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

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

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.

Study Overview

• Status: Completed
• Conditions: Friedreich's Ataxia
• Intervention / Treatment: Drug: Deferiprone oral solution 100mg/mL; Drug: Deferiprone oral solution 100 mg/mL

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.

• Study Type: Interventional
• Enrollment (Actual): 36
• Phase: Phase 2

Contacts and Locations

Study Locations

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

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 7 years to 35 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Dose 1; Deferiprone oral solution 20 mg/kg/day	Drug: Deferiprone oral solution 100mg/mL; Deferiprone oral solution (20 mg/kg/day); Other Names: Ferriprox
Experimental: Dose 2; Deferiprone oral solution 40 mg/kg/day	Drug: Deferiprone oral solution 100 mg/mL; Deferiprone oral solution(40mg/kg/day); Other Names: Ferriprox

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
The patient's long-term tolerance of treatment will be assessed by the occurence of adverse events.	52 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
The long-term efficacy of deferiprone will be assessed. Efficacy measures include the 9HPT, T25FW, LCLA, ICARS and FARS.	52 weeks

Collaborators and Investigators

• Sponsor: ApoPharma
• Investigators: Principal Investigator: Massimo Pandolfo, M.D., Hospital Erasme, Brussels, Belgium; Principal Investigator: Arnold Munnich, M.D., Hospital Necker-Enfants Malades, Paris, France; Principal Investigator: Franco Taroni, M.D., Fondazione IRCCS Istituto Neurologico "C. Besta", Milan, Italy; Principal Investigator: Javier Arpa, M.D., La Fundaction Para la Investigacion Biomedica, Madrid, Spain

Study record dates

Study Major Dates

• Study Start: June 1, 2009
• Primary Completion (Actual): March 1, 2011
• Study Completion (Actual): March 1, 2011

Study Registration Dates

• First Submitted: May 8, 2009
• First Submitted That Met QC Criteria: May 8, 2009
• First Posted (Estimate): May 12, 2009

Study Record Updates

• Last Update Posted (Estimate): June 27, 2011
• Last Update Submitted That Met QC Criteria: June 24, 2011
• Last Verified: June 1, 2011

More Information

Terms related to this study

• Keywords: Friedreich's ataxia
• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Genetic Diseases, Inborn; Neurodegenerative Diseases; Dyskinesias; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Mitochondrial Diseases; Cerebellar Diseases; Spinocerebellar Degenerations; Ataxia; Cerebellar Ataxia; Friedreich Ataxia; Molecular Mechanisms of Pharmacological Action; Chelating Agents; Sequestering Agents; Iron Chelating Agents; Pharmaceutical Solutions; Deferiprone
• Other Study ID Numbers: LA29-EXT