Cerebral PalsyPhase 1NCT01586052
Erythropoietin Therapy for Children With Cerebral Palsy: Phase 1
A Phase 1 study testing Erythropoietin in people with Cerebral Palsy, run by MinYoung Kim, M.D. at 1 site in South Korea.
Trial summary
This purpose of this phase 1 study is to investigate the safety and efficacy of erythropoetin for children with cerebral palsy. Read more about this study
- Who is it for?
- Ages 6 months to 3 years
- Conditions the study is for
- Cerebral Palsy
- What is being tested?
- Erythropoietin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 1 siteSouth Korea
- Study dates
- Jun 2012to Oct 2013
- 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 6 months to 3 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Cerebral Palsy
- Abnormal Muscle Tone
- GMFCS (Gross Motor Functional Classification System): II to IV
- Age: 6 months ~ 3 years
- Abnormal Brain MRI compatible to clinical features and non-progressive
- Willing to Comply with All Study Procedure
Who cannot join
- Known Genetic Disorder
- Baseline Erythropoietin level > 45 mU/mL
- Presence of Drug Hypersensitivity Related to the Study Remedy
- Previous Erythropoietin Treatment before 3 months
- Coagulopathy:
- Family History, Unknown Cerebral Infarction, Thromboembolic Events History
Show all 14Show fewer
- Intractable Seizure Disorder
- Poor Cooperation of Guardian including Inactive Attitude for Rehabilitation
- Uncontrolled Hypertension
- Liver Dysfunction
- Renal Dysfunction
- Absolute Neutrophil Count < 500/dL
- Intracerebral or Intraventricular Hemorrhage
- Malignancy
Trial locations and contacts
1 site in 1 country.
South Korea1 site
CHA Bundang Medical Center, CHA University
Seongnam-si, Gyeonggi-do, 463-712
About this study
Official title: A Phase 1 Clinical Research of Erythropoietin Therapy for Children With Cerebral Palsy: Safety and Efficacy
This purpose of this phase 1 study is to investigate the safety and efficacy of erythropoetin for children with cerebral palsy.
Read the detailed description
Cerebral palsy is a disorder of movement and posture resulted from a nonprogressive lesion or injury of the immature brain. It is a leading cause of childhood onset disability.
Many experimental animal studies have revealed that erythropoietin is useful to repair neurological injury in brain. The main mechanism of erythropoietin is supposed as follows; neuroprotection effect, angiogenesis, and anti-inflammation.
On the basis of many experimental studies, erythropoietin is suggested as a potential therapy for cerebral palsy.
- Conditions
- Cerebral Palsy
- Being tested
- Erythropoietin
- Keywords
- Cerebral Palsy, Erythropoietin, 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.
ErythropoietinDrug
Also called Espogen produced by LG Life Science
250 IU/kg, Twice a week for 4 weeks
| Study group | Role | What they receive |
|---|---|---|
| Erythropoietin and Rehabilitation | Experimental | recombinant human erythropoietin injection and active rehabilitation Receives: Drug: Erythropoietin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events
Monitoring of all adverse events during 8 weeks, the study period We selected specially considered adverse events: encephalopathy, intracranial hemorrhage, seizure, hypertension, thromboembolic event, hypoxia, and acute kidney injury. Other adverse events will be recorded. The list was determined by the clinical experience and all adverse reactions reported by the pharmaceutical company.
Measured: 8 weeks
Secondary outcomes (5)
Changes in Quality of Movement
GMPM (Gross Motor Performance Measure) as a standardized measurement tool for assessing quality of movement regarding 3 properties of 5 ones: alignment, coordination, dissociated movement, stability, and weight shift The interrater reliability of GMPM subscores and total scores was 0.758-0.886 (subject n=75, tester n=10).
Measured: Baseline - 8 weeks
Changes in Gross Motor Function
GMFM (Gross Motor Function Measure) as a standardized measurement tool for assessing Gross Motor Function consisting of 6 sub-scales; lying \& rolling, sitting, crawling \& kneeling, standing, walking, running \& jumping The measured interrater reliability of GMFM subscores and total scores was 0.974 - 0.997 (subject n=101, tester n=10) and intrarater reliability of GMFM subscores and total scores between one most experienced rater and another newly t rained rater was 0.994 - 1.000 (subject n=101, tester n=2).
Measured: Baseline - 8 weeks
Changes in Neurodevelopmental Outcomes
K-BSID-II (Korean version of Bayley Scale of Infant Development-II) Motor and Mental scales The measured intrarater and interrater reliability of K-BSID-II motor and mental scales was 0.92 - 0.99 (subject n=55, tester n=10).
Measured: Baseline - 8 weeks
Changes in Motor Development
AIMS (Alberta Infant Motor Scale) to assess motor development
Measured: Baseline - 8 weeks
Changes in Spasticity
MAS (modified Ashworth Scale) measured at biceps, hip adductor, hamstring, heel cord
Measured: Baseline - 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Apr 2012 (estimated)
- Study startJun 2012
- Primary completionOct 2013
- Study completionOct 2013
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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- MinYoung Kim, M.D.
- Sponsor type
- University, hospital or other
- Collaborators
- LG Life Sciences
- Responsible party
- Sponsor-investigator, MinYoung Kim, M.D., Bundang CHA Hospital
- Investigators
- Minyoung Kim, M.D., Ph.D., Principal investigator, CHA University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01586052
- Sponsor's protocol ID
- CPEPOPhase1
- Status last verified
- Apr 2014
- First submitted
- 23 Apr 2012
- Last updated
- 8 Apr 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Apr 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Clinical Research of Erythropoietin Therapy for Children With Cerebral Palsy: Safety and Efficacy
- ClinicalTrials.gov ID
- NCT01586052
- Acronym / sponsor ID
- CPEPOPhase1
- Conditions
- Cerebral Palsy
- Keywords
- Cerebral Palsy, Erythropoietin, Rehabilitation
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- MinYoung Kim, M.D.University, hospital or other
- Collaborators
- LG Life Sciences
- Study start
- Jun 2012
- Primary completion
- Oct 2013 (actual)
- Study completion
- Oct 2013 (actual)
- First posted
- 26 Apr 2012 (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.
This purpose of this phase 1 study is to investigate the safety and efficacy of erythropoetin for children with cerebral palsy.
Detailed description
Cerebral palsy is a disorder of movement and posture resulted from a nonprogressive lesion or injury of the immature brain. It is a leading cause of childhood onset disability.
Many experimental animal studies have revealed that erythropoietin is useful to repair neurological injury in brain. The main mechanism of erythropoietin is supposed as follows; neuroprotection effect, angiogenesis, and anti-inflammation.
On the basis of many experimental studies, erythropoietin is suggested as a potential therapy for 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 |
|---|---|---|
| Erythropoietin and Rehabilitation | Experimental | Drug: Erythropoietinrecombinant human erythropoietin injection and active rehabilitation |
Who may qualify
- Ages
- 6 months to 3 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Cerebral Palsy
- Abnormal Muscle Tone
- GMFCS (Gross Motor Functional Classification System): II to IV
- Age: 6 months ~ 3 years
- Abnormal Brain MRI compatible to clinical features and non-progressive
Key exclusion criteria (14 listed)
- Known Genetic Disorder
- Baseline Erythropoietin level > 45 mU/mL
- Presence of Drug Hypersensitivity Related to the Study Remedy
- Previous Erythropoietin Treatment before 3 months
- Coagulopathy:
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 |
|---|---|---|
| ErythropoietinAlso: Espogen produced by LG Life Science250 IU/kg, Twice a week for 4 weeks | Drug | Erythropoietin and Rehabilitation |
Primary outcome
Adverse Events
Time frame: 8 weeks
Monitoring of all adverse events during 8 weeks, the study period We selected specially considered adverse events: encephalopathy, intracranial hemorrhage, seizure, hypertension, thromboembolic event, hypoxia, and acute kidney injury. Other adverse events will be recorded. The list was determined by the clinical experience and all adverse reactions reported by the pharmaceutical company.
5 secondary outcomes
Changes in Quality of Movement
Time frame: Baseline - 8 weeks
Changes in Gross Motor Function
Time frame: Baseline - 8 weeks
Changes in Neurodevelopmental Outcomes
Time frame: Baseline - 8 weeks
Changes in Motor Development
Time frame: Baseline - 8 weeks
Changes in Spasticity
Time frame: Baseline - 8 weeks
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 8 Apr 2014.
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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.
Minyoung Kim, M.D., Ph.D. 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 study testing Erythropoietin in people with Cerebral Palsy, run by MinYoung Kim, M.D. 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 14 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 8 Apr 2014. 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 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?
Adverse Events (time frame: 8 weeks). 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 8 Apr 2014.