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Main
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Note: This record shows only the 20 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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17 October 2012 |
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Main ID: |
NCT00897221 |
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Date of registration:
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08/05/2009 |
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Primary sponsor: |
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Public title:
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A Study Investigating the Long-term Safety and Efficacy of Deferiprone in Patients With Friedreich's Ataxia
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Scientific title:
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An Open-label, Single Treatment, Safety and Efficacy, Long-term Study of Deferiprone in Subjects With Friedreich's Ataxia |
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Date of first enrolment:
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June 2009 |
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Target sample size:
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36 |
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Recruitment status: |
Completed |
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URL:
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http://clinicaltrials.gov/show/NCT00897221 |
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Study type:
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Interventional |
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Study design:
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Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
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Countries of recruitment
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Belgium
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Canada
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France
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Italy
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Spain
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Contacts
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Name:
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Franco Taroni, M.D. |
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Address:
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Telephone:
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Email:
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Affiliation:
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Fondazione IRCCS Istituto Neurologico "C. Besta", Milan, Italy |
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Name:
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Massimo Pandolfo, M.D. |
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Address:
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Telephone:
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Email:
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Affiliation:
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Hospital Erasme, Brussels, Belgium |
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Name:
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Arnold Munnich, M.D. |
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Address:
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Email:
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Affiliation:
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Hospital Necker-Enfants Malades, Paris, France |
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Name:
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Javier Arpa, M.D. |
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Address:
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Telephone:
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Email:
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Affiliation:
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La Fundaction Para la Investigacion Biomedica, Madrid, Spain |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
1. Subjects who completed the ApoPharma study LA29-0207
2. Female subjects of childbearing potential must have a negative pregnancy test.
3. 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.
4. 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:
1. Serum Ferritin and Hemoglobin (Hb) levels are below the reference range for age and
sex-matched controls.
2. 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).
3. Doubling of score on 9HPT or T25FW compared to their study baseline results in
LA29-0207.
4. 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.
5. 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.
6. Unable to comply with requirements of the protocol.
7. Pregnant, breastfeeding or planning to become pregnant during the study period.
8. QTc interval >450ms.
9. Have been on antioxidants prior to start of study treatment.
Age minimum:
7 Years
Age maximum:
35 Years
Gender:
Both
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Health Condition(s) or Problem(s) studied
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Friedreich's Ataxia
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Intervention(s)
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Drug: Deferiprone oral solution 100 mg/mL
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Drug: Deferiprone oral solution 100mg/mL
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Primary Outcome(s)
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The patient's long-term tolerance of treatment will be assessed by the occurence of adverse events.
[Time Frame: 52 weeks]
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Secondary Outcome(s)
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The long-term efficacy of deferiprone will be assessed. Efficacy measures include the 9HPT, T25FW, LCLA, ICARS and FARS.
[Time Frame: 52 weeks]
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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