Prospective Pilot Trial to Address Feasibility and Safety of Oral Zinc in GNAO1 Associated Disorders

Launched by CHILDREN'S UNIVERSITY HOSPITAL COLOGNE, GERMANY · May 8, 2024

Keywords

ClinConnect Summary

This clinical trial is looking into whether taking zinc by mouth is a safe and effective treatment for patients with disorders linked to the GNAO1 gene. These disorders can cause issues like movement problems, seizures, and developmental delays. The study will involve participants aged between 6 months and 30 years who have been diagnosed with a GNAO1-related condition. To qualify for the trial, they must have a specific mutation in the GNAO1 gene or show symptoms that align with it. Participants will need to be stable on their current treatments for at least three months before joining the trial.

For six months, participants will take daily zinc supplements and will be monitored through three in-person visits and two phone calls. Researchers will assess any changes in motor skills, behavior, sleep patterns, and overall quality of life. It's important to note that individuals who have taken zinc recently, have other genetic disorders, or are pregnant or nursing cannot participate. This trial aims to see if oral zinc can be a new way to help manage these challenging conditions.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • GNAO1 associated neurological disorder, documented by either
• • Proven pathogenic or likely pathogenic mutation in GNAO1 or
• • 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, clonic), central muscular hypotonia, epilepsy, global developmental delay
• • Age: 6 month - 30 years
• • GMFM ≤ 75
• • written informed consent prior to any trial-related procedure (according to age and status of psycho-intellectual development)
• • of parents or legal guardian
• • of parents or legal guardian and patient
• • of the patient
• • stable on following concomitant treatments for at least 3 months prior to trial inclusion: anti-seizure drugs (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 patients who are not willing to use/ not using a highly effective contraception method with a pearl-index \< 1. Sexually active patients, unless surgically sterile, must be using a highly effective contraception method (including oral, transdermal, injectable or implanted contraceptives, intrauterine device (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