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

Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 6 months
Maximum Age: 30
Healthy Volunteers: f
View:

• 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

Locations
Other Locations
Germany
Children's Hospital, University Hospital Cologne, University of Cologne
RECRUITING
Cologne
Contact Information
Primary
Moritz Thiel, MD
moritz.thiel@uk-koeln.de
+49 221-478-6850
Backup
Kyriakos Matakis, MSc,PhD,MD
kyriakos.martakis@uk-koeln.de
+49 221-478-6853
Time Frame
Start Date: 2024-08-02
Estimated Completion Date: 2026-03
Participants
Target number of participants: 12
Treatments
Experimental: Interventional Arm
Zinc acetate dihydrate in age-adapted dosage ranging from 50mg to 150mg Zn2+ per day according to the recommended dosage in Wilsons Disease.
Sponsors
Leads: Children's University Hospital Cologne, Germany
Collaborators: The Clinical Trials Centre Cologne, University of Cologne, University of Geneva, Switzerland

This content was sourced from clinicaltrials.gov

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