Trial · NCT03263091
Efficacy and Safety of Roxadustat for Treatment of Anemia in Participants With Lower Risk Myelodysplastic Syndrome With Low Red Blood Cell Transfusion Burden
NCT03263091, Phase 3, Terminated, Kyntra Bio; results first posted 2024-08-01
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. Efficacy and Safety of Roxadustat for Treatment of Anemia in Participants With Lower Risk Myelodysplastic Syndrome With Low Red Blood Cell Transfusion Burden. Trials, record NCT03263091. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03263091
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy and Safety of Roxadustat for Treatment of Anemia in Participants With Lower Risk Myelodysplastic Syndrome With Low Red Blood Cell Transfusion Burden
- NCT number
- NCT03263091
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 184 participants, actual
- Lead sponsor
- Kyntra Bio
- Conditions
- Primary MDS (Very Low, Low or Intermediate IPSS-R With <5% Blasts), Anemia
- Interventions
- Roxadustat, Placebo
- First posted
- 2017-08-28
- Last updated
- 2024-08-01
- Results first posted
- 2024-08-01
- Limitations and caveats
- Study did not meet its primary efficacy endpoint; hence, the study was terminated early.
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | OL and OL High-EPO Components: Number of Participants Who Achieved Red Blood Cell (RBC) Transfusion Independence (TI) ≥8 Weeks (≥56 Consecutive Days) Since First Dose in the First 28 Weeks of Treatment | 28 weeks | The RBC TI was defined as the absence of any intravenous (IV) RBC transfusion (packed cell or whole blood) during any consecutive 56 days during the treatment period. Data presented is for number of participants with RBC TI ≥8 weeks (≥56 consecutive days) since first dose in the first 28 weeks of treatment. |
| Primary | DB Component: Number of Participants Who Achieved RBC TI ≥56 Consecutive Days Since First Dose in the First 28 Weeks of Treatment | 28 weeks | RBC TI was defined as the absence of any IV RBC transfusion (packed cell or whole blood) during any consecutive 56 days during the treatment period. Data presented is for number of participants with RBC TI ≥56 consecutive days since first dose in the first 28 weeks of treatment. |
| Secondary | OL and OL High-EPO Components: Number of Participants Who Achieved TI ≥50% Reduction From Baseline in Number of Packs of Red Blood Cells (pRBC) Transfusions Over 8 Weeks | Baseline up to Week 8 | Number of pRBC transfusions at baseline was defined as pRBC transfusions requirement during 8-week period prior to the start of first study medication. Responders were defined as participants with at least a 50% reduction in the number of pRBC transfusions over any 8-week (56 consecutive days) period during the study as compared with the baseline. |
| Secondary | DB Component: Number of Participants Who Achieved TI ≥56 Consecutive Days Since First Dose in 52 Weeks of Treatment | 52 weeks | RBC TI was defined as the absence of any IV RBC transfusion (packed cell or whole blood) during any consecutive 56 days during the treatment period. Data presented is for number of participants with RBC TI ≥56 consecutive days since first dose in the 52 weeks of treatment. |
| Secondary | DB Component: Number of Participants Who Achieved TI ≥56 Consecutive Days Anytime During the Study | Baseline up to Week 56 | RBC TI was defined as the absence of any IV RBC transfusion (packed cell or whole blood) during any consecutive 56 days anytime during the study (up to Week 56). |
| Secondary | DB Component: Number of Participants Who Achieved ≥50% Reduction From Baseline in Number of pRBC Transfusions Over 8 Weeks | Baseline up to Week 8 | Baseline number of transfusions (pRBC/8-weeks) = total number of packs of rRBCs within 16 weeks prior to first dose/2. A pRBC transfusion reduction responder was defined as a participant who achieved ≥50% reduction in number of pRBC transfusions over 8 weeks compared to their baseline for any 8 week period in the duration begining with the first dose date (Day 1) and ending with the end of study or treatment discontinuation due to adverse event (AE)/serious adverse event (SAE) or death, whichever came earlier. |
| Secondary | DB Component: Cumulative Number of Participant Exposure Weeks (PEW) of TI Over the First 28 Weeks of Treatment | 28 weeks | The PEW of TI periods over the first 28 weeks was added up to a cumulative number of weeks. For a participant with at least 1 TI response period over the first 28 weeks, the last TI response period was ended with the date of a subsequent RBC transfusion, visit date at Week 28, date of the end of study or treatment discontinuation due to AE/SAE or death, whichever came earlier. For a participant with no TI response period over the first 28 weeks, the cumulative number of PEW was set to zero. |
| Secondary | DB Component: Change From Baseline in Number of pRBC Packs Transfused Over the First 28 Weeks of Treatment | Baseline, Week 28 | Number of pRBC transfusions at baseline was defined as pRBC transfusions requirement during 8-week period prior to the start of first study medication. |
| Secondary | DB Component: Number of Participants Who Achieved TI ≥20 Consecutive Weeks During the Study | Baseline up to Week 56 | ≥20 consecutive weeks TI was defined as the absence of any IV RBC transfusion (packed cell or whole blood) during any consecutive 140 days anytime during the study (up to 56 weeks). TI was estimated between the first dose date (Day 1) and the end of study (Week 56) or treatment discontinuation due to AE/SAE or death, whichever came earlier. |
| Secondary | DB Component: Mean Change From Baseline in the Patient-Reported Outcomes Measurement Information System-Short Form (PROMIS-SF) v2.0 Physical Function (PF) 10b Score at Week 9 | Baseline, Week 9 | The PROMIS physical function item measures self-reported, current capability to carry out activities that require physical actions, ranging from self-care (activities of daily living) to more complex activities that require a combination of skills, often within a social context. The PF 10-item short form which contains 10 questions was used in this study, and each item was scored on a 5-point rating scale (1 \[unable to do\] to 5 \[without any difficulty\]), with higher scores indicating better functioning. Total raw score was the sum of the response to each question, with the lowest possible raw score 10 (poor physical function) and the highest possible raw score 50 (better physical function). Raw scores converted to T-scores (as detailed in the T-score conversion table for PROMIS-SF v2.0 Physical Function 10b) with a mean of 50 and a standard deviation (SD) of 10. T-scores ranged from minimum 13.8 to maximum 61.3 possible scores with higher scores indicating better physical functioning. |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes12
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | OL and OL High-EPO Components: Number of Participants Who Achieved Red Blood Cell (RBC) Transfusion Independence (TI) ≥8 Weeks (≥56 Consecutive Days) Since First Dose in the First 28 Weeks of Treatment | 28 weeks | The full analysis set (FAS) included all enrolled participants who received at least 1 dose of study medication, and at least 1 corresponding on-treatment hemoglobin (Hb) assessment. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | DB Component: Number of Participants Who Achieved RBC TI ≥56 Consecutive Days Since First Dose in the First 28 Weeks of Treatment | 28 weeks | FAS included all enrolled participants who received at least 1 dose of study medication, and at least 1 corresponding on-treatment Hb assessment. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | OL and OL High-EPO Components: Number of Participants Who Achieved TI ≥50% Reduction From Baseline in Number of Packs of Red Blood Cells (pRBC) Transfusions Over 8 Weeks | Baseline up to Week 8 | FAS included all enrolled participants who received at least 1 dose of study medication, and at least 1 corresponding on-treatment Hb assessment. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | DB Component: Number of Participants Who Achieved TI ≥56 Consecutive Days Since First Dose in 52 Weeks of Treatment | 52 weeks | FAS included all enrolled participants who received at least 1 dose of study medication, and at least 1 corresponding on-treatment Hb assessment. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | DB Component: Number of Participants Who Achieved TI ≥56 Consecutive Days Anytime During the Study | Baseline up to Week 56 | FAS included all enrolled participants who received at least 1 dose of study medication, and at least 1 corresponding on-treatment Hb assessment. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | DB Component: Number of Participants Who Achieved ≥50% Reduction From Baseline in Number of pRBC Transfusions Over 8 Weeks | Baseline up to Week 8 | FAS included all enrolled participants who received at least 1 dose of study medication, and at least 1 corresponding on-treatment Hb assessment. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | DB Component: Cumulative Number of Participant Exposure Weeks (PEW) of TI Over the First 28 Weeks of Treatment | 28 weeks | FAS included all enrolled participants who received at least 1 dose of study medication, and at least 1 corresponding on-treatment Hb assessment. | weeks | MEAN |
| SECONDARY | DB Component: Change From Baseline in Number of pRBC Packs Transfused Over the First 28 Weeks of Treatment | Baseline, Week 28 | FAS included all enrolled participants who received at least 1 dose of study medication, and at least 1 corresponding on-treatment Hb assessment. | pRBC packs | MEAN |
| SECONDARY | DB Component: Number of Participants Who Achieved TI ≥20 Consecutive Weeks During the Study | Baseline up to Week 56 | FAS included all enrolled participants who received at least 1 dose of study medication, and at least 1 corresponding on-treatment Hb assessment. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | DB Component: Mean Change From Baseline in the Patient-Reported Outcomes Measurement Information System-Short Form (PROMIS-SF) v2.0 Physical Function (PF) 10b Score at Week 9 | Baseline, Week 9 | FAS included all enrolled participants who received at least 1 dose of study medication, and at least 1 corresponding on-treatment Hb assessment. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | T-score | MEAN |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 outcomesResults by group28
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
OL and OL High-EPO Components: Number of Participants Who Achieved Red Blood Cell (RBC) Transfusion Independence (TI) ≥8 Weeks (≥56 Consecutive Days) Since First Dose in the First 28 Weeks of Treatment | OL Component (Cohort 1): Roxadustat 1.5 mg/kg | 3 | Participants | ||
OL and OL High-EPO Components: Number of Participants Who Achieved Red Blood Cell (RBC) Transfusion Independence (TI) ≥8 Weeks (≥56 Consecutive Days) Since First Dose in the First 28 Weeks of Treatment | OL Component (Cohort 2): Roxadustat 2.0 mg/kg | 1 | Participants | ||
OL and OL High-EPO Components: Number of Participants Who Achieved Red Blood Cell (RBC) Transfusion Independence (TI) ≥8 Weeks (≥56 Consecutive Days) Since First Dose in the First 28 Weeks of Treatment | OL Component (Cohort 3): Roxadustat 2.5 mg/kg | 5 | Participants | ||
OL and OL High-EPO Components: Number of Participants Who Achieved Red Blood Cell (RBC) Transfusion Independence (TI) ≥8 Weeks (≥56 Consecutive Days) Since First Dose in the First 28 Weeks of Treatment | OL High-EPO Component: Roxadustat 2.5 mg/kg | 3 | Participants | ||
| DB Component: Number of Participants Who Achieved RBC TI ≥56 Consecutive Days Since First Dose in the First 28 Weeks of Treatment | DB Component: Roxadustat | 38 | Participants | ||
| DB Component: Number of Participants Who Achieved RBC TI ≥56 Consecutive Days Since First Dose in the First 28 Weeks of Treatment | DB Component: Placebo | 19 | Participants | ||
| OL and OL High-EPO Components: Number of Participants Who Achieved TI ≥50% Reduction From Baseline in Number of Packs of Red Blood Cells (pRBC) Transfusions Over 8 Weeks | OL Component (Cohort 1): Roxadustat 1.5 mg/kg | 5 | Participants | ||
| OL and OL High-EPO Components: Number of Participants Who Achieved TI ≥50% Reduction From Baseline in Number of Packs of Red Blood Cells (pRBC) Transfusions Over 8 Weeks | OL Component (Cohort 2): Roxadustat 2.0 mg/kg | 3 | Participants | ||
| OL and OL High-EPO Components: Number of Participants Who Achieved TI ≥50% Reduction From Baseline in Number of Packs of Red Blood Cells (pRBC) Transfusions Over 8 Weeks | OL Component (Cohort 3): Roxadustat 2.5 mg/kg | 7 | Participants | ||
| OL and OL High-EPO Components: Number of Participants Who Achieved TI ≥50% Reduction From Baseline in Number of Packs of Red Blood Cells (pRBC) Transfusions Over 8 Weeks | OL High-EPO Component: Roxadustat 2.5 mg/kg | 8 | Participants |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 results by groupParticipant flow72
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| OL Component | STARTED | OL Component (Cohort 1): Roxadustat 1.5 mg/kg | 8 | |
| OL Component | STARTED | OL Component (Cohort 2): Roxadustat 2.0 mg/kg | 8 | |
| OL Component | STARTED | OL Component (Cohort 3): Roxadustat 2.5 mg/kg | 8 | |
| OL Component | STARTED | OL High-EPO Component: Roxadustat 2.5 mg/kg | 0 | |
| OL Component | STARTED | DB Component: Roxadustat | 0 | |
| OL Component | STARTED | DB Component: Placebo | 0 | |
| OL Component | Received at Least 1 Dose of Study Drug | OL Component (Cohort 1): Roxadustat 1.5 mg/kg | 8 | |
| OL Component | Received at Least 1 Dose of Study Drug | OL Component (Cohort 2): Roxadustat 2.0 mg/kg | 8 | |
| OL Component | Received at Least 1 Dose of Study Drug | OL Component (Cohort 3): Roxadustat 2.5 mg/kg | 8 | |
| OL Component | Received at Least 1 Dose of Study Drug | OL High-EPO Component: Roxadustat 2.5 mg/kg | 0 |
1 to 10 of 72Source ClinicalTrials.gov
See all 72 participant flowAdverse events1,074
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 | Haemorrhagic diathesis | Blood and lymphatic system disorders | OL Component (Cohort 1): Roxadustat 1.5 mg/kg | 1 | 8 | |
| other | Haemorrhagic diathesis | Blood and lymphatic system disorders | OL Component (Cohort 2): Roxadustat 2.0 mg/kg | 0 | 8 | |
| other | Haemorrhagic diathesis | Blood and lymphatic system disorders | OL Component (Cohort 3): Roxadustat 2.5 mg/kg | 0 | 8 | |
| other | Haemorrhagic diathesis | Blood and lymphatic system disorders | OL High-EPO Component: Roxadustat 2.5 mg/kg | 0 | 20 | |
| other | Haemorrhagic diathesis | Blood and lymphatic system disorders | DB Component: Roxadustat | 0 | 82 | |
| other | Haemorrhagic diathesis | Blood and lymphatic system disorders | DB Component: Placebo | 0 | 58 | |
| other | Neutropenia | Blood and lymphatic system disorders | OL Component (Cohort 1): Roxadustat 1.5 mg/kg | 0 | 8 | |
| other | Neutropenia | Blood and lymphatic system disorders | OL Component (Cohort 2): Roxadustat 2.0 mg/kg | 0 | 8 | |
| other | Neutropenia | Blood and lymphatic system disorders | OL Component (Cohort 3): Roxadustat 2.5 mg/kg | 0 | 8 | |
| other | Neutropenia | Blood and lymphatic system disorders | OL High-EPO Component: Roxadustat 2.5 mg/kg | 1 | 20 |
1 to 10 of 1,074Source ClinicalTrials.gov
See all 1,074 adverse eventsAdverse event totals18
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | OL Component (Cohort 1): Roxadustat 1.5 mg/kg | 1 | 8 |
| Total, all-cause mortality | OL Component (Cohort 2): Roxadustat 2.0 mg/kg | 0 | 8 |
| Total, all-cause mortality | OL Component (Cohort 3): Roxadustat 2.5 mg/kg | 0 | 8 |
| Total, all-cause mortality | OL High-EPO Component: Roxadustat 2.5 mg/kg | 3 | 20 |
| Total, all-cause mortality | DB Component: Roxadustat | 7 | 82 |
| Total, all-cause mortality | DB Component: Placebo | 4 | 58 |
| Total, other adverse events | OL Component (Cohort 1): Roxadustat 1.5 mg/kg | 7 | 8 |
| Total, other adverse events | OL Component (Cohort 2): Roxadustat 2.0 mg/kg | 8 | 8 |
| Total, other adverse events | OL Component (Cohort 3): Roxadustat 2.5 mg/kg | 6 | 8 |
| Total, other adverse events | OL High-EPO Component: Roxadustat 2.5 mg/kg | 15 | 20 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 34724251 | 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 comparisons5
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| DB Component: Roxadustat vs DB Component: Placebo: DB Component: Number of Participants Who Achieved RBC TI ≥56 Consecutive Days Since First Dose in the First 28 Weeks of Treatment | Trial comparisons |
| DB Component: Roxadustat vs DB Component: Placebo: DB Component: Number of Participants Who Achieved TI ≥56 Consecutive Days Since First Dose in 52 Weeks of Treatment | Trial comparisons |
| DB Component: Roxadustat vs DB Component: Placebo: DB Component: Number of Participants Who Achieved TI ≥56 Consecutive Days Anytime During the Study | Trial comparisons |
| DB Component: Roxadustat vs DB Component: Placebo: DB Component: Number of Participants Who Achieved TI ≥20 Consecutive Weeks During the Study | Trial comparisons |
| DB Component: Roxadustat vs DB Component: Placebo: DB Component: Number of Participants Who Achieved ≥50% Reduction From Baseline in Number of pRBC Transfusions Over 8 Weeks | Trial comparisons |
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/NCT03263091
GET https://conductscience.org/api/trials/v1/NCT03263091/results?page=2