Overview

A Phase IIa Trial to Test Safety and Efficacy Interferon Gamma Treatment in Elevating Frataxin Levels in FRDA Patients

Status:
Completed
Trial end date:
2014-07-30
Target enrollment:
0
Participant gender:
All
Summary
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.
Phase:
Phase 2
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Azienda Policlinico Umberto I
Treatments:
Interferon-gamma
Interferons
Criteria
Inclusion Criteria:

1. FRDA patients should have their diagnosis genetically confirmed.

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 between the ages of > 18 and < 45 years

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Exclusion Criteria:

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.

5. Abnormality in any of the below hematology or chemistry profile values at screening:

- Positive hepatitis B surface antigen (HBsAg), Total hepatitis B core antibody
(HBcAb; also called anti HBc), and/or hepatitis C antibody (HCVAb) with
confirmation by hepatitis C virus ribonucleic acid (HCV RNA).

- ALT/AST levels > or = 1.5X ULN.

- Total bilirubin level > or = 1.5 times the ULN.

- Hemoglobin level < or = 80 gL (8.0 g/dL).

- Platelet count < or = 100 x 109/L (100,000 cells/mm³) or > or = 1000 x 109/L
(1,000,000 cells/mm³).

- White blood cell count < or = 3.5 x 109/L (3500 cells/mm³).

- Absolute neutrophil count (ANC) <2000 cells/mm³.

- Serum creatinine level > or = 177 μmol/ L (2 mg/dL).

- Glycosylated hemoglobin (HbA1c >10%).

6. Current or history of serious psychiatric disorder or alcohol or drug abuse.

7. Participation in other studies within 30 days before screening and/or during study
participation.

8. Other severe acute or chronic medical or psychiatric condition or laboratory
abnormality that may increase the risk associated with study participation or ability
to comply with study procedures, investigational product administration or may
interfere with the interpretation of study results and, in the judgment of the
investigator, would make the subject inappropriate for entry into this study.

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