Phase 1
Completed N=32
A Pilot Study of Inosine in Amyotrophic Lateral Sclerosis (ALS)
Source: ClinicalTrials.gov NCT02288091 ↗Enrolled (actual)
32
Serious AEs
12.0%
Results posted
Oct 2017
Primary outcomePrimary: Number of Participants Experiencing Adverse Events — 22 Participants
Summary
This is a single center, open label, 12-week study of inosine treatment. Inosine treatment leads to an increase in the levels of urate (uric acid) in the blood.
The primary objective of the study is to determine the tolerability of oral administration of inosine.
Secondary study objectives include the measurement of biomarkers of oxidative stress and damage in response to inosine treatment.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Experiencing Adverse Events |
22 | — |
| PRIMARY Tolerability to Complete the Entire 12 Week Study on Study Drug. |
24 | — |
| SECONDARY Blood Biomarkers (GSH) at Baseline and Week 12 |
94.0; 84.5 | — |
| SECONDARY Neuroimaging Biomarkers at Baseline and Week 12 |
0.424; 0.392 | — |
| SECONDARY Blood Biomarkers (FRAP) at Baseline and Week 12 |
765.7; 1188.3 | — |
Eligibility Criteria
Inclusion Criteria
- Age 18 years or older.
- Sporadic or familial ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by revised El Escorial criteria (Appendix 1).
- Capable of providing informed consent and following trial procedures.
- Serum urate 160mmHg or DBP>100mmHg at Screening).
- Contraindications to undergo magnetic resonance spectroscopy (MRS) at Baseline and at Week 12 of the study such as history of claustrophobia, inability to lie flat for approximately one hour, or metal implants (metal pins or plates, extensive non-removable dental work, cerebral aneurysm clips, pacemaker).
- 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 PI judgment, or a history of active substance abuse within the prior year.
- Anything that, in the opinion of the 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.
Data sourced from ClinicalTrials.gov (NCT02288091). 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.