Anemia, NeonatalPhase 1NCT02050971
Autologous Cord Blood Infusion for the Prevention and Treatment of Prematurity Complications In Preterm Neonates
A Phase 1 study testing Umbilical Cord Blood Infusion and Intravenous Infusion of Peripheral Blood or its Components in people with Anemia, Neonatal and Intracranial Hemorrhages, run by Pomeranian Medical University Szczecin at 1 site in Poland.
Trial summary
The purpose of this study is to test the safety and effectiveness of a whole own (autologous) umbilical cord blood transfusion in the first 5 days after birth if the baby is born premature <34 weeks and developed anemia of prematurity. Read more about this study
- Who is it for?
- Ages Up to 30 days
- Conditions the study is for
- Anemia, Neonatal, Intracranial Hemorrhages, Necrotizing Enterocolitis
- What is being tested?
- Umbilical Cord Blood Infusion and Intravenous Infusion of Peripheral Blood or its Components
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 1 sitePoland
- Study dates
- Oct 2010to Dec 2015
- 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 Up to 30 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- preterm neonates less than 34 weeks of gestation, who developed anemia of prematurity,
- available unit of autologous umbilical cord blood
Who cannot join
- major congenital or chromosomal abnormalities,
- intrauterine infection,
- cyanotic heart defect,
- chronic intrauterine hypoxia (defined as growth retardation or pathologies of placental perfusion),
- incompatibilities in main blood groups and Rh antygen,
- lack of parental consent for enrollment to the study,
- contraindications for cord blood collection (lack of consent, amniotic fluid leakage for longer than 6 hours or physical complications in the cord blood harvesting).
Trial locations and contacts
1 site in 1 country.
Poland1 site
Department of Neonatology of Pomeranian Medical University in Szczecin, Poland
Szczecin, 70-111
About this study
Official title: Safety and Effectiveness Phase 1 Study of Autologous Umbilical Cord Blood Transfusion for the Prevention and Treatment of Prematurity Complications In Preterm Neonates
The purpose of this study is to test the safety and effectiveness of a whole own (autologous) umbilical cord blood transfusion in the first 5 days after birth if the baby is born premature <34 weeks and developed anemia of prematurity.
Read the detailed description
The purpose of this pilot study is to conduct the investigation of the safety and efficacy of autologous cord blood infusion in premature neonates who demonstrate anemia due to prematurity (most common prematurity complication). However, premature infants reveal a high risk of other acute complications, including brain injury (e.g., intraventricular hemorrhage; IVH), necrotizing enterocolitis (NEC), and neonatal respiratory distress syndrome (RDS), as well as retinopathy of prematurity (ROP) and bronchopulmonary dysplasia (BPD). Therefore, prematurity is considered one of the main causes of neonatal deaths. The preterm neonates need transfusion of allogenic whole peripheral blood or any of its components at a time of anemia of prematurity development. In contrast, other prematurity complications do not have effective treatment nor preventive strategies. We will enroll premature neonates born premature (<34 weeks of gestation) who developed anemia of prematurity and had their own autologous cord blood collected for subsequent transfusion. Next, we will test tolerability, safety and efficacy of autogenic whole cord blood infusion and evaluate the frequency of premature complications in neonates after transfusion. Besides, this pilot study will test feasibility of technical collection, preparation and infusion of a neonate's own umbilical cord blood within the first 5 days after birth.
- Conditions
- Anemia, Neonatal, Intracranial Hemorrhages, Necrotizing Enterocolitis, Respiratory Distress Syndrome, Newborn, Retinopathy of Prematurity, Bronchopulmonary Dysplasia
- Being tested
- Umbilical Cord Blood Infusion, Intravenous Infusion of Peripheral Blood or its Components
- Keywords
- Umbilical Cord Blood, Autologous Umbilical Cord Blood Transfusion, Prematurity Complications, Infant, Premature, Diseases, Infant, Newborn, Diseases, Hematologic Diseases, Lung Diseases, Central Nervous System Diseases, Gastrointestinal Tract Diseases, Vascular Diseases, Retinopathies, Neonates, Prevention,Treatment
- Medical subject headings
- Anemia, Neonatal, Intracranial Hemorrhages, Enterocolitis, Necrotizing, Respiratory Distress Syndrome, Newborn, Retinopathy of Prematurity, Bronchopulmonary Dysplasia, Infant, Premature, Diseases, Infant, Newborn, Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Umbilical Cord Blood InfusionBiological
Also called Whole Autologous Umbilical Cord Blood Infusion
Cord blood collection after delivery of the baby. Preservation of blood in blood bank. Transfusion of autologous cord blood within the first 5 postnatal days.
Intravenous Infusion of Peripheral Blood or its ComponentsBiological
Also called Allogeneic peripheral blood or its components transfusion
Regular treatment of neonatal anemia with peripheral blood or its components transfusion.
| Study group | Role | What they receive |
|---|---|---|
| Autologous cord blood transfusion | Active comparator | Treatment Group 1 Interventions: collected autologous whole cord blood at birth will be transfused for the preterm neonate Receives: Biological: Umbilical Cord Blood Infusion |
| Standard treatment for neonatal anemia | Sham comparator | Treatment Group 2 Interventions: transfusion of allogeneic whole peripheral blood or any of its components at a time of anemia of prematurity development Receives: Biological: Intravenous Infusion of Peripheral Blood or its Components |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of autologous cord blood infusion in enrolled preterm neonates.
Confirm the safety of autologous cord blood infusion in preterm neonates by repeated follow-up over one year with clinical and laboratory evaluations.
Measured: 1 year
Secondary outcomes (1)
Efficacy of autologous cord blood infusion in enrolled preterm neonates.
Confirm the efficacy of autologous cord blood infusion in preterm neonates by repeated follow-up over one year with clinical and laboratory evaluations for neurodevelopmental and general health outcomes at 3, 6 and 12 months of age. Particularly, analysis of prematurity complications will be performed.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jan 2014 (estimated)
- Study startOct 2010
- Primary completionDec 2014 (estimated)
- Study completionDec 2015 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Anemia, NeonatalStudies 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
- Prevention
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Pomeranian Medical University Szczecin
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Milosz Kawa, Pomeranian Medical University Szczecin
- Investigators
- Boguslaw Machalinski, MD, PhD, BSc, Study chair, Department of General Pathology, Pomeranian Medical University in Szczecin, PolandJacek Rudnicki, MD, PhD, BSc, Study director, Department of Neonatology, Pomeranian Medical University in Szczecin, PolandMilosz Piotr Kawa, MD, PhD, Principal investigator, Department of General Pathology, Pomeranian Medical University in Szczecin, Poland
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02050971
- Sponsor's protocol ID
- ZPO 01
- Other IDs
- CB-P#1
- Status last verified
- Jan 2014
- First submitted
- 29 Jan 2014
- Last updated
- 31 Jan 2014
- Results posted
- No
- Data monitoring committee
- No
Publications
Rudnicki J, Kawa MP, Kotowski M, Michalczyk B, Ustianowski P, Czajka R, Machalinski B. Clinical Evaluation of the Safety and Feasibility of Whole Autologous Cord Blood Transplant as a Source of Stem and Progenitor Cells for Extremely Premature Neonates: Preliminary Report. Exp Clin Transplant. 2015 Dec;13(6):563-72.
PubMed 26643677
Study at a glance
From the registry record, last updated by the sponsor on 31 Jan 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Effectiveness Phase 1 Study of Autologous Umbilical Cord Blood Transfusion for the Prevention and Treatment of Prematurity Complications In Preterm Neonates
- ClinicalTrials.gov ID
- NCT02050971
- Acronym / sponsor ID
- ZPO 01
- Conditions
- Anemia, Neonatal, Intracranial Hemorrhages, Necrotizing Enterocolitis, Respiratory Distress Syndrome, Newborn, Retinopathy of Prematurity, Bronchopulmonary Dysplasia
- Keywords
- Umbilical Cord Blood, Autologous Umbilical Cord Blood Transfusion, Prematurity Complications, Infant, Premature, Diseases, Infant, Newborn, Diseases, Hematologic Diseases, Lung Diseases, Central Nervous System Diseases
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Pomeranian Medical University SzczecinUniversity, hospital or other
- Study start
- Oct 2010
- Primary completion
- Dec 2014 (estimated)
- Study completion
- Dec 2015 (estimated)
- First posted
- 31 Jan 2014 (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.
The purpose of this study is to test the safety and effectiveness of a whole own (autologous) umbilical cord blood transfusion in the first 5 days after birth if the baby is born premature <34 weeks and developed anemia of prematurity.
Detailed description
The purpose of this pilot study is to conduct the investigation of the safety and efficacy of autologous cord blood infusion in premature neonates who demonstrate anemia due to prematurity (most common prematurity complication). However, premature infants reveal a high risk of other acute complications, including brain injury (e.g., intraventricular hemorrhage; IVH), necrotizing enterocolitis (NEC), and neonatal respiratory distress syndrome (RDS), as well as retinopathy of prematurity (ROP) and bronchopulmonary dysplasia (BPD). Therefore, prematurity is considered one of the main causes of neonatal deaths. The preterm neonates need transfusion of allogenic whole peripheral blood or any of its components at a time of anemia of prematurity development. In contrast, other prematurity complications do not have effective treatment nor preventive strategies. We will enroll premature neonates born premature (<34 weeks of gestation) who developed anemia of prematurity and had their own autologous cord blood collected for subsequent transfusion. Next, we will test tolerability, safety and efficacy of autogenic whole cord blood infusion and evaluate the frequency of premature complications in neonates after transfusion. Besides, this pilot study will test feasibility of technical collection, preparation and infusion of a neonate's own umbilical cord blood within the first 5 days after birth.
Study design
- Primary purpose
- Prevention
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Autologous cord blood transfusion | Active comparator | Biological: Umbilical Cord Blood InfusionTreatment Group 1 Interventions: collected autologous whole cord blood at birth will be transfused for the preterm neonate |
| Standard treatment for neonatal anemia | Sham comparator | Biological: Intravenous Infusion of Peripheral Blood or its ComponentsTreatment Group 2 Interventions: transfusion of allogeneic whole peripheral blood or any of its components at a time of anemia of prematurity development |
Who may qualify
- Ages
- Up to 30 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- preterm neonates less than 34 weeks of gestation, who developed anemia of prematurity,
- available unit of autologous umbilical cord blood
Key exclusion criteria (7 listed)
- major congenital or chromosomal abnormalities,
- intrauterine infection,
- cyanotic heart defect,
- chronic intrauterine hypoxia (defined as growth retardation or pathologies of placental perfusion),
- incompatibilities in main blood groups and Rh antygen,
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 |
|---|---|---|
| Umbilical Cord Blood InfusionAlso: Whole Autologous Umbilical Cord Blood InfusionCord blood collection after delivery of the baby. Preservation of blood in blood bank. Transfusion of autologous cord blood within the first 5 postnatal days. | Biological | Autologous cord blood transfusion |
| Intravenous Infusion of Peripheral Blood or its ComponentsAlso: Allogeneic peripheral blood or its components transfusionRegular treatment of neonatal anemia with peripheral blood or its components transfusion. | Biological | Standard treatment for neonatal anemia |
Primary outcome
Safety of autologous cord blood infusion in enrolled preterm neonates.
Time frame: 1 year
Confirm the safety of autologous cord blood infusion in preterm neonates by repeated follow-up over one year with clinical and laboratory evaluations.
1 secondary outcome
Efficacy of autologous cord blood infusion in enrolled preterm neonates.
Time frame: 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jan 2014.
- Derived from this study: PubMed 26643677Rudnicki J, Kawa MP, Kotowski M, Michalczyk B, Ustianowski P, Czajka R, Machalinski B. Clinical Evaluation of the Safety and Feasibility of Whole Autologous Cord Blood Transplant as a Source of Stem and Progenitor Cells for Extremely Premature Neonates: Preliminary Report. Exp Clin Transplant. 2015 Dec;13(6):563-72.
- Website of the Pomeranian Medical University in Szczecin, PolandLink provided by the sponsor.
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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.
Boguslaw Machalinski, MD, PhD, BSc study chair
Department of General Pathology, Pomeranian Medical University in Szczecin, Poland
Jacek Rudnicki, MD, PhD, BSc study director
Department of Neonatology, Pomeranian Medical University in Szczecin, Poland
Milosz Piotr Kawa, MD, PhD principal investigator
Department of General Pathology, Pomeranian Medical University in Szczecin, Poland
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 Umbilical Cord Blood Infusion and Intravenous Infusion of Peripheral Blood or its Components in people with Anemia, Neonatal and Intracranial Hemorrhages, run by Pomeranian Medical University Szczecin at 1 site in Poland.
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 7 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 31 Jan 2014. 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 Poland. 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 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?
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 of autologous cord blood infusion in enrolled preterm neonates. (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02050971, which also lists 1 linked publication.
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 31 Jan 2014.