Polyneuropathy, Inflammatory Demyelinating, Chronic
Conditions
Brief summary
This study is an observational, ambispective, descriptive, non-interventional study of people with a chronic inflammatory demyelinating polyneuropathy/polyradiculoneuropathy (CIDP) diagnosis in the United States with residual impairment, disability, or neurological deficits after at least three months of treatment with standard of care therapy. The study is expected to last two years. Enrollment is expected to continue for one year. Depending on when the participant is enrolled, a participant can be followed for between one and two years, through the end of study, approximately two years after the study starts.
Interventions
None listed
Sponsors
Sanofi
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Participants are eligible to be included in the study only if all of the following criteria apply: * Neurologist-confirmed diagnosis of CIDP found in the medical record, with the last neurologist visit prior to enrollment containing no information that suggests this diagnosis was reversed * Active use of at least one of the following CIDP treatments for three months or longer, with no evidence of discontinuation of this therapy as of the last neurologist visit prior to enrollment * immunoglobulin * corticosteroids, with the exception of prednisone (or equivalent) monotherapy at 10mg or less per day * plasma exchange * efgartigimod alfa * azathioprine * mycophenolate mofetil * cyclosporine * rituximab * methotrexate * Signed informed consent * Residual impairment, disability, or neurological deficits at enrollment, as defined by a raw I-RODS score of 44 or below
Exclusion criteria
Participants are excluded from the study if any of the following criteria apply: * Evidence of participation in any interventional clinical trial with an investigational drug at the time of enrollment * Hyperreflexia (increased reflexes) recorded in the medical record during a neurological exam the year before enrollment and after CIDP diagnosis * Aged under 18 at the time of enrollment The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| I-RODS score at baseline | Baseline | The inflammatory Rasch-built overall disability scale (I-RODS) is a 24-item patient-reported questionnaire that captures disease impact on daily tasks with a score ranging from 0 (maximum disability) to 48 (no disability). It contains 24 items across an activity domain and a social participation domain. Each item is rated as "0": Not possible to perform; "1": possible, but with some difficulty, or "2" possible, without any difficulty. |
| Variations of I-RODS score over the study duration | From Baseline up to End of Study (2 years) | Difference in I-RODS between baseline and: 6 months after enrollment, 12 months after enrollment, end of study. The inflammatory Rasch-built overall disability scale (I-RODS) is a 24-item patient-reported questionnaire that captures disease impact on daily tasks with a score ranging from 0 (maximum disability) to 48 (no disability). It contains 24 items across an activity domain and a social participation domain. Each item is rated as "0": Not possible to perform; "1": possible, but with some difficulty, or "2" possible, without any difficulty. |
| Variations of I-RODS score after treatment change | From baseline up to End of Study (approx. 2 years) | Difference in I-RODS tests between the most recent assessments prior to and subsequent to a change in treatment class. The inflammatory Rasch-built overall disability scale (I-RODS) is a 24-item patient-reported questionnaire that captures disease impact on daily tasks with a score ranging from 0 (maximum disability) to 48 (no disability). It contains 24 items across an activity domain and a social participation domain. Each item is rated as "0": Not possible to perform; "1": possible, but with some difficulty, or "2" possible, without any difficulty. |
| Annualized I-RODS response rate | Up to End of Study (approx. 2 years) | Percent of patients who experienced inflammatory Rasch-built overall disability scale (I-RODS) response over a given time period, normalized to 365 days |
| Annualized I-RODS relapse rate | Up to End of Study (approx. 2 years) | Percent of patients who experienced inflammatory Rasch-built overall disability scale (I-RODS) relapse over a given time period, normalized to 365 days |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Adjusted INCAT (aINCAT) score at baseline and its variations over the study duration and after treatment change | Baseline and throughout the study (up to 2 years) | The adjusted score is identical to the inflammatory neuropathy cause and treatment (INCAT) disability score, except for the exclusion of changes in the upper-limb function from 0 (normal) to 1 (minor symptoms or signs in 1 or both arms, but not affecting any of the functions listed in the scale), or from 1 to 0. |
| Annualized aINCAT response rate | Up to End of Study (approx. 2 years) | Percent of participants who experienced inflammatory neuropathy cause and treatment (INCAT) response over a given time period, normalized to 365 days |
| Annualized aINCAT relapse rate | Up to End of Study (approx. 2 years) | Percent of pparticipantss who experienced inflammatory neuropathy cause and treatment (INCAT) relapse over a given time period, normalized to 365 days |
| Interobserver variability of remote INCAT assessment | Up to End of Study (approx. 2 years) | Intraclass correlation coefficient (ICC) will be used to evaluate interobserver variability. ICC value can range from 0 to 1. Values less than 0.5 are indicative of poor reliability, values between 0.5 and 0.75 indicate moderate reliability, values between 0.75 and 0.9 indicate good reliability, and values greater than 0.90 indicate excellent reliability. |
| Incidence of select comorbidities at baseline and over the study duration | From Baseline up to End of Study (approx. 2 years) | Number of participants with comorbidity events |
| Frequency of select comorbidities at baseline and over the study duration | From Baseline up to End of Study (approx. 2 years) | Events per patient-year |
| Laboratory measurements at baseline and over study duration | From Baseline up to End of Study (approx. 2 years) | Complete Blood Count (CBC), serum lipid profile, liver function tests |
| CAP-PRI score at baseline and its variations over the study duration and after treatment change | From Baseline to End of Study (approx 2 years) | The chronic acquired polyneuropathy- patient reported index \[CAP-PRI(8)\] is a participant reported outcome (PRO) that was developed to capture quality of life in people living with neuropathies. It contains 15 items, with a lower score indicating higher quality of life. Each item is rated as "0": not at all; "1": a little bit, or "2" a lot. |
Countries
United States
Contacts
CONTACTTrial Transparency email recommended (Toll free for US & Canada)
CONTACTPicnicHealth For potential study participants
Outcome results
None listed