Effect of Autologous Cord Blood Mononuclear Cells for Prevention of Bronchopulmonary Dysplasia or Death in Extremely Preterm Neonates

October 30, 2023 updated by: yang jie, Guangdong Women and Children Hospital

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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 Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
140

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • China
    • Guangdong
      • Dongguan, Guangdong, China
        • Recruiting
        • Ren Xuejun
        • Contact:
          • Ren Xuejun, MD
      • Guangzhou, Guangdong, China, 511442
        • Recruiting
        • Jie Yang
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

1 second to 6 months (Child)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: ACBMNC infusion group
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.
Biological: autologous cord blood mononuclear cells
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
Placebo Comparator: control group
Those in control group will receive an infusion of a placebo solution which is normal saline with the same volume.
Biological: normal saline
preterm neonates less than 28 weeks are assigned to receive normal saline within 24 hours after birth

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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.
36 weeks of postmenstrual age or discharge home whichever comes first.

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
mortality
Time Frame: 36 weeks of postmenstrual age or the discharge

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.
36 weeks of postmenstrual age or the discharge

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Guangdong Women and Children Hospital

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Hexian Memorial Affiliated Hospital of Southern Medical University
Foshan Women and Children Hospital
Heyuan Women and Children Hospital
Dongguan Women and Children Hospital
Guangzhou Huadu Women and Children Hospital
Guangdong Cord Blood Bank
Foshan Fuxing Chancheng Central Hospital
Shunde Women and Children Hospital
Huangdu Distric Women and Children Hospital
Longgang Distric Women and Children Hospital,Shenzhen
Zhongshan Boai Hospital
Huizhou first Women and Children Hospital
Huizhou second Women and Children Hospital

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Chair: Jie Yang, Guangdong Women and Children Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
June 20, 2020

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 31, 2023

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 31, 2023

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
June 18, 2020

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 18, 2020

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 22, 2020

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
November 1, 2023

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 30, 2023

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
October 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • Guang dong W C H

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

public

IPD Sharing Time Frame

all time

IPD Sharing Access Criteria

all

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • ANALYTIC_CODE
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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