Phase 2
N=12
Interferon Gamma-1b in Friedreich Ataxia (FRDA)
Friedreich Ataxia
Bottom Line
View on ClinicalTrials.gov: NCT01965327 ↗Enrolled (actual)
12
Serious AEs
0.0%
Results posted
Apr 2015
Primary outcome: Primary: Change in Whole Blood Frataxin Levels — -1.5 percentage of baseline frataxin level — p=0.027
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Interferon Gamma-1b (Drug)
- Age
- Pediatric · 5+ yrs
- Sex
- All
- Sponsor
- Children's Hospital of Philadelphia
- Primary completion
- Mar 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Whole Blood Frataxin Levels |
-1.5 | 0.027 sig |
| SECONDARY Change in Total Friedreich Ataxia Rating Scale (FARS) Score |
-4.98 | 0.0078 sig |
Summary
Friedreich ataxia (FRDA) is a progressive neurodegenerative disease of children and adults for which there is presently no therapy. Recently, a study reported that interferon gamma (IFN-g) could raise frataxin protein levels in both cell lines derived from patients with Friedreich ataxia and in a mouse model with Friedreich ataxia. The present study will test whether IFN-g is safe, tolerated and potentially efficacious in a heterogeneous cohort of children with FRDA.
Eligibility Criteria
Inclusion Criteria
- Subjects with FRDA confirmed by genetic testing with 2 expanded Guanine-adenine-adenine repeats
- Females who are not pregnant or breast feeding, and who do not intend to become pregnant. Females of child-bearing potential must use a reliable method of contraception and must provide a negative urine pregnancy test at screening
- Stable doses of all medications, vitamins and supplements for 30 days prior to study entry and for the duration of the study
- Parent/guardian permission (informed consent) and child assent
Exclusion Criteria
- Any unstable illness that in the investigator's opinion precludes participation in this study
- Use of any investigational product within 30 days prior to enrollment
- Subjects with a history of substance abuse
- Presence of clinically significant cardiac disease
- History of hypersensitivity to IFN-g or E. coli derived products
- Presence of severe renal disease or hepatic disease
- Clinically significant abnormal White blood cell count, hemoglobin or platelet count
- Any subject planning a scheduled surgical procedure during the study
Data sourced from ClinicalTrials.gov (NCT01965327). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.