Neonatal Hypoxic-ischaemic EncephalopathyPhase 1/2NEOSTEMNCT02881970
Neonatal Hypoxic Ischemic Encephalopathy : Safety and Feasibility Study of a Curative Treatment With Autologous Cord Blood Stem Cells
A Phase 1/2 study testing autologous cord blood stem cell in people with Neonatal Hypoxic-ischaemic Encephalopathy, run by Assistance Publique Hopitaux De Marseille at 1 site in France.
Trial summary
Neonatal hypoxic-ischaemic encephalopathy is a dramatic perinatal complication due to brain asphyxia. Neurological and neurosensory sequelae are frequent in survivors, due to neuronal damage and loss. Currently, only total or partial body hypothermia can partially prevent cell loss. Read more about this study
- Who is it for?
- Ages 1 day to 3 days
- Conditions the study is for
- Neonatal Hypoxic-ischaemic Encephalopathy
- What is being tested?
- autologous cord blood stem cell
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteFrance
- Study dates
- 5 Feb 2020to Sep 2028
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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 1 day to 3 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Term ≥ 36 weeks of gestation
- and (2) :
- a blood pH < 7 with base deficit > 12 mmol/l (at birth or within 60 minutes of age)
- or a blood pH between 7,01 and 7,15, with additionnal criteria:
- a history of acute perinatal event (e.g : abnormal fetal cardiac rate, cord prolapse, uterine rupture, maternal hemorrhage)
- and a 5 minutes Apgar score ≤ 5, or a continued need for resuscitation, including endotracheal or mask ventilation at 5 min after birth.
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- signs of encephalopathy within 12 hours of age (Sarnat and Sarnat classification, score ≥ 2)
- ± abnormal electroencephalogram or aEEG within 12 hours of age
- therapeutic hypothermia.
- no maternal infection with VIH, HTLV 1 or 2, Hepatitis B or C virus.
- maternal negative serology for syphilis
- written parental consent
Who cannot join
- presence of known chromosomal anomaly.
- presence of major congenital anomalies. severe intrauterine growth restriction (weight <1800g)
- infants in extremis for whom no additional intensive therapy will be offered by attending neonatologist.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Farid BOUBRED contact
France1 site, 1 recruiting
- Recruiting
Assistance Publique Hopitaux de Marseille
Marseille
FARID BOUBRED, farid.boubred@ap-hm.fr
About this study
Neonatal hypoxic-ischaemic encephalopathy is a dramatic perinatal complication due to brain asphyxia. Neurological and neurosensory sequelae are frequent in survivors, due to neuronal damage and loss.
Currently, only total or partial body hypothermia can partially prevent cell loss. However, no treatment exists to restore neuronal functions.
Read more
Cord blood stem cells are a promising treatment for the near future.
The primary objective of this study is to test the safety and feasibility of a curative treatment with autologous cord blood stem cell in neonatal hypoxic-ischaemic encephalopathy.
The secondary objectives are to test the efficacy of this curative treatment with cell with neurogenic potential on the prevention of neurologic sequelae, as well as to test the optimum timing of cell preparation administration
- Being tested
- autologous cord blood stem cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
autologous cord blood stem cellDrug
Injection of 5.107 / kg autologous mononuclear cells from umbilical cord blood
| Study group | Role | What they receive |
|---|---|---|
| Neonatal hypoxic-ischaemic encephalopathy | Experimental | Not described Receives: Drug: autologous cord blood stem cell |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse clinical or paraclinical event rates due to stem cell preparation
Measured: 2years
Secondary outcomes (1)
- Preliminary efficacy as measured by neurodevelopmental function
Measured: 2years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Aug 2016 (estimated)
- Study start5 Feb 2020
- Primary completionApr 2028 (estimated)
- Study completionSep 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neonatal Hypoxic-ischaemic EncephalopathyStudies of the same condition, open and under way.
- More trials run by Assistance Publique Hopitaux De MarseilleThe sponsor's other registered studies.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique Hopitaux De Marseille
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- CATHERINE GEINDRE, Study director, AP HM
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02881970
- Sponsor's protocol ID
- 2013-A01018-37
- Other IDs
- 2013-32
- Acronym
- NEOSTEM
- Status last verified
- Aug 2024
- First submitted
- 24 Aug 2016
- Last updated
- 5 Aug 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Neonatal Hypoxic Ischemic Encephalopathy : Safety and Feasibility Study of a Curative Treatment With Autologous Cord Blood Stem Cells
- ClinicalTrials.gov ID
- NCT02881970
- Acronym / sponsor ID
- NEOSTEM / 2013-A01018-37
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Assistance Publique Hopitaux De MarseilleUniversity, hospital or other
- Study start
- 5 Feb 2020 (actual)
- Primary completion
- Apr 2028 (estimated)
- Study completion
- Sep 2028 (estimated)
- First posted
- 29 Aug 2016 (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.
Neonatal hypoxic-ischaemic encephalopathy is a dramatic perinatal complication due to brain asphyxia. Neurological and neurosensory sequelae are frequent in survivors, due to neuronal damage and loss.
Currently, only total or partial body hypothermia can partially prevent cell loss. However, no treatment exists to restore neuronal functions.
Cord blood stem cells are a promising treatment for the near future.
The primary objective of this study is to test the safety and feasibility of a curative treatment with autologous cord blood stem cell in neonatal hypoxic-ischaemic encephalopathy.
The secondary objectives are to test the efficacy of this curative treatment with cell with neurogenic potential on the prevention of neurologic sequelae, as well as to test the optimum timing of cell preparation administration
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Neonatal hypoxic-ischaemic encephalopathy | Experimental | Drug: autologous cord blood stem cell |
Who may qualify
- Ages
- 1 day to 3 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (12 listed)
- Term ≥ 36 weeks of gestation
- and (2) :
- a blood pH < 7 with base deficit > 12 mmol/l (at birth or within 60 minutes of age)
- or a blood pH between 7,01 and 7,15, with additionnal criteria:
- a history of acute perinatal event (e.g : abnormal fetal cardiac rate, cord prolapse, uterine rupture, maternal hemorrhage)
Key exclusion criteria (3 listed)
- presence of known chromosomal anomaly.
- presence of major congenital anomalies. severe intrauterine growth restriction (weight <1800g)
- infants in extremis for whom no additional intensive therapy will be offered by attending neonatologist.
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 cord blood stem cellInjection of 5.107 / kg autologous mononuclear cells from umbilical cord blood | Drug | Neonatal hypoxic-ischaemic encephalopathy |
Primary outcome
Adverse clinical or paraclinical event rates due to stem cell preparation
Time frame: 2years
1 secondary outcome
- Preliminary efficacy as measured by neurodevelopmental function
Time frame: 2years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Aug 2024.
- Individual participant dataSharing planned: No
Related trials for Neonatal Hypoxic-ischaemic Encephalopathy
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The PROTECT-HIE Pilot Trial
Hypoxic-ischemic Encephalopathy (HIE)
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A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of RLS-0071 in Newborns With Moderate or Severe Hypoxic-Ischemic Encephalopathy Undergoing Therapeutic Hypothermia
Hypoxic-Ischemic Encephalopathy
Not yet recruiting
Levetiracetam as an Adjuvant Therapy to Hypothermia in Neonatal Asphyxia
Hypoxic Ischemic Encephalopathy of Newborn, Neuro-protection
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A Dose Escalation Study of Levetiracetam in the Treatment of Neonatal Seizures
Neonatal Seizure, Neonatal Encephalopathy
RecruitingObservational study
Continuous Monitoring of Body Temperature in Neonates During Therapeutic Hypothermia
Therapeutic Hypothermia, Neonatal Hypoxic-Ischemic Encephalopathy
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.
Farid BOUBRED contact
CATHERINE GEINDRE study director
AP HM
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing autologous cord blood stem cell in people with Neonatal Hypoxic-ischaemic Encephalopathy, run by Assistance Publique Hopitaux De Marseille at 1 site in France.
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 12 inclusion and 3 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: looking for participants now, as of the sponsor's last update on 5 Aug 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 France. 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Farid BOUBRED (farid.boubrd@ap-hm.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse clinical or paraclinical event rates due to stem cell preparation (time frame: 2years). 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 5 Aug 2024.