Trial · NCT03692052
A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia
NCT03692052, Phase 2, Active not recruiting, Agios Pharmaceuticals, Inc.; results first posted 2021-11-15
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia. Trials, record NCT03692052. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03692052
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia
- NCT number
- NCT03692052
- Status
- Active not recruiting
- Phases
- Phase 2
- Enrollment
- 20 participants, actual
- Lead sponsor
- Agios Pharmaceuticals, Inc.
- Conditions
- Thalassemia
- Interventions
- AG-348
- First posted
- 2018-10-02
- Last updated
- 2026-03-30
- Other registries' IDs
- eudract:2018-002217-35
- Results first posted
- 2021-11-15
Source ClinicalTrials.gov
Registered outcomes21
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants Achieving a Hemoglobin Response (HR) | Up to 12 weeks | HR was defined as a ≥1.0 gram per deciliter (g/dL) increase in Hb concentration from Baseline at 1 or more assessments between Week 4 and Week 12 (inclusive). A participant's Baseline Hb concentration was defined as the average of all the participant's available Hb concentrations during the screening period up to the first dose of study drug. |
| Secondary | Average Change From Baseline in Hb Concentrations From Week 12 to Week 24 | Baseline, Week 12 to Week 24 | A participant's Baseline Hb concentration was defined as the average of all the participant's available Hb concentrations during the screening period up to the first dose of study drug. |
| Secondary | Percentage of Participants Achieving a Sustained Hb Response (sHR) | Week 12 to Week 24 | sHR was defined as achieving HR and achieving a ≥1.0 g/deciliter (dL) increase in Hb concentration at 2 or more evaluable Hb assessments out of the 4 scheduled assessments between the Week 12 visit and Week 24 visit. |
| Secondary | Percentage of Participants Achieving a Delayed Hb Response | Week 12 to Week 24 | Delayed Hb response was defined as not achieving HR and achieving a ≥1.0 g/dL increase in Hb concentration at 1 or more Hb assessments after Week 12. |
| Secondary | Change From Baseline in Hb Concentration Over the Duration of the Extension Period | Baseline up to approximately 10.5 years | |
| Secondary | Time to First ≥1.0 g/dL Increase in Hb Concentration | Up to Week 24 | |
| Secondary | Change From Baseline in Reticulocyte Count | Up to approximately 10.5 years | |
| Secondary | Change From Baseline in Bilirubin | Up to approximately 10.5 years | |
| Secondary | Change From Baseline in Lactate Dehydrogenase (LDH) | Up to approximately 10.5 years | |
| Secondary | Change From Baseline in Haptoglobin | Up to approximately 10.5 years |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 registered outcomesOutcomes21
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants Achieving a Hemoglobin Response (HR) | Up to 12 weeks | FAS included all participants who received at least 1 dose of study treatment. | percentage of participants | NUMBER |
| SECONDARY | Average Change From Baseline in Hb Concentrations From Week 12 to Week 24 | Baseline, Week 12 to Week 24 | FAS included all participants who received at least 1 dose of study treatment. Number analyzed is the number of participants with data available for analysis at the given time point. | grams per liter (g/L) | MEAN |
| SECONDARY | Percentage of Participants Achieving a Sustained Hb Response (sHR) | Week 12 to Week 24 | FAS included all participants who received at least 1 dose of study treatment. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants Achieving a Delayed Hb Response | Week 12 to Week 24 | FAS included all participants who received at least 1 dose of study treatment. | percentage of participants | NUMBER |
| SECONDARY | Change From Baseline in Hb Concentration Over the Duration of the Extension Period | Baseline up to approximately 10.5 years | |||
| SECONDARY | Time to First ≥1.0 g/dL Increase in Hb Concentration | Up to Week 24 | Full Analysis Set (FAS) included all participants who received at least 1 dose of study treatment. Data is reported for the responders. | weeks | MEAN |
| SECONDARY | Change From Baseline in Reticulocyte Count | Up to approximately 10.5 years | |||
| SECONDARY | Change From Baseline in Bilirubin | Up to approximately 10.5 years | |||
| SECONDARY | Change From Baseline in Lactate Dehydrogenase (LDH) | Up to approximately 10.5 years | |||
| SECONDARY | Change From Baseline in Haptoglobin | Up to approximately 10.5 years |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 outcomesResults by group36
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants Achieving a Hemoglobin Response (HR) | AG-348 | 80.0 | 90% Confidence Interval 59.90 to 92.86 | percentage of participants | |
| Average Change From Baseline in Hb Concentrations From Week 12 to Week 24 | AG-348 | Baseline | 79.44 | Standard Deviation 13.690 | grams per liter (g/L) |
| Average Change From Baseline in Hb Concentrations From Week 12 to Week 24 | AG-348 | Average Change from Baseline: Week 12 to 24 | 13.01 | Standard Deviation 6.283 | grams per liter (g/L) |
| Percentage of Participants Achieving a Sustained Hb Response (sHR) | AG-348 | 65.0 | 90% Confidence Interval 44.20 to 82.27 | percentage of participants | |
| Percentage of Participants Achieving a Delayed Hb Response | AG-348 | 10.0 | 90% Confidence Interval 1.81 to 28.26 | percentage of participants | |
| Time to First ≥1.0 g/dL Increase in Hb Concentration | AG-348 | 4.54 | Standard Deviation 3.204 | weeks | |
| Drug Concentrations Over Time for AG-348 | AG-348 | AG-348 50 mg: 0.00 Hour, Day 1 | NA | Geometric Coefficient of Variation NA | nanograms per milliliter (ng/mL) |
| Drug Concentrations Over Time for AG-348 | AG-348 | AG-348 50 mg: 0.50 Hour, Day 1 | NA | Geometric Coefficient of Variation NA | nanograms per milliliter (ng/mL) |
| Drug Concentrations Over Time for AG-348 | AG-348 | AG-348 50 mg: 1 Hour, Day 1 | 785.2 | Geometric Coefficient of Variation 64.6 | nanograms per milliliter (ng/mL) |
| Drug Concentrations Over Time for AG-348 | AG-348 | AG-348 50 mg: 2 Hour, Day 1 | 694.5 | Geometric Coefficient of Variation 26.6 | nanograms per milliliter (ng/mL) |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Core Period (Day 1 to Week 24) | STARTED | AG-348 | 20 | |
| Core Period (Day 1 to Week 24) | COMPLETED | AG-348 | 19 | |
| Core Period (Day 1 to Week 24) | NOT COMPLETED | AG-348 | 1 | |
| Extension Period (Week 25 to 10 Years) | STARTED | AG-348 | 19 | |
| Extension Period (Week 25 to 10 Years) | COMPLETED | AG-348 | 0 | |
| Extension Period (Week 25 to 10 Years) | NOT COMPLETED | AG-348 | 19 |
Source ClinicalTrials.gov
Adverse events81
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Initial insomnia | Psychiatric disorders | AG-348 | 10 | 20 | |
| other | Anxiety | Psychiatric disorders | AG-348 | 1 | 20 | |
| other | Middle insomnia | Psychiatric disorders | AG-348 | 1 | 20 | |
| other | Restlessness | Psychiatric disorders | AG-348 | 1 | 20 | |
| other | Terminal insomnia | Psychiatric disorders | AG-348 | 1 | 20 | |
| other | Dyspepsia | Gastrointestinal disorders | AG-348 | 4 | 20 | |
| other | Abdominal pain | Gastrointestinal disorders | AG-348 | 3 | 20 | |
| other | Diarrhoea | Gastrointestinal disorders | AG-348 | 3 | 20 | |
| other | Abdominal distension | Gastrointestinal disorders | AG-348 | 3 | 20 | |
| other | Nausea | Gastrointestinal disorders | AG-348 | 3 | 20 |
1 to 10 of 81Source ClinicalTrials.gov
See all 81 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | AG-348 | 0 | 20 |
| Total, other adverse events | AG-348 | 17 | 20 |
| Total, serious adverse events | AG-348 | 1 | 20 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 35964609 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03692052
GET https://conductscience.org/api/trials/v1/NCT03692052/registered?page=2