A study to evaluate the efficacy, safety, and tolerability of rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP)

• Register: EUCTR
• Last refreshed on: 9 May 2022
• Main ID: EUCTR2019-000884-26-HR
• Date of registration: 24/11/2021
• Prospective Registration: No
• Primary sponsor: UCB Biopharma SRL
• Public title: A study to evaluate the efficacy, safety, and tolerability of rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP)
• Scientific title: 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) - myOpportunITy1
• Date of first enrolment: 09/09/2021
• Target sample size: 90
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2019-000884-26
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Austria, Belgium, Bulgaria, Croatia, Czech Republic, Czechia, France, Georgia, Greece, Hungary, Italy, Japan, Korea, Republic of, Moldova, Republic of, Netherlands, Poland, Romania, Russian Federation, Spain, Taiwan, Turkey, Ukraine, United Kingdom, United States

Contacts

• Name: Clin Trial Reg & Results Disclosure
• Affiliation: UCB Biosciences GmbH

• Name: Clin Trial Reg & Results Disclosure
• Affiliation: UCB Biosciences GmbH

Key inclusion & exclusion 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:
a. 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
b. 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
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 60
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 30

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 drug is 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


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Primary Immune Thrombocytopenia
MedDRA version: 20.0 Level: PT Classification code 10043554 Term: Thrombocytopenia System Organ Class: 10005329 - Blood and lymphatic system disorders

Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Intervention(s)

Product Name: Rozanolixizumab
Product Code: UCB7665
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: ROZANOLIXIZUMAB
CAS Number: 1584645-37-3
Current Sponsor code: UCB7665
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 140-
Pharmaceutical form of the placebo: Solution for infusion
Route of administration of the placebo: Subcutaneous use

Primary Outcome(s)

Main Objective: Demonstrate the clinical efficacy of rozanolixizumab in maintenance treatment in study participants with primary immune thrombocytopenia (ITP)

Secondary Objective: Assess the safety and tolerability of rozanolixizumab

Timepoint(s) of evaluation of this end point: During the last 12 weeks (Week 13 to Week 25)

Primary end point(s): Durable Clinically Meaningful Platelet Response of =50×10^9/L, for at least 8 out of 12 weeks during the last 12 weeks

Secondary Outcome(s)

Secondary end point(s): 1. Cumulative number of weeks with Clinically Meaningful Platelet Response of =50×10^9/L over the 24-week Treatment Period
2. Time to first Clinically Meaningful Platelet Response of =50×10^9/L: time from starting treatment to achievement of first response of =50×10^9/L
3. Clinically Meaningful Platelet Response of =50×10^9/L by Day 8
4. Response defined as platelet count =30×10^9/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
5. Time to first rescue therapy
6. Change from Baseline to Week 25 in ITP Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score
7. Occurrence of treatment-emergent adverse events (TEAEs)
8. Occurrence of TEAEs leading to withdrawal of investigational medicinal product (IMP) (ie, study discontinuation)

Timepoint(s) of evaluation of this end point: 1.; 4. From Baseline during Treatment Period (up to Week 25)
2. Time from starting treatment to achievement of first response of =50×10^9/L (up to Week 25)
3. Baseline to Day 8
5. From Baseline to first rescue therapy (up to Week 25)
6. From Baseline during Treatment Period (up to Week 25)
7.; 8. From Baseline to end of Safety Follow-Up Period (up to Week 32)

Secondary ID(s)

2019-000884-26-PL

TP0003

NCT04200456

Source(s) of Monetary Support

UCB Biopharma SRL

Ethics review

Status: Approved
Approval date: 24/06/2021
Contact: