ClinicalTrials.gov record NCT06598449

Assessment of Safety of the Use of Fenfluramine in Children With Dravet Syndrome Under 24 Months of Age

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Aug 22, 2026

Assessment of Safety of the Use of Fenfluramine in Children With Dravet Syndrome Under the Age of 24 Months

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 4A study of a treatment that is already approved, to learn more about its long-term effects, safety or use in everyday practice.
Age1 year old to 2 years old
SexAll
LocationUnited States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This phase 4, single-group study gives fenfluramine to children aged 12–23 months with Dravet syndrome to assess safety. Dosing is given twice daily within a specified mg/kg range (with a lower maximum dose if the child is taking stiripentol). Participants are followed for safety outcomes over 12 months.

What it is studying. The study is testing the safety of fenfluramine in infants and toddlers (12–23 months) with Dravet syndrome by recording adverse events during treatment.

What the researchers hope to learn. To measure how often adverse events occur in children 12–23 months old treated with fenfluramine over 12 months and to collect safety data in this age group.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

Dravet syndrome is a genetic epilepsy associated with pathogenic variants in SCN1A that codes for Nav1.1, a protein necessary for sodium channels. Children with Dravet syndrome classically present in the first year of life with prolonged seizures, often hemiclonic and in the setting of fever or temperature changes such as getting in or out of bath water. Many anti-seizure medications are sodium channel blockers and exacerbate seizures in this patient population. This creates some limitations in medication choices for this patient population. Recently fenfluramine was approved for use in Dravet syndrome for people 2 years and older. Randomized studies demonstrated a 74.9% reduction of convulsive motor seizures compared to 19.2% in the placebo group. Additionally, 16% of children treated with fenfluramine were seizure free. Fenfluramine is likely to be as effective in children under the age of 2 years. The current study has proposed an intermediate size patient population expanded access protocol to allow access to fenfluramine for children under 24 months of age.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • fenfluramine

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • Treating physicians must submit a clinical statement of potential benefit, which includes patient's gender, age, diagnosis, genetic pathogenic variant, co-morbidities, seizure history, prior and current therapies, response to prior therapies and reason for request. Echocardiogram (ECHO) results must also be submitted to the lead site prior to final approval. Patients must be between 12 and 23 months old to be eligible. Additional inclusion criteria:
  • SCN1A with a known or presumed pathogenic variant or VUS with a history of prolonged seizure or a clinical diagnosis of Dravet syndrome.
  • Failure of at least one anti-seizure medication that is not a sodium channel blocker (lamotrigine, oxcarbazepine, carbamazepine, eslicarbazepine)

Exclusion criteria

  • Patients with mild or greater mitral valve regurgitation and/or trace or greater aortic valve regurgitation will not be eligible for participation. The clinical statement can be submitted first for initial, conditional approval and then ECHO results can be submitted at a later date for final approval.
  • Patients with failure to thrive will not be eligible for participation as fenfluramine can suppress appetite and has a risk for weight loss. Failure to thrive will be evaluated on the following criteria:
  • Weight less than the 2nd percentile.
  • Lack of weight gain that crosses two or more of the major percentile lines and is not congruent with length.

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants receive fenfluramine oral solution given twice daily; dosing is weight-based (0.2–0.7 mg/kg/day, up to 26 mg/day) and has a lower maximum if the child is taking stiripentol (up to 0.4 mg/kg/day, up to 17 mg/day). The treating physician must submit a clinical statement and an echocardiogram before final approval. Children are followed for safety with monitoring and reporting of adverse events over a 12-month period. No other specific procedures or visit schedule are provided in the registry record.

How the study is structured. Interventional (treatment) study: all participants receive fenfluramine in a single open-label group. There is no masking. Fenfluramine is given twice daily with defined dose ranges and maximum daily doses; a lower maximum applies if the child is taking stiripentol. Safety is tracked over a 12-month period, with the primary measure being whether participants experience any adverse events.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The registry lists this study under Dravet Syndrome (DS); Children Under 2 Years. Does it apply to my diagnosis?
  • The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on CDKL5 deficiency disorder

This trial is also listed on this site under Dravet syndrome.

Clinical trials22 trials listed for CDKL5 deficiency disorderEvery trial the registry lists for CDKL5 deficiency disorder, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in CDKL5 deficiency disorder researchLatest monthly update: September 2026. Includes: EEG background changes. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Oct 22, 2024
Primary completion
December 2026 (estimated)
Study completion
July 2027 (estimated)
Target enrollment
5 participants (estimated)
Last updated on registry
May 15, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
University of Colorado, Denver

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

Terms used on this page · explanations by MedicalUpdates.ai

Randomized
Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
placebo
A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.
expanded access
A way for some people with serious conditions to receive an investigational treatment outside a clinical trial, under specific rules. It is not itself a clinical trial and is not designed to test whether the treatment works.
Echocardiogram
An ultrasound scan of the heart. A transesophageal echocardiogram is done through a probe passed into the food pipe for a closer view.
inclusion criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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