Clinical trials

Clinical trials, made clear.

Find a study, understand its purpose and prepare questions for the medical team.

This is a partial selection. Statuses come from dated sources, not confirmation of an available place. Documented countries and sites are not a complete worldwide list.

6 study/studies in this selection
NCT07503444API RE2T

A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome

This study evaluates the efficacy of fenfluramine hydrochloride (HCl) versus placebo in participants with Rett syndrome (RTT).

Listed as recruitingINTERVENTIONAL
Documented country · BE · ES · FR · HU · IT · JP · PL · USCentre level · 23 Sites named in the sourcesHuman-reviewed
Source updated · Sep 18, 2026 · ClinicalTrials.gov
Source updated · Sep 17, 2026 · Synced · Sep 19, 2026 · Human review · Sep 19, 2026
Understand this trial

Résumé ou traduction assisté par IA : peut contenir des erreurs et ne remplace ni une relecture humaine ni un avis médical.

NCT05606614API RE2T

A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)

The study evaluates the safety, dose selection, efficacy and safety of a single intrathecal dose of TSHA-102 in females with typical Rett syndrome.

Ongoing · not recruitingINTERVENTIONAL
Documented country · CA · USCentre level · 9 Sites named in the sourcesHuman-reviewed
Source updated · Jul 7, 2026 · ClinicalTrials.gov
Source updated · Jul 6, 2026 · Synced · Sep 19, 2026 · Human review · Sep 19, 2026
Understand this trial

Résumé ou traduction assisté par IA : peut contenir des erreurs et ne remplace ni une relecture humaine ni un avis médical.

NCT05898620API RE2T

A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome

This study evaluates the efficacy and safety of the investigational gene therapy NGN-401 in females with typical Rett syndrome.

Ongoing · not recruitingINTERVENTIONAL
Documented country · AU · GB · USCentre level · 16 Sites named in the sourcesHuman-reviewed
Source updated · Jun 10, 2026 · ClinicalTrials.gov
Source updated · Jun 9, 2026 · Synced · Sep 19, 2026 · Human review · Sep 19, 2026
Understand this trial

Résumé ou traduction assisté par IA : peut contenir des erreurs et ne remplace ni une relecture humaine ni un avis médical.

NCT07430046API RE2T

Repurposing Mirtazapine in Rett Syndrome

This phase II study evaluates the safety and efficacy of mirtazapine in females with Rett syndrome, including effects on mood, sleep and motor symptoms.

Listed as recruitingINTERVENTIONAL
Documented country · ITCentre level · 4 Sites named in the sourcesHuman-reviewed
Source updated · Feb 24, 2026 · ClinicalTrials.gov
Source updated · Feb 23, 2026 · Synced · Sep 19, 2026 · Human review · Sep 19, 2026
Understand this trial

Résumé ou traduction assisté par IA : peut contenir des erreurs et ne remplace ni une relecture humaine ni un avis médical.

NCT04988867API RE2T

An Open-Label Study of Trofinetide for Girls Two to Five Years of Age With Rett Syndrome

This study investigates the safety and tolerability of long-term oral trofinetide treatment in girls with Rett syndrome.

Study ended earlyINTERVENTIONAL
Documented country · USCentre level · 7 Sites named in the sourcesHuman-reviewed
Source updated · Sep 24, 2024 · ClinicalTrials.gov
Source updated · Sep 23, 2024 · Synced · Sep 19, 2026 · Human review · Sep 19, 2026
Understand this trial

Résumé ou traduction assisté par IA : peut contenir des erreurs et ne remplace ni une relecture humaine ni un avis médical.

NCT04181723API RE2T

Study of Trofinetide for Girls and Women With Rett Syndrome (LAVENDER™)

This study investigates the efficacy of oral trofinetide versus placebo in females with Rett syndrome.

Study completedINTERVENTIONAL
Documented country · USCentre level · 21 Sites named in the sourcesHuman-reviewed
Source updated · Apr 8, 2024 · ClinicalTrials.gov
Source updated · Apr 7, 2024 · Synced · Sep 19, 2026 · Human review · Sep 19, 2026
Understand this trial

Résumé ou traduction assisté par IA : peut contenir des erreurs et ne remplace ni une relecture humaine ni un avis médical.