NCT03087110

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
12

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Mar 2016

Longer than P75 for phase_1

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 1, 2016

Completed
12 months until next milestone

First Submitted

Initial submission to the registry

February 26, 2017

Completed
24 days until next milestone

First Posted

Study publicly available on registry

March 22, 2017

Completed
2.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2019

Completed
1.3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2020

Completed
Last Updated

January 20, 2021

Status Verified

January 1, 2021

Enrollment Period

3.5 years

First QC Date

February 26, 2017

Last Update Submit

January 17, 2021

Conditions

Outcome Measures

Primary Outcomes (1)

  • Number of participants with abnormal clinical assessment and/or laboratory values

    Safety

    12 months

Secondary Outcomes (8)

  • Preliminary analysis of change in gross motor function

    Baseline, 3 months

  • Preliminary analysis of change in gross motor function

    Baseline, 12 months

  • Preliminary analysis of change in fine motor function

    Baseline, 3 months

  • Preliminary analysis of change in fine motor function

    Baseline, 12 months

  • Preliminary analysis of change in cognitive function

    Baseline, 12 months

  • +3 more secondary outcomes

Study Arms (1)

Cord blood infusion

EXPERIMENTAL

Matched sibling donor cord blood cell infusion

Biological: Matched sibling donor cord blood cell infusion

Interventions

Single dose intravenous infusion of 12/12 HLA matched sibling donor cord blood cells (\>1x10\^7 cells/kg)

Cord blood infusion

Eligibility Criteria

Age1 Year - 16 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

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

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Lady Cilento Children's Hospital

Brisbane, Queensland, 4101, Australia

Location

The Royal Children's Hospital

Melbourne, Victoria, 3052, Australia

Location

Related Publications (2)

  • 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.

MeSH Terms

Conditions

Cerebral Palsy

Condition Hierarchy (Ancestors)

Brain Damage, ChronicBrain DiseasesCentral Nervous System DiseasesNervous System Diseases

Study Officials

  • Dinah Reddihough, MBChB, MD

    Group leader

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 26, 2017

First Posted

March 22, 2017

Study Start

March 1, 2016

Primary Completion

September 1, 2019

Study Completion

December 1, 2020

Last Updated

January 20, 2021

Record last verified: 2021-01

Data Sharing

IPD Sharing
Will share

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.

Shared Documents
STUDY PROTOCOL, SAP, ICF
Time Frame
6 months after publication of primary outcome
Access Criteria
1\) Data access agreement; 2) approval by Trial Steering Committee; 3) recognised research institutions.

Locations