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

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified January 2014 by Azienda Policlinico Umberto I.
Recruitment status was:  Recruiting
Information provided by (Responsible Party):
Carlo Casali, Azienda Policlinico Umberto I Identifier:
First received: January 12, 2014
Last updated: January 13, 2014
Last verified: January 2014
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.

Condition Intervention Phase
Friedreich Ataxia.
Drug: gamma interferon
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase IIa Clinical Trial to Test the Safety and Efficacy of Interferon Gamma Treatment in Elevating Frataxin Levels in Friedreich's Ataxia (FRDA) Patients

Resource links provided by NLM:

Further study details as provided by Azienda Policlinico Umberto I:

Primary Outcome Measures:
  • Change in cellular frataxin [ Time Frame: 24 hours and 7 days from each study drug administration ]
    The primary endpoint is to test the increase of cellular frataxin after treatment with IFN gamma. Quantitation of cellular frataxin will be performed after 24 hours and 7 days from each study drug administration

Secondary Outcome Measures:
  • Safety Blood sample [ Time Frame: day 0-14-28-35 ]
    Secondary endpoint is 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

Estimated Enrollment: 10
Study Start Date: May 2013
Estimated Study Completion Date: December 2014
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Gamma interferon
IFN gamma 1b (Immukin ®) will be administered by subcutaneous route at day 0, 14 and 28 at a dose of 100, 150 and 200 ug respectively.
Drug: gamma interferon
IFN gamma 1b (Immukin ®) will be administered by subcutaneous route at day 0, 14 and 28 at a dose of 100, 150 and 200 ug respectively.
Other Name: Imukin

Detailed Description:
This is a Phase 2 clinical trial. A total of 10 FRDA patients will be recruited All subjects will be treated with a dose of 100-150-200-micrograms of IFN gamma 1b (Imukin®) subcutaneously, with an interval of 14 days, for a total of 3 injections.

Ages Eligible for Study:   18 Years to 45 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

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.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT02035020

Contact: Barbara Tomassini, MD ++39-3387504282
Contact: Carlo Casali, PhD +39-3355247345

Policlinico Umberto I° Recruiting
Rome, Italy/Rome, Italy, 00161
Contact: Carlo Casali, MD, Prof.   
Contact: Barbara Tomassini, PhD    +39-3387504282   
Principal Investigator: Carlo Casali, PROF         
Sponsors and Collaborators
Azienda Policlinico Umberto I
Principal Investigator: Carlo Casali, MD Policlinico Umberto I°
  More Information

Responsible Party: Carlo Casali, Professor, Azienda Policlinico Umberto I Identifier: NCT02035020     History of Changes
Other Study ID Numbers: GIFT/1
Study First Received: January 12, 2014
Last Updated: January 13, 2014

Keywords provided by Azienda Policlinico Umberto I:
frataxin, gamma interferon, Friedreich ataxia

Additional relevant MeSH terms:
Cerebellar Ataxia
Friedreich Ataxia
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinocerebellar Degenerations
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Antineoplastic Agents
Antiviral Agents
Anti-Infective Agents processed this record on March 30, 2017