Phase 2
Completed N=23
Safety of Urate Elevation in Amyotrophic Lateral Sclerosis (ALS)
Source: ClinicalTrials.gov NCT03168711 ↗Enrolled (actual)
23
Serious AEs
21.7%
Results posted
Feb 2021
Primary outcomePrimary: Number of Participants With Adverse Events — 11; 7 participants
Summary
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
1. Safety, as measured by adverse events
2. 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).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Adverse Events |
11; 7 | — |
| PRIMARY Tolerability to Complete the Entire 20 Week Study on Study Drug |
12; 7 | — |
Eligibility Criteria
Inclusion Criteria
- Age 18-85.
- Sporadic or familial ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by revised El Escorial criteria (Appendix 1).
- Slow vital capacity (SVC) ≥ 60% of predicted for age, height, and gender at the Screening Visit.
- Capable of providing informed consent and following trial procedures.
- Serum urate 160mmHg or DBP>100mmHg at Screening).
- Women who are pregnant or lactating.
- The presence of unstable psychiatric disease, cognitive impairment, or dementia that would impair ability of the subject to provide informed consent, according to Site Investigator judgment, or a history of active substance abuse within the prior year.
- Anything that, in the opinion of the Site Investigator, would place the subject at increased risk or preclude the subject's full compliance with or completion of the study.
- Use of the following within 30 days prior to Screening: inosine, allopurinol, probenecid, more than 300mg vitamin C daily (note that a subject may take a standard multivitamin up to one tablet or capsule daily). Use of thiazides is permissible as long as the subject is on a stable dose from 1 week prior to Screening.
- Known hypersensitivity or intolerability to inosine.
- Renal insufficiency as defined by eGFR < 60 mL/min/1.73m2 at the time of screening.
Data sourced from ClinicalTrials.gov (NCT03168711). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.