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

• Register: ClinicalTrials.gov
• Last refreshed on: 21 August 2023
• Main ID: NCT04224688
• Date of registration: 08/01/2020
• Prospective Registration: Yes
• Primary sponsor: UCB Biopharma SRL
• Public title: A Study to Assess the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP) myOpportunITy2
• 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)
• Date of first enrolment: June 3, 2020
• Target sample size: 30
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/ct2/show/NCT04224688
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Austria, Belgium, Bulgaria, Canada, China, Czechia, Denmark, France, Germany, Hong Kong, Italy, Poland, Romania, Russian Federation, Serbia, Spain, Taiwan, Turkey, Ukraine, United Kingdom, United States

Contacts

• Name: UCB Cares
• Affiliation: 001 844 599 2273

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:

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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Primary Immune Thrombocytopenia

Intervention(s)

Drug: Rozanolixizumab

Other: Placebo

Primary Outcome(s)

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 [Time Frame: During the last 12 weeks (Week 13 to Week 25)]

Secondary Outcome(s)

Cumulative Number of Weeks With Clinically Meaningful Platelet Response of =50×10^9/L Over the 24-week Treatment Period [Time Frame: Week 1 up to Week 25]

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 [Time Frame: Time from starting treatment to achievement of first response of =50×10^9/L (up to Week 25)]

Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) [Time Frame: From Baseline to end of Safety Follow-Up Period (up to Week 31)]

Change From Baseline to Week 25 in Primary Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score [Time Frame: From Baseline during Treatment Period (up to Week 25)]

Time to First Rescue Therapy [Time Frame: From Baseline to first rescue therapy (up to Week 25)]

Percentage of Participants With TEAEs Leading to Withdrawal of Investigational Medicinal Product (ie, Study Discontinuation) [Time Frame: From Baseline to end of Safety Follow-Up Period (up to Week 31)]

Percentage of Participants With Clinically Meaningful Platelet Response of =50×10^9/L by Day 8 [Time Frame: Baseline to Day 8]

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 [Time Frame: From Baseline during Treatment Period (up to Week 25)]

Secondary ID(s)

TP0006

2019-003451-11

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 08/08/2023
• URL: https://clinicaltrials.gov/ct2/show/results/NCT04224688