Clinical trial • Phase III • Immunology|Rare Disease

NIPOCALIMAB for Hemolytic disease of the fetus and newborn (HDFN)

Phase III trial of NIPOCALIMAB for Hemolytic disease of the fetus and newborn (HDFN).

Overview

Trial Therapeutic Area
Immunology|Rare Disease
Trial Disease
Hemolytic disease of the fetus and newborn (HDFN)
Trial Stage
Phase III
Drug Modality
Monoclonal antibody
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
11-09-2023
First CTIS Authorization Date
18-01-2024

Trial design

Randomised, saline, 0.9% sodium chloride solution for injection (placebo) administered as placebo comparator; product listed as 'saline, 0.9% sodium chloride solution for injection' (no dose/schedule specified in ctis record).-controlled Phase III trial in Austria, Spain, Sweden and others.

Randomised
Yes
Comparator
Saline, 0.9% Sodium Chloride Solution for Injection (placebo) administered as placebo comparator; product listed as 'Saline, 0.9% Sodium Chloride Solution for Injection' (no dose/schedule specified in CTIS record).
Target Sample Size
92

Eligibility

Recruits 92 Vulnerable population flag is selected. The trial includes pregnant participants; informed consent materials and procedures are provided specifically for maternal participants (SIS and ICF Main Maternal / Maternal Participant). Subject information and informed consent forms for Parent/Guardian are available (REDACTED_L1_SIS and ICF Parent Guardian / Parent Legal Guardian) and eConsent materials are provided. Multiple language translations and parent/guardian consent documents are included in the submission packages, indicating provision for vulnerable-subject consent processes and parent/legal-guardian documentation where applicable..

Pregnancy Exclusion
Currently pregnant with a multiple gestation (twins or more).
Vulnerable Population
Vulnerable population flag is selected. The trial includes pregnant participants; informed consent materials and procedures are provided specifically for maternal participants (SIS and ICF Main Maternal / Maternal Participant). Subject information and informed consent forms for Parent/Guardian are available (REDACTED_L1_SIS and ICF Parent Guardian / Parent Legal Guardian) and eConsent materials are provided. Multiple language translations and parent/guardian consent documents are included in the submission packages, indicating provision for vulnerable-subject consent processes and parent/legal-guardian documentation where applicable.

Inclusion criteria

  • {"criterion_text":"-Female, 18 (or the legal age of consent if above 18 in local regions) to 45 years of age, inclusive, at the time of informed consent."}
  • {"criterion_text":"-Pregnant and an estimated GA (based on ultrasound dating) from Week 13^0/7 and Week 18^6/7 at randomization."}
  • {"criterion_text":"-History of severe HDFN in a prior pregnancy, defined as documented: a. fetal anemia* s result of HDFN* or fetal hydrops** as a result of HDFN, or received ≥1 IUT as a result of HDFN. *defined as fetal hemoglobin level <0.84 MoM (Section 8.2.1.2). Fetal hemoglobin (g/dL) can be calculated using hematocrit (%)/3. **defined as the presence of ≥2 abnormal fluid collections in the fetus as ascites, plural effusions, pericardial effusion, and generalized skin edema (skin thickness >5 mm. OR b. Fetal loss or neonatal death as a result of HDFN, with maternal alloantibody titers for RhD, Kell, Rhc, RhE, or RhC antigen above the critical levels (anti-Kell ≥4; other ≥16) and evidence of an antigen-positive fetus (When these tests have not been performed as part of regional/local clinical practice, alternative confirmation of HDFN, after consultation with the sponsor, may be considered)."}
  • {"criterion_text":"-During the current pregnancy, presence of maternal alloantibody to RhD, Kell, Rhc, RhE, or RhC antigen with titers above the critical level (anti-Kell ≥4; other ≥16) based on the designated central lab results at screening."}
  • {"criterion_text":"-Evidence of antigen-positivity corresponding to the current maternal alloantibody (RhD, Kell, Rhc, RhE, or RhC) confirmed by non-invasive antigen cffDNA performed at the central laboratory."}

Exclusion criteria

  • {"criterion_text":"-Currently pregnant with a multiple gestation (twins or more)."}
  • {"criterion_text":"-2.1 Criterion amended per Amendment 6.Evidence of fetal anemia by ultrasound or repeated MCA-PSV ≥1.5 MoM* prior to randomization in the current pregnancy. *In the absence of other evidence of fetal anemia on ultrasound, inability to measure MCA-PSV before GA 14 weeks 0 days, secondary to concerns of safety/accuracy, will not be considered an exclusion."}
  • {"criterion_text":"-3.2 Criterion amended per Amendment 6. History of severe preeclampsia prior to GA Week 34 or severe FGR (EFW<3rd percentile, based on local fetal growth normative standards) in a previous pregnancy. Note: for countries where the percentiles are not used in clinical practice, local country measures (standard deviations) equivalent to <3rd percentile may be considered after consultation with the sponsor."}
  • {"criterion_text":"-Current uncontrolled hypertension."}
  • {"criterion_text":"-History of myocardial infarction, unstable ischemic heart disease, or stroke."}

Endpoints

Primary endpoints

  • {"endpoint_text":"-The proportion of pregnancies that did not result in fetal loss (due to any reason), IUT, hydrops fetalis, or neonatal death (due to any reason) during the neonatal period (through 4 weeks of age or 41 weeks PMA, whichever is later).","definition_or_measurement_approach":"Neonatal period defined as through 4 weeks of age or 41 weeks postmenstrual age (PMA), whichever is later; endpoint is the proportion of pregnancies without fetal loss, intrauterine transfusion (IUT), hydrops fetalis, or neonatal death during that neonatal period."}

Recruitment

Digital Remote Recruitment
Yes
Planned Sample Size
92
Recruitment Window Months
65
Consent Approach
Informed consent is provided by the maternal participant (Maternal Participant ICF / SIS and ICF Main Maternal). eConsent is available (eConsent materials and screenshots referenced). Parent/legal guardian ICFs and SIS for Parent/Guardian are provided where applicable. Consent and participant materials are provided in multiple languages (documentation includes English, Spanish, French, Dutch, German, Arabic, Romanian, Tigrinya, Polish, Italian, Swedish and others as per country-specific submission packages).

Methods

  • Use of patient-facing recruitment materials including Participant Recruitment Brochures and Clinical Trial Patient Education Booklets (K2_Clinical Trial Patient Education Booklet) distributed at sites.
  • Digital/web advertising: Participant Recruitment Web Ads for multiple countries (web ads referenced in K2 materials).
  • Print advertising and posters: Participant Recruitment Print Ads and Professional Recruitment Posters (K2_Participant Recruitment Print Ad; K2_Professional Recruitment Poster).
  • Social media advertisements and site social toolkits (documents labelled Social Media Advertisement / Social_ToolKit).
  • Patient database letters and telephone screens to contact potential participants (Patient Database Letter, Telephone Screen materials).
  • Colleague referral and professional outreach (Colleague Referral Letter, Professional Recruitment Brochure).
  • Site-based recruitment via hospital/clinic referral and local study staff.
  • Use of eConsent and electronic participant-facing materials (eConsent submission materials and eConsent screenshots) to facilitate remote consent.

Geography

Total Number Of Sites
22
Total Number Of Participants
31

Austria

Earliest CTIS Part Ii Submission Date
05-04-2024
Latest Decision Or Authorization Date
06-05-2024
Processing Time Days
31
Number Of Sites
2
Number Of Participants
2

Sites

Site Name
Medical University of Graz, Division of Obstetrics, Neue Stiftingtalstraße 6
Department Name
Department of Obstetrics and Gynecology
Principal Investigator Name
Philip Klaritsch
Principal Investigator Email
philipp.klaritsch@medunigraz.at
Contact Person Name
Philip Klaritsch
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

Spain

Earliest CTIS Part Ii Submission Date
31-10-2023
Latest Decision Or Authorization Date
22-01-2024
Processing Time Days
83
Number Of Sites
3
Number Of Participants
6

Sites

Site Name
Hospital Universitario La Paz
Department Name
gynecology
Principal Investigator Name
Jose Luis Bartha Rasero
Principal Investigator Email
josel.bartha@salud.madrid.org
Contact Person Name
Jose Luis Bartha Rasero
Contact Person Email
josel.bartha@salud.madrid.org
Site Name
University Hospital Virgen Del Rocio S.L.
Department Name
gynecology
Principal Investigator Name
Lucas Ángel Cerrillos Gonzalez
Principal Investigator Email
lucas.cerrillos.sspa@juntadeandalucia.es
Contact Person Name
Lucas Ángel Cerrillos Gonzalez
Site Name
Hospital Universitari Vall D Hebron
Department Name
gynecology
Principal Investigator Name
Maria Ángeles Sánchez Durán
Principal Investigator Email
angeles.sanchezduran@vallhebron.cat
Contact Person Name
Maria Ángeles Sánchez Durán

Sweden

Earliest CTIS Part Ii Submission Date
05-12-2023
Latest Decision Or Authorization Date
19-01-2024
Processing Time Days
45
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
Karolinska University Hospital
Department Name
Center for Fetal Medicine
Principal Investigator Name
Gunilla Ajne
Principal Investigator Email
gunilla.ajne@regionstockholm.se
Contact Person Name
Gunilla Ajne

Italy

Earliest CTIS Part Ii Submission Date
24-10-2023
Latest Decision Or Authorization Date
18-01-2024
Processing Time Days
86
Number Of Sites
2
Number Of Participants
2

Sites

Site Name
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Department Name
Dipartimento di Scienze della Salute della Donna, del Bambino e di Sanità Pubblica
Principal Investigator Name
Marco De Santis
Principal Investigator Email
marco.desantis@unicatt.it
Contact Person Name
Marco De Santis
Contact Person Email
marco.desantis@unicatt.it
Site Name
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Department Name
UOC Ostetricia
Principal Investigator Name
Irene Cetin
Principal Investigator Email
irene.cetin@policlinico.mi.it
Contact Person Name
Irene Cetin
Contact Person Email
irene.cetin@policlinico.mi.it

Germany

Earliest CTIS Part Ii Submission Date
14-12-2023
Latest Decision Or Authorization Date
18-01-2024
Processing Time Days
35
Number Of Sites
3
Number Of Participants
2

Sites

Site Name
University Medical Center Hamburg-Eppendorf
Department Name
Department of Obstetrics and Fetal Medicine
Principal Investigator Name
Stefan Verlohren
Principal Investigator Email
s.verlohren@uke.de
Contact Person Name
Stefan Verlohren
Contact Person Email
s.verlohren@uke.de
Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Department of Obstetrics
Principal Investigator Name
Iris Dressler-Steinbach
Principal Investigator Email
iris.dressler-steinbach@charite.de
Contact Person Name
Iris Dressler-Steinbach
Site Name
Justus-Liebig-Universitaet Giessen
Department Name
Universitätsklinikum Gießen und Marburg GmbH, Sektion Hämostaseologie
Principal Investigator Name
Ulrich Sachs
Principal Investigator Email
Ulrich.sachs@med.uni-giessen.de
Contact Person Name
Ulrich Sachs

Belgium

Earliest CTIS Part Ii Submission Date
04-12-2023
Latest Decision Or Authorization Date
18-01-2024
Processing Time Days
45
Number Of Sites
2
Number Of Participants
4

Sites

Site Name
Association Hospitaliere De Bruxelles Et De Schaerbeek Centre Hospitalier Universitaire Brugmann
Department Name
Department of Gynecology and Obstetrics
Principal Investigator Name
Jacques Jani
Principal Investigator Email
jacques.jani@chu-brugmann.be
Contact Person Name
Jacques Jani
Contact Person Email
jacques.jani@chu-brugmann.be
Site Name
UZ Leuven
Department Name
Department of Obstetrics and Gynaecology
Principal Investigator Name
Roland Devlieger
Principal Investigator Email
roland.devlieger@uzleuven.be
Contact Person Name
Roland Devlieger
Contact Person Email
roland.devlieger@uzleuven.be

Netherlands

Earliest CTIS Part Ii Submission Date
20-12-2023
Latest Decision Or Authorization Date
22-01-2024
Processing Time Days
33
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
Academisch Ziekenhuis Leiden
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

Ireland

Earliest CTIS Part Ii Submission Date
05-04-2024
Latest Decision Or Authorization Date
03-05-2024
Processing Time Days
28
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
Rotunda Hospital
Department Name
Rotunda Private Clinics
Principal Investigator Name
Fergal Malone
Principal Investigator Email
fmalone@rcsi.ie
Contact Person Name
Fergal Malone
Contact Person Email
fmalone@rcsi.ie

France

Earliest CTIS Part Ii Submission Date
28-09-2023
Latest Decision Or Authorization Date
19-01-2024
Processing Time Days
113
Number Of Sites
3
Number Of Participants
4

Sites

Site Name
Assistance Publique Hopitaux De Paris
Department Name
Fetal Medecine Department
Principal Investigator Name
Jean-Marie Jouannic
Principal Investigator Email
Jean-marie.jouannic@aphp.fr
Contact Person Name
Jean-Marie Jouannic
Contact Person Email
Jean-marie.jouannic@aphp.fr
Site Name
Hospices Civils De Lyon
Department Name
Department of obstetrics and fetal medicine
Principal Investigator Name
Anthony ATALLAH
Principal Investigator Email
anthony.atallah@chu-lyon.fr
Contact Person Name
Anthony ATALLAH
Contact Person Email
anthony.atallah@chu-lyon.fr
Site Name
Centre Hospitalier Universitaire De Lille
Department Name
Gynecology-Obstetrics Department
Principal Investigator Name
Veronique Houfflin-Debarge
Principal Investigator Email
Veronique.DEBARGE@chu-lille.fr
Contact Person Name
Veronique Houfflin-Debarge
Contact Person Email
Veronique.DEBARGE@chu-lille.fr

Poland

Earliest CTIS Part Ii Submission Date
02-04-2026
Latest Decision Or Authorization Date
23-04-2026
Processing Time Days
21
Number Of Sites
3
Number Of Participants
4

Sites

Site Name
Instytut Centrum Zdrowia Matki Polki
Department Name
Klinika Perinatologii Poloznictwa i Ginekologii
Principal Investigator Name
Grzesiak Mariusz
Principal Investigator Email
mariusz.grzesiak@iczmp.edu.pl
Contact Person Name
Grzesiak Mariusz
Contact Person Email
mariusz.grzesiak@iczmp.edu.pl
Site Name
Panstwowy Instytut Medyczny Ministerstwa Spraw Wewnetrznych I Administracji
Department Name
Klinika Poloznictwa i Perinatologii
Principal Investigator Name
Mirosław Wielgos
Principal Investigator Email
miroslaw.wielgos@cskmswia.gov.pl
Contact Person Name
Mirosław Wielgos
Site Name
Instytut Matki I Dziecka
Department Name
Klinika Poloznictwa i Ginekologii
Principal Investigator Name
Tadeusz Issat
Principal Investigator Email
tadeusz.issat@imid.med.pl
Contact Person Name
Tadeusz Issat
Contact Person Email
tadeusz.issat@imid.med.pl

Czechia

Earliest CTIS Part Ii Submission Date
26-01-2026
Latest Decision Or Authorization Date
20-03-2026
Processing Time Days
53
Number Of Sites
1
Number Of Participants
1

Sites

Site Name
Vseobecna Fakultni Nemocnice V Praze
Department Name
Klinika gynekologie, porodnictvi a neonatologie 1. LF a VFN
Principal Investigator Name
Pavel Calda
Principal Investigator Email
pavel.calda@vfn.cz
Contact Person Name
Pavel Calda
Contact Person Email
pavel.calda@vfn.cz

Sponsor

Primary sponsor

Full Name
Janssen - Cilag International
Organisation Type
Pharmaceutical company
Country Of Registered Address
Belgium

Contract research organisations

Name
IQVIA Limited
Responsibilities
eConsent
Name
Iqvia Rds Inc.
Responsibilities
NCC Management
Name
Pharmaceutical Research Associates Group B.V.
Responsibilities
Operational/site services (sponsor duty code 4)
Name
4g Clinical LLC
Responsibilities
Site services / study support (sponsor duty code 3)

Third parties

  • {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"eConsent","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Patient transportation and subject compensation service","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"PROs/ClinROs","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Qd Solutions Inc.","duties_or_roles":"IPE Materials","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Marken LLP","duties_or_roles":"HHS","organisation_type":"Pharmaceutical company"}
  • {"country":"Netherlands","full_name":"Sanquin Diagnostiek B.V.","duties_or_roles":"Laboratory services (sponsor duty code 4)","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Fortrea Inc.","duties_or_roles":"Reviewer","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Iqvia Rds Inc.","duties_or_roles":"NCC Management","organisation_type":"Pharmaceutical company"}
  • {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services S.a.r.l.","duties_or_roles":"Laboratory services (sponsor duty code 4)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"Data capture / eCOA (sponsor duty code 7)","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"Netherlands","full_name":"Pharmaceutical Research Associates Group B.V.","duties_or_roles":"Laboratory/services (sponsor duty code 4)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Ancillare LP","duties_or_roles":"Study supplies","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Grifols Laboratory Solutions Inc.","duties_or_roles":"Laboratory services (sponsor duty code 4)","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"4g Clinical LLC","duties_or_roles":"Site management / other (sponsor duty code 3)","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Billiontoone Inc.","duties_or_roles":"Laboratory services (sponsor duty code 4)","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"Laboratory services (sponsor duty code 4)","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
JNJ-80202135 (SOLUTION FOR INFUSION)
Active Substance
NIPOCALIMAB
Modality
Monoclonal antibody
Routes Of Administration
INTRAVENOUS USE
Route
Intravenous
Orphan Designation
Yes
Investigational Product Name
JNJ-80202135 (SOLUTION FOR INJECTION)
Active Substance
NIPOCALIMAB
Modality
Monoclonal antibody
Routes Of Administration
INTRAVENOUS USE
Route
Intravenous
Investigational Product Name
Saline, 0.9% Sodium Chloride Solution for Injection (placebo)
Modality
Other

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