NCT06412653

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 13 (actual)
• Sponsor: Children's University Hospital Cologne, Germany · Academic / Other
• Sex: All
• Age: 6 Months – 30 Years
• Healthy volunteers: Not accepted

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.

Conditions

• GNAO1
• Dystonia
• Epilepsy
• Development Delay
• Developmental and Epileptic Encephalopathy 17
• Neurodevelopmental Disorder With Involuntary Movements
• Choreoathetosis

Interventions

Type	Name	Description
DRUG	Zinc Acetate Dihydrate	In this single arm trial, all participants will be receive the trial drug zinc acetate dihydrate orally. The Investigational medicinal product (IMP) will be given one hour after meal in a dosage which is recommended in Wilson Disease and has been given in this condition without observing severe adverse effects. If oral administration is not possible due to the disability level of the patient, the IMP can be mortared and suspended and can then be given as suspension orally or via the Percutaneous endoscopic gastrostomy. The total treatment duration in each patient is 6 months with stable dosage over the duration of the trial. If the therapy shows effects, the parents and participants may continue medication after the end of the trial. If not, they will stop the medication after the last visit at the trial site.

Timeline

• Start date: 2024-08-02
• Primary completion: 2025-08-04
• Completion: 2025-08-04
• First posted: 2024-05-14
• Last updated: 2026-02-03

Locations

1 site across 1 country: Germany