Trial Outcomes & Findings for A Study to Assess the Efficacy, Safety and Tolerability of Rozanolixizumab in Subjects With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (NCT NCT03861481)

Last Updated: 2023-08-01

Results Overview

iRODS is a linearly weighted patient-reported outcome measure (questionnaire) that captures activity and social participation limitations in participants with chronic inflammatory demyelinating polyradiculoneuropathy. Questionnaire consisted of 24 items (including eating, taking a shower, walking a flight of stairs, standing for hours, etc.) and assesses a participant's ability to perform daily and social activities. Participants had 3 response options: 0=impossible to perform; 1=performed with difficulty; 2=easily performed, performed without difficulty. Raw sum scores of iRODS (range 0 to 48, where 0=worse and 48=best) were translated to log odds units (logits) scale, placing participant' estimates on same logit scale, which had a score range of -6.95 (most severe activity and social participation restrictions) to 8.11 (no activity and social participation limitations). A positive change is associated with a better outcome of less disease activity and more social activity.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

34 participants

Primary outcome timeframe

From Baseline up to Week 13 (Day 85)

Results posted on

2023-08-01

Participant Flow

The study started to enroll study participants in March 2019 and concluded in March 2021.

Participant Flow refers to the Randomized Set.

Participant milestones

Participant milestones
Measure	Placebo Participants received placebo matched to rozanolixizumab as a subcutaneous injection once weekly for 12 weeks.	Rozanolixizumab Participants received rozanolixizumab Dose A as a subcutaneous injection once weekly for 12 weeks.
Overall Study STARTED	17	17
Overall Study COMPLETED	11	10
Overall Study NOT COMPLETED	6	7

Reasons for withdrawal

Reasons for withdrawal
Measure	Placebo Participants received placebo matched to rozanolixizumab as a subcutaneous injection once weekly for 12 weeks.	Rozanolixizumab Participants received rozanolixizumab Dose A as a subcutaneous injection once weekly for 12 weeks.
Overall Study COVID-19 pandemic circumstances	0	1
Overall Study Participant not plan to participate in the study CIDP04	1	0
Overall Study Relapse	1	0
Overall Study Withdrawal by Subject	0	1
Overall Study Lack of Efficacy	4	5

Baseline Characteristics

A Study to Assess the Efficacy, Safety and Tolerability of Rozanolixizumab in Subjects With Chronic Inflammatory Demyelinating Polyradiculoneuropathy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Placebo n=17 Participants Participants received placebo matched to rozanolixizumab as a subcutaneous injection once weekly for 12 weeks.	Rozanolixizumab n=17 Participants Participants received rozanolixizumab Dose A as a subcutaneous injection once weekly for 12 weeks.	Total n=34 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	16 Participants n=5 Participants	12 Participants n=7 Participants	28 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants	5 Participants n=7 Participants	6 Participants n=5 Participants
Age, Continuous	56.4 years STANDARD_DEVIATION 7.4 • n=5 Participants	57.3 years STANDARD_DEVIATION 13.3 • n=7 Participants	56.8 years STANDARD_DEVIATION 10.6 • n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants	8 Participants n=7 Participants	16 Participants n=5 Participants
Sex: Female, Male Male	9 Participants n=5 Participants	9 Participants n=7 Participants	18 Participants n=5 Participants
Race/Ethnicity, Customized Asian	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race/Ethnicity, Customized Black	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race/Ethnicity, Customized White	11 Participants n=5 Participants	15 Participants n=7 Participants	26 Participants n=5 Participants
Race/Ethnicity, Customized Other/mixed	4 Participants n=5 Participants	1 Participants n=7 Participants	5 Participants n=5 Participants

PRIMARY outcome

Timeframe: From Baseline up to Week 13 (Day 85)

Population: The Full Analysis Set (FAS) consisted of all participants who received at least one dose of treatment and who had a Baseline and at least one valid post-Baseline iRODS measurement up to Week 13 (Day 85)/premature end of treatment (inclusively).

Outcome measures

Outcome measures
Measure	Placebo n=17 Participants Participants received placebo matched to rozanolixizumab as a subcutaneous injection once weekly for 12 weeks.	Rozanolixizumab n=16 Participants Participants received rozanolixizumab Dose A as a subcutaneous injection once weekly for 12 weeks.
Change From Baseline to Week 13 (Day 85) in Inflammatory Rasch-built Overall Disability Scale (iRODS) Score	0.234 score on a scale (logits) Standard Error 0.379	0.181 score on a scale (logits) Standard Error 0.468

Adverse Events

Placebo

Serious events: 0 serious events

Other events: 14 other events

Deaths: 0 deaths

Rozanolixizumab

Serious events: 2 serious events

Other events: 15 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Placebo n=17 participants at risk Participants received placebo matched to rozanolixizumab as a subcutaneous injection once weekly for 12 weeks.	Rozanolixizumab n=17 participants at risk Participants received rozanolixizumab Dose A as a subcutaneous injection once weekly for 12 weeks.
Nervous system disorders Chronic inflammatory demyelinating polyradiculoneuropathy	0.00% 0/17 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.	11.8% 2/17 • Number of events 2 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.

Other adverse events

Other adverse events
Measure	Placebo n=17 participants at risk Participants received placebo matched to rozanolixizumab as a subcutaneous injection once weekly for 12 weeks.	Rozanolixizumab n=17 participants at risk Participants received rozanolixizumab Dose A as a subcutaneous injection once weekly for 12 weeks.
Gastrointestinal disorders Diarrhoea	17.6% 3/17 • Number of events 5 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.	11.8% 2/17 • Number of events 2 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.
Gastrointestinal disorders Nausea	11.8% 2/17 • Number of events 6 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.	0.00% 0/17 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.
General disorders Infusion site erythema	0.00% 0/17 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.	17.6% 3/17 • Number of events 4 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.
General disorders Peripheral swelling	0.00% 0/17 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.	17.6% 3/17 • Number of events 3 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.
General disorders Fatigue	17.6% 3/17 • Number of events 3 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.	0.00% 0/17 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.
General disorders Pain	11.8% 2/17 • Number of events 2 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.	0.00% 0/17 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.
Infections and infestations Urinary tract infection	17.6% 3/17 • Number of events 4 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.	0.00% 0/17 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.
Injury, poisoning and procedural complications Fall	11.8% 2/17 • Number of events 2 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.	0.00% 0/17 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.
Investigations Bacterial test positive	0.00% 0/17 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.	11.8% 2/17 • Number of events 2 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.
Musculoskeletal and connective tissue disorders Arthralgia	0.00% 0/17 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.	17.6% 3/17 • Number of events 3 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.
Nervous system disorders Headache	29.4% 5/17 • Number of events 10 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.	35.3% 6/17 • Number of events 9 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.
Nervous system disorders Chronic inflammatory demyelinating polyradiculoneuropathy	23.5% 4/17 • Number of events 4 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.	23.5% 4/17 • Number of events 4 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.
Nervous system disorders Hypoaesthesia	11.8% 2/17 • Number of events 2 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.	0.00% 0/17 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.
Skin and subcutaneous tissue disorders Rash	11.8% 2/17 • Number of events 3 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.	0.00% 0/17 • From Baseline until the Safety Follow-up Visit (up to Week 24) A TEAE is defined as any event that was not present prior the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment until 8 weeks following the last administration of IMP.

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