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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00530127




Registration number
NCT00530127
Ethics application status
Date submitted
13/09/2007
Date registered
17/09/2007
Date last updated
2/06/2010

Titles & IDs
Public title
A Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia
Scientific title
A Six-month Double-blind, Randomized, Placebo-controlled Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia
Secondary ID [1] 0 0
LA29-0207
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Friedreich's Ataxia 0 0
Condition category
Condition code
Neurological 0 0 0 0
Other neurological disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Neurodegenerative diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - placebo
Treatment: Drugs - deferiprone
Treatment: Drugs - deferiprone
Treatment: Drugs - placebo
Treatment: Drugs - deferiprone

Placebo comparator: A - Placebo solution

Experimental: B - Deferiprone oral solution 20 mg/kg/day

Experimental: C - Deferiprone oral solution 40 mg/kg/day

Placebo comparator: D - Placebo solution

Experimental: E - deferiprone oral solution 60 mg/kg/day


Treatment: Drugs: placebo
Same dose and frequency as treatment

Treatment: Drugs: deferiprone
100 mg/mL

Treatment: Drugs: deferiprone
100 mg/mL

Treatment: Drugs: placebo
Same dosage and frequency as study drug

Treatment: Drugs: deferiprone
100 mg/mL
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
The patient's tolerance of treatment will be determined, as assessed by the occurrence of adverse events
Timepoint [1] 0 0
6 months
Secondary outcome [1] 0 0
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).
Timepoint [1] 0 0
6 months

Eligibility
Key inclusion criteria
1. Diagnosis of FRDA, with confirmed mutation (excludes point mutation) in the frataxin (FXN) gene and GAA repeats = 400 on the shorter allele.
2. Males or females aged 7 to 35 years.
3. 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.
4. Neurological testing: A FARS score >20 and <85 at Screening and Baseline.
5. Female subjects of childbearing potential must have a negative pregnancy test at Baseline.
6. 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.
7. 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.
Minimum age
7 Years
Maximum age
35 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Iron deficiency defined as ferritin levels below the reference range for age- and sex-matched controls
2. 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).
3. Abnormal ALT, greater than 2.0 times the upper limit of normal on two consecutive assessments.
4. Serum creatinine outside the normal reference range.
5. 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.
6. Refusal to participate in screening procedures or unable to participate in screening procedures or unable to comply with the requirements of the protocol.
7. Receiving any investigational drug products or having received any investigational product within 30 days prior to enrollment into this study.
8. Subjects who have previously taken deferiprone.
9. 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.
10. Pregnant, breastfeeding or planning to become pregnant during the study period.
11. History of malignancy.
12. History of alcohol or drug abuse.
13. 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.
14. Hypersensitivity to the active substance (deferiprone) or any of the excipients in the oral solution.
15. QT interval > 450 msec at Baseline.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/04/2008
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/07/2009
Sample size
Target
80
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Murdoch Children's Research Institute - Victoria
Recruitment postcode(s) [1] 0 0
3052 - Victoria
Recruitment outside Australia
Country [1] 0 0
Belgium
State/province [1] 0 0
Brussels
Country [2] 0 0
Canada
State/province [2] 0 0
Ontario
Country [3] 0 0
France
State/province [3] 0 0
Paris
Country [4] 0 0
Italy
State/province [4] 0 0
Milan
Country [5] 0 0
Spain
State/province [5] 0 0
Madrid

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
ApoPharma
Address
Country

Ethics approval
Ethics application status

Summary
Brief 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 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:

1. 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),
2. quality of life using quality-of-life surveys, and
3. functional status using Activities of Daily Living (ADL).
Trial website
https://clinicaltrials.gov/study/NCT00530127
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Massimo Pandolfo, M.D.
Address 0 0
Hospital Erasme, Brussels, Belgium
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT00530127