Clinical trials: Dup15q Syndrome
Dup15q Syndrome, Autism Spectrum Disorder
Investigating the Therapeutic Efficacy of All-trans Retinoic Acid in Autism Spectrum Disorder Patients With 15q11-13 Duplication Syndrome
Autism spectrum disorder (ASD) , hereafter referred to as autism, is a group of neurodevelopmental disorders caused by genetic and environmental factors.
- Conditions
- Dup15q Syndrome, Autism Spectrum Disorder, Therapy, All-trans Retinoic Acid
- Trial name
- NCT07079696
- Testing
- ATRA
- Where
- China
- Volunteers
- 90 people
- Sponsor
- Second Affiliated Hospital, Zhejiang University, School of Medicine
Epilepsy, Dravet Syndrome
A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic Encephalopathies
The purpose of this study is to investigate the effect on the frequency of all seizures (convulsive and drop) in participants treated with TAK-935 compared to placebo.
- Conditions
- Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome
- Trial name
- ELEKTRA (NCT03650452)
- Testing
- TAK-935
- Where
- 44 sites in United States, Australia and 6 more
- Volunteers
- 141 people
- Sponsor
- Takeda
15q Duplication Syndrome, CDKL5 Deficiency Disease
A Multicenter, Open-label, Pilot Study of Soticlestat (TAK-935/OV935) in Participants With 15Q Duplication Syndrome (Dup 15q) or Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder (ARCADE STUDY)
The purpose of this study is to investigate the effect of soticlestat on the frequency of motor seizures for participants with Dup15q or CDD during the Maintenance Period.
- Trial name
- NCT03694275
- Testing
- Soticlestat
- Where
- 8 sites in United States
- Volunteers
- 20 people
- Sponsor
- Takeda
Angelman Syndrome, Chromosome 15q Duplication (dup15q) Syndrome
Angelman Syndrome (AS) Biomarker Study
The primary objective of this study is to measure ubiquitin-protein ligase E3A (UBE3A) protein levels in cerebrospinal fluid (CSF) and to evaluate its utility as a biomarker in support of the development of therapies for AS.
- Trial name
- NCT04103333
- Testing
- Lumbar Puncture, Blood Collection
- Where
- 5 sites in United States
- Volunteers
- 20 people
- Sponsor
- Biogen
Dup15Q Syndrome
A Phase II Study of Basmisanil in Children and Adolescents With Dup15q Syndrome
The primary goal of this Phase 2 clinical trial is to determine the effects of a selective negative allosteric modulator (NAM) of GABAA α5 receptor, Basmisanil (NTX-1511), on brain wave (EEG) characteristics in children and…
- Trial name
- NCT07846215
- Testing
- Basmisanil
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Newleos Therapeutics, Inc.
Epilepsy, Angelman Syndrome
Structural-functional Connectome in Drug-resistant Epilepsies and Neurodevelopmental Syndromes With Epilepsy
Recent studies have shown that the aperiodic part of the signal (neuronal avalanches) of electroencephalography (EEG) contains important information about the dynamics of neuronal networks.
- Conditions
- Epilepsy, Angelman Syndrome, Dup15q Syndrome
- Trial name
- NCT06353620
- Testing
- High Density EEG recording, Rey Complex Figure Test
- Where
- Italy
- Volunteers
- 120 people
- Sponsor
- IRCCS Eugenio Medea
Dup15q Syndrome
A Study to Evaluate the Safety and Efficacy of Basmisanil Treatment in Children Aged 2-14 Years With Dup15q Syndrome
This study consists of two parts. Part 1 will evaluate the safety, efficacy, and pharmacodynamics of 52-weeks of basmisanil treatment in children and adolescents (aged 2-14 years) with Dup15q syndrome.
- Trial name
- NCT05307679
- Testing
- Basmisanil
- Where
- 5 sites in United States, Spain and 1 more
- Volunteers
- 7 people
- Sponsor
- Hoffmann-La Roche
Fragile X Syndrome, Angelman Syndrome
Parent and Infant Inter(X)Action Intervention (PIXI)
The objective is to develop and test, through an iterative process, an intervention to address and support the development of infants with a confirmed diagnosis of a neurogenetic disorder with associated developmental delays or…
- Conditions
- Fragile X Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Dup15Q Syndrome
- Trial name
- NCT03836300
- Testing
- Parent-Infant Inter(X)action Intervention (PIXI)
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- RTI International
Autism, Treatment Adherence
A Clinical Study Evaluating the Efficacy and Safety of Retinoic Acid in Patients With 15q11-q13 Duplication Syndrome
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.
- Conditions
- Autism, Treatment Adherence, Retinoic Acid, Dup15Q Syndrome
- Trial name
- NCT05281965
- Testing
- Retinoic acid
- Where
- 2 sites in China
- Volunteers
- 20 people
- Sponsor
- Second Affiliated Hospital, Zhejiang University, School of Medicine