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| ID | Type | Description | Link |
|---|---|---|---|
| U1111-1179-9253 | Other Identifier | WHO Universal Trial Number |
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| Name | Class |
|---|---|
| Royal Children's Hospital | OTHER |
| Children's Health Queensland | UNKNOWN |
| Monash Health | OTHER |
| Sydney Children's Hospitals Network |
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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.
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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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cord blood infusion | Experimental | Matched sibling donor cord blood cell infusion |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Matched sibling donor cord blood cell infusion | Biological | Single dose intravenous infusion of 12/12 HLA matched sibling donor cord blood cells (>1x10^7 cells/kg) |
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| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with abnormal clinical assessment and/or laboratory values | Safety | 12 months |
| 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) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Dinah Reddihough, MBChB, MD | Group leader | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Lady Cilento Children's Hospital | Brisbane | Queensland | 4101 | Australia | ||
| The Royal Children's Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 35193825 | Derived | 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. | |
| 32152173 |
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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.
6 months after publication of primary outcome
1) Data access agreement; 2) approval by Trial Steering Committee; 3) recognised research institutions.
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Mar 6, 2017 | Jul 3, 2018 | Prot_000.pdf |
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| ID | Term |
|---|---|
| D002547 | Cerebral Palsy |
| ID | Term |
|---|---|
| D001925 | Brain Damage, Chronic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
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| OTHER |
| Cerebral Palsy Alliance | OTHER |
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| 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 |
| Melbourne |
| Victoria |
| 3052 |
| Australia |
| 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. |