Combined febuxostat and inosine treatment for people with early Parkinson's

A Phase Ib Trial of Combined Febuxostat and Inosine Therapy in Patients With Parkinson's Disease

We will test whether taking febuxostat plus inosine twice daily for 12 weeks is safe for adults with early-stage Parkinson's disease.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 24 (estimated)
• Ages: 18 Years to 80 Years
• Sex: All
• Sponsor: Fujita Health University (Academic / other)
• Locations: 1 site (Toyoake, Aichi-ken)
• Trial ID: NCT07170475 on ClinicalTrials.gov

What this trial studies

This Phase Ib, open-label trial enrolls 24 people with early Parkinson's disease to receive one of four oral dose regimens of febuxostat plus inosine taken twice daily for 12 weeks. Participants attend clinic visits at baseline and at weeks 4, 8, and 12 for blood tests (including hypoxanthine), neurological exams, and questionnaires, and keep a brief diary of side effects and daily changes. The main goal is to collect safety and tolerability data and to measure biochemical effects on purine metabolites. Results will inform whether the combination is safe enough to test in larger efficacy trials.

Who should consider this trial

Good fit: Adults aged 18–80 with early-stage Parkinson's disease who have been on a stable Parkinson's medication regimen for at least three months, have Hoehn-Yahr stage 1–3, MDS-UPDRS Part III score 10–35 (ON state), and MMSE ≥ 24 are ideal candidates.

Not a fit: People with advanced disability who cannot walk or stand unaided, significant cognitive impairment, or current/ recent use of excluded drugs or inosine supplements are unlikely to benefit from this small safety trial.

Why it matters

Potential benefit: If the combination is safe, it could enable larger trials aimed at slowing Parkinson's progression by modifying purine metabolism.

How similar studies have performed: Prior trials that raised urate with inosine did not demonstrate clear slowing of Parkinson's progression, so the combined febuxostat-plus-inosine approach is relatively novel and largely untested in PD.

Eligibility criteria

Inclusion Criteria:

1. Able to provide voluntary written informed consent.
2. Receiving stable Parkinson's disease medication (no changes in type or dose) for at least 3 months before enrollment.
3. Age 18 to 80 years at the time of consent.
4. Diagnosed with Parkinson's disease by a specialist according to MDS-PD diagnostic criteria, and at pre-enrollment screening, all of the following are met:

   1. Hoehn-Yahr stage (ON state) 1 to 3
   2. MDS-UPDRS Part III (ON state) score 10 to 35
   3. Mini-Mental State Examination (MMSE) score ≥ 24

Exclusion Criteria:

1. Requires almost total assistance in daily life and is unable to walk or stand unaided.
2. Currently taking azathioprine, mercaptopurine hydrate, vidarabine, didanosine, or rosuvastatin.
3. Used febuxostat, allopurinol, or topiroxostat within 3 months before study start.
4. Taking any supplement containing inosine.
5. Started any new Parkinson's disease medication or therapy within 3 months before enrollment.
6. Serum creatinine \>1.5× upper limit of normal (ULN), or AST (GOT) or ALT (GPT) \>2× ULN at screening.
7. History of surgical treatment for Parkinson's disease.
8. History of or comorbid hypersensitivity/allergy to any ingredient of the investigational drugs.
9. Participation in another clinical trial involving an unapproved drug within 30 days before consent, or currently enrolled in another interventional study.
10. Pregnant or breastfeeding, or unwilling/unable to use reliable contraception during the study period.
11. Positive test at screening for HIV, HBV, HTLV-1, or syphilis; \*\*HCV antibody-positive with undetectable HCV RNA\*\* is allowed.
12. Unable to take the investigational drugs orally without changing the dosage form.
13. Gastrointestinal disease or prior GI surgery that may affect drug absorption, as judged by the investigator.
14. Psychiatric disorder or symptoms that interfere with daily life and make study participation difficult.
15. Unable to complete assessments or questionnaires independently.
16. Any other condition that, in the investigator's judgment, would make participation unsafe or inappropriate.

Where this trial is running

Toyoake, Aichi-ken

• Fujita Health University — Toyoake, Aichi-ken, Japan (Recruiting)

Conditions Parkinson's Disease; Parkinson Disease; Febuxostat; Inosine; Hypoxanthine; Combination Therapy; Safety Study