Fenfluramine for Infantile Spasms
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a medication called fenfluramine to determine if it helps children with infantile spasms, a type of seizure disorder. The focus is on children whose spasms haven't improved with standard treatments like vigabatrin and ACTH. Participants will begin with a 21-day treatment, and if effective, they may continue for up to 6 months. Eligible children have ongoing spasms despite trying these common treatments. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering a chance to benefit from a potentially effective new therapy.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but you cannot start or change doses of second-line treatments for infantile spasms within 14 days before the trial. Also, you should not have used any cannabinoid products in the 14 days before the trial.
Is there any evidence suggesting that fenfluramine is likely to be safe for humans?
Research has shown that fenfluramine has been tested for safety in conditions like Dravet Syndrome and Lennox-Gastaut Syndrome. In these studies, patients generally tolerated fenfluramine well. For instance, one study on Lennox-Gastaut Syndrome found that participants taking fenfluramine experienced fewer seizures than those on a placebo, with safety results aligning with previous findings.
Another study demonstrated that long-term use of fenfluramine for Dravet Syndrome was effective and safe. Some side effects occurred, but they were consistent with earlier research.
As this trial closely monitors safety, earlier research suggests some confidence in the treatment's safety. However, like any medication, side effects may occur, so monitoring participants' responses during the trial is crucial.12345Why do researchers think this study treatment might be promising for infantile spasms?
Fenfluramine is unique because it offers a new approach for treating infantile spasms by utilizing a different mechanism of action. Most treatments for this condition, like ACTH and vigabatrin, work by modulating neurotransmitter activity, but Fenfluramine acts on the serotonin system to reduce seizure activity. Researchers are excited about Fenfluramine because it may provide rapid relief, with potential effectiveness in just 21 days, and offers a longer-term treatment option for patients who respond well. This could be a game-changer for infants with spasms, providing an alternative when current therapies fall short.
What evidence suggests that fenfluramine might be an effective treatment for infantile spasms?
Research shows that fenfluramine can help treat certain seizure disorders. In people with Dravet syndrome, a type of epilepsy, studies have found that fenfluramine greatly reduces seizures. For instance, one study showed that 64.2% of patients responded well, with many experiencing fewer seizures. Another study on patients with Lennox-Gastaut syndrome, another severe form of epilepsy, found that about half of the patients had their seizures reduced by 50% or more. Based on these promising results, this trial will investigate fenfluramine for treating infantile spasms, especially when other treatments don't work.16789
Who Is on the Research Team?
Daniel Shrey, MD
Principal Investigator
Children's Hospital of Orange County
Are You a Good Fit for This Trial?
This trial is for children aged 1 to 3 with infantile spasms that haven't improved after treatment with ACTH and vigabatrin. Kids can't join if they have serious heart problems, used cannabinoids recently, started a new spasm treatment or diet, or got a vagal nerve stimulator in the last few weeks.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive fenfluramine treatment for 21 days, with dosage titrated to 0.8 mg/kg/day
Extension
Participants with favorable response may continue treatment for up to 6 months
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Fenfluramine
Fenfluramine is already approved in United States, European Union for the following indications:
- Seizures associated with Dravet syndrome
- Seizures associated with Lennox-Gastaut syndrome
- Seizures associated with Dravet syndrome
- Seizures associated with Lennox-Gastaut syndrome
Find a Clinic Near You
Who Is Running the Clinical Trial?
University of California, Los Angeles
Lead Sponsor
Children's Hospital of Orange County
Lead Sponsor