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A Pilot Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Multiple Oral Doses of AG-348 in Subjects With Stable Sickle Cell Disease

Trial Profile

A Pilot Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Multiple Oral Doses of AG-348 in Subjects With Stable Sickle Cell Disease

Status: Completed
Phase of Trial: Phase 0

Latest Information Update: 13 Jul 2022

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At a glance

  • Drugs Mitapivat (Primary)
  • Indications Sickle cell anaemia
  • Focus Adverse reactions; Proof of concept

Most Recent Events

  • 16 May 2022 This study reports protocol amendment as the dose was escalated to 100 mg BID in 9 subjects
  • 16 May 2022 Results assessing and tolerability of multiple ascending doses of mitapivat in adults with SCD (HbSS) with no recent blood transfusions or changes in hydroxyurea (HU) or L-glutamine therapy published in the Blood
  • 04 Nov 2021 According to an Agios Pharmaceuticals media release, the company will host a virtual investor event on Dec. 14, 2021 to review the key clinical oral and poster presentations from the ASH meeting.

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