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Soticlestat

Soticlestat is an investigational drug being studied as a potential treatment for rare forms of epilepsy, including Dravet syndrome and Lennox-Gastaut syndrome. Clinical trials are evaluating its safety, effectiveness, and optimal dosing when used as an add-on therapy to existing anti-seizure medications. This article summarizes key findings from recent clinical trials of soticlestat in patients with rare epilepsies.

Table of Contents

What is Soticlestat?

Soticlestat, also known as TAK-935, is a new medication being studied for the treatment of various types of epilepsy and complex regional pain syndrome (CRPS)[1][2]. It is currently in clinical trials and is not yet approved for general use. Soticlestat is being developed as an add-on therapy, which means it is intended to be used alongside other medications to improve treatment outcomes.

What Conditions Does Soticlestat Treat?

Soticlestat is being studied for the treatment of several rare and difficult-to-treat conditions, including:

  • Dravet Syndrome (DS): A severe form of epilepsy that begins in infancy and is characterized by frequent, prolonged seizures[3]
  • Lennox-Gastaut Syndrome (LGS): A complex, rare form of epileptic encephalopathy that usually begins in early childhood[3]
  • CDKL5 Deficiency Disorder (CDD): A rare genetic disorder characterized by seizures and developmental delays[4]
  • 15q Duplication Syndrome (Dup15q): A rare chromosomal disorder that can cause epilepsy, autism, and intellectual disability[4]
  • Complex Regional Pain Syndrome (CRPS): A chronic pain condition that usually affects an arm or leg after an injury or trauma[2]

How Does Soticlestat Work?

While the exact mechanism of action is not fully understood, soticlestat is believed to work by inhibiting an enzyme called 24-hydroxycholesterol (24HC) in the brain[4]. This enzyme is involved in cholesterol metabolism, and by reducing its activity, soticlestat may help to decrease excessive brain activity that leads to seizures and other neurological symptoms.

How is Soticlestat Administered?

Soticlestat is taken orally in the form of tablets or mini-tablets. The dosage depends on the patient’s weight and the specific condition being treated. In most clinical trials, the medication is given twice daily[3]. For patients who have difficulty swallowing, the medication can also be administered through a feeding tube[5].

Current Clinical Trials

Several clinical trials are currently underway to evaluate the safety and effectiveness of soticlestat:

  • Phase 3 trials for Dravet Syndrome and Lennox-Gastaut Syndrome in children and adults[6][5]
  • Phase 2 trials for CDKL5 Deficiency Disorder and 15q Duplication Syndrome[4]
  • Phase 2 trial for Complex Regional Pain Syndrome in adults[2]

These trials are assessing various outcomes, including reduction in seizure frequency, improvement in quality of life, and changes in pain intensity.

Potential Side Effects

As with any medication, soticlestat may cause side effects. The full range of potential side effects is still being studied in clinical trials. Common methods for monitoring side effects in these trials include:

  • Regular blood tests and physical examinations
  • Monitoring of vital signs
  • Electrocardiograms (ECGs) to check heart function
  • Assessment of suicidal thoughts or behaviors using the Columbia-Suicide Severity Rating Scale (C-SSRS)[7]

Patients in clinical trials are closely monitored for any adverse events, which are carefully recorded and analyzed.

Future Research

Ongoing and future research on soticlestat includes:

  • Long-term safety and efficacy studies[3]
  • Studies on the effects of soticlestat in patients with liver impairment[8]
  • Investigations into potential drug interactions, such as with rifampin[9]
  • Evaluation of different formulations and dosing strategies[7]

These studies will help researchers better understand how soticlestat works in different patient populations and how it can be used most effectively and safely.

Aspect Details
Primary Conditions Studied Dravet syndrome, Lennox-Gastaut syndrome
Other Conditions CDKL5 deficiency disorder, Chromosome 15q duplication syndrome
Main Study Objectives Efficacy in reducing seizure frequency, Long-term safety and tolerability, Optimal dosing, Quality of life improvements
Dosage Forms Oral tablets, Mini-tablets, Crushed tablets mixed with food
Typical Dose Range 100-300 mg twice daily, adjusted based on body weight
Study Designs Randomized controlled trials, Open-label extension studies, Pharmacokinetic studies
Key Outcome Measures Percent change in seizure frequency, Responder rates, Safety assessments, Quality of life scores
Patient Age Groups Children, adolescents, and adults
Treatment Duration Main studies: 4-6 months, Extension studies: Up to several years

FAQ

  • What is soticlestat? Soticlestat (also known as TAK-935) is an experimental medication being studied as a potential treatment for rare forms of epilepsy. It is taken orally as a tablet or mini-tablet and is designed to be used as an add-on therapy alongside other anti-seizure medications.
  • What types of epilepsy is soticlestat being studied for? Soticlestat is primarily being investigated for Dravet syndrome and Lennox-Gastaut syndrome. Some studies have also included patients with other rare epilepsies like CDKL5 deficiency disorder and chromosome 15q duplication syndrome.
  • How is soticlestat taken? Soticlestat is taken orally, usually twice daily. The dosage depends on the patient's body weight. It can be given as tablets, mini-tablets, or crushed and mixed with food like applesauce. Some studies have also tested administration via feeding tubes for patients who cannot swallow pills.
  • What are the main goals of the soticlestat clinical trials? The main goals are to evaluate soticlestat's effectiveness in reducing seizure frequency, assess its safety and tolerability when used long-term, determine optimal dosing, and measure improvements in quality of life and other non-seizure symptoms in patients with rare epilepsies.
  • How long do the soticlestat clinical trials typically last? The duration varies, but many of the trials last 4-6 months for the main treatment period. Some studies include an open-label extension phase where patients can continue treatment for several years to gather long-term safety and efficacy data.

Ongoing Clinical Trials on Soticlestat

  • Study on Long-Term Safety of Soticlestat for Patients with Dravet or Lennox-Gastaut Syndromes

    Not recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium France Germany Greece Hungary Italy +4

  • Study on Soticlestat for Children and Adults with Dravet or Lennox-Gastaut Syndrome Previously Treated with Fenfluramine

    Not recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    Denmark

  • Study on Long-Term Safety of Soticlestat for Adults and Children with Developmental Epileptic Encephalopathies

    Not recruiting

    2 1 1
    Investigated diseases:
    Investigated drugs:
    Poland Portugal Spain

Glossary

  • Soticlestat: An investigational oral medication being studied as a potential treatment for rare forms of epilepsy. Also known as TAK-935.
  • Dravet syndrome: A rare, severe form of epilepsy that begins in infancy and is characterized by frequent, prolonged seizures and developmental delays.
  • Lennox-Gastaut syndrome (LGS): A severe form of epilepsy that usually begins in childhood and is characterized by multiple types of seizures and intellectual disability.
  • CDKL5 deficiency disorder: A rare genetic condition characterized by seizures beginning in infancy and severe developmental delays.
  • Chromosome 15q duplication syndrome: A rare chromosomal disorder associated with developmental delays, intellectual disability, and epilepsy.
  • Adjunctive therapy: An additional treatment used together with the primary treatment to increase its effectiveness.
  • Open-label extension study: A study phase where all participants receive the active study drug, often following a placebo-controlled phase, to gather long-term safety and efficacy data.
  • Pharmacokinetics: The study of how a drug is absorbed, distributed, metabolized, and eliminated by the body.
  • Bioequivalence: The property of two drug products having the same rate and extent of absorption when administered at the same dose.
  • Placebo: An inactive substance designed to look like the study drug but containing no active medication, used as a control in clinical trials.

References

  1. https://clinicaltrials.eu/trial/study-on-long-term-safety-of-soticlestat-for-adults-and-children-with-developmental-epileptic-encephalopathies/
  2. https://clinicaltrials.gov/study/NCT03990649
  3. https://clinicaltrials.gov/study/NCT05163314
  4. https://clinicaltrials.gov/study/NCT03694275
  5. https://clinicaltrials.gov/study/NCT04938427
  6. https://clinicaltrials.gov/study/NCT04940624
  7. https://clinicaltrials.gov/study/NCT05284760
  8. https://clinicaltrials.gov/study/NCT05098054
  9. https://clinicaltrials.gov/study/NCT05098041