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

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

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.

Study Overview

• Status: Completed
• Conditions: Cerebral Palsy
• Intervention / Treatment: Biological: Matched sibling donor cord blood cell infusion

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 Type: Interventional
• Enrollment (Actual): 12
• Phase: Phase 1

Contacts and Locations

Study Locations

• Australia
  • Queensland
    • Brisbane, Queensland, Australia, 4101
      • Lady Cilento Children's Hospital
  • Victoria
    • Melbourne, Victoria, Australia, 3052
      • The Royal Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 16 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / 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)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with abnormal clinical assessment and/or laboratory values	Safety	12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Preliminary analysis of change in gross motor function	Gross Motor Function Measure (GMFM-66)	Baseline, 3 months
Preliminary analysis of change in gross motor function	Gross Motor Function Measure (GMFM-66)	Baseline, 12 months
Preliminary analysis of change in fine motor function	Quality of Upper Extremity Skills Test (QUEST)	Baseline, 3 months
Preliminary analysis of change in fine motor function	Quality of Upper Extremity Skills Test (QUEST)	Baseline, 12 months
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
Preliminary analysis of change in quality of life	Cerebral Palsy Quality of Life (CP-QoL-CHILD)	Baseline, 3 months
Preliminary analysis of change in quality of life	Cerebral Palsy Quality of Life (CP-QoL-CHILD)	Baseline,12 months
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

Collaborators and Investigators

• Sponsor: Murdoch Childrens Research Institute
• Collaborators: Sydney Children's Hospitals Network; Monash Health; Cerebral Palsy Alliance; The Royal Children's Hospital; Children's Health Queensland
• Investigators: Principal Investigator: Dinah Reddihough, MBChB, MD, Group Leader

Publications and helpful links

General Publications

• Crompton K, Novak I, Fahey M, Badawi N, Lee KJ, Mechinaud-Heloury F, Edwards P, Colditz P, Soosay Raj T, Hough J, Wang X, Paget S, Hsiao KC, Anderson P, Reddihough D. Safety of sibling cord blood cell infusion for children with cerebral palsy. Cytotherapy. 2022 Sep;24(9):931-939. doi: 10.1016/j.jcyt.2022.01.003. Epub 2022 Feb 19.
• Crompton K, Novak I, Fahey M, Badawi N, Wallace E, Lee K, Mechinaud-Heloury F, Colditz PB, Elwood N, Edwards P, Reddihough D. Single group multisite safety trial of sibling cord blood cell infusion to children with cerebral palsy: study protocol and rationale. BMJ Open. 2020 Mar 8;10(3):e034974. doi: 10.1136/bmjopen-2019-034974.

Study record dates

Study Major Dates

• Study Start (Actual): March 1, 2016
• Primary Completion (Actual): September 1, 2019
• Study Completion (Actual): December 1, 2020

Study Registration Dates

• First Submitted: February 26, 2017
• First Submitted That Met QC Criteria: March 15, 2017
• First Posted (Actual): March 22, 2017

Study Record Updates

• Last Update Posted (Actual): January 20, 2021
• Last Update Submitted That Met QC Criteria: January 17, 2021
• Last Verified: January 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Brain Damage, Chronic; Cerebral Palsy
• Other Study ID Numbers: HREC/14/RCHM/38; RCH ID 34210; U1111-1179-9253 (Other Identifier: WHO Universal Trial Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Yes
• IPD Plan Description: The de-identified data set collected for this analysis of the SCUBI-CP trial will be available six months after publication of the primary outcome. The study protocol, analysis plan and consent forms will also be available. The data may be obtained from the Murdoch Children's Research Institute. Prior to releasing any data the following are required: a data access agreement must be signed between relevant parties, the SCUBI-CP Trial Steering Committee must see and approve the analysis plan describing how the data will be analysed, there must be an agreement around appropriate acknowledgement and any additional costs involved must be covered. Should the Trial Steering Committee be unavailable, this role is delegated to the Murdoch Children's Research Institute. Data will only be shared with a recognised research institution which has approved the proposed analysis plan.
• IPD Sharing Time Frame: 6 months after publication of primary outcome
• IPD Sharing Access Criteria: 1) Data access agreement; 2) approval by Trial Steering Committee; 3) recognised research institutions.
• IPD Sharing Supporting Information Type: Study Protocol; Statistical Analysis Plan (SAP); Informed Consent Form (ICF)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No