A Study Investigating the Long-term Safety and Efficacy of Deferiprone in Patients With Friedreich's Ataxia

Friedreich's Ataxia Clinical Trial

Official title:

An Open-label, Single Treatment, Safety and Efficacy, Long-term Study of Deferiprone in Subjects With Friedreich's Ataxia

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00897221
• Other study ID #: LA29-EXT
• Status: Completed
• Phase: Phase 2
• First received: May 8, 2009
• Last updated: June 24, 2011
• Start date: June 2009
• Est. completion date: March 2011

Study information

• Verified date: June 2011
• Source: ApoPharma
• Contact: n/a
• Is FDA regulated: No
• Health authority: Belgium: Federal Agency for Medicinal Products and Health ProductsFrance: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)Italy: Ethics CommitteeSpain: Spanish Agency of Medicines
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 36
• Est. completion date: March 2011
• Est. primary completion date: March 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 7 Years to 35 Years

Eligibility

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.

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Ataxia
• Cerebellar Ataxia
• Friedreich Ataxia
• Friedreich's Ataxia

Intervention

Drug:
• Deferiprone oral solution 100mg/mL
Deferiprone oral solution (20 mg/kg/day)
• Deferiprone oral solution 100 mg/mL
Deferiprone oral solution(40mg/kg/day)

Locations

Country	Name	City	State
Belgium	Hospital Erasme	Brussels
France	Hospital Necker-Enfants Malades	Paris
Italy	Fondazione IRCCS Istituto Neurologico "C. Besta"	Milan
Spain	La Fundacion Para la Investigacion Biomedica	Madrid

Sponsors (1)

Lead Sponsor	Collaborator
ApoPharma

Countries where clinical trial is conducted

Belgium,  France,  Italy,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The patient's long-term tolerance of treatment will be assessed by the occurence of adverse events.		52 weeks	Yes
Secondary	The long-term efficacy of deferiprone will be assessed. Efficacy measures include the 9HPT, T25FW, LCLA, ICARS and FARS.		52 weeks	No