Hypoxic-Ischemic EncephalopathyPhase 1NCT03352310
Feasibility and Safety of Umbilical Cord Blood Transfusion in the Treatment of Neonatal Cerebral Ischemia and Anemia
A Phase 1 study testing autologous umbilical cord blood (UCB) in people with Hypoxic-Ischemic Encephalopathy and Hypoxia Neonatal, run by Mononuclear Therapeutics Ltd. at 1 site in Hong Kong.
Trial summary
The study is to investigate the feasibility and safety of autologous umbilical cord blood transfusion to treat the newborn infants with presence of clinical indications of neonatal hypoxic-ischemia encephalopathy (HIE) and anemia. Read more about this study
- Who is it for?
- Ages Up to 48 hours
- Conditions the study is for
- Hypoxic-Ischemic Encephalopathy, Hypoxia Neonatal, Cerebral Ischemia of Newborn
- What is being tested?
- autologous umbilical cord blood (UCB)
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteHong Kong
- Study dates
- 16 Apr 2018to 30 Dec 2020
- 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 48 hours
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- evidence of asphyxiation, defined by 5-minute Apgar score ≤ 5;
- evidence of HIE, defined by UCB pH <7.15 or base excess ≤ 10mM;
- subjects with HIE confirmed by clinical features and initial investigations;
- subjects with evidence of anemia, defined by hematocrit < 40% or hemoglobin ≤ 13g/dL within the first 96 hours of life;
- obtain the informed consent from parents
Who cannot join
- congestive cardiac failure;
- microcephaly, anencephaly, encephalocele, or other abnormality
- conjoint twins;
- chromosomal disorders
- fetal alcohol syndrome
- spinal bifida or other neural tube defects
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- subjects have other neurological deficit conditions
- polycythemia
- congenital hematological malignancy
- investigator decision
Trial locations and contacts
1 site in 1 country.
Ronald Wang, MD contact
Hong Kong1 site, 1 recruiting
- Recruiting
The Chinese University of Hong Kong
Shatin
Simon Lam, M.D., +852 35052851
Simon Lam, MD
About this study
Official title: Umbilical Cord Blood Mononuclear Cell Bank in Hong Kong and Treatment of Neonatal Cerebral Ischemia and Anemia - Part IV Clinical Trial
The study is to investigate the feasibility and safety of autologous umbilical cord blood transfusion to treat the newborn infants with presence of clinical indications of neonatal hypoxic-ischemia encephalopathy (HIE) and anemia. Umbilical cord blood (UCB) is collected following labor and is transfused intravenously within 48 hours after the birth. Newborn infant without UCB available recieves the standard care will be enrolled as control group.
Following the autologous UCB transfusion in the study group or standard care in the control group, HIE subjects will be followed for 2 years for survival and neurodevelopmental outcomes and anemia subjects will be followed for 6 months to assess the survival and change of hematocrit and hemoglobin levels.
- Conditions
- Hypoxic-Ischemic Encephalopathy, Hypoxia Neonatal, Cerebral Ischemia of Newborn, Anemia, Neonatal
- Being tested
- autologous umbilical cord blood (UCB), standard care
- Keywords
- umbilical cord blood
- Medical subject headings
- Hypoxia-Ischemia, Brain, Asphyxia Neonatorum, Anemia, Neonatal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
autologous umbilical cord blood (UCB)Biological
autologous UCB transfusion to the newborn infants presence of HIE and/or anemia within 48 hours after the birth
standard careProcedure or surgery
standard care procedure to the newborn infants presence of HIE and/or anemia
| Study group | Role | What they receive |
|---|---|---|
| Study Group | Experimental | autologous UCB transfusion Receives: Biological: autologous umbilical cord blood (UCB) |
| Control Group | Other | standard care Receives: Procedure: standard care |
What is being measured
The primary outcome is the main question the study is designed to answer.
HIE: Mortality
Mortality Rate of the HIE Subjects
Measured: 6 months
Anemia: Change from Baseline Hematocrit
Change from Baseline Hematocrit of the Anemia Subjects
Measured: 48 hours, 1 week, 3 months, 6 months
Secondary outcomes (9)
HIE: HINE
Hammersmith Infant Neurological Examination (HINE) of the HIE Subjects
Measured: 6 months, 1 year and 2 years
HIE: HNNE
Hammersmith Neonatal Neurological Examination (HNNE) of the HIE Subjects
Measured: -1 day, 3 months (before discharge)
HIE: GMDS
Griffiths Mental Development Scale (GMDS) of the HIE Subjects
Measured: 6 months, 1 year and 2 years
HIE: CBCL
Child Behavior Checklist for Attention Deficit of the HIE Subjects
Measured: 2 years
HIE: Q-CHAT
Quantitative Checklist for Autism in Toddlers of the HIE Subjects
Measured: 2 years
Anemia: hemoglobin
Change from Baseline hemoglobin of the Anemia Subjects
Measured: 48 hours, 1 week, 3 months and 6 months
Anemia: Oxygenation level
Change from Baseline SpO2 of the Anemia Subjects
Measured: 48 hours, 1 week
Anemia: Oxidative Stress Level
Change of Baseline Isoprostane of the Anemia Subjects
Measured: 48 hours, 1 week, 3 months, and 6 months
Anemia: Requirements of Packed Cell Transfusion
Frequency of Requirements of Packed Cell Transfusion by the Anemia Subjects
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Nov 2017
- Study start16 Apr 2018
- Primary completion30 Jun 2020 (estimated)
- Study completion30 Dec 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hypoxic-Ischemic 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
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- Study Group - autologous umbilcial cord blood transfusion Control Group - standard care
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Mononuclear Therapeutics Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Chinese University of Hong Kong, China Spinal Cord Injury Network
- Responsible party
- Sponsor
- Investigators
- Simon Lam, MD, Principal investigator, Chinese University of Hong Kong
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03352310
- Sponsor's protocol ID
- UCB-HIEA-01
- Status last verified
- Jun 2018
- First submitted
- 15 Nov 2017
- Last updated
- 7 Jun 2018
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 7 Jun 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Umbilical Cord Blood Mononuclear Cell Bank in Hong Kong and Treatment of Neonatal Cerebral Ischemia and Anemia - Part IV Clinical Trial
- ClinicalTrials.gov ID
- NCT03352310
- Acronym / sponsor ID
- UCB-HIEA-01
- Conditions
- Hypoxic-Ischemic Encephalopathy, Hypoxia Neonatal, Cerebral Ischemia of Newborn, Anemia, Neonatal
- Keywords
- umbilical cord blood
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Mononuclear Therapeutics Ltd.Industry (pharma, biotech, devices)
- Collaborators
- Chinese University of Hong Kong, China Spinal Cord Injury Network
- Study start
- 16 Apr 2018 (actual)
- Primary completion
- 30 Jun 2020 (estimated)
- Study completion
- 30 Dec 2020 (estimated)
- First posted
- 24 Nov 2017 (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.
The study is to investigate the feasibility and safety of autologous umbilical cord blood transfusion to treat the newborn infants with presence of clinical indications of neonatal hypoxic-ischemia encephalopathy (HIE) and anemia. Umbilical cord blood (UCB) is collected following labor and is transfused intravenously within 48 hours after the birth. Newborn infant without UCB available recieves the standard care will be enrolled as control group.
Following the autologous UCB transfusion in the study group or standard care in the control group, HIE subjects will be followed for 2 years for survival and neurodevelopmental outcomes and anemia subjects will be followed for 6 months to assess the survival and change of hematocrit and hemoglobin levels.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Study Group - autologous umbilcial cord blood transfusion Control Group - standard care
| Arm | Type | Receives |
|---|---|---|
| Study Group | Experimental | Biological: autologous umbilical cord blood (UCB)autologous UCB transfusion |
| Control Group | Other | Procedure: standard carestandard care |
Who may qualify
- Ages
- Up to 48 hours
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- evidence of asphyxiation, defined by 5-minute Apgar score ≤ 5;
- evidence of HIE, defined by UCB pH <7.15 or base excess ≤ 10mM;
- subjects with HIE confirmed by clinical features and initial investigations;
- subjects with evidence of anemia, defined by hematocrit < 40% or hemoglobin ≤ 13g/dL within the first 96 hours of life;
- obtain the informed consent from parents
Key exclusion criteria (10 listed)
- congestive cardiac failure;
- microcephaly, anencephaly, encephalocele, or other abnormality
- conjoint twins;
- chromosomal disorders
- fetal alcohol syndrome
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 (UCB)autologous UCB transfusion to the newborn infants presence of HIE and/or anemia within 48 hours after the birth | Biological | Study Group |
| standard carestandard care procedure to the newborn infants presence of HIE and/or anemia | Procedure or surgery | Control Group |
Primary outcome
HIE: Mortality
Time frame: 6 months
Mortality Rate of the HIE Subjects
Anemia: Change from Baseline Hematocrit
Time frame: 48 hours, 1 week, 3 months, 6 months
Change from Baseline Hematocrit of the Anemia Subjects
9 secondary outcomes
HIE: HINE
Time frame: 6 months, 1 year and 2 years
HIE: HNNE
Time frame: -1 day, 3 months (before discharge)
HIE: GMDS
Time frame: 6 months, 1 year and 2 years
HIE: CBCL
Time frame: 2 years
HIE: Q-CHAT
Time frame: 2 years
Anemia: hemoglobin
Time frame: 48 hours, 1 week, 3 months and 6 months
Anemia: Oxygenation level
Time frame: 48 hours, 1 week
Anemia: Oxidative Stress Level
Time frame: 48 hours, 1 week, 3 months, and 6 months
Anemia: Requirements of Packed Cell Transfusion
Time frame: 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jun 2018.
- Individual participant dataSharing planned: Undecided
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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.
Ronald Wang, MD contact
Simon Lam, MD principal investigator
Chinese University of Hong Kong
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 autologous umbilical cord blood (UCB) in people with Hypoxic-Ischemic Encephalopathy and Hypoxia Neonatal, run by Mononuclear Therapeutics Ltd. at 1 site in Hong Kong.
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 5 inclusion and 10 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 7 Jun 2018. 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 Hong Kong. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Simon Lam, MD (+852 35052851, hshslam@cuhk.edu.hk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
HIE: Mortality (time frame: 6 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 7 Jun 2018.