AG348-C-003 Phase II Study in Patients with Pyruvate Kinase Deficiency
Research type
Research Study
Full 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
IRAS ID
179396
Contact name
Mark Layton
Contact email
Sponsor organisation
Agios Pharmaceuticals
Eudract number
2015-000484-13
Duration of Study in the UK
1 years, 8 months, 31 days
Research summary
PK deficiency is a rare blood disease. It is also a genetic disease which means that it may be transmitted from a parent to a child. PK deficiency affects both children and adults and its severity ranges from mild to serious or even life threatening.
The purpose of this study is to evaluate the safety of different dose levels of AG-348 ( the study drug) when given for 24 weeks (6 months). The study will evaluate the amount of study drug that gets into the blood and how long it stays in the blood (pharmacokinetics or PK); what the study drug does in the body (pharmacodynamics or PD); and look for signs as to whether the study drug is working (activity or efficacy).
Initially, patients will be assigned to one of two twice-daily doses of AG-348. Patients will receive either 300mg or 50mg of study drug orally every twelve hours. The patients will be selected to receive either dose depending on their PKR mutation so that there is a balance of specific mutations across both dose levels. The study is open – label so therefore both patients and study doctors will know what treatment patients are assigned to receive. Because PK Deficiency is a rare disease with a limited eligible patient population and because the physical characteristics of affected patients are very different due to the wide variety of mutations in PKR that cause the disease, it is important to focus closely on dose findings in this first-in-patient study. Therefore, in addition to initiating this study with 2 different doses of study drug, a Data Review Team will be established to review study data on a regular basis and adapt the study design, dose and schedule of study drug.
REC name
London - Westminster Research Ethics Committee
REC reference
15/LO/0863
Date of REC Opinion
2 Jun 2015
REC opinion
Favourable Opinion