PRAX-628 for Epilepsy

Recruiting at 10 trial locations

Overseen By Head of Pharmacovigilance

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Praxis Precision Medicines

Must be taking: ASMs

Must not be taking: Vigabatrin, Felbamate

Disqualifiers: Schizophrenia, OCD, Cardiac disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 8 weeks

50 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial does not specify that you need to stop taking your current medications. In fact, you must be on stable doses of 1 to 3 allowable anti-seizure medications (ASMs) to participate.

What data supports the effectiveness of the drug PRAX-628 for epilepsy?

Research on similar drugs, like perampanel, which is an AMPA receptor antagonist, shows that targeting these receptors can help control seizures in epilepsy patients. Additionally, studies on LY3130481, another AMPA receptor antagonist, suggest that selectively blocking certain brain receptors can prevent seizures without causing motor side effects. ^{1 2 3 4 5}

How is the drug PRAX-628 different from other epilepsy treatments?

PRAX-628 is unique because it targets specific AMPA receptors in the brain, potentially reducing seizures without causing motor side effects, unlike other AMPA receptor antagonists that can cause dizziness and motor impairment. ^{3 4 6 7 8}

What is the purpose of this trial?

An Open Label Clinical Trial to Evaluate the Efficacy and Safety of PRAX-628 in Adult Patients With Focal Onset or Primary Generalized Tonic-Clonic Seizures

Research Team

Medical Director

Principal Investigator

Praxis Precision Medicines

Eligibility Criteria

Adults with focal onset or tonic-clonic seizures who have had at least 2 seizures per month and are on stable epilepsy medication can join. Those pregnant, planning pregnancy, with serious mental health issues, recent neurosurgery, cardiac problems, cancer history within 5 years, or recent experimental therapy use cannot participate.

Inclusion Criteria

Evidence by computed tomography (CT) or magnetic resonance imaging (MRI) ruling out a progressive cause of epilepsy

I have been diagnosed with focal onset or generalized tonic-clonic seizures.

I am on 1 to 3 stable seizure medications.

Exclusion Criteria

Pregnant or breastfeeding at Screening, positive serum pregnancy test at Screening, or planning pregnancy during the trial or within 14 days of the last study drug dose

Schizophrenia, obsessive-compulsive disorder, or other serious mental health disorders

I have had specific types of seizures or seizure emergencies in the last year.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 30mg/day of PRAX-628 for 8 weeks

8 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• PRAX-628

Trial Overview The trial is testing the effectiveness and safety of a new medication called PRAX-628 (30mg) for adults with certain types of seizures. It's an open label study where all participants know they're receiving the actual drug being tested.

Participant Groups

Group I: Open-label 30mg/day PRAX-628 for 8 weeks Experimental Treatment 1 Intervention

Participants who meet all eligibility criteria will receive 30mg of PRAX-628 for 8 weeks.

Who Is Running the Clinical Trial?

Praxis Precision Medicines

Lead Sponsor

Trials

11

Recruited

1,300+

References

1. United States pubmed.ncbi.nlm.nih.gov

Adjunctive perampanel for refractory partial-onset seizures: randomized phase III study 304. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

Efficacy and safety of adjunctive perampanel for the treatment of refractory partial seizures: a pooled analysis of three phase III studies. [2022]

3. Netherlands pubmed.ncbi.nlm.nih.gov

The value of human epileptic tissue in the characterization and development of novel antiepileptic drugs: The example of CERC-611 and KRM-II-81. [2020]

4. United States pubmed.ncbi.nlm.nih.gov

Forebrain-selective AMPA-receptor antagonism guided by TARP γ-8 as an antiepileptic mechanism. [2021]

5. Denmark pubmed.ncbi.nlm.nih.gov

Tolerability and safety of perampanel: two randomized dose-escalation studies. [2021]

6. United States pubmed.ncbi.nlm.nih.gov

Effects of 2-[N-(4-chlorophenyl)-N-methylamino]-4H-pyrido[3.2-e]-1,3-thiazin-4-one (YM928), an orally active alpha-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid receptor antagonist, in models of generalized epileptic seizure in mice and rats. [2004]

7. United Arab Emirates pubmed.ncbi.nlm.nih.gov

Targeted Blockade of TARP-γ8-Associated AMPA Receptors: Anticonvulsant Activity with the Selective Antagonist LY3130481 (CERC-611). [2019]

8. England pubmed.ncbi.nlm.nih.gov

Perampanel markedly improved clinical seizures in a patient with a Rett-like phenotype and 960-kb deletion on chromosome 9q34.11 including the STXBP1. [2022]