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Multiple N-of-1 trials to investigate hypoxia therapy in Parkinson’s disease: study rationale and protocol

Janssen Daalen, JM, Meinders, MJ, Giardina, F, Roes, KCB, Stunnenberg, BC, Mathur, S, Ainslie, PN, Thijssen, DHJ and Bloem, BR (2022) Multiple N-of-1 trials to investigate hypoxia therapy in Parkinson’s disease: study rationale and protocol. BMC Neurology, 22 (1). ISSN 1471-2377

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Open Access URL: https://doi.org/10.1186/s12883-022-02770-7 (Published version)


Background: Parkinson’s disease (PD) is a neurodegenerative disease, for which no disease-modifying therapies exist. Preclinical and clinical evidence suggest that hypoxia-based therapy might have short- and long-term benefits in PD. We present the contours of the first study to assess the safety, feasibility and physiological and symptomatic impact of hypoxia-based therapy in individuals with PD. Methods/Design: In 20 individuals with PD, we will investigate the safety, tolerability and short-term symptomatic efficacy of continuous and intermittent hypoxia using individual, double-blind, randomized placebo-controlled N-of-1 trials. This design allows for dose finding and for including more individualized outcomes, as each individual serves as its own control. A wide range of exploratory outcomes is deployed, including the Movement Disorders Society Unified Parkinson’s Disease Rating scale (MDS-UPDRS) part III, Timed Up & Go Test, Mini Balance Evaluation Systems (MiniBES) test and wrist accelerometry. Also, self-reported impression of overall symptoms, motor and non-motor symptoms and urge to take dopaminergic medication will be assessed on a 10-point Likert scale. As part of a hypothesis-generating part of the study, we also deploy several exploratory outcomes to probe possible underlying mechanisms of action, including cortisol, erythropoietin and platelet-derived growth factor β. Efficacy will be assessed primarily by a Bayesian analysis. Discussion: This evaluation of hypoxia therapy could provide insight in novel pathways that may be pursued for PD treatment. This trial also serves as a proof of concept for deploying an N-of-1 design and for including individualized outcomes in PD research, as a basis for personalized treatment approaches. Trial registration: ClinicalTrials.gov Identifier: NCT05214287 (registered January 28, 2022).

Item Type: Article
Uncontrolled Keywords: Clinical trial; Disease-modifying; Hypoxia; Mitochondrial dysfunction; Parkinson’s disease; Treatment; Neurology & Neurosurgery; 1109 Neurosciences; 1702 Cognitive Sciences
Subjects: R Medicine > R Medicine (General)
R Medicine > RM Therapeutics. Pharmacology
Divisions: Sport & Exercise Sciences
Publisher: BioMed Central
SWORD Depositor: A Symplectic
Date Deposited: 29 Jul 2022 14:21
Last Modified: 29 Jul 2022 14:30
DOI or ID number: 10.1186/s12883-022-02770-7
URI: https://researchonline.ljmu.ac.uk/id/eprint/17308
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