Double-blind, Randomized, Placebo-controlled Study Evaluating the Safety and Efficacy of Nipocalimab in Reducing the Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT) in At-risk Pregnancies

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Janssen - Cilag International

Participant type

Patients

Age range

18-64 years

Gender

Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

20 Feb 2024 → ongoing

Decision date (initial)

2024-01-25

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Pharmacogenomic, Pharmacodynamic, Pharmacokinetic, Safety

To evaluate the superiority of nipocalimab compared with placebo in reducing the risk of severe FNAIT

Secondary objectives 2

1. To evaluate the efficacy of nipocalimab compared with placebo on FNAIT-related thrombocytopenia
2. To evaluate the efficacy of nipocalimab compared with placebo in reducing the risk of FNAIT-related bleeding

Conditions and MedDRA coding

Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

Plan to share IPD

Yes

IPD plan description

The data sharing policy of the Janssen Pharmaceutical Companies of Johnson & Johnson is available at www.janssen.com/clinical-trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. 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.
2. Pregnant and an estimated GA (based on ultrasound dating) from Week 13^0/7 to 18^6/7 at randomization.
3. 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
4. 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
5. 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 5

1. Currently pregnant with multiple gestations (twins or more)
2. History of severe preeclampsia in a previous pregnancy
3. History of severe FGR (birth weight <3rd percentile for GA) in a previous pregnancy.
4. History of myocardial infarction, unstable ischemic heart disease, or stroke
5. Known allergies, hypersensitivity, or intolerance to nipocalimab or its excipients (refer to the IB)

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. 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.

Secondary endpoints 2

1. Platelet count at birth in a neonate
2. Adjudicated bleeding in utero up to the first week post birth in a fetus/neonate

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Janssen - Cilag International

Sponsor organisation

Janssen - Cilag International

Address

Turnhoutseweg 30

City

Beerse

Postcode

2340

Country

Belgium

Scientific contact point

Organisation

Janssen - Cilag International

Contact name

CTIS Point of Contact

Public contact point

Organisation

Janssen - Cilag International

Contact name

CTIS Point of Contact

Third parties 10

Locations

10 EU/EEA countries · 17 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 127 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

17 submissions — initial application plus substantial / non-substantial modifications