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Study on the Effects of Mirtazapine for Improving Symptoms in Patients with Rett Syndrome

Verified siteRegistered drugNo placebo
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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 the medication mirtazapine in individuals with Rett syndrome. Rett syndrome is a rare genetic disorder that affects brain development, leading to severe physical and mental disabilities. The study aims to see if mirtazapine can help improve social behavior, facial and breathing functions, and physical movements in those affected by this condition.

Participants in the study will receive mirtazapine, which is given as an oral solution. The study will observe how the medication affects symptoms based on the age and severity of the condition in each participant. The goal is to determine which symptoms mirtazapine can improve and to what extent.

The study will take place over a period of time, during which participants will be monitored for changes in their condition. The effectiveness of the treatment will be assessed using various scales and measurements, such as the Motor-Behavior Assessment Scale and the Anxiety, Depression, and Mood Scale. The study will also look at improvements in sleep patterns, hand function, and overall behavior. The results will help understand the potential benefits of mirtazapine for those living with Rett syndrome.

The research process

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

  1. Step 1

    Introduction to the study

    The study is designed to evaluate the effectiveness of mirtazapine in improving social behavior, orofacial/respiratory, and motor/physical areas in patients with Rett syndrome.

    The study will assess which symptoms mirtazapine can improve, depending on the age and severity of the condition.

  2. Step 2

    Medication administration

    The medication used in the study is Mirtapil, an oral solution containing mirtazapine at a concentration of 15 mg/ml.

    The dosage, frequency, and duration of administration will be determined by the study protocol and communicated to the caregiver.

  3. Step 3

    Monitoring and assessment

    The effectiveness of the treatment will be evaluated using various scales and measurements, including the Motor-Behavior Assessment Scale (MBAS), Anxiety, Depression, and Mood Scale (ADAMS), and the Rett Syndrome Behaviour Questionnaire (RSBQ).

    Additional assessments will include measurements from medical devices for remote sensing, such as the Youcare Smart T-shirt and Actigraphy, as well as the Sleep Disturbances Scale for Children (SDSC).

    The Purposeful Hand Function scale (PHF) and the Clinical Global Impression of Change scale (CGI-C) will also be used to assess improvements.

    The Rett Syndrome Severity Scale (RCSS) and the Parenting Stress Index (PSI-SF) will be used to evaluate changes in severity and parental stress, respectively.

    Biomarker levels, including BDNF, GDNF, and PDGF, will be measured to assess the biological response to the treatment.

  4. Step 4

    Study duration

    The estimated end date for the study is August 31, 2025.

    The recruitment for the study is expected to start on November 1, 2024.

Who can join the trial?

15 criteria

  • Only female patients can participate.
  • Participants must be older than 5 years.
  • The person taking care of the patient must understand the study's purpose and be able to help with the study.
  • The patient must weigh more than 10 kg.
  • The patient must have a confirmed diagnosis of Rett Syndrome (RTT) due to a change in the MECP2 gene.
  • The patient must have breathing problems, such as:
    • Stopping breathing for short periods (periodic apnea).
    • Breathing too fast at times (intermittent hyperventilation).
    • Holding their breath.
    • Swallowing air.
    • Forcing air or saliva out.
    • The patient must have at least ten episodes of breathing problems during the day in the week before the first study visit, as reported by the caregiver.
    • The patient must have been on a stable medication plan for 4 weeks before starting the study.
    • If the patient is of childbearing age, they must have a negative pregnancy test.
    • A written consent form must be signed by the patient's parent, legal guardian, or representative before the first study visit.
    • The patient must be willing and able to complete the study with the help of their caregiver.

Who cannot join the trial?

4 criteria

  • Only females can participate in the study. Males are not eligible.
  • Participants must be within a specific age range. If you are outside this range, you cannot join the study.
  • Individuals with certain medical conditions other than Rett Syndrome may not be eligible.
  • Participants should not be part of any other vulnerable population that might be at risk during the study.
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Investigated drugs

Mirtazapine is a medication being studied for its potential benefits in treating symptoms of Rett syndrome. This study is exploring how it might improve social behavior, as well as orofacial, respiratory, and motor functions in individuals with this condition. The goal is to understand which symptoms Mirtazapine can help with, considering the age and severity of the condition in each participant.

What is already known about the treatment

Mirtazapine – Mirtazapine is administered orally, typically in tablet form. It is currently being studied in a Phase II clinical trial for its potential use in treating Rett Syndrome, a rare neurological disorder. The main therapeutic goal is to improve social behavior, as well as orofacial, respiratory, and motor functions in affected individuals. Mirtazapine works by modulating neurotransmitters in the brain, particularly by enhancing the release of norepinephrine and serotonin, which can help improve mood and behavior. It is classified as a tetracyclic antidepressant.

Investigated diseases

Rett Syndrome – Rett Syndrome is a rare genetic neurological disorder that primarily affects girls. It typically becomes apparent after 6 to 18 months of age, following a period of normal development. The condition is characterized by a loss of purposeful hand skills, speech, and motor abilities, along with the development of repetitive hand movements. Individuals with Rett Syndrome often experience problems with walking, coordination, and breathing. As the syndrome progresses, it can lead to severe impairments in communication and social interaction. The disorder is caused by mutations in the MECP2 gene, which is crucial for brain development.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-515411-21-00Protocol codeMirtaRettEstimated enrolment54 patientsSponsorUniversity Of Trieste

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