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NCT05281965
A Clinical Study Evaluating the Efficacy and Safety of Retinoic Acid in Patients With 15q11-q13 Duplication Syndrome
EARLY_PHASE1 trial testing Retinoic acid in Autism in 20 participants. Status unknown.
1 June 2023
Quick facts
| Lead sponsor | Second Affiliated Hospital, School of Medicine, Zhejiang University |
|---|---|
| Phase | EARLY_PHASE1 |
| Status | Status unknown |
| Study type | INTERVENTIONAL |
| Allocation | randomized |
| Design | crossover |
| Masking | single |
| Primary purpose | treatment |
| Enrollment | 20 |
| Start date | 1 April 2022 |
| Primary completion | 1 June 2023 |
| Estimated completion | 1 June 2024 |
| Sites | 2 locations across China |
Drugs / interventions tested
- Retinoic acid — full drug profile →
Conditions studied
- Autism — all drugs for Autism →
- Treatment Adherence — all drugs for Treatment Adherence →
- Retinoic Acid — all drugs for Retinoic Acid →
- Dup15Q Syndrome — all drugs for Dup15Q Syndrome →
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.
Verify or expand the search:
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Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT05281965 (US National Library of Medicine, public domain)
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Second Affiliated Hospital, School of Medicine, Zhejiang University
- Last refreshed: 16 March 2022
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