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

Primary Immune Thrombocytopenia Clinical Trial

— myOpportunITy2  

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)

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT04224688
• Other study ID #: TP0006
• Secondary ID: 2019-003451-11
• Status: Terminated
• Phase: Phase 3
• Start date: June 3, 2020
• Est. completion date: May 5, 2022

Study information

• Verified date: August 2023
• Source: UCB Pharma
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 30
• Est. completion date: May 5, 2022
• Est. primary completion date: April 25, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Primary Immune Thrombocytopenia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

Drug:
• Rozanolixizumab
Study participants receive rozanolixizumab by subcutaneous infusion at pre-specified time points.

Other:
• Placebo
Study participants receive placebo by subcutaneous infusion at pre-specified time points.

Locations

Country	Name	City	State
Bulgaria	Tp0006 40189	Plovdiv
Bulgaria	Tp0006 40008	Sofia
China	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
France	Tp0006 40364	Lille
Germany	Tp0006 40369	Berlin
Germany	Tp0006 40366	Rostock
Poland	Tp0006 40219	Slupsk
Poland	Tp0006 40223	Warszawa
Russian Federation	Tp0006 20052	Moscow
Russian Federation	Tp0006 20054	Moscow
Russian Federation	Tp0006 20053	Saint Petersburg
Spain	Tp0006 40231	Madrid
Spain	Tp0006 40268	Madrid
Spain	Tp0006 40381	Zaragoza
Taiwan	Tp0006 20094	Tainan
Taiwan	Tp0006 20095	Taipei City
Ukraine	Tp0006 20061	Cherkasy
Ukraine	Tp0006 20064	Kyiv
United Kingdom	Tp0006 40239	Leeds
United States	Tp0006 50243	Boston	Massachusetts
United States	Tp0006 50412	Saint Petersburg	Florida

Sponsors (1)

Lead Sponsor	Collaborator
UCB Biopharma SRL

Countries where clinical trial is conducted

United States,  Bulgaria,  China,  France,  Germany,  Poland,  Russian Federation,  Spain,  Taiwan,  Ukraine,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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 were reported.	During the last 12 weeks (Week 13 to Week 25)
Secondary	Cumulative Number of Weeks With Clinically Meaningful Platelet Response of =50×10^9/L Over the 24-week Treatment Period	Total number of weeks with platelet counts =50×10^9/L over the 24-week Treatment Period of the study (Week 1 to Week 25) were reported.	Week 1 up to Week 25
Secondary	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 from starting treatment to achievement of first Clinically Meaningful Platelet Response of =50×10^9/L was defined as date of first clinically meaningful response - date of first treatment + 1. Median was calculated based upon the Kaplan-Meier estimate.	Time from starting treatment to achievement of first response of =50×10^9/L (up to Week 25)
Secondary	Percentage of Participants With Clinically Meaningful Platelet Response of =50×10^9/L by Day 8	Clinically meaningful platelet response was defined as platelet count of =50×10^9/L.	Baseline to Day 8
Secondary	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	Response was 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.	From Baseline during Treatment Period (up to Week 25)
Secondary	Time to First Rescue Therapy	Time to first rescue therapy was defined as date of first rescue therapy use - date of first treatment + 1. Median was calculated based upon the Kaplan-Meier estimate.	From Baseline to first rescue therapy (up to Week 25)
Secondary	Change From Baseline to Week 25 in Primary Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score	The ITP-PAQ Version 1 is a 44 item disease-specific Health-Related Quality of Life questionnaire developed for use in adults with chronic ITP. It includes 10 scales, Four of the scales measure physical health: Symptoms (6 items), Bother (3 items), Fatigue (4 items), and Activity (2 items). Two of the scales measure emotional health: Fear (5 items) and Psychological (5 items) Health. The remaining four scales measure other aspects of quality of life (QOL): Work QOL (4 items), Social QOL (4 items), Women's Reproductive QOL (6 items) and Overall QOL (5 items). Each item is rated on a Likert-type scale containing 4 to 7 responses. All item scores are transformed to a 0 to 100 continuum and are weighted equally to derive individual scale scores and the total score (0-100) is calculated as per the formula: Sum of item scores within the scale/raw sum range*100. Higher scores indicate better health status.	From Baseline during Treatment Period (up to Week 25)
Secondary	Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)	An adverse event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of investigational medicinal product (IMP), whether or not considered related to the IMP. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IMP. TEAEs are defined as AEs starting after the time of first IMP administration up to and including 8 weeks (56 days) after the final dose.	From Baseline to end of Safety Follow-Up Period (up to Week 31)
Secondary	Percentage of Participants With TEAEs Leading to Withdrawal of Investigational Medicinal Product (ie, Study Discontinuation)	An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of IMP, whether or not considered related to the IMP. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IMP. TEAEs are defined as AEs starting after the time of first IMP administration up to and including 8 weeks (56 days) after the final dose.	From Baseline to end of Safety Follow-Up Period (up to Week 31)

External Links

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