Prospective pilot trial to address the feasibility and safety of treatment with oral Zinc in GNAO1 associated disorders (ZINCGNAO1)

Phase 1

Recruiting

Conditions: GNAO1 associated disorders
MedDRA version: 26.0Level: PTClassification code: 10064062Term: Neurodevelopmental disorder Class: 100000004873
Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Registration Number: CTIS2024-512735-72-00

Lead Sponsor: niversity Of Cologne

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: All

Target Recruitment: 12

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