Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
Conditions
Keywords
Chronic inflammatory demyelinating polyradiculoneuropathy, CIDP, UCB7665, rozanolixizumab
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).
Interventions
DRUGRozanolixizumab
Subjects will receive rozanolixizumab in a specified sequence during the treatment period.
OTHERPlacebo
Subjects will receive placebo in a specified sequence during the treatment period.
Sponsors
UCB Biopharma S.P.R.L.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline to Week 13 (Day 85) in Inflammatory Rasch-built Overall Disability Scale (iRODS) Score | From Baseline up to Week 13 (Day 85) | 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. |
Countries
Belgium, Denmark, France, Germany, Netherlands, Spain, United Kingdom, United States
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.
Participants by arm
| Arm | Count |
|---|---|
| Placebo Participants received placebo matched to rozanolixizumab as a subcutaneous injection once weekly for 12 weeks. | 17 |
| Rozanolixizumab Participants received rozanolixizumab Dose A as a subcutaneous injection once weekly for 12 weeks. | 17 |
| Total | 34 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | COVID-19 pandemic circumstances | 0 | 1 |
| Overall Study | Lack of Efficacy | 4 | 5 |
| 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 |
Baseline characteristics
| Characteristic | Placebo | Total | Rozanolixizumab |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 1 Participants | 6 Participants | 5 Participants |
| Age, Categorical Between 18 and 65 years | 16 Participants | 28 Participants | 12 Participants |
| Age, Continuous | 56.4 years STANDARD_DEVIATION 7.4 | 56.8 years STANDARD_DEVIATION 10.6 | 57.3 years STANDARD_DEVIATION 13.3 |
| Race/Ethnicity, Customized Asian | 1 Participants | 1 Participants | 0 Participants |
| Race/Ethnicity, Customized Black | 1 Participants | 2 Participants | 1 Participants |
| Race/Ethnicity, Customized Other/mixed | 4 Participants | 5 Participants | 1 Participants |
| Race/Ethnicity, Customized White | 11 Participants | 26 Participants | 15 Participants |
| Sex: Female, Male Female | 8 Participants | 16 Participants | 8 Participants |
| Sex: Female, Male Male | 9 Participants | 18 Participants | 9 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 17 | 0 / 17 |
| other Total, other adverse events | 14 / 17 | 15 / 17 |
| serious Total, serious adverse events | 0 / 17 | 2 / 17 |
Outcome results
Primary
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.
Time frame: 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).
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Placebo | 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 |
| Rozanolixizumab | Change From Baseline to Week 13 (Day 85) in Inflammatory Rasch-built Overall Disability Scale (iRODS) Score | 0.181 score on a scale (logits) | Standard Error 0.468 |
90% CI: [-0.892, 0.788]