TriCelX Nabs FDA Clearance for Phase 1/2 of First-in-Indication Cell Therapy Trial for CTE
Developed in hand with the DoD, the company is hoping to lay the groundwork for cell therapy in concussive injuries for soldiers, athletes, and the broader population.
Key Points
- TriCelX has received FDA clearance to begin a Phase 1/2 trial of XytriX in chronic traumatic encephalopathy, or CTE.
- The company says this is the first time a cell therapy will be studied in this condition.
- The open-label dose-escalation study will test safety, tolerability, and early signs of activity in adults with probable CTE, including veterans and former contact-sport athletes.
TriCelX, a Texas-based biotech company, has received FDA clearance to start a Phase 1/2 clinical trial of its allogeneic wharton’s jelly-derived MSC therapy, XytriX, in chronic traumatic encephalopathy, which they say is the first cell therapy trial in this condition.
XytriX was developed with the Department of Defense under the Blast Overpressure Safety Act, which aims to study and treat concussive injuries in the armed forces.
Trial design and patient population
The trial is an open-label, three-cohort dose-escalation study using a 3+3 design in adults with probable CTE. Participants will receive XytriX by intravenous and intrathecal administration. Eligibility will be based on a formal diagnosis of Traumatic Encephalopathy Syndrome at the probable level by a qualified neurologist using the 2021 NINDS TES criteria.
The study is designed to assess both safety and preliminary efficacy over 24 months. The main evaluations include:
- Treatment-emergent adverse events
- Serious adverse events
- Dose-limiting toxicities
- Changes from baseline in neurocognitive outcomes
- Changes from baseline in behavioral outcomes
- Changes from baseline in functional outcomes
The protocol also includes exploratory biomarker and imaging measures intended to help characterize biological activity and support planning for a later controlled trial. These measures include:
- Serum neurofilament light chain, or NfL
- GFAP
- Plasma phosphorylated-tau species
- Structural MRI
- Diffusion tensor imaging, or DTI
- Functional MRI
The trial will enroll adults with probable CTE from two groups highlighted in the release:
- Blast-exposed service members and veterans
- Former contact-sport athletes
Why the study matters
CTE is a progressive tauopathy associated with repeated head trauma and blast exposure, and there are no approved treatments that address the underlying disease. The disease burdens both military and sports populations, including prior findings in former NFL players and Navy SEALs. TriCelX says XytriX is designed to target chronic neuroinflammation and tau pathology linked to CTE.
Because this is the first cell therapy study in CTE, the trial may help define an early framework for evaluating future programs in the indication.
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