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7408: Natural History of Pearson Syndrome

Status: Recruiting

 

Pearson Syndrome has not been studied in a prospective fashion. Most of what is known about Pearson Syndrome is from case reports. Pearson Syndrome is a rare mitochondrial disorder and only about 60 patients have been described in these reports. Children typically present in their 1st two years of life (most in infancy) with anemia with or without pancreatitis (Inflammation of the pancreas.

The objective of this study is to characterize the natural history of Pearson Syndrome.

 

This Study is for: Pearson Syndrome

 

Summary

Background

The purpose of this observational study is to obtain information on the natural history of Pearson Syndrome for the purpose of educating the scientific community and for development of potential treatment options through clinical trials.

 

About this Study

The multi-center clinical research network conducting this research will include several NAMDC sites with a total planned enrollment of 25 patients. Patients will remain in the study for a minimum of 3 years and up to 5 years dependent on continued funding.

 

Target Enrollment

To be eligible to participate, you must:

  1. Be an individual of any age with confirmed Pearson Syndrome.
  2. Agree to participate in the NAMDC Clinical Registry

You are not eligible to participate if:

  1. Your disease can not be confirmed for Pearson Syndrome
  2. You are not willing to participate in the NAMDC clinical Registry
 

How to participate:

In order to participate in a study, you may contact the study coordinator:

Sumit Parikh, M.D.
Cleveland Clinic, Cleveland, OH
Phone: 216-444-1994
parikhs@ccf.org