Drug Trial Snapshot: DIACOMIT

HOW TO USE THIS SNAPSHOT
The information provided in Snapshots highlights who participated in the clinical trials that supported the FDA approval of this drug, and whether there were differences among sex, race and age groups. The “MORE INFO” bar shows more detailed, technical content for each section. The Snapshot is intended as one tool for consumers to use when discussing the risks and benefits of the drugs.

LIMITATIONS OF THIS SNAPSHOT:
Do not rely on Snapshots to make decisions regarding medical care. Always speak to your health provider about the risks and benefits of a drug. Refer to the DIACOMIT Package Insert for complete information.

DIACOMIT (stiripentol)
dī-ə-ka-mət
Biocodex
Approval date: August 20, 2018

DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

DIACOMIT is a drug used along with another drug (clobazam) to treat seizures in patients with Dravet syndrome who are 2 years of age or older. Dravet syndrome is a rare and severe form of epilepsy that starts in early childhood. It is associated with difficult to control seizures and various degrees of development disability.

How is this drug used?

The total daily dose of DIACOMIT is based on a patient’s weight, and it is then divided into two or three doses. DIACOMIT can be taken as a capsule or as a powder. DIACOMIT capsules should be swallowed whole with a glass of water during a meal. DIACOMIT powder is mixed in a glass of water and should be taken right away after mixing during a meal.

What are the benefits of this drug?

Patients with Dravet syndrome taking DIACOMIT along with other seizure medications experienced fewer seizures (generalized clonic or tonic-clonic type) than patients taking placebo with other seizure medications. In Trial 1, 71% of the patients taking DIACOMIT responded to treatment compared with 5% of the placebo group. In the second trial, 67% of the patients taking DIACOMIT responded to treatment and 9% to placebo. A patient was classified as a ‘responder’ if the number of seizures in the second month of treatment was at least 50% lower than the number in the month before treatment was started.

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Were there any differences in how well the drug worked in clinical trials among sex, race and age?

Sex: DIACOMIT worked similarly in males and females.
Race: Data on race were not collected per local regulatory authorities.
Age: Age groups were too small to allow a determination of whether there were any differences among them in how well the drug works.

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What are the possible side effects?

DIACOMIT may cause serious side effects, including excessive sleepiness, loss of appetite, weight loss, decrease in blood cell counts, and thoughts about suicide or dying.

The most common side effects of DIACOMIT are sleepiness, decreased appetite, agitation, ataxia (impaired balance or coordination), weight loss, low muscle tone, nausea, shaking, dysarthria (difficulty speaking words; difficulty forming words during speech), and insomnia (difficulty sleeping).

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Were there any differences in side effects among sex, race and age?

Sex: The occurrence of side effects was similar in males and females.
Race: Data on race were not collected per local regulatory authorities.
Age: Age groups were too small to allow a determination of whether there were any differences among them in occurrence of side effects.

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WHO WAS IN THE STUDIES?

Who participated in the clinical trials?

The FDA approved DIACOMIT based primarily on evidence from two clinical trials (Trial 1 and Trial 2) of 64 patients with Dravet syndrome. Trial 1 was conducted in 15 centers in France and Trial 2 in 6 centers in Italy.

The demographics of these patients are presented below.

Figure 1 summarizes how many men and women were enrolled in the clinical trials.

Figure 1. Baseline Demographics by Sex (safety population)

Pie chart summarizing how many males and females were in the clinical trials. In total, 30 males (47%) and 34 females (53%) participated in the clinical trials.

FDA Review

Baseline Demographics by Race (safety population)

Data were not collected per local regulatory authority.

Figure 2 summarizes the percentage of patients by age in the clinical trials used to evaluate safety.

Figure 2. Baseline Demographics by Age (safety population)

Pie charts summarizing how many individuals of certain age groups were in the clinical trials. In total, 47 patients were younger than 12 years (73%), and 17 patients were 12 years and older (27 %)

FDA Review

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How were the trials designed?

The benefits and side effects of DIACOMIT were primarily evaluated in two clinical trials. These trials enrolled patients with Dravet syndrome whose seizures were not well controlled on their current treatment, which included clobazam and valproate.

All enrolled patients used their current treatment for seizures for one month. After this baseline period, patients were randomly assigned to receive DIACOMIT or placebo two to three times a day in addition to their baseline treatment. Patients were then observed on that treatment for two months. Neither the patients nor the health care providers knew whether DIACOMIT or placebo was being given until after the trial was completed.

The benefit of DIACOMIT was evaluated by measuring the frequency of seizures (generalized clonic or tonic- clonic) as recorded by patients and/or their caregivers, using a diary and then comparing it to the frequency of seizures in placebo treated patients. The primary assessment of benefit was based on the percentage of patients who ‘responded’ to treatment. A patient was classified as a ‘responder’ if the number of seizures in the second month of treatment was at least 50% lower than the number in the month before treatment was started.

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GLOSSARY

CLINICAL TRIAL: Voluntary research studies conducted in people and designed to answer specific questions about the safety or effectiveness of drugs, vaccines, other therapies, or new ways of using existing treatments.
COMPARATOR: A previously available treatment or placebo used in clinical trials that is compared to the actual drug being tested.
EFFICACY: How well the drug achieves the desired response when it is taken as described in a controlled clinical setting, such as during a clinical trial.
PLACEBO: An inactive substance or “sugar pill” that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo.
SUBGROUP: A subset of the population studied in a clinical trial. Demographic subsets include sex, race, and age groups.