Fetal and Neonatal Alloimmune ThrombocytopeniaNCT03561909
Kinetics of Blood Platelets Transfused to Healthy Subjects
A study testing Platelet transfusion in people with Fetal and Neonatal Alloimmune Thrombocytopenia, run by Prophylix Pharma AS at 1 site in Germany.
Trial summary
The current phase 0 trial is preceding the phase 1/2 trial of a newly developed drug, NAITgam, for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT) - a rare, but potentially very severe bleeding condition in the fetus or newborn. Read more about this study
- Who is it for?
- Ages 18 to 50 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Fetal and Neonatal Alloimmune Thrombocytopenia
- What is being tested?
- Platelet transfusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 1 siteGermany
- Study dates
- 17 Apr 2018to 10 Dec 2018
- Phase
- Interventional
- 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 18 to 50 years
- Men only
- Healthy volunteers accepted
- Adult
Who may be able to join
- Written informed consent must be obtained before any trial related procedures are performed
- Healthy, male subjects
- Age ≥18 and < 50 years old
- BMI < 30kg/mˆ2
- HLA-A2 and/or HLA-A9 negative
Who cannot join
- History of hypersensitivity to platelet concentrates or human plasma protein
- Subjects with known IgA deficiency and anti-IgA antibodies
- Blood donation received within 3 weeks
- Platelet counts < 150 × 10ˆ9/L or > 450 × 10ˆ9/L
- Any type of known platelet function disorder
- Treatment with non-steroidal anti-inflammatory drugs (NSAIDs, e.g. acetylsalicylic acid) or selective serotonin reuptake inhibitors within 7 days prior to Visit 1
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- Chronic or ongoing active infectious disease requiring systemic treatment including, but not limited to, chronic and renal infection, chronic chest infection with bronchiectasis, and tuberculosis
- Participation in any other interventional clinical trial during the trial period
- Subjects known or suspected of not being able to comply with this trial protocol (e.g. due to alcoholism, drug dependency or psychological disorder)
- Presence of HLA-antibodies class I (MFI level > 3000)
- Signs of previous or ongoing infection with HIV and/or Hepatitis B and/or C virus
Trial locations and contacts
1 site in 1 country.
Germany1 site
Fraunhofer Institute for Molecular Biology and Applied Ecology IME
Frankfurt am Main, Hessia, 60596
About this study
Official title: An Open-label, Single Centre, Exploratory Trial Investigating the Kinetics of Platelets Transfused to Healthy, Male Subjects
The current phase 0 trial is preceding the phase 1/2 trial of a newly developed drug, NAITgam, for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT) - a rare, but potentially very severe bleeding condition in the fetus or newborn. FNAIT may occur in women whose blood platelets do not express HPA-1a. If the fetus has inherited HPA-1a from the father, the mother's immune system may be stimulated to produce HPA-1a antibodies if HPA-1a positive fetal blood platelets enter the maternal circulation during delivery. In a subsequent pregnancy, such antibodies will cross the placenta and may reduce the number of HPA-1a positive blood platelets in the fetus, which in turn may result in severe bleeding in the fetus or newborn.
The phase 1/2 study of NAITgam will examine NAITgam's ability to eliminate HPA-1a positive blood platelets that has been transfused to healthy male subjects, whose blood platelet do not express HPA-1a. The ability to quickly eliminate transfused HPA-1a positive platelets is considered as a surrogate endpoint for NAITgam's ability to prevent formation of antibodies against HPA-1a after delivery of an HPA-1a positive child.
Read more
The current phase 0 trial will examine the survival of blood platelets transfused to healthy male individuals without subsequent administration of NAITgam. The natural survival of transfused platelet, as determined in the phase 0 trial, will be compared with the survival of transfused HPA-1a positive platelets after administration of NAITgam in the phase 1/2 trial. The aim of the phase 0 trial is first, to determine the dose of blood platelet that should be transfused to the healthy subjects in the phase 1/2 trial; and secondly, to determine the optimal time point, after transfusion of platelets, for administration of NAITgam in the phase 1/2 trial.
Eight to 24 healthy male subjects will be included in the phase 0 trial. After transfusion of platelets, blood samples will be collected at regular intervals to determine the proportion of transfused blood platelets. Differences between tissue type antigens between donor and recipient will be used to determine the proportion of transfused platelets. Survival of transfused platelets will be performed by flow cytometry - a method that can be used to quantify very small proportions of cells in the blood. Fluorochrome-conjugated monoclonal antibodies against HLA-A2 and HLA-A9 will be used for flow cytometric identification the transfused platelets.
- Being tested
- Platelet transfusion
- Medical subject headings
- Thrombocytopenia, Neonatal Alloimmune
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Platelet transfusionOther
Transfusion of a platelet dose from 20 × 10ˆ9 to 100 × 10ˆ9.
| Study group | Role | What they receive |
|---|---|---|
| Platelet transfusion | Other | HLA-A2 and/or HLA A9 negative healthy study subjects will be transfused with a small dose of platelets from an HLA-A2 and/or HLA-A9 positive donor. Receives: Other: Platelet transfusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Terminal elimination half live
Determination of the terminal elimination half live of a single platelet dose transfused to healthy male subjects
Measured: The terminal elimination half live of transfused platelets will be determined based on the survival of platelets within the first 5 days after trandfusion
Secondary outcomes (3)
Cmax
The peak platelet concentration
Measured: Will be determined within the first 5 days after transfusion
AUC
The area under the platelet concentration versus time curve
Measured: Will be determined within the first 5 days after transfusion
Clearance
Natural clearance of platelets from the circulation
Measured: Will be determined within the first 5 days after transfusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jun 2018
- Study start17 Apr 2018
- Primary completion10 Dec 2018
- Study completion10 Dec 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fetal and Neonatal Alloimmune ThrombocytopeniaStudies 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 N/A
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Prophylix Pharma AS
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Larix A/S, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Deutsches Rotes Kreuz DRK-Blutspendedienst Baden-Wurttemberg-Hessen, Bioscientia Central Laboratory
- Responsible party
- Sponsor
- Investigators
- Jens Kjeldsen-Kragh, MD, PhD, Study chair, Prophylix Pharma ASMichaela Köhm, MD, Principal investigator, Fraunhofer Institute for Molecular Biology and Applied Ecology IME
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03561909
- Sponsor's protocol ID
- PX-0
- Status last verified
- May 2019
- First submitted
- 4 Jun 2018
- Last updated
- 10 May 2019
- 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 10 May 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Single Centre, Exploratory Trial Investigating the Kinetics of Platelets Transfused to Healthy, Male Subjects
- ClinicalTrials.gov ID
- NCT03561909
- Acronym / sponsor ID
- PX-0
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Prophylix Pharma ASIndustry (pharma, biotech, devices)
- Collaborators
- Larix A/S, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Deutsches Rotes Kreuz DRK-Blutspendedienst Baden-Wurttemberg-Hessen, Bioscientia Central Laboratory
- Study start
- 17 Apr 2018 (actual)
- Primary completion
- 10 Dec 2018 (actual)
- Study completion
- 10 Dec 2018 (actual)
- First posted
- 19 Jun 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 current phase 0 trial is preceding the phase 1/2 trial of a newly developed drug, NAITgam, for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT) - a rare, but potentially very severe bleeding condition in the fetus or newborn. FNAIT may occur in women whose blood platelets do not express HPA-1a. If the fetus has inherited HPA-1a from the father, the mother's immune system may be stimulated to produce HPA-1a antibodies if HPA-1a positive fetal blood platelets enter the maternal circulation during delivery. In a subsequent pregnancy, such antibodies will cross the placenta and may reduce the number of HPA-1a positive blood platelets in the fetus, which in turn may result in severe bleeding in the fetus or newborn.
The phase 1/2 study of NAITgam will examine NAITgam's ability to eliminate HPA-1a positive blood platelets that has been transfused to healthy male subjects, whose blood platelet do not express HPA-1a. The ability to quickly eliminate transfused HPA-1a positive platelets is considered as a surrogate endpoint for NAITgam's ability to prevent formation of antibodies against HPA-1a after delivery of an HPA-1a positive child.
The current phase 0 trial will examine the survival of blood platelets transfused to healthy male individuals without subsequent administration of NAITgam. The natural survival of transfused platelet, as determined in the phase 0 trial, will be compared with the survival of transfused HPA-1a positive platelets after administration of NAITgam in the phase 1/2 trial. The aim of the phase 0 trial is first, to determine the dose of blood platelet that should be transfused to the healthy subjects in the phase 1/2 trial; and secondly, to determine the optimal time point, after transfusion of platelets, for administration of NAITgam in the phase 1/2 trial.
Eight to 24 healthy male subjects will be included in the phase 0 trial. After transfusion of platelets, blood samples will be collected at regular intervals to determine the proportion of transfused blood platelets. Differences between tissue type antigens between donor and recipient will be used to determine the proportion of transfused platelets. Survival of transfused platelets will be performed by flow cytometry - a method that can be used to quantify very small proportions of cells in the blood. Fluorochrome-conjugated monoclonal antibodies against HLA-A2 and HLA-A9 will be used for flow cytometric identification the transfused platelets.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Platelet transfusion | Other | Other: Platelet transfusionHLA-A2 and/or HLA A9 negative healthy study subjects will be transfused with a small dose of platelets from an HLA-A2 and/or HLA-A9 positive donor. |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- Written informed consent must be obtained before any trial related procedures are performed
- Healthy, male subjects
- Age ≥18 and < 50 years old
- BMI < 30kg/mˆ2
- HLA-A2 and/or HLA-A9 negative
Key exclusion criteria (11 listed)
- History of hypersensitivity to platelet concentrates or human plasma protein
- Subjects with known IgA deficiency and anti-IgA antibodies
- Blood donation received within 3 weeks
- Platelet counts < 150 × 10ˆ9/L or > 450 × 10ˆ9/L
- Any type of known platelet function disorder
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 |
|---|---|---|
| Platelet transfusionTransfusion of a platelet dose from 20 × 10ˆ9 to 100 × 10ˆ9. | Other | Platelet transfusion |
Primary outcome
Terminal elimination half live
Time frame: The terminal elimination half live of transfused platelets will be determined based on the survival of platelets within the first 5 days after trandfusion
Determination of the terminal elimination half live of a single platelet dose transfused to healthy male subjects
3 secondary outcomes
Cmax
Time frame: Will be determined within the first 5 days after transfusion
AUC
Time frame: Will be determined within the first 5 days after transfusion
Clearance
Time frame: Will be determined within the first 5 days after transfusion
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 May 2019.
- Individual participant dataSharing planned: No
Related trials for Fetal and Neonatal Alloimmune Thrombocytopenia
RecruitingPhase 3
A Study of Nipocalimab or Intravenous Immunoglobulin (IVIG) in Pregnancies At Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)
Thrombocytopenia, Neonatal Alloimmune
RecruitingPhase 3
A Study of Nipocalimab in Reducing the Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)
Thrombocytopenia, Neonatal Alloimmune
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.
Jens Kjeldsen-Kragh, MD, PhD study chair
Prophylix Pharma AS
Michaela Köhm, MD principal investigator
Fraunhofer Institute for Molecular Biology and Applied Ecology IME
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Platelet transfusion in people with Fetal and Neonatal Alloimmune Thrombocytopenia, run by Prophylix Pharma AS at 1 site in Germany.
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 5 inclusion and 11 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: finished, as of the sponsor's last update on 10 May 2019. 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 Germany. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Terminal elimination half live (time frame: The terminal elimination half live of transfused platelets will be determined based on the survival of platelets within the first 5 days after trandfusion). 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 10 May 2019.