Global Developmental DelayNCT01601158
Umbilical Cord Blood Therapy for Children With Global Developmental Delay
A study testing Umbilical Cord Blood administration and Active Rehabilitation in people with Global Developmental Delay, run by MinYoung Kim, M.D. at 1 site in South Korea.
Trial summary
This open-label study aims to evaluate the safety and efficacy of umbilical cord blood therapy for children with global developmental delay. Read more about this study
- Who is it for?
- Ages 6 months to 15 years
- Conditions the study is for
- Global Developmental Delay
- What is being tested?
- Umbilical Cord Blood administration and Active Rehabilitation
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteSouth Korea
- Study dates
- May 2012to Aug 2013
- 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 6 months to 15 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Global developmental delay
- Willing to comply with all study procedure
Who cannot join
- Medical instability including pneumonia or renal function at enrollment
- Poor cooperation of guardian,including inactive attitude for rehabilitation and visits for follow-up
- Uncontrolled persistent epilepsy
- Not eligible according to the principal investigator
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: Safety and Efficacy of Allogeneic Umbilical Cord Blood Therapy for Children With Global Developmental Delay
This open-label study aims to evaluate the safety and efficacy of umbilical cord blood therapy for children with global developmental delay.
Read the detailed description
Global developmental delay (GDD) is a subset of developmental disabilities defined as significant delay in two or more of the following developmental domains: gross/fine motor, speech/language, cognition, social/personal, and activities of daily living.
Umbilical cord blood has been used for inherited metabolic diseases that feature global developmental delay and many experimental animal studies have revealed umbilical cord blood is useful to repair neurological impairments in brain.
- Conditions
- Global Developmental Delay
- Being tested
- Umbilical Cord Blood administration, Active Rehabilitation
- Keywords
- Global developmental delay, Umbilical cord blood, Rehabilitation
- Medical subject headings
- Learning Disabilities
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Umbilical Cord Blood administrationBiological
Also called Donated Umbilical Cord Blood Units from Affiliated Cord Blood Bank
The subjects will be undertaken allogeneic umbilical cord blood infusion intravenously or intraarterially under non-myeloablative immunosuppression.
Active RehabilitationOther
All subjects should participate in active rehabilitation. They will receive two physical and occupational therapy sessions per day. Post discharge, each participant should continue to receive rehabilitation therapy at least 3 days per week until the study completion.
| Study group | Role | What they receive |
|---|---|---|
| Umbilical Cord Blood and Rehabilitation | Experimental | Allogeneic Umbilical Cord Blood infusion and Active Rehabilitation Receives: Biological: Umbilical Cord Blood administration; Other: Active Rehabilitation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes in Cognition
Korean Wechsler Preschool and Primay Scale of Intelligence (K-WPPSI) (Higher value means better cognitive function (K-WPPSI: below 70 - worst, over 130 - best). The investigators will report K-WPPSI at each assessment time points.
Measured: Baseline - 6 months - 12 months
Changes in Cognitive Neurodevelopmental Outcome
Korean version of Bayley Scale of Infant Development-II (K-BSID-II) Mental Scales (Higher value means better cognitive function, K-BSID-II mental scale raw score: 0 - worst, 178 - best). The investigators will report K-BSID-II Mental Scale raw scores at each assessment time points.
Measured: Baseline - 1 month - 3 months - 6 months - 12 months
Secondary outcomes (8)
Changes in Motor Performance
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 (range: 0~100, Higher value means better motor quality). The investigators will report GMPM scores at each assessment time points.
Measured: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Standardized Gross Motor Function
GMFM (Gross Motor Function Measure) as a standardized measurement tool for assessing Gross Motor Function consisting of sub-scales; lying \& rolling, sitting, crawling \& kneeling, standing, walking, running \& jumping (range: 0~100 , Higher value means better gross motor function). The investigators will report GMFM scores at each assessment time points.
Measured: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Motor Neurodevelopmental Outcome
Korean version of Bayley Scale of Infant Development-II (K-BSID-II) Motor Scales (higher value means better motor function: 0 - worst, 111 - best). The investigators will report K-BSID-II Motor Scale raw scores at each assessment time points.
Measured: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Functional Independence in Daily Activities
WeeFIM (Functional Independence Measure for Children) measures functional independence in daily activities. WeeFIM contains 18 items and each item is ranked from complete dependence (scored as 1) to complete independence (scored as 7). The range is from 18 to 126 and higher scores mean more independent performance in daily activities. The investigators will report total WeeFIM scores measured at each assessment time points.
Measured: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Visual Perception Test
We will evaluate visual perception function with one of three measures: DTVP (Developmental Test of Visual Perception), MVPT (Motor-free Visual Perception Test), and VMI (Visual-Motor Integration, Visual Perception and Motor Coordination). All can be scored as percentile rank from 0 to 100. Higher values mean better visual perception ability
Measured: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Muscle Strength
Summation of MMT (manual muscle strength test score): summated scores of the manual muscle strength test (zero=0, trace=1, poor=2, fair=3, good=4, normal=5) for flexors, extensors, abductors, and adductors of bilateral shoulder and hip joints; flexors and extensors of bilateral elbow, wrist, and knee; dorsiflexors and plantar flexors of the ankles (range: 0 ~ 160) Higher scores mean better muscle strength. Categories of outcome table will be summation of MMT scores measured at each assessment time point.
Measured: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Functional Performance in Daily Activities
Pediatric Evaluation of Disability Inventory (PEDI) for assessing functional performance in daily activities in children (All values are adjusted and higher value means better functional performance, 0 - worst, 100 - best). We will report 2 scales and 3 domains of each scale: a Functional Skill Scale (FSS) and a Caregiver Assistance Scale (CAS) which are divided respectively into 3 domains: self care, mobility, and social function. Categories of outcome table will be each domain scores measured at each assessment time point.
Measured: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Language Evaluation
Sequenced Language Scale for Infant (SELSI), Preschool Receptive-Expressive Language Scale (PRES) or Korean Western Aphasia Battery (K-WAB) will be used depending on age and function.
Measured: Baseline - 6 months - 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 May 2012 (estimated)
- Study startMay 2012
- Primary completionAug 2013
- Study completionAug 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Global Developmental DelayStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- MinYoung Kim, M.D.
- Sponsor type
- University, hospital or other
- 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
- NCT01601158
- Sponsor's protocol ID
- GDDUCB
- Status last verified
- Jan 2014
- First submitted
- 16 May 2012
- Last updated
- 9 Jan 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Jan 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of Allogeneic Umbilical Cord Blood Therapy for Children With Global Developmental Delay
- ClinicalTrials.gov ID
- NCT01601158
- Acronym / sponsor ID
- GDDUCB
- Conditions
- Global Developmental Delay
- Keywords
- Global developmental delay, Umbilical cord blood, Rehabilitation
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- MinYoung Kim, M.D.University, hospital or other
- Study start
- May 2012
- Primary completion
- Aug 2013 (actual)
- Study completion
- Aug 2013 (actual)
- First posted
- 17 May 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 open-label study aims to evaluate the safety and efficacy of umbilical cord blood therapy for children with global developmental delay.
Detailed description
Global developmental delay (GDD) is a subset of developmental disabilities defined as significant delay in two or more of the following developmental domains: gross/fine motor, speech/language, cognition, social/personal, and activities of daily living.
Umbilical cord blood has been used for inherited metabolic diseases that feature global developmental delay and many experimental animal studies have revealed umbilical cord blood is useful to repair neurological impairments in brain.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Umbilical Cord Blood and Rehabilitation | Experimental | Biological: Umbilical Cord Blood administration, Other: Active RehabilitationAllogeneic Umbilical Cord Blood infusion and Active Rehabilitation |
Who may qualify
- Ages
- 6 months to 15 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Global developmental delay
- Willing to comply with all study procedure
Key exclusion criteria (4 listed)
- Medical instability including pneumonia or renal function at enrollment
- Poor cooperation of guardian,including inactive attitude for rehabilitation and visits for follow-up
- Uncontrolled persistent epilepsy
- Not eligible according to the principal investigator
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 |
|---|---|---|
| Umbilical Cord Blood administrationAlso: Donated Umbilical Cord Blood Units from Affiliated Cord Blood BankThe subjects will be undertaken allogeneic umbilical cord blood infusion intravenously or intraarterially under non-myeloablative immunosuppression. | Biological | Umbilical Cord Blood and Rehabilitation |
| Active RehabilitationAll subjects should participate in active rehabilitation. They will receive two physical and occupational therapy sessions per day. Post discharge, each participant should continue to receive rehabilitation therapy at least 3 days per week until the study completion. | Other | Umbilical Cord Blood and Rehabilitation |
Primary outcome
Changes in Cognition
Time frame: Baseline - 6 months - 12 months
Korean Wechsler Preschool and Primay Scale of Intelligence (K-WPPSI) (Higher value means better cognitive function (K-WPPSI: below 70 - worst, over 130 - best). The investigators will report K-WPPSI at each assessment time points.
Changes in Cognitive Neurodevelopmental Outcome
Time frame: Baseline - 1 month - 3 months - 6 months - 12 months
Korean version of Bayley Scale of Infant Development-II (K-BSID-II) Mental Scales (Higher value means better cognitive function, K-BSID-II mental scale raw score: 0 - worst, 178 - best). The investigators will report K-BSID-II Mental Scale raw scores at each assessment time points.
8 secondary outcomes
Changes in Motor Performance
Time frame: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Standardized Gross Motor Function
Time frame: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Motor Neurodevelopmental Outcome
Time frame: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Functional Independence in Daily Activities
Time frame: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Visual Perception Test
Time frame: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Muscle Strength
Time frame: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Functional Performance in Daily Activities
Time frame: Baseline - 1 month - 3 months - 6 months - 12 months
Changes in Language Evaluation
Time frame: Baseline - 6 months - 12 months
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 9 Jan 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 study testing Umbilical Cord Blood administration and Active Rehabilitation in people with Global Developmental Delay, 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 2 inclusion and 4 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 9 Jan 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.
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?
Changes in Cognition (time frame: Baseline - 6 months - 12 months). The study lists 2 primary outcomes in all. 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 9 Jan 2014.