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Trial record 1 of 1 for:    NCT04596995
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A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP) (myOpportunITy3)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04596995
Recruitment Status : Completed
First Posted : October 22, 2020
Last Update Posted : January 18, 2023
Sponsor:
Information provided by (Responsible Party):
UCB Pharma ( UCB Biopharma SRL )

Study Description
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Brief Summary:
The purpose of this study is to assess the long-term safety, tolerability and clinical efficacy of treatment with rozanolixizumab.

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

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 43 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Extension Study to Investigate the Long-Term Safety, Tolerability, and Efficacy of Rozanolixizumab in Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)
Actual Study Start Date : December 30, 2020
Actual Primary Completion Date : December 23, 2022
Actual Study Completion Date : December 23, 2022

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Rozanolixizumab Treatment Arm
All study participants 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 during the Treatment Period.
Other Name: UCB7665


Outcome Measures
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Primary Outcome Measures :
  1. Occurrence of treatment-emergent adverse events (TEAEs) [ Time Frame: From Baseline to end of Safety Follow-Up Period (up to Week 60) ]
    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.

  2. Occurrence of TEAEs leading to permanent withdrawal of rozanolixizumab (ie, study discontinuation) [ Time Frame: From Baseline to end of Safety Follow-Up Period (up to Week 60) ]
    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.


Secondary Outcome Measures :
  1. Stable Clinically Meaningful Response without rescue therapy at ≥70% of the visits over the planned 52-week Treatment Period starting at Week 4 [ Time Frame: Over the planned 52-week Treatment Period (starting at Week 4) ]
    Stable Clinically Meaningful Response, defined as Clinically Meaningful Response (ie, platelet count ≥50×10^9/L) without rescue therapy at ≥70% of the visits over the planned 52-week Treatment Period starting at Week 4

  2. Change from Baseline to Week 53 including all intermediate timepoints for ITP Patient Assessment Questionnaire (ITP-PAQ) Symptoms domain score [ Time Frame: From Baseline during the Treatment Period (up to Week 53) ]
    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.

  3. Area under the curve (AUC) of the oral steroid dose over time [ Time Frame: From Baseline during the Treatment Period (up to Week 53) ]
    Area under the curve (AUC) of the oral steroid dose over time

  4. Change in dose and/or frequency of concomitant ITP medications (excluding corticosteroids) over time [ Time Frame: From Baseline during the Treatment Period (up to Week 53) ]
    Change in dose and/or frequency of concomitant ITP medications (excluding corticosteroids) over time will be measured.


Eligibility Criteria
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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 completed TP0003 [NCT04200456] or TP0006 [NCT04224688] until Visit 27 (Week 25) and, in the opinion of the investigator, has been compliant with the TP0003 or TP0006 study assessments
  • The study participant is considered reliable and capable of adhering to the protocol, visit schedule, or medication intake according to the judgment of the investigator

  • 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 urine 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 final dose of study treatment

Exclusion Criteria:

  • Study participant has any ongoing investigational medicinal product (IMP)-related serious adverse event (SAE) or ongoing severe IMP-related treatment-emergent adverse event (TEAE) experienced during TP0003 or TP0006
  • Study participant has, at last available assessment of TP0003 or TP0006, 3.0x upper limit of normal (ULN) of any of the following: alanine aminotransferase (ALT), aspartate aminotransferase (AST), or alkaline phosphatase (ALP)
Contacts and Locations
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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): NCT04596995


Locations
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Sponsors and Collaborators
UCB Biopharma SRL
Investigators
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Study Director: UCB Cares 001 844 599 2273
More Information
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Responsible Party: UCB Biopharma SRL
ClinicalTrials.gov Identifier: NCT04596995    
Other Study ID Numbers: TP0004
2019-000883-40 ( EudraCT Number )
First Posted: October 22, 2020    Key Record Dates
Last Update Posted: January 18, 2023
Last Verified: January 2023
Individual Participant Data (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
Statistical Analysis Plan (SAP)
Clinical Study Report (CSR)
Time Frame: 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.
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

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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