Clinical Trial: A Study of AG-348 in Adult Patients With Pyruvate Kinase Deficiency
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: A Phase 2, Open Label, Randomized, Dose Ranging, Safety, Efficacy, Pharmacokinetic and Pharmacodynamic Study of AG-348 in Adult Patients With Pyruvate Kinase Deficiency
Brief Summary: Study AG348-C-003 is a multicenter study designed to evaluate the safety and efficacy of different dose levels of AG-348 in patients with PK deficiency.
Detailed Summary: This is a Phase 2, open label, two arm, multicenter, randomized, dose-ranging study during which adult patients with PK deficiency will receive multiple doses of AG-348 for up to 24 weeks (Core Period); eligible patients may enter an Extension Period to receive AG-348 for up to 2 additional years. Up to 25 patients will be randomized to two doses of AG-348; a third arm (up to 25 patients) may be added, if required. AG-348 will be administered orally at various doses; if implemented, the dose of Arm 3 will be determined based on ongoing review. Data will be reviewed on a regular basis and study design, dose and schedule will be adapted based on these reviews. The study will evaluate the safety and tolerability of multiple doses of AG-348, pharmacokinetic (PK) and pharmacodynamic (PD) profile of AG-348 and early indicators of clinical efficacy.
Sponsor: Agios Pharmaceuticals, Inc.
Current Primary Outcome: Safety: incidence of adverse events [ Time Frame: 24 weeks, on average ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Pharmacokinetic parameters of AG-348 and its metabolite area-under-the-curve (AUC) 0-12hr [ Time Frame: 24 weeks ]
- Pharmacokinetic parameters of AG-348 and its metabolite (Tmax) [ Time Frame: 24 weeks ]
- Pharmacokinetic parameters of AG-348 and its metabolite (Cmax) [ Time Frame: 24 weeks ]
- Pharmacokinetic parameters of AG-348 and its metabolite (Cl/F) [ Time Frame: 24 weeks ]
- Change from baseline in whole blood concentration of adenosine triphosphate (ATP) [ Time Frame: 24 weeks ]
- Change from baseline in whole blood concentration of 2,3 - diphosphoglycerate (2,3-DPG) [ Time Frame: 24 weeks ]
- Change from baseline in hemoglobin. [ Time Frame: 24 weeks ]
Original Secondary Outcome: Same as current
Information By: Agios Pharmaceuticals, Inc.
Dates:
Date Received: June 10, 2015
Date Started: June 2015
Date Completion: May 2019
Last Updated: April 6, 2017
Last Verified: November 2016