- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03078621
Bone Marrow-Derived Stem Cell Transplantation for the Treatment of Cerebral Palsy
Safety and Efficacy of Transplantation of Specific Populations of Stem Cells and Mesenchymal Stem Cells for the Treatment of Cerebral Palsy
Study Overview
Detailed Description
Cerebral palsy (CP) is a chronic childhood disorder defined by a range of motor and cognitive impairments and results in a substantial suffering to the patient and a tremendous socio-economic burden to the individual, family, and healthcare system. With no effective treatments or interventions, therapies for CP are currently focused on supportive and management strategies. Stem cell transplantation has been suggested as a putative intervention for neural pathology, as mesenchymal and neural stem cells, as well as olfactory ensheathing glia and Schwann cells, which have shown some regenerative and functional efficacy in experimental central nervous system disorders.
In this trial, the investigators study the safety and efficacy of the intrathecal transplantation of specific populations of purified autologous bone marrow-derived stem cells and mesenchymal stem cells in patients with cerebral palsy.
Study Type
Enrollment (Anticipated)
Phase
- Phase 2
- Phase 1
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- Must be more than 2 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.
- Willing to comply with all study procedures.
- Gross Motor Function Classification Score level II-V
Exclusion Criteria:
Known history of:
- Intractable seizures
- Traumatic brain injury
- Genetic disorder
- Current Infection
- Renal insufficiency
- Hepatic disease
- HIV+ (as demonstrated by positive blood test)
- Immunosuppression
- Infectious related neurological injury
- Other etiologies such as degenerative, mitochondrial, and metabolic disorders
- Normal brain MRI
- Evidence of acute illness such as fever (temperature > 37.5 C), vomiting, diarrhea, wheezing or crackles
- Progressing neurological disease (Batten Disease, Leukodystrophies, Neurotransmitter disorders)
- Microcephaly, macrocephaly, cortical malformations, or genetic disorders of dysgenesis
- Pulmonary disease requiring ventilator support
- Unwillingness to return for follow-up visits
- Contraindications to MRI
Study Plan
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: NA
- Interventional Model: SINGLE_GROUP
- Masking: NONE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
EXPERIMENTAL: Stem Cells
Intravenous and Intrathecal transplantation of specific populations of purified bone marrow-derived stem cells and mesenchymal stem cells.
|
Intravenous and Intrathecal transplantation of purified autologous bone marrow-derived stem cells and mesenchymal stem cells.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Changes in Motor Performance.
Time Frame: 6 months
|
GMPM (Gross Motor Performance Measure) as a standard measurement tool for assessing quality of movement.
|
6 months
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Motor function studies
Time Frame: 1 year
|
Total volumes and specific tract lesions will be studied and correlated with functional outcomes.
|
1 year
|
Specific white matter tract analysis using MRI
Time Frame: 6 months
|
Changes on brain Diffusion Tensor Image (DTI).
|
6 months
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start
Primary Completion (ANTICIPATED)
Study Completion (ANTICIPATED)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (ACTUAL)
Study Record Updates
Last Update Posted (ACTUAL)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- SCA-CP1
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
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
-
East Carolina UniversityRecruitingHemiplegic Cerebral Palsy | Unilateral Cerebral Palsy | Remote Ischemic ConditioningUnited States
-
MTI UniversityEnrolling by invitationSpastic Diplegic Cerebral PalsyEgypt
-
October 6 UniversityCompletedSpastic Cerebral Palsy | Spastic Hemiplegic Cerebral PalsyEgypt
-
Marmara UniversityUnknownCerebral Palsy, Spastic | Cerebral Palsy, Spastic, DiplegicTurkey
Clinical Trials on Stem Cell Therapy
-
Mongolian National University of Medical SciencesNot yet recruiting
-
Hackensack Meridian HealthActive, not recruitingDiabetes Mellitus Type 1United States
-
General Hospital of Chinese Armed Police ForcesUnknownHereditary Cerebellar Ataxia.China
-
University Medical Centre LjubljanaCompletedHeart Failure | Dilated CardiomyopathySlovenia
-
University Hospital, Basel, SwitzerlandUnknownWound Healing | Chronic Wounds | Hematopoietic Stem Cells | Pressure SoresSwitzerland
-
Johann Wolfgang Goethe University HospitalCompletedArterial Occlusive DiseasesGermany
-
Johann Wolfgang Goethe University HospitalCompletedCongestive Heart FailureGermany
-
University Medical Centre LjubljanaRecruitingHeart Failure | Mechanical Circulatory SupportSlovenia
-
Universidad de ZaragozaHospital Universitario Fundación Jiménez DíazCompleted
-
University of HelsinkiUnknownMyocardial Infarction | Coronary Artery Disease | Heart FailureFinland