Clinical trial NCT00897221: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Friedreich's Ataxia
Keywords: Friedreich's ataxia
Lead sponsor: ApoPharma
Sponsor class: INDUSTRY
First posted: 2009-05-12
Enrollment: 36
Sex: ALL
Minimum age (years): 7
Maximum age (years): 35
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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:

1. cardiac function,
2. quality of life, and
3. 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.

Eligibility criteria: 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.

Primary outcomes:
measure: The patient's long-term tolerance of treatment will be assessed by the occurence of adverse events.
timeFrame: 52 weeks
Secondary outcomes:
measure: The long-term efficacy of deferiprone will be assessed. Efficacy measures include the 9HPT, T25FW, LCLA, ICARS and FARS.
timeFrame: 52 weeks
Other outcomes:
Locations:
city: Brussels
country: Belgium
facility: Hospital Erasme
city: Paris
country: France
facility: Hospital Necker-Enfants Malades
city: Milan
country: Italy
facility: Fondazione IRCCS Istituto Neurologico "C. Besta"
city: Madrid
country: Spain
facility: La Fundacion Para la Investigacion Biomedica
Study officials:
affiliation: Hospital Erasme, Brussels, Belgium
name: Massimo Pandolfo, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Hospital Necker-Enfants Malades, Paris, France
name: Arnold Munnich, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Fondazione IRCCS Istituto Neurologico "C. Besta", Milan, Italy
name: Franco Taroni, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: La Fundaction Para la Investigacion Biomedica, Madrid, Spain
name: Javier Arpa, M.D.
role: PRINCIPAL_INVESTIGATOR
References: