The first patient has been dosed in Stoke Therapeutics‘ Phase 1/2a MONARCH trial, which is investigating the safety, tolerability, and efficacy of STK-001 as a treatment for children and adolescents with Dravet syndrome. Enrollment in the trial is ongoing.
People with Dravet, a severe type of epilepsy, have one healthy copy and one mutated copy of the SCN1A gene, which provides instructions for a sodium channel protein vital for the proper functioning of nerve cells.
STK-001 is an antisense nucleotide that allows protein production from the gene’s functional copy, thus restoring normal levels of the sodium channel protein in the nerve cells.
Part A of the MONARCH study (NCT04442295), in which the first dosed patient is enrolled, is evaluating lower doses of STK-001. The second phase, Part B, which will test higher doses, is on hold pending the results of Part A.
The trial has had open enrollment since late June. Stoke expects to enroll approximately 40 Dravet patients, ages 2-18, at 20 test centers across the United States. More information about contacts and locations can be found here.
To participate in the trial, patients must be taking anti-epileptic medication. They also must have taken at least two anti-epileptic medications but stopped due to ineffectiveness or side effects.
The participants will undergo one month of baseline observation before receiving a single dose of STK-001 via lumbar puncture (spinal tap). This type of administration allows the treatment to directly access the cerebrospinal fluid (CSF), which is the liquid surrounding the brain and spinal cord.
After receiving STK-001, the patients will be monitored over a six-month period for adverse reactions to the therapy, to measure STK-001 levels in blood plasma and in the CSF, and to assess the frequency of seizures. Changes in quality of life and clinical status also will be evaluated.
In total, the study will require patients to visit their testing centers roughly eight times throughout the seven-to-nine months. The costs of medical treatments will be covered for qualified participants, and compensation for expenses may be included as well.
Dravet syndrome is a genetic condition characterized by seizures and cognitive disability and is caused by a mutation in one of the two copies of the SCN1A gene.
STK-001 was designed using Stoke’s proprietary TANGO technology to increase the number of healthy sodium channels created from the functional copy of the SCN1A gene.
In preclinical studies with Dravet mouse models, STK-001 significantly reduced seizure frequency and prolonged survival.
If approved, STK-001 would become the first treatment for Dravet that targets its underlying cause and not just the syndrome’s symptoms.
“Today we are announcing that the first patient has been dosed with STK-001, which we believe has the potential to be the first-disease modifying medicine for Dravet syndrome, a severe and progressive genetic epilepsy that is characterized by developmental delays and cognitive impairment, in addition to seizure activity,” Edward Kaye, MD, CEO of Stoke, said in a press release.
“The start of MONARCH also marks Stoke’s official transition to a clinical-stage biotech company,” Kaye added. “We enter this new stage in a strong financial position to execute on our plans for STK-001 in Dravet syndrome and continue to advance the potential of our TANGO platform for additional genetic diseases.”
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