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

A Phase 3 Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)

Sponsors

Lead Sponsor: UCB Biopharma SRL

Source UCB Pharma
Brief Summary

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

Overall Status Recruiting
Start Date January 31, 2020
Completion Date February 2022
Primary Completion Date December 2021
Phase Phase 3
Study Type Interventional
Primary Outcome
Measure Time Frame
Durable Clinically Meaningful Platelet Response of ≥50x10^9/L during the last 12 weeks During the last 12 weeks (Week 13 to Week 25)
Secondary Outcome
Measure Time Frame
Cumulative number of visits with Clinically Meaningful Platelet Response of ≥50x10^9/L From Baseline during Treatment Period (up to Week 25)
Response defined as platelet count ≥30x10^9/L and at least a 2-fold increase of the Baseline count confirmed on at least 2 separate occasions at two adjacent nominal visits at least 7 days apart, and absence of bleeding by visit From Baseline during Treatment Period (up to Week 25)
Complete Response defined as platelet count ≥100x10^9/L confirmed on at least 2 separate occasions at two adjacent nominal visits at least 7 days apart, and absence of bleeding by visit From Baseline during Treatment Period (up to Week 25)
Time to Clinically Meaningful Platelet Response of ≥50x10^9/L: time from starting treatment to achievement of first response of ≥50x10^9/L Time from starting treatment to achievement of first response of ≥50x10^9/L (up to Week 25)
Clinically Meaningful Platelet Response of ≥50x10^9/L by Day 8 Baseline to Day 8
Time to first rescue therapy From Baseline to first rescue therapy (up to Week 25)
Response defined as change from Baseline at or above the defined threshold for ITP Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score From Baseline during Treatment Period (up to Week 25)
Occurrence of treatment-emergent adverse events (TEAEs) From Baseline to end of Safety Follow-Up Period (up to Week 31)
Occurrence of TEAEs leading to withdrawal of investigational medicinal product (IMP) From Baseline to end of Safety Follow-Up Period (up to Week 31)
Enrollment 105
Condition
Intervention

Intervention Type: Drug

Intervention Name: Rozanolixizumab

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

Arm Group Label: Rozanolixizumab

Other Name: UCB7665

Intervention Type: Other

Intervention Name: Placebo

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

Arm Group Label: Placebo

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 documented intolerance or insufficient response to one or more appropriate courses of standard of care ITP medication prior to Screening - Study participants must have prior history of a response to a previous ITP therapy. - If taking allowed immunosuppressive 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 (<30x10^9/L) prior to Screening - Study participant has a platelet count measurement at Screening and at Baseline (Day 1) with an average of the two <30x10^9/L and no single count may be >35x10^9/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 or 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 attach, myocardial infarction, deep vein thrombosis or pulmonary embolism) within the 6 months prior to randomization or requires 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 investigational medicinal product (IMP) or comparative drugs (and/or an investigational device) as stated in this protocol - Study participant has evidence of a secondary cause of immune thrombocytopenia from the past medical history (eg, bacterial or viral infection, past medical history of leukemia, lymphoma, common variable immunodeficiency, systemic lupus erythematosus, autoimmune thyroid disease) or to drug treatments (eg, heparin, quinine, antimicrobials, anticonvulsants) or 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 dose of 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 has a Karnofsky Performance Status rating <60% at the Screening Visit - 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

Gender: All

Minimum Age: 18 Years

Maximum Age: N/A

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
UCB Cares Study Director +1-844-599-2273 (UCB)
Overall Contact

Last Name: UCB Cares

Phone: +1844599

Phone Ext.: 2273

Email: [email protected]

Location
Facility: Status:
Tp0003 50241 | Syracuse, New York, 13210, United States Recruiting
Tp0003 40184 | Haine-Saint-Paul, Belgium Recruiting
Tp0003 40182 | Yvoir, Belgium Recruiting
Tp0003 40188 | Sofia, Bulgaria Recruiting
Tp0003 40190 | Sofia, Bulgaria Recruiting
Tp0003 40342 | Angers, France Recruiting
Tp0003 40196 | Pessac, France Recruiting
Tp0003 20050 | Tbilisi, Georgia Recruiting
Tp0003 40178 | Nyiregyhaza, Hungary Recruiting
Tp0003 40204 | Pécs, Hungary Recruiting
Tp0003 20068 | Chiba, Japan Recruiting
Tp0003 20039 | Hidaka, Japan Recruiting
Tp0003 20167 | Meguro, Japan Recruiting
Tp0003 20176 | Nagasaki, Japan Recruiting
Tp0003 20175 | Nagoya, Japan Recruiting
Tp0003 20159 | Shibuya-Ku, Japan Recruiting
Tp0003 20158 | Shinagawa-Ku, Japan Recruiting
Tp0003 20140 | Yokohama, Japan Recruiting
Tp0003 20051 | Chisinau, Moldova, Republic of Recruiting
Tp0003 40218 | Gdańsk, Poland Recruiting
Tp0003 40222 | Skorzewo, Poland Recruiting
Tp0003 40221 | Łódź, Poland Recruiting
Tp0003 20093 | Moscow, Russian Federation Recruiting
Tp0003 20057 | Saint Petersburg, Russian Federation Recruiting
Tp0003 40049 | Sevilla, Spain Recruiting
Tp0003 40230 | Valencia, Spain Recruiting
Tp0003 20062 | Ivano-Frankivs'k, Ukraine Recruiting
Tp0003 20063 | Kyiv, Ukraine Recruiting
Tp0003 40234 | Plymouth, United Kingdom Recruiting
Location Countries

Belgium

Bulgaria

France

Georgia

Hungary

Japan

Moldova, Republic of

Poland

Russian Federation

Spain

Ukraine

United Kingdom

United States

Verification Date

October 2020

Responsible Party

Type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 2
Arm Group

Label: Rozanolixizumab

Type: Experimental

Description: 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.

Label: Placebo

Type: Placebo Comparator

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

Acronym myOpportunITy1
Patient Data Yes
Study Design Info

Allocation: Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Treatment

Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Masking Description: Investigators are blinded to the treatment code, they will see the platelet values.

Source: ClinicalTrials.gov