Observational Study of Deferiprone (Ferriprox®) in the Treatment of Superficial Siderosis

Completed

Conditions studied: Superficial Siderosis

In brief

Superficial siderosis is a progressive neurological disease caused by iron deposition in the central nervous system (CNS) from chronic subarachnoid bleeding. Until 2011, there has been no effective treatment for this progressive condition that leads to hearing loss, spasticity, weakness, loss of bowel/bladder function, incoordination, dementia and ultimately death. Last year, the investigators demonstrated that a lipid soluble iron chelator, deferiprone, can reduce hemosiderin deposition in patients with superficial siderosis by MRI in as little as 3 months. As the only therapy that can improve this condition, chelation with deferiprone is the standard of care for treatment of superficial siderosis. Now that the FDA has approved deferiprone in the United States for thalassemia, the investigators propose documenting the clinical effect of deferiprone over 2 years in superficial siderosis patients. The investigators' goal is to understand how the clinical course of this disease is altered using standard of care chelation therapy with deferiprone. Patients with superficial siderosis who are taking deferiprone for chelation therapy at doses consistent with the standard of care will be offered enrollment into this observational study. Patients will be treated and monitored locally by participating neurologists who have agreed to help the investigators collect information for this study. The investigators are interested in documenting the clinical effect of deferiprone on hearing, ataxia and myelopathy using standardized scales performed and documenting the effect of deferiprone on hemosiderin deposition in the CNS by MRI, all performed according to standard of care.

Key facts

Study ID

NCT01284127

Run by

Johns Hopkins University

People needed

38

Starts

2012-03-01

Expected to finish

2018-10-04

Last updated by the study team

2019-09-23

Who can join

Age: 18 and older, up to 100. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Confirmed diagnosis of superficial siderosis by MRI.
• Must be receiving deferiprone according to standard of care under the supervision of the treating physician.
• Must be able to understand and sign the informed consent form.

You may not qualify if…

• If the patient is unwilling or unable to comply with the requirements of the study.

Where it is running

• Johns Hopkins University — Baltimore, Maryland, United States

Full record on ClinicalTrials.gov

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