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NCT05281965

A Clinical Study Evaluating the Efficacy and Safety of Retinoic Acid in Patients With 15q11-q13 Duplication Syndrome

Status unknown EARLY_PHASE1 Last updated 16 March 2022
What this trial tests

EARLY_PHASE1 trial testing Retinoic acid in Autism in 20 participants. Status unknown.

Timeline
1 April 2022
Primary endpoint
1 June 2023
1 June 2024

Quick facts

Lead sponsorSecond Affiliated Hospital, School of Medicine, Zhejiang University
PhaseEARLY_PHASE1
StatusStatus unknown
Study typeINTERVENTIONAL
Allocationrandomized
Designcrossover
Maskingsingle
Primary purposetreatment
Enrollment20
Start date1 April 2022
Primary completion1 June 2023
Estimated completion1 June 2024
Sites2 locations across China

Drugs / interventions tested

Conditions studied

Sponsor

Second Affiliated Hospital, School of Medicine, Zhejiang University

Who can join

Adults 6 to 18, any sex, with Autism or Treatment Adherence. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Autism Spectrum Disorders (ASD), with its core symptoms of communication and repetitive behaviors, is a serious neurodevelopmental disorder common in childhood and affects about 1% of children. So far, autism remains a clinical dilemma with no effective therapy. The most common chromsomal ability among ASD patients is 15q11-13q duplication syndrome(dup15q syndrome).Clinical phenotypes of dup15q syndrome include autism, mental retardation, epilepsy (usually refractory epilepsy, often manifested as infantile spasm), congenital heart disease, mild facial abnormalities, etc. UBE3A is one of the most important genes in the 15q11-q13 region.Biochemistry and molecular biology of the Chinese Academy of Sciences Hu Ronggui group found a new kind of autism in mechanisms and potential therapeutic targets - describe the ubiquitin ligase UBE3A protein and retinoic acid.Previous studies have shown that the basis of the relevant treatment measures can effectively relieve the mouse model of autism characteristics. Therefore, retinoic acid supplementation in the treatment of dup15q syndrome is a potential therapeutic target.

Publications & conference data

No peer-reviewed publications indexed yet for this trial.

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Other trials of Retinoic acid

Trials testing the same drug.

Other recruiting trials for Autism

Currently open trials in the same condition.

Other Second Affiliated Hospital, School of Medicine, Zhejiang University trials

Trials by the same sponsor.

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Data sources for this page

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