Could a common antidepressant ease rett syndrome?
NCT ID NCT07430046
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
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 54 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jul 2025
- Expected to finish
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Dec 2026
An estimate. End dates often move.
- Lead sponsor
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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
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5 to 40 years
- Sex
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Female participants only
- Healthy volunteers
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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 Hide 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.
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.
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The places running it
4 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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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
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Centro Epilessia - Unità Neurologia Pediatrica, ASST Ospedale Santi Carlo Paolo - Dipartimento Scienze della Salute, Università di Milano
RECRUITINGMilan, 20142, Italy
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UOC di Neuropsichiatria Infantile, Policlinico Universitario "Gaetano Martino" di Messina, University of Messina. Messina
RECRUITINGMessina, 98125, Italy
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Unità di Neuropsichiatria Infantile, IRCCS, Istituto Giannina Gaslini, Genova
RECRUITINGGenova, 16147, Italy
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Unità di Pediatria, Dipartimento della Donna e dei Bambini - Policlinico S. M. alle Scotte. Siena
RECRUITINGSiena, 53100, Italy
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