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Nuovo impiego del farmaco Mirtazapina nella cura di pazienti affette da sindrome di Rett, con disturbi tipici della patologia.
EU CTIS ID: 2024-515411-21-00
What this study is testing
The study aims to evaluate the efficacy of Mirtazapine (MTZ) to induce improving changes in social behaviour, orofacial/respiratory and motor/physical area. This study will provide information on what symptoms will Mirtazapine be able to correct, depending on the age and severity of the subject.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Female patients aged > 5 years
- The caregiver is able to understand the purpose of the protocol and to cooperate in the study
- Body weight > 10 kg
- Diagnosis of RTT confirmed by MECP2 gene mutation
- Respiratory dysfunction (at least one of the following): periodic apnoea, intermittent hyperventilation, breath holding, swallowing of air, forced expulsion of air and/or saliva
- Ten or more episodes per day of respiratory dysfunction during wakefulness in the week preceding the screening visit (reported by the caregiver)
You likely can't join if
- They are participating in another investigational clinical trial
- Hypersensitivity to MTZ
- 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 at ECG > 450 msec
- Scheduled surgery during the study
- Severe diabetes mellitus (hyperglycaemia with values above 250/300 mg/dL)
See the full eligibility criteria
- Female patients aged > 5 years
- The caregiver is able to understand the purpose of the protocol and to cooperate in the study
- Body weight > 10 kg
- Diagnosis of RTT confirmed by MECP2 gene mutation
- Respiratory dysfunction (at least one of the following): periodic apnoea, intermittent hyperventilation, breath holding, swallowing of air, forced expulsion of air and/or saliva
- Ten or more episodes per day of respiratory dysfunction during wakefulness in the week preceding the screening visit (reported by the caregiver)
- Stable medication regimen for 4 weeks prior to the start of the study
- Females of childbearing age only with negative pregnancy test
- Written consent signed by parent/legal guardian/representative prior to screening visit
- The patient is cooperative, willing and able to complete the study with caregiver assistance
- They are participating in another investigational clinical trial
- Hypersensitivity to MTZ
- 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 at ECG > 450 msec
- Scheduled surgery 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) <13
The study team makes the final eligibility decision.
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 listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling female, 0-17 years, 18-64 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.