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NCT01965327 · ClinicalTrials.gov registry record · Phase 2
Interferon Gamma-1b in Friedreich Ataxia (FRDA)
A Phase 2 study, sponsored by Children's Hospital of Philadelphia.
- Completed
- Registry status
- Phase 2
- Development phase
- 12
- Enrollment target
NCT01965327: Completed Phase 2 study, sponsored by Children's Hospital of Philadelphia.
NCT01965327 is a Phase 2 study that has completed, run by Children's Hospital of Philadelphia. The registered enrollment target is 12 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (91% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01965327, a Phase 2 study, has completed, sponsored by Children's Hospital of Philadelphia.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 12 participants
- Enrollment target
Study 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.
Primary Outcome
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.
Interventions
- DRUG Interferon Gamma-1b
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2013-08 |
| Est. Completion | 2014-10 |
| Phase | Phase 2 |
What the finished NCT01965327 record still lists
NCT01965327 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (91% lower).
The record links to 0 conditions, and to 1 intervention - of which Interferon Gamma-1b is the first listed.
NCT01965327 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01965327 about?
NCT01965327 is a clinical study titled "Interferon Gamma-1b in Friedreich Ataxia (FRDA)". 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 mo...
What is the current status of trial NCT01965327?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 12 participants. The study started on 2013-08. Estimated completion is 2014-10.
What interventions are being tested in trial NCT01965327?
The interventions under investigation include: Interferon Gamma-1b (DRUG).
Who is sponsoring clinical trial NCT01965327?
This trial is sponsored by Children's Hospital of Philadelphia, which has 431 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01965327's enrollment target sits among peer trials
12 1948th of 2000 higher than 34 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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