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NCT Number: NCT00897221

A Study Investigating the Long-term Safety and Efficacy of Deferiprone in Patients 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.

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Key information

Conditions

Friedreich's Ataxia Brain Diseases Central Nervous System Diseases Cerebellar Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Friedreich Ataxia Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolic Diseases Mitochondrial Diseases Nervous System Diseases Neurodegenerative Diseases Nutritional and Metabolic Diseases Spinal Cord Diseases Spinocerebellar Degenerations

Age range

7 year–35 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Hospital Erasme, Brussels, Belgium

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Deferiprone oral solution 100mg/mL

Drug

Deferiprone oral solution (20 mg/kg/day)

Other names: Ferriprox

Deferiprone oral solution 100 mg/mL

Drug

Deferiprone oral solution(40mg/kg/day)

Other names: Ferriprox

Primary outcomes

  1. The patient's long-term tolerance of treatment will be assessed by the occurence of adverse events.

    Time frame: 52 weeks

Secondary outcomes

  1. The long-term efficacy of deferiprone will be assessed. Efficacy measures include the 9HPT, T25FW, LCLA, ICARS and FARS.

    Time frame: 52 weeks

Sponsors and collaborators

Lead sponsor

ApoPharma

Industry

Registry information

Official study title

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

Important dates

Study start
2009
Primary completion
2011
Study completion
2011
First posted
May 12, 2009
Registry last updated
Jun 27, 2011

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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