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Prospective pilot trial to address the feasibility and safety of treatment with oral Zinc in GNAO1 associated disorders (ZINCGNAO1)
EU CTIS ID: 2024-512735-72-00
What this study is testing
To investigate feasibility and safety of an oral therapy with zinc in patients affected by GNAO1.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- GNAO1 associated neurological disorder, documented by either (1)Proven pathogenic or likely pathogenic mutation in GNAO1 or (2)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, stereotypic movements, clonic), central muscular hypotonia, epilepsy, global developmental delay
- Age: 6month-30years
- Gross-Motor-Function measure-66 (GMFM-66) ≤ 75
- Written informed consent prior to any trial-related procedure by parents or legal guardian
- Stable on following concomitant treatments for at least 3 months prior to trial inclusion: anti-seizure medication (ASD); baclofen, Deep brain stimulation settings
You likely can't join if
- 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
See the full eligibility criteria
- GNAO1 associated neurological disorder, documented by either (1)Proven pathogenic or likely pathogenic mutation in GNAO1 or (2)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, stereotypic movements, clonic), central muscular hypotonia, epilepsy, global developmental delay
- Age: 6month-30years
- Gross-Motor-Function measure-66 (GMFM-66) ≤ 75
- Written informed consent prior to any trial-related procedure by parents or legal guardian
- Stable on following concomitant treatments for at least 3 months prior to trial inclusion: anti-seizure medication (ASD); baclofen, Deep brain stimulation settings
- 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 participants who are not willing to use/ not using a highly effective contraception method with a pearl-index < 1. Sexually active participants resp. their partner, unless surgically sterile, must be using a highly effective contraception method (including oral, transdermal, injectable or implanted contraceptives, 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
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.