Phase 2 Epilepsy Trial, Recruiting NCT07531745 Sponsor: Ionis Pharmaceuticals, Inc. Condition: Epilepsy
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Phase 2 Epilepsy Trial, Recruiting

NCT07531745
Recruiting Phase 2

Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

  • The child must be between 2 and 12 years old at the time of signing up for the study.
  • The child must have at least one parent or caregiver who is 18 years or older, is willing to give written consent, and can attend all study appointments.
  • The child must have been formally diagnosed with Dravet Syndrome (DS) according to recognised international medical guidelines.
  • The child must have a confirmed genetic change (variant) in a specific gene called SCN1A that is known or likely to cause their condition.
  • The child must currently be taking at least one seizure medication at a steady, unchanged dose for at least 4 weeks before joining the study.
  • Any other epilepsy treatments (such as a special diet or a nerve stimulation device), other medications, supplements, or nutritional support must have been at a stable level for at least 4 weeks before joining; if a nerve stimulation device was implanted, this must have happened at least 6 months before joining.
  • The child must experience a required number of specific types of seizures during the initial screening period of the study (confirm with trial site for exact number).

Who may not be able to join:

  • The child has a known brain or spinal condition that could make a spinal fluid collection procedure (a lumbar puncture) unsafe or difficult.
  • The child has a confirmed genetic change in a different gene (other than SCN1A) that is known to cause epilepsy.
  • The child has previously received, or is currently taking part in a trial for, a gene therapy or a similar type of drug called an antisense oligonucleotide (ASO) for Dravet Syndrome.
  • The child has taken part in a trial of any other experimental drug, biological treatment, or medical device within the past 30 days (or longer, depending on the specific treatment involved).
  • The child is currently taking a seizure medication that works mainly by blocking sodium channels in the brain as a regular ongoing treatment (confirm with trial site for specific medications).
  • The child has previously had certain brain surgeries, such as an operation to cut connections between the two sides of the brain, deep brain stimulation device implantation, or any other brain surgery aimed at reducing seizures.

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.
Last synced 20 July 2026
Phase 2: approximately ~30% of drugs entering this phase reach regulatory approval, based on published industry-wide historical data. This is not specific to this trial.
Phase success rates shown are historical industry-wide averages based on published data. They are not a prediction for this specific trial or your individual situation.

Contact this trial

Phone: (844) 867-4309

Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.

GP referral letter

Print a one-page summary to share with your doctor.

Trial details

Status
Recruiting
Phase
Phase 2
Sponsor
Ionis Pharmaceuticals, Inc.
Registry
ClinicalTrials.gov
Start date
21 May 2026
Est. completion
1 December 2030

Where this trial is recruiting

🇺🇸 United States

Primary endpoints

Parts 1 and 2: Number of Participants with Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs; Number of Participants With Clinically Significant Change From Baseline in Safety Laboratory Values; Parts 1 and 2: Number of Participants With Clinically Significant Change From Baseline in Vital Signs; Parts 1 and 2: Number of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG); Parts 1 and 2: Number of Participants With Clinically Significant Change From Baseline in Physical and Neurological Examination Findings; Parts 1 and 2: Number of Participants w...

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 20 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov