Cerebral PalsyNCT03414697
Transplantation of Umbilical Cord-derived Mesenchymal Stem Cells Via Different Routes
A study testing UC-MSCs in people with Cerebral Palsy, run by The First Affiliated Hospital of Dalian Medical University at 1 site in China.
Trial summary
To preliminarily evaluate the efficacy and safety of umbilical cord-derived mesenchymal stem cells (UC-MSCs), and compare the efficacy of UC-MSCs administered through the intravenous, intrathecal, and intranasal routes, in the treatment of cerebral palsy in children. Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- Cerebral Palsy
- What is being tested?
- UC-MSCs
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 44 participants
- Where
- 1 siteChina
- Study dates
- Apr 2018to Dec 2020
- Phase
- Interventional
- 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 2 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients diagnosed with hypoxic/ischemic cerebral palsy, primarily including birth asphyxia and premature children
- Patients with spastic quadriplegia
- Patients with moderate to severe cerebral palsy, GMFM scores of 2-3
- Age at 2-18 years
- Provision of signed informed consent by legal representatives of the child prior to start of the study
Who cannot join
- Those with systemic diseases that likely interfere with the treatment or child's compliance
- Those complicated by life-threatening diseases of any organ
- Those with brain deformity
- Those with uncontrolled epilepsy
- Those with abnormal behavior or mood disorders
- Those with allergies especially those who are allergic to blood products
- Those are infected with infectious diseases
- Those who had underwent a craniocerebral surgery in other clinical trials
Trial locations and contacts
1 site in 1 country.
China1 site
The First Affiliated Hospital of Dalian Medical University
Dalian, Liaoning, 116011
Jing Liu, Ph.D, 86041184394568, liujing.dlrmc@hotmail.com
About this study
Official title: Transplantation of Umbilical Cord-derived Mesenchymal Stem Cells Via Different Routes for the Treatment of Cerebral Palsy in Children
To preliminarily evaluate the efficacy and safety of umbilical cord-derived mesenchymal stem cells (UC-MSCs), and compare the efficacy of UC-MSCs administered through the intravenous, intrathecal, and intranasal routes, in the treatment of cerebral palsy in children.
Read the detailed description
Cerebral palsy refers to a neurological disorder caused by a non-progressive brain injury or malformation that occurs in early childhood. It can result in central motor deficits, developmental retardation, abnormal posture, abnormal muscular strength, abnormal muscle tone and/or dysreflexia. It has a high disability rate. There is currently no effective treatment for cerebral palsy. Conventional treatments for cerebral palsy are often tiresome and expensive and have a slow onset of action. Stem cells have been recently used in the treatment of cerebral palsy. This provides a novel method for the treatment of cerebral palsy. According to the existing clinical studies, neural stem cells, bone marrow-derived mesenchymal stem cells, and umbilical cord-derived mesenchymal stem cells (UC-MSCs) are mainly used as the seed cells for the treatment of cerebral palsy. UC-MSCs are the most promising seed cells for the treatment of cerebral palsy because of the advantages including rich sources, ease of harvesting, short doubling time, low immunogenicity, long-time survival post-transplantation, and no ethical issues. UC-MSCs have been widely used to treat Parkinson's disease, rheumatoid arthritis, traumatic brain injury, aplastic anemia, and decompensated liver disease. However,only two studies, and performed only in China, are reported on the treatment of cerebral palsy with UC-MSCs. One from the Hospital 463 of PLA reported 51 patients with cerebral palsy whose symptoms had not been obviously improved after 1 year of rehabilitative treatment. These patients received intrathecal injection of UC-MSCs for three times, once a week, followed by one injection of UC-MSCs via the peripheral vein in the fourth week. Four UC-MSCs injections, once per week, were considered as one course of treatment. Activities of daily living scale score was compared between before and after treatment to evaluate efficacy. Another study is a case report from China. In this report, a combined intravenous and intrathecal injection of UC-MSCs was used to treat cerebral palsy in a 5-year-old child. 28-month follow-up results revealed that the child's gross motor function, immune function, muscle strength, and language ability improved and adverse reactions were not obvious.
- Conditions
- Cerebral Palsy
- Being tested
- Control group, UC-MSCs
- Keywords
- umbilical cord-derived mesenchymal stem cells, cerebral palsy
- Medical subject headings
- Cerebral Palsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Control groupOther
The control group received routine rehabilitation treatments including acupuncture, massage, Bobath therapy, and Vojat therapy.
UC-MSCsProcedure or surgery
Injection of umbilical cord-derived mesenchymal stem cells (UC-MSCs) (at least 1×10^7), once every 2 weeks, one course of treatment including two injections within 4 weeks, a total of two courses of treatment.
| Study group | Role | What they receive |
|---|---|---|
| Control group | Other | Routine rehabilitation treatments Receives: Other: Control group |
| Intravenous UC-MSCs group | Experimental | Injection of UC-MSCs via the peripheral vein. Receives: Procedure: UC-MSCs |
| Intrathecal UC-MSCs group | Experimental | Injection of UC-MSCs via the intrathecal route. Receives: Procedure: UC-MSCs |
| Intranasal UC-MSCs group | Experimental | Injection of UC-MSCs via the nasal route. Receives: Procedure: UC-MSCs |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Gross Motor Function Measure (GMFM)-88 item score
a 4-point scale that is divided into five categories including lying and rolling, sitting, crawling and kneeling, standing, walking, running, and jumping. A score of 0 indicates that the child does not initiate the task; 1 indicates that the child initiates the task (completes < 10% of the activity); 2 indicates that the child partially completes the task (completes from 10 to 99% of the activity); 3 indicates that the child completes the task (100%). The sum of score of the five categories is used as the evaluation outcome.It is used to evaluate gross motor function.
Measured: 12 months after 2 courses of treatment with UC-MSCs.
Secondary outcomes (4)
Fine Motor Function Measure scale (FMFM) score
To evaluate fine motor function.The scale is divided into 45 items covering visual tracing (7 items), upper extremity range of motion (8 items), grab ability (8 items), operational ability (10 items), and hand-eye coordination (12 items). The FMFM is a 4-point scale with the score range of 0-3. The original total score is 135. The summed score for evaluating fine motor function measure is 0-100.
Measured: 1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.
The Modified Ashworth scale (MAS) score
To evaluate muscle tone. The MAS score is divided into six grades: 0, 1, 1+, 2, 3 and 4. Passive full ranges of motion exercises are performed by moving the knee and ankle until the patient feel a slight resistance from hamstrings and plantar flexor muscles (soleus muscle and gastrocnemius muscle). The average score is calculated across three repeated measurements.
Measured: 1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.
Gesell Development Schedule score
To evaluate neurobehavioral development.
Measured: 1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.
Incidence of adverse reactions
Liver and kidney dysfunction, fever, infection, vomiting.
Measured: 1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jan 2018
- Study startApr 2018 (estimated)
- Primary completionSep 2020 (estimated)
- Study completionDec 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 44 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The First Affiliated Hospital of Dalian Medical University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Liu Jing, The First Affiliated Hospital of Dalian Medical University
- Investigators
- Jing Liu, Ph.D, Principal investigator, The First Affiliated Hospital of Dalian Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03414697
- Sponsor's protocol ID
- FirstHospitalDalianMU004
- Status last verified
- Jan 2018
- First submitted
- 22 Jan 2018
- Last updated
- 30 Jan 2018
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Jan 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Transplantation of Umbilical Cord-derived Mesenchymal Stem Cells Via Different Routes for the Treatment of Cerebral Palsy in Children
- ClinicalTrials.gov ID
- NCT03414697
- Acronym / sponsor ID
- FirstHospitalDalianMU004
- Conditions
- Cerebral Palsy
- Keywords
- umbilical cord-derived mesenchymal stem cells, cerebral palsy
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 44 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital of Dalian Medical UniversityUniversity, hospital or other
- Study start
- Apr 2018 (estimated)
- Primary completion
- Sep 2020 (estimated)
- Study completion
- Dec 2020 (estimated)
- First posted
- 30 Jan 2018 (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.
To preliminarily evaluate the efficacy and safety of umbilical cord-derived mesenchymal stem cells (UC-MSCs), and compare the efficacy of UC-MSCs administered through the intravenous, intrathecal, and intranasal routes, in the treatment of cerebral palsy in children.
Detailed description
Cerebral palsy refers to a neurological disorder caused by a non-progressive brain injury or malformation that occurs in early childhood. It can result in central motor deficits, developmental retardation, abnormal posture, abnormal muscular strength, abnormal muscle tone and/or dysreflexia. It has a high disability rate. There is currently no effective treatment for cerebral palsy. Conventional treatments for cerebral palsy are often tiresome and expensive and have a slow onset of action. Stem cells have been recently used in the treatment of cerebral palsy. This provides a novel method for the treatment of cerebral palsy. According to the existing clinical studies, neural stem cells, bone marrow-derived mesenchymal stem cells, and umbilical cord-derived mesenchymal stem cells (UC-MSCs) are mainly used as the seed cells for the treatment of cerebral palsy. UC-MSCs are the most promising seed cells for the treatment of cerebral palsy because of the advantages including rich sources, ease of harvesting, short doubling time, low immunogenicity, long-time survival post-transplantation, and no ethical issues. UC-MSCs have been widely used to treat Parkinson's disease, rheumatoid arthritis, traumatic brain injury, aplastic anemia, and decompensated liver disease. However,only two studies, and performed only in China, are reported on the treatment of cerebral palsy with UC-MSCs. One from the Hospital 463 of PLA reported 51 patients with cerebral palsy whose symptoms had not been obviously improved after 1 year of rehabilitative treatment. These patients received intrathecal injection of UC-MSCs for three times, once a week, followed by one injection of UC-MSCs via the peripheral vein in the fourth week. Four UC-MSCs injections, once per week, were considered as one course of treatment. Activities of daily living scale score was compared between before and after treatment to evaluate efficacy. Another study is a case report from China. In this report, a combined intravenous and intrathecal injection of UC-MSCs was used to treat cerebral palsy in a 5-year-old child. 28-month follow-up results revealed that the child's gross motor function, immune function, muscle strength, and language ability improved and adverse reactions were not obvious.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Control group | Other | Other: Control groupRoutine rehabilitation treatments |
| Intravenous UC-MSCs group | Experimental | Procedure: UC-MSCsInjection of UC-MSCs via the peripheral vein. |
| Intrathecal UC-MSCs group | Experimental | Procedure: UC-MSCsInjection of UC-MSCs via the intrathecal route. |
| Intranasal UC-MSCs group | Experimental | Procedure: UC-MSCsInjection of UC-MSCs via the nasal route. |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Patients diagnosed with hypoxic/ischemic cerebral palsy, primarily including birth asphyxia and premature children
- Patients with spastic quadriplegia
- Patients with moderate to severe cerebral palsy, GMFM scores of 2-3
- Age at 2-18 years
- Provision of signed informed consent by legal representatives of the child prior to start of the study
Key exclusion criteria (8 listed)
- Those with systemic diseases that likely interfere with the treatment or child's compliance
- Those complicated by life-threatening diseases of any organ
- Those with brain deformity
- Those with uncontrolled epilepsy
- Those with abnormal behavior or mood disorders
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 |
|---|---|---|
| Control groupThe control group received routine rehabilitation treatments including acupuncture, massage, Bobath therapy, and Vojat therapy. | Other | Control group |
| UC-MSCsInjection of umbilical cord-derived mesenchymal stem cells (UC-MSCs) (at least 1×10^7), once every 2 weeks, one course of treatment including two injections within 4 weeks, a total of two courses of treatment. | Procedure or surgery | Intranasal UC-MSCs group, Intrathecal UC-MSCs group, Intravenous UC-MSCs group |
Primary outcome
The Gross Motor Function Measure (GMFM)-88 item score
Time frame: 12 months after 2 courses of treatment with UC-MSCs.
a 4-point scale that is divided into five categories including lying and rolling, sitting, crawling and kneeling, standing, walking, running, and jumping. A score of 0 indicates that the child does not initiate the task; 1 indicates that the child initiates the task (completes < 10% of the activity); 2 indicates that the child partially completes the task (completes from 10 to 99% of the activity); 3 indicates that the child completes the task (100%). The sum of score of the five categories is used as the evaluation outcome.It is used to evaluate gross motor function.
4 secondary outcomes
Fine Motor Function Measure scale (FMFM) score
Time frame: 1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.
The Modified Ashworth scale (MAS) score
Time frame: 1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.
Gesell Development Schedule score
Time frame: 1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.
Incidence of adverse reactions
Time frame: 1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jan 2018.
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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.
Jing Liu, Ph.D principal investigator
The First Affiliated Hospital of Dalian Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing UC-MSCs in people with Cerebral Palsy, run by The First Affiliated Hospital of Dalian Medical University at 1 site in China.
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 5 inclusion and 8 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: status not confirmed, as of the sponsor's last update on 30 Jan 2018. 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 China. 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.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Jing Liu, Ph.D (86041184394568, liujing.dlrmc@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The Gross Motor Function Measure (GMFM)-88 item score (time frame: 12 months after 2 courses of treatment with UC-MSCs.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 30 Jan 2018.