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Name:
UMIN ID:

Recruitment status Preinitiation
Unique ID issued by UMIN UMIN000033645
Receipt No. R000038363
Official scientific title of the study 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 2018/08/06

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Basic information
Official scientific title of the study 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
Title of the study (Brief title) A Phase 3 Study of AG-348 in Not Regularly Transfused Adult Subjects with Pyruvate Kinase Deficiency
Region
Japan Asia(except Japan) North America
Europe

Condition
Condition Pyruvate Kinase Deficiency
Classification by specialty
Hematology and clinical oncology Pediatrics
Classification by malignancy Others
Genomic information YES

Objectives
Narrative objectives1 To evaluate the efficacy of treatment with AG-348 compared with placebo in increasing hemoglobin (Hb) concentrations
Basic objectives2 Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes 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.
Key secondary outcomes

Base
Study type Interventional

Study design
Basic design Parallel
Randomization Randomized
Randomization unit Individual
Blinding Double blind -all involved are blinded
Control Placebo
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms 2
Purpose of intervention Treatment
Type of intervention
Medicine
Interventions/Control_1 AG-348 5 mg, 20 mg or 50 mg BID
Interventions/Control_2 placebo
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit
18 years-old <=
Age-upper limit

Not applicable
Gender Male and Female
Key inclusion criteria 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.

Key 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.
Target sample size 76

Research contact person
Name of lead principal investigator Hirohide Kawasaki
Organization Kansai Medical University Hospital
Division name Pediatrics
Address 3-1 Shinmachi 2 Chome, Hirakata City, Osaka, Japan
TEL 072-804-0101
Email kouhouh@hirakata.kmu.ac.jp

Public contact
Name of contact person Yoshitaka Ando
Organization Integrated Development Associates Co., Ltd.
Division name Clinical Operations
Address 3-2-5, Kasumigaseki, Chiyoda-ku, Tokyo
TEL 03-6811-2323
Homepage URL
Email yoshitaka.ando@i-d-a.com

Sponsor
Institute Integrated Development Associates Co., Ltd.
Institute
Department

Funding Source
Organization Agios Pharmaceuticals, Inc.
Organization
Division
Category of Funding Organization Profit organization
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funder(s)

Secondary IDs
Secondary IDs YES
Study ID_1 2017-003823-31
Org. issuing International ID_1 European Medicines Agency
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions

Other administrative information
Date of disclosure of the study information
2018 Year 08 Month 06 Day

Progress
Recruitment status Preinitiation
Date of protocol fixation
2018 Year 02 Month 26 Day
Anticipated trial start date
2018 Year 09 Month 01 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Related information
URL releasing protocol
Publication of results Unpublished
URL releasing results
Results
Other related information

Management information
Registered date
2018 Year 08 Month 06 Day
Last modified on
2018 Year 08 Month 06 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000038363

Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name


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