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NCT04076943

Evaluation of Efficacy and Safety of Roxadustat for the Treatment of Chemotherapy Induced Anemia

Completed Phase 2 Results posted Last updated 3 June 2022
What this trial tests

Phase 2 trial testing Roxadustat in Chemotherapy Induced Anemia in 92 participants. Completed in 23 April 2021.

Timeline
20 August 2019
Primary endpoint
26 March 2021
23 April 2021

Quick facts

Lead sponsorKyntra Bio
PhasePhase 2
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment92
Start date20 August 2019
Primary completion26 March 2021
Estimated completion23 April 2021
Sites15 locations across United States

Drugs / interventions tested

Conditions studied

Sponsor

Kyntra Bio — full company profile →

Who can join

18 and older, any sex, with Chemotherapy Induced Anemia. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Maximum Change in Hb Within 16 Weeks From Baseline Without Red Blood Cell (RBC) Transfusion Primary · Baseline, up to Week 16

Baseline Hb was defined as the mean of the assessments from central lab prior to first dose of the study treatment, which included up to 2 latest screening values prior to Day 1 and a value on Day 1. All central lab assessments from Day 1 to end of treatment (EOT) or early termination (ET) were included in the evaluation of this endpoint. Hb values within 4 weeks after an RBC transfusion were excluded.

GroupValue95% CI
Roxadustat2.47± 1.510
Mean Change in Hb Level From Baseline to Week 16 (Without RBC Transfusion) Secondary · Baseline, Week 16

Baseline Hb was defined as the mean of the assessments from central lab prior to first dose of the study treatment, which included up to 2 latest screening values prior to Day 1 and a value on Day 1. Mean Hb during treatment was computed using the mean area-under-the-curve trapezoid method, from Day 1 to EOT or ET Hb assessment.

GroupValue95% CI
Roxadustat1.20± 1.012
Change in Hb From Baseline at Weeks 9, 13, and 16 (Without RBC Transfusion) Secondary · Baseline, Weeks 9, 13, and 16

Baseline Hb was defined as the mean of the assessments from central lab prior to first dose of the study treatment, which included up to 2 latest screening values prior to Day 1 and a value on Day 1.

Change at Week 9
GroupValue95% CI
Roxadustat1.43± 1.421
Change at Week 13
GroupValue95% CI
Roxadustat2.15± 1.921
Change at Week 16
GroupValue95% CI
Roxadustat2.52± 1.700
Percentage of Participants Who Achieved a ≥1 g/dL Increase in Hb From Baseline Through Week 16 Secondary · Baseline through Week 16

The 95% confidence interval (CI) was based on the exact method of Clopper-Pearson method. All central lab assessments from Day 1 to EOT or ET were included in the analysis. Hb values within 4 weeks after an RBC transfusion was excluded.

GroupValue95% CI
Roxadustat82.072.5 – 89.4
Time to Achieve a ≥1 g/dL Increase in Hb From Baseline Through Week 16 Secondary · Baseline through Week 16

Median was calculated using Kaplan-Meier product limit method. 95% CI was calculated using the method of Brookmeyer and Crowley. All central lab assessments from Day 1 to EOT or ET were included in the analysis. Hb values within 4 weeks after an RBC transfusion were excluded.

GroupValue95% CI
Roxadustat41.029.0 – 46.0
Percentage of Participants Who Achieved a ≥1.5 g/dL Increase in Hb From Baseline Through Week 16 Secondary · Baseline through Week 16

The 95% CI was based on the exact method of Clopper-Pearson method. All central lab assessments from Day 1 to EOT or ET were included in the analysis. Hb values within 4 weeks after an RBC transfusion was excluded.

GroupValue95% CI
Roxadustat73.062.6 – 81.9
Percentage of Participants Who Achieved a Hematopoietic Response Secondary · Baseline through Week 16

Hematopoietic response was defined as an increase in Hb of 1.5 g/dL from baseline or attaining a Hb of 11 g/dL. The 95% CI was based on the exact method of Clopper-Pearson method. All central lab assessments from Day 1 to EOT or ET were included in the analysis. Hb values within 4 weeks after an RBC transfusion was excluded.

GroupValue95% CI
Roxadustat75.365.0 – 83.8
Percentage of Participants Who Achieved a ≥2 g/dL Increase in Hb From Baseline Through Week 16 Secondary · Baseline through Week 16

The 95% CI was based on the exact method of Clopper-Pearson method. All central lab assessments from Day 1 to EOT or ET were included in the analysis. Hb values within 4 weeks after an RBC transfusion was excluded.

GroupValue95% CI
Roxadustat60.749.7 – 70.9
Percentage of Participants Who Had an RBC Transfusion From Beginning of Week 5 (Day 29) to Week 16 Secondary · Week 5 to Week 16
GroupValue95% CI
Roxadustat14.5

Adverse events — posted to ClinicalTrials.gov

Time frame: Baseline up to Week 20. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Roxadustat
Serious: 40/92 (43%)
Deaths: 17/92

Serious adverse events (53 terms)

ReactionSystemRoxadustat
Pancreatic carcinoma metastaticNeoplasms benign, malignant and unspecified (incl cysts and polyps)
Pulmonary embolismRespiratory, thoracic and mediastinal disorders
Febrile neutropeniaBlood and lymphatic system disorders
AstheniaGeneral disorders
Disease progressionGeneral disorders
SepsisInfections and infestations
Mental status changesPsychiatric disorders
Deep vein thrombosisVascular disorders
AnaemiaBlood and lymphatic system disorders
Acute myocardial infarctionCardiac disorders
DiarrhoeaGastrointestinal disorders
VomitingGastrointestinal disorders
DehydrationMetabolism and nutrition disorders
SeizureNervous system disorders
Acute kidney injuryRenal and urinary disorders
Respiratory failureRespiratory, thoracic and mediastinal disorders
HypotensionVascular disorders
NeutropeniaBlood and lymphatic system disorders
PancytopeniaBlood and lymphatic system disorders
Atrial fibrillationCardiac disorders
Ventricular tachycardiaCardiac disorders
AscitesGastrointestinal disorders
Gastrointestinal haemorrhageGastrointestinal disorders
NauseaGastrointestinal disorders
OdynophagiaGastrointestinal disorders
Other adverse events (23 terms — click to expand)

ReactionSystemRoxadustat
FatigueGeneral disorders
NauseaGastrointestinal disorders
DyspnoeaRespiratory, thoracic and mediastinal disorders
DiarrhoeaGastrointestinal disorders
VomitingGastrointestinal disorders
Deep vein thrombosisVascular disorders
NeutropeniaBlood and lymphatic system disorders
ThrombocytopeniaBlood and lymphatic system disorders
ConstipationGastrointestinal disorders
Decreased appetiteMetabolism and nutrition disorders
Oedema peripheralGeneral disorders
Abdominal painGastrointestinal disorders
PyrexiaGeneral disorders
DehydrationMetabolism and nutrition disorders
HypokalaemiaMetabolism and nutrition disorders
DizzinessNervous system disorders
CoughRespiratory, thoracic and mediastinal disorders
RashSkin and subcutaneous tissue disorders
FallInjury, poisoning and procedural complications
ArthralgiaMusculoskeletal and connective tissue disorders
Muscular weaknessMusculoskeletal and connective tissue disorders
HeadacheNervous system disorders
Pulmonary embolismRespiratory, thoracic and mediastinal disorders

Most-reported serious reactions: Pancreatic carcinoma metastatic, Pulmonary embolism, Febrile neutropenia, Asthenia, Disease progression, Sepsis, Mental status changes, Deep vein thrombosis.

Data from ClinicalTrials.gov NCT04076943 adverse events section.

Sponsor's own description

The purpose of this study is to find out if roxadustat (also known as FG-4592) is safe and effective for the treatment of anemia in participants receiving chemotherapy treatment for cancer.

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Hypoxia-inducible factor-prolyl hydroxylase inhibitors in the treatment of anemia of chronic kidney disease.
    Haase VH. · · 2021 · cited 115× · PMID 33777492 · DOI 10.1016/j.kisu.2020.12.002
  2. The Epigenetic Profile of Tumor Endothelial Cells. Effects of Combined Therapy with Antiangiogenic and Epigenetic Drugs on Cancer Progression.
    Ciesielski O, Biesiekierska M, Panthu B, Vialichka V, et al · · 2020 · cited 36× · PMID 32283668 · DOI 10.3390/ijms21072606
  3. The impact of myelosuppression on quality of life of patients treated with chemotherapy.
    Crawford J, Herndon D, Gmitter K, Weiss J. · · 2024 · cited 31× · PMID 38587388 · DOI 10.2217/fon-2023-0513
  4. Improving Outcomes of Chemotherapy: Established and Novel Options for Myeloprotection in the COVID-19 Era.
    Lyman GH, Kuderer NM, Aapro M. · · 2021 · cited 15× · PMID 34307165 · DOI 10.3389/fonc.2021.697908
  5. A critical review of Roxadustat formulations, solid state studies, and analytical methodology.
    Mahajan R, Samanthula G, Srivastava S, Asthana A. · · 2023 · cited 3× · PMID 37346363 · DOI 10.1016/j.heliyon.2023.e16595
  6. Hypoxia-inducible factor-prolyl hydroxylase inhibitors in treatment of anemia with chronic disease.
    Li Z, Shen L, Tu Y, Lu S, et al · · 2025 · cited 2× · PMID 40405347 · DOI 10.1097/cm9.0000000000003470
  7. [Efficacy and safety of roxadustat in the treatment of refractory non-severe aplastic anemia].
    Xu L, Hu QL, Yang C, Chen M, et al · · 2024 · cited 1× · PMID 38716598 · DOI 10.3760/cma.j.cn121090-20230902-00101

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