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

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ClinicalTrials.gov Identifier: NCT03861481

Recruitment Status : Completed

First Posted : March 4, 2019

Results First Posted : April 4, 2022

Last Update Posted : August 1, 2023

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Sponsor:

Information provided by (Responsible Party):

UCB Pharma ( UCB Biopharma S.P.R.L. )

Study Type	Interventional
Study Design	Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
Condition	Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
Interventions	Drug: Rozanolixizumab Other: Placebo
Enrollment	34

Participant Flow

Recruitment Details	The study started to enroll study participants in March 2019 and concluded in March 2021.
Pre-assignment Details	Participant Flow refers to the Randomized Set.


Arm/Group Title	Placebo	Rozanolixizumab
Arm/Group Description	Participants received placebo match...	Participants received rozanolixizum...
Arm/Group Description	Participants received placebo matched to rozanolixizumab as a subcutaneous injection once weekly for 12 weeks.	Participants received rozanolixizumab Dose A as a subcutaneous injection once weekly for 12 weeks.
Period Title: Overall Study
Started	17	17
Completed	11	10
Not Completed	6	7
Reason Not Completed
COVID-19 pandemic circumstances	0	1
Participant not plan to participate in the study CIDP04	1	0
Relapse	1	0
Withdrawal by Subject	0	1
Lack of Efficacy	4	5

Baseline Characteristics

Arm/Group Title		Placebo	Rozanolixizumab	Total
Arm/Group Description		Participants received placebo match...	Participants received rozanolixizum...	Total of all reporting groups
Arm/Group Description		Participants received placebo matched to rozanolixizumab as a subcutaneous injection once weekly for 12 weeks.	Participants received rozanolixizumab Dose A as a subcutaneous injection once weekly for 12 weeks.	Total of all reporting groups
Overall Number of Baseline Participants		17	17	34
Baseline Analysis Population Description		...
Baseline Analysis Population Description		The Randomized Set consisted of all participants randomized into the study.
Age, Categorical Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	17 participants	17 participants	34 participants
	<=18 years	0 0.0%	0 0.0%	0 0.0%
	Between 18 and 65 years	16 94.1%	12 70.6%	28 82.4%
	>=65 years	1 5.9%	5 29.4%	6 17.6%
Age, Continuous Mean (Standard Deviation) Unit of measure: Years
	Number Analyzed	17 participants	17 participants	34 participants
		56.4 (7.4)	57.3 (13.3)	56.8 (10.6)
Sex: Female, Male Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	17 participants	17 participants	34 participants
	Female	8 47.1%	8 47.1%	16 47.1%
	Male	9 52.9%	9 52.9%	18 52.9%
Race/Ethnicity, Customized Measure Type: Count of Participants Unit of measure: Participants	Number Analyzed	17 participants	17 participants	34 participants
Asian		1 5.9%	0 0.0%	1 2.9%
Black		1 5.9%	1 5.9%	2 5.9%
White		11 64.7%	15 88.2%	26 76.5%
Other/mixed		4 23.5%	1 5.9%	5 14.7%

Outcome Measures

1.Primary Outcome


Title	Change From Baseline to Week 13 (Day 85) in Inflammatory Rasch-built Overall Disability Scale (iRODS) Score
Description	iRODS is a linearly weighted patient-reported outcome measure (questio...
Description	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.
Time Frame	From Baseline up to Week 13 (Day 85)

Outcome Measure Data

Analysis Population Description

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).


Arm/Group Title	Placebo	Rozanolixizumab
Arm/Group Description:	Participants received placebo match...	Participants received rozanolixizum...
Arm/Group Description:	Participants received placebo matched to rozanolixizumab as a subcutaneous injection once weekly for 12 weeks.	Participants received rozanolixizumab Dose A as a subcutaneous injection once weekly for 12 weeks.
Overall Number of Participants Analyzed	17	16
Least Squares Mean (Standard Error) Unit of Measure: score on a scale (logits)
	0.234 (0.379)	0.181 (0.468)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Placebo, Rozanolixizumab
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Method of Estimation	Estimation Parameter	LS Mean difference (Rozimab - Placebo)
	Estimated Value	-0.052
	Confidence Interval	(2-Sided) 90% -0.892 to 0.788
	Estimation Comments	MMRM analysis (fixed effect terms: treatment, baseline iRODS score, prior Ig therapy administration route, assessment week, treatment by week interaction; random effect term: study participant). LS Mean Difference > 0 favours rozanolixizumab.

Adverse Events

Time Frame	From Baseline until the Safety Follow-up Visit (up to Week 24)
Adverse Event Reporting Description	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.

Arm/Group Title	Placebo		Rozanolixizumab
Arm/Group Description	Participants received placebo match...		Participants received rozanolixizum...
Arm/Group Description	Participants received placebo matched to rozanolixizumab as a subcutaneous injection once weekly for 12 weeks.		Participants received rozanolixizumab Dose A as a subcutaneous injection once weekly for 12 weeks.
All-Cause Mortality
	Placebo		Rozanolixizumab
	Affected / at Risk (%)		Affected / at Risk (%)
Total	0/17 (0.00%)		0/17 (0.00%)
Serious Adverse Events Serious Adverse Events
	Placebo		Rozanolixizumab
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/17 (0.00%)		2/17 (11.76%)
Nervous system disorders
Chronic inflammatory demyelinating polyradiculoneuropathy ^* 1	0/17 (0.00%)	0	2/17 (11.76%)	2
1 Term from vocabulary, MedDRA v24.0 * Indicates events were collected by non-systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	Placebo		Rozanolixizumab
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	14/17 (82.35%)		15/17 (88.24%)
Gastrointestinal disorders
Diarrhoea ^* 1	3/17 (17.65%)	5	2/17 (11.76%)	2
Nausea ^* 1	2/17 (11.76%)	6	0/17 (0.00%)	0
General disorders
Infusion site erythema ^* 1	0/17 (0.00%)	0	3/17 (17.65%)	4
Peripheral swelling ^* 1	0/17 (0.00%)	0	3/17 (17.65%)	3
Fatigue ^* 1	3/17 (17.65%)	3	0/17 (0.00%)	0
Pain ^* 1	2/17 (11.76%)	2	0/17 (0.00%)	0
Infections and infestations
Urinary tract infection ^* 1	3/17 (17.65%)	4	0/17 (0.00%)	0
Injury, poisoning and procedural complications
Fall ^* 1	2/17 (11.76%)	2	0/17 (0.00%)	0
Investigations
Bacterial test positive ^* 1	0/17 (0.00%)	0	2/17 (11.76%)	2
Musculoskeletal and connective tissue disorders
Arthralgia ^* 1	0/17 (0.00%)	0	3/17 (17.65%)	3
Nervous system disorders
Headache ^* 1	5/17 (29.41%)	10	6/17 (35.29%)	9
Chronic inflammatory demyelinating polyradiculoneuropathy ^* 1	4/17 (23.53%)	4	4/17 (23.53%)	4
Hypoaesthesia ^* 1	2/17 (11.76%)	2	0/17 (0.00%)	0
Skin and subcutaneous tissue disorders
Rash ^* 1	2/17 (11.76%)	3	0/17 (0.00%)	0
1 Term from vocabulary, MedDRA v24.0 * Indicates events were collected by non-systematic assessment

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact

Layout table for Results Point of Contact information

Name/Title:	UCB
Organization:	Cares
Phone:	001-844-599-2273
EMail:	UCBCares@ucb.com

Layout table for additonal information

Responsible Party:	UCB Pharma ( UCB Biopharma S.P.R.L. )
ClinicalTrials.gov Identifier:	NCT03861481
Other Study ID Numbers:	CIDP01 2016-002411-17 ( EudraCT Number )
First Submitted:	March 1, 2019
First Posted:	March 4, 2019
Results First Submitted:	March 9, 2022
Results First Posted:	April 4, 2022
Last Update Posted:	August 1, 2023

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