Trial Outcomes & Findings for A Study to Assess Long-term Safety, Tolerability and Efficacy of Rozanolixizumab in Subjects With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (NCT NCT04051944)

NCT ID: NCT04051944

Last Updated: 2022-11-03

Results Overview

An Adverse Event (AE) is any untoward medical occurrence in a participant or clinical investigation subject administered a pharmaceutical product, which does not necessarily had a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. A TEAE was defined as any event that was not present prior the first administration of investigational medicinal product (IMP) in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

21 participants

Primary outcome timeframe

From Baseline until Follow-Up Visit (up to Week 84)

Results posted on

2022-11-03

Participant Flow

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

Participant Flow refers to the Enrolled Set. Participants from parent study CIDP01 (NCT03861481) who had completed the Treatment Period without a relapse of chronic inflammatory demyelinating polyradiculoneuropathy were directly enrolled into this study. Newly treated participants are participants treated with placebo in parent study CIDP01 (NCT03861481). Previously treated participants are participants treated with rozanolixizumab in parent study CIDP01 (NCT03861481).

Participant milestones

Participant milestones
Measure	Rozanolixizumab (Newly Treated) Participants received rozanolixizumab Dose A as a subcutaneous infusion once weekly up to Week 76.	Rozanolixizumab (Previously Treated) Participants received rozanolixizumab Dose A as a subcutaneous infusion once weekly up to Week 76.
Overall Study STARTED	11	10
Overall Study COMPLETED	3	6
Overall Study NOT COMPLETED	8	4

Reasons for withdrawal

Reasons for withdrawal
Measure	Rozanolixizumab (Newly Treated) Participants received rozanolixizumab Dose A as a subcutaneous infusion once weekly up to Week 76.	Rozanolixizumab (Previously Treated) Participants received rozanolixizumab Dose A as a subcutaneous infusion once weekly up to Week 76.
Overall Study Study ending	1	1
Overall Study Early study termination by participant	1	0
Overall Study Withdrawal by Subject	2	2
Overall Study Lack of Efficacy	3	0
Overall Study Adverse event, non- fatal	1	1

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Rozanolixizumab (Newly Treated) n=11 Participants Participants received rozanolixizumab Dose A as a subcutaneous infusion once weekly up to Week 76.	Rozanolixizumab (Previously Treated) n=10 Participants Participants received rozanolixizumab Dose A as a subcutaneous infusion once weekly up to Week 76.	Total n=21 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Age, Categorical Between 18 and 65 years	8 Participants n=39 Participants	6 Participants n=41 Participants	14 Participants n=35 Participants
Age, Categorical >=65 years	3 Participants n=39 Participants	4 Participants n=41 Participants	7 Participants n=35 Participants
Age, Continuous	59.8 years STANDARD_DEVIATION 5.1 • n=39 Participants	59.1 years STANDARD_DEVIATION 15.9 • n=41 Participants	59.5 years STANDARD_DEVIATION 11.3 • n=35 Participants
Sex: Female, Male Female	4 Participants n=39 Participants	6 Participants n=41 Participants	10 Participants n=35 Participants
Sex: Female, Male Male	7 Participants n=39 Participants	4 Participants n=41 Participants	11 Participants n=35 Participants
Race/Ethnicity, Customized Asian	1 Participants n=39 Participants	0 Participants n=41 Participants	1 Participants n=35 Participants
Race/Ethnicity, Customized White	8 Participants n=39 Participants	9 Participants n=41 Participants	17 Participants n=35 Participants
Race/Ethnicity, Customized Other/Mixed	0 Participants n=39 Participants	1 Participants n=41 Participants	1 Participants n=35 Participants
Race/Ethnicity, Customized Missing	2 Participants n=39 Participants	0 Participants n=41 Participants	2 Participants n=35 Participants
Race/Ethnicity, Customized Not Hispanic or Latino	9 Participants n=39 Participants	10 Participants n=41 Participants	19 Participants n=35 Participants

PRIMARY outcome

Timeframe: From Baseline until Follow-Up Visit (up to Week 84)

Population: The Safety Set (SS) consisted of all enrolled study participants who were administered at least one dose of rozanolixizumab in CIDP04.

Outcome measures

Outcome measures
Measure	Rozanolixizumab (Newly Treated) n=11 Participants Participants received rozanolixizumab Dose A as a subcutaneous infusion once weekly up to Week 76.	Rozanolixizumab (Previously Treated) n=10 Participants Participants received rozanolixizumab Dose A as a subcutaneous infusion once weekly up to Week 76.
Number of Participants With Treatment-emergent Adverse Event (TEAEs)	10 Participants	10 Participants

Adverse Events

Rozanolixizumab (Newly Treated)

Serious events: 2 serious events

Other events: 10 other events

Deaths: 0 deaths

Rozanolixizumab (Previously Treated)

Serious events: 2 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Rozanolixizumab (Newly Treated) n=11 participants at risk Participants received rozanolixizumab Dose A as a subcutaneous infusion once weekly up to Week 76.	Rozanolixizumab (Previously Treated) n=10 participants at risk Participants received rozanolixizumab Dose A as a subcutaneous infusion once weekly up to Week 76.
Infections and infestations Urinary tract infection	0.00% 0/11 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	10.0% 1/10 • Number of events 1 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Malignant melanoma	9.1% 1/11 • Number of events 1 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	0.00% 0/10 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.
Nervous system disorders Chronic inflammatory demyelinating polyradiculoneuropathy	0.00% 0/11 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	10.0% 1/10 • Number of events 1 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.
Nervous system disorders Sensory loss	9.1% 1/11 • Number of events 1 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	0.00% 0/10 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.

Other adverse events

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

Measure	Rozanolixizumab (Newly Treated) n=11 participants at risk Participants received rozanolixizumab Dose A as a subcutaneous infusion once weekly up to Week 76.	Rozanolixizumab (Previously Treated) n=10 participants at risk Participants received rozanolixizumab Dose A as a subcutaneous infusion once weekly up to Week 76.
Gastrointestinal disorders Diarrhoea	9.1% 1/11 • Number of events 2 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	10.0% 1/10 • Number of events 1 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.
Gastrointestinal disorders Constipation	0.00% 0/11 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	20.0% 2/10 • Number of events 2 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.
General disorders Fatigue	27.3% 3/11 • Number of events 3 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	30.0% 3/10 • Number of events 4 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.
General disorders Pyrexia	18.2% 2/11 • Number of events 2 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	0.00% 0/10 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.
General disorders Peripheral swelling	9.1% 1/11 • Number of events 1 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	10.0% 1/10 • Number of events 1 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.
Infections and infestations Influenza	18.2% 2/11 • Number of events 2 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	0.00% 0/10 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.
Infections and infestations Nasopharyngitis	18.2% 2/11 • Number of events 2 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	20.0% 2/10 • Number of events 2 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.
Infections and infestations Upper respiratory tract infection	18.2% 2/11 • Number of events 2 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	0.00% 0/10 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.
Infections and infestations Urinary tract infection	18.2% 2/11 • Number of events 2 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	0.00% 0/10 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.
Injury, poisoning and procedural complications Muscle strain	9.1% 1/11 • Number of events 1 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	20.0% 2/10 • Number of events 2 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.
Injury, poisoning and procedural complications Fall	0.00% 0/11 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	20.0% 2/10 • Number of events 4 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.
Investigations Blood immunoglobulin G decreased	36.4% 4/11 • Number of events 10 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	40.0% 4/10 • Number of events 8 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.
Musculoskeletal and connective tissue disorders Joint swelling	9.1% 1/11 • Number of events 1 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	20.0% 2/10 • Number of events 2 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.
Musculoskeletal and connective tissue disorders Muscle spasms	0.00% 0/11 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	20.0% 2/10 • Number of events 2 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.
Musculoskeletal and connective tissue disorders Muscular weakness	18.2% 2/11 • Number of events 4 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	0.00% 0/10 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.
Musculoskeletal and connective tissue disorders Back pain	9.1% 1/11 • Number of events 1 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	10.0% 1/10 • Number of events 1 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.
Nervous system disorders Headache	36.4% 4/11 • Number of events 7 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	10.0% 1/10 • Number of events 5 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.
Nervous system disorders Neuralgia	9.1% 1/11 • Number of events 1 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	20.0% 2/10 • Number of events 2 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.
Nervous system disorders Sensory loss	18.2% 2/11 • Number of events 3 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	0.00% 0/10 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	9.1% 1/11 • Number of events 1 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	10.0% 1/10 • Number of events 2 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.
Skin and subcutaneous tissue disorders Rash	9.1% 1/11 • Number of events 1 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.	10.0% 1/10 • Number of events 1 • From Baseline until Follow-Up Visit (up to Week 84) A TEAE was defined as any event that was not present prior the first administration of IMP in CIDP04 study or any unresolved event already present before the first administration of IMP in CIDP04 study that worsened in intensity following exposure to treatment until 8 weeks following the last administration of IMP in CIDP04 study.