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A Study to Evaluate the Efficacy, Safety and Pharmacokinetics of FYU-981 in Hyperuricemic Pediatric Patients with or without Gout (Phase II Study)

A Phase II Study of FYU-981 in Hyperuricemic Pediatric Patients with or without Gout

30

Hyperuricemic pediatric patients with or without gout (6 to 18 years old)

Informed consent: 35 [Body weight >= 40 kg] Number of subjects who received the study treatment: 20 Number of subjects who discontinued the study: 2 Number of subjects who completed the study: 18 [Body weight < 40 kg] Number of subjects who received the study treatment: 10 Number of subjects who discontinued the study: 0 Number of subjects who completed the study: 10

The incidence of adverse events was 86.7% (26/30 subjects) in the overall subjects, 90.0% (18/20 subjects) in subjects weighing >= 40 kg, and 80.0% (8/10 subjects) in subjects weighing < 40 kg. The incidence of adverse drug reactions was 10.0% (3/30 subjects) in the overall subjects, 10.0% (2/20 subjects) in subjects weighing >= 40 kg, and 10.0% (1/10 subjects) in subjects weighing < 40 kg. There were no deaths, serious adverse events, or adverse events leading to discontinuation of the investigational product. The incidence of adverse events leading to investigational product interruption in the overall subjects was 3.3% (1/30 subjects). This event was a rash and was considered not related to the investigational product in the opinion by investigator.

[Primary Outcome] In the overall subjects (29 subjects), the Percentage (95% confidence interval) of subjects with serum uric acid of <= 6.0 mg/dL at the end of administration was 82.8% (64.2% to 94.2%), and the lower limit of the 95% confidence interval exceeded the threshold population of 30% as predefined. [Secondary Outcome] The mean (standard deviation) serum uric acid was 9.36 (1.17) mg/dL at baseline and 5.15 (1.56) mg/dL at the end of administration, with a reduction rate (standard deviation) of 44.29 (18.28) % from baseline, and the change (standard deviation) -4.21 (1.92) mg/dL. [Pharmacokinetics (PK) assessment] Plasma unchanged drug concentration of FYU-981 will be separately evaluated using a population pharmacokinetic analysis that combines the data obtained from this clinical study with data from studies conducted in adults.

In this study, FYU-981 was administered orally to hyperuricemic pediatric patients with or without gout, at a dosage and administration bordered by 40 kg body weight for repeated 30 weeks. The efficacy of FYU-981 was verified, and its safety and pharmacokinetics were confirmed.

Sept. 11, 2025

No

https://jrct.mhlw.go.jp/latest-detail/jRCT2031220036

Masahiko Fushimi

FUJI YAKUHIN CO., LTD.

4-383, Sakuragi-cho, Omiya-ku, Saitama-shi, Saitama, Japan

clinical-trials@fujiyakuhin.co.jp

Development Department

FUJI YAKUHIN CO., LTD.

4-383, Sakuragi-cho, Omiya-ku, Saitama-shi, Saitama, Japan

+81-48-644-3403

clinical-trials@fujiyakuhin.co.jp

Complete

May. 01, 2022

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

-Hyperuricemic pediatric patients with or without gout
-Serum urate level:
>= 7.0 mg/dL in patients with history of gout, or
>= 8.0 mg/dL in patients complicated with kidney disease, hypertension, diabetes, metabolic syndrome or ischemic heart disease, or
>= 9.0 mg/dL in patients with not applicable the above

-Gouty arthritis within two weeks before start of study treatment
-Renal overload type of hyperuricemia
-HbA1c: >= 9.0%
-Clinically significant cardiac, and hepatic disease
-Kidney calculi or clinically significant urinary calculi
-eGFR: < 30 mL/min/1.73 m^2
-AST: >3xULN (upper limit of normal) or ALT: >3xULN
-Systolic blood pressure: >= 180 mmHg or diastolic blood pressure: >= 110 mmHg
etc.

Both

Hyperuricemic pediatric patients with or without gout

FYU-981 is administered orally once daily starting with the initial dose of 0.25 or 0.5 mg/day, followed by the maintenance doses of 0.5 to 4 mg/day.

Percentage of subjects with serum urate <=6.0 mg/dL at the end of administration

+81-263-58-4567

April. 14, 2022

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