Trial · NCT04200456
A Study to Evaluate the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)
NCT04200456, Phase 3, Terminated, UCB Biopharma SRL; results first posted 2023-08-08
3 sourcestrials-2026-11-12-0.4.0
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ConductScience Library. A Study to Evaluate the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP). Trials, record NCT04200456. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04200456
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Evaluate the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)
- NCT number
- NCT04200456
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 33 participants, actual
- Lead sponsor
- UCB Biopharma SRL
- Conditions
- Primary Immune Thrombocytopenia
- Interventions
- Rozanolixizumab, Placebo
- First posted
- 2019-12-16
- Last updated
- 2025-03-18
- Other registries' IDs
- eudract:2019-000884-26
- Results first posted
- 2023-08-08
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | 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 | 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. |
| Secondary | Cumulative Number of Weeks With Clinically Meaningful Platelet Response of ≥50×10^9/L Over the 24-week Treatment Period | 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. |
| Secondary | 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 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. |
| Secondary | Percentage of Participants With Clinically Meaningful Platelet Response of ≥50×10^9/L by Day 8 | Baseline to Day 8 | Clinically meaningful platelet response was defined as platelet count of ≥50×10\^9/L. |
| Secondary | 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 | 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. |
| Secondary | Time to First Rescue Therapy | 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. The probability of requiring rescue medication did not reach 0.5 so the KM median in the rozanolixizumab arm could not be estimated. |
| Secondary | Change From Baseline to Week 25 in Primary Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score | 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. |
| Secondary | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) | 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. |
| Secondary | Percentage of Participants With TEAEs Leading to Withdrawal of Investigational Medicinal Product (ie, Study Discontinuation) | 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. |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | 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 | During the last 12 weeks (Week 13 to Week 25) | Randomized Set consisted of all enrolled study participants who were randomized. No formal analysis was carried out as the program was terminated. | Percentage of participants | NUMBER |
| SECONDARY | Cumulative Number of Weeks With Clinically Meaningful Platelet Response of ≥50×10^9/L Over the 24-week Treatment Period | Week 1 up to Week 25 | Randomized Set consisted of all enrolled study participants who were randomized. | Weeks | MEDIAN |
| SECONDARY | 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 response of ≥50×10^9/L (up to Week 25) | Randomized Set consisted of all enrolled study participants who were randomized. | days | MEDIAN |
| SECONDARY | Percentage of Participants With Clinically Meaningful Platelet Response of ≥50×10^9/L by Day 8 | Baseline to Day 8 | Randomized Set consisted of all enrolled study participants who were randomized. | Percentage of participants | NUMBER |
| SECONDARY | 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 | From Baseline during Treatment Period (up to Week 25) | Randomized Set consisted of all enrolled study participants who were randomized. | Percentage of participants | NUMBER |
| SECONDARY | Time to First Rescue Therapy | From Baseline to first rescue therapy (up to Week 25) | Randomized Set consisted of all enrolled study participants who were randomized. | Days | MEDIAN |
| SECONDARY | Change From Baseline to Week 25 in Primary Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score | From Baseline during Treatment Period (up to Week 25) | Randomized Set consisted of all enrolled study participants who were randomized. Here, Number of Participants analyzed signifies those participants who were evaluable for this outcome measure. | units on a scale | MEAN |
| SECONDARY | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) | From Baseline to end of Safety Follow-Up Period (up to Week 31) | Safety set included all randomized study participants who received at least one dose of IMP. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With TEAEs Leading to Withdrawal of Investigational Medicinal Product (ie, Study Discontinuation) | From Baseline to end of Safety Follow-Up Period (up to Week 31) | Safety set included all randomized study participants who received at least one dose of IMP. | Percentage of participants | NUMBER |
Source ClinicalTrials.gov
Results by group18
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| 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 | Placebo | 0 | Percentage of participants | ||
| 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 | Rozanolixizumab | 19.0 | Percentage of participants | ||
| Cumulative Number of Weeks With Clinically Meaningful Platelet Response of ≥50×10^9/L Over the 24-week Treatment Period | Placebo | 0.0 | Full Range 0 to 7 | Weeks | |
| Cumulative Number of Weeks With Clinically Meaningful Platelet Response of ≥50×10^9/L Over the 24-week Treatment Period | Rozanolixizumab | 3.0 | Full Range 0 to 24 | Weeks | |
| 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 | Placebo | 44.0 | 95% Confidence Interval 6.0 to NA | days | |
| 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 | Rozanolixizumab | 8.0 | 95% Confidence Interval 6.0 to NA | days | |
| Percentage of Participants With Clinically Meaningful Platelet Response of ≥50×10^9/L by Day 8 | Placebo | 16.7 | Percentage of participants | ||
| Percentage of Participants With Clinically Meaningful Platelet Response of ≥50×10^9/L by Day 8 | Rozanolixizumab | 52.4 | Percentage of participants | ||
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 | Placebo | 8.3 | Percentage of participants | ||
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 | Rozanolixizumab | 33.3 | Percentage of participants |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Placebo | 12 | |
| Overall Study | STARTED | Rozanolixizumab | 21 | |
| Overall Study | COMPLETED | Placebo | 9 | |
| Overall Study | COMPLETED | Rozanolixizumab | 15 | |
| Overall Study | NOT COMPLETED | Placebo | 3 | |
| Overall Study | NOT COMPLETED | Rozanolixizumab | 6 |
Source ClinicalTrials.gov
Adverse events62
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Placebo | 2 | 12 | 2 |
| other | Anaemia | Blood and lymphatic system disorders | Rozanolixizumab | 3 | 21 | 12 |
| other | Tinnitus | Ear and labyrinth disorders | Placebo | 1 | 12 | 1 |
| other | Tinnitus | Ear and labyrinth disorders | Rozanolixizumab | 0 | 21 | 0 |
| other | Nausea | Gastrointestinal disorders | Placebo | 1 | 12 | 1 |
| other | Nausea | Gastrointestinal disorders | Rozanolixizumab | 5 | 21 | 6 |
| other | Vomiting | Gastrointestinal disorders | Placebo | 0 | 12 | 0 |
| other | Vomiting | Gastrointestinal disorders | Rozanolixizumab | 4 | 21 | 5 |
| other | Diarrhoea | Gastrointestinal disorders | Placebo | 2 | 12 | 2 |
| other | Diarrhoea | Gastrointestinal disorders | Rozanolixizumab | 2 | 21 | 9 |
1 to 10 of 62Source ClinicalTrials.gov
See all 62 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo | 0 | 12 |
| Total, all-cause mortality | Rozanolixizumab | 0 | 21 |
| Total, other adverse events | Placebo | 9 | 12 |
| Total, other adverse events | Rozanolixizumab | 16 | 21 |
| Total, serious adverse events | Placebo | 1 | 12 |
| Total, serious adverse events | Rozanolixizumab | 2 | 21 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 39552477 | RESULT |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Rozanolixizumab vs Placebo: deaths from any cause | Trial comparisons |
| Rozanolixizumab vs Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Source
- Release
- trials-2026-11-12-0.4.0
- Release manifest SHA-256
- 0f4a8d8c48aee2a7573ae0fa94ecbeab70c1adbe449bbc95c424bc7d3e97b9d4
- Registry capture
- 2026-11-12
- Results copy, AACT
- 2026-03-23
- NIH clinical studies file
- 2026-11-13
Source ClinicalTrials.gov
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNone stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04200456
GET https://conductscience.org/api/trials/v1/NCT04200456/events?page=2