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

Last updated: August 28, 2024
Sponsor: Children's University Hospital Cologne, Germany
Overall Status: Active - Recruiting

Phase

2

Condition

Dystonia

Epilepsy

Tic Disorders

Treatment

Zinc Acetate Dihydrate

Clinical Study ID

NCT06412653
Uni-Koeln-5275
  • Ages 6-30
  • All Genders

Study 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 Criteria

Inclusion

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 beconsistent 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, globaldevelopmental delay

  • Age: 6 month - 30 years

  • GMFM ≤ 75

  • written informed consent prior to any trial-related procedure (according to age andstatus 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 trialinclusion: anti-seizure drugs (ASD); baclofen, Deep brain stimulation settings

Exclusion

Exclusion Criteria:

  • Treatment of Zinc in the last 4 months before inclusion

  • known other genetic variants that are known to cause symptoms like observed inGNAO1-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 withcompeting interventions

  • sexually active patients who are not willing to use/ not using a highly effectivecontraception method with a pearl-index < 1. Sexually active patients, unlesssurgically sterile, must be using a highly effective contraception method (includingoral, transdermal, injectable or implanted contraceptives, intrauterine device (IUD), using a condom of the sexual partner or sterile sexual partner) and mustagree to continue using such precautions during the whole study period.

  • Pregnant women and nursing mothers

Study Design

Total Participants: 12
Treatment Group(s): 1
Primary Treatment: Zinc Acetate Dihydrate
Phase: 2
Study Start date:
August 02, 2024
Estimated Completion Date:
March 31, 2026

Connect with a study center

  • Children's Hospital, University Hospital Cologne, University of Cologne

    Cologne, 50937
    Germany

    Active - Recruiting

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