Asset profile
STK-001
RNA / oligonucleotide
Evidence outlook · no controlled comparison
NCT04442295 · Dravet Syndrome
The lead study is single-arm or otherwise uncontrolled, so it produced no formal comparison against a control. This tier describes the design, not an outcome.
NCT04442295: The study was non-randomized, open-label, sequential, and had no comparator arm, with 62 participants. Its primary outcomes were safety, pharmacokinetic, and cerebrospinal-fluid exposure measures, and no primary-endpoint results are available.
How the evidence outlook works ↗
Featured trials
The studies most instrumental to this drug's clinical progression, individually graded on design and result. Pick an indication; Research unlocks the trials, endpoints, key results and grades.
Competitive landscape
61 competitors in this universe: 1 direct rival (same mechanism and indication), 0 same-class, 60 same-indication; 60 further along in development.
| Drug | Stage | Modality | Mechanism | Evidence signal | Reported efficacygraded featured trials |
|---|---|---|---|---|---|
| STK-001: this drug | Phase 1/2 | RNA / oligonucleotide | SCN1A mRNA | ||
| ION337 | Phase 1/2 | RNA / oligonucleotide | SCN1A mRNA | ||
| Brivaracetam | Approved | Small molecule | Synaptic vesicle glycoprotein 2A (SV2A) | ||
| Cannabidiol (Epidiolex) | Approved | Small molecule | GPR55 / TRPV1 / adenosine transport (multi-target), cannabinoid | ||
| Carbamazepine | Approved | Small molecule | Sodium channels | ||
| Cenobamate (Xcopri) | Approved | Small molecule | Sodium channels + GABA-A receptors | ||
| Clobazam (Onfi) | Approved | Small molecule | GABA-A receptor | ||
| Clobazam (Sympazan) | Approved | Small molecule | GABA-A receptor (benzodiazepine site) | ||
| Diastat | Approved | Combination | GABA-A receptor | ||
| Diazepam (Valium) | Approved | Small molecule | GABA-A receptor | ||
| Diazepam (Valtoco) | Approved | Small molecule | GABA-A receptor | ||
| Divalproex Sodium (Depakote) | Approved | Small molecule | GABAergic neurotransmission | ||
| Eslicarbazepine Acetate (Aptiom) | Approved | Small molecule | Voltage-gated sodium channel | ||
| Ethosuximide | Approved | Small molecule | T-type calcium channel | ||
| Everolimus | Approved | Small molecule | mTOR kinase, mTOR (mTORC1) (inhibitor) | ||
| Ezogabine (Potiga) | Approved | Small molecule | Kv7 potassium channel |
…and 46 more. Research shows the complete landscape.
Unlock the full comparison
Research adds each competitor's evidence grade and sourced trial results, aligned per race, so you can see who actually has the data.
Compare head-to-head
Upcoming events
(1 total)
Estimated primary-completion dates from ClinicalTrials.gov. These are registry estimates, never confirmed readout dates; they can move as trials progress.
2029 (1)
March 2029 (1)
What to watch
All registered studies
Every ClinicalTrials.gov study of this drug's own program, grouped by indication and phase. Research unlocks the studies themselves: titles, statuses, dates and featured-trial cross-links.