Anemia, Myelodysplastic Syndrome
Conditions
Keywords
Myelodysplastic Syndromes, Anemia, Hemoglobin (Hb), Low Risk Myelodysplastic Syndrome
Brief summary
The objective of this study to evaluate the efficacy FG-4592 for the treatment of anemia in Chinese participants with lower risk MDS.
Detailed description
This is a Phase 2/3 trial with sequential Phase 2 and Phase 3 components. In each part, there is an up to 4 weeks screening period followed by a treatment period of 26 weeks and a 4 week follow-up period.
Interventions
DRUGFG-4592
Oral
DRUGPlacebo
Oral
Sponsors
AstraZeneca
Kyntra Bio
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Masking description
Phase 2 part is an open label, single arm, dose-escalation study. Phase 3 component is a randomized, double blind, placebo-controlled study.
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Diagnosis of primary MDS classified as very low, low or intermediate risk with \<5% blasts (documented within 12 weeks prior to Day 1) * Screening Hb \<10 g/dL and ≥6g/dL * Transfusion independent defined as no red blood cell transfusions within 12 weeks of Day 1 * Erythropoiesis-stimulating agent (ESA)-naïve (not within 30 days of Day 1) * Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2 Key
Exclusion criteria
* Diagnosis of secondary MDS * Significant myelofibrosis (\>2+fibrosis) * Prior therapy with azacitidine, decitabine, antithymocyte globulin, cyclosporine, thalidomide, or lenalidomide within 12 weeks prior to Day 1 * Baseline erythropoietin level of \>400 units (U)/liter (L) * Clinically significant anemia due to non-MDS etiologies Note: Other inclusion and
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With a Hemoglobin (Hb) Response to FG-4592 Without Transfusion | 26 weeks | Hemoglobin response to FG-4592 is defined as an increase in mean Hb of ≥1.0 grams (g)/deciliter (dL) from baseline within any 8-week period during the study without transfusion. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Time to First Hb Response | 26 weeks | Hb response to FG-4592 is defined as an increase in mean Hb of ≥1.0 g/dL from baseline within any 8-week period during the study without transfusion. |
| Change From Baseline in Mean Hb in Weeks 25 to 27 | Baseline, Weeks 25 to 27 | — |
| Number of Participants With Hb Increase ≥1.0 g/dL From Baseline | 26 weeks | Hb response to FG-4592 is defined as an increase in mean Hb of ≥1.0 g/dL from baseline within any 8-week period during the study without transfusion. |
| Number of Participants that Require Transfusions or Hb <6 g/dL up to Week 27 | Up to Week 27 | — |
| Mean Change From Baseline in Functional Assessment of Cancer Therapy - Anemia (FACT-An) and Functional Assessment of Chronic Illness Therapy Fatigue (FACIT-F) Scores at Week 27 | 30 weeks | Frame: Baseline, Week 27 |
| Percentage of Participants With Mean Hb ≥10.0 g/dL Within Any 8-week Period During the Study Without Transfusion | 26 weeks | — |
Countries
China
Outcome results
None listed