SCUBI-CP: Stem Cells in Umbilical Blood Infusion for CP

• Sponsor: Murdoch Childrens Research Institute (Other)
• Overall Status: Completed
• CT.gov ID: NCT03087110
• Collaborator: The Royal Children's Hospital (Other), Children's Health Queensland (Other), Monash Health (Other), Sydney Children's Hospitals Network (Other), Cerebral Palsy Alliance (Other)
• Enrollment: 12
• Locations: 2
• Arm: 1
• Actual Duration (Months): 57
• Patients Per Site: 6
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief 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.

Condition or Disease	Intervention/Treatment	Phase
Cerebral Palsy	Biological: Matched sibling donor cord blood cell infusion	Phase 1

Detailed 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.

Study Design

• Study Type: Interventional
• Actual Enrollment: 12 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Safety Study of Sibling Cord Blood Cell Infusion to Children With Cerebral Palsy
• Actual Study Start Date: Mar 1, 2016
• Actual Primary Completion Date: Sep 1, 2019
• Actual Study Completion Date: Dec 1, 2020

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Cord blood infusion Matched sibling donor cord blood cell infusion	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)

Outcome Measures

Primary Outcome Measures

1. Number of participants with abnormal clinical assessment and/or laboratory values [12 months]

   Safety

Secondary Outcome Measures

1. Preliminary analysis of change in gross motor function [Baseline, 3 months]

   Gross Motor Function Measure (GMFM-66)

2. Preliminary analysis of change in gross motor function [Baseline, 12 months]

   Gross Motor Function Measure (GMFM-66)

3. Preliminary analysis of change in fine motor function [Baseline, 3 months]

   Quality of Upper Extremity Skills Test (QUEST)

4. Preliminary analysis of change in fine motor function [Baseline, 12 months]

   Quality of Upper Extremity Skills Test (QUEST)

5. Preliminary analysis of change in cognitive function [Baseline, 12 months]

   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.

6. Preliminary analysis of change in quality of life [Baseline, 3 months]

   Cerebral Palsy Quality of Life (CP-QoL-CHILD)

7. Preliminary analysis of change in quality of life [Baseline,12 months]

   Cerebral Palsy Quality of Life (CP-QoL-CHILD)

8. Digital PCR analysis of peripheral blood cellular DNA to determine the fraction of donor DNA in circulation [3 months]

   Chimerism study to detect the longevity of infused cells

Eligibility Criteria

Criteria

• Ages Eligible for Study: 1 Year to 16 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Lady Cilento Children's Hospital	Brisbane	Queensland	Australia	4101
2	The Royal Children's Hospital	Melbourne	Victoria	Australia	3052

Sponsors and Collaborators

• Murdoch Childrens Research Institute
• The Royal Children's Hospital
• Children's Health Queensland
• Monash Health
• Sydney Children's Hospitals Network
• Cerebral Palsy Alliance

Investigators

• Principal Investigator: Dinah Reddihough, MBChB, MD, Group leader

Study Documents (Full-Text)

• Study Protocol - Mar 6, 2017

More Information

Publications

None provided.

• Responsible Party: Murdoch Childrens Research Institute
• ClinicalTrials.gov Identifier: NCT03087110
• Other Study ID Numbers: HREC/14/RCHM/38; RCH ID 34210; U1111-1179-9253
• First Posted: Mar 22, 2017
• Last Update Posted: Jan 20, 2021
• Last Verified: Jan 1, 2021

• Individual Participant Data (IPD) Sharing Statement: Yes
• Plan to Share IPD: Yes
• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Cerebral Palsy

Study Results

No Results Posted as of Jan 20, 2021