CTIS2024-512735-72-00
Recruiting
Phase 1
Prospective pilot trial to address the feasibility and safety of treatment with oral Zinc in GNAO1 associated disorders (ZINCGNAO1) - Uni-Koeln-5275
niversity Of Cologne0 sites12 target enrollmentApril 3, 2024
ConditionsGNAO1 associated disordersMedDRA version: 26.0Level: PTClassification code: 10064062Term: Neurodevelopmental disorder Class: 100000004873Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- GNAO1 associated disorders
- Sponsor
- niversity Of Cologne
- Enrollment
- 12
- Status
- Recruiting
- Last Updated
- last year
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •GNAO1 associated neurological disorder, documented by either (1\)Proven pathogenic or likely pathogenic mutation in GNAO1 or (2\)a variant of unknown significance in GNAO1 and clinical symptoms likely to be consistent with GNAO1 as determined by the investigators and at least one of the common symptoms of GNAO1: Movement disorder (dystonia, chorea, ataxia, stereotypic movements, clonic), central muscular hypotonia, epilepsy, global developmental delay, Age: 6month\-30years, Gross\-Motor\-Function measure\-66 (GMFM\-66\) \= 75, Written informed consent prior to any trial\-related procedure by parents or legal guardian, Stable on following concomitant treatments for at least 3 months prior to trial inclusion: anti\-seizure medication (ASD); baclofen, Deep brain stimulation settings
Exclusion Criteria
- •Treatment of Zinc in the last 4 months before inclusion, Known other genetic variants that are known to cause symptoms like observed in GNAO1\-related disorders, additional to the proven GNAO1 mutation, Implantation of Deep brain stimulation planned during the duration of the trial, i.e. in the six months after inclusion, Start of intrathecal baclofen therapy planned during the duration of the trial, i.e. in the six months after inclusion, Known allergy/hypersensitivity to the scheduled trial drug, Concomitant participation in other clinical drugs with investigational drugs or with competing interventions, Sexually active participants who are not willing to use/ not using a highly effective contraception method with a pearl\-index \< 1\. Sexually active participants resp. their partner, unless surgically sterile, must be using a highly effective contraception method (including oral, transdermal, injectable or implanted contraceptives, IUD, using a condom of the sexual partner or sterile sexual partner) and must agree to continue using such precautions during the whole study period, Pregnant women and nursing mothers
Outcomes
Primary Outcomes
Not specified
Similar Trials
Not yet recruiting
Phase 4
Point-of-care Urine Monitoring of Adherence (PUMA): Testing a Real-Time Urine Assay of Tenofovir in PrEPPACTR202003901399017niversity of California San Fransisco100
Completed
Not Applicable
Pilot trial to assess the feasibility and response rates for an RCT evaluating the effectiveness of a computer tailored intervention for smoking cessation in general practiceISRCTN34254423Record Provided by the NHSTCT Register - 2004 Update - Department of Health (UK)800
Completed
Not Applicable
Dignity Therapy: A Psychotherapeutic Intervention to Enhance the End of Life Experience for Persons with Motor Neurone Disease and their Family CarersMotor Neurone DiseaseAmyotrophic Lateral SclerosisNeurological - Neurodegenerative diseasesACTRN12611000410954School of Psychology and Speech Pathology50
Completed
Not Applicable
A pilot study to assess the feasibility, acceptability, and impact of an intervention to address the potential effects of social networking site use on well-being.ACTRN12622001057774niversity of Adelaide59
Completed
Not Applicable
A pilot study to assess the feasibility of a prospective randomised controlled trial of a patient-centred medicines management approach to reduce the burden of iatrogenic symptoms in palliative careAll patients referred to pallaitive care, irrespective of underlying diagnosis.Other - Conditions of unknown or disputed aetiology (such as chronic fatigue syndrome/myalgic encephalomyelitis)ACTRN12611001262998Flinders University40