A phase II clinical trial to evaluate the safety and efficacy of interferon gamma in elevating frataxin levels in Friedreich ataxia patients

• Conditions: Friedreich's Ataxia; MedDRA version: 14.1Level: SOCClassification code 10029205Term: Nervous system disordersSystem Organ Class: 10029205 - Nervous system disorders; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2012-001881-14-IT
• Lead Sponsor: AZIENDA UNIVERSITARIA POLICLINICO UMBERTO I DI ROMA

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-01-11
• Last Update Posted: 27 January 2014

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

1. Genetically confirmed diagnosis of Friederich’s Ataxia 2. Evidence of a personally signed and dated informed consent document indicating that the subject (or a legally acceptable representative) has been informed of all pertinent aspects of the study. 3. Subjects who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures. 4. Male and/or female subjects >/=18 .
Are the trial subjects under 18? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 15
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Subjects presenting with any of the following will not be included in the study: Medical History 1. Pregnant or breastfeeding women. 2. Significant concurrent medical conditions at the time of screening or baseline visit, including, but not limited to, the following: • Any major illness/condition or evidence of an unstable clinical condition (eg, renal, hepatic, hematologic, GI, endocrine, pulmonary, immunologic, or local active infection/infectious illness) that, in the investigator’s judgment, will substantially increase the risk to the subject if he or she participates in the study. • Class III or IV congestive heart failure as defined by the New York Heart Association. • Acute coronary syndrome (eg, myocardial infarction, unstable angina pectoris) and any history of significant cerebrovascular disease within 24 weeks before screening. 3. Presence of a transplanted organ. 4. Previous assumption of IFN gamma 1b.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective of this study is to investigate whether the treatment with IFN gamma can induce significant accumulation of frataxin in FRDA patients, a possibility suggested by pre-clinical evidence in an animal model of the disease.;Secondary Objective: Secondary objectives include the assessment of the safety and tolerability of IFN gamma in FRDA patients during the treatment period.;Primary end point(s): The primary endpoint is the increase of cellular frataxin after treatment with IFN gamma.;Timepoint(s) of evaluation of this end point: Blood sample for the quantitation of frataxin will include in total 9 blood samples. Blood samples will be taken at the beginning of the treatment, 0 timepoint, and at days 1-7-14-15-21-28-29-35.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Secondary endpoints are the safety and tolerability of IFN gamma in FRDA patients. The on treatment adverse events and withdrawals due to adverse effects will be reported. Any subject who receives at least 1 dose of investigational product will be included in the evaluation for safety.;Timepoint(s) of evaluation of this end point: Patients will be evaluated at the inclusion and after 14, 28 and 58 days.