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New treatment option for RETT Syndrome With Proven MECP2 Mutation
Official title Repurposing Mirtazapine in Rett Syndrome
ClinicalTrials.gov ID: NCT07430046
What this study is testing
What is mirtazapine?
mirtazapine is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for rett syndrome with proven mecp2 mutation.
Also referred to as MirtaPil oral solution, Remeron oral solution.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Rett Syndrome (RTT) is a rare neurodevelopmental disorder caused by an MECP2 gene mutation on the X chromosome, primarily affecting females. It causes progressive motor and cognitive decline, loss of speech, repetitive hand movements, breathing issues, seizures, and sleep problems.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 5 years
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 5 to 40, women only
You may be able to join if
- 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...
You likely can't join if
- 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...
- QTcF interval on the ECG greater than 450 msec
- Surgery planned during the study.
See the full eligibility 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...
- 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...
- 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.
- 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...
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Genova, Italy
- Messina, Italy
- Milan, Italy
- Siena, Italy
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 5 years per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling female, 5 years to 40 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Genova, Italy; Messina, Italy; Milan, Italy; Siena, Italy. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.