Friedreich's AtaxiaPhase 1/2NCT00530127
A Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia
A Phase 1/2 study testing deferiprone in people with Friedreich's Ataxia, run by ApoPharma at 6 sites in 6 countries.
Trial summary
The primary objective of this study is to demonstrate the safety and tolerability of deferiprone in subjects with Friedreich's ataxia (FRDA). The secondary objective is to evaluate the efficacy of deferiprone for the treatment of FRDA, as assessed by a 9-Hole Peg Test (9HPT), Timed 25-Foot Walk (T25FW), Low-Contrast… Read more about this study
- Who is it for?
- Ages 7 to 35 years
- Conditions the study is for
- Friedreich's Ataxia
- What is being tested?
- deferiprone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 80 participants
- Where
- 6 sitesin 6 countries
- Study dates
- Apr 2008to Jul 2009
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 7 to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of FRDA, with confirmed mutation (excludes point mutation) in the frataxin (FXN) gene and GAA repeats ≥ 400 on the shorter allele.
- Males or females aged 7 to 35 years.
- No exposure to idebenone, coenzyme Q10, vitamin C, vitamin E or other antioxidants as a supplement or as a drug therapy for a period of at least one month prior to start of treatment and during the study.
- Neurological testing: A FARS score >20 and <85 at Screening and Baseline.
- Female subjects of childbearing potential must have a negative pregnancy test at Baseline.
- If the subject is a heterosexual, sexually-active male, he confirms 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 schedule.
Who cannot join
- Iron deficiency defined as ferritin levels below the reference range for age- and sex-matched controls
- Unable to complete T25FW AND with score > 5 minutes in the 9HPT. (Subjects who can complete T25FW or with a score ≤ 5 minutes in the 9HPT will be allowed to enroll if the score has not doubled compared to screening).
- Abnormal ALT, greater than 2.0 times the upper limit of normal on two consecutive assessments.
- Serum creatinine outside the normal reference range.
- History or evidence of neutropenia defined by an absolute neutrophil count (ANC) < 1.5 x 109/L or thrombocytopenia defined by a platelet count <150 x 109/L.
- Refusal to participate in screening procedures or unable to participate in screening procedures or unable to comply with the requirements of the protocol.
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- Receiving any investigational drug products or having received any investigational product within 30 days prior to enrollment into this study.
- Subjects who have previously taken deferiprone.
- Subjects who, in the opinion of the Investigator, represent poor medical, psychological or psychiatric risks, and for whom participation in an investigational trial would be unwise.
- Pregnant, breastfeeding or planning to become pregnant during the study period.
- History of malignancy.
- History of alcohol or drug abuse.
- Investigators, site personnel directly affiliated with this study and their immediate families. Immediate family is defined as a spouse, parent, child or sibling, whether biological or legally adopted.
- Hypersensitivity to the active substance (deferiprone) or any of the excipients in the oral solution.
- QT interval > 450 msec at Baseline.
Trial locations and contacts
6 sites in 6 countries.
6 sites in 6 countries
About this study
Official title: A Six-month Double-blind, Randomized, Placebo-controlled Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia
The primary objective of this study is to demonstrate the safety and tolerability of deferiprone in subjects with Friedreich's ataxia (FRDA).
The secondary objective is to evaluate the efficacy of deferiprone for the treatment of FRDA, as assessed by a 9-Hole Peg Test (9HPT), Timed 25-Foot Walk (T25FW), Low-Contrast Letter Acuity test (LCLA), International Cooperative Ataxia Rating Scale (ICARS), and Friedreich's Ataxia Rating Scale (FARS).
Read more
The tertiary objectives are to evaluate the effect of deferiprone on:
- cardiac function as measured by changes in Left Ventricular Shortening Fraction (LVSF), Left Ventricular Ejection Fraction (LVEF) and Left Ventricular (LV) mass using echocardiogram (ECHO),
- quality of life using quality-of-life surveys, and
- functional status using Activities of Daily Living (ADL).
Read the detailed description
This will be a multi-centre, double-blind, randomized, placebo-controlled clinical trial. A total of 80 patients with Friedreich's ataxia will be enrolled. Eligible patients will receive deferiprone oral solution or placebo at a total daily dose of 20 mg/kg/day, 40 mg/kg/day or 60 mg/kg/day, divided into two-daily doses for 6 months.
- Conditions
- Friedreich's Ataxia
- Being tested
- placebo, deferiprone
- Keywords
- Friedreich's ataxia
- Medical subject headings
- Friedreich Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
placeboDrug
Same dose and frequency as treatment
deferiproneDrug
100 mg/mL
deferiproneDrug
100 mg/mL
placeboDrug
Same dosage and frequency as study drug
deferiproneDrug
100 mg/mL
| Study group | Role | What they receive |
|---|---|---|
| A | Placebo comparator | Placebo solution Receives: Drug: placebo |
| B | Experimental | Deferiprone oral solution 20 mg/kg/day Receives: Drug: deferiprone |
| C | Experimental | Deferiprone oral solution 40 mg/kg/day Receives: Drug: deferiprone |
| D | Placebo comparator | Placebo solution Receives: Drug: placebo |
| E | Experimental | deferiprone oral solution 60 mg/kg/day Receives: Drug: deferiprone |
What is being measured
The primary outcome is the main question the study is designed to answer.
The patient's tolerance of treatment will be determined, as assessed by the occurrence of adverse events
Measured: 6 months
Secondary outcomes (1)
The efficacy endpoints will be change in the score for 9-Hole Peg Test (9HPT), Timed 25-Foot Walk (T25FW), Low-Contrast Letter Acuity test (LCLA), International Cooperative Ataxia Rating Scale (ICARS), and Friedreich's Ataxia Rating Scale (FARS).
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Sep 2007 (estimated)
- Study startApr 2008
- Primary completionJul 2009
- Study completionJul 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Friedreich's AtaxiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- ApoPharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Massimo Pandolfo, M.D., Principal investigator, Hospital Erasme, Brussels, BelgiumArnold Munnich, M.D., Principal investigator, Hospital Necker-Enfants Malades, Paris, FranceFranco Taroni, Principal investigator, Fondazione IRCCS Istituto Neurologico "C. Besta"Martin Delatycki, Principal investigator, Murdoch Children's Research Institute, Vicotria, AustraliaJavier Arpa, Principal investigator, La Fundaction Para la Investigacion Biomedica, Madrid, SpainMark Tarnopolsky, MD, Principal investigator, McMaster University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00530127
- Sponsor's protocol ID
- LA29-0207
- Status last verified
- May 2010
- First submitted
- 13 Sep 2007
- Last updated
- 2 Jun 2010
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 2 Jun 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Six-month Double-blind, Randomized, Placebo-controlled Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia
- ClinicalTrials.gov ID
- NCT00530127
- Acronym / sponsor ID
- LA29-0207
- Conditions
- Friedreich's Ataxia
- Keywords
- Friedreich's ataxia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- ApoPharmaIndustry (pharma, biotech, devices)
- Study start
- Apr 2008
- Primary completion
- Jul 2009 (actual)
- Study completion
- Jul 2009 (actual)
- First posted
- 17 Sep 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of this study is to demonstrate the safety and tolerability of deferiprone in subjects with Friedreich's ataxia (FRDA).
The secondary objective is to evaluate the efficacy of deferiprone for the treatment of FRDA, as assessed by a 9-Hole Peg Test (9HPT), Timed 25-Foot Walk (T25FW), Low-Contrast Letter Acuity test (LCLA), International Cooperative Ataxia Rating Scale (ICARS), and Friedreich's Ataxia Rating Scale (FARS).
The tertiary objectives are to evaluate the effect of deferiprone on:
- cardiac function as measured by changes in Left Ventricular Shortening Fraction (LVSF), Left Ventricular Ejection Fraction (LVEF) and Left Ventricular (LV) mass using echocardiogram (ECHO),
- quality of life using quality-of-life surveys, and
- functional status using Activities of Daily Living (ADL).
Detailed description
This will be a multi-centre, double-blind, randomized, placebo-controlled clinical trial. A total of 80 patients with Friedreich's ataxia will be enrolled. Eligible patients will receive deferiprone oral solution or placebo at a total daily dose of 20 mg/kg/day, 40 mg/kg/day or 60 mg/kg/day, divided into two-daily doses for 6 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| A | Placebo comparator | Drug: placeboPlacebo solution |
| B | Experimental | Drug: deferiproneDeferiprone oral solution 20 mg/kg/day |
| C | Experimental | Drug: deferiproneDeferiprone oral solution 40 mg/kg/day |
| D | Placebo comparator | Drug: placeboPlacebo solution |
| E | Experimental | Drug: deferipronedeferiprone oral solution 60 mg/kg/day |
Who may qualify
- Ages
- 7 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Diagnosis of FRDA, with confirmed mutation (excludes point mutation) in the frataxin (FXN) gene and GAA repeats ≥ 400 on the shorter allele.
- Males or females aged 7 to 35 years.
- No exposure to idebenone, coenzyme Q10, vitamin C, vitamin E or other antioxidants as a supplement or as a drug therapy for a period of at least one month prior to start of treatment and during the study.
- Neurological testing: A FARS score >20 and <85 at Screening and Baseline.
- Female subjects of childbearing potential must have a negative pregnancy test at Baseline.
Key exclusion criteria (15 listed)
- Iron deficiency defined as ferritin levels below the reference range for age- and sex-matched controls
- Unable to complete T25FW AND with score > 5 minutes in the 9HPT. (Subjects who can complete T25FW or with a score ≤ 5 minutes in the 9HPT will be allowed to enroll if the score has not doubled compared to screening).
- Abnormal ALT, greater than 2.0 times the upper limit of normal on two consecutive assessments.
- Serum creatinine outside the normal reference range.
- History or evidence of neutropenia defined by an absolute neutrophil count (ANC) < 1.5 x 109/L or thrombocytopenia defined by a platelet count <150 x 109/L.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| placeboSame dose and frequency as treatment | Drug | A |
| deferiprone100 mg/mL | Drug | B |
| deferiprone100 mg/mL | Drug | C |
| placeboSame dosage and frequency as study drug | Drug | D |
| deferiprone100 mg/mL | Drug | E |
Primary outcome
The patient's tolerance of treatment will be determined, as assessed by the occurrence of adverse events
Time frame: 6 months
1 secondary outcome
The efficacy endpoints will be change in the score for 9-Hole Peg Test (9HPT), Timed 25-Foot Walk (T25FW), Low-Contrast Letter Acuity test (LCLA), International Cooperative Ataxia Rating Scale (ICARS), and Friedreich's Ataxia Rating Scale (FARS).
Time frame: 6 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jun 2010.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Massimo Pandolfo, M.D. principal investigator
Hospital Erasme, Brussels, Belgium
Arnold Munnich, M.D. principal investigator
Hospital Necker-Enfants Malades, Paris, France
Franco Taroni principal investigator
Fondazione IRCCS Istituto Neurologico "C. Besta"
6 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing deferiprone in people with Friedreich's Ataxia, run by ApoPharma at 6 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Jun 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Australia, Belgium, Canada, France, Italy and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The patient's tolerance of treatment will be determined, as assessed by the occurrence of adverse events (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Jun 2010.