Clinical trials: GNAO1
GNAO1
GNAO1 Natural History Study
The purpose of the GNAO1 Natural History Study is to establish the clinical phenotype of GNAO1 associated neurologic disease, its association with genotype, and areas of clinical importance within the disease.
- Trial name
- GNAO1 (NCT04950946)
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Washington University School of Medicine
GNAO1, Dystonia
Prospective Pilot Trial to Address Feasibility and Safety of Oral Zinc in GNAO1 Associated Disorders
The goal of this clinical trial is to investigate feasibility and safety of an oral therapy with zinc in patients affected by Guanine nucleotide-binding protein G(o) subunit alpha (GNAO1) associated disorders.
- Conditions
- GNAO1, Dystonia, Epilepsy, Development Delay
- Trial name
- ZINCGNAO1 (NCT06412653)
- Testing
- Zinc Acetate Dihydrate
- Where
- Germany
- Volunteers
- 13 people
- Sponsor
- Children's University Hospital Cologne, Germany
TOR1A, PANK2
Deep Brain Stimulation (DBS) MatchMaker
DBS Matchmaker (https://www.dbsmatchmaker.com) is a platform designed to connect clinicians worldwide who are treating rare movement disorders with deep brain stimulation (DBS).
- Conditions
- TOR1A, PANK2, HPRT1, EIF2AK2
- Trial name
- DBSMatchMaker (NCT06912841)
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- Boston Children's Hospital
Epilepsy-Dyskinesia, Epilepsy
Registry and Natural History of Epilepsy-Dyskinesia Syndromes
The Registry and Natural History of Epilepsy-Dyskinesia Syndromes is focused on gathering longitudinal clinical data as well as biological samples (blood, urine, and/or skin/tissue) from male and female patients, of all ages, who…
- Conditions
- Epilepsy-Dyskinesia, Epilepsy, Dyskinesia, EDS
- Trial name
- NCT06967727
- Where
- United States
- Volunteers
- 700 people
- Sponsor
- Boston Children's Hospital
Epilepsy in Children, Dyskinesias
A Retrospective Survey-based Multicenter Study to Delineate the Molecular and Phenotypic Spectrum of Epilepsy-dyskinesia Syndromes
The Epilepsy-Dyskinesia Study aims to advance the understanding of the clinical and molecular spectrum of epilepsy-dyskinesia syndromes, monogenic diseases that cause both movement disorders and epilepsy.
- Conditions
- Epilepsy in Children, Dyskinesias, Movement Disorders in Children, Neurologic Disorder
- Trial name
- NCT06585605
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- Boston Children's Hospital
GNAO1, Epilepsy
Tianasen (ASO-GNAO1) for GNAO1-Encephalopathy With Epilepsy and Movement Disorders.
The goal of this clinical trial is to evaluate the efficacy and safety of the investigational drug ASO-GNAO1 (Tianasen) in pediatric patients with c.607G>A mutation in the GNAO1 gene associated with epilepsy and…
- Conditions
- GNAO1, Epilepsy, Hyperkinesis
- Trial name
- ASO-GNAO1 (NCT07363603)
- Testing
- Antisense oligonucleotide treatment (ASO)
- Where
- Russia
- Volunteers
- 5 people
- Sponsor
- Pirogov Russian National Research Medical University