A study sponsored by Massachusetts General Hospital began earlier this year investigating the safety and tolerability of orally administered inosine. Inosine is a chemical which, when ingested, elevates levels of uric acid (urate) in the blood. Urate is an endogenous antioxidant, meaning it is produced in our bodies, rather than taken through diet. It may help in the defense against oxidative stress which is believed to play an important part in neuron degeneration. Low urate levels have been associated with the progression of Parkinson’s disease and is currently being investigated in other neurological diseases like ALS, Huntington’s disease, Alzheimer’s disease and multiple sclerosis. Inosine is currently used by some to improve athletic performance but clinical trials conducted examining this showed no improvement.

The study’s principal investigator, Sabrina Paganoni, MD, PhD, completed a short Phase 1 pilot study, which concluded last year, demonstrating initial safety and feasibility of urate elevation in people with ALS. This current Phase 2, multi-center, placebo controlled study aims to confirm these findings with more subjects over a longer exposure time. The study aims to enroll 30 participants who will take either inosine or the placebo for 20 weeks. Treatment will be titrated between 1-6 capsules of 500mg of inosine per day aiming for urate levels of 7-8mg/dL.

Uric acid has been shown to increase risk of kidney stones and gout, therefore, safety will be monitored to detect these adverse events. Since the risk is elevated, those with a history of these conditions will not be considered for enrollment. Those with a history of heart disease are also excluded.  Inclusion criteria states that participants must be between the ages of 18 and 85, have a SVC ≥ 60% of predicted for age, height, and gender at screening and serum urate levels below the median of 5.5mg/dL. This study is aiming to test the feasibility of using a smartphone app to monitor disease progression. Those who wish to be considered for enrollment must also agree to participate in this aspect of the trial. For the full list of criteria please visit our clinical trial page.

This study will enroll at three sites in the US; Holy Cross Hospital, FL, Massachusetts General Hospital, MA and University of Minnesota, MN. None of these centers are currently recruiting. Please sign up for our clinical trials mailing list to keep up-to-date on recruitment and enrollment criteria for all ALS clinical trials.

Helpful links:

Clinical Trial Design and Contact Information

Pilot Trial

ClinicalTrials.gov