A Phase 2 interventional study of Interferon Gamma-1b in Friedreich Ataxia, sponsored by Children's Hospital of Philadelphia. Completed at 1 site in United States. Open to participants aged 5 Years to 17 Years. Per ClinicalTrials.gov, last updated 2021-04-13.
Sponsored by Children's Hospital of Philadelphia · Phase 2, Interventional, and Treatment
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.
Study Objectives:
Primary:
Secondary:
Study Phases:
Screening - During screening, subjects will be assessed for inclusion and exclusion criteria.
Intervention - Subjects will begin treatment at baseline visit and the dose of study medication will be increased to the maximum dose over four weeks. The subjects will be maintained at the maximum dose for 8 weeks. After 12 weeks, treatment will stop. Study medication will be administered via subcutaneous injections three times per week for 12 weeks.
Follow-up - Follow-up visits will occur at 7 and 28 days after the subject has completed the 12 weeks of active treatment.
295 studies on the registry are indexed under Ataxia; 51 are open to participants now.
This study's enrollment of 12 is below the median of 26 across 216 interventional studies indexed under Ataxia.
Browse Ataxia studies →Children's Hospital of Philadelphia is the lead sponsor of 480 studies on the registry; 85 are open to participants now.
Of its 28 completed or terminated interventional studies of FDA-regulated products, 22 (79%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
All individuals in this study will be given active medication (interferon gamma-1b) for 12 weeks. This will be administered according to a dose-escalation schedule.
Drug: Interferon Gamma-1b
Subjects will begin by taking 10 mcg/m2 of IFN-g-1b for the first two weeks of the study. Dose will be escalated to 25 mcg/m2 of IFN-g-1b for weeks three and four of the study. Finally, the dose will be escalated to 50 mcg/m2 of IFN-g-1b for the last eight weeks of the study, which is the current dose approved by the FDA for children. All doses will be administered via subcutaneous injection.
Also known as: Actimmune™, IFN-g-1b
Change in Whole Blood Frataxin Levels
Assessment of the change in whole blood frataxin levels as assessed by lateral flow assay using an immunoassay for frataxin. Frataxin levels in the blood were measured at each study visit. Change in frataxin level at the end of treatment (week 12) relative to frataxin level at baseline was analyzed.
Time frame: Frataxin levels were measured at the beginning and conclusion of treatment (baseline and 12 weeks)
Change in Total Friedreich Ataxia Rating Scale (FARS) Score
The Friedreich Ataxia Rating Scale (FARS) is neurological rating scale specifically developed and validated for FRDA. The FARS includes assessments of stance, gait, upper and lower limb coordination, speech, proprioception and strength. In addition to the standard neurological examination, the FARS contains three quantitative performance measures and a component that assesses activities of daily living (ADL). Quantitative performance measures include the nine-hole peg test, and a timed 25-foot walk. FARS scores correlate significantly with functional disability, activities of daily living scores and disease duration. The scores from the three subscales are added to generate a total score ranging from 0 to 159, with a higher score indicating a greater level of disability.
Time frame: FARS score was calculated at the beginning and conclusion of treatment (baseline and 12 weeks)
12 subjects were screened and all were enrolled in the study between September - December 2013.
| Milestone | Interferon Gamma-1b (ACTIMMUNE) |
|---|---|
| Started | 12 |
| Completed | 10 |
| Not completed | 2 |
| Withdrew: Physician decision | 2 |
Assessment of the change in whole blood frataxin levels as assessed by lateral flow assay using an immunoassay for frataxin. Frataxin levels in the blood were measured at each study visit. Change in frataxin level at the end of treatment (week 12) relative to frataxin level at baseline was analyzed.
| percentage of baseline frataxin level | Interferon Gamma-1b (ACTIMMUNE) |
|---|---|
| Change in Whole Blood Frataxin Levels | -1.5 ± 1.9 |
The Friedreich Ataxia Rating Scale (FARS) is neurological rating scale specifically developed and validated for FRDA. The FARS includes assessments of stance, gait, upper and lower limb coordination, speech, proprioception and strength. In addition to the standard neurological examination, the FARS contains three quantitative performance measures and a component that assesses activities of daily living (ADL). Quantitative performance measures include the nine-hole peg test, and a timed 25-foot walk. FARS scores correlate significantly with functional disability, activities of daily living scores and disease duration. The scores from the three subscales are added to generate a total score ranging from 0 to 159, with a higher score indicating a greater level of disability.
| units on a scale | Interferon Gamma-1b (ACTIMMUNE) |
|---|---|
| Change in Total Friedreich Ataxia Rating Scale (FARS) Score | -4.98 ± 3.6 |
Collected over 16 weeks. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Interferon Gamma-1b (ACTIMMUNE) | — | 0/12 (0%) | 11/12 (91.7%) |
| Event | Interferon Gamma-1b (ACTIMMUNE) |
|---|---|
| NauseaGastrointestinal disorders | 7/12 |
| Influenza like illnessInfections and infestations | 6/12 |
| VomitingGastrointestinal disorders | 4/12 |
| MyalgiaGeneral disorders | 4/12 |
| Injection Site BruisingGeneral disorders | 4/12 |
| FatigueGeneral disorders | 3/12 |
| HeadacheGeneral disorders | 3/12 |
| Injection Site PainInvestigations | 3/12 |
| Absominal PainGastrointestinal disorders | 2/12 |
| PyrexiaGeneral disorders | 2/12 |
| Age, Categorical(Participants) | Interferon Gamma-1b (ACTIMMUNE) |
|---|---|
| <=18 years | 12 |
| Between 18 and 65 years | 0 |
| >=65 years | 0 |
| Age, Continuous(years) | Interferon Gamma-1b (ACTIMMUNE) |
|---|---|
| Mean | 12 (8 to 17) |
| Sex: Female, Male(Participants) | Interferon Gamma-1b (ACTIMMUNE) |
|---|---|
| Female | 3 |
| Male | 9 |
| Ethnicity (NIH/OMB)(Participants) | Interferon Gamma-1b (ACTIMMUNE) |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 12 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | Interferon Gamma-1b (ACTIMMUNE) |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 1 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 11 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(participants) | Interferon Gamma-1b (ACTIMMUNE) |
|---|---|
| United States | 12 |
This study is completed, as verified in Mar 2021. You cannot join it, but the record below documents what was studied.
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