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

NCT02035020 | Completed Phase 2

• 1 other identifier: GIFT/1
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Friedreich Ataxia

Keywords

frataxin, gamma interferon, Friedreich ataxia

Outcome Measures

Primary Outcomes (1)

• Change in cellular frataxin

  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

  24 hours and 7 days from each study drug administration

Secondary Outcomes (1)

• Safety Blood sample

  day 0-14-28-35

Study Arms (1)

Gamma interferon

EXPERIMENTAL

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

Interventions

gamma interferon DRUG

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.

Also known as: Imukin

Gamma interferon

Eligibility Criteria

• Age: 18 Years - 45 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• FRDA patients should have their diagnosis genetically confirmed.
• 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.
• Subjects who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
• Male and/or female subjects between the ages of \> 18 and \< 45 years

You may not qualify if:

• Pregnant or breastfeeding women.
• 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.
• Presence of a transplanted organ.
• Previous assumption of IFN gamma 1b.
• 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.

Sponsors & Collaborators

• Azienda Policlinico Umberto I: lead

Study Sites (1)

Policlinico Umberto I°

Rome, Italy/Rome, 00161, Italy

Location

Related Publications (1)

• Tomassini B, Arcuri G, Fortuni S, Sandi C, Ezzatizadeh V, Casali C, Condo I, Malisan F, Al-Mahdawi S, Pook M, Testi R. Interferon gamma upregulates frataxin and corrects the functional deficits in a Friedreich ataxia model. Hum Mol Genet. 2012 Jul 1;21(13):2855-61. doi: 10.1093/hmg/dds110. Epub 2012 Mar 23.

  PMID: 22447512 BACKGROUND

MeSH Terms

Conditions

Friedreich Ataxia

Interventions

Interferon-gamma

Condition Hierarchy (Ancestors)

Spinocerebellar Degenerations; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Interferons; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Macrophage-Activating Factors; Lymphokines; Proteins; Biological Factors

Study Officials

• Carlo Casali, MD

  Policlinico Umberto I°

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: January 12, 2014
• First Posted: January 14, 2014
• Study Start: May 1, 2013
• Primary Completion: July 30, 2014
• Study Completion: July 30, 2014
• Last Updated: April 21, 2017

Record last verified: 2017-04

Locations

IT (1)