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Clinical trial intelligence report
A Phase 2 Clinical Trial to Test the Safety and Efficacy of Etravirine in Friedreich Ataxia Patients
Source-linked diligence brief with registry provenance, taxonomy normalization and premium analytical context.
Generated
Jul 28, 2026
Report code
NCT04273165-Jul 28, 2026
NCT ID
NCT04273165
Status
COMPLETED
Phase
Phase 2
Sponsor
IRCCS Eugenio Medea
Executive brief
Investment-Ready Snapshot
Etravirine, an FDA-approved non-nucleoside reverse transcriptase inhibitor for HIV, is being repositioned for the treatment of Friedreich Ataxia (FA), a rare neurodegenerative disorder with no current approved therapies. The trial aims to assess its safety and efficacy in a small cohort of FA patients, potentially opening a new therapeutic avenue in a niche market. Given the favorable safety profile of Etravirine and the unmet medical need in FA, successful outcomes could lead to significant market opportunities, particularly in rare disease segments. The competitive landscape remains limited due to the rarity of FA, but diligence should focus on ongoing and future studies targeting Frataxin and related pathways.
Source & freshness
Provenance
https://clinicaltrials.gov/study/NCT04273165
Indication
Friedreich Ataxia
Modality
small molecule
Target
Frataxin protein upregulation via enhancement of mRNA translation.
Intervention
Etravirine Tablets
Source record
Protocol Description
Detailed source ingestion pending.
Source record
Outcome Measures
Detailed source ingestion pending.
Source record
Eligibility
Detailed source ingestion pending.
AI analysis
Known Results And Readout Context
Detailed source ingestion pending.
IP intelligence
Patent And IP Landscape
Detailed source ingestion pending.
Source record
Contacts
Detailed source ingestion pending.