, 2019
Study Completion (Actual)
February 28, 2019
Study Registration Dates
First Submitted
February 16, 2010
First Submitted That Met QC Criteria
February 18, 2010
First Posted (Estimate)
February 22, 2010
Study Record Updates
Last Update Posted (Actual)
October 7, 2022
Last Update Submitted That Met QC Criteria
October 6, 2022
Last Verified
October 1, 2022
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- ACBSC09
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Cerebral Palsy
-
Gazi UniversityCompletedCerebral Palsy | Cerebral Palsy, Spastic | Cerebral Palsy Spastic Diplegia | Cerebral Palsy Quadriplegic | Cerebral Palsy, MonoplegicTurkey
-
Northwestern UniversityActive, not recruitingCerebral Palsy | Diplegic Cerebral Palsy | Bilateral Cerebral PalsyUnited States
-
Centre Médico-Chirurgical de Réadaptation des Massues...RecruitingCerebral Palsy, Dyskinetic | Cerebral Palsy, Spastic | Infantile Hemiplegic Cerebral PalsyFrance
-
St Mary's University CollegeUniversity of GloucestershireUnknownCerebral Palsy | Cerebral Palsy Ataxic | Cerebral Palsy, MixedUnited Kingdom
-
Hilde FeysHasselt University; ETH Zurich; Curtin UniversityRecruitingHemiplegic Cerebral Palsy | Cerebral Palsy, SpasticBelgium
-
University of California, San FranciscoNational Institutes of Health (NIH)RecruitingDystonic Cerebral Palsy | Dyskinetic Cerebral PalsyUnited States
-
MTI UniversityEnrolling by invitationSpastic Diplegic Cerebral PalsyEgypt
-
East Carolina UniversityRecruitingHemiplegic Cerebral Palsy | Unilateral Cerebral Palsy | Remote Ischemic ConditioningUnited States
-
October 6 UniversityCompletedSpastic Cerebral Palsy | Spastic Hemiplegic Cerebral PalsyEgypt
-
Marmara UniversityUnknownCerebral Palsy, Spastic | Cerebral Palsy, Spastic, DiplegicTurkey
Clinical Trials on Cord Blood Infusion
-
Joanne Kurtzberg, MDAvailableCerebral Palsy | Apraxia of Speech | Hydrocephalus | Hypoxia Ischemia, Cerebral | Drowning; AnoxiaUnited States
-
Joanne Kurtzberg, MDCompletedAutism Spectrum Disorder | Autism | ASD | PDDUnited States
-
James Baumgartner, MDCord Blood Registry, Inc.SuspendedPerinatal Arterial Ischemic StrokeUnited States
-
Bundang CHA HospitalWithdrawnCerebral PalsyKorea, Republic of
-
MinYoung Kim, M.D.Completed
-
Pomeranian Medical University SzczecinUnknownNecrotizing Enterocolitis | Intracranial Hemorrhages | Retinopathy of Prematurity | Respiratory Distress Syndrome, Newborn | Anemia, Neonatal | Bronchopulmonary DysplasiaPoland
-
Joanne Kurtzberg, MDThe Marcus FoundationCompleted
-
Michael CottenThe Robertson FoundationCompletedModerate or Severe Hypoxic-ischemic Encephalopathy in NewbornsUnited States
-
Mesoblast, Ltd.CompletedNon-Hodgkin's Lymphoma | Acute Lymphoblastic Leukemia | Acute Myelogenous Leukemia | Hodgkin's DiseaseUnited States
-
Murdoch Childrens Research InstituteSydney Children's Hospitals Network; Monash Health; Cerebral Palsy Alliance; The... and other collaboratorsCompleted