Clinical trial NCT03078621: Bone Marrow-Derived Stem Cell Transplantation for the Treatment of Cerebral Palsy
Official title: Safety and Efficacy of Transplantation of Specific Populations of Stem Cells and Mesenchymal Stem Cells for the Treatment of Cerebral Palsy
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Cerebral Palsy
Keywords: stem cells
Lead sponsor: Stem Cells Arabia
Sponsor class: OTHER
First posted: 2017-03-13
Enrollment: 50
Sex: ALL
Minimum age (years): 2
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study is a single arm, single center trial to study the safety and efficacy of purified autologous bone marrow derived stem cells and mesenchymal stem cells for the treatment of children with cerebral palsy.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Changes in Motor Performance.
timeFrame: 6 months
Secondary outcomes:
measure: Motor function studies
timeFrame: 1 year
measure: Specific white matter tract analysis using MRI
timeFrame: 6 months
Other outcomes:
Locations:
Study officials:
References: