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

NCT ID: NCT04224688

Last Updated: 2023-08-08

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE3
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-06-03
• Study Completion Date: 2022-05-05

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, of 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) etc.
• 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

Tp0006 50412

St. Petersburg, Florida, United States

Tp0006 50243

Boston, Massachusetts, United States

Tp0006 40189

Plovdiv, , Bulgaria

Tp0006 40008

Sofia, , Bulgaria

Tp0006 20201

Bengbu, , China

Tp0006 20189

Changchun, , China

Tp0006 20188

Changsha, , China

Tp0006 20186

Changzhou, , China

Tp0006 20179

Fuzhou, , China

Tp0006 20187

Hangzhou, , China

Tp0006 20177

Jinan, , China

Tp0006 20185

Jinan, , China

Tp0006 20194

Wuxi, , China

Tp0006 40364

Lille, , France

Tp0006 40369

Berlin, , Germany

Tp0006 40366

Rostock, , Germany

Tp0006 40219

Słupsk, , Poland

Tp0006 40223

Warsaw, , Poland

Tp0006 20052

Moscow, , Russia

Tp0006 20054

Moscow, , Russia

Tp0006 20053

Saint Petersburg, , Russia

Tp0006 40231

Madrid, , Spain

Tp0006 40268

Madrid, , Spain

Tp0006 40381

Zaragoza, , Spain

Tp0006 20094

Tainan City, , Taiwan

Tp0006 20095

Taipei, , Taiwan

Tp0006 20061

Cherkasy, , Ukraine

Tp0006 20064

Kyiv, , Ukraine

Tp0006 40239

Leeds, , United Kingdom

Countries

United States; Bulgaria; China; France; Germany; Poland; Russia; Spain; Taiwan; Ukraine; United Kingdom

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

2019-003451-11

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

TP0006

Identifier Type: -

Identifier Source: org_study_id