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

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ClinicalTrials.gov Identifier: NCT04224688
Recruitment Status : Recruiting
First Posted : January 13, 2020
Last Update Posted : April 9, 2021
Sponsor:
Information provided by (Responsible Party):
UCB Pharma ( UCB Biopharma SRL )

Tracking Information
First Submitted Date  ICMJE January 8, 2020
First Posted Date  ICMJE January 13, 2020
Last Update Posted Date April 9, 2021
Actual Study Start Date  ICMJE June 3, 2020
Estimated Primary Completion Date July 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 4, 2021)
Durable Clinically Meaningful Platelet Response of ≥50x10^9/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) ]
Durable Clinically Meaningful Platelet Response of ≥50x10^9/L, for at least 8 out of 12 weeks during the last 12 weeks (Week 13 to 25)
Original Primary Outcome Measures  ICMJE
 (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)
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: February 4, 2021)
  • Cumulative number of weeks with Clinically Meaningful Platelet Response of ≥50x10^9/L over the 24-week Treatment Period [ Time Frame: From Baseline during Treatment Period (up to Week 25) ]
    Total number of weeks with platelet counts ≥50x10^9/L over the 24-week Treatment Period of the study (Week 1 to Week 25).
  • Time to first 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 first 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.
  • 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.
  • 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.
  • Change from Baseline to Week 25, including all intermediate timepoints, in ITP Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score [ Time Frame: From Baseline during Treatment Period, including intermediate timepoints Week 1; 5; 9; 13; 17; 21 and 25 (up to Week 25) ]
    The ITP-PAQ is a 44-item disease-specific Health-Related Quality of life (HRQoL) questionnaire developed for use in adults with chronic ITP. It includes 11 scales: Symptoms, Fatigue, Physical Health - Bother, Physical Health - Activity, Emotional Health - Psychological, Emotional Health - Fear, Overall QoL, Social Activity, Women's Reproductive Health - Fertility, Women's Reproductive Health - Menstrual Symptoms, and Work. 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. Higher scores indicate better health status.
  • 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) (ie, study discontinuation) [ 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.
Original Secondary Outcome Measures  ICMJE
 (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  ICMJE 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  ICMJE 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  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE 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  ICMJE Primary Immune Thrombocytopenia
Intervention  ICMJE
  • 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  ICMJE
  • 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

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: February 4, 2021)
90
Original Estimated Enrollment  ICMJE
 (submitted: January 8, 2020)
105
Estimated Study Completion Date  ICMJE August 2022
Estimated Primary Completion Date July 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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 medications 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 (<30x10^9/L) prior to Screening
  • Study participant has a platelet count measurement at Screening and at Baseline (Day 1) with an average of the two <30x10^9/L and no single count may be >35x10^9/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 or 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 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 (eg, past medical history of untreated H. pylori infection, leukemia, lymphoma, common variable immunodeficiency, systemic lupus erythematosus, autoimmune thyroid disease or to 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 has a Karnofsky Performance Status rating <60% at the Screening Visit
  • 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  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: UCB Cares +1844599 ext 2273 UCBCares@ucb.com
Listed Location Countries  ICMJE Austria,   Belgium,   Bulgaria,   Canada,   China,   Czechia,   France,   Germany,   Poland,   Russian Federation,   Spain,   Taiwan,   Ukraine,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04224688
Other Study ID Numbers  ICMJE 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  ICMJE
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
Responsible Party UCB Pharma ( UCB Biopharma SRL )
Study Sponsor  ICMJE UCB Biopharma SRL
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: UCB Cares +1-844-599-2273 (UCB)
PRS Account UCB Pharma
Verification Date April 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP