A Dose-Finding Study of AG-348 in Sickle Cell Disease
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ClinicalTrials.gov Identifier: NCT04000165 |
Recruitment Status :
Completed
First Posted : June 27, 2019
Results First Posted : July 12, 2022
Last Update Posted : July 12, 2022
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Sponsor:
National Institutes of Health Clinical Center (CC)
Collaborators:
National Heart, Lung, and Blood Institute (NHLBI)
Agios Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Swee Lay Thein, M.D., National Institutes of Health Clinical Center (CC)
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Study Type | Interventional |
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Study Design | Allocation: N/A; Intervention Model: Sequential Assignment; Masking: None (Open Label); Primary Purpose: Basic Science |
Condition |
Sickle Cell Disease |
Intervention |
Drug: AG-348 |
Enrollment | 17 |
Participant Flow
Recruitment Details | Participants were enrolled at the National Institutes of Health in Bethesda, Maryland from July 2019 to June 2021. |
Pre-assignment Details |
Arm/Group Title | AG-348 in Participants With Sickle Cell Disease |
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Intra-patient dose escalating study, starting with 5 mg twice a day, increasing to 20 mg twice a day, to maximum 50 mg or 100 mg twice a day. Dosing period is every 2 weeks at each dose level. Dose taper will start on Day 42 (50 mg) or Day 56 (100 mg) with dose reduced over 12 to 15 days. |
Period Title: Overall Study | |
Started | 17 |
Completed Study at 50 mg Dose | 7 |
Escalated to 100 mg | 10 |
Completed | 15 |
Not Completed | 2 |
Reason Not Completed | |
Physician Decision | 1 |
Withdrawal by Subject | 1 |
Baseline Characteristics
Arm/Group Title | AG-348 in Participants With Sickle Cell Disease | |
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Intra-patient dose escalating study, starting with 5 mg twice a day, increasing to 20 mg twice a day, to maximum 50 mg or 100 mg twice a day. Dosing period is every 2 weeks at each dose level. Dose taper will start on Day 42 (50 mg) or Day 56 (100 mg) with dose reduced over 12 to 15 days. | |
Overall Number of Baseline Participants | 17 | |
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[Not Specified]
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Age, Categorical
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 17 participants | |
<=18 years |
0 0.0%
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Between 18 and 65 years |
17 100.0%
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>=65 years |
0 0.0%
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Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 17 participants | |
Female |
6 35.3%
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Male |
11 64.7%
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Ethnicity (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 17 participants | |
Hispanic or Latino |
0 0.0%
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Not Hispanic or Latino |
17 100.0%
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Unknown or Not Reported |
0 0.0%
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Race (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 17 participants | |
American Indian or Alaska Native |
0 0.0%
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Asian |
0 0.0%
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Native Hawaiian or Other Pacific Islander |
0 0.0%
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Black or African American |
17 100.0%
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White |
0 0.0%
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More than one race |
0 0.0%
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Unknown or Not Reported |
0 0.0%
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Region of Enrollment
Measure Type: Number Unit of measure: Participants |
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United States | Number Analyzed | 17 participants |
17 |
Outcome Measures
Adverse Events
Limitations and Caveats
[Not Specified]
More Information
Results Point of Contact
Name/Title: | Dr. Swee Lay Thein, Chief of Sickle Cell Branch |
Organization: | The National Institutes of Health / The National Heart, Lung, and Blood Institute |
Phone: | 301.402.6699 |
EMail: | sweelay.thein@nih.gov |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | Swee Lay Thein, M.D., National Institutes of Health Clinical Center (CC) |
ClinicalTrials.gov Identifier: | NCT04000165 |
Other Study ID Numbers: |
190097 19-H-0097 |
First Submitted: | June 22, 2019 |
First Posted: | June 27, 2019 |
Results First Submitted: | May 6, 2022 |
Results First Posted: | July 12, 2022 |
Last Update Posted: | July 12, 2022 |