Clinical trial
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
Name
PRAX-222-111
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.
Trial arms
Trial start
2023-04-13
Estimated PCD
2025-07-01
Trial end
2026-02-01
Status
Active (not recruiting)
Phase
Early phase I
Treatment
PRAX-222 - Initial Dose
PRAX-222
Arms:
Preliminary Safety
PRAX-222 - Initial Ascending Doses
Ascending doses of PRAX-222
Arms:
Dose Escalation - PRAX-222
PRAX-222 - Optional Ascending Doses
Escalation of PRAX-222 dose(s)
Arms:
Optional Dose Escalation - PRAX-222
PRAX-222 - Fixed Doses
Fixed-dose(s) of PRAX-222 not to exceed the maximum tolerated dose of PRAX-222
Arms:
Confirmatory Dosing - PRAX-222, Open-label PRAX-222
Placebo
Placebo procedure
Arms:
Confirmatory Dosing - Placebo, Dose Escalation - Placebo, Optional Dose Escalation - Placebo
Size
60
Primary endpoint
Number of participants with treatment-emergent adverse events (Preliminary Safety, Dose Escalation)
Screening (-8 weeks) through up to 92 weeks
Seizure frequency (Confirmatory Phase)
36 to 40 weeks
Eligibility criteria
Inclusion Criteria:
* Has onset of seizures prior to 3 months of age.
* Has a minimum weight of at least 10 kg at screening.
* 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.
* Additional inclusion criteria apply and will be assessed by the study team
Exclusion Criteria:
* 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.
* Is taking more than 2 sodium channel blocking anti-seizure medications
* Additional exclusion criteria apply and will be assessed by the study team
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Seamless study design with sequential study parts, including an open-label safety part; a double-blind, randomized dose-escalation part; a double-blind, randomized confirmatory part; and an open-label extension part.', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'QUADRUPLE', 'maskingDescription': 'No masking in the 2 open-label parts, masking as indicated in the 2 double-blind, randomized parts.', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}}, 'enrollmentInfo': {'count': 60, 'type': 'ESTIMATED'}}
Updated at
2024-01-10
1 organization
1 product
2 indications
Organization
Praxis Precision MedicinesProduct
PRAX-222Indication
SCN2A-DEEIndication
Epilepsy