UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000033645 |
|---|---|
| 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 |
* This page includes information on clinical trials registered in UMIN clinical trial registed system.
* We don't aim to advertise certain products or treatments
Basic information
Public 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
Acronym
A Phase 3 Study of AG-348 in Not Regularly Transfused Adult Subjects with Pyruvate Kinase Deficiency
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
Scientific Title:Acronym
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
| 1st name | Hirohide |
| Middle name | |
| Last name | Kawasaki |
Organization
Kansai Medical University Hospital
Division name
Pediatrics
Zip code
573-1191
Address
3-1 Shinmachi 2 Chome, Hirakata City, Osaka, Japan
TEL
072-804-0101
kouhouh@hirakata.kmu.ac.jp
Public contact
Name of contact person
| 1st name | Ryoko |
| Middle name | |
| Last name | Makino |
Organization
Labcorp Development Japan K.K.
Division name
Clinical Development Services
Zip code
104-6108
Address
Harumi Triton Square Office Tower Y 8F, 1-8-11 Harumi, Chuo-ku, Tokyo
TEL
03-6837-9500
Homepage URL
Ryoko.Makino@labcorp.com
Sponsor or person
Institute
Labcorp Development Japan K.K.
Institute
Department
Personal name
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)
IRB Contact (For public release)
Organization
Kansai Medical University Hospital IRB
Address
2-3-1 Shinmachi, Hirakata, Osaka
Tel
072-804-2808
chiken@hirakata.kmu.ac.jp
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 |
Related information
URL releasing protocol
Publication of results
Published
Result
URL related to results and publications
https://www.clinicaltrialsregister.eu/ctr-search/trial/2017-003823-31/results
Number of participants that the trial has enrolled
6
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description
Progress
Recruitment status
Main results already published
Date of protocol fixation
| 2018 | Year | 02 | Month | 26 | Day |
Date of IRB
| 2018 | Year | 07 | Month | 08 | Day |
Anticipated trial start date
| 2018 | Year | 09 | Month | 01 | Day |
Last follow-up date
| 2020 | Year | 12 | Month | 31 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
Management information
Registered date
| 2018 | Year | 08 | Month | 06 | Day |
Last modified on
| 2021 | Year | 11 | Month | 10 | Day |
Link to view the page
Value
https://center6.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 |
