Pharmacoepidemiologic General Research Extension: PGRx-sIBM Study

NCT ID: NCT02735447

Last Updated: 2016-04-18

Basic Information

• Recruitment Status: UNKNOWN
• Total Enrollment: 190 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2016-05-31
• Study Completion Date: 2017-02-28

Brief Summary

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).

Conditions

Sporadic Inclusion Body Myositis

Study Design

• Observational Model Type: CASE_ONLY
• Study Time Perspective: CROSS_SECTIONAL

Eligibility Criteria

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.

• Minimum Eligible Age: 45 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

La-ser Europe Limited

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Lamiae Grimaldi-Bensouda, PharmD, PhD

Role: STUDY_DIRECTOR

LASER

Central Contacts

Clémentine Nordon, MD, PhD

Role: CONTACT

Clementine.Nordon@la-ser.com

+33 (0) 1 83 62 91 53

Other Identifiers

LASER-PGRx

Identifier Type: -

Identifier Source: org_study_id