Oral zinc therapy for patients with GNAO1 associated disorders

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial investigates the feasibility and safety of administering oral zinc therapy to patients with GNAO1 associated disorders. Participants will receive zinc acetate dihydrate for six months, during which their motor skills, behavior, well-being, dyskinesia, seizure frequency, quality of life, and microbiome changes will be assessed through visits and phone calls. The study aims to determine if this treatment is safe and effective for improving symptoms associated with GNAO1 disorders.

Why it matters

Who can join: the full rules

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

Where it's running

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

Study contacts

• Principal investigator: Moritz Thiel, MD — Children's Hospital, University Hospital Cologne, University of Cologne
• Study coordinator: Moritz Thiel, MD
• Email: moritz.thiel@uk-koeln.de
• Phone: +49 221-478-6850

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.