A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Subjects with Pyruvate Kinase Deficiency

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Subjects with Pyruvate Kinase Deficiency - A Phase 3 Study of AG-348 in Not Regularly Transfused Adult Subjects with Pyruvate Kinase Deficiency

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

JPRN

Registry ID

JPRN-UMIN000033645

Enrollment

Registered

2018-08-06

Start date

2018-09-01

Completion date

Unknown

Last updated

2025-07-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Pyruvate Kinase Deficiency

Interventions

AG-348 5 mg, 20 mg or 50 mg BID placebo

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria:

Exclusion criteria

Exclusion criteria: 1.Are homozygous for the R479H mutation or have 2 non-missense mutations, without the presence of another missense mutation, in the PKLR gene as determined per the genotyping performed by the study central genotyping laboratory. 2.Have a significant medical condition that confers an unacceptable risk to participating in the study, and/or that could confound the interpretation of the study data. Such significant medical conditions.

Design outcomes

Primary

Measure	Time frame
The primary endpoint is the hemoglobin responce (HR), defined as a 1.5 g/dL or more increase in Hb concentration from baseline thar is sustained at 2 or more scheduled assessments at Weeks 16, 20, and 24 during the FIxed Dose Period. The individual subject's baseline Hb concentration is defined as the averege of all available Hb concentrations from the central laboratoly for that subject during the Screening Period up to the first dose of treatment.	—

Measure

Time frame

The primary endpoint is the hemoglobin responce (HR), defined as a 1.5 g/dL or more increase in Hb concentration from baseline thar is sustained at 2 or more scheduled assessments at Weeks 16, 20, and 24 during the FIxed Dose Period. The individual subject's baseline Hb concentration is defined as the averege of all available Hb concentrations from the central laboratoly for that subject during the Screening Period up to the first dose of treatment.

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Countries

Japan,Asia(except Japan),North America,Europe

Contacts

Public ContactRyoko Makino

Labcorp Development Japan K.K. Clinical Development Services

Ryoko.Makino@labcorp.com03-6837-9500

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Feb 4, 2026