Cerebral PalsyPhase 1/2NCT03130816
Mechanism of Allogeneic UCB Therapy in Cerebral Palsy
A Phase 1/2 study testing allogeneic cord blood transplantation in people with Cerebral Palsy and Child Development, run by Bundang CHA Hospital at 1 site in South Korea.
Trial summary
In our prior study on the therapeutic mechanism of UCB, changes in cytokine levels were observed but the results are inconclusive and further studies on animal models and changes of protein expression before and after UCB therapy in the clinical settings are required. Read more about this study
- Who is it for?
- Ages 10 months to 20 years
- Conditions the study is for
- Cerebral Palsy, Child Development
- What is being tested?
- allogeneic cord blood transplantation
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 90 participants
- Where
- 1 siteSouth Korea
- Study dates
- 29 Jul 2015to 21 May 2019
- Phase
- Phase 1/2
- 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 10 months to 20 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosed with cerebral palsy
- Age of ≥10 months and ≤20 years
- Mismatch in HLA-A, B, and DR ≤3, and total nucleated cell count ≥2x107/kg. If the cell count is less than given values, more than 2 units may be used.
- Voluntary decision to participation in the study with informed consent agreed and obtained from the subject's representative.
- Patient and/or representatives are both willing and capable of being hospitalized according to the schedule specified in the protocol and continue the study for 12 months after study entry.
- If the patient has participated in another clinical trial, at least 3 months should have passed since end of the study.
Who cannot join
- Current aspiration pneumonia
- Known genetic disease
- History of hypersensitivity reaction to any study drugs pertinent to the study
- Patient with severe convulsion disease who has clinical convulsion despite combination therapy with 3 or more agents
- Uncontrolled hypertension defined as systolic blood pressure >115 mmHg and/or diastolic blood pressure >70 mmHg
- Hepatic impairment defined as asparate aminotransferase (AST) >55 IU/L and/or alanine aminotrasferase (ALT) >45 IU/L
Show all 10Show fewer
- Renal impairment defined as creatinine (Cr) ≥1.3 mg/dL
- Presence of diagnosed or suspected malignant tumor and/or hematologic malignancy
- Non-compliance with study visits specified in the protocol or poor compliance of care-giver.
- Any factors not specified above that the principal investigator determines medically inadequate for participation in this study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Mechanism of Allogeneic Umbilical Cord Blood Therapy in Cerebral Palsy
In our prior study on the therapeutic mechanism of UCB, changes in cytokine levels were observed but the results are inconclusive and further studies on animal models and changes of protein expression before and after UCB therapy in the clinical settings are required.
The changes in protein expression will be assessed by multiplex RT-PCR mRNA assay. Clinical efficacy of UCB therapy will be evaluated with various functional assessment tools. Factors regarding UCB therapy (number of transplanted cells, HLA matching status, serum level of immunosuppressant, etc.) and patient factors (age, functional status, etc.) will be analyzed for correlation with protein expression after UCB therapy. Several target proteins for analysis are available. Pentraxin and toll-like receptor (TLR) 4 are receptors modulating intrinsic immune reaction and was shown to have a significant correlation with clinical efficacy of stem cell therapy. Ubiquitine is a regulatory protein that combines with the target protein and affects its degradation, interaction, localization and activation. The ubiquitine system controls total protein quantity for homeostasis and can be found in all tissues. Deubiquitination (DUB) enzyme down-regulates this ubiquitine and is known to modulate all cellular changes
- Conditions
- Cerebral Palsy, Child Development
- Being tested
- allogeneic cord blood transplantation
- Keywords
- Umbilical cord blood transplantation, Cytokine changes
- Medical subject headings
- Cerebral Palsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
allogeneic cord blood transplantationBiological
UCB with total nucleated cell count ≤ 7x108/kg will be used for this clinical trial. Suitable UCB (i.e., containing total nucleated cell count ≥2x107/kg with three or less mismatch among HLA-A, -B, and -DR) will be selected. This criterion was selected upon the rationale that even though minimal HLA mismatch is preferred, prior studies indicate significant effects of UCB therapy for patients with 3 HLA mismatches.
| Study group | Role | What they receive |
|---|---|---|
| allogeneic cord blood transplantation | Experimental | Intravenous(IV) infusion will be done by the following method A. After 4 hours of fasting, subjects will be sedated with chloral hydrate (Pocral®) syrup B. Intravenous infusion will be conducted in stem cell center, CHA Bundang Medical Center and the therapy will be performed by the Principal Investigator or a physician delegated from the Principal Investigator. The physician conducting the infusion will not participate in the efficacy and result analysis of this study.
C. Oxygen saturation will be monitored during therapy. Receives: Biological: allogeneic cord blood transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change of GMFM
Gross motor function measure measured at baseline before UCB administration is compared to the score measured at months 3, 6, and 12 after UCB treatment.
Measured: Baseline before UCB administration, months 3, 6, and 12 after UCB treatment
Secondary outcomes (2)
Change of mRNA assay
Separate peripheral blood mononuclear cell (PBMC) from the patients' blood sample and screen for changes in protein enzymes including those related to DUB at the mRNA level after UCB therapy.
Measured: Change between the baseline level before UCB therapy and levels after UCB administration at 2 days, 1 week, 5 weeks, and 12 months
Change of GMPM
Dissociated Movement, Coordination, Alignment, Weight shift, and Stability are rated with GMPM (Gross Motor Performance Measure). Each raw score (1-5 point) and converted percent scores are evaluated. Total score is 100(%), higher scores indicate better function. GMPM measured at baseline before UCB administration is compared to the score measured at months 3, 6, and 12 after UCB treatment.
Measured: Baseline before UCB administration, months 3, 6, and 12 after UCB treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Apr 2017
- Study start29 Jul 2015
- Primary completion21 May 2019
- Study completion21 May 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cerebral PalsyStudies of the same condition, open and under way.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Patients diagnosed with cerebral palsy volunteered for participation in this study.
- Masking
- None (open label)
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Bundang CHA Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, MinYoung Kim, MD, PhD, Bundang CHA Hospital
- Investigators
- MinYoung Kim, MD, PhD, Principal investigator, CHA University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03130816
- Sponsor's protocol ID
- 2015-06-093
- Status last verified
- Nov 2020
- First submitted
- 20 Apr 2017
- Last updated
- 4 Nov 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Suh MR, Min K, Cho KH, Kim J, Lim I, Park M, Noh EM, Kim MY. Maintenance of the synergistic effects of cord blood cells and erythropoietin combination therapy after additional cord blood infusion in children with cerebral palsy: 1-year open-label extension study of randomized placebo-controlled trial. Stem Cell Res Ther. 2023 Dec 12;14(1):362. doi: 10.1186/s13287-023-03600-4.
PubMed 38087394
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Mechanism of Allogeneic Umbilical Cord Blood Therapy in Cerebral Palsy
- ClinicalTrials.gov ID
- NCT03130816
- Acronym / sponsor ID
- 2015-06-093
- Conditions
- Cerebral Palsy, Child Development
- Keywords
- Umbilical cord blood transplantation, Cytokine changes
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 90 participants (estimated)
- Lead sponsor
- Bundang CHA HospitalUniversity, hospital or other
- Study start
- 29 Jul 2015 (actual)
- Primary completion
- 21 May 2019 (actual)
- Study completion
- 21 May 2019 (actual)
- First posted
- 27 Apr 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.
In our prior study on the therapeutic mechanism of UCB, changes in cytokine levels were observed but the results are inconclusive and further studies on animal models and changes of protein expression before and after UCB therapy in the clinical settings are required.
The changes in protein expression will be assessed by multiplex RT-PCR mRNA assay. Clinical efficacy of UCB therapy will be evaluated with various functional assessment tools. Factors regarding UCB therapy (number of transplanted cells, HLA matching status, serum level of immunosuppressant, etc.) and patient factors (age, functional status, etc.) will be analyzed for correlation with protein expression after UCB therapy. Several target proteins for analysis are available. Pentraxin and toll-like receptor (TLR) 4 are receptors modulating intrinsic immune reaction and was shown to have a significant correlation with clinical efficacy of stem cell therapy. Ubiquitine is a regulatory protein that combines with the target protein and affects its degradation, interaction, localization and activation. The ubiquitine system controls total protein quantity for homeostasis and can be found in all tissues. Deubiquitination (DUB) enzyme down-regulates this ubiquitine and is known to modulate all cellular changes
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Patients diagnosed with cerebral palsy volunteered for participation in this study.
| Arm | Type | Receives |
|---|---|---|
| allogeneic cord blood transplantation | Experimental | Biological: allogeneic cord blood transplantationIntravenous(IV) infusion will be done by the following method A. After 4 hours of fasting, subjects will be sedated with chloral hydrate (Pocral®) syrup B. Intravenous infusion will be conducted in stem cell center, CHA Bundang Medical Center and the therapy will be performed by the Principal Investigator or a physician delegated from the Principal Investigator. The physician conducting the infusion will not participate in the efficacy and result analysis of this study. C. Oxygen saturation will be monitored during therapy. |
Who may qualify
- Ages
- 10 months to 20 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Diagnosed with cerebral palsy
- Age of ≥10 months and ≤20 years
- Mismatch in HLA-A, B, and DR ≤3, and total nucleated cell count ≥2x107/kg. If the cell count is less than given values, more than 2 units may be used.
- Voluntary decision to participation in the study with informed consent agreed and obtained from the subject's representative.
- Patient and/or representatives are both willing and capable of being hospitalized according to the schedule specified in the protocol and continue the study for 12 months after study entry.
Key exclusion criteria (10 listed)
- Current aspiration pneumonia
- Known genetic disease
- History of hypersensitivity reaction to any study drugs pertinent to the study
- Patient with severe convulsion disease who has clinical convulsion despite combination therapy with 3 or more agents
- Uncontrolled hypertension defined as systolic blood pressure >115 mmHg and/or diastolic blood pressure >70 mmHg
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 |
|---|---|---|
| allogeneic cord blood transplantationUCB with total nucleated cell count ≤ 7x108/kg will be used for this clinical trial. Suitable UCB (i.e., containing total nucleated cell count ≥2x107/kg with three or less mismatch among HLA-A, -B, and -DR) will be selected. This criterion was selected upon the rationale that even though minimal HLA mismatch is preferred, prior studies indicate significant effects of UCB therapy for patients with 3 HLA mismatches. | Biological | allogeneic cord blood transplantation |
Primary outcome
Change of GMFM
Time frame: Baseline before UCB administration, months 3, 6, and 12 after UCB treatment
Gross motor function measure measured at baseline before UCB administration is compared to the score measured at months 3, 6, and 12 after UCB treatment.
2 secondary outcomes
Change of mRNA assay
Time frame: Change between the baseline level before UCB therapy and levels after UCB administration at 2 days, 1 week, 5 weeks, and 12 months
Change of GMPM
Time frame: Baseline before UCB administration, months 3, 6, and 12 after UCB treatment
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 4 Nov 2020.
- Derived from this study: PubMed 38087394Suh MR, Min K, Cho KH, Kim J, Lim I, Park M, Noh EM, Kim MY. Maintenance of the synergistic effects of cord blood cells and erythropoietin combination therapy after additional cord blood infusion in children with cerebral palsy: 1-year open-label extension study of randomized placebo-controlled trial. Stem Cell Res Ther. 2023 Dec 12;14(1):362. doi: 10.1186/s13287-023-03600-4.
- Individual participant dataSharing planned: Undecided
Related trials for Cerebral Palsy
Not yet recruiting
Feasibility of a Robot-Guided Active Play Intervention for Children With Cerebral Palsy
Cerebral Palsy
RecruitingObservational study
Turkish Translation, Cross-Cultural Adaptation, Validity, and Reliability of the ABILHAND-Kids Young CP Scale
Cerebral Palsy
Not yet recruiting
Video-based Visual Feedback Combined With Task-oriented Physiotherapy Among Cerebral Palsy
Cerebral Palsy (CP)
Recruiting
Sensory Integration Therapy vs Conventional Physical Therapy in Children With Mixed Cerebral Palsy
Cerebral Palsy
Recruiting
Additional Effects on tDCS With AOT on Lower Limb Functions on Children With Diplegic CP
Diplegic Cerebral Palsy
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.
MinYoung Kim, MD, PhD principal investigator
CHA University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing allogeneic cord blood transplantation in people with Cerebral Palsy and Child Development, run by Bundang CHA Hospital at 1 site in South Korea.
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 10 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 4 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in South Korea. 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Change of GMFM (time frame: Baseline before UCB administration, months 3, 6, and 12 after UCB treatment). 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 NCT03130816, which also lists 1 linked publication.
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 4 Nov 2020; AzTrials last refreshed its copy on 8 Nov 2026.