Back to resultsA Study Investigating the Long-term Safety and Efficacy of Deferiprone in Patients With Friedreich's Ataxia
Primary Purpose
Friedreich's Ataxia
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Deferiprone oral solution 100mg/mL
Deferiprone oral solution 100 mg/mL
Sponsored by
About this trial
This is an interventional treatment trial for Friedreich's Ataxia focused on measuring Friedreich's ataxia
Eligibility Criteria
Inclusion Criteria:
- Subjects who completed the ApoPharma study LA29-0207
- Female subjects of childbearing potential must have a negative pregnancy test.
- Male subjects must confirm that he and/or his female partner will use an effective method of contraception for the length of the trial and for 30 days following completion of the study or early termination.
- Signed and witnessed written informed consent/assent, obtained prior to the first study intervention, as well as the ability to adhere to study restrictions, appointments and evaluation schedules.
Exclusion Criteria:
- Serum Ferritin and Hemoglobin (Hb) levels are below the reference range for age and sex-matched controls.
- Unable to complete T25FW AND with a 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).
- Doubling of score on 9HPT or T25FW compared to their study baseline results in LA29-0207.
- History or evidence of neutropenia/agranulocytosis defined by a confirmed absolute neutrophil count (ANC) < 1.5 x 109/L or thrombocytopenia defined by a platelet count <150 x 109/L.
- Occurrence of SAEs or any other AEs during the LA29-0207 study, which in the opinion of the investigator cause the patient's participation in the extension study to be inappropriate.
- Unable to comply with requirements of the protocol.
- Pregnant, breastfeeding or planning to become pregnant during the study period.
- QTc interval >450ms.
- Have been on antioxidants prior to start of study treatment.
Sites / Locations
- Hospital Erasme
- Hospital Necker-Enfants Malades
- Fondazione IRCCS Istituto Neurologico "C. Besta"
- La Fundacion Para la Investigacion Biomedica
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
Dose 1
Dose 2
Arm Description
Deferiprone oral solution 20 mg/kg/day
Deferiprone oral solution 40 mg/kg/day
Outcomes
Primary Outcome Measures
The patient's long-term tolerance of treatment will be assessed by the occurence of adverse events.
Secondary Outcome Measures
The long-term efficacy of deferiprone will be assessed. Efficacy measures include the 9HPT, T25FW, LCLA, ICARS and FARS.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00897221
Brief Title
A Study Investigating the Long-term Safety and Efficacy of Deferiprone in Patients With Friedreich's Ataxia
Official Title
An Open-label, Single Treatment, Safety and Efficacy, Long-term Study of Deferiprone in Subjects With Friedreich's Ataxia
Study Type
Interventional
2. Study Status
Record Verification Date
June 2011
Overall Recruitment Status
Completed
Study Start Date
June 2009 (undefined)
Primary Completion Date
March 2011 (Actual)
Study Completion Date
March 2011 (Actual)
3. Sponsor/Collaborators
Name of the Sponsor
ApoPharma
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The primary objective of this study is to evaluate the long-term safety and tolerability of deferiprone in subjects with Friedreich's ataxia (FRDA).
The secondary objective is to evaluate the long-term efficacy of deferiprone for the treatment of FRDA.
The tertiary objectives are to evaluate the effect of deferiprone on:
cardiac function,
quality of life, and
functional status.
Detailed Description
This is a multi-centre, open-label, non-randomized, single treatment, safety and efficacy study. All subjects who completed the LA29-0207 study are eligible for participation. Participants will receive deferiprone oral solution at the same dose (20 or 40 mg/kg/day) that they were assigned for LA29-0207. The duration of treatment will be 52 weeks.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Friedreich's Ataxia
Keywords
Friedreich's ataxia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
36 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Dose 1
Arm Type
Experimental
Arm Description
Deferiprone oral solution 20 mg/kg/day
Arm Title
Dose 2
Arm Type
Experimental
Arm Description
Deferiprone oral solution 40 mg/kg/day
Intervention Type
Drug
Intervention Name(s)
Deferiprone oral solution 100mg/mL
Other Intervention Name(s)
Ferriprox
Intervention Description
Deferiprone oral solution (20 mg/kg/day)
Intervention Type
Drug
Intervention Name(s)
Deferiprone oral solution 100 mg/mL
Other Intervention Name(s)
Ferriprox
Intervention Description
Deferiprone oral solution(40mg/kg/day)
Primary Outcome Measure Information:
Title
The patient's long-term tolerance of treatment will be assessed by the occurence of adverse events.
Time Frame
52 weeks
Secondary Outcome Measure Information:
Title
The long-term efficacy of deferiprone will be assessed. Efficacy measures include the 9HPT, T25FW, LCLA, ICARS and FARS.
Time Frame
52 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
7 Years
Maximum Age & Unit of Time
35 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Subjects who completed the ApoPharma study LA29-0207
Female subjects of childbearing potential must have a negative pregnancy test.
Male subjects must confirm that he and/or his female partner will use an effective method of contraception for the length of the trial and for 30 days following completion of the study or early termination.
Signed and witnessed written informed consent/assent, obtained prior to the first study intervention, as well as the ability to adhere to study restrictions, appointments and evaluation schedules.
Exclusion Criteria:
Serum Ferritin and Hemoglobin (Hb) levels are below the reference range for age and sex-matched controls.
Unable to complete T25FW AND with a 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).
Doubling of score on 9HPT or T25FW compared to their study baseline results in LA29-0207.
History or evidence of neutropenia/agranulocytosis defined by a confirmed absolute neutrophil count (ANC) < 1.5 x 109/L or thrombocytopenia defined by a platelet count <150 x 109/L.
Occurrence of SAEs or any other AEs during the LA29-0207 study, which in the opinion of the investigator cause the patient's participation in the extension study to be inappropriate.
Unable to comply with requirements of the protocol.
Pregnant, breastfeeding or planning to become pregnant during the study period.
QTc interval >450ms.
Have been on antioxidants prior to start of study treatment.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Massimo Pandolfo, M.D.
Organizational Affiliation
Hospital Erasme, Brussels, Belgium
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Arnold Munnich, M.D.
Organizational Affiliation
Hospital Necker-Enfants Malades, Paris, France
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Franco Taroni, M.D.
Organizational Affiliation
Fondazione IRCCS Istituto Neurologico "C. Besta", Milan, Italy
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Javier Arpa, M.D.
Organizational Affiliation
La Fundaction Para la Investigacion Biomedica, Madrid, Spain
Official's Role
Principal Investigator
Facility Information:
Facility Name
Hospital Erasme
City
Brussels
Country
Belgium
Facility Name
Hospital Necker-Enfants Malades
City
Paris
Country
France
Facility Name
Fondazione IRCCS Istituto Neurologico "C. Besta"
City
Milan
Country
Italy
Facility Name
La Fundacion Para la Investigacion Biomedica
City
Madrid
Country
Spain
12. IPD Sharing Statement
Learn more about this trial
A Study Investigating the Long-term Safety and Efficacy of Deferiprone in Patients With Friedreich's Ataxia
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