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

NCT03861481 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: CIDP012016-002411-17
• Study Type: interventional
• Target: 34
• Locations: 8 countries
• Sites: 22

Brief Summary

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

Conditions

Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)

Keywords

Chronic inflammatory demyelinating polyradiculoneuropathy; CIDP; UCB7665; rozanolixizumab

Outcome Measures

Primary Outcomes (1)

• 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)

Study Arms (2)

Rozanolixizumab

EXPERIMENTAL

Subjects will be randomized to receive predefined subcutaneous doses of rozanolixizumab at a specified frequency

Drug: Rozanolixizumab

Placebo

PLACEBO COMPARATOR

Subjects will be randomized to receive predefined subcutaneous doses of placebo at a specified frequency

Other: Placebo

Interventions

Rozanolixizumab DRUG

Subjects will receive rozanolixizumab in a specified sequence during the treatment period.

Rozanolixizumab

Placebo OTHER

Subjects will receive placebo in a specified sequence during the treatment period.

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• UCB Biopharma S.P.R.L.: lead

Study Sites (22)

Cidp01 902

Phoenix, Arizona, 85018, United States

Location

Cidp01 905

Los Angeles, California, 90033, United States

Location

Cidp01 901

Tampa, Florida, 33612, United States

Location

Cidp01 907

Augusta, Georgia, 30912, United States

Location

Cidp01 911

Lexington, Kentucky, 40536, United States

Location

Cidp01 903

Charlotte, North Carolina, 28210, United States

Location

Cidp01 912

Durham, North Carolina, 27710, United States

Location

Cidp01 101

Ghent, Belgium

Location

Cidp01 102

Leuven, Belgium

Location

Cidp01 103

Liège, Belgium

Location

Cidp01 302

Copenhagen, Denmark

Location

Cidp01 402

Bordeaux, France

Location

Cidp01 404

Nice, France

Location

Cidp01 401

Strasbourg, France

Location

Cidp01 501

Berlin, Germany

Location

Cidp01 503

Essen, Germany

Location

Cidp01 505

Göttingen, Germany

Location

Cidp01 502

Würzburg, Germany

Location

Cidp01 601

Amsterdam, Netherlands

Location

Cidp01 701

Barcelona, Spain

Location

Cidp01 702

Barcelona, Spain

Location

Cidp01 802

Sheffield, United Kingdom

Location

MeSH Terms

Conditions

Polyradiculoneuropathy, Chronic Inflammatory Demyelinating

Interventions

rozanolixizumab

Condition Hierarchy (Ancestors)

Polyradiculoneuropathy; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: UCB
• Organization: Cares

UCBCares@ucb.com

001-844-599-2273

Study Officials

• UCB Cares

  +1 844 599 2273 (UCB)

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: March 1, 2019
• First Posted: March 4, 2019
• Study Start: March 26, 2019
• Primary Completion: March 31, 2021
• Study Completion: March 31, 2021
• Last Updated: August 1, 2023
• Results First Posted: April 4, 2022

Record last verified: 2023-07

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• 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.
• 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.

Locations

BE (3); US (7); DK (1); GB (1); FR (3); DE (4); ES (2); NL (1)