Safety IssuesPhase 1NCT03791372
Clinical Effect and Safety of Autologous Umbilical Cord Blood Transfusion in the Treatment of Cerebral Palsy
A Phase 1 study testing 0.9% Sodium Chloride and Autologous Umbilical Cord Blood in people with Safety Issues and Effect of Drugs, run by Guangdong Women and Children Hospital at 1 site in China.
Trial summary
To study the clinical efficacy and safety of autologous umbilical cord blood transfusion in the treatment of cerebral palsy. Read more about this study
- Who is it for?
- Ages 6 months to 10 years
- Conditions the study is for
- Safety Issues, Effect of Drugs
- What is being tested?
- 0.9% Sodium Chloride and Autologous Umbilical Cord Blood
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 25 participants
- Where
- 1 siteChina
- Study dates
- 10 Oct 2017to 10 Apr 2021
- 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 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- A patient who was diagnosed with a mild case, and severity case of cerebral palsy or developmental disability through developmental assessment from more than two specialists including a specialist in pediatrics and rehabilitation medicine..
- In case there is a sign of the general decrease in white matter, or periventricular leukomalacia near cerebral ventricles in MRI DTI test.
- A subject who did a written consent to participation in this clinical trial through the very person and a legal representative.
Who cannot join
- In case a patient underwent a surgical procedure, or was administered autologous cord blood within one year before participating in a clinical trial.
- Accompanied by a serious disease, such as chromosome abnormality, etc.
- In case where a patient's medical condition is judged to be maladapted by a researcher.
- In case a patient or his or her legal representative doesn't agree to participation in a clinical trial.
- A patient having a predisposition to allergies.
- A patient having serious disorders in the liver, kidney, and cardiopulmonary function.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Guangdong Women and Children's Hospital and Health Institute
Guangzhou, Guangdong, 510010
Chang Yanqun, Phd, +862039151772, sfycyq@sina.com
About this study
To study the clinical efficacy and safety of autologous umbilical cord blood transfusion in the treatment of cerebral palsy.
Read the detailed description
This is a Phase 1 clinical trial that constitutes two time points cohorts with participants who will receive intravenous autologous umbilical cord blood and 0.9% sodium chloride respectively. The timing of treatment is any time after the diagnosis of cerebral palsy. The investigator will proceed the groups during the same period.
- Demographic Data and Baseline Characteristics of the Studied Group were collected:
- Basic patient's information survey
- Medical history
- Physical examination
- Basic blood test result
- Children's developmental disorders evaluation before the treatment
- Brain Magnetic Resonance Imaging-Diffusion Tensor Imaging (MRI-DTI) before the treatment
- Neurocognitive function test before the treatment
- Assessment of clinical condition in the course by measurement of blood pressure, heart and respiratory rates, temperature and adverse events was recorded.
- Autologous cord blood doses is 20-30ml (total Mononuclear cells>1*10^7/kg)，the infusion speed is 1ml/min, and with same volume of 0.9% sodium chloride as placebo.
- The follow-up: clinical test until 30th month in 3 month gaps.
- Conditions
- Safety Issues, Effect of Drugs
- Being tested
- 0.9% Sodium Chloride, Autologous Umbilical Cord Blood
- Keywords
- Cerebral palsy, Autologous Umbilical Cord Blood, safety, effect
- Medical subject headings
- Cerebral Palsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
0.9% Sodium ChlorideDrug
0.9% Sodium Chloride in control group
Autologous Umbilical Cord BloodBiological
Also called Autologous Umbilical Cord Blood Mononuclear Cells Therapy
Autologous Umbilical Cord Blood Mononuclear Cells Therapy in Cerebral Palsy for safety and effect evaluation.
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | 0.9% sodium chloride infusion any time after entering the Placebo Group, doses is 20-30ml. The infusion speed is 1ml/min. Receives: Drug: 0.9% Sodium Chloride |
| Cord Blood Mononuclear Cells(CBMNC) | Experimental | Autologous Umbilical Cord Blood Mononuclear Cells Therapy any time after the diagnosis of cerebral palsy, doses is 20-30ml (total Mononuclear cells>1*10^7/kg). The infusion speed is 1ml/min. Receives: Biological: Autologous Umbilical Cord Blood |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Change of Gross Motor Function Classification System(GMFCS) Score
GMFCS is used to assess the gross motor function of the children who were diagnosised as cerebral palsy.The results of GMFCS are divided into five levels. Grade I represents the best outcome while grade Ⅴ represents the worst.
Measured: up to 30 months after therapy at a 3-month interval
The Change of Gesell Developmental Scale(GDS) Score
The GDS is used to measure the gross motor,fine action,adaptability，language and individual-society function of the children whose age ranges from 1-month to 6-year-old. The score less than 75 for each subscale represent the worse outcome and more than 75 represent the better.
Measured: every 3-month after therapy until 30 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jan 2019
- Study start10 Oct 2017
- Primary completion10 Oct 2020 (estimated)
- Study completion10 Apr 2021 (estimated)
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 Safety IssuesStudies 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
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- Parallel assignment
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 25 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Guangdong Women and Children Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Xiao Huimei, Guangdong Women and Children Hospital
- Investigators
- Chang Yanqun, Phd, Study director, Guangdong Women and Children Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03791372
- Sponsor's protocol ID
- GuangdongWCHRD
- Status last verified
- Jan 2019
- First submitted
- 18 Dec 2018
- Last updated
- 4 Jan 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Sun JM, Song AW, Case LE, Mikati MA, Gustafson KE, Simmons R, Goldstein R, Petry J, McLaughlin C, Waters-Pick B, Chen LW, Wease S, Blackwell B, Worley G, Troy J, Kurtzberg J. Effect of Autologous Cord Blood Infusion on Motor Function and Brain Connectivity in Young Children with Cerebral Palsy: A Randomized, Placebo-Controlled Trial. Stem Cells Transl Med. 2017 Dec;6(12):2071-2078. doi: 10.1002/sctm.17-0102. Epub 2017 Oct 28.
PubMed 29080265
Study at a glance
From the registry record, last updated by the sponsor on 4 Jan 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Effect and Safety of Autologous Umbilical Cord Blood Transfusion in the Treatment of Cerebral Palsy
- ClinicalTrials.gov ID
- NCT03791372
- Acronym / sponsor ID
- GuangdongWCHRD
- Conditions
- Safety Issues, Effect of Drugs
- Keywords
- Cerebral palsy, Autologous Umbilical Cord Blood, safety, effect
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- Guangdong Women and Children HospitalUniversity, hospital or other
- Study start
- 10 Oct 2017 (actual)
- Primary completion
- 10 Oct 2020 (estimated)
- Study completion
- 10 Apr 2021 (estimated)
- First posted
- 2 Jan 2019 (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 study the clinical efficacy and safety of autologous umbilical cord blood transfusion in the treatment of cerebral palsy.
Detailed description
This is a Phase 1 clinical trial that constitutes two time points cohorts with participants who will receive intravenous autologous umbilical cord blood and 0.9% sodium chloride respectively. The timing of treatment is any time after the diagnosis of cerebral palsy. The investigator will proceed the groups during the same period.
- Demographic Data and Baseline Characteristics of the Studied Group were collected:
- Basic patient's information survey
- Medical history
- Physical examination
- Basic blood test result
- Children's developmental disorders evaluation before the treatment
- Brain Magnetic Resonance Imaging-Diffusion Tensor Imaging (MRI-DTI) before the treatment
- Neurocognitive function test before the treatment
- Assessment of clinical condition in the course by measurement of blood pressure, heart and respiratory rates, temperature and adverse events was recorded.
- Autologous cord blood doses is 20-30ml (total Mononuclear cells>1*10^7/kg)，the infusion speed is 1ml/min, and with same volume of 0.9% sodium chloride as placebo.
- The follow-up: clinical test until 30th month in 3 month gaps.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
- Model description
- Parallel assignment
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: 0.9% Sodium Chloride0.9% sodium chloride infusion any time after entering the Placebo Group, doses is 20-30ml. The infusion speed is 1ml/min. |
| Cord Blood Mononuclear Cells(CBMNC) | Experimental | Biological: Autologous Umbilical Cord BloodAutologous Umbilical Cord Blood Mononuclear Cells Therapy any time after the diagnosis of cerebral palsy, doses is 20-30ml (total Mononuclear cells>1*10^7/kg). The infusion speed is 1ml/min. |
Who may qualify
- Ages
- 6 months to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- A patient who was diagnosed with a mild case, and severity case of cerebral palsy or developmental disability through developmental assessment from more than two specialists including a specialist in pediatrics and rehabilitation medicine..
- In case there is a sign of the general decrease in white matter, or periventricular leukomalacia near cerebral ventricles in MRI DTI test.
- A subject who did a written consent to participation in this clinical trial through the very person and a legal representative.
Key exclusion criteria (6 listed)
- In case a patient underwent a surgical procedure, or was administered autologous cord blood within one year before participating in a clinical trial.
- Accompanied by a serious disease, such as chromosome abnormality, etc.
- In case where a patient's medical condition is judged to be maladapted by a researcher.
- In case a patient or his or her legal representative doesn't agree to participation in a clinical trial.
- A patient having a predisposition to allergies.
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 |
|---|---|---|
| 0.9% Sodium Chloride0.9% Sodium Chloride in control group | Drug | Placebo |
| Autologous Umbilical Cord BloodAlso: Autologous Umbilical Cord Blood Mononuclear Cells TherapyAutologous Umbilical Cord Blood Mononuclear Cells Therapy in Cerebral Palsy for safety and effect evaluation. | Biological | Cord Blood Mononuclear Cells(CBMNC) |
Primary outcome
The Change of Gross Motor Function Classification System(GMFCS) Score
Time frame: up to 30 months after therapy at a 3-month interval
GMFCS is used to assess the gross motor function of the children who were diagnosised as cerebral palsy.The results of GMFCS are divided into five levels. Grade I represents the best outcome while grade Ⅴ represents the worst.
The Change of Gesell Developmental Scale(GDS) Score
Time frame: every 3-month after therapy until 30 months
The GDS is used to measure the gross motor,fine action,adaptability，language and individual-society function of the children whose age ranges from 1-month to 6-year-old. The score less than 75 for each subscale represent the worse outcome and more than 75 represent the better.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jan 2019.
- Background: PubMed 29080265Sun JM, Song AW, Case LE, Mikati MA, Gustafson KE, Simmons R, Goldstein R, Petry J, McLaughlin C, Waters-Pick B, Chen LW, Wease S, Blackwell B, Worley G, Troy J, Kurtzberg J. Effect of Autologous Cord Blood Infusion on Motor Function and Brain Connectivity in Young Children with Cerebral Palsy: A Randomized, Placebo-Controlled Trial. Stem Cells Transl Med. 2017 Dec;6(12):2071-2078. doi: 10.1002/sctm.17-0102. Epub 2017 Oct 28.
- Individual participant dataSharing planned: Undecided
Related trials for Safety Issues
Active, not recruitingPhase 1
E-CEL UVEC Cells as an Adjunct Cell Therapy for the Arthroscopic Rotator Cuff Repair in Adults
Rotator Cuff Tears, Muscle Atrophy or Weakness
Not yet recruitingPhase 1
Phase 1 Study to Evaluate RG002C0106 Injection in Healthy Participants
Safety Issues
Enrolling by invitation
Pediatric Trauma Centers RE-AIM at Gun Safety
Firearm Injury, Safety Issues
RecruitingObservational study
Efficacy and Safety of a New Sperm Capacitation Method
Reproductive Issues, Male Infertility
Active, not recruitingPhase 1/2
Role of a CCK Receptor Antagonist Proglumide in Management of Chronic Pancreatitis
Safety Issues, Pain
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.
Chang Yanqun, Phd study director
Guangdong Women and Children Hospital
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 0.9% Sodium Chloride and Autologous Umbilical Cord Blood in people with Safety Issues and Effect of Drugs, run by Guangdong Women and Children Hospital 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 3 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: status not confirmed, as of the sponsor's last update on 4 Jan 2019. 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. Some participants in this study may receive a placebo. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Xiao Huimei, MD (+862039151772 ext. +86, xiaowu2010727@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The Change of Gross Motor Function Classification System(GMFCS) Score (time frame: up to 30 months after therapy at a 3-month interval). 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?
On the ClinicalTrials.gov record for NCT03791372, 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 Jan 2019.