BPDPhase 3NCT04440670
Effect of Autologous Cord Blood Mononuclear Cells for Prevention of Bronchopulmonary Dysplasia or Death in Extremely Preterm Neonates
A Phase 3 study testing autologous cord blood mononuclear cells and normal saline in people with BPD, run by Guangdong Women and Children Hospital at 2 sites in China.
Trial summary
This is the first and largest randomized, controlled, blinded trial that evaluates the efficacy of autologous cord blood mononuclear cells infusion as a prevention therapy for BPD or death. Read more about this study
- Who is it for?
- Ages 0 to 28 weeks
- Conditions the study is for
- BPD
- What is being tested?
- autologous cord blood mononuclear cells and normal saline
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 140 participants
- Where
- 2 sitesChina
- Study dates
- 20 Jun 2020to 31 Dec 2023
- Phase
- Phase 3
- 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 28 weeks
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Infants fulfilling all the following inclusion criteria will be enrolled in this trial: 1. born at study hospital; 2. singleton birth; 3. less than 28 weeks GA 4.Signed informed consent obtained; 5. had available umbilical cord blood (UCB).
Who cannot join
- Those infants are excluded if they were 1. with severe congenital abnormalities; 2.with maternal clinical chorioamnionitis 3. the mother was positive for hepatitis B (HBsAg and/or HBeAg) or C virus (anti-HCV), syphilis, HIV (anti-HIV-1 and -2) or IgM against cytomegalovirus, rubella, toxoplasma and herpes simplex virus.
Trial locations and contacts
2 sites in 1 country.
China2 sites, 2 recruiting
- Recruiting
Ren Xuejun
Dongguan, Guangdong
Ren Xuejun, MD
- Recruiting
Jie Yang
Guangzhou, Guangdong, 511442
Jie Yang, PHD, 39151777 ext. 020, jieyang@126.com
About this study
Official title: Effect of Autologous Cord Blood Mononuclear Cells for Prevention of Bronchopulmonary Dysplasia or Death in Extremely Preterm Neonates: a Placebo-controlled Randomized Multicenter Trial
This is the first and largest randomized, controlled, blinded trial that evaluates the efficacy of autologous cord blood mononuclear cells infusion as a prevention therapy for BPD or death. The results of this trial will provide valuable clinical evidence for recommendations on the management of BPD in extremely preterm infants. In this prospective, randomized controlled double-blind multi-center clinical trial, 140 extremely preterm neonates less than 28 weeks are randomly assigned to receive intravenous autologous cord blood mononuclear cells infusion (targeted dose of 5×107cells/kg but no less than 1×107cells/kg) or placebo ( normal saline) within 24 hours after birth in a 1:1 ratio using a central randomization system. The primary outcome is survival without bronchopulmonary dysplasia at 36 weeks of postmenstrual age or discharge home. The secondary outcomes will include mortality rate, BPD severity, other common preterm complication rate, respiratory support duration, the length and cost of hospitalization and long term outcomes after two years follow up post infusion.
Read the detailed description
Study design and settings:
This present study will be a randomized, placebo-controlled, double-blinded, multi-center trial to be conducted at 12 medical centers in tertiary hospitals with Neonatal Intensive Care Unit that were selected by the expert committee. A total of 140 neonates fulfilling the eligibility criteria will be enrolled. Subsequently, the participants will be randomly divided into two groups (ACBMNC infusion group and control (placebo) group ) in a ratio of 1:1.
Sample size:
Based on our previous study and others' study, we found the ACBMNC infusion was effective in reducing respiratory support duration in preterm infants. The rate of BPD among extremely preterm infants in our NICU was 60% (pA). What we expect to be an intended (or at least acceptable) effect of the ACBMNC infusion is 25 % reduction in frequency of BPD(pB:35%). To detect this difference with a sensitivity of 80% and an error probability of 5%, at least 59 patients per randomization group will be required using the following formula:
n=(pA(1-pA)/κ+pB(1-pB))((z1-α/2+z1-β)/(pA-pB))2 To account for the possibility of as high as 20% loss to follow-up, our estimated sample size is 140 cases totally.
Objectives:
Primary objective： The primary objective of this trial is to evaluate the efficacy of ACBMNC infusion in preventing bronchopulmonary dysplasia or death at 36 weeks of postmenstrual age or discharge home in extremely preterm infants.
Secondary objectives：
- To compare the mortality rate at 36 weeks of postmenstrual age.
- To compare the BPD severity
- To compare the rate of other common preterm complications included intraventricular hemorrhage (IVH), necrotizing enterocolitis (NEC), retinopathy of prematurity (ROP), respiratory distress syndrome (RDS), ventilation-associated pneumonia (VAP), hypoxic ischemic encephalopathy (HIE), late onset sepsis (LOS) and anemia.To compare the duration of mechanical ventilation and oxygen therapy in two groups
- To determine re-intubation rate and time return to BW
- To compare the duration of antibiotic usage
- To determine the long term outcomes after two years follow up
Participants:
Inclusion criteria:
Infants fulfilling all the following inclusion criteria will be enrolled in this trial: 1. born at study hospital; 2. singleton birth; 3. less than 28 weeks GA 4.Signed informed consent obtained; 5. had available umbilical cord blood (UCB).
Exclusion criteria:
Those infants are excluded if they were 1. with severe congenital abnormalities; 2.with maternal clinical chorioamnionitis 3. the mother was positive for hepatitis B (HBsAg and/or HBeAg) or C virus (anti-HCV), syphilis, HIV (anti-HIV-1 and -2) or IgM against cytomegalovirus, rubella, toxoplasma and herpes simplex virus.
Trial treatment methods:
Soon after the preterm infant was deliveried, written consent was signed by the parents, and autologous cord blood infusion was applied to the baby in addition to routine pulmonary surfactant replacement, and mechanical ventilation support as indicated. Those assigned to the ACBMNC group received an infusion of ACBMNC with 24 h after birth. Those in control group received an infusion of a placebo solution which is normal saline with the same volume. Cell dose for all patients was targeted at 5×107 cells per kilogram.
- Conditions
- BPD
- Being tested
- autologous cord blood mononuclear cells, normal saline
- Keywords
- Autologous cord blood mononuclear cells, bronchopulmonary dysplasia, extremely preterm neonates
- Medical subject headings
- Bronchopulmonary Dysplasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
autologous cord blood mononuclear cellsBiological
preterm neonates less than 28 weeks are assigned to receive intravenous autologous cord blood mononuclear cells infusion (5×107cells/kg) within 24 hours after birth
normal salineBiological
preterm neonates less than 28 weeks are assigned to receive normal saline within 24 hours after birth
| Study group | Role | What they receive |
|---|---|---|
| ACBMNC infusion group | Experimental | Those assigned to the ACBMNC group will receive intravenous autologous cord blood mononuclear cells infusion within 24 h after birth. Cell dose for all patients was targeted at 5×107 cells per kilogram. Receives: Biological: autologous cord blood mononuclear cells |
| control group | Placebo comparator | Those in control group will receive an infusion of a placebo solution which is normal saline with the same volume. Receives: Biological: normal saline |
What is being measured
The primary outcome is the main question the study is designed to answer.
frequency of bronchopulmonary dysplasia or death
The frequency of bronchopulmonary dysplasia or death at 36 weeks of postmenstrual age or discharge home whichever comes first.
Measured: 36 weeks of postmenstrual age or discharge home whichever comes first.
Secondary outcomes (1)
mortality
The mortality rate.
- Incidence of other preterm complications including intraventricular hemorrhage (IVH), periventricular leukomalacia (PVL), necrotizing enterocolitis (NEC), retinopathy of prematurity (ROP), respiratory distress syndrome (RDS), ventilation-associated pneumonia (VAP), late onset sepsis (LOS) and anemia .
- Duration of hospitalization.
- Duration of mechanical ventilation and oxygen therapy
- The frequency of re-intubation.
- The time (days) return to BW.
Measured: 36 weeks of postmenstrual age or the discharge
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jun 2020
- Study start20 Jun 2020
- Primary completion31 Dec 2023 (estimated)
- Study completion31 Dec 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for BPDStudies 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 3
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- In this prospective, randomized controlled double-blind multi-center clinical trial, 140 preterm neonates less than 28 weeks are randomly assigned to receive intravenous autologous cord blood mononuclear cells infusion (5×107cells/kg) or placebo ( normal saline) within 24 hours after birth in a 1:1 ratio using a central randomization system.
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Outcomes assessor
- Enrolment
- 140 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Guangdong Women and Children Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Foshan Fuxing Chancheng Central Hospital, Foshan Women's and Children's Hospital, Hexian Memorial Affiliated Hospital of Southern Medical University, Heyuan Women and Children Hospital, Dongguan Women and Children Hospital, Guangzhou Huadu Women and Children Hospital, Shunde Women and Children Hospital, Guangdong Cord Blood Bank, Huangdu Distric Women and Children Hospital, Longgang Distric Women and Children Hospital,Shenzhen, BoAi Hospital of Zhongshan, Huizhou first Women and Children Hospital, Huizhou second Women and Children Hospital
- Responsible party
- Principal investigator, yang jie, Guangdong Women and Children Hospital
- Investigators
- Jie Yang, Study chair, Guangdong Women and Children Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04440670
- Sponsor's protocol ID
- Guang dong W C H
- Status last verified
- Oct 2023
- First submitted
- 18 Jun 2020
- Last updated
- 1 Nov 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: public
Study at a glance
From the registry record, last updated by the sponsor on 1 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Autologous Cord Blood Mononuclear Cells for Prevention of Bronchopulmonary Dysplasia or Death in Extremely Preterm Neonates: a Placebo-controlled Randomized Multicenter Trial
- ClinicalTrials.gov ID
- NCT04440670
- Acronym / sponsor ID
- Guang dong W C H
- Conditions
- BPD
- Keywords
- Autologous cord blood mononuclear cells, bronchopulmonary dysplasia, extremely preterm neonates
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 140 participants (estimated)
- Lead sponsor
- Guangdong Women and Children HospitalUniversity, hospital or other
- Collaborators
- Foshan Fuxing Chancheng Central Hospital, Foshan Women's and Children's Hospital, Hexian Memorial Affiliated Hospital of Southern Medical University, Heyuan Women and Children Hospital, Dongguan Women and Children Hospital, Guangzhou Huadu Women and Children Hospital, Shunde Women and Children Hospital, Guangdong Cord Blood Bank, Huangdu Distric Women and Children Hospital, Longgang Distric Women and Children Hospital,Shenzhen, BoAi Hospital of Zhongshan, Huizhou first Women and Children Hospital, Huizhou second Women and Children Hospital
- Study start
- 20 Jun 2020 (actual)
- Primary completion
- 31 Dec 2023 (estimated)
- Study completion
- 31 Dec 2023 (estimated)
- First posted
- 22 Jun 2020 (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.
This is the first and largest randomized, controlled, blinded trial that evaluates the efficacy of autologous cord blood mononuclear cells infusion as a prevention therapy for BPD or death. The results of this trial will provide valuable clinical evidence for recommendations on the management of BPD in extremely preterm infants. In this prospective, randomized controlled double-blind multi-center clinical trial, 140 extremely preterm neonates less than 28 weeks are randomly assigned to receive intravenous autologous cord blood mononuclear cells infusion (targeted dose of 5×107cells/kg but no less than 1×107cells/kg) or placebo ( normal saline) within 24 hours after birth in a 1:1 ratio using a central randomization system. The primary outcome is survival without bronchopulmonary dysplasia at 36 weeks of postmenstrual age or discharge home. The secondary outcomes will include mortality rate, BPD severity, other common preterm complication rate, respiratory support duration, the length and cost of hospitalization and long term outcomes after two years follow up post infusion.
Detailed description
Study design and settings:
This present study will be a randomized, placebo-controlled, double-blinded, multi-center trial to be conducted at 12 medical centers in tertiary hospitals with Neonatal Intensive Care Unit that were selected by the expert committee. A total of 140 neonates fulfilling the eligibility criteria will be enrolled. Subsequently, the participants will be randomly divided into two groups (ACBMNC infusion group and control (placebo) group ) in a ratio of 1:1.
Sample size:
Based on our previous study and others' study, we found the ACBMNC infusion was effective in reducing respiratory support duration in preterm infants. The rate of BPD among extremely preterm infants in our NICU was 60% (pA). What we expect to be an intended (or at least acceptable) effect of the ACBMNC infusion is 25 % reduction in frequency of BPD(pB:35%). To detect this difference with a sensitivity of 80% and an error probability of 5%, at least 59 patients per randomization group will be required using the following formula:
n=(pA(1-pA)/κ+pB(1-pB))((z1-α/2+z1-β)/(pA-pB))2 To account for the possibility of as high as 20% loss to follow-up, our estimated sample size is 140 cases totally.
Objectives:
Primary objective： The primary objective of this trial is to evaluate the efficacy of ACBMNC infusion in preventing bronchopulmonary dysplasia or death at 36 weeks of postmenstrual age or discharge home in extremely preterm infants.
Secondary objectives：
- To compare the mortality rate at 36 weeks of postmenstrual age.
- To compare the BPD severity
- To compare the rate of other common preterm complications included intraventricular hemorrhage (IVH), necrotizing enterocolitis (NEC), retinopathy of prematurity (ROP), respiratory distress syndrome (RDS), ventilation-associated pneumonia (VAP), hypoxic ischemic encephalopathy (HIE), late onset sepsis (LOS) and anemia.To compare the duration of mechanical ventilation and oxygen therapy in two groups
- To determine re-intubation rate and time return to BW
- To compare the duration of antibiotic usage
- To determine the long term outcomes after two years follow up
Participants:
Inclusion criteria:
Infants fulfilling all the following inclusion criteria will be enrolled in this trial: 1. born at study hospital; 2. singleton birth; 3. less than 28 weeks GA 4.Signed informed consent obtained; 5. had available umbilical cord blood (UCB).
Exclusion criteria:
Those infants are excluded if they were 1. with severe congenital abnormalities; 2.with maternal clinical chorioamnionitis 3. the mother was positive for hepatitis B (HBsAg and/or HBeAg) or C virus (anti-HCV), syphilis, HIV (anti-HIV-1 and -2) or IgM against cytomegalovirus, rubella, toxoplasma and herpes simplex virus.
Trial treatment methods:
Soon after the preterm infant was deliveried, written consent was signed by the parents, and autologous cord blood infusion was applied to the baby in addition to routine pulmonary surfactant replacement, and mechanical ventilation support as indicated. Those assigned to the ACBMNC group received an infusion of ACBMNC with 24 h after birth. Those in control group received an infusion of a placebo solution which is normal saline with the same volume. Cell dose for all patients was targeted at 5×107 cells per kilogram.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, outcomes assessor)
- Number of arms
- 2
- Model description
- In this prospective, randomized controlled double-blind multi-center clinical trial, 140 preterm neonates less than 28 weeks are randomly assigned to receive intravenous autologous cord blood mononuclear cells infusion (5×107cells/kg) or placebo ( normal saline) within 24 hours after birth in a 1:1 ratio using a central randomization system.
| Arm | Type | Receives |
|---|---|---|
| ACBMNC infusion group | Experimental | Biological: autologous cord blood mononuclear cellsThose assigned to the ACBMNC group will receive intravenous autologous cord blood mononuclear cells infusion within 24 h after birth. Cell dose for all patients was targeted at 5×107 cells per kilogram. |
| control group | Placebo comparator | Biological: normal salineThose in control group will receive an infusion of a placebo solution which is normal saline with the same volume. |
Who may qualify
- Ages
- 0 to 28 weeks
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Infants fulfilling all the following inclusion criteria will be enrolled in this trial: 1. born at study hospital; 2. singleton birth; 3. less than 28 weeks GA 4.Signed informed consent obtained; 5. had available umbilical cord blood (UCB).
Key exclusion criteria (1 listed)
- Those infants are excluded if they were 1. with severe congenital abnormalities; 2.with maternal clinical chorioamnionitis 3. the mother was positive for hepatitis B (HBsAg and/or HBeAg) or C virus (anti-HCV), syphilis, HIV (anti-HIV-1 and -2) or IgM against cytomegalovirus, rubella, toxoplasma and herpes simplex virus.
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 mononuclear cellspreterm neonates less than 28 weeks are assigned to receive intravenous autologous cord blood mononuclear cells infusion (5×107cells/kg) within 24 hours after birth | Biological | ACBMNC infusion group |
| normal salinepreterm neonates less than 28 weeks are assigned to receive normal saline within 24 hours after birth | Biological | control group |
Primary outcome
frequency of bronchopulmonary dysplasia or death
Time frame: 36 weeks of postmenstrual age or discharge home whichever comes first.
The frequency of bronchopulmonary dysplasia or death at 36 weeks of postmenstrual age or discharge home whichever comes first.
1 secondary outcome
mortality
Time frame: 36 weeks of postmenstrual age or the discharge
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Nov 2023.
- Individual participant dataSharing planned: Yes. public
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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.
Jie Yang study chair
Guangdong Women and Children Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing autologous cord blood mononuclear cells and normal saline in people with BPD, run by Guangdong Women and Children Hospital at 2 sites in China.
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 1 inclusion and 1 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 1 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in China. 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. Some participants in this study may receive a placebo. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 zhuxiao Ren, MD (+8613538984634, renzhx1990@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
frequency of bronchopulmonary dysplasia or death (time frame: 36 weeks of postmenstrual age or discharge home whichever comes first.). 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 1 Nov 2023.