NCT06412653CompletedPhase 2
Prospective Pilot Trial to Address Feasibility and Safety of Oral Zinc in GNAO1 Associated Disorders
Prospective Pilot Trial to Address the Feasibility and Safety of Treatment With Oral Zinc in GNAO1 Associated Disorders
Source: ClinicalTrials.gov · Last updated 3 Feb 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Completed
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 13 (actual)
- First submitted
- 29 Feb 2024
- Start date
- 2 Aug 2024
- Primary completion date
- 4 Aug 2025
- Lead sponsor
- Children's University Hospital Cologne, Germany
- Diseases
- Epilepsy
- NCT number
- NCT06412653
What you commit to
- Study type: Interventional · Phase 2
- Participants enrolled: 13
- From 2 Aug 2024 to the primary completion date, 4 Aug 2025
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Treatment of Zinc in the last 4 months before inclusion
- start of intrathecal baclofen therapy planned during the duration of the trial, i.e. in the six months after inclusion
- Concomitant participation in other clinical drugs with investigational drugs or with competing interventions
Other illness
- known other genetic variants that are known to cause symptoms like observed in GNAO1-related disorders, additional to the proven GNAO1 mutation
- Known allergy/hypersensitivity to the scheduled trial drug
Other
- implantation of Deep brain stimulation planned during the duration of the trial, i.e. in the six months after inclusion
- 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
Who the study is looking for
- 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
Age in the registry: 6 months to 30 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Children's Hospital, University Hospital Cologne, University of Cologne Coordinator of this site? Confirm its status | Cologne | Germany | Unknown status |
Related publications
- Safety and feasibility of oral zinc for patients with GNAO1-related disorders (ZINCGNAO1): an open-label, single-arm, single-centre, pilot trial in Germany.EClinicalMedicine · 2026 · PMID 42542632
This page is information, not medical advice. Discuss any trial with your doctor.