CompletedPhase 2

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

Germany13 participantsStarted 2024-08-02

Plain-language summary

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. The main questions it aims to answer are: * Is a daily oral therapy with zinc in GNAO1 associated disorders a safe and feasible therapy? * Are there potential changes in general motor skills, general behaviour and well being, day/night rhythm, level of dyskinesia and dystonia, frequency of seizures, quality of life and changes in the microbiome of the patients. Participants with GNAO1 associated disorders will be given an oral zinc therapy for 6 month and will be assessed in 3 visits and 2 phone calls within this trial.

Who can participate

Age range

6 Months – 30 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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 w…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Feasibility of daily treatment with oral zinc in GNAO1 as assessed by diary.

Timeframe: From first visit at Inclusion to visit after 6 month

Safety of daily administered zinc in GNAO1 as assessed by regular evaluation of the side effects

Timeframe: From first visit at inclusion until last phone call after 7 month

Safety of daily administered zinc in GNAO1 as assessed by regular blood tests

Timeframe: Blood analysis at baseline, after 3 and 6 month

Trial details

NCT IDNCT06412653

SponsorChildren's University Hospital Cologne, Germany

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2025-08-04

View on ClinicalTrials.gov More GNAO1 trials