Trial Outcomes & Findings for A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP) (NCT NCT04596995)

Last Updated: 2024-02-05

Results Overview

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.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

43 participants

Primary outcome timeframe

From Baseline to end of Safety Follow-Up Period (up to Week 60)

Results posted on

2024-02-05

Participant Flow

The study started to enroll study participants in January 2021 and completed prematurely in December 2022. Study participants from TP0003 (NCT04200456) or TP0006 (NCT04224688) who had completed the 24-week Treatment Period (irrespective of rescue therapy) and met eligibility criteria for TP0004 were enrolled in this study.

Participant Flow refers to the Enrolled Set.

Participant milestones

Participant milestones
Measure	Rozanolixizumab In TP0004, participants received a fixed unit dose of rozanolixizumab subcutaneous (sc) infusion at the assigned dose level in the parent studies (TP0003 and TP0006), for one year, starting from Day 1 (which corresponds to Week 25 of the parent studies). The dose of rozanolixizumab could be increased or decreased based upon the platelet count and across body weight tiers.
Overall Study STARTED	43
Overall Study COMPLETED	14
Overall Study NOT COMPLETED	29

Reasons for withdrawal

Reasons for withdrawal
Measure	Rozanolixizumab In TP0004, participants received a fixed unit dose of rozanolixizumab subcutaneous (sc) infusion at the assigned dose level in the parent studies (TP0003 and TP0006), for one year, starting from Day 1 (which corresponds to Week 25 of the parent studies). The dose of rozanolixizumab could be increased or decreased based upon the platelet count and across body weight tiers.
Overall Study Lack of Efficacy	8
Overall Study Lost to Follow-up	3
Overall Study Withdrawal by Subject	12
Overall Study Sponsor decision	3
Overall Study Enrolled in managed access program	3

Baseline Characteristics

A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Rozanolixizumab n=43 Participants In TP0004, participants received a fixed unit dose of rozanolixizumab sc infusion at the assigned dose level in the parent studies (TP0003 and TP0006), for one year, starting from Day 1 (which corresponds to Week 25 of the parent studies). The dose of rozanolixizumab could be increased or decreased based upon the platelet count and across body weight tiers.
Age, Continuous	42.7 years STANDARD_DEVIATION 13.7 • n=5 Participants
Age, Customized 18 - <65 years	41 Participants n=5 Participants
Age, Customized >=65 - <85 years	2 Participants n=5 Participants
Age, Customized >=85 years	0 Participants n=5 Participants
Sex: Female, Male Female	28 Participants n=5 Participants
Sex: Female, Male Male	15 Participants n=5 Participants
Race/Ethnicity, Customized Asian	8 Participants n=5 Participants
Race/Ethnicity, Customized White	33 Participants n=5 Participants
Race/Ethnicity, Customized Other or Mixed	1 Participants n=5 Participants
Race/Ethnicity, Customized Missing	1 Participants n=5 Participants
Race/Ethnicity, Customized Hispanic or Latino	2 Participants n=5 Participants
Race/Ethnicity, Customized Not Hispanic or Latino	41 Participants n=5 Participants

PRIMARY outcome

Timeframe: From Baseline to end of Safety Follow-Up Period (up to Week 60)

Population: Safety Set included all study participants who received at least 1 dose of IMP (partial or full).

Outcome measures

Outcome measures
Measure	Rozanolixizumab n=43 Participants In TP0004, participants received a fixed unit dose of rozanolixizumab sc infusion at the assigned dose level in the parent studies (TP0003 and TP0006), for one year, starting from Day 1 (which corresponds to Week 25 of the parent studies). The dose of rozanolixizumab could be increased or decreased based upon the platelet count and across body weight tiers.
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)	90.7 percentage of Participants

PRIMARY outcome

Timeframe: From Baseline to end of Safety Follow-Up Period (up to Week 60)

Population: Safety Set included all study participants who received at least 1 dose of IMP (partial or full).

Outcome measures

Outcome measures
Measure	Rozanolixizumab n=43 Participants In TP0004, participants received a fixed unit dose of rozanolixizumab sc infusion at the assigned dose level in the parent studies (TP0003 and TP0006), for one year, starting from Day 1 (which corresponds to Week 25 of the parent studies). The dose of rozanolixizumab could be increased or decreased based upon the platelet count and across body weight tiers.
Percentage of Participants With TEAEs Leading to Permanent Withdrawal of Rozanolixizumab (ie, Study Discontinuation)	0 percentage of Participants

SECONDARY outcome

Timeframe: Over the 52-week Treatment Period (starting at Week 4)

Population: Safety Set included all study participants who received at least 1 dose of IMP (partial or full).

Stable Clinically Meaningful Response was 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.

Outcome measures

Outcome measures
Measure	Rozanolixizumab n=43 Participants In TP0004, participants received a fixed unit dose of rozanolixizumab sc infusion at the assigned dose level in the parent studies (TP0003 and TP0006), for one year, starting from Day 1 (which corresponds to Week 25 of the parent studies). The dose of rozanolixizumab could be increased or decreased based upon the platelet count and across body weight tiers.
Percentage of Participants With Stable Clinically Meaningful Response Without Rescue Therapy at ≥70% of the Visits Over the Planned 52-week Treatment Period Starting at Week 4	16.3 percentage of participants

SECONDARY outcome

Timeframe: Week 53 or 55, compared to Baseline

Population: Safety Set included all study participants who received at least 1 dose of IMP (partial or full). Here, number of participants analyzed signifies those who were evaluable for this outcome measure and number analyzed signifies participants who were evaluable at specified time points. Week 53 was used for study participants who finished the study on weekly dosing, and Week 55 was used for study participants who finished the study on biweekly dosing.

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.

Outcome measures

Outcome measures
Measure	Rozanolixizumab n=12 Participants In TP0004, participants received a fixed unit dose of rozanolixizumab sc infusion at the assigned dose level in the parent studies (TP0003 and TP0006), for one year, starting from Day 1 (which corresponds to Week 25 of the parent studies). The dose of rozanolixizumab could be increased or decreased based upon the platelet count and across body weight tiers.
Change From Baseline in Immune Thrombocytopenia-Patient Assessment Questionnaire (ITP-PAQ) to Week 53 or 55 Symptoms Domain Score Week 53	2.98 score on a scale Standard Deviation 11.21
Change From Baseline in Immune Thrombocytopenia-Patient Assessment Questionnaire (ITP-PAQ) to Week 53 or 55 Symptoms Domain Score Week 55	4.17 score on a scale Standard Deviation 12.50

Adverse Events

Rozanolixizumab

Serious events: 9 serious events

Other events: 31 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Rozanolixizumab n=43 participants at risk In TP0004, participants received a fixed unit dose of rozanolixizumab sc infusion at the assigned dose level in the parent studies (TP0003 and TP0006), for one year, starting from Day 1 (which corresponds to Week 25 of the parent studies). The dose of rozanolixizumab could be increased or decreased based upon the platelet count and across body weight tiers.
Blood and lymphatic system disorders Immune thrombocytopenia	2.3% 1/43 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
General disorders Pyrexia	2.3% 1/43 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
Infections and infestations COVID-19	4.7% 2/43 • Number of events 2 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
Injury, poisoning and procedural complications Joint dislocation	2.3% 1/43 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
Injury, poisoning and procedural complications Radius fracture	2.3% 1/43 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
Investigations Platelet count decreased	2.3% 1/43 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Leiomyoma	2.3% 1/43 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
Reproductive system and breast disorders Uterine haemorrhage	4.7% 2/43 • Number of events 2 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
Skin and subcutaneous tissue disorders Purpura	2.3% 1/43 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
Skin and subcutaneous tissue disorders Skin haemorrhage	2.3% 1/43 • Number of events 2 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
Vascular disorders Haemorrhage	2.3% 1/43 • Number of events 1 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.

Other adverse events

Other adverse events
Measure	Rozanolixizumab n=43 participants at risk In TP0004, participants received a fixed unit dose of rozanolixizumab sc infusion at the assigned dose level in the parent studies (TP0003 and TP0006), for one year, starting from Day 1 (which corresponds to Week 25 of the parent studies). The dose of rozanolixizumab could be increased or decreased based upon the platelet count and across body weight tiers.
Blood and lymphatic system disorders Anaemia	7.0% 3/43 • Number of events 11 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
Gastrointestinal disorders Diarrhoea	11.6% 5/43 • Number of events 5 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
Gastrointestinal disorders Nausea	11.6% 5/43 • Number of events 9 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
Gastrointestinal disorders Vomiting	7.0% 3/43 • Number of events 7 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
General disorders Fatigue	11.6% 5/43 • Number of events 6 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
General disorders Pyrexia	20.9% 9/43 • Number of events 26 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
Infections and infestations COVID-19	16.3% 7/43 • Number of events 8 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
Infections and infestations Pharyngitis	9.3% 4/43 • Number of events 4 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
Infections and infestations Upper respiratory tract infection	7.0% 3/43 • Number of events 4 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
Injury, poisoning and procedural complications Contusion	7.0% 3/43 • Number of events 3 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
Investigations Body temperature increased	11.6% 5/43 • Number of events 8 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
Musculoskeletal and connective tissue disorders Pain in extremity	7.0% 3/43 • Number of events 3 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
Nervous system disorders Headache	41.9% 18/43 • Number of events 58 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
Skin and subcutaneous tissue disorders Petechiae	7.0% 3/43 • Number of events 3 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.
Skin and subcutaneous tissue disorders Rash	7.0% 3/43 • Number of events 4 • From Baseline to end of Safety Follow-Up Period (up to Week 60) Treatment-emergent AEs are defined as AEs starting after the time of first investigational medicinal product (IMP) administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.

Additional Information

UCB

Cares

Phone: 001 844 599 2273

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: GT60