A Randomized, Placebo-controlled Clinical Trial Evaluating the Efficacy and Safety of Glycine and Magnesium+Thiamine Supplements, Alone or Combined, Administered for 6 Months to Patients With Primary Ciliary Dyskinesia

What this Trial Is About

This research aims to evaluate whether oral supplements of glycine and magnesium with thiamine can improve symptoms and lung function in patients with primary ciliary dyskinesia (PCD), a condition where mucus clearance is impaired and can lead to severe lung damage. Currently, treatment options for PCD are limited to supportive care, and this study investigates if these supplements, alone or combined, can reduce respiratory exacerbations and inflammation while improving quality of life. Participants will be randomly assigned to one of four groups to receive for 6 months either glycine, magnesium plus thiamine, a combination of glycine and magnesium plus thiamine, or a placebo. Glycine is given at 0.5 g/kg/day up to 25 g daily, magnesium (as magnesium citrate) up to 400 mg/day, and thiamine (as benfotiamine) up to 10 mg/day, all divided into three daily doses. Treatments are provided as a whitish powder in identical bottles, and participants continue their usual care alongside the study supplements. Throughout the 6-month study, participants will be monitored regularly every two months with various assessments including pulmonary function tests, oxygen saturation, saliva tests for inflammatory markers, and quality of life questionnaires. Weekly online symptom questionnaires will track respiratory exacerbations. The main outcome is the number of respiratory exacerbations during the study, with additional measures including lung function, physical growth, walking ability, and inflammatory compounds in saliva. The total participation time is 6 months with careful safety and adherence monitoring.

Glycine and Magnesium+Thiamine for the Treatment of Primary Ciliary Dyskinesia