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

PHASE2RecruitingINTERVENTIONAL
Enrollment

12

Participants

Timeline

Start Date

August 2, 2024

Primary Completion Date

December 31, 2025

Study Completion Date

March 31, 2026

Conditions
GNAO1DystoniaEpilepsyDevelopment DelayDevelopmental and Epileptic Encephalopathy 17Neurodevelopmental Disorder With Involuntary MovementsChoreoathetosis
Interventions
DRUG

Zinc Acetate Dihydrate

In this single arm trial, all participants will be receive the trial drug zinc acetate dihydrate orally. The Investigational medicinal product (IMP) will be given one hour after meal in a dosage which is recommended in Wilson Disease and has been given in this condition without observing severe adverse effects. If oral administration is not possible due to the disability level of the patient, the IMP can be mortared and suspended and can then be given as suspension orally or via the Percutaneous endoscopic gastrostomy. The total treatment duration in each patient is 6 months with stable dosage over the duration of the trial. If the therapy shows effects, the parents and participants may continue medication after the end of the trial. If not, they will stop the medication after the last visit at the trial site.

Trial Locations (1)

50937

RECRUITING

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

All Listed Sponsors
collaborator

The Clinical Trials Centre Cologne

OTHER

collaborator

University of Cologne

OTHER

collaborator

University of Geneva, Switzerland

OTHER

lead

Children's University Hospital Cologne, Germany

OTHER