A Study to Assess the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP) (myOpportunITy2)

• ClinicalTrials.gov Identifier: NCT04224688
• Recruitment Status: Terminated
• First Posted: January 13, 2020
• Results First Posted: August 8, 2023
• Last Update Posted: August 8, 2023
• Sponsor: UCB Biopharma SRL
• Information provided by (Responsible Party): UCB Pharma ( UCB Biopharma SRL )

Tracking Information

• First Submitted Date: January 8, 2020
• First Posted Date: January 13, 2020
• Results First Submitted Date: May 15, 2023
• Results First Posted Date: August 8, 2023
• Last Update Posted Date: August 8, 2023
• Actual Study Start Date: June 3, 2020
• Actual Primary Completion Date: April 25, 2022 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: May 15, 2023)

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) ]

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.

Original Primary Outcome Measures (submitted: January 8, 2020)

Durable Clinically Meaningful Platelet Response of ≥50x10^9/L during the last 12 weeks [ Time Frame: During the last 12 weeks (Week 13 to Week 25) ]

Durable Clinically Meaningful Platelet Response of ≥50x10^9/L, as defined by proportion of study participants who have platelet responses for at least 8 out of 12 weeks during the last 12 weeks (Week 13 to 25)

Current Secondary Outcome Measures (submitted: August 4, 2023)

• 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 ]
  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.
• 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) ]
  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.
• Percentage of Participants With Clinically Meaningful Platelet Response of ≥50×10^9/L by Day 8 [ Time Frame: Baseline to Day 8 ]
  Clinically meaningful platelet response was defined as platelet count of ≥50×10^9/L.
• 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) ]
  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.
• Time to First Rescue Therapy [ Time Frame: From Baseline to first rescue therapy (up to Week 25) ]
  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.
• 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) ]
  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.
• Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) [ Time Frame: From Baseline to end of Safety Follow-Up Period (up to Week 31) ]
  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.
• 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) ]
  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.

Original Secondary Outcome Measures (submitted: January 8, 2020)

• Cumulative number of visits with Clinically Meaningful Platelet Response of ≥50x10^9/L [ Time Frame: From Baseline during Treatment Period (up to Week 25) ]
  Number of weeks (visits) with platelet counts ≥50x10^9/L over the Treatment Period of the study (Week 1 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 [ Time Frame: 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.
• 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 [ Time Frame: 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.
• Time to Clinically Meaningful Platelet Response of ≥50x10^9/L: time from starting treatment to achievement of first response of ≥50x10^9/L [ Time Frame: Time from starting treatment to achievement of first response of ≥50x10^9/L (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
• Clinically Meaningful Platelet Response of ≥50x10^9/L by Day 8 [ Time Frame: Baseline to Day 8 ]
  Clinically meaningful Response defined as: platelet count ≥50x10^9/L.
• Time to first rescue therapy [ Time Frame: From Baseline to first rescue therapy (up to Week 25) ]
  Time to first rescue therapy use will be analyzed using a Cox Proportional Hazards model with fixed terms for treatment, splenectomy,degree of thrombocytopenia (platelet count < or ≥ 15x10^9/L), and geographical region.
• Response defined as change from Baseline at or above the defined threshold for ITP Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score [ Time Frame: From Baseline during Treatment Period (up to Week 25) ]
  The ITP Patient Assessment Questionnaire (ITP-PAQ) responder rates will always be calculated relative to the Baseline and will be limited to the participants with a Baseline ITP-PAQ score at or above the applicable threshold score.
• Occurrence of treatment-emergent adverse events (TEAEs) [ Time Frame: From Baseline to end of Safety Follow-Up Period (up to Week 31) ]
  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.
• Occurrence of TEAEs leading to withdrawal of investigational medicinal product (IMP) [ Time Frame: From Baseline to end of Safety Follow-Up Period (up to Week 31) ]
  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.

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: 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)
• 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).
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3

Study Design

Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description:

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

Primary Purpose: Treatment

• Condition: Primary Immune Thrombocytopenia

Intervention

• Drug: Rozanolixizumab
  Study participants receive rozanolixizumab by subcutaneous infusion at pre-specified time points.
  Other Name: UCB7665
• Other: Placebo
  Study participants receive placebo by subcutaneous infusion at pre-specified time points.

Study Arms

• Experimental: Rozanolixizumab
  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.
  Intervention: Drug: Rozanolixizumab
• Placebo Comparator: Placebo
  Study participants randomized to this arm receive placebo at pre-specified time points during the Treatment Period.
  Intervention: Other: Placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Terminated
• Actual Enrollment (submitted: August 17, 2022): 30
• Original Estimated Enrollment (submitted: January 8, 2020): 105
• Actual Study Completion Date: May 5, 2022
• Actual Primary Completion Date: April 25, 2022 (Final data collection date for primary outcome measure)

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Bulgaria, China, France, Germany, Poland, Russian Federation, Spain, Taiwan, Ukraine, United Kingdom, United States
• Removed Location Countries: Austria, Belgium, Canada, Czechia, Denmark, Hong Kong, Italy, Romania, Serbia, Turkey

Administrative Information

• NCT Number: NCT04224688
• Other Study ID Numbers: TP0006; 2019-003451-11 ( EudraCT Number )
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Product Manufactured in and Exported from the U.S.: No

IPD Sharing Statement

• Plan to Share IPD: Yes
• Plan Description: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.
• Supporting Materials: Study Protocol
• Supporting Materials: Statistical Analysis Plan (SAP)
• Supporting Materials: Clinical Study Report (CSR)
• Time Frame: Data from this study may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
• Access Criteria: Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.
• URL: https://www.Vivli.org

• Current Responsible Party: UCB Pharma ( UCB Biopharma SRL )
• Original Responsible Party: Same as current
• Current Study Sponsor: UCB Biopharma SRL
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: UCB Cares; 001 844 599 2273

• PRS Account: UCB Pharma
• Verification Date: August 2023