Congenital Diaphragmatic HerniaPhase 1NCT03526588
Umbilical Cord Blood Mononuclear Cells for Hypoxic Neurologic Injury in Infants With Congenital Diaphragmatic Hernia (CDH)
A Phase 1 study testing Autologous umbilical cord blood in people with Congenital Diaphragmatic Hernia, run by The University of Texas Health Science Center, Houston at 1 site in United States.
Trial summary
The purpose of this study is to investigate the use of autologous umbilical cord blood (UCB) mononuclear cells to mitigate hypoxic neurologic injury among infants with high-risk congenital diaphragmatic hernia (CDH). Read more about this study
- Who is it for?
- Ages 10 minutes to 7 days
- Conditions the study is for
- Congenital Diaphragmatic Hernia
- What is being tested?
- Autologous umbilical cord blood
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Aug 2018to 1 Jul 2024
- 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 10 minutes to 7 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Diagnosis of CDH between 20 and 36 weeks estimated gestational age (EGA)
- Only one of the following fetal criteria and one of the following postnatal criteria must be met for enrollment. Fetal criteria: an ultrasound (US)-obtained observed to expected lung to head ratio (o/e LHR) less than or equal to 35% or 2) a fetal magnetic resonance imaging (fMRI)- obtained observed to expected total fetal lung volume (o/e TFLV) less than or equal to 35%. Postnatal criteria: 1) Cord blood gas (CBG) with potenital hydrogen (pH) <7.0, 2) Arterial blood gas (ABG) with pH <7.2 on 2 gasses within the first 24 hours, 3) Preductal oxygen saturation (O2 sat) <90% x 2 total hours (not necessarily consecutive) within the first 24 hours, or 4) Oxygenation Index (OI) >20 x 2 total hours (not necessarily consecutive) within the first 24 hours.
Who cannot join
- Genetic/chromosomal abnormality: Trisomy 21, Trisomy 18, Trisomy 13 or other, significant genetic abnormality. Microdeletions or other mild genetic abnormalities are not considered exclusionary.
- Severe/major cardiac anomaly: coarctation of the aorta, combined atrial and ventricular septal defects, hypoplastic left heart syndrome, tetralogy of fallot, double outlet right ventricle, atrioventricular canal defects, or other hemodynamically significant defects.
- Moderate/severe neurologic / intracranial abnormality: Grade III or IV intraparenchymal hemorrhage, space occupying mass or lesion, or clinically significant traumatic lesion such as a subdural or epidural hemorrhage.
- Prematurity <30 weeks estimated gestational age (EGA): Birth at 29 6/7 weeks or before
- Participation in an alternative prenatal intervention study: Fetoscopic Endotracheal Occlusion (FETO)
- Unwillingness / inability to return for follow-up evaluation and assessment
Trial locations and contacts
1 site in 1 country.
United States1 site
The University of Texas Health Science Center at Houston
Houston, Texas, 77030
About this study
The purpose of this study is to investigate the use of autologous umbilical cord blood (UCB) mononuclear cells to mitigate hypoxic neurologic injury among infants with high-risk congenital diaphragmatic hernia (CDH).
- Conditions
- Congenital Diaphragmatic Hernia
- Being tested
- Autologous umbilical cord blood
- Keywords
- CDH
- Medical subject headings
- Hernias, Diaphragmatic, Congenital
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous umbilical cord bloodBiological
6×10^6 mononuclear cells isolated from the patient's own umbilical cord blood per dose. 4 total doses administered intravenously over 7 days.
| Study group | Role | What they receive |
|---|---|---|
| Autologous umbilical cord blood | Experimental | Not described Receives: Biological: Autologous umbilical cord blood |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety as assessed by vital sign monitoring (heart rate)
Measured: daily for 7 days following the initial infusion
Safety as assessed by vital sign monitoring (systolic blood pressure)
Measured: daily for 7 days following the initial infusion
Safety as assessed by vital sign monitoring (diastolic blood pressure)
Measured: daily for 7 days following the initial infusion
Safety as assessed by vital sign monitoring (temperature)
Measured: daily for 7 days following the initial infusion
Safety as assessed by pulmonary status (indicated by peak inspiratory pressure (PIP))
Measured: daily for 7 days following the initial infusion
Safety as assessed by pulmonary status (indicated by positive end expiratory pressure (PEEP))
Measured: daily for 7 days following the initial infusion
Safety as assessed by pulmonary status (indicated by respiratory rate (RR))
Measured: daily for 7 days following the initial infusion
Safety as assessed by pulmonary status (indicated by Fraction of inspired oxygen (FiO2))
Measured: daily for 7 days following the initial infusion
Safety as assessed by presence of new infiltrates or altered aeration upon chest radiography
Measured: daily for 7 days following the initial infusion
Safety as assessed by cardiovascular status (indicated by heart rate)
Measured: daily for 7 days following the initial infusion
Safety as assessed by cardiovascular status (indicated by systolic blood pressure)
Measured: daily for 7 days following the initial infusion
Safety as assessed by cardiovascular status (indicated by diastolic blood pressure)
Measured: daily for 7 days following the initial infusion
Safety as assessed by cardiovascular status (indicated by changes in cardiovascular pharmacologic support)
Measured: daily for 7 days following the initial infusion
Safety as assessed by infection status (indicated by body temperature)
Measured: daily for 7 days following the initial infusion
Safety as assessed by infection status (indicated by white blood cell count)
Measured: 7 days following the initial infusion
Safety as assessed by infection status (indicated by physical signs of infection)
Measured: daily for 7 days following the initial infusion
Safety as assessed by liver function (indicated by Alanine aminotransferase (ALT) levels)
Measured: 7 days following the initial infusion
Safety as assessed by liver function (indicated by aspartate aminotransferase (AST) levels
Measured: 7 days following the initial infusion
Safety as assessed by liver function (indicated by bilirubin levels)
Measured: 7 days following the initial infusion
Safety as assessed by liver function (indicated by albumin levels)
Measured: 7 days following the initial infusion
Safety as assessed by blood urea nitrogen (BUN) levels
Measured: 7 days following the initial infusion
Safety as assessed by creatinine levels
Measured: 7 days following the initial infusion
Safety as assessed by carbon dioxide (CO2) levels
Measured: 7 days following the initial infusion
Safety as assessed by glucose levels
Measured: 7 days following the initial infusion
Safety as assessed by serum chloride levels
Measured: 7 days following the initial infusion
Safety as assessed by serum potassium levels
Measured: 7 days following the initial infusion
Safety as assessed by serum sodium levels
Measured: 7 days following the initial infusion
Neurologic/neurodevelopmental status as assessed by intracranial abnormalities upon magnetic resonance imaging (MRI)
Measured: within 14 days of discharge (discharge occurs at about 2-4 months after birth)
Neurologic/neurodevelopmental status as assessed by receipt of neurologic pharmacologic medications
Measured: at the time of discharge (which is about 2-4 months after birth)
Neurologic/neurodevelopmental status as assessed by Bayley Scales of Infant and Toddler Development-III (BSID-III)
The Bayley-III is an individually-administered examination that assesses the current developmental functioning of infants and young children from birth to 42 months of age. The Bayley is a standardized, norm-referenced measure that assesses development in Cognitive, Language and Motor domains. Composite standard scores can be derived that have a mean of 100 and a standard deviation of 15.
Measured: 2 years after birth
Secondary outcomes (5)
Mortality
Measured: 2 years after birth
Length of stay in hospital
Measured: from birth to discharge or death, whichever occurs first (discharge occurs at about 2-4 months after birth)
Progression of pulmonary hypertension as assessed by echocardiography
Measured: within 24 hours of birth, prior to operative repair (occurs between day 2 & 14 of life), prior to discharge (usually 2-6 months), and after discharge (2wks-6 months following discharge)
Duration of extracorporeal membrane oxygenation (ECMO) support
Measured: days from ECMO initiation until decannulation (an average of 3 weeks)
Duration of ventilatory support
Measured: from initiation of ventilation until extubation (an average of 8 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 May 2018
- Study start1 Aug 2018
- Primary completion1 Jul 2022
- Study completion1 Jul 2024
Why it stopped: Competing randomized trial of an experimental therapy limited initial enrollment. The trial was completed, published, and the experimental therapy became broadly desired. Given this, the investigators felt it best to terminate this competing study.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital Diaphragmatic HerniaStudies of the same condition, open and under way.
- More trials run by The University of Texas Health Science Center, HoustonThe 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Texas Medical Center Regenerative Medicine Consortium
- Responsible party
- Principal investigator, Matthew T Harting, The University of Texas Health Science Center, Houston
- Investigators
- Matthew T. Harting, MD, MS, Principal investigator, The University of Texas Health Science Center, Houston
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03526588
- Sponsor's protocol ID
- HSC-MS-18-0148
- Status last verified
- Jul 2024
- First submitted
- 27 Apr 2018
- Last updated
- 11 Jul 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Umbilical Cord Blood Mononuclear Cells for Hypoxic Neurologic Injury in Infants With Congenital Diaphragmatic Hernia (CDH)
- ClinicalTrials.gov ID
- NCT03526588
- Acronym / sponsor ID
- HSC-MS-18-0148
- Conditions
- Congenital Diaphragmatic Hernia
- Keywords
- CDH
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Competing randomized trial of an experimental therapy limited initial enrollment. The trial was completed, published, and the experimental therapy became broadly desired. Given this, the investigators felt it best to terminate this competing study.
- Enrolment
- 3 participants (actual)
- Lead sponsor
- The University of Texas Health Science Center, HoustonUniversity, hospital or other
- Collaborators
- Texas Medical Center Regenerative Medicine Consortium
- Study start
- 1 Aug 2018 (actual)
- Primary completion
- 1 Jul 2022 (actual)
- Study completion
- 1 Jul 2024 (actual)
- First posted
- 16 May 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.
The purpose of this study is to investigate the use of autologous umbilical cord blood (UCB) mononuclear cells to mitigate hypoxic neurologic injury among infants with high-risk congenital diaphragmatic hernia (CDH).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Autologous umbilical cord blood | Experimental | Biological: Autologous umbilical cord blood |
Who may qualify
- Ages
- 10 minutes to 7 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Diagnosis of CDH between 20 and 36 weeks estimated gestational age (EGA)
- Only one of the following fetal criteria and one of the following postnatal criteria must be met for enrollment. Fetal criteria: an ultrasound (US)-obtained observed to expected lung to head ratio (o/e LHR) less than or equal to 35% or 2) a fetal magnetic resonance imaging (fMRI)- obtained observed to expected total fetal lung volume (o/e TFLV) less than or equal to 35%. Postnatal criteria: 1) Cord blood gas (CBG) with potenital hydrogen (pH) <7.0, 2) Arterial blood gas (ABG) with pH <7.2 on 2 gasses within the first 24 hours, 3) Preductal oxygen saturation (O2 sat) <90% x 2 total hours (not necessarily consecutive) within the first 24 hours, or 4) Oxygenation Index (OI) >20 x 2 total hours (not necessarily consecutive) within the first 24 hours.
Key exclusion criteria (6 listed)
- Genetic/chromosomal abnormality: Trisomy 21, Trisomy 18, Trisomy 13 or other, significant genetic abnormality. Microdeletions or other mild genetic abnormalities are not considered exclusionary.
- Severe/major cardiac anomaly: coarctation of the aorta, combined atrial and ventricular septal defects, hypoplastic left heart syndrome, tetralogy of fallot, double outlet right ventricle, atrioventricular canal defects, or other hemodynamically significant defects.
- Moderate/severe neurologic / intracranial abnormality: Grade III or IV intraparenchymal hemorrhage, space occupying mass or lesion, or clinically significant traumatic lesion such as a subdural or epidural hemorrhage.
- Prematurity <30 weeks estimated gestational age (EGA): Birth at 29 6/7 weeks or before
- Participation in an alternative prenatal intervention study: Fetoscopic Endotracheal Occlusion (FETO)
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 blood6×10^6 mononuclear cells isolated from the patient's own umbilical cord blood per dose. 4 total doses administered intravenously over 7 days. | Biological | Autologous umbilical cord blood |
Primary outcome
Safety as assessed by vital sign monitoring (heart rate)
Time frame: daily for 7 days following the initial infusion
Safety as assessed by vital sign monitoring (systolic blood pressure)
Time frame: daily for 7 days following the initial infusion
Safety as assessed by vital sign monitoring (diastolic blood pressure)
Time frame: daily for 7 days following the initial infusion
Safety as assessed by vital sign monitoring (temperature)
Time frame: daily for 7 days following the initial infusion
Safety as assessed by pulmonary status (indicated by peak inspiratory pressure (PIP))
Time frame: daily for 7 days following the initial infusion
Safety as assessed by pulmonary status (indicated by positive end expiratory pressure (PEEP))
Time frame: daily for 7 days following the initial infusion
Safety as assessed by pulmonary status (indicated by respiratory rate (RR))
Time frame: daily for 7 days following the initial infusion
Safety as assessed by pulmonary status (indicated by Fraction of inspired oxygen (FiO2))
Time frame: daily for 7 days following the initial infusion
Safety as assessed by presence of new infiltrates or altered aeration upon chest radiography
Time frame: daily for 7 days following the initial infusion
Safety as assessed by cardiovascular status (indicated by heart rate)
Time frame: daily for 7 days following the initial infusion
Safety as assessed by cardiovascular status (indicated by systolic blood pressure)
Time frame: daily for 7 days following the initial infusion
Safety as assessed by cardiovascular status (indicated by diastolic blood pressure)
Time frame: daily for 7 days following the initial infusion
Safety as assessed by cardiovascular status (indicated by changes in cardiovascular pharmacologic support)
Time frame: daily for 7 days following the initial infusion
Safety as assessed by infection status (indicated by body temperature)
Time frame: daily for 7 days following the initial infusion
Safety as assessed by infection status (indicated by white blood cell count)
Time frame: 7 days following the initial infusion
Safety as assessed by infection status (indicated by physical signs of infection)
Time frame: daily for 7 days following the initial infusion
Safety as assessed by liver function (indicated by Alanine aminotransferase (ALT) levels)
Time frame: 7 days following the initial infusion
Safety as assessed by liver function (indicated by aspartate aminotransferase (AST) levels
Time frame: 7 days following the initial infusion
Safety as assessed by liver function (indicated by bilirubin levels)
Time frame: 7 days following the initial infusion
Safety as assessed by liver function (indicated by albumin levels)
Time frame: 7 days following the initial infusion
Safety as assessed by blood urea nitrogen (BUN) levels
Time frame: 7 days following the initial infusion
Safety as assessed by creatinine levels
Time frame: 7 days following the initial infusion
Safety as assessed by carbon dioxide (CO2) levels
Time frame: 7 days following the initial infusion
Safety as assessed by glucose levels
Time frame: 7 days following the initial infusion
Safety as assessed by serum chloride levels
Time frame: 7 days following the initial infusion
Safety as assessed by serum potassium levels
Time frame: 7 days following the initial infusion
Safety as assessed by serum sodium levels
Time frame: 7 days following the initial infusion
Neurologic/neurodevelopmental status as assessed by intracranial abnormalities upon magnetic resonance imaging (MRI)
Time frame: within 14 days of discharge (discharge occurs at about 2-4 months after birth)
Neurologic/neurodevelopmental status as assessed by receipt of neurologic pharmacologic medications
Time frame: at the time of discharge (which is about 2-4 months after birth)
Neurologic/neurodevelopmental status as assessed by Bayley Scales of Infant and Toddler Development-III (BSID-III)
Time frame: 2 years after birth
The Bayley-III is an individually-administered examination that assesses the current developmental functioning of infants and young children from birth to 42 months of age. The Bayley is a standardized, norm-referenced measure that assesses development in Cognitive, Language and Motor domains. Composite standard scores can be derived that have a mean of 100 and a standard deviation of 15.
5 secondary outcomes
Mortality
Time frame: 2 years after birth
Length of stay in hospital
Time frame: from birth to discharge or death, whichever occurs first (discharge occurs at about 2-4 months after birth)
Progression of pulmonary hypertension as assessed by echocardiography
Time frame: within 24 hours of birth, prior to operative repair (occurs between day 2 & 14 of life), prior to discharge (usually 2-6 months), and after discharge (2wks-6 months following discharge)
Duration of extracorporeal membrane oxygenation (ECMO) support
Time frame: days from ECMO initiation until decannulation (an average of 3 weeks)
Duration of ventilatory support
Time frame: from initiation of ventilation until extubation (an average of 8 weeks)
Results and safety
The study was stopped early (reason given: Competing randomized trial of an experimental therapy limited initial enrollment. The trial was completed, published, and the experimental therapy became broadly desired. Given this, the investigators felt it best to terminate this competing study.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jul 2024.
- The Comprehensive Center for CDH Care at Children's Memorial Hermann Hospital and The University of Texas Health Science Center in Houston (Texas Medical Center)Link provided by the sponsor.
Related trials for Congenital Diaphragmatic Hernia
Enrolling by invitationObservational study
North American Fetal Therapy Network Prospective Registry for Long-term Outcome Following Fetoscopic Endoluminal Tracheal Occlusion in Severe Left and Right Congenital Diaphragmatic Hernia
Congenital Diaphragmatic Hernia
Active, not recruiting
Fetoscopic Endoluminal Tracheal Occlusion in Severe Left Congenital Diaphragmatic Hernia
Congenital Diaphragmatic Hernia, Pulmonary Hypoplasia
Active, not recruitingPhase 2
Effect of a Parenteral Emulsion With Omega3 on Neonates With PPHN and CDH
Pulmonary Hypertension of Newborn, Diaphragm Defect
RecruitingPhase 4
De-implementing Inhaled Nitric Oxide for Congenital Diaphragmatic Hernia
Congenital Diaphragmatic Hernia
Active, not recruiting
Fetoscopic Endoluminal Tracheal Occlusion (FETO) for Severe Left Diaphragmatic Hernia (CDH)
Congenital Diaphragmatic Hernia
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.
Matthew T. Harting, MD, MS principal investigator
The University of Texas Health Science Center, Houston
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 in people with Congenital Diaphragmatic Hernia, run by The University of Texas Health Science Center, Houston 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 2 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: stopped early, as of the sponsor's last update on 11 Jul 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 was stopped early (reason given: Competing randomized trial of an experimental therapy limited initial enrollment. The trial was completed, published, and the experimental therapy became broadly desired. Given this, the investigators felt it best to terminate this competing study.). Results are not available in the source record.
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?
Safety as assessed by vital sign monitoring (heart rate) (time frame: daily for 7 days following the initial infusion). The study lists 30 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 11 Jul 2024.