Clinical trial NCT04224688: A Study to Assess the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)
Official 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)
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Primary Immune Thrombocytopenia
Keywords: ITP
UCB7665
Rozanolixizumab
Primary Immune Thrombocytopenia
Lead sponsor: UCB Pharma
Sponsor class: INDUSTRY
First posted: 2020-01-13
Enrollment: 30
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).


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

Primary outcomes:
measure: Percentage of Participants With Durable Clinically Meaningful Platelet Response of ≥50×10^9/L, for at Least 8 Out of 12 Weeks During the Last 12 Weeks
timeFrame: During the last 12 weeks (Week 13 to Week 25)
Secondary outcomes:
measure: Cumulative Number of Weeks With Clinically Meaningful Platelet Response of ≥50×10^9/L Over the 24-week Treatment Period
timeFrame: Week 1 up to Week 25
measure: Time to First Clinically Meaningful Platelet Response (CMPR) of ≥50×10^9/L: Time From Starting Treatment to Achievement of First Response of ≥50×10^9/L
timeFrame: Time from starting treatment to achievement of first response of ≥50×10^9/L (up to Week 25)
measure: Percentage of Participants With Clinically Meaningful Platelet Response of ≥50×10^9/L by Day 8
timeFrame: Baseline to Day 8
measure: Percentage of Participants With Response Defined as Platelet Count ≥30*10^9/L and at Least Doubling of Baseline, at Least 2 Separate Occasions at Two Adjacent Nominal Visits at Least 7 Days Apart, and Absence of Bleeding
timeFrame: From Baseline during Treatment Period (up to Week 25)
measure: Time to First Rescue Therapy
timeFrame: From Baseline to first rescue therapy (up to Week 25)
measure: Change From Baseline to Week 25 in Primary Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score
timeFrame: From Baseline during Treatment Period (up to Week 25)
measure: Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
timeFrame: From Baseline to end of Safety Follow-Up Period (up to Week 31)
measure: Percentage of Participants With TEAEs Leading to Withdrawal of Investigational Medicinal Product (ie, Study Discontinuation)
timeFrame: From Baseline to end of Safety Follow-Up Period (up to Week 31)
Other outcomes:
Locations:
city: Saint Petersburg
country: United States
facility: Tp0006 50412
state: Florida
city: Boston
country: United States
facility: Tp0006 50243
state: Massachusetts
city: Plovdiv
country: Bulgaria
facility: Tp0006 40189
city: Sofia
country: Bulgaria
facility: Tp0006 40008
city: Bengbu
country: China
facility: Tp0006 20201
city: Changchun
country: China
facility: Tp0006 20189
city: Changsha
country: China
facility: Tp0006 20188
city: Changzhou
country: China
facility: Tp0006 20186
city: Fuzhou
country: China
facility: Tp0006 20179
city: Hangzhou
country: China
facility: Tp0006 20187
city: Jinan
country: China
facility: Tp0006 20177
city: Jinan
country: China
facility: Tp0006 20185
city: Wuxi
country: China
facility: Tp0006 20194
city: Lille
country: France
facility: Tp0006 40364
city: Berlin
country: Germany
facility: Tp0006 40369
city: Rostock
country: Germany
facility: Tp0006 40366
city: Slupsk
country: Poland
facility: Tp0006 40219
city: Warszawa
country: Poland
facility: Tp0006 40223
city: Moscow
country: Russian Federation
facility: Tp0006 20052
city: Moscow
country: Russian Federation
facility: Tp0006 20054
city: Saint Petersburg
country: Russian Federation
facility: Tp0006 20053
city: Madrid
country: Spain
facility: Tp0006 40231
city: Madrid
country: Spain
facility: Tp0006 40268
city: Zaragoza
country: Spain
facility: Tp0006 40381
city: Tainan
country: Taiwan
facility: Tp0006 20094
city: Taipei City
country: Taiwan
facility: Tp0006 20095
city: Cherkasy
country: Ukraine
facility: Tp0006 20061
city: Kyiv
country: Ukraine
facility: Tp0006 20064
city: Leeds
country: United Kingdom
facility: Tp0006 40239
Study officials:
affiliation: 001 844 599 2273
name: UCB Cares
role: STUDY_DIRECTOR
References: