Clinical trial • Phase III • Immunology|Rare Disease
NIPOCALIMAB for Fetal and neonatal alloimmune thrombocytopenia
Phase III trial of NIPOCALIMAB for Fetal and neonatal alloimmune thrombocytopenia.
Overview
- Trial Therapeutic Area
- Immunology|Rare Disease
- Trial Disease
- Fetal and neonatal alloimmune thrombocytopenia
- Trial Stage
- Phase III
- Drug Modality
- Monoclonal antibody|Peptide/protein/enzyme|Small molecule
Key dates
- Initial CTIS Submission Date
- 23-08-2024
- First CTIS Authorization Date
- 10-12-2024
Trial design
Randomised, open-label, prednisone (oral tablet; dose/schedule not specified in provided data) and human normal immunoglobulin (ivig) (intravenous; dose/schedule not specified in provided data).-controlled Phase III trial in Austria, Germany, Poland and others.
- Randomised
- Yes
- Open Label
- Yes
- Comparator
- PREDNISONE (oral tablet; dose/schedule not specified in provided data) and Human normal immunoglobulin (IVIG) (intravenous; dose/schedule not specified in provided data).
- Target Sample Size
- 36
Eligibility
Recruits 36 Pregnant women are identified as a vulnerable population (isVulnerablePopulationSelected = true). Participants must be adults (Aged 18 or legal age of consent) and provide informed consent. Parent-Guardian ICF documents are included among uploaded documents (Parent-Guardian ICF entries present for country-specific packages), and ICF/SIS documents and translation certificates are provided for multiple languages..
- Pregnancy Exclusion
- Currently pregnant with multiple gestations (twins or more).
- Vulnerable Population
- Pregnant women are identified as a vulnerable population (isVulnerablePopulationSelected = true). Participants must be adults (Aged 18 or legal age of consent) and provide informed consent. Parent-Guardian ICF documents are included among uploaded documents (Parent-Guardian ICF entries present for country-specific packages), and ICF/SIS documents and translation certificates are provided for multiple languages.
Inclusion criteria
- {"criterion_text":"- Aged 18 (or the legal age of consent if above 18 years in local region) to 45 years at the time of informed consent.\n- Pregnant and an estimated GA from Week 13^0/7 to 18^6/7 at Visit 1. (Note: Screening may start at GA Week 8).\n- Current pregnancy with presence of maternal anti-HPA-1a and/or anti-HPA-5b alloantibody and positive fetal HPA-1a and/or anti-HPA-5b genotype as confirmed by cell-free fetal DNA in maternal blood.\n- Health status considered stable by the investigator on the basis of physical examination, medical history, vital signs, 12-lead ECG, and clinical laboratory tests performed at screening."}
Exclusion criteria
- {"criterion_text":"- Currently pregnant with multiple gestations (twins or more).\n- History of severe preeclampsia in a previous pregnancy.\n- History of severe FGR (birth weight <3rd percentile for GA) in a previous pregnancy.\n- History of myocardial infarction, unstable ischemic heart disease, or stroke."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Adverse outcome of death or adjudicated severe bleeding in utero, and up to the first week post birth, or platelet count at birth <30×10^9/L in a fetus/neonate","definition_or_measurement_approach":"Occurrence of death or adjudicated severe bleeding in utero up to the first week post birth, or platelet count at birth <30×10^9/L in the fetus/neonate (platelet count threshold explicitly specified)."}
Recruitment
- Digital Remote Recruitment
- Yes
- Planned Sample Size
- 36
- Recruitment Window Months
- 59
- Consent Approach
- Informed consent is obtained from the pregnant participant (minimum age 18, or legal age of consent in local region). Multiple Subject Information Sheets and Informed Consent Forms (SIS and ICF) are included among uploaded documents (country-specific ICFs and Parent-Guardian ICFs are present). Translation certificates and multi-language ICFs are provided for different country packages (e.g. DE/ENG, PL, NL), indicating consent materials are available in multiple languages.
Methods
- Digital outreach posts / Digital Outreach Post documents (social media/digital channels) targeting potential participants; country-specific digital outreach documents present for Austria/Germany/Poland/Netherlands (document titles include 'Digital Outreach Post' and 'Digital Outreach Post_PL_DE_NL').
- Clinical Trials Brochure / Participant brochures (printed or downloadable brochures) for outreach to potential participants; country-specific brochure documents available (e.g. 'Clinical Trials Brochure', 'Brochure_PL').
- Database Letter (contacting potential participants via site or registry databases) — documents titled 'Database Letter' present in country dossier packages (AT/DE/PL/NL).
- Recruitment Fact Sheets / Participant Fact Sheet documents (informational materials for potential participants) present in country materials.
- Social Toolkit (materials for partners and community dissemination) — documents titled 'Social Toolkit' present in country materials.
- Subject Wallet Card and L2 materials provided to participants (documents titled 'Subject Wallet Card' present).
Geography
- Total Number Of Sites
- 11
- Total Number Of Participants
- 36
Austria
- Earliest CTIS Part Ii Submission Date
- 09-09-2024
- Latest Decision Or Authorization Date
- 20-04-2026
- Processing Time Days
- 587
- Number Of Sites
- 2
- Number Of Participants
- 2
Sites
- Site Name
- Medical University Of Vienna
- Department Name
- Department of Obstetrics and Gynecology Division of Obstetrics and Feto-Maternal Medicine
- Principal Investigator Name
- Alex Farr
- Principal Investigator Email
- alex.farr@meduniwien.ac.at
- Contact Person Name
- Alex Farr
- Contact Person Email
- alex.farr@meduniwien.ac.at
- Site Name
- Medical University Of Graz
- Department Name
- Clinical Department of Obstetrics
- Principal Investigator Name
- Philipp Klaritsch
- Principal Investigator Email
- philipp.klaritsch@medunigraz.at
- Contact Person Name
- Philipp Klaritsch
- Contact Person Email
- philipp.klaritsch@medunigraz.at
Germany
- Earliest CTIS Part Ii Submission Date
- 04-10-2024
- Latest Decision Or Authorization Date
- 21-04-2026
- Processing Time Days
- 564
- Number Of Sites
- 5
- Number Of Participants
- 5
Sites
- Site Name
- Universitaetsklinikum Tuebingen AöR
- Department Name
- Department für Frauengesundheit
- Principal Investigator Name
- Karl-Oliver Kagan
- Principal Investigator Email
- Karl.kagan@med.uni-tuebingen.de
- Contact Person Name
- Karl-Oliver Kagan
- Contact Person Email
- Karl.kagan@med.uni-tuebingen.de
- Site Name
- Justus-Liebig-Universitaet Giessen
- Department Name
- Sektion Haemostaseologie
- Principal Investigator Name
- Ulrich Sachs
- Principal Investigator Email
- ulrich.sachs@med.uni-giessen.de
- Contact Person Name
- Ulrich Sachs
- Contact Person Email
- ulrich.sachs@med.uni-giessen.de
- Site Name
- Charite Universitaetsmedizin Berlin KöR
- Department Name
- Campus Charité Mitte (CCM), Klinik für Geburtsmedizin
- Principal Investigator Name
- Beate Mayer
- Principal Investigator Email
- beate.mayer@charite.de
- Contact Person Name
- Beate Mayer
- Contact Person Email
- beate.mayer@charite.de
- Site Name
- Medizinische Hochschule Hannover
- Department Name
- Pränatalmedizin und Geburtshilfe im Perinatalzentrum
- Principal Investigator Name
- Constantin von Kaisenberg
- Principal Investigator Email
- vonkaisenberg.constantin@mh-hannover.de
- Contact Person Name
- Constantin von Kaisenberg
- Contact Person Email
- vonkaisenberg.constantin@mh-hannover.de
- Site Name
- Friedrich-Schiller-Universitaet Jena
- Department Name
- Klinik fur Frauenheilkunde und Geburtshilfe
- Principal Investigator Name
- Ekkehard Schleußner
- Principal Investigator Email
- ekkehard.schleussner@med.uni-jena.de
- Contact Person Name
- Ekkehard Schleußner
- Contact Person Email
- ekkehard.schleussner@med.uni-jena.de
Poland
- Earliest CTIS Part Ii Submission Date
- 11-11-2024
- Latest Decision Or Authorization Date
- 21-04-2026
- Processing Time Days
- 526
- Number Of Sites
- 3
- Number Of Participants
- 4
Sites
- Site Name
- Panstwowy Instytut Medyczny Ministerstwa Spraw Wewnetrznych I Administracji
- Department Name
- Klinika Położnictwa i Perinatologii
- Principal Investigator Name
- Marzena Dębska
- Principal Investigator Email
- poloznictwo@cskmswia.gov.pl
- Contact Person Name
- Marzena Dębska
- Contact Person Email
- poloznictwo@cskmswia.gov.pl
- Site Name
- Instytut Matki I Dziecka
- Department Name
- Klinika Położnictwa i Ginekologii
- Principal Investigator Name
- Tadeusz Issat
- Principal Investigator Email
- klinika.poloznictwa@imid.med.pl
- Contact Person Name
- Tadeusz Issat
- Contact Person Email
- klinika.poloznictwa@imid.med.pl
- Site Name
- Instytut Centrum Zdrowia Matki Polki
- Department Name
- Klinika Perinatologii Położnictwa i Ginekologii
- Principal Investigator Name
- Mariusz Grzesiak
- Principal Investigator Email
- sek66@iczmp.edu.pl
- Contact Person Name
- Mariusz Grzesiak
- Contact Person Email
- sek66@iczmp.edu.pl
Netherlands
- Earliest CTIS Part Ii Submission Date
- 18-09-2025
- Latest Decision Or Authorization Date
- 17-04-2026
- Processing Time Days
- 211
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Leids Universitair Medisch Centrum (LUMC)
- Department Name
- Department of Obstetrics and Gynaecology
- Principal Investigator Name
- Joanne (E.J.T.) Verweij
- Principal Investigator Email
- e.j.t.verweij@lumc.nl
- Contact Person Name
- Joanne (E.J.T.) Verweij
- Contact Person Email
- e.j.t.verweij@lumc.nl
Sponsor
Primary sponsor
- Full Name
- Janssen Cilag International
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Belgium
Contract research organisations
- Name
- Parexel International (IRL) Limited
- Responsibilities
- sponsorDuties codes: [6]
- Name
- Icon Development Solutions LLC
- Responsibilities
- sponsorDuties codes: [4]
- Name
- Cytel Inc.
- Responsibilities
- sponsorDuties codes: [10]
- Name
- 4g Clinical LLC
- Responsibilities
- sponsorDuties codes: [3]
- Name
- Imperial Clinical Research Services International Limited
- Responsibilities
- sponsorDuties codes: [12]
Third parties
- {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"sponsorDuties codes: [6]","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"sponsorDuties codes: [7]","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Qd Solutions Inc.","duties_or_roles":"sponsorDuties codes: [15]; value: Recruitment materials","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Cytel Inc.","duties_or_roles":"sponsorDuties codes: [10]","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"sponsorDuties codes: [4]","organisation_type":"Pharmaceutical company"}
- {"country":"Netherlands","full_name":"Sanquin Diagnostiek B.V.","duties_or_roles":"sponsorDuties codes: [4]","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United Kingdom","full_name":"Imperial Clinical Research Services International Limited","duties_or_roles":"sponsorDuties codes: [12]","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"United States","full_name":"4g Clinical LLC","duties_or_roles":"sponsorDuties codes: [3]","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"sponsorDuties codes: [15]; value: Reimbursement","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Icon Development Solutions LLC","duties_or_roles":"sponsorDuties codes: [4]","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- Imaavy 185 mg/mL concentrate for solution for infusion (nipocalimab)
- Active Substance
- NIPOCALIMAB
- Modality
- Monoclonal antibody
- Routes Of Administration
- Intravenous
- Route
- Intravenous
- Authorisation Status
- Authorised (marketing authorisation EU/1/25/1989/001 as listed for product Imaavy)
- Investigational Product Name
- PREDNISONE
- Active Substance
- PREDNISONE
- Modality
- Small molecule
- Routes Of Administration
- Oral
- Route
- Oral
- Investigational Product Name
- IMMUNOGLOBULINS, NORMAL HUMAN, FOR INTRAVASCULAR ADM. (IVIG)
- Active Substance
- HUMAN NORMAL IMMUNOGLOBULIN (IV)
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- Intravenous
- Route
- Intravenous
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