Phase II Pilot Study of Aminoimidazole Carboxamide Riboside (AICAR), a Precursor of Purine Synthesis, for Lesch-Nyhan Disease

Summary

OBJECTIVES: I. Determine the effects of aminoimidazole carboxamide riboside (AICAR) on hematologic manifestations of Lesch-Nyhan disease. II. Assess the behavioral and neurological benefits of AICAR in patients with Lesch-Nyhan disease. III. Examine the effect of AICAR on purine production.

Timeline

Start

1996-02

Primary completion

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Completion

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Publications

• Background Page T, Barshop B, Yu AL, Nyhan WL. Treatment of Lesch-Nyhan syndrome with AICAR. Adv Exp Med Biol. 1994;370:353-6. doi: 10.1007/978-1-4615-2584-4_76. No abstract available.

Drugs

Indications