RecruitingRare disease

Study on the Effectiveness and Safety of Apremilast for Patients Aged 6 and Older with Generalized Epidermolysis Bullosa Simplex

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the effects of a medication called Otezla, which contains the active ingredient apremilast. The study is designed to evaluate the safety and effectiveness of this medication in treating a rare skin condition known as epidermolysis bullosa simplex (EBS). EBS is a genetic disorder that causes the skin to be very fragile, leading to the formation of blisters from minor injuries or friction. The trial will involve patients who are 6 years of age or older and have a confirmed diagnosis of a severe form of EBS due to specific genetic mutations.

The purpose of the study is to assess how well apremilast works in reducing the symptoms of EBS, such as the number of new blisters, and to evaluate its safety for patients. The study will follow a specific design where patients will receive the medication in different phases to observe its effects. Participants will take Otezla in the form of film-coated tablets, which are taken orally. The maximum daily dose is 60 milligrams, and the treatment period can last up to 16 weeks. During the study, some participants may receive a placebo, which is a substance with no active medication, to compare the effects.

Throughout the trial, researchers will monitor various aspects of the patients' health, including the severity of their condition, any itching or pain they experience, and their overall quality of life. The study also aims to validate a new scale for measuring the severity of EBS. By the end of the trial, the researchers hope to gather valuable information on the potential benefits and risks of using apremilast for treating EBS, which could lead to improved treatment options for those affected by this challenging condition.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Participation begins after meeting the criteria: age 6 or older, confirmed diagnosis of epidermolysis bullosa simplex (EBS) due to specific genetic mutations, and a daily average of more than three new blisters.
Agreement to avoid any topical treatments not approved by the study investigator is required.
Step 2
Medication administration
The medication used in the study is Otezla, which contains the active substance apremilast.
The medication is taken orally in the form of film-coated tablets, available in dosages of 10 mg, 20 mg, and 30 mg.
Step 3
Treatment periods
The study follows a challenge-dechallenge-rechallenge design over a period of 20 weeks.
The primary goal is to evaluate the effectiveness of apremilast in treating EBS.
Step 4
Safety and efficacy assessment
Safety and tolerability are monitored by observing specific events during the treatment periods and for an additional week.
Secondary measures include assessing severity, itch, pain, dressing duration, quality of life, and compliance.
A new severity scale for EBS patients is validated during the study.

Who can join the trial?

5 criteria

Patients must be 6 years or older.
Both male and female patients can participate.
Patients must have a confirmed diagnosis of a specific type of epidermolysis bullosa simplex (EBS-sev) caused by a change in certain genes called KRT5 or 14.
Patients should have an average of more than 3 new blisters each day.
The patient or their caregiver must agree not to use any skin treatments unless they are approved by the study doctor.

Who cannot join the trial?

3 criteria

Patients who are younger than 6 years old cannot participate.
Patients who do not have a diagnosis of epidermolysis bullosa simplex cannot participate. This is a skin condition that causes the skin to be very fragile and to blister easily.
Patients who are part of a vulnerable population cannot participate. A vulnerable population includes groups of people who may need special protection, like children or people with certain disabilities.

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Verified sites

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Centre Hospitalier Regional Et Universitaire De Brest
Brest, France
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Centre Hospitalier Universitaire De Nice
Nice, France
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Hopital Beaujon
Clichy, France

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Investigated drugs

Apremilast is a medication being studied for its effectiveness and safety in treating patients aged 6 years and older with a skin condition called epidermolysis bullosa simplex generalized. This condition causes the skin to be very fragile and to blister easily. The trial aims to see how well Apremilast works in reducing the symptoms of this condition over different periods of treatment.

Apremilast

What is already known about the treatment

Apremilast – Apremilast is administered orally in tablet form. It is currently being studied in clinical trials for its effectiveness in treating epidermolysis bullosa simplex, a rare skin condition. The medication works by inhibiting an enzyme called phosphodiesterase 4 (PDE4), which helps reduce inflammation in the body. Apremilast is classified as a phosphodiesterase inhibitor and is being evaluated for its safety and efficacy in patients aged six years and older with this condition.

Investigated diseases

Epidermolysis Bullosa Simplex – This is a genetic skin disorder characterized by fragile skin that blisters easily, often in response to minor injuries or friction. The condition primarily affects the outer layer of the skin, leading to the formation of blisters and erosions. Blisters typically appear on the hands and feet but can occur anywhere on the body. The severity of the condition can vary, with some individuals experiencing mild symptoms and others having more extensive blistering. Over time, repeated blistering can lead to thickened skin and scarring. The condition is usually present from birth or early childhood and is caused by mutations in genes responsible for skin integrity.