Cerebral PalsyPhase 3CPNCT01929434
Efficacy of Stem Cell Transplantation Compared to Rehabilitation Treatment of Patients With Cerebral Paralysis
A Phase 3 study testing rehabilitation and stem cell injection in people with Cerebral Palsy, run by General Hospital of Chinese Armed Police Forces at 1 site in China.
Trial summary
Cerebral palsy (CP) is described as a group of permanent disorders affecting motor development and posture, resulting in activity limitation attributed to nonprogressive disturbances of the fetal or infant brain. Read more about this study
- Who is it for?
- Ages 1 year to 14 years
- Conditions the study is for
- Cerebral Palsy
- What is being tested?
- rehabilitation and stem cell injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 300 participants
- Where
- 1 siteChina
- Study dates
- Oct 2013to Dec 2016
- Phase
- Phase 3
- 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 14 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patients with diagnosis of cerebral palsy.
- Patients' curator must be able to give voluntary consent.
Who cannot join
- Intracranial infection.
- Severe respiratory and circulatory system diseases.
- Hematologic malignancies.
- Positive serological tests such as AIDS, hepatitis B virus, hepatitis C virus and syphilis （antigen or antibody）.
- Tumors.
- Genetic and metabolic diseases.
Trial locations and contacts
1 site in 1 country.
China1 site
General Hospital of Chinese People's Armed Police Forces
Beijing, Beijing Municipality, 100050
About this study
Official title: Efficacy of Stem Cell Transplantation Compared to Rehabilitation Treatment of Children With Cerebral Paralysis
Cerebral palsy (CP) is described as a group of permanent disorders affecting motor development and posture, resulting in activity limitation attributed to nonprogressive disturbances of the fetal or infant brain. The prevalence of cerebral palsy has increased among the children with low birth-weight, jaundice, respiratory distress and intrauterine infection and so on. The incidence of cerebral palsy is increasing gradually with increased neonatal survival rate. Although there are many kinds of functional therapy programs especially the rehabilitation treatment for cerebral palsy, their effects are limited. Increasing cerebral palsy patients become a heavy burden to the family and society. Stem cell based therapy, a new prospective therapy for central nervous system disorders, has the potential to repair the damaged brain tissue in patients with cerebral palsy.
In this study, 300 patients with cerebral palsy will be divided into three groups and the investigators will use mesenchymal stem cells derived from umbilical cord to treat 100 CP patients of them randomly. We will also follow up the other 100 patients who only receive rehabilitation treatment and another 100 patients who accept neither stem cell therapy nor rehabilitation treatment. On this basis, as the investigators we can compare the efficacy of cell therapy and rehabilitation treatments for cerebral palsy patients.
Multiple sources of assessment were used to ascertain and classify all cases of cerebral palsy. Particularly the Gross Motor Function Measure (GMFM) as an important valid and reliable outcome measure, has made it possible to evaluate the severity of movement disability，change over time and the effects of clinical interventions. It also will be the primary outcome measure in follow-up analysis of this study.
Read the detailed description
Patients enrolled in this study need to finish our whole follow-up survey for 12 months, which is carried out by clinical doctors, rehabilitators and epidemiologist.
- Conditions
- Cerebral Palsy
- Being tested
- rehabilitation, stem cell injection
- Keywords
- cerebral palsy, cell transplantation, rehabilitation
- Medical subject headings
- Cerebral Palsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rehabilitationOther
Also called phsical exercise rehabilitation
Patients only receive rehabilitation of physical therapy and occupational therapy.
stem cell injectionBiological
Mesenchymal stem cells derived from umbilical cord are transplanted directly into subarachnoid by Lumbar puncture.
| Study group | Role | What they receive |
|---|---|---|
| rehabilitation | Active comparator | Patients in the group accept rehabilitation for three weeks in hospital and other eleven months in their home under the guidance of physical therapist. Receives: Other: rehabilitation |
| control | No intervention | Patients receive no professional treatment in hospital or rehabilitation centre. |
| stem cell injection | Experimental | Patients in the group accept cell therapy including four times stem cells transplant via intrathecal injection. Receives: Biological: stem cell injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Gross Motor Function Measure Score
Gross Motor Function Measure-88 and Gross Motor Function Measure-66
Measured: Change from baseline at 12 monthes after enrollment or transplantation
Secondary outcomes (1)
Routine Blood Test and Biochemical Test
- red blood cell
- white blood cell
- platelet count
- glutamic pyruvic transaminase
- glutamic oxaloacetic transaminase
Measured: Change from baseline at 12 monthes after enrollment or transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Aug 2013 (estimated)
- Study startOct 2013
- Primary completionDec 2015
- Study completionDec 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 300 participants (actual)
Sponsor and investigators
- Lead sponsor
- General Hospital of Chinese Armed Police Forces
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Yihua An, Doctor, Study director, the General Hospital of Chinese People's Armed Police Forces
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01929434
- Sponsor's protocol ID
- 2013-05-13 CP III
- Acronym
- CP
- Status last verified
- Jul 2015
- First submitted
- 20 Aug 2013
- Last updated
- 27 Dec 2017
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Pharoah PO, Platt MJ, Cooke T. The changing epidemiology of cerebral palsy. Arch Dis Child Fetal Neonatal Ed. 1996 Nov;75(3):F169-73. doi: 10.1136/fn.75.3.f169.
PubMed 8976681Richards CL, Malouin F. Cerebral palsy: definition, assessment and rehabilitation. Handb Clin Neurol. 2013;111:183-95. doi: 10.1016/B978-0-444-52891-9.00018-X.
PubMed 23622163Himmelmann K. Epidemiology of cerebral palsy. Handb Clin Neurol. 2013;111:163-7. doi: 10.1016/B978-0-444-52891-9.00015-4. No abstract available.
PubMed 23622160Reddihough DS, Collins KJ. The epidemiology and causes of cerebral palsy. Aust J Physiother. 2003;49(1):7-12. doi: 10.1016/s0004-9514(14)60183-5.
PubMed 12600249Rethlefsen SA, Ryan DD, Kay RM. Classification systems in cerebral palsy. Orthop Clin North Am. 2010 Oct;41(4):457-67. doi: 10.1016/j.ocl.2010.06.005.
PubMed 20868878Lubis MU, Tjipta GD, Marbun MD, Saing B. Cerebral palsy. Paediatr Indones. 1990 Mar-Apr;30(3-4):65-70.
PubMed 2075008
Study at a glance
From the registry record, last updated by the sponsor on 27 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy of Stem Cell Transplantation Compared to Rehabilitation Treatment of Children With Cerebral Paralysis
- ClinicalTrials.gov ID
- NCT01929434
- Acronym / sponsor ID
- CP / 2013-05-13 CP III
- Conditions
- Cerebral Palsy
- Keywords
- cerebral palsy, cell transplantation, rehabilitation
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 300 participants (actual)
- Lead sponsor
- General Hospital of Chinese Armed Police ForcesUniversity, hospital or other
- Study start
- Oct 2013
- Primary completion
- Dec 2015 (actual)
- Study completion
- Dec 2016 (actual)
- First posted
- 28 Aug 2013 (estimated)
- 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.
Cerebral palsy (CP) is described as a group of permanent disorders affecting motor development and posture, resulting in activity limitation attributed to nonprogressive disturbances of the fetal or infant brain. The prevalence of cerebral palsy has increased among the children with low birth-weight, jaundice, respiratory distress and intrauterine infection and so on. The incidence of cerebral palsy is increasing gradually with increased neonatal survival rate. Although there are many kinds of functional therapy programs especially the rehabilitation treatment for cerebral palsy, their effects are limited. Increasing cerebral palsy patients become a heavy burden to the family and society. Stem cell based therapy, a new prospective therapy for central nervous system disorders, has the potential to repair the damaged brain tissue in patients with cerebral palsy.
In this study, 300 patients with cerebral palsy will be divided into three groups and the investigators will use mesenchymal stem cells derived from umbilical cord to treat 100 CP patients of them randomly. We will also follow up the other 100 patients who only receive rehabilitation treatment and another 100 patients who accept neither stem cell therapy nor rehabilitation treatment. On this basis, as the investigators we can compare the efficacy of cell therapy and rehabilitation treatments for cerebral palsy patients.
Multiple sources of assessment were used to ascertain and classify all cases of cerebral palsy. Particularly the Gross Motor Function Measure (GMFM) as an important valid and reliable outcome measure, has made it possible to evaluate the severity of movement disability，change over time and the effects of clinical interventions. It also will be the primary outcome measure in follow-up analysis of this study.
Detailed description
Patients enrolled in this study need to finish our whole follow-up survey for 12 months, which is carried out by clinical doctors, rehabilitators and epidemiologist.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| rehabilitation | Active comparator | Other: rehabilitationPatients in the group accept rehabilitation for three weeks in hospital and other eleven months in their home under the guidance of physical therapist. |
| control | No intervention | –Patients receive no professional treatment in hospital or rehabilitation centre. |
| stem cell injection | Experimental | Biological: stem cell injectionPatients in the group accept cell therapy including four times stem cells transplant via intrathecal injection. |
Who may qualify
- Ages
- 1 year to 14 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Patients with diagnosis of cerebral palsy.
- Patients' curator must be able to give voluntary consent.
Key exclusion criteria (6 listed)
- Intracranial infection.
- Severe respiratory and circulatory system diseases.
- Hematologic malignancies.
- Positive serological tests such as AIDS, hepatitis B virus, hepatitis C virus and syphilis （antigen or antibody）.
- Tumors.
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 |
|---|---|---|
| rehabilitationAlso: phsical exercise rehabilitationPatients only receive rehabilitation of physical therapy and occupational therapy. | Other | rehabilitation |
| stem cell injectionMesenchymal stem cells derived from umbilical cord are transplanted directly into subarachnoid by Lumbar puncture. | Biological | stem cell injection |
Primary outcome
Gross Motor Function Measure Score
Time frame: Change from baseline at 12 monthes after enrollment or transplantation
Gross Motor Function Measure-88 and Gross Motor Function Measure-66
1 secondary outcome
Routine Blood Test and Biochemical Test
Time frame: Change from baseline at 12 monthes after enrollment or transplantation
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 27 Dec 2017.
- Background: PubMed 8976681Pharoah PO, Platt MJ, Cooke T. The changing epidemiology of cerebral palsy. Arch Dis Child Fetal Neonatal Ed. 1996 Nov;75(3):F169-73. doi: 10.1136/fn.75.3.f169.
- Background: PubMed 23622163Richards CL, Malouin F. Cerebral palsy: definition, assessment and rehabilitation. Handb Clin Neurol. 2013;111:183-95. doi: 10.1016/B978-0-444-52891-9.00018-X.
- Background: PubMed 23622160Himmelmann K. Epidemiology of cerebral palsy. Handb Clin Neurol. 2013;111:163-7. doi: 10.1016/B978-0-444-52891-9.00015-4. No abstract available.
- Background: PubMed 12600249Reddihough DS, Collins KJ. The epidemiology and causes of cerebral palsy. Aust J Physiother. 2003;49(1):7-12. doi: 10.1016/s0004-9514(14)60183-5.
- Background: PubMed 20868878Rethlefsen SA, Ryan DD, Kay RM. Classification systems in cerebral palsy. Orthop Clin North Am. 2010 Oct;41(4):457-67. doi: 10.1016/j.ocl.2010.06.005.
- Background: PubMed 2075008Lubis MU, Tjipta GD, Marbun MD, Saing B. Cerebral palsy. Paediatr Indones. 1990 Mar-Apr;30(3-4):65-70.
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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.
Yihua An, Doctor study director
the General Hospital of Chinese People's Armed Police Forces
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing rehabilitation and stem cell injection in people with Cerebral Palsy, run by General Hospital of Chinese Armed Police Forces 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 2 inclusion and 6 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 27 Dec 2017. 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Gross Motor Function Measure Score (time frame: Change from baseline at 12 monthes after enrollment or transplantation). 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 NCT01929434, which also lists 6 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 27 Dec 2017.