Clinical trial • Phase III • Immunology|Rare Disease
NIPOCALIMAB for Fetal and neonatal alloimmune thrombocytopenia (FNAIT)
Phase III trial of NIPOCALIMAB for Fetal and neonatal alloimmune thrombocytopenia (FNAIT).
Overview
- Trial Therapeutic Area
- Immunology|Rare Disease
- Trial Disease
- Fetal and neonatal alloimmune thrombocytopenia (FNAIT)
- Trial Stage
- Phase III
- Drug Modality
- Monoclonal antibody
Key dates
- Initial CTIS Submission Date
- 15-09-2023
- First CTIS Authorization Date
- 23-01-2024
Trial design
Randomised, saline, 0.9% sodium chloride solution for injection (placebo) — route: injection/infusion; dose and schedule not specified in the provided record.-controlled Phase III trial in Italy, Sweden, Norway and others.
- Randomised
- Yes
- Comparator
- Saline, 0.9% Sodium Chloride Solution for Injection (placebo) — route: injection/infusion; dose and schedule not specified in the provided record.
- Target Sample Size
- 18
Eligibility
Recruits 18 Pregnant participants are designated as a vulnerable population (isVulnerablePopulationSelected = true). Informed consent is obtained from the maternal participant (maternal participant ICFs present). Parent/guardian consent documents are available for infant/child-related components (multiple "Parent/Guardian" ICFs listed). Optional genetic research consent forms are present. Consent materials and information sheets are available in multiple country-specific language versions as indicated in the document list..
- Pregnancy Exclusion
- Currently pregnant with multiple gestations (twins or more)
- Vulnerable Population
- Pregnant participants are designated as a vulnerable population (isVulnerablePopulationSelected = true). Informed consent is obtained from the maternal participant (maternal participant ICFs present). Parent/guardian consent documents are available for infant/child-related components (multiple "Parent/Guardian" ICFs listed). Optional genetic research consent forms are present. Consent materials and information sheets are available in multiple country-specific language versions as indicated in the document list.
Inclusion criteria
- {"criterion_text":"- Aged 18 (or the legal age of consent if above 18 years of agein local region) to 45 years at the time of informed consent.\n- Pregnant and an estimated GA (based on ultrasound dating) from Week 13^0/7 to 18^6/7 at randomization.\n- Has a history of ≥1 prior pregnancy with FNAIT (including neonatal platelet count <150×109/L), with none of them affected by fetal/neonatal ICH or severe hemorrhage based on medical records\n- Current pregnancy with presence of maternal anti-HPA-1a alloantibody and positive fetal HPA-1a 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\n- Known allergies, hypersensitivity, or intolerance to nipocalimab or its excipients (refer to the IB)"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Adverse outcome of death or adjudicated severe bleeding in utero up to the first week post birth, or platelet count at birth <30×10⁹/l in a fetus/neonate.","definition_or_measurement_approach":"Adjudicated assessments of death or severe bleeding in utero up to 1 week after birth; measurement of platelet count at birth (<30 × 10⁹/L) in fetus/neonate."}
Secondary endpoints
- {"endpoint_text":"- Platelet count at birth in a neonate","definition_or_measurement_approach":"Laboratory measurement of neonatal platelet count at birth."}
- {"endpoint_text":"- Adjudicated bleeding in utero up to the first week post birth in a fetus/neonate","definition_or_measurement_approach":"Adjudicated assessment of in utero bleeding events up to 1 week post birth."}
Recruitment
- Registry Or Advocacy Recruitment
- True, ClinLife Registry (referenced in recruitment material titles for Hungary as "EC-Notes_ClinLife_Registry_HU_HUN").
- Digital Remote Recruitment
- True, digital methods include Digital Outreach Posts and Social Media Toolkits; digital recruitment materials are provided country-specifically (document titles indicate multiple countries).
- Planned Sample Size
- 18
- Recruitment Window Months
- 70
- Consent Approach
- Informed consent is obtained from the maternal participant via Maternal Participant ICFs. Parent/guardian ICFs are available for infant/child-related components and for optional genetic research. Multiple country- and language-specific ICFs and subject information sheets are provided (documents listed for EN, SE/SWE, BE Eng/Dut/Fre, HU, NO, SK, ITA, FR, ES, SI, etc.), indicating localized consent materials.
Methods
- Digital outreach posts / Social media toolkit (digital channels) — country-specific digital outreach materials listed (e.g., Digital Outreach Post documents for HU, NO, SK, etc.) aimed at raising awareness among potential participants and the public.
- Patient-facing materials: Patient Fact Sheet, Placebo Response Sheet, Participant Study Guide — materials available per country to inform pregnant participants.
- Clinical Trial Education Sheets and site-level recruitment arrangements — materials for healthcare providers and local site recruitment processes.
- Site-based recruitment arrangements (site contact, wallets cards, local translations) — country-specific recruitment arrangement documents present (e.g., K1 recruitment arrangements files for BE, SE, SK, NO, ES, FR, IT, HU, SI).
Geography
- Total Number Of Sites
- 16
- Total Number Of Participants
- 26
Italy
- Earliest CTIS Part Ii Submission Date
- 30-10-2023
- Latest Decision Or Authorization Date
- 20-03-2026
- Processing Time Days
- 872
- Number Of Sites
- 2
- Number Of Participants
- 6
Sites
- Site Name
- Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
- Department Name
- OUC Ostetricia
- Contact Person Name
- Irene Cetin
- Contact Person Email
- irene.cetin@policlinico.mi.it
- Site Name
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Department Name
- Dip scienze salute donna, bambino e di sanità pubblica - UOC Ostetricia e Patologia Ostetrica
- Contact Person Name
- Marco De Santis
- Contact Person Email
- Marco.DeSantis@unicatt.it
Sweden
- Earliest CTIS Part Ii Submission Date
- 07-12-2023
- Latest Decision Or Authorization Date
- 20-03-2026
- Processing Time Days
- 834
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Karolinska University Hospital
- Department Name
- Center for fetal medicine, K66
- Contact Person Name
- Gunilla Ajne
- Contact Person Email
- gunilla.ajne@regionstockholm.se
Norway
- Earliest CTIS Part Ii Submission Date
- 11-12-2023
- Latest Decision Or Authorization Date
- 19-03-2026
- Processing Time Days
- 829
- Number Of Sites
- 4
- Number Of Participants
- 7
Sites
- Site Name
- St. Olavs Hospital HF
- Department Name
- Fetal medicine
- Contact Person Name
- Kjell Å. B. Salvesen
- Contact Person Email
- Kjell.asmund.salvesen@stolav.no
- Site Name
- Oslo University Hospital HF
- Department Name
- Leader Fetal Medicine Department
- Contact Person Name
- Sitras Vasilis
- Contact Person Email
- vsit@ous-hf.no
- Site Name
- Helse Bergen HF
- Department Name
- Obstetrics and Gynecology department
- Contact Person Name
- Cathrine Ebbing
- Contact Person Email
- Cathrine.Ebbing@helse-bergen.no
- Site Name
- Universitetssykehuset Nord-Norge HF
- Department Name
- Obstetrics and gynecology/ Clinical medicine
- Contact Person Name
- Heidi Tiller
- Contact Person Email
- heidi.tiller@unn.no
Belgium
- Earliest CTIS Part Ii Submission Date
- 19-12-2023
- Latest Decision Or Authorization Date
- 13-04-2026
- Processing Time Days
- 846
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- UZ Leuven
- Department Name
- Maternal-Fetal medicine
- Contact Person Name
- Roland Devlieger
- Contact Person Email
- roland.devlieger@uzleuven.be
Slovakia
- Earliest CTIS Part Ii Submission Date
- 14-11-2023
- Latest Decision Or Authorization Date
- 20-04-2026
- Processing Time Days
- 888
- Number Of Sites
- 3
- Number Of Participants
- 3
Sites
- Site Name
- Fakultna Nemocnica S Poliklinikou Nove Zamky
- Department Name
- Obstetrical and Gynecological Clinic
- Contact Person Name
- Oleksander Grygoryk
- Contact Person Email
- oleksander.grygoryk@nspnz.sk
- Site Name
- Univerzitna nemocnica L. Pasteura Kosice
- Department Name
- Gynecology and obstetrics
- Contact Person Name
- Peter Urdzik
- Contact Person Email
- Peter.urdzik@upjs.sk
- Site Name
- Univerzitna Nemocnica Martin
- Department Name
- Gynecology and Obstetrics Clinic
- Contact Person Name
- Kamil Biringer
- Contact Person Email
- kamil.biringer@uniba.sk
Spain
- Earliest CTIS Part Ii Submission Date
- 14-11-2023
- Latest Decision Or Authorization Date
- 23-03-2026
- Processing Time Days
- 860
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- University Hospital Virgen Del Rocio S.L.
- Department Name
- Hematology
- Contact Person Name
- Maria Eva Mingot Castellano
- Contact Person Email
- mariae.mingot.sspa@juntadeandalucia.es
Hungary
- Earliest CTIS Part Ii Submission Date
- 10-06-2024
- Latest Decision Or Authorization Date
- 19-03-2026
- Processing Time Days
- 648
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Semmelweis University
- Department Name
- Szülészeti és Nőgyógyászati Klinika
- Contact Person Name
- Nándor Ács
- Contact Person Email
- acsnandor@gmail.com
France
- Earliest CTIS Part Ii Submission Date
- 30-01-2024
- Latest Decision Or Authorization Date
- 19-03-2026
- Processing Time Days
- 780
- Number Of Sites
- 2
- Number Of Participants
- 2
Sites
- Site Name
- Centre Hospitalier Universitaire De Lille
- Department Name
- Gynecology-Obstetrics Department
- Contact Person Name
- Veronique Houfflin-Debarge
- Contact Person Email
- Veronique.DEBARGE@chu-lille.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Fetal Medecine Department
- Contact Person Name
- Jean-Marie Jouannic
- Contact Person Email
- Jean-marie.jouannic@aphp.fr
Slovenia
- Earliest CTIS Part Ii Submission Date
- 30-01-2024
- Latest Decision Or Authorization Date
- 17-03-2026
- Processing Time Days
- 777
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- University Medical Center Ljubljana
- Department Name
- Porodnišnica Ljubljana
- Contact Person Name
- Vesna Fabjan Vodusek
- Contact Person Email
- Vesna.fabjan@mf.uni-lj.si
Sponsor
Primary sponsor
- Full Name
- Janssen - Cilag International
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Belgium
Contract research organisations
- Name
- Cytel Inc.
- Name
- 4g Clinical LLC
- Name
- Iqvia Rds Inc.
- Responsibilities
- Support/coordinate investigator/site contract negotiations; study drug materials; regulatory and DPO submissions in Slovenia and Ireland
- Name
- Icon Development Solutions LLC
- Name
- Parexel International (IRL) Limited
- Name
- Imperial Clinical Research Services International Limited
Third parties
- {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"reimbursement","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Cytel Inc.","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"4g Clinical LLC","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Iqvia Rds Inc.","duties_or_roles":"IQVIA is tasked to support/coordinate activities concerning investigator/site related contract negotiations; study drug materials; regulatory and DPO submissions in Slovenia and Ireland","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"Netherlands","full_name":"Sanquin Diagnostiek B.V.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Imperial Clinical Research Services International Limited","duties_or_roles":"","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"United States","full_name":"Icon Development Solutions LLC","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- JNJ-80202135
- Active Substance
- NIPOCALIMAB
- Modality
- Monoclonal antibody
- Routes Of Administration
- Intravenous use
- Route
- Intravenous
- Authorisation Status
- 1
- Investigational Product Name
- Saline, 0.9% Sodium Chloride Solution for Injection
- Modality
- Other
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