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Key PointsQuestionDoes treatment with l-citrulline and metformin combination therapy reduce motor function decline in ambulant patients with Duchenne muscular dystrophy?FindingsIn this randomized clinical trial of 47 ambulant male children aged 6.5 to 10 years with Duchenne muscular dystrophy, treatment with a combination of l-citrulline and metformin therapies provided a clinically relevant but not statistically significant reduction in motor function decline, as assessed by the transfer and standing posture dimension of the Motor Function Measure scale. No indications of harm were found in the intention-to-treat population.MeaningTreatment with a combination of l-citrulline and metformin therapies may slow muscle function decline in a specific subgroup of patients with Duchenne muscular dystrophy, but additional clinical trials with greater statistical power are warranted.