Stem Cells in Umbilical Blood Infusion for CP

Cerebral Palsy Clinical Trial

— SCUBI-CP  

Official title:

Safety Study of Sibling Cord Blood Cell Infusion to Children With Cerebral Palsy

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03087110
• Other study ID #: HREC/14/RCHM/38; RCH ID 34210
• Secondary ID: U1111-1179-9253
• Status: Completed
• Phase: Phase 1
• Start date: March 2016
• Est. completion date: December 2020

Study information

• Verified date: January 2021
• Source: Murdoch Childrens Research Institute
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 12
• Est. completion date: December 2020
• Est. primary completion date: September 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 16 Years

Eligibility

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

Related Conditions & MeSH terms

• Cerebral Palsy

Intervention

Biological:
• Matched sibling donor cord blood cell infusion
Single dose intravenous infusion of 12/12 HLA matched sibling donor cord blood cells (>1x10^7 cells/kg)

Locations

Country	Name	City	State
Australia	Lady Cilento Children's Hospital	Brisbane	Queensland
Australia	The Royal Children's Hospital	Melbourne	Victoria

Sponsors (6)

Lead Sponsor	Collaborator
Murdoch Childrens Research Institute	Cerebral Palsy Alliance, Children's Health Queensland, Monash Health, Sydney Children's Hospitals Network, The Royal Children's Hospital

Country where clinical trial is conducted

Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of participants with abnormal clinical assessment and/or laboratory values	Safety	12 months
Secondary	Preliminary analysis of change in gross motor function	Gross Motor Function Measure (GMFM-66)	Baseline, 3 months
Secondary	Preliminary analysis of change in gross motor function	Gross Motor Function Measure (GMFM-66)	Baseline, 12 months
Secondary	Preliminary analysis of change in fine motor function	Quality of Upper Extremity Skills Test (QUEST)	Baseline, 3 months
Secondary	Preliminary analysis of change in fine motor function	Quality of Upper Extremity Skills Test (QUEST)	Baseline, 12 months
Secondary	Preliminary analysis of change in cognitive function	Age appropriate cognitive testing: Bayley Scales of Infant and Toddler Development (BSID-III), Wechsler Preschool Primary Scale of Intelligence (WPPSI-IV), Wechsler Intelligence Scale for Children (WISC-V). Change from baseline will be compared using z-scores across measures.	Baseline, 12 months
Secondary	Preliminary analysis of change in quality of life	Cerebral Palsy Quality of Life (CP-QoL-CHILD)	Baseline, 3 months
Secondary	Preliminary analysis of change in quality of life	Cerebral Palsy Quality of Life (CP-QoL-CHILD)	Baseline,12 months
Secondary	Digital PCR analysis of peripheral blood cellular DNA to determine the fraction of donor DNA in circulation	Chimerism study to detect the longevity of infused cells	3 months