Could a common mineral ease rare genetic disorder? zinc trial shows promise
NCT ID NCT06412653
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This pilot trial tested whether taking oral zinc every day for 6 months is safe and doable for people with GNAO1 disorders, a rare genetic condition that causes movement problems, seizures, and developmental delays. 13 participants aged 6 months to 30 years took zinc acetate. The study checked if they could stick to the daily dose and monitored side effects through blood tests and check-ins. It's a first step to see if zinc might help manage symptoms.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- zinc acetate dihydrate (oral zinc)
- What this could lead to
- If it works, this could point toward a simple, safe treatment to help manage movement problems and seizures in GNAO1 disorders.
- What could go wrong
- This is a very small, early pilot study with only 13 participants and no placebo group. It only tests feasibility and safety, not whether zinc actually improves symptoms.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
-
13 people
The number who actually took part.
- Started
-
Aug 2024
- Finished
-
Aug 2025
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
6 months to 30 years
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Children's Hospital, University Hospital Cologne, University of Cologne
Cologne, 50937, Germany
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