Friedreich's Ataxia MedDRA version: 9.1 Level: LLT Classification code 10017374 Term: Friedreich's ataxia
Conditions
Interventions
Product Name: Deferiprone 100 mg/mL oral solution
Product Code: L1, AP0-066, CP20
Pharmaceutical Form: Oral solution
INN or Proposed INN: deferiprone
CAS Number: 30652-11-0
Current Sponsor code: L1, A
Sponsors
ApoPharma Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Diagnosis of FRDA, with confirmed mutation (excludes point mutation) in the frataxin (FXN) gene and GAA repeats = 400 on the shorter allele. 2. Males or females aged 7 to 35 years 3. No exposure to idebenone, coenzyme Q10, vitamin C, vitamin E or other antioxidants as a supplement or as a drug therapy for a period of at least one month prior to start of treatment and during the study. 4. Neurological testing: A FARS score >20 and =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Iron deficiency defined as ferritin levels below the reference range for age- and sex-matched controls 2. Unable to complete T25FW AND with score > 5 minutes in the 9HPT. (Subjects who can complete T25FW or with a score = 5 minutes in the 9HPT will be allowed to enrol if the score has not doubled compared to screening). 3. Abnormal ALT, greater than 2.0 times the upper limit of normal on two consecutive assessments. 4. Serum creatinine outside the normal reference range. 5. History or evidence of neutropenia/agranulocytosis defined by an absolute neutrophil count (ANC) < 1.5 x 10E9/L or thrombocytopenia defined by a platelet count <150 x 10E9/L. 6. Refusal to participate in screening procedures or unable to participate in screening procedures or unable to comply with the requirements of the protocol. 7. Receiving any investigational drug products or having received any investigational product within 30 days prior to enrolment into this study. 8. Subjects who have previously taken deferiprone. 9. Subjects who, in the opinion of the Investigator, represent poor medical, psychological or psychiatric risks, and for whom participation in an investigational trial would be unwise. 10. Pregnant, breastfeeding or planning to become pregnant during the study period. 11. History of malignancy. 12. History of alcohol or drug abuse. 13. Investigators, site personnel directly affiliated with this study and their immediate families. Immediate family is defined as a spouse, parent, child or sibling, whether biological or legally adopted. 14. Hypersensitivity to the active substance (deferiprone) or any of the excipients in the oral solution. 15. QT Interval Greater than 450 ms at Baseline.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of this study is to demonstrate the safety and tolerability of deferiprone in subjects with Friedreich’s ataxia (FRDA).;Secondary Objective: The secondary objective is to evaluate the efficacy of deferiprone for the treatment of FRDA, as assessed by a 9-Hole Peg Test (9HPT), Timed 25-Food Walk (T25FW), Low-Contrast Letter Acuity test (LCLA), International Cooperative Ataxia Rating Scale (ICARS), and Friedreich’s Ataxia Rating Scale (FARS). The tertiary objects are to evaluate the effect of deferiprone on: 1) cardiac function as measured by changes in Left Ventricular Shortening Fraction (LVSF), Left Ventricular Ejection Fraction (LVEF) and Left Ventricular (LV) mass using echocardiogram (ECHO), 2) quality of life using quality-of-life surveys, and 3) functional status using Activities of Daily Living (ADL). ;Primary end point(s): 9-Hole Peg Test: The 9HPT is a quantitative test of the functionality of the upper extremities (see Appendix 4). The test will be administered by a trained examiner (not necessarily the Investigator) in accordance with the Schedule of Events. The subject is instructed to place pegs into set holes as quickly as possible and then remove all the pegs. The test is performed two consecutive times with the dominant hand, followed by two consecutive times with the non-dominant hand. The maximum time allotted for each test (dominant or non-dominant hand) will be 5 minutes. The total time in seconds it takes the subject to complete the task is recorded. The average time for the four tests will be evaluated as a measure of treatment efficacy. Timed 25-Foot Walk: The T25FW is a quantitative mobility and leg function performance test (see Appendix 5). The test will be administered by a trained examiner (not necessarily the Investigator) in accordance with the Schedule of Events. The subject is instructed to walk 25 feet along a pre-defined straight course as quickly as possible. The task will be immediately repea | — |
Countries
Belgium, France, Italy, Spain, United Kingdom
Outcome results
None listed