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Double-blind, randomized, placebo-controlled study of Leriglitazone in pediatric Rett Syndrome
EU CTIS ID: 2024-514684-26-00
What this study is testing
To evaluate the safety and tolerability of leriglitazone in RTT subjects.
- 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
- Free written Informed Consent Form (ICF) by their parent(s)/legal guardian(s) or authorized legal representative(s) prior to any procedure required by the study.
- Female subjects aged ≥5 and ≤12 years, at the time of signing the ICF.
- Diagnosis of classical/typical RTT, according to 2010 criteria with a documented mutation of the MECP2 gene
- Severity rating of between 10 and 36 (RTT Natural History/Clinical Severity Scale).
- Be able to swallow the study treatment provided as an oral liquid suspension.
- A female subject is eligible if she meets one of the following criteria: − is of non-childbearing potential, see Contraception Requirements; or − is of childbearing potential and agrees to use an accepted contraceptive method from at least 4 weeks prior to Enrolment until 4 weeks after the End of Treatment (EOT) or Premature Discontinuation Visit (PDV), see Contraception Requirements
You likely can't join if
- Actively undergoing neurological regression.
- Conditions that could modify absorption of the study drug.
- Known hereditary fructose intolerance (HFI).
- Positive result in serum beta human chorionic gonadotropin (hCG) pregnancy test, if childbearing potential or lactating girl.
- Other medical, neurologic, psychiatric, or social condition that, in the opinion of the Investigator, is likely to unfavourably alter risk-benefit of study participation, confound interpretation of safety or efficacy results, or interfere with the satisfactory completion of study requirements.
- Has any of the following: − QT interval (corrected by Bazett’s formula) of >460 ms at Screening or Baseline (before dosing). − History of a risk factor for torsade de pointes (e.g., heart failure or family history of long QT syndrome). − History of clinically significant QT prolongation that is deemed to put the subject at increased risk of clinically significant QT prolongation or treatment for known QTc prolongation with drugs such as betablockers
See the full eligibility criteria
Who can join
- Free written Informed Consent Form (ICF) by their parent(s)/legal guardian(s) or authorized legal representative(s) prior to any procedure required by the study.
- Female subjects aged ≥5 and ≤12 years, at the time of signing the ICF.
- Diagnosis of classical/typical RTT, according to 2010 criteria with a documented mutation of the MECP2 gene
- Severity rating of between 10 and 36 (RTT Natural History/Clinical Severity Scale).
- Be able to swallow the study treatment provided as an oral liquid suspension.
- A female subject is eligible if she meets one of the following criteria: − is of non-childbearing potential, see Contraception Requirements; or − is of childbearing potential and agrees to use an accepted contraceptive method from at least 4 weeks prior to Enrolment until 4 weeks after the End of Treatment (EOT) or Premature Discontinuation Visit (PDV), see Contraception Requirements
What rules you out
- Actively undergoing neurological regression.
- Conditions that could modify absorption of the study drug.
- Known hereditary fructose intolerance (HFI).
- Positive result in serum beta human chorionic gonadotropin (hCG) pregnancy test, if childbearing potential or lactating girl.
- Other medical, neurologic, psychiatric, or social condition that, in the opinion of the Investigator, is likely to unfavourably alter risk-benefit of study participation, confound interpretation of safety or efficacy results, or interfere with the satisfactory completion of study requirements.
- Has any of the following: − QT interval (corrected by Bazett’s formula) of >460 ms at Screening or Baseline (before dosing). − History of a risk factor for torsade de pointes (e.g., heart failure or family history of long QT syndrome). − History of clinically significant QT prolongation that is deemed to put the subject at increased risk of clinically significant QT prolongation or treatment for known QTc prolongation with drugs such as betablockers
- Reduced left-ventricular ejection fraction (LVEF) ˂ 50%, or other clinically significant cardiac abnormalities on echocardiogram that in the Investigator’s opinion could predispose the subject to volume overload or its attendant consequences.
- Non-stable epilepsy/status epilepticus in the last 8 weeks.
- Previous or current history of cancer, unless surgically resected and without evidence of recurrence for a minimum of 5 years.
- Known type 1 or type 2 diabetes.
- Use of pioglitazone or other thiazolidinediones within the past 6 months prior to screening.
- Known hypersensitivity/allergic reaction or intolerance to pioglitazone or any other thiazolidinedione.
- Known hypersensitivity/allergic reaction to the study drug substance or any of the excipients.
- Has a requirement for treatment with a prohibited concomitant medication, specified in Protocol (Previous and Concomitant Medication), including the antiepileptic medication (Carbamazepine, Clobazam, Perampanel and/or Zonisamide), the antipsychotic medication (Risperidone), and medications known to prolong QTc interval.
- Clinically significant anemia with hemoglobin <10 g/dL.
- Abnormal liver enzyme tests for aspartate transaminase (AST) or alanine transaminase (ALT) of >2.5 × the upper limit of normal (ULN).
- Subject has moderate to severe renal impairment (GFR ˂ 60 mL/min)
- Participation in any clinical trial within the previous 3 months.
- Subject has mild, moderate, or severe hepatic impairment (Child-Pugh classification groups A, B or C).
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. 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.