RECRUITING

A Clinical Trial of PRAX-222 in Pediatric Participants with Early Onset SCN2A Developmental and Epileptic Encephalopathy

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Conditions

SCN2A-DEEEpilepsyAntisense OligonucleotideNaV1.2 Voltage-Gated Sodium ChannelVoltage-Gated Sodium Channel BlockersSCN2A variantPediatric epilepsyDevelopmental and Epileptic EncephalopathyDEE

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

The goal of this trial is to learn about the effect of PRAX-222 in pediatric participants with early onset SCN2A developmental and epileptic encephalopathy (DEE), aged 2 to 18 years.

Official Title

A Seamless, Clinical Trial to Investigate the Safety and Efficacy of Multiple Doses of PRAX-222 in Pediatric Participants with Early Onset SCN2A Developmental and Epileptic Encephalopathy

Quick Facts

Study Start:2023-04-13
Study Completion:2027-09
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05737784

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:2 Years to 18 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT
Inclusion CriteriaExclusion Criteria
  1. * Has onset of seizures prior to 3 months of age.
  2. * Has a minimum weight of at least 10 kg at screening.
  3. * Has a documented SCN2A variant through genetic testing obtained via a laboratory accredited per Clinical Laboratory Improvement Amendments (CLIA) or College of American Pathologists (CAP) or equivalent.
  4. * Additional inclusion criteria apply and will be assessed by the study team
  1. * Has any clinically significant or known pathogenic genetic variant other than in the SCN2A gene, or a genetic variant that may explain or contribute to the participant's epilepsy and/or developmental disorder.
  2. * Is taking more than 2 sodium channel blocking anti-seizure medications
  3. * Additional exclusion criteria apply and will be assessed by the study team

Contacts and Locations

Study Contact

Head of Pharmacovigilance
CONTACT
617-300-8460
clinicaltrials@praxismedicines.com

Principal Investigator

Medical Director
STUDY_DIRECTOR
Praxis Precision Medicines

Study Locations (Sites)

Le Bonheur Childrens Hospital
Memphis, Tennessee, 38103
United States

Collaborators and Investigators

Sponsor: Praxis Precision Medicines

  • Medical Director, STUDY_DIRECTOR, Praxis Precision Medicines

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-04-13
Study Completion Date2027-09

Study Record Updates

Study Start Date2023-04-13
Study Completion Date2027-09

Terms related to this study

Keywords Provided by Researchers

  • Antisense Oligonucleotide
  • NaV1.2 Voltage-Gated Sodium Channel
  • Voltage-Gated Sodium Channel Blockers
  • SCN2A variant
  • Pediatric epilepsy
  • Developmental and Epileptic Encephalopathy
  • DEE

Additional Relevant MeSH Terms

  • SCN2A-DEE
  • Epilepsy