Clinicians: KCNT1 Clinical Trials
An overview of current & upcoming KCNT1-focused studies for quick clinician referral. Share the patient education page with families and invite them to sign up for trial updates.
Quick Navigation
Access key resources for clinicians and families
Understanding KCNT1 Epilepsy
- Access key resources for clinicians and families
- Clinical Trials Education page (parent-friendly)
- Trial Interest Signup (families)
- Refer a Family (Clinician form)
- Sign up for Clinician Updates
KCNT1-Specific Studies
| Trial name (link) | Sponsor | Modality | Phase | Overall Status* | Countries / Active sites | Coordinator / Contact |
|---|---|---|---|---|---|---|
| [ABS-1230] | Actio Biosciences | Small molecule (channel modulator) | 1 | Not yet recruiting | US (site activation varies) | |
| [ASO Program] | Servier | ASO (intrathecal) | TBD | Recruiting (site-dependent) | US / [others] | |
| [ATL-201] | Atalanta | di-siRNA (intrathecal) | TBD | Preclinical | (TBD) | |
| [Natural History / Registry] | Multicenter | Observational | – | Not yet recruiting | US /international | n |
*Overall status reflects registry labels (e.g., Not yet recruiting, Recruiting, Active—not recruiting). Individual hospitals enroll only after site activation (contracts, ethics, training, drug on site).
Other Clinical Trials (not KCNT1-specific)
| Sponsor | Therapy Name (link) | Phase | Class of therapeutic | Delivery Target | Status | |
|---|---|---|---|---|---|---|
| Lundbeck | Bexicaserin | Phase 3 | Small molecule | Oral | 5-HT2C (not KCNT1) | Enrolling |
Quick Guidance for Referring Clinicians
Referral Best Practices
Confirm site activation before referral
Country authorization ≠ site readiness.
Screening packet
Recent EEG/MRI, KCNT1 variant report, med list with doses, baseline seizure diary (4–8 weeks if feasible).
Medication washouts/holds may be required
Coordinate safety plans with the PI/site.
Design basics
Randomization, placebo, blinding, typical primary endpoints (often seizure reduction).
Expanded Access (EA)
Not guaranteed; regulators prefer enrollment in the trial. EA needs sponsor agreement, regulatory/IRB approval and can be limited by drug supply.
Variant (Re)classification for Trial Eligibility
Many KCNT1 trials require a documented pathogenic/likely pathogenic variant (sometimes with gain-of-function evidence). If the report is VUS or incomplete:
Complete genetics
Order trio testing when possible (clarifies de novo vs inherited, mosaicism).
Gather Clinical/Segregation Data
Phenotype, EEG/MRI, Family History.
Request Lab Reinterpretation (ACMG/AMP)
De novo confirmation, allele frequency (gnomAD), in silico support, ClinVar, literature.
Functional Evidence If Required
Published electrophysiology or sponsor-validated assays for GOF.
Close the loop
Obtain updated report/addendum and include in the screening packet.
Tip: Start trio testing early for children likely to be considered for trials in the next 6–12 months.
For Your Patients & Families
Resources
- Clinical Trials Education (plain-language)
- Trial Interest List (email updates as sites open)
- Printable FAQ & Readiness Checklist
Contact
- Refer a Family (secure form)
- Clinician line / study questions
- Join clinician directory & roundtables
Disclaimer
Understanding which phase a trial is in helps you know what to expect in terms of safety data, effectiveness evidence, and the number of other participants.
