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

NCT01284127 | Completed Results

• 1 other identifier: NA_00067749
• Study Type: observational
• Target: 38
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Superficial Siderosis

Keywords

superficial siderosis

Outcome Measures

Primary Outcomes (5)

• Number of Participants Who Show Stability, Improvement or Decline in Self Reported Hearing

  Number of participants who show stability, improvement or decline in self reported hearing.

  At the end of the 2-year period

• Number of Participants Who Show Stability, Improvement or Decline in Self Reported Coordination

  Number of participants who show stability, improvement or decline in self reported coordination.

  At the end of the 2-year period

• Number of Participants Who Show Stability, Improvement or Decline in Self Reported Walking

  Number of participants who show stability, improvement or decline in self reported walking.

  At the end of the 2-year period

• Number of Participants Who Show Stability, Improvement or Decline in Self Reported Fine Motor Function

  Number of participants who show stability, improvement or decline in self reported fine motor function.

  At the end of the 2-year period

• Number of Participants Who Show Stability, Improvement or Decline in Self Reported Bowel/Bladder Function

  Number of participants who show stability, improvement or decline in self reported bowel/bladder function.

  At the end of the 2-year period

Secondary Outcomes (1)

• Number of Participants With MRI of the Brain and Spinal Cord Showing Changes in Hemosiderin Deposition

  Baseline, At the end of the 2-year period

Study Arms (1)

Deferiprone

All patients must have MRI evidence of superficial siderosis and be treated with deferiprone according to standard of care guidelines.

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

All patients with MRI-confirmed superficial siderosis currently residing in the United States.

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.

Sponsors & Collaborators

• Johns Hopkins University: lead

Study Sites (1)

Johns Hopkins University

Baltimore, Maryland, 21287, United States

Location

Related Publications (1)

• Levy M, Llinas RH. Deferiprone reduces hemosiderin deposits in the brain of a patient with superficial siderosis. AJNR Am J Neuroradiol. 2011 Jan;32(1):E1-2. doi: 10.3174/ajnr.A2331. Epub 2010 Nov 4.

  PMID: 21051507 BACKGROUND

Limitations and Caveats

The major limitations of this study relate to the observational design.

Results Point of Contact

• Title: Michael Levy
• Organization: Johns Hopkins University

mlevy@jhmi.edu

443-287-4612

Study Officials

• Michael Levy, MD, PhD

  Johns Hopkins University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 20, 2011
• First Posted: January 26, 2011
• Study Start: March 1, 2012
• Primary Completion: January 8, 2018
• Study Completion: October 4, 2018
• Last Updated: September 23, 2019
• Results First Posted: September 23, 2019

Record last verified: 2019-08

Locations

US (1)