Superficial Siderosis Clinical Trial
Official title:
Phase IV Observational Study of Deferiprone (Ferriprox®) in the Treatment of Superficial Siderosis
NCT number | NCT01284127 |
Other study ID # | NA_00067749 |
Secondary ID | |
Status | Completed |
Phase | |
First received | |
Last updated | |
Start date | March 2012 |
Est. completion date | October 4, 2018 |
Verified date | August 2019 |
Source | Johns Hopkins University |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
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.
Status | Completed |
Enrollment | 38 |
Est. completion date | October 4, 2018 |
Est. primary completion date | January 8, 2018 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years to 100 Years |
Eligibility |
Inclusion Criteria: 1. Confirmed diagnosis of superficial siderosis by MRI. 2. Must be receiving deferiprone according to standard of care under the supervision of the treating physician. 3. Must be able to understand and sign the informed consent form. Exclusion Criteria: 1. If the patient is unwilling or unable to comply with the requirements of the study. |
Country | Name | City | State |
---|---|---|---|
United States | Johns Hopkins University | Baltimore | Maryland |
Lead Sponsor | Collaborator |
---|---|
Johns Hopkins University |
United States,
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. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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 | |
Primary | 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 | |
Primary | 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 | |
Primary | 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 | |
Primary | 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 | Number of Participants With MRI of the Brain and Spinal Cord Showing Changes in Hemosiderin Deposition | MRI of the brain and spinal cord without contrast to monitor for changes in hemosiderin deposition in terms of worsening or improvement. | Baseline, At the end of the 2-year period |
Status | Clinical Trial | Phase | |
---|---|---|---|
Not yet recruiting |
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