Trial · NCT01965327
Interferon Gamma-1b in Friedreich Ataxia (FRDA)
NCT01965327, Phase 2, Completed, Children's Hospital of Philadelphia; results first posted 2015-04-30
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Interferon Gamma-1b in Friedreich Ataxia (FRDA). Trials, record NCT01965327. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01965327
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Interferon Gamma-1b in Friedreich Ataxia (FRDA)
- NCT number
- NCT01965327
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 12 participants, actual
- Lead sponsor
- Children's Hospital of Philadelphia
- Conditions
- Friedreich Ataxia
- Interventions
- Interferon Gamma-1b
- First posted
- 2013-10-18
- Last updated
- 2021-04-13
- Results first posted
- 2015-04-30
- Limitations and caveats
- This study was performed in a small population of children with Friedreich ataxia (n=12) and without a placebo arm.
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in Whole Blood Frataxin Levels | Frataxin levels were measured at the beginning and conclusion of treatment (baseline and 12 weeks) | 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. |
| Secondary | Change in Total Friedreich Ataxia Rating Scale (FARS) Score | FARS score was calculated at the beginning and conclusion of treatment (baseline and 12 weeks) | 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. |
Source ClinicalTrials.gov
Outcomes2
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change in Whole Blood Frataxin Levels | Frataxin levels were measured at the beginning and conclusion of treatment (baseline and 12 weeks) | The primary analysis was based on an intent-to-treat approach including all subjects who had baseline frataxin blood levels collected. | percentage of baseline frataxin level | MEAN |
| SECONDARY | Change in Total Friedreich Ataxia Rating Scale (FARS) Score | FARS score was calculated at the beginning and conclusion of treatment (baseline and 12 weeks) | The analysis was based on an intent-to-treat approach and included all subjects with baseline FARS assessment. | units on a scale | MEAN |
Source ClinicalTrials.gov
Results by group2
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change in Whole Blood Frataxin Levels | Interferon Gamma-1b (ACTIMMUNE) | -1.5 | Standard Deviation 1.9 | percentage of baseline frataxin level | |
| Change in Total Friedreich Ataxia Rating Scale (FARS) Score | Interferon Gamma-1b (ACTIMMUNE) | -4.98 | Standard Deviation 3.6 | units on a scale |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Interferon Gamma-1b (ACTIMMUNE) | 12 | |
| Overall Study | COMPLETED | Interferon Gamma-1b (ACTIMMUNE) | 10 | |
| Overall Study | NOT COMPLETED | Interferon Gamma-1b (ACTIMMUNE) | 2 |
Source ClinicalTrials.gov
Adverse events21
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Absominal Pain | Gastrointestinal disorders | Interferon Gamma-1b (ACTIMMUNE) | 2 | 12 | |
| other | Diarrhea | Gastrointestinal disorders | Interferon Gamma-1b (ACTIMMUNE) | 1 | 12 | |
| other | Nausea | Gastrointestinal disorders | Interferon Gamma-1b (ACTIMMUNE) | 7 | 12 | |
| other | Vomiting | Gastrointestinal disorders | Interferon Gamma-1b (ACTIMMUNE) | 4 | 12 | |
| other | Chills | General disorders | Interferon Gamma-1b (ACTIMMUNE) | 1 | 12 | |
| other | Fatigue | General disorders | Interferon Gamma-1b (ACTIMMUNE) | 3 | 12 | |
| other | Influenza like illness | Infections and infestations | Interferon Gamma-1b (ACTIMMUNE) | 6 | 12 | |
| other | Pyrexia | General disorders | Interferon Gamma-1b (ACTIMMUNE) | 2 | 12 | |
| other | Contusion | Skin and subcutaneous tissue disorders | Interferon Gamma-1b (ACTIMMUNE) | 1 | 12 | |
| other | Elevated transaminases | Investigations | Interferon Gamma-1b (ACTIMMUNE) | 1 | 12 |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Interferon Gamma-1b (ACTIMMUNE) | ||
| Total, other adverse events | Interferon Gamma-1b (ACTIMMUNE) | 11 | 12 |
| Total, serious adverse events | Interferon Gamma-1b (ACTIMMUNE) | 0 | 12 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01965327
GET https://conductscience.org/api/trials/v1/NCT01965327/events?page=2