RWE-based Treatment Patterns and Outcomes in CIDP

NCT05940922 | Unknown

• 1 other identifier: RWH_LS_CIDPv2
• Study Type: observational
• Target: 170
• Locations: 1 country
• Sites: 1

Brief Summary

To describe the demographics, clinical characteristics, treatment patterns and clinical outcomes of chronic inflammatory demyelinating polyneuropathy (CIDP), Guillain-Barre Syndrome (GBS), and heredofamilial amyloidosis (hATTR) adult patients at a single U.K. centre.

Conditions

Chronic Inflammatory Demyelinating Polyneuropathy; Guillain-Barre Syndrome; Hereditary Amyloidosis

Keywords

Inflammatory polyneuropathies

Outcome Measures

Primary Outcomes (8)

• Patient baseline characteristics

  Patient demographics, clinical characteristics, weight (kg), and medical history.

  At diagnosis

• Grip strength test

  Average measurement of grip strength using dynamometer over 3 trials per hand, measured in kilograms of force.

  At diagnosis or pre-Ig treatment initiation.

• 9-hole peg test

  Average of four measurements (two per hand) in seconds, measured using stopwatch.

  At diagnosis or pre-Ig treatment initiation.

• 10 meter walk test

  Average speed in meters/second over 10 meters, measured using stopwatch.

  At diagnosis or pre-Ig treatment initiation.

• Overall neuropathy limitations scale

  The ONLS allows semi-objective measurement of function, which is useful to detect changes with therapy, and relatively earlier changes may be detected than on the standard five-point Medical Research Council scale used in routine examination of muscle power. The ONLS is graded separately for upper and lower limbs as follows: Arm scale score (0 to 5) + Leg scale score (0 to 7) for the total out of 12.

  At diagnosis or pre-Ig treatment initiation.

• Berg balance test

  The Berg balance uses a stopwatch, a ruler or a measuring tape, a chair, a step, and an object that can be picked up. It takes \~15 min to complete and includes 14 tasks scored 0-4 and scores are added for a total out of 56. Berg balance test scoring system: 0 to 20: Score - need the assistance of a wheelchair to move around safely 21 to 40: Score - need some type of walking assistance, cane or a walker 41 to 56: Score - considered independent and able to move around safely without assistance

  At diagnosis or pre-Ig treatment initiation.

• Medical research council muscle strength score

  The Medical Research Council (MRC) Scale for Muscle Strength is a commonly used scale for assessing muscle strength from Grade 5 (normal) to Grade 0 (no visible contraction) The Criteria requires that each of the six muscle groups listed below are examined bilaterally, each with a score from 0 to 5 according to the scale: Shoulder abductors, Elbow flexors, Wrist extensors, Hip flexors, Knee extensors, Foot dorsiflexors.

  At diagnosis or pre-Ig treatment initiation.

• Inflammatory neuropathy cause and treatment (INCAT) sensory sum (ISS) score

  The ISS score is inversely related to function, with 0 representing no functional impairment and 10 representing inability to make any purposeful movement with either arms or legs.

  At diagnosis or pre-Ig treatment initiation up to December 31, 2022 or death or lost-to-follow-up, whichever comes first.

Secondary Outcomes (4)

• Time to treatment initiation

  From diagnosis to day 1 of treatment

• Treatment patterns

  From the date of first immunoglobulin up to December 31, 2022 or death or lost-to-follow-up, whichever comes first.

• Treatment outcomes

  Treatment outcomes to be measured at 6 week intervals after the date of first immunoglobulin up to December 31, 2022 or death or lost-to-follow-up, whichever comes first.

• Relapse

  From 6 weeks after the date of first immunoglobulin treatment to the date of first documented relapse up to a maximum of 173 months of follow-up

Study Arms (1)

CIDP GBS hATTR

Adult patients diagnosed and treated at the study centre for chronic inflammatory demyelinating polyneuropathy (CIDP), or Guillain-Barre syndrome (GBS), or heredofamilial amyloidosis (hATTR).

Other: No intervention. Retrospective observation only.

Interventions

No intervention. Retrospective observation only. OTHER

No intervention. Retrospective observation only.

CIDP GBS hATTR

Eligibility Criteria

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

Study Population

Retrospective medical data review of patients diagnosed with chronic inflammatory demyelinating polyneuropathy (CIDP), Guillain-Barre syndrome (GBS), or heredofamilial amyloidosis (hATTR) at a single centre in the United Kingdom, and who received immunoglobulin treatment.

You may qualify if:

• Patients diagnosed or treated at the study centre with a diagnosis of chronic inflammatory demyelinating polyneuropathy (CIDP), Guillain-Barre syndrome (GBS), or heredofamilial amyloidosis (hATTR).
• Patients aged 18 years and over.
• Patients who did not "opt out" of their health data being used for research.

You may not qualify if:

• Patients who "opted out" of their health data being used for research.

Sponsors & Collaborators

• RwHealth: lead
• Alnylam Pharmaceuticals: collaborator
• Northern Care Alliance NHS Foundation Trust: collaborator

Study Sites (1)

Northern Care Alliance NHS Foundation Trust

Salford, M6 8HD, United Kingdom

Location

MeSH Terms

Conditions

Polyradiculoneuropathy, Chronic Inflammatory Demyelinating; Guillain-Barre Syndrome; Amyloidosis, Familial; Neuritis

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; Post-Infectious Disorders; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases; Amyloidosis; Proteostasis Deficiencies

Central Study Contacts

Mike Hughes, Ph.D.

CONTACT

07462249069; mike.hughes@realworld.health

Scott Fletcher, BSc

CONTACT

scott.fletcher@realworld.health

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 22, 2023
• First Posted: July 11, 2023
• Study Start: August 17, 2023
• Primary Completion: October 30, 2023
• Study Completion: October 30, 2023
• Last Updated: July 11, 2023

Record last verified: 2023-07

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1)