A Study to Evaluate the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)

NCT ID: NCT04200456

Last Updated: 2025-03-18

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE3
• Total Enrollment: 33 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-01-31
• Study Completion Date: 2022-04-27

Brief Summary

The purpose of this study is to demonstrate the clinical efficacy of rozanolixizumab in maintenance treatment and assess safety and tolerability of rozanolixizumab in adult study participants with primary immune thrombocytopenia (ITP).

Conditions

Primary Immune Thrombocytopenia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Rozanolixizumab

Study participants randomized to this arm will receive fixed-unit doses of rozanolixizumab across body weight tiers at pre-specified time points during the Treatment Period. Doses will be adjusted based on platelet count values or medical needs.

Group Type: EXPERIMENTAL

Rozanolixizumab

Intervention Type: DRUG

Study participants receive rozanolixizumab by subcutaneous infusion at pre-specified time points.

Placebo

Study participants randomized to this arm receive placebo at pre-specified time points during the Treatment Period.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: OTHER

Study participants receive placebo by subcutaneous infusion at pre-specified time points.

Interventions

Rozanolixizumab

Study participants receive rozanolixizumab by subcutaneous infusion at pre-specified time points.

Intervention Type: DRUG

Placebo

Study participants receive placebo by subcutaneous infusion at pre-specified time points.

Intervention Type: OTHER

Other Intervention Names

UCB7665

Eligibility Criteria

Inclusion Criteria

• Study participant must be ≥18 years of age at the time of the Screening Visit
• Study participant has a diagnosis of persistent (>3 months duration) or chronic (>12 months duration) primary immune thrombocytopenia (ITP) at the Screening Visit
• Study participant has a documented intolerance or insufficient response to two or more appropriate standard of care ITP treatments prior to Screening
• Study participants must have prior history of a response to a previous ITP therapy
• If taking allowed drugs, study participant must be on stable doses during defined time periods prior to Baseline (Day 1)
• Study participant has a documented history of low platelet count (<30×10^9/L) prior to Screening
• Study participant has a platelet count measurement at Screening and at Baseline (Day 1) with an average of the two <30×10^9/L and no single count may be >35×10^9/L (using local laboratories)
• Study participant has a current or history of a peripheral blood smear consistent with ITP
• Study participants may be male or female:

1. A male participant must agree to use contraception during the Treatment Period and for at least 3 months after the final dose of study treatment and refrain from donating sperm during this period

2. A female participant is eligible to participate if she is not pregnant as confirmed by a negative serum pregnancy test and not planning to get pregnant during the participation in the study, not breastfeeding, and at least one of the following conditions applies:

Not a woman of childbearing potential (WOCBP) OR A WOCBP who agrees to follow the contraceptive guidance during the Treatment Period and for at least 3 months after the dose of study treatment

Exclusion Criteria

• Participant has a history of arterial or venous thromboembolism (eg, stroke, transient ischemic attack, myocardial infarction, deep vein thrombosis or pulmonary embolism) within the 6 months prior to randomization or requires current anticoagulant treatment
• Study participant has clinically significant bleeding that warrants immediate platelet adjustment (eg, menorrhagia with significant drop in hemoglobin)
• Study participant has a known hypersensitivity to any components of the study medication or any other anti-neonatal Fc receptor (FcRn) medications
• Study participant has evidence of a secondary cause of immune thrombocytopenia (clear association with other medical conditions, eg, untreated H. pylori infection, leukemia, lymphoma, common variable immunodeficiency, systemic lupus erythematosus, autoimmune thyroid disease or is drug induced), participant has a multiple immune cytopenia (eg, Evan's syndrome)
• Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess) in the opinion of the investigator, or had a serious infection (resulting in hospitalization or requiring parenteral antibiotic treatment) within 6 weeks prior to the first dose of investigational medicinal product (IMP)
• Study participant with a known tuberculosis (TB) infection, at high risk of acquiring TB infection, or latent tuberculosis infection (LTBI), or current/history of nontuberculous mycobacterial infection (NTMBI)
• Study participant has a history of a major organ transplant or hematopoietic stem cell/marrow transplant
• Study participant has experienced intracranial bleed in the last 6 months prior to the Screening Visit
• Study participant has a history of coagulopathy disorders other than ITP
• Study participant with current or medical history of immunoglobulin A (IgA) deficiency, or a measurement of IgA <50 mg/dL at the Screening Visit
• Study participant has undergone a splenectomy in the 2 years prior to the Baseline Visit

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

UCB Biopharma SRL

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

UCB Cares

Role: STUDY_DIRECTOR

001 844 599 2273

Locations

Tp0003 50244

Seattle, Washington, United States

Tp0003 40188

Sofia, , Bulgaria

Tp0003 40197

Amiens, , France

Tp0003 40196

Pessac, , France

Tp0003 20050

Tbilisi, , Georgia

Tp0003 40558

Thessaloniki, , Greece

Tp0003 40202

Győr, , Hungary

Tp0003 40178

Nyíregyháza, , Hungary

Tp0003 40204

Pécs, , Hungary

Tp0003 40208

Florence, , Italy

Tp0003 20030

Chūōku, , Japan

Tp0003 20039

Iruma-gun, , Japan

Tp0003 20159

Shibuya-ku, , Japan

Tp0003 20051

Chisinau, , Moldova

Tp0003 40218

Gdansk, , Poland

Tp0003 40221

Lodz, , Poland

Tp0003 40222

Skorzewo, , Poland

Tp0003 40225

Bucharest, , Romania

Tp0003 40226

Bucharest, , Romania

Tp0003 20055

Saint Petersburg, , Russia

Tp0003 20218

Daegu, , South Korea

Tp0003 20207

Seoul, , South Korea

Tp0003 20099

Taipei, , Taiwan

Tp0003 20060

Dnipropetrovsk, , Ukraine

Tp0003 20062

Ivano-Frankivsk, , Ukraine

Tp0003 20063

Kyiv, , Ukraine

Tp0003 20100

Zaporizhzhia, , Ukraine

Tp0003 40238

London, , United Kingdom

Tp0003 40234

Plymouth, , United Kingdom

Countries

United States; Bulgaria; France; Georgia; Greece; Hungary; Italy; Japan; Moldova; Poland; Romania; Russia; South Korea; Taiwan; Ukraine; United Kingdom

References

Cooper N, Bussel JB, Kazmierczak M, Miyakawa Y, Cluck S, Lledo Garcia R, Haier B, Lavrov A, Singh P, Snipes R, Kuter DJ. Inhibition of FcRn with rozanolixizumab in adults with immune thrombocytopenia: Two randomised, double-blind, placebo-controlled phase 3 studies and their open-label extension. Br J Haematol. 2025 Feb;206(2):675-688. doi: 10.1111/bjh.19858. Epub 2024 Nov 18.

Reference Type: RESULT

PMID: 39552477 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

http://www.fda.gov/Safety/MedWatch/SafetyInformation/default.htm

FDA Safety Alerts and Recalls

Other Identifiers

2019-000884-26

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

TP0003

Identifier Type: -

Identifier Source: org_study_id