Safety of Urate Elevation in Amyotrophic Lateral Sclerosis (ALS) (SURE-ALS2)
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|ClinicalTrials.gov Identifier: NCT03168711|
Recruitment Status : Recruiting
First Posted : May 30, 2017
Last Update Posted : February 1, 2018
This is a multi-center, 20-week study of inosine treatment.
Study Objectives and Endpoints The primary objective of the study is to determine the safety and tolerability of oral administration of inosine (administered daily) dosed to moderately elevate serum urate over 20 weeks.
The primary outcome measures will be
- Safety, as measured by adverse events
- Tolerability, defined as the ability of subjects to complete the entire 20-week study.
As an exploratory objective, we will test the feasibility and utility of a smartphone application for monitoring symptoms and disease progression in patients with amyotrophic lateral sclerosis (ALS).
|Condition or disease||Intervention/treatment||Phase|
|Amyotrophic Lateral Sclerosis||Drug: Inosine Drug: Placebo||Phase 2|
Amyotrophic lateral sclerosis (ALS) is a fatal, neurodegenerative disease for which there is no cure. Multiple lines of evidence have implicated oxidative stress in the pathophysiology of ALS. Urate (uric acid) is an endogenous antioxidant system, and urate may serve as a major defense against oxidative stress. Urate has emerged as a promising neuro-protectant and therapeutic target based on convergent epidemiological, laboratory, and clinical data in multiple neurodegenerative diseases, most notably Parkinson's disease (PD). In PD, urate elevation has been pursued as a potential therapy by administration of inosine, a urate precursor that is available as an over-the-counter supplement. Administration of inosine results in a predictable elevation of urate levels and has been shown to be safe and well tolerated in PD.
Analysis of ALS databases revealed that higher urate levels are an independent predictor of slower progression and prolonged survival in ALS. However, whether elevating urate in people with ALS would result in better outcomes is unknown.
The Principal Investigator has recently concluded a Pilot Study of Inosine in ALS, which was a short, open label, single center study involving 25 subjects [NCT02288091]. The study showed safety and feasibility of urate elevation in patients with ALS. The Principal Investigator is now pursuing a multi-center Phase II trial to confirm these findings with longer exposure time.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||30 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||Safety of Urate Elevation in Amyotrophic Lateral Sclerosis (ALS)|
|Actual Study Start Date :||October 1, 2017|
|Estimated Primary Completion Date :||October 2019|
|Estimated Study Completion Date :||January 2020|
U.S. FDA Resources
Subjects will be administered oral inosine daily. The dose of inosine will be titrated to obtain serum urate levels of 7 - 8 mg/dL.
Subjects on inosine will receive 1-6 capsules a day of 500 mg inosine titrated to target urate levels of 7 - 8 mg/dL.
Placebo Comparator: Placebo
Subjects will be administered oral placebo daily. The dose of placebo will be titrated to obtain serum urate levels of 7 - 8 mg/dL.
Subjects on placebo will receive 1-6 capsules a day of 500 mg placebo (sugar pill) titrated to target urate levels of 7 - 8 mg/dL.
- Safety Will be Assessed by the Occurrence of Adverse Events [ Time Frame: Baseline to Week 24 ]Safety will be assessed by the occurrence of adverse events such as kidney stones and gout (expected adverse events) in all participants receiving at least 1 dose of study drug
- Tolerability to Complete the Entire 20 Week Study on Study Drug [ Time Frame: Baseline to Week 20 ]Tolerance of study drug will be defined as the number of participants who able to complete the 20-week study without permanently discontinuing study drug or suspending study drug for greater than 28 days
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03168711
|United States, Florida|
|Holy Cross Hospital||Recruiting|
|Fort Lauderdale, Florida, United States, 33308|
|Contact: Franco Salas 954-229-7965 firstname.lastname@example.org|
|Principal Investigator: Eduardo Locatelli, MD, MPH|
|United States, Massachusetts|
|Massachusetts General Hospital||Recruiting|
|Boston, Massachusetts, United States, 02114|
|Contact: Jianing (Ning) Liu 617-726-1880 email@example.com|
|Principal Investigator: Katharine Nicholson, MD|
|United States, Minnesota|
|University of Minnesota||Recruiting|
|Minneapolis, Minnesota, United States, 55455|
|Contact: Valerie Ferment 612-301-1535 firstname.lastname@example.org|
|Principal Investigator: David Walk, MD|
|Principal Investigator:||Sabrina Paganoni, MD, PhD||Massachusetts General Hospital|