Stem Cells in Umbilical Blood Infusion for CP (SCUBI-CP)

Murdoch Childrens Research Institute

Status and phase: Completed; Phase 1

Conditions: Cerebral Palsy

Treatments: Biological: Matched sibling donor cord blood cell infusion

Study type: Interventional

Funder types: Other

Identifiers

NCT03087110

U1111-1179-9253 (Other Identifier)

HREC/14/RCHM/38 RCH ID 34210;

Details and patient eligibility

About

This study will investigate the safety of single dose intravenous infusion of cord blood cells which were cryopreserved after the birth of a brother or sister to a child with cerebral palsy.

Full description

Cerebral palsy (CP) is the most common physical disability of childhood, affecting 2 per 1000 live births across the world. CP describes permanent non-progressive motor disorders arising from damage to the developing brain.

Preclinical studies of different types of stem cells in models of acute brain injury similar to CP have shown significant functional improvement. The variety of stem cells available in umbilical cord blood (UCB), an ethically uncomplicated source of stem cells, has led to a focus on UCB stem cell therapy as a quick-to-clinic option. Previous studies indicate that autologous or unrelated donor UCBC infusion is safe and feasible for children with CP, and may lead to improved motor functioning, but there is no information about the safety and effects of matched sibling cord blood. Therefore, this trial will study the safety of infusing matched sibling cord blood cells to children with cerebral palsy.

Enrollment: 12 patients

Sex: All

Ages: 1 to 16 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Diagnosis of any type of CP
2. CP of any severity
3. A record of sibling CBU in storage at a TGA accredited private cord blood bank
4. Ability to travel to one of the trial centres
5. Ability to participate in assessments
6. Informed consent by parent/guardian

Exclusion criteria

1. presence of progressive neurological disease
2. known genetic disorder
3. known brain dysplasia
4. immune system disorder or immune deficiency syndrome
5. infectious disease markers showing up on virology screen
6. evidence of cord blood unit contamination, or fewer than 10^7 cells/kg body mass
7. ventilator support
8. ill health, or if the participant's medical condition does not allow safe travel
9. previous cell therapy
10. Botulinum toxin A within 3 months before or after infusion
11. surgery within 3 months before or after infusion
12. cannot obtain parent/guardian consent

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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12 participants in 1 patient group

Cord blood infusion

Experimental group

Description:

Matched sibling donor cord blood cell infusion

Treatment:

Biological: Matched sibling donor cord blood cell infusion