Study Based on Electronic Health RecOrds to Identify Patients at High-risk of Fabry DiseasE (HOPE Fabry)

NCT ID: NCT05671770

Last Updated: 2024-03-06

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 15 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2023-07-28
• Study Completion Date: 2023-12-14

Brief Summary

Primary objective:

To estimate the prevalence of patients who are at high-risk for Fabry Disease (FD) in the Cleveland Clinic, Abu Dhabi (CCAD) United Arab Emirates (EMR) database from May 2016 to May 2022, according to the predictive algorithm (i.e., feasibility assessment eligibility criteria)

Secondary objectives:

• To estimate the prevalence of FD among patients at high-risk for FD (i.e., among enrolled patients)
• To characterize the patient profile, overall and in Cohorts 1 and 2
• To describe the most common characteristics among positive FD patients and negative FD patients

Detailed Description

Patients at high-risk for FD will be consecutively enrolled over approximately 6 months

Conditions

Fabry Disease

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: RETROSPECTIVE

Study Groups

Cohort 1

Patients with chronic kidney disease

No interventions assigned to this group

Cohort 2

Patients with hypertrophic cardiomyopathy

No interventions assigned to this group

Eligibility Criteria

Inclusion Criteria

• Patients who were identified as high-risk for FD via the predictive algorithm per the feasibility assessment
• Patients who have signed an informed consent form

Cohort 1 (chronic kidney disease)

• Adult male patient ≤60 years old or adult female patient of any age
• Having chronic kidney disease
• Having proteinuria
• Having one or more of the following conditions:

• Neuralgia and neuritis, unspecified
• Disturbances in skin sensation
• Anhidrosis, and
• Hypertrophic cardiomyopathy.

Cohort 2 (hypertrophic cardiomyopathy)

• Adult male patient ≤50 years old or adult female patient of any age
• Having hypertrophic cardiomyopathy
• Having one or more of the following conditions:

• transient cerebral ischemic attack,
• chronic kidney disease.

Exclusion Criteria

• Patients with an established diagnosis of FD.
• Pregnant subject at the time of the study.

The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Sanofi

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Sciences & Operations

Role: STUDY_DIRECTOR

Sanofi

Locations

Cleveland Clinic AbuDhabi

Abu Dhabi, , United Arab Emirates

Countries

United Arab Emirates

Other Identifiers

OBS17546

Identifier Type: -

Identifier Source: org_study_id