A Study Investigating the Long-term Safety and Efficacy o... | NCT00897221

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

NCT00897221

Sponsor

ApoPharma

Status

Completed

Last Update Posted

Jun 27, 2011Estimated

Enrollment

36Actual

Phase

Phase 2

Conditions

Friedreich's Ataxia

Interventions

Deferiprone oral solution 100mg/mL
Deferiprone oral solution 100 mg/mL

Countries

Belgium
France
Italy
Spain

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Jun 2011

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jun 2009

Primary Completion Date

Mar 2011Actual

Completion Date

Mar 2011Actual

First Submitted Date

May 8, 2009

First Submission Date that Met QC Criteria

May 8, 2009

First Posted Date

May 12, 2009Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jun 24, 2011

Last Update Posted Date

Jun 27, 2011Estimated

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief 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:

cardiac function,
quality of life, and
functional status.

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

36Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Dose 1	Experimental	Deferiprone oral solution 20 mg/kg/day	Drug: Deferiprone oral solution 100mg/mL
Dose 2	Experimental	Deferiprone oral solution 40 mg/kg/day	Drug: Deferiprone oral solution 100 mg/mL

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Deferiprone oral solution 100mg/mL	Drug	Deferiprone oral solution (20 mg/kg/day)	Dose 1

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
The patient's long-term tolerance of treatment will be assessed by the occurence of adverse events.		52 weeks

Secondary Outcomes

Measure	Description	Time Frame
The long-term efficacy of deferiprone will be assessed. Efficacy measures include the 9HPT, T25FW, LCLA, ICARS and FARS.		52 weeks

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

7 Years

Maximum Age

35 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

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

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Hospital Erasme		Brussels			Belgium
Hospital Necker-Enfants Malades

ID	Term
D013132	Spinocerebellar Degenerations
D002526	Cerebellar Diseases
D001927	Brain Diseases
D002493	Central Nervous System Diseases

ID	Term
D011728	Pyridones
D011725	Pyridines
D006573	Heterocyclic Compounds, 1-Ring
D006571	Heterocyclic Compounds