Intracerebroventricular anaerobic dopamine in Parkinson's disease with L-dopa-related complications: a phase 1/2 randomized-controlled trial.

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Tác giả: Simon Baigne, Christine Barthelemy, Natacha Carta, Louise Carton, Alexandre Demailly, Dominique Deplanque, Jean Christophe Devedjian, David Devos, Alain Duhamel, Kathy Dujardin, Matthieu Fisichella, Véronique Foutel, Bastien Gouges, Julien Labreuche, Damien Lannoy, Céline Leclercq, Felix Marchand, Caroline Moreau, Pascal Odou, Thomas Ollivier, Thavarak Ouk, Benjamin Palas, Camille Potey, Nicolas Reyns, Anne Sophie Rolland, Gustavo Touzet, Michèle Vasseur

Ngôn ngữ: eng

Ký hiệu phân loại:

Thông tin xuất bản: United States : Nature medicine , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 720845

Continuous compensation for cerebral dopamine deficiency represents an ideal treatment for Parkinson's disease. Dopamine does not cross the digestive and blood-brain barriers and is rapidly oxidized. The new concept is the intracerebroventricular administration of anaerobic dopamine (A-dopamine) using an abdominal pump connected to a subcutaneous catheter implanted in the third ventricle, near the striatum. An open-label phase 1 study showed no serious adverse reactions induced by A-dopamine in 12 patients. A randomized, controlled, open-label, crossover phase 2 study of 1 month of A-dopamine versus 1 month of optimized oral antiparkinsonian therapy was conducted in 9 patients. The primary endpoint, a blinded assessment of the percentage over target (that is, time with dyskinesia or bradykinesia), recorded by home actimetry using a wristwatch, was significantly reduced on A-dopamine compared with that on oral treatment alone (P = 0.027), with a median within-patient difference of -10.4 (Hedge g = -0.62 (95% confidence interval: -1.43, -0.08)). Home diaries were also significantly improved. These initial data on the feasibility, safety and effects of this new device-assisted therapy suggest validation by a large randomized double-blind trial. ClinicalTrials.gov registration: NCT04332276 .
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