Primary MDS (Very Low, Low or Intermediate IPSS-R With <5% Blasts)Phase 3NCT03263091
Efficacy and Safety of Roxadustat for Treatment of Anemia in Participants With Lower Risk Myelodysplastic Syndrome With Low Red Blood Cell Transfusion Burden
A Phase 3 study testing Roxadustat in people with Primary MDS (Very Low, Low or Intermediate IPSS-R With <5% Blasts) and Anemia, run by Kyntra Bio at 124 sites in 16 countries.
Trial summary
The purpose of this study is to determine whether FG-4592 is safe and effective in the treatment of anemia in participants with lower risk MDS and low red blood cell transfusion burden. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary MDS (Very Low, Low or Intermediate IPSS-R With <5% Blasts), Anemia
- What is being tested?
- Roxadustat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 184 participants
- Where
- 124 sitesin 16 countries
- Study dates
- 29 Jan 2018to 20 Jun 2023
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of primary MDS classified by the International Prognostic Scoring System - Revised (IPSS-R) as very low, low or intermediate risk with <5% bone marrow blasts. There is no minimum time from diagnosis to registration/randomization except to allow for proper IPSS-R classification to be made (within 16 weeks prior to randomization), and to show transfusion dependence for participants in both portions of the study.
- RBC transfusion of either 2-4 pRBC units during the 8 weeks prior to registration/randomization or 1 pRBC in two consecutive periods of 8 weeks within the 16 weeks prior to registration/randomization. Open-Label Lead-in participants only, the requirement to demonstrate transfusion dependence can also be met by a Principal Investigator starting this particular participant on pRBC transfusion during the screening period.
- No restriction on prior use of recombinant erythropoietins or analogues (erythropoiesis-stimulating agents [ESAs]), except no ESA use within 8 weeks prior to Day 1 registration/randomization.
- Hemoglobin (Hb) ≤10.0 grams/deciliter (g/dL) during screening
- Eastern Cooperative Oncology Group (ECOG) of 0-2 at screening
Who cannot join
- Diagnosis of secondary MDS associated with prior chemotherapy, extensive radiation therapy (>25% of bone marrow reserve), and or/other significant chemical or radiation exposure
- Significant myelofibrosis (>2+ fibrosis)
- MDS associated with 5q(del) cytogenetic abnormality
- Screen serum erythropoietin level > 400 milli-international units (mIU)/milliliter (mL) • Clinically significant anemia, as determined by the investigator, due to non-MDS etiologies such as iron deficiency, vitamin B12 or folate deficiency, autoimmune or hereditary hemolysis or anemia or hemorrhage or hereditary anemia such as sickle cell anemia or thalassemia.
Trial locations and contacts
124 sites in 16 countries.
124 sites in 16 countries
United States30 sites
Investgational site
Atlanta, Georgia, 30322
Investigational Site
Augusta, Georgia, 30912
Investigational Site
Bakersfield, California, 93309
Investigational Site
Beverly Hills, California, 90212
Investigational Site
Burbank, California, 90212
Investigational Site
Canton, Ohio, 44718
Investigational Site
Charleston, South Carolina, 29414
Investigational Site
Charlotte, North Carolina, 28204
Investigational Site
Charlottesville, Virginia, 22908
Investigational Site
Cleveland, Ohio, 44111
Investigational Site
Cleveland, Ohio, 44195
Investigational Site
Encino, California, 91436
Investigational Site
Grand Junction, Colorado, 81501
Investigational Site
Houston, Texas, 77030
Investigational Site
Laguna Hills, California, 92653
Investigational Site
Los Angeles, California, 90095
Investigational Site
Los Angeles, California, 91326
Investigational Site
Mayfield Heights, Ohio, 44124
Investigational Site
Oceanside, California, 92056
Investigational Site
Pasadena, California, 91105
Investigational Site
Pembroke Pines, Florida, 33028
Investigational Site
Philadelphia, Pennsylvania, 19106
Investigational Site
Santa Maria, California, 93454
Investigational Site
St Louis, Missouri, 63110
Investigational Site
Torrance, California, 90505
Investigational Site
Valencia, California, 91355
Investigational Site
Ventura, California, 93003
Investigational Site
Westlake Village, California, 91361
Investiational Site
Weston, Florida, 33331
Investigational Site
Whittier, California, 90603
Italy14 sites
Investigational Site
Alessandria, 15121
Investigational Site
Bologna, 40138
Investigational Site
Florence, 50134
Investigational Site
Genova, 16132
Investigational Site
Lecce, 73100
Investigational Site
Meldola, Ravenna, 47014
Investigational Site
Milan, 20089
Investigational Site
Monza, Lombardy, 20900
Investigational Site
Reggio Calabria, 89124
Investigational Site
Rimini
Investigational Site
Roma, 00133
Investigational Site
Terni, 5100
Investigational Site
Torino
Investigational Site
Varese, 21100
Spain13 sites
Investigational Site
Barcelona, Catalonia, 08003
Investigational Site
Barcelona, 08035
Investigational Site
Barcelona, 08041
Investigational Site
Barcelona, 08908
Investigational Site
Madrid, 28007
Investigational Site
Madrid, 28034
Investigational Site
Madrid, 28050
Investigational Site
Pamplona, Navarre, 31008
Investigational Site
Sabadell, Barcelona, 08208
Investigational Site
Salamanca, 37007
Investigational Site
Seville, 41009
Investigational Site
Seville, 41013
Investigational Site
Valencia, 46026
Russia9 sites
Investigational Site
Kaluga, Russian Federation, 248007
Investigational Site
Moscow, Russian Federation, 111123
Investigational Site
Moscow, Russian Federation, 123182
Investigational Site
Moscow, Russian Federation, 129110
Investigational Site
Omsk, Russian Federation, 644013
Investigational Site
Saint Petersburg, Russian Federation, 191024
Investigational Site
Saint Petersburg, Russian Federation, 197089
Investigational Site
Saint Petersburg, Russian Federation, 197341
Investigational Site
Saint Petersburg, 197022
Germany7 sites
Investigational Site
Dresden, Saxony, 01307
Investigational Site
Düsseldorf, 40225
Investigational Site
Düsseldorf, 40479
Investigational Site
Freiburg im Breisgau, Baden-Wurttemberg, 79106
Investigational Site
Leipzig, Saxony, 04103
Investigational Site
München, Bavaria, 81675
Investigational Site
Münster, North Rhine-Westphalia, 48149
Israel7 sites
Investigational Site
Haifa, 3436212
Investigational Site
Kfar Saba, Central District, 4428164
Investigational Site
Nahariya, Northern District, 22100
Investigational Site
Ramat Gan, 5265601
Investigational Site
Tel Aviv, 49372
Investigational site
Tel HaShomer
Investigational Site
Tsrifin, Central District, 70300
Poland7 sites
Investigational Site
Bialystok, 15-732
Investigational Site
Bydgoszcz, 85-168
Investigational Site
Krakow, 31-513
Investigational Site
Piła, 64-920
Investigational Site
Skorzewo, 60-185
Investigational Site
Słupsk, 76-200
Investigational Site
Warsaw, 02-172
Turkey (Türkiye)7 sites
Investigational Site
Ankara, 6500
Investigational Site
Dikimevi, 06590
Investigational Site
Izmir, 35340
Investigational Site
Izmir, 59100
Investigational Site
Kayseri, 38039
Investigational Site
Tekirdağ, 59100
Investigational Site
Yenişehir, 33110
Belgium6 sites
Investigational Site
Bruges, West-Vlaanderen, 8000
Investigational Site
Brussels, Brussels Capital, 1200
Investigational Site
Brussels
Investigational Site
Hasselt, Limburg, 3500
Investigational Site
Wilrijk, Antwerpen, 2610
Investigational Site
Yvoir, 5530
Australia5 sites
Investigational Site
Darlinghurst, New South Wales, 2010
Investigational Site
Hobart, Tasmania, 7000
Investigational Site
Liverpool, New South Wales, 2170
Investigational Site
South Brisbane, Queensland, 4101
Investigational Site
Victoria Park, Saint Albans, 3021
South Korea5 sites
Investigational Site
Hwasun, Jeonranamdo, 58128
Investigational Site
Incheon, Incheon Gwang'yeogsi, 21565
Investigational Site
Incheon, Incheon Gwangyeogsi, 21565
Investigational Site
Seoul, Seoul Teugbyeolsi, 06351
Investigational Site
Seoul, 03080
United Kingdom5 sites
Investigational Site
Boston, Lincolnshire, PE21 9QS
Investigational Site
Harrow, HA1 3UJ
Investigational Site
Headington, Oxford, OX3 7LE
Investigational Site
London, SE5 9RS
Investigational Site
Manchester, M20 4BX
France4 sites
Investigational Site
Grenoble, Isère, 38043
Investigational Site
Nice, Alpes-Maritimes, 06200
Investigational Site
Paris
Investigational Site
Tours, 37044
Canada2 sites
Investigational Site
London, Ontario, N6A5W9
Investigational Site
Vancouver, British Columbia, V6E 1M7
India2 sites
Investigational Site
Kolkata, West Bengal, 700014
Investigational Site
Vellore, Tamil Nadu, 632 004
About this study
Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Investigating the Efficacy and Safety of Roxadustat (FG-4592) for Treatment of Anemia in Patients With Lower Risk Myelodysplastic Syndrome (MDS) With Low Red Blood Cell (RBC) Transfusion Burden (LTB)
The purpose of this study is to determine whether FG-4592 is safe and effective in the treatment of anemia in participants with lower risk MDS and low red blood cell transfusion burden.
Read the detailed description
This study includes an Open-Label Lead in, a Double-Blind component, and an Open-Label High Erythropoietin component. There is a screening period of up to 42 days followed by a treatment period of 52 weeks and a 4-week end of treatment assessment.
- Being tested
- Roxadustat, Placebo
- Keywords
- Myelodysplastic Syndromes, Anemia, Hemoglobin (Hb), Low Risk Myelodysplastic Syndrome, Low Risk MDS
- Medical subject headings
- Anemia, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RoxadustatDrug
Also called FG-4592, ASP1517, AZD9941
Oral tablets
PlaceboDrug
Oral tablets
| Study group | Role | What they receive |
|---|---|---|
| Roxadustat | Experimental | Open-label, lead-in: Participants will receive sequential escalating roxadustat doses (1.5 milligrams/kilograms [mg/kg], 2.0 mg/kg and 2.5 mg/kg), three times a week (TIW) based upon their actual weight at the randomization visit to identify the starting dose for double-blind period.
Double-blind: Participants will receive roxadustat 2.5 mg/kg TIW based upon their body weight for a duration of 52 weeks.
Open-label: Participants with high serum erythropoietin levels (>400 milli-international units [mIU]/milliliter [mL] mIU/mL) will receive roxadustat 2.5 mg/kg TIW based upon their body weight for a duration of 52 weeks. Receives: Drug: Roxadustat |
| Placebo | Placebo comparator | Double-blind: Participants will receive placebo matching to roxadustat for a duration of 52 weeks. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
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.
Measured: 28 weeks
DB Component: Number of Participants Who Achieved RBC TI ≥56 Consecutive Days Since First Dose in the First 28 Weeks of Treatment
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.
Measured: 28 weeks
Secondary outcomes (10)
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
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.
Measured: Baseline up to Week 8
DB Component: Number of Participants Who Achieved TI ≥56 Consecutive Days Since First Dose in 52 Weeks of Treatment
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.
Measured: 52 weeks
DB Component: Number of Participants Who Achieved TI ≥56 Consecutive Days Anytime During the Study
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).
Measured: Baseline up to Week 56
DB Component: Number of Participants Who Achieved ≥50% Reduction From Baseline in Number of pRBC Transfusions Over 8 Weeks
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.
Measured: Baseline up to Week 8
DB Component: Cumulative Number of Participant Exposure Weeks (PEW) of TI Over the First 28 Weeks of Treatment
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.
Measured: 28 weeks
DB Component: Change From Baseline in Number of pRBC Packs Transfused Over the First 28 Weeks of Treatment
Number of pRBC transfusions at baseline was defined as pRBC transfusions requirement during 8-week period prior to the start of first study medication.
Measured: Baseline, Week 28
DB Component: Number of Participants Who Achieved TI ≥20 Consecutive Weeks During the Study
≥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.
Measured: Baseline up to Week 56
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
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.
Measured: Baseline, Week 9
DB Component: Mean Change From Baseline in the PROMIS-SF v1.0 Fatigue 13a Score at Week 9
Fatigue was measured using the 13-item fatigue scale of the Functional Assessment of Chronic Illness Therapy (FACIT) Measurement System, each item was scored on a 5-point rating scale ranging from 1 "not at all" to 5 "very much", with lower scores indicating better functioning. Total raw score was the sum of the response to each question, with the lowest possible raw score 13 (lowest level of fatigue) and the highest possible raw score 65 (highest level of fatigue), with lower scores indicating better functioning. Raw scores converted to T-scores (as detailed in the T-score conversion table for PROMIS-SF v1.0 Fatigue 13a) with a mean of 50 and a SD of 10. T-scores ranged from minimum 30.3 to maximum 83.5 possible scores with lower scores indicating better functioning.
Measured: Baseline, Week 9
DB Component: Mean Change From Baseline in the European Quality of Life Five Dimensional Five Level Health Questionnaire (EQ-5D-5L) Visual Analogue Scale Score at Week 9
The EQ-5D questionnaire is designed for self-completion by participants. The EQ-5D-5L descriptive system comprises the following 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 levels: no problems, slight problem, moderate problems, severe problems, and unable to/extreme problems. The questionnaire also included a visual analogue scale, where the participant was asked to rate current health status on a scale of 0-100, with 0 being the worst imaginable health state and 100 being the best imaginable health.
Measured: Baseline, Week 9
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Aug 2017
- Study start29 Jan 2018
- Primary completion9 Mar 2023
- Study completion20 Jun 2023
Why it stopped: Study did not meet its primary efficacy endpoint.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary MDS (Very Low, Low or Intermediate IPSS-R With <5% Blasts)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 184 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kyntra Bio
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- AstraZeneca, Astellas Pharma Inc
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03263091
- Sponsor's protocol ID
- FGCL-4592-082
- Status last verified
- Jul 2024
- First submitted
- 22 Aug 2017
- Last updated
- 1 Aug 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Henry DH, Glaspy J, Harrup R, Mittelman M, Zhou A, Carraway HE, Bradley C, Saha G, Modelska K, Bartels P, Leong R, Yu KP. Roxadustat for the treatment of anemia in patients with lower-risk myelodysplastic syndrome: Open-label, dose-selection, lead-in stage of a phase 3 study. Am J Hematol. 2022 Feb 1;97(2):174-184. doi: 10.1002/ajh.26397. Epub 2021 Nov 9.
PubMed 34724251
Study at a glance
From the registry record, last updated by the sponsor on 1 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Investigating the Efficacy and Safety of Roxadustat (FG-4592) for Treatment of Anemia in Patients With Lower Risk Myelodysplastic Syndrome (MDS) With Low Red Blood Cell (RBC) Transfusion Burden (LTB)
- ClinicalTrials.gov ID
- NCT03263091
- Acronym / sponsor ID
- FGCL-4592-082
- Keywords
- Myelodysplastic Syndromes, Anemia, Hemoglobin (Hb), Low Risk Myelodysplastic Syndrome, Low Risk MDS
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study did not meet its primary efficacy endpoint.
- Enrolment
- 184 participants (actual)
- Lead sponsor
- Kyntra BioIndustry (pharma, biotech, devices)
- Collaborators
- AstraZeneca, Astellas Pharma Inc
- Study start
- 29 Jan 2018 (actual)
- Primary completion
- 9 Mar 2023 (actual)
- Study completion
- 20 Jun 2023 (actual)
- First posted
- 28 Aug 2017 (actual)
- Results
- Posted 1 Aug 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine whether FG-4592 is safe and effective in the treatment of anemia in participants with lower risk MDS and low red blood cell transfusion burden.
Detailed description
This study includes an Open-Label Lead in, a Double-Blind component, and an Open-Label High Erythropoietin component. There is a screening period of up to 42 days followed by a treatment period of 52 weeks and a 4-week end of treatment assessment.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Roxadustat | Experimental | Drug: RoxadustatOpen-label, lead-in: Participants will receive sequential escalating roxadustat doses (1.5 milligrams/kilograms [mg/kg], 2.0 mg/kg and 2.5 mg/kg), three times a week (TIW) based upon their actual weight at the randomization visit to identify the starting dose for double-blind period. Double-blind: Participants will receive roxadustat 2.5 mg/kg TIW based upon their body weight for a duration of 52 weeks. Open-label: Participants with high serum erythropoietin levels (>400 milli-international units [mIU]/milliliter [mL] mIU/mL) will receive roxadustat 2.5 mg/kg TIW based upon their body weight for a duration of 52 weeks. |
| Placebo | Placebo comparator | Drug: PlaceboDouble-blind: Participants will receive placebo matching to roxadustat for a duration of 52 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Diagnosis of primary MDS classified by the International Prognostic Scoring System - Revised (IPSS-R) as very low, low or intermediate risk with <5% bone marrow blasts. There is no minimum time from diagnosis to registration/randomization except to allow for proper IPSS-R classification to be made (within 16 weeks prior to randomization), and to show transfusion dependence for participants in both portions of the study.
- RBC transfusion of either 2-4 pRBC units during the 8 weeks prior to registration/randomization or 1 pRBC in two consecutive periods of 8 weeks within the 16 weeks prior to registration/randomization. Open-Label Lead-in participants only, the requirement to demonstrate transfusion dependence can also be met by a Principal Investigator starting this particular participant on pRBC transfusion during the screening period.
- No restriction on prior use of recombinant erythropoietins or analogues (erythropoiesis-stimulating agents [ESAs]), except no ESA use within 8 weeks prior to Day 1 registration/randomization.
- Hemoglobin (Hb) ≤10.0 grams/deciliter (g/dL) during screening
- Eastern Cooperative Oncology Group (ECOG) of 0-2 at screening
Key exclusion criteria (4 listed)
- Diagnosis of secondary MDS associated with prior chemotherapy, extensive radiation therapy (>25% of bone marrow reserve), and or/other significant chemical or radiation exposure
- Significant myelofibrosis (>2+ fibrosis)
- MDS associated with 5q(del) cytogenetic abnormality
- Screen serum erythropoietin level > 400 milli-international units (mIU)/milliliter (mL) • Clinically significant anemia, as determined by the investigator, due to non-MDS etiologies such as iron deficiency, vitamin B12 or folate deficiency, autoimmune or hereditary hemolysis or anemia or hemorrhage or hereditary anemia such as sickle cell anemia or thalassemia.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RoxadustatAlso: FG-4592, ASP1517, AZD9941Oral tablets | Drug | Roxadustat |
| PlaceboOral tablets | Drug | Placebo |
Primary outcome
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
Time frame: 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.
DB Component: Number of Participants Who Achieved RBC TI ≥56 Consecutive Days Since First Dose in the First 28 Weeks of Treatment
Time frame: 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.
10 secondary outcomes
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
Time frame: Baseline up to Week 8
DB Component: Number of Participants Who Achieved TI ≥56 Consecutive Days Since First Dose in 52 Weeks of Treatment
Time frame: 52 weeks
DB Component: Number of Participants Who Achieved TI ≥56 Consecutive Days Anytime During the Study
Time frame: Baseline up to Week 56
DB Component: Number of Participants Who Achieved ≥50% Reduction From Baseline in Number of pRBC Transfusions Over 8 Weeks
Time frame: Baseline up to Week 8
DB Component: Cumulative Number of Participant Exposure Weeks (PEW) of TI Over the First 28 Weeks of Treatment
Time frame: 28 weeks
DB Component: Change From Baseline in Number of pRBC Packs Transfused Over the First 28 Weeks of Treatment
Time frame: Baseline, Week 28
DB Component: Number of Participants Who Achieved TI ≥20 Consecutive Weeks During the Study
Time frame: Baseline up to Week 56
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
Time frame: Baseline, Week 9
DB Component: Mean Change From Baseline in the PROMIS-SF v1.0 Fatigue 13a Score at Week 9
Time frame: Baseline, Week 9
DB Component: Mean Change From Baseline in the European Quality of Life Five Dimensional Five Level Health Questionnaire (EQ-5D-5L) Visual Analogue Scale Score at Week 9
Time frame: Baseline, Week 9
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Aug 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 34724251Henry DH, Glaspy J, Harrup R, Mittelman M, Zhou A, Carraway HE, Bradley C, Saha G, Modelska K, Bartels P, Leong R, Yu KP. Roxadustat for the treatment of anemia in patients with lower-risk myelodysplastic syndrome: Open-label, dose-selection, lead-in stage of a phase 3 study. Am J Hematol. 2022 Feb 1;97(2):174-184. doi: 10.1002/ajh.26397. Epub 2021 Nov 9.
- Individual participant dataSharing planned: No
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
124 sites in 16 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Roxadustat in people with Primary MDS (Very Low, Low or Intermediate IPSS-R With <5% Blasts) and Anemia, run by Kyntra Bio at 124 sites in 16 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 1 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 124 sites in United States, Italy, Spain, Russia, Germany and 11 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 1 Aug 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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 (time frame: 28 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03263091, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Aug 2024.