Unique ID issued by UMIN | UMIN000033645 |
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Receipt number | R000038363 |
Scientific Title | 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 |
Date of disclosure of the study information | 2018/08/06 |
Last modified on | 2021/11/10 15:30:50 |
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
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
Japan | Asia(except Japan) | North America |
Europe |
Pyruvate Kinase Deficiency
Hematology and clinical oncology | Pediatrics |
Others
YES
To evaluate the efficacy of treatment with AG-348 compared with placebo in increasing hemoglobin (Hb) concentrations
Safety,Efficacy
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.
Interventional
Parallel
Randomized
Individual
Double blind -all involved are blinded
Placebo
2
Treatment
Medicine |
AG-348 5 mg, 20 mg or 50 mg BID
placebo
18 | years-old | <= |
Not applicable |
Male and Female
1.Have provided signed written informed consent prior to performing any study procedure, including screening procedures.
2.Be aged 18 years or older.
3.Have documented clinical laboratory confirmation of PK deficiency, defined as documented presence of at least 2 mutant alleles in the PKLR gene, of which at least 1 is a missense mutation, as determined per the genotyping performed by the study central genotyping laboratory.
4. Have an Hb concentration less than or equal to 10.0 g/dL regardless of gender (average of at least 2 Hb measurements (separated by a minimum of 7 days) during the Screening Period performed by the study central laboratory).
5. Be considered not regularly transfused, defined as having had no more than 4 transfusion episodes in the 12 month period up to the first day of study treatment and no transfusions in the 3 months prior to the first day of study treatment.
6. Have received at least 0.8 mg oral folic acid daily for at least 21 days prior to the first dose of study treatment, to be continued daily during study participation.
7. Have adequate organ function.
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.
76
1st name | Hirohide |
Middle name | |
Last name | Kawasaki |
Kansai Medical University Hospital
Pediatrics
573-1191
3-1 Shinmachi 2 Chome, Hirakata City, Osaka, Japan
072-804-0101
kouhouh@hirakata.kmu.ac.jp
1st name | Ryoko |
Middle name | |
Last name | Makino |
Labcorp Development Japan K.K.
Clinical Development Services
104-6108
Harumi Triton Square Office Tower Y 8F, 1-8-11 Harumi, Chuo-ku, Tokyo
03-6837-9500
Ryoko.Makino@labcorp.com
Labcorp Development Japan K.K.
Agios Pharmaceuticals, Inc.
Profit organization
Kansai Medical University Hospital IRB
2-3-1 Shinmachi, Hirakata, Osaka
072-804-2808
chiken@hirakata.kmu.ac.jp
YES
2017-003823-31
European Medicines Agency
2018 | Year | 08 | Month | 06 | Day |
Published
https://www.clinicaltrialsregister.eu/ctr-search/trial/2017-003823-31/results
6
Main results already published
2018 | Year | 02 | Month | 26 | Day |
2018 | Year | 07 | Month | 08 | Day |
2018 | Year | 09 | Month | 01 | Day |
2020 | Year | 12 | Month | 31 | Day |
2018 | Year | 08 | Month | 06 | Day |
2021 | Year | 11 | Month | 10 | Day |
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000038363
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