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A Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia

Primary Purpose

Friedreich's Ataxia

Status

Completed

Phase

Phase 1

Locations

International

Study Type

Interventional

Intervention

placebo

deferiprone

placebo

deferiprone

About this trial

This is an interventional treatment trial for Friedreich's Ataxia focused on measuring Friedreich's ataxia

Eligibility Criteria

7 Years - 35 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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.

Exclusion Criteria:

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.
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.

Sites / Locations

Murdoch Children's Research Institute
Hospital Erasme
McMaster University
Hospital Necker-Enfants Malades
Fondazione IRCCS Istituto Neurologico "C. Besta"
La Fundacion Para la Investigacion Biomedica

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm Type

Placebo Comparator

Experimental

Placebo Comparator

Experimental

Arm Label

Arm Description

Placebo solution

Deferiprone oral solution 20 mg/kg/day

Deferiprone oral solution 40 mg/kg/day

Placebo solution

deferiprone oral solution 60 mg/kg/day

Outcomes

Primary Outcome Measures

The patient's tolerance of treatment will be determined, as assessed by the occurrence of adverse events

Secondary Outcome Measures

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).

Full Information

NCT ID

NCT00530127

First Posted

September 13, 2007

Last Updated

May 31, 2010

Sponsor

ApoPharma

1. Study Identification

Unique Protocol Identification Number

NCT00530127

Brief Title

A Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia

Official Title

A Six-month Double-blind, Randomized, Placebo-controlled Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia

Study Type

Interventional

2. Study Status

Record Verification Date

May 2010

Overall Recruitment Status

Completed

Study Start Date

April 2008 (undefined)

Primary Completion Date

July 2009 (Actual)

Study Completion Date

July 2009 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor

ApoPharma

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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: 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Friedreich's Ataxia

Keywords

Friedreich's ataxia

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantInvestigator

Allocation

Randomized

Enrollment

80 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Arm Type

Placebo Comparator

Arm Description

Placebo solution

Arm Title

Arm Type

Experimental

Arm Description

Deferiprone oral solution 20 mg/kg/day

Arm Title

Arm Type

Experimental

Arm Description

Deferiprone oral solution 40 mg/kg/day

Arm Title

Arm Type

Placebo Comparator

Arm Description

Placebo solution

Arm Title

Arm Type

Experimental

Arm Description

deferiprone oral solution 60 mg/kg/day

Intervention Type

Drug

Intervention Name(s)

placebo

Intervention Description

Same dose and frequency as treatment

Intervention Type

Drug

Intervention Name(s)

deferiprone

Intervention Description

100 mg/mL

Intervention Type

Drug

Intervention Name(s)

deferiprone

Intervention Description

100 mg/mL

Intervention Type

Drug

Intervention Name(s)

placebo

Intervention Description

Same dosage and frequency as study drug

Intervention Type

Drug

Intervention Name(s)

deferiprone

Intervention Description

100 mg/mL

Primary Outcome Measure Information:

Title

The patient's tolerance of treatment will be determined, as assessed by the occurrence of adverse events

Time Frame

6 months

Secondary Outcome Measure Information:

Title

Time Frame

6 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

7 Years

Maximum Age & Unit of Time

35 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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. Exclusion Criteria: 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. 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.

Overall Study Officials: