TrialSignal
Clinical trial intelligence report
A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS)
Source-linked diligence brief with registry provenance, taxonomy normalization and premium analytical context.
Generated
Jul 28, 2026
Report code
NCT03506425-Jul 28, 2026
NCT ID
NCT03506425
Status
COMPLETED
Phase
Phase 1/2
Sponsor
Richard Bedlack, M.D., Ph.D.
Executive brief
Investment-Ready Snapshot
The pilot trial of Triheptanoin for ALS represents a potential breakthrough in the treatment of a disease with significant unmet medical need. Given the limited therapeutic options currently available for ALS, which primarily focus on symptom management rather than disease modification, Triheptanoin could carve out a niche in the market if proven effective. The collaboration with Ultragenyx Pharmaceutical Inc suggests a strategic partnership that may enhance development capabilities and market access. The trial's completion and forthcoming results could influence investor interest and competitive positioning within the neurodegenerative disease space, particularly against existing therapies like Riluzole and Radicava.
Source & freshness
Provenance
https://clinicaltrials.gov/study/NCT03506425
Indication
ALS
Modality
small molecule
Target
Mitochondrial function improvement and energy production enhancement via Triheptanoin, a medium-chain triglyceride.
Intervention
Triheptanoin
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.