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NCT02120235 Clinical Trial

Investigating Lysosomal Storage Diseases in Minority Groups

Fabry Disease Clinical Trial

Clinical Trial Details — Status: Recruiting

Administrative data
NCT number NCT02120235
Other study ID # 14-CFCT-11
Secondary ID
Status Recruiting
Phase N/A
First received April 17, 2014
Last updated April 3, 2017
Start date February 2014
Est. completion date December 2018

Study information
Verified date April 2017
Source O & O Alpan LLC
Contact Ozlem Goker-Alpan, M.D.
Phone 571-308-1900
Email ogokeralpan@oandoalpan.com
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

Although lysosomal storage disorders, such as Fabry disease, Gaucher disease, and Pompe disease, represent serious challenges in the healthcare system, no study has yet investigated the prevalence of these diseases in the US. Frequently, patients show progressive worsening of symptoms for several years before they get diagnosed. Since many of these diseases can be managed therapeutically, it is important to identify and treat patients in order to avoid organ damage. The investigators aim to undertake a screening study that identifies undiagnosed patients with lysosomal storage disorders and determine the prevalence of these diseases with special focus on underrepresented minority groups.

Recruitment information / eligibility
Status Recruiting
Enrollment 20000
Est. completion date December 2018
Est. primary completion date February 2018
Accepts healthy volunteers Accepts Healthy Volunteers
Gender All
Age group N/A to 100 Years
Eligibility Inclusion Criteria:

- Subject is greater than or equal to 1 day of age and less than or equal to 100 years of age

- Subject is managed by a physician in the Washington, D.C metro area

- Subject is getting blood work as part of standard clinical care and there is at least 60 uL blood remained in a tube after all clinical tests were run

Exclusion Criteria:

- Absolute contraindication for blood drawing

- Subject cannot be traced back by the referring physician upon a positive screening result

Study Design

Related Conditions & MeSH terms

Locations
Country Name City State
United States O&O Alpan, LLC Fairfax Virginia

Sponsors (1)
Lead Sponsor Collaborator
O & O Alpan LLC

Country where clinical trial is conducted

United States, 

Outcome
Type Measure Description Time frame Safety issue
Primary Number of patients identified with lysosomal storage disorders 2 years
See also
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