Not recruitingRare disease

Study on the Safety and Feasibility of Oral Zinc Acetate Dihydrate for Patients with GNAO1-Related Disorders

Verified siteRegistered drugNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a group of rare conditions known as GNAO1 associated disorders. These disorders are linked to changes in the GNAO1 gene and can lead to symptoms such as movement problems, muscle weakness, seizures, and developmental delays. The trial will explore the use of a treatment called Wilzin, which contains the active ingredient zinc acetate dihydrate. Wilzin is available in two forms: 25 mg and 50 mg hard capsules. The purpose of the study is to assess how feasible and safe it is to use oral zinc as a treatment for individuals with GNAO1 associated disorders.

Participants in the study will take the zinc capsules by mouth, and the treatment period will last for several months. Throughout the study, researchers will monitor how often the zinc is taken correctly and will check for any side effects. The study will also look at changes in motor skills, quality of life, and other symptoms related to GNAO1 disorders. Caregivers will help by keeping a daily log of the participant's behavior, sleep patterns, and any seizures. Blood tests will be conducted to measure zinc levels and to ensure there are no deficiencies in other important substances like ferritin and copper.

The study will also involve analyzing stool samples to see if there are any changes in the gut related to the GNAO1 disorders. Researchers will compare the results to see if different genetic variants of GNAO1 affect the outcomes. The trial aims to provide valuable information on whether zinc can be a safe and effective treatment option for those affected by these rare genetic disorders.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the trial
Participation begins after meeting specific criteria, including having a GNAO1 associated disorder and being between 6 months and 30 years old.
Consent must be provided by a parent or legal guardian before any trial-related procedures.
Step 2
Initial assessment
An initial assessment is conducted to evaluate motor skills, quality of life, and other health parameters.
Measurements include the Gross-Motor-Function measure-66 (GMFM-66) and the Burke-Fahn-Marsden Dystonia Rating scale (BFMDRS).
Step 3
Medication administration
The treatment involves taking Wilzin capsules, which contain zinc acetate dihydrate.
Capsules are available in 50 mg and 25 mg doses and are taken orally or through a feeding tube.
Step 4
Ongoing monitoring
Regular evaluations are conducted to monitor safety and effectiveness.
Parents are required to maintain a daily logbook to record general behavior, sleep patterns, and seizure activity.
Step 5
Follow-up visits
Follow-up visits occur at specified intervals to assess motor skills, quality of life, and serum levels of zinc, ferritin, and copper.
The microbiome in the stool is analyzed to detect any changes related to the treatment.
Step 6
Completion of trial
The trial is expected to conclude by December 31, 2025.
Final assessments are conducted to evaluate the overall impact of the treatment.

Who can join the trial?

12 criteria

The patient must have a **GNAO1 associated neurological disorder**. This means they have a specific genetic issue related to GNAO1, which can be confirmed by:
- A proven or likely harmful change in the GNAO1 gene.
- A change in the GNAO1 gene that is not clearly understood, but the patient shows symptoms that match GNAO1 issues, as decided by the study doctors.
- The patient must have at least one common symptom of GNAO1, such as:
  - **Movement disorder**: Problems with controlling movements, which can include dystonia (muscle contractions), chorea (jerky movements), ataxia (lack of coordination), stereotypic movements (repetitive movements), or clonic movements (muscle spasms).
  - **Central muscular hypotonia**: Low muscle tone.
  - **Epilepsy**: Seizure disorder.
  - **Global developmental delay**: Slower development in multiple areas.
  - The patient must be between **6 months and 30 years** old.
  - The patient must have a **Gross-Motor-Function measure-66 (GMFM-66) score of 75 or less**. This is a test that measures how well a person can perform certain physical activities.
  - The patient's parents or legal guardian must provide **written informed consent** before any trial-related activities begin. This means they agree to let the patient participate in the study after understanding what it involves.
  - The patient must have been stable on certain treatments for at least **3 months** before joining the trial. These treatments include:
    
    **Anti-seizure medication (ASD)**: Medicines used to control seizures.
    **Baclofen**: A medication used to treat muscle spasms.
    **Deep brain stimulation settings**: A treatment involving electrical impulses to the brain.

Who cannot join the trial?

5 criteria

Patients who do not have **GNAO1 associated disorders** cannot participate. **GNAO1 associated disorders** are a group of conditions caused by changes in the GNAO1 gene, which can affect the brain and movement.
Patients who are not within the specified age range cannot participate. The age range includes children and adolescents.
Patients who are not part of the specified clinical trial group cannot participate. This means the study is looking for specific characteristics in participants.
Both **male** and **female** patients are eligible, so exclusion is not based on gender.
Patients who are not considered part of a **vulnerable population** cannot participate. A vulnerable population includes groups that may need special protection or care.

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Verified sites

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University Hospital Cologne
Verified
Cologne, Germany

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Investigated drugs

Zinc is being studied in this trial to see if it is a safe and practical treatment for patients with GNAO1 associated disorders. Zinc is a mineral that is important for many bodily functions, including the immune system and cell growth. In this trial, zinc is given orally to see if it can help manage symptoms or improve the condition of patients with GNAO1 disorders.

Zinc Acetate Dihydrate

What is already known about the treatment

Zinc – Zinc is administered orally and is currently being studied in clinical trials for its feasibility and safety in treating GNAO1 associated disorders. It is not yet widely recognized in medical literature for this specific use, as research is ongoing. The main therapeutic indication being explored is its potential benefit in managing symptoms related to GNAO1 disorders. Zinc is believed to work at the molecular level by supporting enzyme function and cellular metabolism, which may help in stabilizing neurological functions. It is classified as a dietary supplement and essential mineral.

Investigated diseases

GNAO1 Associated Disorders – These are a group of rare genetic conditions caused by mutations in the GNAO1 gene. The disorders can lead to a variety of symptoms, including movement abnormalities such as dystonia and dyskinesia, which are involuntary muscle contractions and movements. Individuals may also experience developmental delays and seizures. The severity and specific symptoms can vary widely among affected individuals. These disorders are typically identified in early childhood and can impact both motor and cognitive functions. The progression of symptoms can be unpredictable, with some individuals experiencing worsening of symptoms over time.