The MONARCH trial (NCT04442295) will recruit up to 48 patients with Dravet, from ages 2–18, at multiple centers across the United States. Currently, recruitment is ongoing at the Multicare Institute for Research and Innovation, in Tacoma, Washington. More information on trial locations and contacts can be found here.
To be eligible, patients must currently be on an anti-epileptic medication and have previously taken at least two anti-epileptic medications but stopped due to ineffectiveness or side effects.
Patients will be observed for at least one month, after which they will be given a single injection of STK-001 (given through the spinal canal) and observed for six months to study the effects of the treatment. The trial itself will run from 7–9 months, during which the trial participants will have to visit their study center about eight times. For those who qualify, the cost of medical treatments will be covered, with possible compensation for time and travel.
Dravet syndrome is a genetic condition caused by a mutation in one of the two copies of the SCN1A gene, which codes for a sodium channel protein necessary for nerve cell function.
In preclinical studies using mouse models of Dravet, STK-001 significantly prolonged survival and reduced the frequency of seizures.
MONARCH is split into two parts, with part A testing two comparatively low doses of STK-001 and part B evaluating higher doses. Part B is on hold and is pending the outcome of part A, which is currently ongoing.
Both parts will test two different (undisclosed) doses on at least two patients divided into two age groups: from 2–12 and from 13–18 years. The trial is designed as such to determine the best dosage of STK-001 to optimize benefits and reduce side effects going forward.
The study’s primary goals include documenting any adverse reactions to treatment and the levels of STK-001 in the blood and cerebrospinal fluid (that which surrounds the brain and spinal cord).
Over the course of six months following the initial dosing, researchers will monitor overall seizure frequency, clinical status, and quality of life.
Though it is not yet known, it is estimated that patients will require STK-001 injections 2–3 times per year. Those enrolled in the MONARCH study may also be eligible for future trials.
STK-001 had previously received an orphan drug designation from the U.S. Food and Drug Administration, which supports the development of treatments for diseases that affect less than 200,000 people in the U.S.
In addition to MONARCH, Stoke is also supporting another clinical study called BUTTERFLY, an observational study of children and adolescents with Dravet syndrome that will help to evaluate the natural course of the disease.
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