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Could a common antidepressant ease rett syndrome?

NCT ID NCT07430046

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase II trial is testing whether mirtazapine, an antidepressant, can improve mood, sleep, and motor symptoms in people with Rett syndrome. The study will enroll 54 females aged 5 to 40 with a confirmed MECP2 mutation. Participants will receive increasing doses of mirtazapine over six months, and researchers will monitor safety and symptom changes.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
mirtazapine (an antidepressant)
What this could lead to
If it works, this could point toward a treatment to improve mood, sleep, and hand control in people with Rett syndrome.
What could go wrong
This is an early, small, open-label trial with no placebo group, so results may be less reliable. It is not a cure and may not work for everyone.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 54 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jul 2025

Expected to finish

Dec 2026

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

5 to 40 years

Sex

Female participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

INCLUSION CRITERIA * 1\. Female aged 5 to 39 years inclusive, at the time of signing the informed consent. * 2\. Girls of childbearing age negative to pregnancy test; * 3\. Body weight \> 10 kg. and within the expected range for RTT, based on age and height. * 4\. Diagnosis of RTT based on consensus clinical criteria (Neul, 2010) and a confirmed mutation in MECP2 gene. * 5\. Breathing dysfunction (at least one of the following): period apnoea, intermittent hyperventilation, breath holding spells, air swallowing, forced expulsion of air and /or saliva. * 6\. Ten episodes or more/day of breathing dysfunction during wakefulness in the week prior to the screening visit (parents report). 7\. Stable medication regimen for 4 weeks prior to beginning the study (if receiving services - physical, occupational, or speech therapy - subjects must be on a stable regimen of these services for 3 months prior to beginning the study). * 8\. Female patients of childbearing potential must use a highly effective contraceptive method such as combined hormonal contraception (containing estrogen and progestin) associated with ovulation suppression (oral, intravaginal, transdermal); progestin-only hormonal contraception associated with ovulation suppression (oral, injectable, implantable); intrauterine device; hormone-releasing intrauterine system. Sexual abstinence is considered a highly effective contraceptive method if it aligns with the individual's usual lifestyle. Female patients of childbearing potential are to use adequate contraception as recommended by their Health Care Provider. * 9\. Written consent signed by parent/legal guardian/representative prior to screening visit * 10\. Patient is cooperative, willing to complete the study, and capable of doing so with assistance of a caregiver. * 11\. Caregiver is able to understand the instructions and fully participate. EXCLUSION CRITERIA Participants are excluded from the study if any of the following criteria apply: * Patient is participating to another investigational clinical trial. * Hypersensitivity to MTZ or any of the other ingredients of Mirtapil®. * Clinically significant (as determined by the investigator) cardiovascular, respiratory, gastrointestinal, renal, hepatic, haematological pathologies or other pathologies, in addition to those directly related to RTT. In particular, patients with the following parameters will be excluded: leucocyte count is \< 4000/mm2; neutrophil count is \< 2000/mm3; hyponatremia (\< 125 mmol/L); renal dysfunction (creatinine \> 2 X ULN), hepatic dysfunction (AST, ALT, bilirubin \> 2 X ULN); or if severe diabetes mellitus is present. * QTcF interval on the ECG greater than 450 msec * Surgery planned during the study. * Severe diabetes mellitus (hyperglycaemia with values above 250/300 mg/dL); * Pregnancy, breastfeeding. * Evidence of clinically significant malnutrition with BMI (or BMI) (kg/m2) \< * Patients who manifested prior suicidal ideation.

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Conditions

The condition(s) this trial relates to.

Cognitive Dysfunction Neurologic Manifestations Rett syndrome sleep-wake disorder

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    4 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Centro Epilessia - Unità Neurologia Pediatrica, ASST Ospedale Santi Carlo Paolo - Dipartimento Scienze della Salute, Università di Milano

    RECRUITING

    Milan, 20142, Italy

  • UOC di Neuropsichiatria Infantile, Policlinico Universitario "Gaetano Martino" di Messina, University of Messina. Messina

    RECRUITING

    Messina, 98125, Italy

  • Unità di Neuropsichiatria Infantile, IRCCS, Istituto Giannina Gaslini, Genova

    RECRUITING

    Genova, 16147, Italy

  • Unità di Pediatria, Dipartimento della Donna e dei Bambini - Policlinico S. M. alle Scotte. Siena

    RECRUITING

    Siena, 53100, Italy

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