Bone Marrow-Derived Stem Cell Transplantation for the Treatment of Cerebral Palsy

Phase 1

• Conditions: Cerebral Palsy
• Interventions: Biological: Stem Cell Therapy

• Registration Number: NCT03078621
• Lead Sponsor: Stem Cells Arabia

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.

Study Timeline

• Study Start: 2016-09
• Study First Submitted: 2017-02-25
• Study First Submitted QC: 2017-03-07
• Study First Posted: 2017-03-13
• Status Verified: 2020-03
• Last Update Submitted: 2020-03-15
• Last Update Posted: 2020-03-17
• Primary Completion: 2020-10
• Study Completion: 2021-01

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 50

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Stem Cells	Stem Cell Therapy	Intravenous and Intrathecal transplantation of specific populations of purified bone marrow-derived stem cells and mesenchymal stem cells.

Primary Outcome Measures

Name	Time	Method
Changes in Motor Performance.	6 months	GMPM (Gross Motor Performance Measure) as a standard measurement tool for assessing quality of movement.

Secondary Outcome Measures

Name	Time	Method
Motor function studies	1 year	Total volumes and specific tract lesions will be studied and correlated with functional outcomes.
Specific white matter tract analysis using MRI	6 months	Changes on brain Diffusion Tensor Image (DTI).