CClinicalTrials.gg
Sign In
CompletedNCT00897221Updated Jun 27, 2011

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

A Phase 2 interventional study of Deferiprone oral solution 100mg/mL and Deferiprone oral solution 100 mg/mL in Friedreich's Ataxia, sponsored by ApoPharma. Completed at 4 sites in 4 countries. Open to participants aged 7 Years to 35 Years. Per ClinicalTrials.gov, last updated 2011-06-27.

Sponsored by ApoPharma · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
36
Allocation
Non-randomized
Ages
7 Years to 35 Years
Sex
All
01

Study 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.
Read the 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.

02

Conditions studied

  • Friedreich's Ataxia

Keywords

  • Friedreich's ataxia
03

In context

Ataxia

295 studies on the registry are indexed under Ataxia; 51 are open to participants now.

This study's enrollment of 36 is above the median of 26 across 216 interventional studies indexed under Ataxia.

Browse Ataxia studies →

Lead sponsor

ApoPharma is the lead sponsor of 23 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
7 Years to 35 Years
Sexes eligible
All
Accepts healthy volunteers
No

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

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.
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
36 participants (actual)

Study arms

  • Experimental
    Dose 1

    Deferiprone oral solution 20 mg/kg/day

    Drug: Deferiprone oral solution 100mg/mL

  • Experimental
    Dose 2

    Deferiprone oral solution 40 mg/kg/day

    Drug: Deferiprone oral solution 100 mg/mL

Interventions

  • DrugDeferiprone oral solution 100mg/mL

    Deferiprone oral solution (20 mg/kg/day)

    Also known as: Ferriprox

  • DrugDeferiprone oral solution 100 mg/mL

    Deferiprone oral solution(40mg/kg/day)

    Also known as: Ferriprox

06

What researchers measure

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

07

Study locations

4 sites
  • Hospital Erasme
    Brussels, Belgium
  • Hospital Necker-Enfants Malades
    Paris, France
  • Fondazione IRCCS Istituto Neurologico "C. Besta"
    Milan, Italy
  • La Fundacion Para la Investigacion Biomedica
    Madrid, Spain
08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jun 27, 2011, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT00897221
Lead sponsor
ApoPharma
First posted
May 12, 2009
Start date
Jun 2009
Primary completion
Mar 2011
Completion
Mar 2011
Last update
Jun 27, 2011

Study contacts

Massimo Pandolfo, M.D.
principal investigator · Hospital Erasme, Brussels, Belgium
Arnold Munnich, M.D.
principal investigator · Hospital Necker-Enfants Malades, Paris, France
Franco Taroni, M.D.
principal investigator · Fondazione IRCCS Istituto Neurologico "C. Besta", Milan, Italy
Javier Arpa, M.D.
principal investigator · La Fundaction Para la Investigacion Biomedica, Madrid, Spain

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Jun 2011. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Other studies in this condition

Ataxia→

Other studies from this sponsor

ApoPharma

Start the discussion