Cerebral PalsyPhase 1SCUBI-CPNCT03087110
Stem Cells in Umbilical Blood Infusion for CP
A Phase 1 study testing Matched sibling donor cord blood cell infusion in people with Cerebral Palsy, run by Murdoch Childrens Research Institute at 2 sites in Australia.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 year to 16 years
- Conditions the study is for
- Cerebral Palsy
- What is being tested?
- Matched sibling donor cord blood cell infusion
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 2 sitesAustralia
- Study dates
- Mar 2016to Dec 2020
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Show all 12Show fewer
- 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
Trial locations and contacts
2 sites in 1 country.
Australia2 sites
Lady Cilento Children's Hospital
Brisbane, Queensland, 4101
The Royal Children's Hospital
Melbourne, Victoria, 3052
About this study
Official title: 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.
Read the 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.
- Conditions
- Cerebral Palsy
- Being tested
- Matched sibling donor cord blood cell infusion
- Medical subject headings
- Cerebral Palsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Matched sibling donor cord blood cell infusionBiological
Single dose intravenous infusion of 12/12 HLA matched sibling donor cord blood cells (>1x10^7 cells/kg)
| Study group | Role | What they receive |
|---|---|---|
| Cord blood infusion | Experimental | Matched sibling donor cord blood cell infusion Receives: Biological: Matched sibling donor cord blood cell infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with abnormal clinical assessment and/or laboratory values
Safety
Measured: 12 months
Secondary outcomes (8)
Preliminary analysis of change in gross motor function
Gross Motor Function Measure (GMFM-66)
Measured: Baseline, 3 months
Preliminary analysis of change in gross motor function
Gross Motor Function Measure (GMFM-66)
Measured: Baseline, 12 months
Preliminary analysis of change in fine motor function
Quality of Upper Extremity Skills Test (QUEST)
Measured: Baseline, 3 months
Preliminary analysis of change in fine motor function
Quality of Upper Extremity Skills Test (QUEST)
Measured: 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.
Measured: Baseline, 12 months
Preliminary analysis of change in quality of life
Cerebral Palsy Quality of Life (CP-QoL-CHILD)
Measured: Baseline, 3 months
Preliminary analysis of change in quality of life
Cerebral Palsy Quality of Life (CP-QoL-CHILD)
Measured: 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
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Mar 2017
- Study startMar 2016
- Primary completionSep 2019
- Study completionDec 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Cerebral PalsyStudies of the same condition, open and under way.
- More trials run by Murdoch Childrens Research InstituteThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Murdoch Childrens Research Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Royal Children's Hospital, Children's Health Queensland, Monash Health, Sydney Children's Hospitals Network, Cerebral Palsy Alliance
- Responsible party
- Sponsor
- Investigators
- Dinah Reddihough, MBChB, MD, Principal investigator, Group leader
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03087110
- Sponsor's protocol ID
- HREC/14/RCHM/38; RCH ID 34210
- Other IDs
- U1111-1179-9253
- Acronym
- SCUBI-CP
- Status last verified
- Jan 2021
- First submitted
- 26 Feb 2017
- Last updated
- 20 Jan 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
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.
PubMed 35193825Crompton 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.
PubMed 32152173
Study at a glance
From the registry record, last updated by the sponsor on 20 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety Study of Sibling Cord Blood Cell Infusion to Children With Cerebral Palsy
- ClinicalTrials.gov ID
- NCT03087110
- Acronym / sponsor ID
- SCUBI-CP / HREC/14/RCHM/38; RCH ID 34210
- Conditions
- Cerebral Palsy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Murdoch Childrens Research InstituteUniversity, hospital or other
- Collaborators
- Royal Children's Hospital, Children's Health Queensland, Monash Health, Sydney Children's Hospitals Network, Cerebral Palsy Alliance
- Study start
- Mar 2016 (actual)
- Primary completion
- Sep 2019 (actual)
- Study completion
- Dec 2020 (actual)
- First posted
- 22 Mar 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cord blood infusion | Experimental | Biological: Matched sibling donor cord blood cell infusionMatched sibling donor cord blood cell infusion |
Who may qualify
- Ages
- 1 year to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (12 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Matched sibling donor cord blood cell infusionSingle dose intravenous infusion of 12/12 HLA matched sibling donor cord blood cells (>1x10^7 cells/kg) | Biological | Cord blood infusion |
Primary outcome
Number of participants with abnormal clinical assessment and/or laboratory values
Time frame: 12 months
Safety
8 secondary outcomes
Preliminary analysis of change in gross motor function
Time frame: Baseline, 3 months
Preliminary analysis of change in gross motor function
Time frame: Baseline, 12 months
Preliminary analysis of change in fine motor function
Time frame: Baseline, 3 months
Preliminary analysis of change in fine motor function
Time frame: Baseline, 12 months
Preliminary analysis of change in cognitive function
Time frame: Baseline, 12 months
Preliminary analysis of change in quality of life
Time frame: Baseline, 3 months
Preliminary analysis of change in quality of life
Time frame: Baseline,12 months
Digital PCR analysis of peripheral blood cellular DNA to determine the fraction of donor DNA in circulation
Time frame: 3 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jan 2021.
- Derived from this study: PubMed 35193825Crompton 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.
- Derived from this study: PubMed 32152173Crompton 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.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Dinah Reddihough, MBChB, MD principal investigator
Group leader
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Matched sibling donor cord blood cell infusion in people with Cerebral Palsy, run by Murdoch Childrens Research Institute at 2 sites in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with abnormal clinical assessment and/or laboratory values (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03087110, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jan 2021.