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Trial record 1 of 1 for:    NCT04224688
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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 : November 6, 2020
Sponsor:
Information provided by (Responsible Party):
UCB Pharma ( UCB Biopharma SRL )

Brief Summary:
The purpose of this study is to demonstrate the clinical efficacy of rozanolixizumab in maintenance treatment and asses safety and tolerability of rozanolixizumab in adult study participants with primary immune thrombocytopenia (ITP).

Condition or disease Intervention/treatment Phase
Primary Immune Thrombocytopenia Drug: Rozanolixizumab Other: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 105 participants
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
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)
Actual Study Start Date : June 3, 2020
Estimated Primary Completion Date : July 2022
Estimated Study Completion Date : August 2022


Arm Intervention/treatment
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.
Drug: Rozanolixizumab
Study participants receive rozanolixizumab by subcutaneous infusion at pre-specified time points.
Other Name: UCB7665

Placebo Comparator: Placebo
Study participants randomized to this arm receive placebo at pre-specified time points during the Treatment Period.
Other: Placebo
Study participants receive placebo by subcutaneous infusion at pre-specified time points.




Primary Outcome Measures :
  1. 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)


Secondary Outcome Measures :
  1. 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).

  2. 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.

  3. 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.

  4. 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

  5. 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.

  6. 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.

  7. 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.

    The ITP-PAQ is a 44-item disease-specific quality of life (QoL) 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.


  8. 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.

  9. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 documented intolerance or insufficient response to one or more appropriate courses of standard of care ITP medication prior to Screening
  • Study participants must have prior history of a response to a previous ITP therapy.
  • If taking allowed immunosuppressive 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 attach, 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 investigational medicinal product (IMP) or comparative drugs (and/or an investigational device) as stated in this protocol
  • Study participant has evidence of a secondary cause of immune thrombocytopenia from the past medical history (eg, bacterial or viral infection, past medical history of leukemia, lymphoma, common variable immunodeficiency, systemic lupus erythematosus, autoimmune thyroid disease) or to drug treatments (eg, heparin, quinine, antimicrobials, anticonvulsants) or participant has a multiple immune cytopenia, eg, Evan's syndrome
  • 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 dose of 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04224688


Contacts
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Contact: UCB Cares +1844599 ext 2273 UCBCares@ucb.com

Locations
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United States, Massachusetts
Tp0006 50243 Recruiting
Boston, Massachusetts, United States, 02114
Bulgaria
Tp0006 40008 Recruiting
Sofia, Bulgaria
Canada
Tp0006 50309 Recruiting
Saint John's, Canada
France
Tp0006 40374 Recruiting
Dijon, France
Germany
Tp0006 40367 Recruiting
Frankfurt, Germany
Poland
Tp0006 40219 Recruiting
Słupsk, Poland
Tp0006 40223 Recruiting
Warszawa, Poland
Russian Federation
Tp0006 20054 Recruiting
Moscow, Russian Federation
Tp0006 20053 Recruiting
Saint Petersburg, Russian Federation
Spain
Tp0006 40232 Recruiting
Palma De Mallorca, Spain
Taiwan
Tp0006 20096 Recruiting
Tainan, Taiwan
Ukraine
Tp0006 20061 Recruiting
Cherkasy, Ukraine
Tp0006 20064 Recruiting
Kyiv, Ukraine
United Kingdom
Tp0006 40055 Recruiting
Norwich, United Kingdom
Sponsors and Collaborators
UCB Biopharma SRL
Investigators
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Study Director: UCB Cares +1-844-599-2273 (UCB)
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Responsible Party: UCB Biopharma SRL
ClinicalTrials.gov Identifier: NCT04224688    
Other Study ID Numbers: TP0006
2019-003451-11 ( EudraCT Number )
First Posted: January 13, 2020    Key Record Dates
Last Update Posted: November 6, 2020
Last Verified: November 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: 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. Investigators 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.clinicalstudydatarequest.com 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. This plan may change if a determination is made that the data cannot be adequately anonymized.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
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.clinicalstudydatarequest.com 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.clinicalstudydatarequest.com/

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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
Keywords provided by UCB Pharma ( UCB Biopharma SRL ):
ITP
UCB7665
Rozanolixizumab
Primary Immune Thrombocytopenia
Additional relevant MeSH terms:
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Thrombocytopenia
Purpura, Thrombocytopenic, Idiopathic
Blood Platelet Disorders
Hematologic Diseases
Purpura, Thrombocytopenic
Purpura
Blood Coagulation Disorders
Thrombotic Microangiopathies
Hemorrhagic Disorders
Autoimmune Diseases
Immune System Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations
Rozanolixizumab
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs