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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03087110
Recruitment Status : Active, not recruiting
First Posted : March 22, 2017
Last Update Posted : September 16, 2019
Sponsor:
Collaborators:
The Royal Children's Hospital Melbourne
Children's Health Queensland
Monash Health
Sydney Children's Hospitals Network
Cerebral Palsy Alliance
Information provided by (Responsible Party):
Murdoch Childrens Research Institute

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.


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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 12 participants
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 : March 2016
Estimated Primary Completion Date : September 2019
Estimated Study Completion Date : May 2020

Resource links provided by the National Library of Medicine


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)




Primary Outcome Measures :
  1. Number of participants with abnormal clinical assessment and/or laboratory values [ Time Frame: 12 months ]
    Safety


Secondary Outcome Measures :
  1. Preliminary analysis of change in gross motor function [ Time Frame: Baseline, 3 months ]
    Gross Motor Function Measure (GMFM-66)

  2. Preliminary analysis of change in gross motor function [ Time Frame: Baseline, 12 months ]
    Gross Motor Function Measure (GMFM-66)

  3. Preliminary analysis of change in fine motor function [ Time Frame: Baseline, 3 months ]
    Quality of Upper Extremity Skills Test (QUEST)

  4. Preliminary analysis of change in fine motor function [ Time Frame: Baseline, 12 months ]
    Quality of Upper Extremity Skills Test (QUEST)

  5. Preliminary analysis of change in cognitive function [ Time Frame: 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 [ Time Frame: Baseline, 3 months ]
    Cerebral Palsy Quality of Life (CP-QoL-CHILD)

  7. Preliminary analysis of change in quality of life [ Time Frame: 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 [ Time Frame: 3 months ]
    Chimerism study to detect the longevity of infused cells



Information from the National Library of Medicine

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03087110


Locations
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Australia, Queensland
Lady Cilento Children's Hospital
Brisbane, Queensland, Australia, 4101
Australia, Victoria
The Royal Children's Hospital
Melbourne, Victoria, Australia, 3052
Sponsors and Collaborators
Murdoch Childrens Research Institute
The Royal Children's Hospital Melbourne
Children's Health Queensland
Monash Health
Sydney Children's Hospitals Network
Cerebral Palsy Alliance
Investigators
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Principal Investigator: Dinah Reddihough, MBChB, MD Group leader
  Study Documents (Full-Text)

Documents provided by Murdoch Childrens Research Institute:
Study Protocol  [PDF] March 6, 2017


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Responsible Party: Murdoch Childrens Research Institute
ClinicalTrials.gov Identifier: NCT03087110     History of Changes
Other Study ID Numbers: HREC/14/RCHM/38; RCH ID 34210
U1111-1179-9253 ( Other Identifier: WHO Universal Trial Number )
First Posted: March 22, 2017    Key Record Dates
Last Update Posted: September 16, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
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.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (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.

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cerebral Palsy
Brain Damage, Chronic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases