Pharmacoepidemiologic General Research Extension: PGRx-sIBM Study

Sporadic inclusion body myositis (sIBM) is a rare idiopathic inflammatory myopathy characterized by chronic proximal leg and distal arm asymmetric muscle weakness. Its prevalence is estimated between 10 and 100 cases per million, over the age of 50. Little is known about the natural course of the disease, the delay between onset and diagnosis, the patterns of disease management and treatment in routine practice, as well as the burden for patients.

The objectives of the PGRx-sIBM study will be to describe:

• The epidemiology of the disease (estimation of the prevalence, age at onset, delay between onset and diagnosis, diagnosis process, etc.)
• The patterns of disease management and treatment in routine practice
• The burden of the disease for patients and their informal caregiver, as well as the quality of life of patients
• The burden of the disease for informal caregivers

This will be an observational (non-interventional), cross-sectional (transversal) study.

Study setting The PGRx-sIBM will take place in France, Italy, Switzerland and Spain. Recruitment centers will be public and private practices and hospitals from urban or rural areas. In each recruitment center, at least one physician will act as a study investigator, in charge of identifying and recruiting patients.

Inclusion of patients

Patients will be included by their specialized physician (neurologist, Internal Medicine) during a regular outpatient visit, provided that they meet the following inclusion criteria:

• The patient has been diagnosed with sIBM, as per the physician judgement and regardless of the date of diagnosis
• Male or female
• Age ≥ 45 years old
• The patient does regularly live in the country of inclusion
• The patient or his/her proxy can read and respond to a telephone interview
• The patient agrees to participate

Exclusion criteria will be:

• The patient refuses to participate or the written Consent Form is not signed
• The patient or his/her proxy cannot be reached by telephone. All eligible patients will be invited to participate in the study, during a 3 to 6-month period of time.

The target sample size will be of 190 patients (≈15-20 in Switzerland, ≈50 in Spain, ≈50 in Italy and ≈70-75 in France).

Study Overview

• Status: Unknown
• Conditions: Sporadic Inclusion Body Myositis

• Study Type: Observational
• Enrollment (Anticipated): 190

Contacts and Locations

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 45 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Patients diagnosed with sporadic inclusion body myositis.

Description

Inclusion Criteria:

• The patient has been diagnosed with sIBM, as per the physician judgement and regardless of the date of diagnosis
• Male or female
• Age ≥ 45 years old
• The patient does regularly live in the country of inclusion
• The patient or his/her proxy can read and respond to a telephone interview
• The patient agrees to participate

Exclusion Criteria:

• The patient refuses to participate or the written Consent Form is not signed
• The patient or his/her proxy cannot be reached by telephone.

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Only
• Time Perspectives: Cross-Sectional

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Disability	Measured by the Clinical Global Impression scale and the use of assisitive devices	Within the last 12 months
Quality of Life	EQ-D5	Within the last 7 days

Collaborators and Investigators

• Sponsor: La-ser Europe Limited
• Investigators: Study Director: Lamiae Grimaldi-Bensouda, PharmD, PhD, LASER

Study record dates

Study Major Dates

• Study Start: May 1, 2016
• Primary Completion (Anticipated): February 1, 2017
• Study Completion (Anticipated): February 1, 2017

Study Registration Dates

• First Submitted: April 6, 2016
• First Submitted That Met QC Criteria: April 6, 2016
• First Posted (Estimate): April 12, 2016

Study Record Updates

• Last Update Posted (Estimate): April 18, 2016
• Last Update Submitted That Met QC Criteria: April 14, 2016
• Last Verified: April 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Myositis; Myositis, Inclusion Body
• Other Study ID Numbers: LASER-PGRx