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

A Multicenter, Randomized, Subject-Blind, Investigator-Blind, Placebo-Controlled, Parallel-Group Study Evaluating the Efficacy, Safety, and Tolerability of Rozanolixizumab in Subjects With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)

The purpose of the study is to evaluate clinical efficacy of rozanolixizumab as a treatment for subjects with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP).

Study Overview

• Status: Completed
• Conditions: Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
• Intervention / Treatment: Drug: Rozanolixizumab; Other: Placebo

• Study Type: Interventional
• Enrollment (Actual): 34
• Phase: Phase 2

Expanded Access

Available outside the clinical trial. See expanded access record.

Contacts and Locations

Study Locations

• Belgium
  • Gent, Belgium
    • Cidp01 101
  • Leuven, Belgium
    • Cidp01 102
  • Liège, Belgium
    • Cidp01 103
• Denmark
  • Copenhagen, Denmark
    • Cidp01 302
• France
  • Bordeaux, France
    • Cidp01 402
  • Nice, France
    • Cidp01 404
  • Strasbourg, France
    • Cidp01 401
• Germany
  • Berlin, Germany
    • Cidp01 501
  • Essen, Germany
    • Cidp01 503
  • Göttingen, Germany
    • Cidp01 505
  • Würzburg, Germany
    • Cidp01 502
• Netherlands
  • Amsterdam, Netherlands
    • Cidp01 601
• Spain
  • Barcelona, Spain
    • Cidp01 701
  • Barcelona, Spain
    • Cidp01 702
• United Kingdom
  • Sheffield, United Kingdom
    • Cidp01 802
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85018
      • Cidp01 902
  • California
    • Los Angeles, California, United States, 90033
      • Cidp01 905
  • Florida
    • Tampa, Florida, United States, 33612
      • Cidp01 901
  • Georgia
    • Augusta, Georgia, United States, 30912
      • Cidp01 907
  • Kentucky
    • Lexington, Kentucky, United States, 40536
      • Cidp01 911
  • North Carolina
    • Charlotte, North Carolina, United States, 28210
      • Cidp01 903
    • Durham, North Carolina, United States, 27710
      • Cidp01 912

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Subject is ≥ 18 years of age with a minimum body weight of ≥42 kg at Visit 1 (Screening)
• Subject has a documented definite or probable diagnosis of Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) according to the European Federation of Neurological Societies (EFNS)/ Peripheral Nerve Society (PNS) criteria 2010
• Subject has an immunoglobulin-dependency confirmed by clinical examination during therapy or upon interruption or reduction of therapy within 18 months prior to Screening and documented in medical history
• Subject is on a stable dosage (not more than ±20% deviation) for subcutaneous immunoglobulin (SCIg) or intravenous immunoglobulin (IVIg) and a fixed interval for at least 4 months of either treatment
• Female subjects of childbearing potential must agree to use a highly effective method of birth control, during the study and for a period of 3 months after their final dose of investigational medicinal product (IMP)
• Male subjects with a partner of childbearing potential must be willing to use a condom when sexually active during the study and for 3 months after the final administration of IMP

Exclusion Criteria:

• Previously received treatment in this study or subject has previously been exposed to rozanolixizumab
• Current diagnosis or has a history of Type 1 or Type 2 diabetes mellitus and/or hemoglobin A1c level >6.0 %
• Known immunoglobulin M (IgM)-mediated neuropathy
• Clinical or known evidence of associated systemic diseases that might cause neuropathy or treatment with agents that might lead to neuropathy
• History of clinically relevant ongoing chronic infections
• Family history of primary immunodeficiency
• Received a live vaccination within 8 weeks prior to the Baseline Visit; or intends to have a live vaccination during the course of the study or within 7 weeks following the final dose of IMP
• Received any experimental biological agent within or outside of a clinical study in the past 3 months or within 5 half-lives prior to Baseline
• Prior treatment with rituximab, ofatumumab, or ocrelizumab in the 6 months prior to the Baseline Visit or subject has had prior treatment with rituximab, ofatumumab, or ocrelizumab in the 12 months prior to Baseline and B cells are not within the normal range
• Female subject who is pregnant or lactating

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Rozanolixizumab; Subjects will be randomized to receive predefined subcutaneous doses of rozanolixizumab at a specified frequency	Drug: Rozanolixizumab; Subjects will receive rozanolixizumab in a specified sequence during the treatment period.
Placebo Comparator: Placebo; Subjects will be randomized to receive predefined subcutaneous doses of placebo at a specified frequency	Other: Placebo; Subjects will receive placebo in a specified sequence during the treatment period.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline to Week 13 (Day 85) in Inflammatory Rasch-built Overall Disability Scale (iRODS) Score	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.	From Baseline up to Week 13 (Day 85)

Collaborators and Investigators

• Sponsor: UCB Biopharma S.P.R.L.
• Investigators: Study Director: UCB Cares, +1 844 599 2273 (UCB)

Study record dates

Study Major Dates

• Study Start (Actual): March 26, 2019
• Primary Completion (Actual): March 31, 2021
• Study Completion (Actual): March 31, 2021

Study Registration Dates

• First Submitted: March 1, 2019
• First Submitted That Met QC Criteria: March 1, 2019
• First Posted (Actual): March 4, 2019

Study Record Updates

• Last Update Posted (Actual): August 1, 2023
• Last Update Submitted That Met QC Criteria: July 28, 2023
• Last Verified: July 1, 2023

More Information

Terms related to this study

• Keywords: rozanolixizumab; CIDP; Chronic inflammatory demyelinating polyradiculoneuropathy; UCB7665
• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Immune System Diseases; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Autoimmune Diseases; Disease Attributes; Neuromuscular Diseases; Peripheral Nervous System Diseases; Polyneuropathies; Chronic Disease; Polyradiculoneuropathy; Polyradiculoneuropathy, Chronic Inflammatory Demyelinating; Physiological Effects of Drugs; Immunosuppressive Agents; Immunologic Factors; Rozanolixizumab
• Other Study ID Numbers: CIDP01; 2016-002411-17 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.
• IPD Sharing Time Frame: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
• IPD Sharing Access Criteria: Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No