Objective: This is the first study that will assess the potential impact of hypoxia-based therapy in individuals with PD on respiratory parameters and PD symptoms.

Background: Parkinson’s disease (PD) is a neurodegenerative disease, for which currently no disease-modifying therapies exist. Preclinical and clinical evidence suggests that hypoxia-based therapy might have short- and long-term effects in PD by the resultant adaptive bodily responses. Hypoxia therapy is believed to exert these effects through hypoxic preconditioning, which induces a hypoxia-inducible factor (HIF)-mediated cascade of local hypoxia-mitigating neuroprotective reactions. These might influence the dopaminergic and noradrenergic system on the short-term and the oxidative stress response and mitochondrial dysfunction on the long-term. Therefore, it has been raised as a promising novel basis for therapeutic options in PD.

Method: In 20 individuals with clinical PD, we will investigate the safety, tolerability, short-term symptomatic efficacy of continuous and intermittent hypoxia using individual, double-blind, randomized placebo-controlled N-of-1 trials. This design, often deployed in research concerning rare disease, 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 Unified Parkinson’s Disease Rating scale (MDS-UPDRS) part III, Timed Up & Go Test, MiniBES balance test and wrist accelerometry. Additionally, 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 a hypothesis-generating addition, cortisol, erythropoietin and platelet-derived growth factor will be measured to explore potentially involved mechanisms. Efficacy will be primarily assessed by Bayesian analysis.

Results: N/A

Conclusion: This application of intermittent and continuous hypoxia therapy could provide insights in novel pathways that may be pursued for PD treatment and explores whether hypoxia-based therapy might serve as personalized therapy in some individuals with PD. This trial may additionally serve as a proof of concept of deploying an N-of-1 design and inclusion of individualized outcomes in PD research.

References: ClinicalTrials.gov Identifier: NCT05214287

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MDS Abstracts - https://www.mdsabstracts.org/abstract/multiple-n-of-1-trials-to-investigate-hypoxia-therapy-in-parkinsons-disease-talisman-study-rationale-and-protocol/