Clinical trial NCT03087110: Stem Cells in Umbilical Blood Infusion for CP
Official title: Safety Study of Sibling Cord Blood Cell Infusion to Children With Cerebral Palsy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Cerebral Palsy
Lead sponsor: Murdoch Childrens Research Institute
Sponsor class: OTHER
Collaborators: name: Royal Children's Hospital
name: Children's Health Queensland
name: Monash Health
name: Sydney Children's Hospitals Network
name: Cerebral Palsy Alliance
First posted: 2017-03-22
Enrollment: 12
Sex: ALL
Minimum age (years): 1
Maximum age (years): 16
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Number of participants with abnormal clinical assessment and/or laboratory values
timeFrame: 12 months
Secondary outcomes:
measure: Preliminary analysis of change in gross motor function
timeFrame: Baseline, 3 months
measure: Preliminary analysis of change in gross motor function
timeFrame: Baseline, 12 months
measure: Preliminary analysis of change in fine motor function
timeFrame: Baseline, 3 months
measure: Preliminary analysis of change in fine motor function
timeFrame: Baseline, 12 months
measure: Preliminary analysis of change in cognitive function
timeFrame: Baseline, 12 months
measure: Preliminary analysis of change in quality of life
timeFrame: Baseline, 3 months
measure: Preliminary analysis of change in quality of life
timeFrame: Baseline,12 months
measure: Digital PCR analysis of peripheral blood cellular DNA to determine the fraction of donor DNA in circulation
timeFrame: 3 months
Other outcomes:
Locations:
city: Brisbane
country: Australia
facility: Lady Cilento Children's Hospital
state: Queensland
city: Melbourne
country: Australia
facility: The Royal Children's Hospital
state: Victoria
Study officials:
affiliation: Group leader
name: Dinah Reddihough, MBChB, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: 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.