Open Label Extension Clinical Trial of Vormatrigine in Adult Patients with Epilepsy.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Praxis Precision Medicines Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

16 Oct 2025 → ongoing

Decision date (initial)

2025-10-08

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

Praxis Precision Medicines

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Safety, Efficacy

To assess the safety and tolerability of vormatrigine in adults with focal or primary generalized tonic-clonic seizures.

Secondary objectives 3

1. (EXPLORATORY) To evaluate the efficacy of vormatrigine on focal or primary generalized tonic-clonic seizure frequency in adults.
2. (EXPLORATORY) To characterize the effect of vormatrigine on global impressions in adults with focal or primary generalized tonic-clonic seizures.
3. (EXPLORATORY) To determine vormatrigine exposure at selected timepoints after dosing in adults with focal onset seizures or primary generalized tonic clonic seizures.

Conditions and MedDRA coding

Focal onset seizures or primary generalized tonic-clonic seizures.

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. 1. Participant and caregiver (if applicable), is willing to sign an informed consent document in accordance with International Council for Harmonisation (ICH) Good Clinical Practice (GCP) guidelines, indicating that they understand the purpose of the clinical trial; understands and can perform, complete, and comply with all the procedures and assessments that are required during the clinical trial, including the seizure diary and contraception, and is willing to participate in the clinical trial.
2. 2. Participant who completed an eligible clinical trial (unless agreed by the sponsor medical monitor), including PRAX-628-212, PRAX-628-321 or other vormatrigine trial, or participant who has received vormatrigine outside of an eligible clinical trial (such as an expanded access program).
3. 3. Participant who had adequate seizure diary and vormatrigine adherence and had been otherwise adherent to the study procedures in the opinion of the investigator unless deemed unacceptable by sponsor.

Exclusion criteria 4

1. 1. Participant who withdrew or discontinued treatment from the treatment period of an eligible clinical trial unless deemed acceptable by the sponsor.
2. 2. Participant who plans to enter another interventional investigational study or is on any prohibited medication listed in Table 7.
3. 3. Participant who is pregnant, lactating or planning to become pregnant.
4. 4. Participant who is ineligible for the extension clinical trial in the view of the investigator.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. - Incidence and severity of treatment-emergent adverse events (TEAEs), including discontinuation of study drug due to TEAEs. - Changes in vital sign measurements. - Changes in clinical laboratory results. - Changes in electrocardiogram parameters. - Changes in suicidality, as assessed by Columbia Suicide Severity Rating Scale (C-SSRS)

Secondary endpoints 4

1. (EXPLORATORY) Percent change in monthly (28 days) focal or generalized seizure frequency from the Screening/Observation Period of the primary trial.
2. (EXPLORATORY) Change from baseline over time in PGI-S and CGI-S.
3. (EXPLORATORY) Vormatrigine plasma concentrations
4. (EXPLORATORY) PGI-C, CGI-C, and CaGI-C (if applicable) assessed overtime post-screening.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 4

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Praxis Precision Medicines Inc.

Sponsor organisation

Praxis Precision Medicines Inc.

Address

99 High Street Floor 30th

City

Boston

Postcode

02110-2345

Country

United States

Scientific contact point

Organisation

Praxis Precision Medicines Inc.

Contact name

Kate Touse

Public contact point

Organisation

Praxis Precision Medicines Inc.

Contact name

Kate Touse

Third parties 7

Locations

4 EU/EEA countries · 36 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 52 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications