Trial Outcomes & Findings for Observational Study of Deferiprone (Ferriprox®) in the Treatment of Superficial Siderosis (NCT NCT01284127)

NCT ID: NCT01284127

Last Updated: 2019-09-23

Results Overview

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

• Recruitment status: COMPLETED
• Target enrollment: 38 participants
• Primary outcome timeframe: At the end of the 2-year period
• Results posted on: 2019-09-23

Participant Flow

Participant milestones

Measure	Deferiprone All patients must have MRI evidence of superficial siderosis and be treated with deferiprone according to standard of care guidelines.
Overall Study STARTED	38
Overall Study COMPLETED	31
Overall Study NOT COMPLETED	7

Reasons for withdrawal

Measure	Deferiprone All patients must have MRI evidence of superficial siderosis and be treated with deferiprone according to standard of care guidelines.
Overall Study Adverse Event	2
Overall Study Withdrawal by Subject	4
Overall Study Physician Decision	1

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	Deferiprone n=38 Participants All patients must have MRI evidence of superficial siderosis and be treated with deferiprone according to standard of care guidelines.
Age, Continuous	64 years n=5 Participants
Sex: Female, Male Female	18 Participants n=5 Participants
Sex: Female, Male Male	20 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	36 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Region of Enrollment United States	38 Participants n=5 Participants
Cause of Bleed Dural Tear	11 Participants n=5 Participants
Cause of Bleed Prior Neurosurgical Procedure	8 Participants n=5 Participants
Cause of Bleed Central Nervous System (CNS) Tumor/Cyst	4 Participants n=5 Participants
Cause of Bleed Undetermined	15 Participants n=5 Participants
Duration of Clinical Disease	7 years n=5 Participants
Participants with Repair of Bleed	7 Participants n=5 Participants

PRIMARY outcome

Timeframe: At the end of the 2-year period

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

Outcome measures

Measure	Deferiprone n=31 Participants All patients must have MRI evidence of superficial siderosis and be treated with deferiprone according to standard of care guidelines.
Number of Participants Who Show Stability, Improvement or Decline in Self Reported Hearing Stable	13 Participants
Number of Participants Who Show Stability, Improvement or Decline in Self Reported Hearing Worse	18 Participants
Number of Participants Who Show Stability, Improvement or Decline in Self Reported Hearing Improved	0 Participants

PRIMARY outcome

Timeframe: At the end of the 2-year period

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

Outcome measures

Measure	Deferiprone n=31 Participants All patients must have MRI evidence of superficial siderosis and be treated with deferiprone according to standard of care guidelines.
Number of Participants Who Show Stability, Improvement or Decline in Self Reported Coordination Stable	8 Participants
Number of Participants Who Show Stability, Improvement or Decline in Self Reported Coordination Worse	21 Participants
Number of Participants Who Show Stability, Improvement or Decline in Self Reported Coordination Improved	2 Participants

PRIMARY outcome

Timeframe: At the end of the 2-year period

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

Outcome measures

Measure	Deferiprone n=31 Participants All patients must have MRI evidence of superficial siderosis and be treated with deferiprone according to standard of care guidelines.
Number of Participants Who Show Stability, Improvement or Decline in Self Reported Walking Improved	1 Participants
Number of Participants Who Show Stability, Improvement or Decline in Self Reported Walking Stable	9 Participants
Number of Participants Who Show Stability, Improvement or Decline in Self Reported Walking Worse	21 Participants

PRIMARY outcome

Timeframe: At the end of the 2-year period

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

Outcome measures

Measure	Deferiprone n=31 Participants All patients must have MRI evidence of superficial siderosis and be treated with deferiprone according to standard of care guidelines.
Number of Participants Who Show Stability, Improvement or Decline in Self Reported Fine Motor Function Stable	9 Participants
Number of Participants Who Show Stability, Improvement or Decline in Self Reported Fine Motor Function Worse	22 Participants
Number of Participants Who Show Stability, Improvement or Decline in Self Reported Fine Motor Function Improved	0 Participants

PRIMARY outcome

Timeframe: At the end of the 2-year period

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

Outcome measures

Measure	Deferiprone n=31 Participants All patients must have MRI evidence of superficial siderosis and be treated with deferiprone according to standard of care guidelines.
Number of Participants Who Show Stability, Improvement or Decline in Self Reported Bowel/Bladder Function Stable	12 Participants
Number of Participants Who Show Stability, Improvement or Decline in Self Reported Bowel/Bladder Function Worse	18 Participants
Number of Participants Who Show Stability, Improvement or Decline in Self Reported Bowel/Bladder Function Improved	1 Participants

SECONDARY outcome

Timeframe: Baseline, At the end of the 2-year period

Population: All participants with follow up MRIs (16) were evaluated.

MRI of the brain and spinal cord without contrast to monitor for changes in hemosiderin deposition in terms of worsening or improvement.

Outcome measures

Measure	Deferiprone n=16 Participants All patients must have MRI evidence of superficial siderosis and be treated with deferiprone according to standard of care guidelines.
Number of Participants With MRI of the Brain and Spinal Cord Showing Changes in Hemosiderin Deposition Worse	8 Participants
Number of Participants With MRI of the Brain and Spinal Cord Showing Changes in Hemosiderin Deposition Improved	8 Participants

Adverse Events

Deferiprone

Serious events: 0 serious events

Other events: 10 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Deferiprone n=38 participants at risk All patients must have MRI evidence of superficial siderosis and be treated with deferiprone according to standard of care guidelines.
Nervous system disorders Fatigue	26.3% 10/38 • 2 years
Musculoskeletal and connective tissue disorders Joint Pain	5.3% 2/38 • 2 years
Skin and subcutaneous tissue disorders Mouth sores	5.3% 2/38 • 2 years

Additional Information

Michael Levy

Johns Hopkins University

Phone: 443-287-4612

Email: mlevy@jhmi.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place