Phase 1 / Phase 2: testing ION337 for Dravet Syndrome
The primary purpose of this study is to measure the whether it is safe and how hard side effects are to live with of ION337 in participants with Dravet syndrome (DS).
Official registry: https://clinicaltrials.gov/study/NCT07531745 · Ionis Pharmaceuticals, Inc. · enrollment listed as 32
What this phase means
Phase 2: researchers already know a roughly safe dose (from phase 1). This stage asks whether the treatment actually helps the condition, and watches side effects more closely.
Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.
What the registry says happens
If you join, the listed intervention(s) are ION337. The registry lists allocation as randomized. The primary purpose of this study is to measure the whether it is safe and how hard side effects are to live with of ION337 in participants with Dravet syndrome (DS).
They plan to enroll about 32 people at 7 sites. Taking part typically means taking a study drug (or dummy pill) plus check-ins.
Official title (unrewritten)
Phase 1-2, Open-Label, Single and Multiple Ascending Dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION337 in Patients With Dravet Syndrome
Interventions: ION337
Primary outcome (registry wording): Parts 1 and 2: Number of Participants with Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs
Who it is for (from inclusion criteria)
- Ages 2 Years to 12 Years
- Has a documented diagnosis of DS according to the International League Against Epilepsy (ILAE) criteria and as agreed by the Epilepsy Study Consortium, Inc (ESCI)
- Has confirmation of a pathogenic or likely pathogenic SCN1A variant
- Experiences the required number of major motor seizures during the Screening Period
Who the registry excludes
- Known brain or spinal disease that would interfere with the LP procedure or CSF circulation, or presence of other factors that would affect the safety of the LP procedure
- Pathogenic or likely pathogenic variant in another gene that causes epilepsy
- Has had prior treatment with or is currently enrolled in an treatment clinical trial for a gene therapy or for another antisense oligonucleotide (ASO) for the treatment of DS
- Has had treatment with or is currently enrolled in an treatment clinical trial of any other investigational drug, biological agent, or device within 30 days prior to Screening, or 5 half-lives of investigational agent, whichever is longer
- Current treatment with an anti-seizure medication (ASM) acting primarily as a sodium channel blocker, as maintenance treatment
- Prior brain surgeries including: corpus callosotomy, implantation of device for deep brain stimulation or any other palliative brain surgery intended to reduce seizure burden
- Note: Other protocol pre-specified inclusion/
Public study contact (from the registry)
Ionis Pharmaceuticals, Inc.
IonisDravetSyndrome@clinicaltrialmedia.com
Published on ClinicalTrials.gov. Contact them as a possible participant. They decide eligibility.
Sites listed on ClinicalTrials.gov
7 listed- University of Michigan Health System — Ann Arbor, Michigan (recruiting)
- Mayo Clinic — Rochester, Minnesota (recruiting)
- Duke University Health Systems — Durham, North Carolina (recruiting)
- The Children's Hospital of Philadelphia (CHOP) — Philadelphia, Pennsylvania (recruiting)
- Le Bonheur Children's Hospital — Memphis, Tennessee (recruiting)
- Seattle Children's Hospital — Seattle, Washington (recruiting)
+ 1 sites outside the US on the same record
FAQ about this NCT record
Is NCT07531745 still recruiting?
The sponsor posted status “Recruiting” on ClinicalTrials.gov at last ingest. Sites can pause independently. Confirm with the official record and the listed contact.
What phase is NCT07531745?
Phase 1 / Phase 2. Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.
Can PlainTrials tell me if I qualify for NCT07531745?
No. The inclusion and exclusion lists on this page are readable extracts of the public eligibility block. Only the study team can screen you.
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