Back to resultsA Study to Evaluate the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP) (myOpportunITy1)
Primary Purpose
Primary Immune Thrombocytopenia
Status
Terminated
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Rozanolixizumab
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Primary Immune Thrombocytopenia focused on measuring ITP, UCB7665, Rozanolixizumab, Primary immune thrombocytopenia
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:
- 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
- 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, 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)
- 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
Sites / Locations
- Tp0003 50244
- Tp0003 40188
- Tp0003 40197
- Tp0003 40196
- Tp0003 20050
- Tp0003 40558
- Tp0003 40202
- Tp0003 40178
- Tp0003 40204
- Tp0003 40208
- Tp0003 20030
- Tp0003 20039
- Tp0003 20159
- Tp0003 20218
- Tp0003 20207
- Tp0003 20051
- Tp0003 40218
- Tp0003 40221
- Tp0003 40222
- Tp0003 40226
- Tp0003 40225
- Tp0003 20055
- Tp0003 20099
- Tp0003 20060
- Tp0003 20062
- Tp0003 20063
- Tp0003 20100
- Tp0003 40238
- Tp0003 40234
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Rozanolixizumab
Placebo
Arm Description
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.
Study participants randomized to this arm receive placebo at pre-specified time points during the Treatment Period.
Outcomes
Primary Outcome Measures
Percentage of Participants With Durable Clinically Meaningful Platelet Response of ≥50x10^9/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.
Secondary Outcome Measures
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.
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.
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.
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.
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. The probability of requiring rescue medication did not reach 0.5 so the KM median in the rozanolixizumab arm could not be estimated.
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.
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.
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04200456
Brief Title
A Study to Evaluate the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)
Acronym
myOpportunITy1
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)
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Terminated
Why Stopped
Strategic Business Decision; Not a safety decision
Study Start Date
January 31, 2020 (Actual)
Primary Completion Date
March 21, 2022 (Actual)
Study Completion Date
April 27, 2022 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
UCB Biopharma SRL
4. Oversight
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
Data Monitoring Committee
Yes
5. Study Description
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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Immune Thrombocytopenia
Keywords
ITP, UCB7665, Rozanolixizumab, Primary immune thrombocytopenia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Masking Description
Investigators are blinded to the treatment code, they will see the platelet values.
Allocation
Randomized
Enrollment
33 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Rozanolixizumab
Arm Type
Experimental
Arm Description
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.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Study participants randomized to this arm receive placebo at pre-specified time points during the Treatment Period.
Intervention Type
Drug
Intervention Name(s)
Rozanolixizumab
Other Intervention Name(s)
UCB7665
Intervention Description
Study participants receive rozanolixizumab by subcutaneous infusion at pre-specified time points.
Intervention Type
Other
Intervention Name(s)
Placebo
Intervention Description
Study participants receive placebo by subcutaneous infusion at pre-specified time points.
Primary Outcome Measure Information:
Title
Percentage of Participants With Durable Clinically Meaningful Platelet Response of ≥50x10^9/L, for at Least 8 Out of 12 Weeks During the Last 12 Weeks
Description
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.
Time Frame
During the last 12 weeks (Week 13 to Week 25)
Secondary Outcome Measure Information:
Title
Cumulative Number of Weeks With Clinically Meaningful Platelet Response of ≥50×10^9/L Over the 24-week Treatment Period
Description
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 Frame
Week 1 up to Week 25
Title
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
Description
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 Frame
Time from starting treatment to achievement of first response of ≥50×10^9/L (up to Week 25)
Title
Percentage of Participants With Clinically Meaningful Platelet Response of ≥50×10^9/L by Day 8
Description
Clinically meaningful platelet response was defined as platelet count of ≥50×10^9/L.
Time Frame
Baseline to Day 8
Title
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
Description
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 Frame
From Baseline during Treatment Period (up to Week 25)
Title
Time to First Rescue Therapy
Description
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. The probability of requiring rescue medication did not reach 0.5 so the KM median in the rozanolixizumab arm could not be estimated.
Time Frame
From Baseline to first rescue therapy (up to Week 25)
Title
Change From Baseline to Week 25 in Primary Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score
Description
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.
Time Frame
From Baseline during Treatment Period (up to Week 25)
Title
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
Description
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.
Time Frame
From Baseline to end of Safety Follow-Up Period (up to Week 31)
Title
Percentage of Participants With TEAEs Leading to Withdrawal of Investigational Medicinal Product (ie, Study Discontinuation)
Description
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.
Time Frame
From Baseline to end of Safety Follow-Up Period (up to Week 31)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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:
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
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, 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)
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
UCB Cares
Organizational Affiliation
001 844 599 2273
Official's Role
Study Director
Facility Information:
Facility Name
Tp0003 50244
City
Seattle
State/Province
Washington
ZIP/Postal Code
98104
Country
United States
Facility Name
Tp0003 40188
City
Sofia
Country
Bulgaria
Facility Name
Tp0003 40197
City
Amiens
Country
France
Facility Name
Tp0003 40196
City
Pessac Cedex
Country
France
Facility Name
Tp0003 20050
City
Tbilisi
Country
Georgia
Facility Name
Tp0003 40558
City
Thessaloniki
Country
Greece
Facility Name
Tp0003 40202
City
Győr
Country
Hungary
Facility Name
Tp0003 40178
City
Nyíregyháza
Country
Hungary
Facility Name
Tp0003 40204
City
Pécs
Country
Hungary
Facility Name
Tp0003 40208
City
Firenze
Country
Italy
Facility Name
Tp0003 20030
City
Chuo-ku
Country
Japan
Facility Name
Tp0003 20039
City
Iruma-gun
Country
Japan
Facility Name
Tp0003 20159
City
Shibuya-ku
Country
Japan
Facility Name
Tp0003 20218
City
Daegu
Country
Korea, Republic of
Facility Name
Tp0003 20207
City
Seoul
Country
Korea, Republic of
Facility Name
Tp0003 20051
City
Chisinau
Country
Moldova, Republic of
Facility Name
Tp0003 40218
City
Gdansk
Country
Poland
Facility Name
Tp0003 40221
City
Lodz
Country
Poland
Facility Name
Tp0003 40222
City
Skorzewo
Country
Poland
Facility Name
Tp0003 40226
City
Bucharest
Country
Romania
Facility Name
Tp0003 40225
City
Bucuresti
Country
Romania
Facility Name
Tp0003 20055
City
Sankt-peterburg
Country
Russian Federation
Facility Name
Tp0003 20099
City
Taipei
Country
Taiwan
Facility Name
Tp0003 20060
City
Dnipropetrovsk
Country
Ukraine
Facility Name
Tp0003 20062
City
Ivano-frankivsk
Country
Ukraine
Facility Name
Tp0003 20063
City
Kyiv
Country
Ukraine
Facility Name
Tp0003 20100
City
Zaporizhzhia
Country
Ukraine
Facility Name
Tp0003 40238
City
London
Country
United Kingdom
Facility Name
Tp0003 40234
City
Plymouth
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing 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.
IPD Sharing 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.
IPD Sharing 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.
IPD Sharing URL
https://www.Vivli.org
Links:
URL
http://www.fda.gov/Safety/MedWatch/SafetyInformation/default.htm
Description
FDA Safety Alerts and Recalls
Learn more about this trial
A Study to Evaluate the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)
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