Moderate to Severe Hypoxic-ischemic EncephalopathyPhase 1NCT03635450
Study of hCT-MSC in Newborn Infants With Moderate or Severe HIE
A Phase 1 study testing Infusion of hCT-MSC in people with Moderate to Severe Hypoxic-ischemic Encephalopathy, run by Joanne Kurtzberg, MD at 1 site in United States.
Trial summary
To determine the safety of single and repeated intravenous doses of hCT-MSC in newborn infants with HIE. Read more about this study
- Who is it for?
- Ages 0 to 48 hours
- Conditions the study is for
- Moderate to Severe Hypoxic-ischemic Encephalopathy
- What is being tested?
- Infusion of hCT-MSC
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- 27 Dec 2018to 28 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 0 to 48 hours
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 36 0/7th weeks gestation or older at the time of delivery.
- Able to receive one dose of hCT-MSCs in the first 48 postnatal hours
- Willingness to return for one year assessments.
- Signs of encephalopathy within 6 hours of age
Who cannot join
- Major congenital or chromosomal abnormalities
- Severe growth restriction (birth weight <1800 g)
- Opinion by attending neonatologist that the study may interfere with clinical treatment or safety of subject
- Moribund neonates for whom no further treatment is planned
- Infants whose mothers have unknown serologies for Hepatitis B or HIV
- Infants born to mothers are known to be HIV, Hepatitis B, Hepatitis C or who have active syphilis or CMV infection in pregnancy
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- Infants suspected of overwhelming sepsis
- ECMO initiated or likely in the first 48 hours of life
- Mother suspected to have intraamniotic infection at time of birth.
- ALL blood gases (cord and postnatal) done within the first 60 minutes had a pH > 7.15 AND base deficit < 10 mEq/L (source can be arterial, venous or capillary)
- Mother with documented Zika infection during this pregnancy
- Availability of autologous cord blood collected and usable in the randomized trial of autologous volume- and red blood cell-reduced cord blood cells (Duke IRB Pro00066647; clinical trials.gov link: https://clinicaltrials.gov/ct2/show/NCT02612155 )
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase I Study of hCT-MSC, an Umbilical Cord-Derived Mesenchymal Stromal Cell Product, in Newborn Infants With Moderate or Severe Hypoxic- Ischemic Neonatal Encephalopathy.
To determine the safety of single and repeated intravenous doses of hCT-MSC in newborn infants with HIE.
Read the detailed description
The purpose of this study is to assess the safety of one and two intravenous infusions of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC), the first administered in the first 48 postnatal hours, and the second at two months postnatal age, in term and near term infants with moderate to severe neonatal hypoxic-ischemic encephalopathy (HIE). This is a phase I, prospective, open-label trial designed to assess the safety of one or two intravenous doses of hCT-MSC in newborn infants with moderate to severe HIE who are recipients of therapeutic hypothermia. Infants born at 36 0/7 weeks gestation or later who have moderate to severe hypoxic-ischemic encephalopathy and are receiving therapeutic hypothermia will be eligible to participate. Investigators project an accrual of 6 patients. All infants will receive intravenous infusion(s) of hCT-MSCs. The first cohort of three infants will receive a single dose in the first 48 postnatal hours. If there are no safety concerns, the second cohort of three infants will receive two doses, with the first dose given in the first 48 postnatal hours and the second dose given approximately two months after the first dose.
The potential risks associated with infusion of MSCs include a reaction to the product (rash, shortness of breath, wheezing, difficulty breathing, hypotension, swelling around the mouth, throat or eyes, tachycardia, diaphoresis), transmission of infection, and HLA sensitization. Another risk of this study is loss of confidentiality or privacy. Every effort will be made to keep the infant's medical record confidential. The results will be summarized using descriptive statistics and statistical testing as appropriate. Continuous secondary endpoints will be summarized using mean, standard deviation, CV%, median, minimum, and maximum.
- Being tested
- Infusion of hCT-MSC
- Keywords
- hypoxic-ischemic encephalopathy, newborn infants, therapeutic hypothermia, hCT-MSC, an Umbilical Cord Tissue-Derived Mesenchymal Stromal Cell Product
- 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.
Infusion of hCT-MSCBiological
Infants who meet enrollment criteria for moderate to severe hypoxic ischemic encephalopathy will receive 1 infusion of hCT-MSC within the first 48 postnatal hours. hCT-MSCs are a product of allogeneic cells manufactured from digested umbilical cord tissue that is expanded in culture, cryopreserved and banked. hCT-MSCs are manufactured from umbilical cord tissue donated to the Carolinas Cord Blood Bank, an FDA-licensed, FACT-accredited, public cord blood bank at Duke University Medical Center, after written informed consent from the baby's mother. Cord tissue is harvested from the placentas of male babies delivered by elective C-section after a normal, full- term pregnancy.
| Study group | Role | What they receive |
|---|---|---|
| First cohort of 3 subjects enrolled | Experimental | The first cohort of three patients will receive a single dose in the first 48 postnatal hours. Receives: Biological: Infusion of hCT-MSC |
| Second cohort of 3 subjects enrolled | Experimental | If there are no safety concerns after the first cohort of 3 subjects are infused then the second cohort of three patients will receive two doses, with the first dose given in the first 48 postnatal hours and the second dose given approximately two months after the first dose. Receives: Biological: Infusion of hCT-MSC |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of infusion reactions
for this study, infusion reactions are defined as anaphylactic or anaphylactoid reactions with clinical signs inclusive of skin rashes, bronchospasm, angioedema, myocardial infarcts, arrhythmias, and acute lung injury.
Measured: 24 hours after each infusion
Incidence of Infections post-infusion
for this study, infections recorded as safety endpoints will be defined as bacterial, viral or fungal infections identified by culture or molecular methodologies within two weeks after administration of hCT-MSC.
Measured: Up to 2 Weeks
Secondary outcomes (2)
Survival
Death prior to discharge from initial hospitalization
Measured: Up to 6 months
Neurodevelopmental Assessments
1 year (12 - 16 postnatal months) Bayley Scales of Infant and Toddler Development, Third Edition (Bayley III) assessments in cognitive, language and motor development. Moderate to Severe CP will be assigned with cognitive score ,70, motor score ,70 and with Gross Motor Function Classification System >=2.
Measured: Up to 16 postnatal months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2018
- Study start27 Dec 2018
- Primary completion28 Jul 2019
- Study completion28 Dec 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Moderate to Severe 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
- The first cohort of three patients will receive a single dose in the first 48 postnatal hours. If there are no safety concerns, the second cohort of three patients will receive two doses, with the first dose given in the first 48 postnatal hours and the second dose given approximately two months after the first dose.
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Joanne Kurtzberg, MD
- Sponsor type
- University, hospital or other
- Collaborators
- Duke Clinical and Translational Science Institute (CTSI), part of the NIH Clinical and Translational Science Awards (CTSA)
- Responsible party
- Sponsor-investigator, Joanne Kurtzberg, MD, Duke University
- Investigators
- Michael Cotten, MD, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03635450
- Sponsor's protocol ID
- Pro00100253
- Status last verified
- Apr 2024
- First submitted
- 15 Aug 2018
- Last updated
- 12 Apr 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Study of hCT-MSC, an Umbilical Cord-Derived Mesenchymal Stromal Cell Product, in Newborn Infants With Moderate or Severe Hypoxic- Ischemic Neonatal Encephalopathy.
- ClinicalTrials.gov ID
- NCT03635450
- Acronym / sponsor ID
- Pro00100253
- Keywords
- hypoxic-ischemic encephalopathy, newborn infants, therapeutic hypothermia, hCT-MSC, an Umbilical Cord Tissue-Derived Mesenchymal Stromal Cell Product
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Joanne Kurtzberg, MDUniversity, hospital or other
- Collaborators
- Duke Clinical and Translational Science Institute (CTSI), part of the NIH Clinical and Translational Science Awards (CTSA)
- Study start
- 27 Dec 2018 (actual)
- Primary completion
- 28 Jul 2019 (actual)
- Study completion
- 28 Dec 2020 (actual)
- First posted
- 17 Aug 2018 (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 determine the safety of single and repeated intravenous doses of hCT-MSC in newborn infants with HIE.
Detailed description
The purpose of this study is to assess the safety of one and two intravenous infusions of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC), the first administered in the first 48 postnatal hours, and the second at two months postnatal age, in term and near term infants with moderate to severe neonatal hypoxic-ischemic encephalopathy (HIE). This is a phase I, prospective, open-label trial designed to assess the safety of one or two intravenous doses of hCT-MSC in newborn infants with moderate to severe HIE who are recipients of therapeutic hypothermia. Infants born at 36 0/7 weeks gestation or later who have moderate to severe hypoxic-ischemic encephalopathy and are receiving therapeutic hypothermia will be eligible to participate. Investigators project an accrual of 6 patients. All infants will receive intravenous infusion(s) of hCT-MSCs. The first cohort of three infants will receive a single dose in the first 48 postnatal hours. If there are no safety concerns, the second cohort of three infants will receive two doses, with the first dose given in the first 48 postnatal hours and the second dose given approximately two months after the first dose.
The potential risks associated with infusion of MSCs include a reaction to the product (rash, shortness of breath, wheezing, difficulty breathing, hypotension, swelling around the mouth, throat or eyes, tachycardia, diaphoresis), transmission of infection, and HLA sensitization. Another risk of this study is loss of confidentiality or privacy. Every effort will be made to keep the infant's medical record confidential. The results will be summarized using descriptive statistics and statistical testing as appropriate. Continuous secondary endpoints will be summarized using mean, standard deviation, CV%, median, minimum, and maximum.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- The first cohort of three patients will receive a single dose in the first 48 postnatal hours. If there are no safety concerns, the second cohort of three patients will receive two doses, with the first dose given in the first 48 postnatal hours and the second dose given approximately two months after the first dose.
| Arm | Type | Receives |
|---|---|---|
| First cohort of 3 subjects enrolled | Experimental | Biological: Infusion of hCT-MSCThe first cohort of three patients will receive a single dose in the first 48 postnatal hours. |
| Second cohort of 3 subjects enrolled | Experimental | Biological: Infusion of hCT-MSCIf there are no safety concerns after the first cohort of 3 subjects are infused then the second cohort of three patients will receive two doses, with the first dose given in the first 48 postnatal hours and the second dose given approximately two months after the first dose. |
Who may qualify
- Ages
- 0 to 48 hours
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- 36 0/7th weeks gestation or older at the time of delivery.
- Able to receive one dose of hCT-MSCs in the first 48 postnatal hours
- Willingness to return for one year assessments.
- Signs of encephalopathy within 6 hours of age
Key exclusion criteria (12 listed)
- Major congenital or chromosomal abnormalities
- Severe growth restriction (birth weight <1800 g)
- Opinion by attending neonatologist that the study may interfere with clinical treatment or safety of subject
- Moribund neonates for whom no further treatment is planned
- Infants whose mothers have unknown serologies for Hepatitis B or HIV
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 |
|---|---|---|
| Infusion of hCT-MSCInfants who meet enrollment criteria for moderate to severe hypoxic ischemic encephalopathy will receive 1 infusion of hCT-MSC within the first 48 postnatal hours. hCT-MSCs are a product of allogeneic cells manufactured from digested umbilical cord tissue that is expanded in culture, cryopreserved and banked. hCT-MSCs are manufactured from umbilical cord tissue donated to the Carolinas Cord Blood Bank, an FDA-licensed, FACT-accredited, public cord blood bank at Duke University Medical Center, after written informed consent from the baby's mother. Cord tissue is harvested from the placentas of male babies delivered by elective C-section after a normal, full- term pregnancy. | Biological | First cohort of 3 subjects enrolled, Second cohort of 3 subjects enrolled |
Primary outcome
Incidence of infusion reactions
Time frame: 24 hours after each infusion
for this study, infusion reactions are defined as anaphylactic or anaphylactoid reactions with clinical signs inclusive of skin rashes, bronchospasm, angioedema, myocardial infarcts, arrhythmias, and acute lung injury.
Incidence of Infections post-infusion
Time frame: Up to 2 Weeks
for this study, infections recorded as safety endpoints will be defined as bacterial, viral or fungal infections identified by culture or molecular methodologies within two weeks after administration of hCT-MSC.
2 secondary outcomes
Survival
Time frame: Up to 6 months
Neurodevelopmental Assessments
Time frame: Up to 16 postnatal 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 12 Apr 2024.
- Individual participant dataSharing planned: No
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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.
Michael Cotten, MD principal investigator
Duke 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 Infusion of hCT-MSC in people with Moderate to Severe Hypoxic-ischemic Encephalopathy, run by Joanne Kurtzberg, MD at 1 site in United States.
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 4 inclusion and 12 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 12 Apr 2024. 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 United States. 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?
Incidence of infusion reactions (time frame: 24 hours after each infusion). 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 12 Apr 2024.