Neonatal EncephalopathyPhase 1NCT02256618
Autologous Cord Blood Cell Therapy for Neonatal Encephalopathy
A Phase 1 study testing Autologous umbilical cord blood cells in people with Neonatal Encephalopathy and Hypoxic-ischemic Encephalopathy, run by Neonatal Encephalopathy Consortium, Japan at 6 sites in Japan.
Trial summary
This is a pilot study to test feasibility and safety of intravenous infusion of autologous umbilical cord blood cells in the first 72 hours after birth if a neonate is born with signs of encephalopathy. Read more about this study
- Who is it for?
- Ages Up to 24 hours
- Conditions the study is for
- Neonatal Encephalopathy, Hypoxic-ischemic Encephalopathy
- What is being tested?
- Autologous umbilical cord blood cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 6 sitesJapan
- Study dates
- Aug 2014to Jul 2019
- 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 Up to 24 hours
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Infants are eligible if they meet all the following inclusion criteria except 4.
- ≥36 weeks gestation
- Either a 10-minute Apgar score ≤5, continued need for resuscitation for at least 10 minutes, or severe acidosis, defined as pH <7.0 or base deficit ≥16 mmol/L in a sample of umbilical cord blood or any blood during the first hour after birth
- Moderate to severe encephalopathy (Sarnat II to III)
- A moderately or severely abnormal background amplitude-integrated EEG (aEEG) voltage, or seizures identified by aEEG, if monitored
- Up to 24 hours of age
- Autologous umbilical cord blood available to infuse within 3 days after birth
- A person with parental authority must have consented for the study.
Who cannot join
- Known major congenital anomalies, such as chromosomal anomalies, heart diseases
- Major intracranial hemorrhage identified by brain ultrasonography or computed tomography
- Severe growth restriction, with birth-weight less than 1800 g
- Severe infectious disease, such as sepsis
- Hyperkalemia
- Outborn infants (Infants born at hospitals other than the study sites)
- Volume of collected cord blood <40 ml
- Infants judged critically ill and unlikely to benefit from neonatal intensive care by the attending neonatologist
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
Japan6 sites
Saitama Medical University
Kawagoe, Saitama, 350-0495
Kurashiki Central Hospital
Kurashiki, Okayama-ken, 710-8602
Nagoya University Hospital
Nagoya, Aichi-ken, 466-8560
Yodogawa Christian Hospital
Osaka, 533-0032
Osaka City General Hospital
Osaka, 534-0021
Osaka City University
Osaka, 545-8585
About this study
Official title: A Pilot Feasibility and Safety Study of Autologous Umbilical Cord Blood Cell Therapy in Infants With Neonatal Encephalopathy
This is a pilot study to test feasibility and safety of intravenous infusion of autologous umbilical cord blood cells in the first 72 hours after birth if a neonate is born with signs of encephalopathy.
Read the detailed description
This is a multicenter pilot study to evaluate the feasibility and safety of intravenous infusions of autologous (the patient's own) umbilical cord blood cells in term gestation newborns with neonatal encephalopathy (hypoxic-ischemic encephalopathy). If a neonate is born with signs of moderate to severe encephalopathy and cooled for the encephalopathy, the neonate can receive their own non-cryopreserved volume- and red blood cell-reduced cord blood cells. The cord blood cells are divided into 3 doses and infused at 12-24, 36-48, and 60-72 hours after the birth. Infants will be followed for safety and neurodevelopmental outcome up to 18 months.
- Being tested
- Autologous umbilical cord blood cells
- Keywords
- neonatal encephalopathy, hypoxic-ischemic encephalopathy, newborn infants, neonates, umbilical cord blood cells
- Medical subject headings
- Hypoxia-Ischemia, Brain
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous umbilical cord blood cellsOther
Autologous non-cryopreserved volume- and red blood cell-reduced cord blood cells will be intravenously infused
| Study group | Role | What they receive |
|---|---|---|
| Cell therapy | Experimental | Infants who are born at the study sites, have moderate to severe encephalopathy, and have cord blood available for infusion Receives: Other: Autologous umbilical cord blood cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse event rates
Adverse event rates (combined rate of death, continuous respiratory support, and continuous use of vasopressor) will be compared between the cell recipients and historical controls at 30 days of age.
Measured: first 30 postnatal days
Secondary outcomes (1)
Efficacy
Neuroimaging at 12 months of age and neurodevelopmental function at 18 months of age will be compared between the cell recipients and historical controls.
Measured: 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Oct 2014 (estimated)
- Study startAug 2014
- Primary completionOct 2017
- Study completionJul 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Neonatal EncephalopathyStudies 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
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Neonatal Encephalopathy Consortium, Japan
- Sponsor type
- Research network
- Collaborators
- Osaka City University, Yodogawa Christian Hospital, Kurashiki Central Hospital, Nagoya University, Osaka City General Hospital, Saitama Medical University, National Cerebral and Cardiovascular Center, Japan, National Center for Child Health and Development, Japan, Tokyo University, Tokyo Women's Medical University
- Responsible party
- Sponsor
- Investigators
- Haruo Shintaku, MD, PhD, Study director, Osaka City University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02256618
- Sponsor's protocol ID
- UMIN000014903
- Status last verified
- Oct 2019
- First submitted
- 1 Oct 2014
- Last updated
- 29 Oct 2019
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Tsuji M, Taguchi A, Ohshima M, Kasahara Y, Sato Y, Tsuda H, Otani K, Yamahara K, Ihara M, Harada-Shiba M, Ikeda T, Matsuyama T. Effects of intravenous administration of umbilical cord blood CD34(+) cells in a mouse model of neonatal stroke. Neuroscience. 2014 Mar 28;263:148-58. doi: 10.1016/j.neuroscience.2014.01.018. Epub 2014 Jan 18.
PubMed 24444827Ohshima M, Taguchi A, Tsuda H, Sato Y, Yamahara K, Harada-Shiba M, Miyazato M, Ikeda T, Iida H, Tsuji M. Intraperitoneal and intravenous deliveries are not comparable in terms of drug efficacy and cell distribution in neonatal mice with hypoxia-ischemia. Brain Dev. 2015 Apr;37(4):376-86. doi: 10.1016/j.braindev.2014.06.010. Epub 2014 Jul 14.
PubMed 25034178Taguchi A, Soma T, Tanaka H, Kanda T, Nishimura H, Yoshikawa H, Tsukamoto Y, Iso H, Fujimori Y, Stern DM, Naritomi H, Matsuyama T. Administration of CD34+ cells after stroke enhances neurogenesis via angiogenesis in a mouse model. J Clin Invest. 2004 Aug;114(3):330-8. doi: 10.1172/JCI20622.
PubMed 15286799
Study at a glance
From the registry record, last updated by the sponsor on 29 Oct 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Feasibility and Safety Study of Autologous Umbilical Cord Blood Cell Therapy in Infants With Neonatal Encephalopathy
- ClinicalTrials.gov ID
- NCT02256618
- Acronym / sponsor ID
- UMIN000014903
- Keywords
- neonatal encephalopathy, hypoxic-ischemic encephalopathy, newborn infants, neonates, umbilical cord blood cells
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Neonatal Encephalopathy Consortium, JapanResearch network
- Collaborators
- Osaka City University, Yodogawa Christian Hospital, Kurashiki Central Hospital, Nagoya University, Osaka City General Hospital, Saitama Medical University, National Cerebral and Cardiovascular Center, Japan, National Center for Child Health and Development, Japan, Tokyo University, Tokyo Women's Medical University
- Study start
- Aug 2014
- Primary completion
- Oct 2017 (actual)
- Study completion
- Jul 2019 (actual)
- First posted
- 3 Oct 2014 (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 is a pilot study to test feasibility and safety of intravenous infusion of autologous umbilical cord blood cells in the first 72 hours after birth if a neonate is born with signs of encephalopathy.
Detailed description
This is a multicenter pilot study to evaluate the feasibility and safety of intravenous infusions of autologous (the patient's own) umbilical cord blood cells in term gestation newborns with neonatal encephalopathy (hypoxic-ischemic encephalopathy). If a neonate is born with signs of moderate to severe encephalopathy and cooled for the encephalopathy, the neonate can receive their own non-cryopreserved volume- and red blood cell-reduced cord blood cells. The cord blood cells are divided into 3 doses and infused at 12-24, 36-48, and 60-72 hours after the birth. Infants will be followed for safety and neurodevelopmental outcome up to 18 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cell therapy | Experimental | Other: Autologous umbilical cord blood cellsInfants who are born at the study sites, have moderate to severe encephalopathy, and have cord blood available for infusion |
Who may qualify
- Ages
- Up to 24 hours
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- Infants are eligible if they meet all the following inclusion criteria except 4.
- ≥36 weeks gestation
- Either a 10-minute Apgar score ≤5, continued need for resuscitation for at least 10 minutes, or severe acidosis, defined as pH <7.0 or base deficit ≥16 mmol/L in a sample of umbilical cord blood or any blood during the first hour after birth
- Moderate to severe encephalopathy (Sarnat II to III)
- A moderately or severely abnormal background amplitude-integrated EEG (aEEG) voltage, or seizures identified by aEEG, if monitored
Key exclusion criteria (8 listed)
- Known major congenital anomalies, such as chromosomal anomalies, heart diseases
- Major intracranial hemorrhage identified by brain ultrasonography or computed tomography
- Severe growth restriction, with birth-weight less than 1800 g
- Severe infectious disease, such as sepsis
- Hyperkalemia
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 |
|---|---|---|
| Autologous umbilical cord blood cellsAutologous non-cryopreserved volume- and red blood cell-reduced cord blood cells will be intravenously infused | Other | Cell therapy |
Primary outcome
Adverse event rates
Time frame: first 30 postnatal days
Adverse event rates (combined rate of death, continuous respiratory support, and continuous use of vasopressor) will be compared between the cell recipients and historical controls at 30 days of age.
1 secondary outcome
Efficacy
Time frame: 18 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 29 Oct 2019.
- Background: PubMed 24444827Tsuji M, Taguchi A, Ohshima M, Kasahara Y, Sato Y, Tsuda H, Otani K, Yamahara K, Ihara M, Harada-Shiba M, Ikeda T, Matsuyama T. Effects of intravenous administration of umbilical cord blood CD34(+) cells in a mouse model of neonatal stroke. Neuroscience. 2014 Mar 28;263:148-58. doi: 10.1016/j.neuroscience.2014.01.018. Epub 2014 Jan 18.
- Background: PubMed 25034178Ohshima M, Taguchi A, Tsuda H, Sato Y, Yamahara K, Harada-Shiba M, Miyazato M, Ikeda T, Iida H, Tsuji M. Intraperitoneal and intravenous deliveries are not comparable in terms of drug efficacy and cell distribution in neonatal mice with hypoxia-ischemia. Brain Dev. 2015 Apr;37(4):376-86. doi: 10.1016/j.braindev.2014.06.010. Epub 2014 Jul 14.
- Background: PubMed 15286799Taguchi A, Soma T, Tanaka H, Kanda T, Nishimura H, Yoshikawa H, Tsukamoto Y, Iso H, Fujimori Y, Stern DM, Naritomi H, Matsuyama T. Administration of CD34+ cells after stroke enhances neurogenesis via angiogenesis in a mouse model. J Clin Invest. 2004 Aug;114(3):330-8. doi: 10.1172/JCI20622.
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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.
Haruo Shintaku, MD, PhD study director
Osaka City University
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Autologous umbilical cord blood cells in people with Neonatal Encephalopathy and Hypoxic-ischemic Encephalopathy, run by Neonatal Encephalopathy Consortium, Japan at 6 sites in Japan.
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 8 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: finished, as of the sponsor's last update on 29 Oct 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Japan. 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 event rates (time frame: first 30 postnatal days). 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 NCT02256618, which also lists 3 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 29 Oct 2019.