Low and Int 1-risk Myelodysplastic SyndromePhase 2NCT01868477

Combination Study of Deferasirox and Erythropoietin in Patients With Low- and Int-1-risk Myelodysplastic Syndrome.

A Phase 2 study testing Deferasirox DFX, DT, Erythropoietin alpha and Deferasirox DFX, FCT in people with Low and Int 1-risk Myelodysplastic Syndrome, run by Novartis Pharmaceuticals at 30 sites in 10 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

Who is it for?: Ages 18 years and older
Conditions the study is for: Low and Int 1-risk Myelodysplastic Syndrome
What is being tested?: Deferasirox DFX, DT, Erythropoietin alpha and Deferasirox DFX, FCT
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 28 participants
Where: 30 sitesin 10 countries
Study dates: 28 Jan 2014to 5 Apr 2017
Phase: Phase 2
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

Patients who had low- and Int-1-risk myelodysplastic syndrome
Documented diagnosis of the following:
Myelodysplastic syndrome that lasted ≥ 3 months and < 3 years Disease must not have been secondary to treatment with radiotherapy, chemotherapy, and/or immunotherapy for malignant or autoimmune diseases
A hemoglobin < 10 g/dL and ≥ 8 g/dL
History of transfusions < 10 RBC units and must not have been RBC transfusion dependent
300 ng/mL < serum ferritin < 1,500 ng/mL (Values within 10% difference above 1500 ng/ml or 10% difference below 300 ng/ml could have been accepted at the investigator's discretion.

Show all 9Show fewer

Endogenous erythropoietin levels < 500 units/L
Serum creatinine ≤ 1.5 times upper limit of normal (ULN)
Creatinine clearance above the concentration limit in locally approved prescribing information (PI). Patients with creatinine clearance between 40 and less than 60 mL/min, who did not present with additional risk factors that might impair renal function, were eligible at the discretion of the investigator

Who cannot join

Patients who had MDS with isolated del(5q)
Patients who had received prior EPO treatment or other recombinant growth factors regardless of the outcome (Patient who had received prior EPO treatment or other recombinant growth factors for less than 4 weeks and not within 3 months before screening without a documented response are allowed)
Patients who had received steroids or immunosuppressive therapy for the improvement of hematological parameters (stable steroid treatment for adrenal failure or chronic medical conditions, and intermittent dexamethasone as antiemetics were allowed).
B12 and folate deficient patients with and without clinical symptoms (patients were rescreened after successful therapy of B12 and folate deficiency)
Uncontrolled seizures or uncontrolled hypertension

Trial locations and contacts

30 sites in 10 countries.

Country

City, postcode or hospital

30 sites in 10 countries

China6 sites

Novartis Investigative Site
Beijing, Beijing Municipality, 100730
Novartis Investigative Site
Chengdu, Sichuan, 610041
Novartis Investigative Site
Guangzhou, Guangdong, 51000
Novartis Investigative Site
Hangzhou, Zhejiang, 310003
Novartis Investigative Site
Nanjing, Jiangsu
Novartis Investigative Site
Shanghai, 200025

Other trials in China

Germany5 sites

Novartis Investigative Site
Berlin, 12203
Novartis Investigative Site
Dresden, 01307
Novartis Investigative Site
Düsseldorf, 40225
Novartis Investigative Site
Lütten Klein, 18107
Novartis Investigative Site
Würzburg, 97080

Other trials in Germany

Sweden4 sites

Novartis Investigative Site
Gothenburg, 413 45
Novartis Investigative Site
Linköping, SE-581 85
Novartis Investigative Site
Luleå, SE 971 80
Novartis Investigative Site
Stockholm, SE-141 86

Other trials in Sweden

Canada3 sites

Novartis Investigative Site
Hamilton, Ontario, L8V 5C2
Novartis Investigative Site
Toronto, Ontario, M4N 3M5
Novartis Investigative Site
Vancouver, British Columbia, V6Z1Y6

Other trials in Canada

Italy3 sites

Novartis Investigative Site
Cagliari, CA, 09126
Novartis Investigative Site
Reggio Calabria, RC, 89124
Novartis Investigative Site
Roma, RM, 00161

Other trials in Italy

Spain3 sites

Novartis Investigative Site
Badalona, Catalonia, 08916
Novartis Investigative Site
Girona, Catalonia, 17007
Novartis Investigative Site
L'Hospitalet de Llobregat, Catalonia, 08907

Other trials in Spain

Algeria2 sites

Novartis Investigative Site
Oran, 31000
Novartis Investigative Site
Sidi Bel Abbes, 22000

Other trials in Algeria

Argentina2 sites

Novartis Investigative Site
CABA, Buenos Aires, C1425DND
Novartis Investigative Site
La Plata, Buenos Aires, B1900AWT

Other trials in Argentina

South Korea1 site

Novartis Investigative Site
Seoul, Korea, 06351

Other trials in South Korea

United Kingdom1 site

Novartis Investigative Site
Oldham, Lancashire, OL1 2JH

Other trials in United Kingdom

About this study

Official title: An Open-label, Phase II, Randomized, Pilot Study to Assess the Effect in Term of Erythroid Improvement of Deferasirox Combined With Erythropoietin Compared to Erythropoietin Alone in Patients With low-and Int-1-risk Myelodysplastic Syndrome.

The primary purpose of this trial was is to assess the effect of treatment with deferasirox combined with erythropoietin vs. erythropoietin alone on erythropoiesis in patients with low- and int-1-risk myelodysplastic syndrome. The addition of deferasirox to erythropoietin can lead to a potential synergism with the reduction of reactive oxygen species, through both the NF-kB pathway and the control of free toxic iron. This may create a better environment in the bone marrow for a better response with erythropoietin.

This study was designed to test in a prospective way the combination of deferasirox with erythropoietin in terms of their effect on hematopoiesis.

Read the detailed description

This study did not meet the original enrollment objective of 60 patients and was terminated without extending enrollment past original planned LPFV of 31-Oct-2016.

Conditions: Low and Int 1-risk Myelodysplastic Syndrome
Being tested: Deferasirox DFX, DT, Erythropoietin alpha, Deferasirox DFX, FCT
Keywords: myelodysplastic syndrome, MDS, myelodysplasia, blood disorder, cytopenias, low blood counts, progressive bone marrow failure, adult, ICL570, deferasirox, erythropoietin, erythropoiesis, NF-kB pathway, hematopoiesis.
Medical subject headings: Myelodysplastic Syndromes, Anemia, Refractory, with Excess of Blasts, Hematologic Diseases, Cytopenia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Deferasirox DFX, DTDrug
provided as dispersible tablets for oral use in 125 and 250, 500 mg
Erythropoietin alphaDrug
Deferasirox DFX, FCTDrug
provided as film-coated tablet for oral use in 90, 180, 360 mg strengths

Study groups in NCT01868477
Study group	Role	What they receive
Erythropoietin alpha	Experimental	Patients will receive erythropoietin 40,000 units/week. If after 4 weeks erythroid improvement is inadequate, dose will be escalated to 60,000 units/week. If after 12 weeks of treatment, erythroid improvement in inadequate, patients will be switched to the combination arm. At any time when erythroid response is achieved, erythropoietin treatment will be stopped until end of study. Receives: Drug: Erythropoietin alpha
Deferasirox + Erythropoietin alpha	Experimental	Patients will receive deferasirox dispersible tablet (DT) 10 mg/kg/day or deferasirox film-coated tablet (FCT) 7 mg/kg/day in combination with erythropoietin 40,000 units/week. If after 4 weeks erythroid improvement is inadequate, erythropoietin dose will be escalated to 60,000 units/week. If after 12 weeks of treatment, erythroid improvement in inadequate, patients will be discontinued from the study. At any time when erythroid response is achieved, erythropoietin treatment will be stopped until end of study. Patients will continue deferasirox treatment. Receives: Drug: Deferasirox DFX, DT; Drug: Deferasirox DFX, FCT

What is being measured

The primary outcome is the main question the study is designed to answer.

Difference in Percentage of Patients Achieving Erythroid Response Within 12 Weeks, by Treatment Group (Full Analysis Set)
Difference in percentage of patients achieving an erythroid response within 12 weeks of treatment between the two arms according to modified IWG 2006 criteria increase in hemoglobin (Hb) ≥ 1.5 g/dL. Erythroid response is defined as the increase in Hb from baseline ≥ 1.5 g/dL. Patients achieving erythroid response at least once within 12 weeks were considered responders
Measured: Baseline up to 12 weeks

Secondary outcomes (12)

Absolute Change From Baseline to Post-baseline Value for Hemoglobin(g/dL)(Full Analysis Set)
Hematological response criteria defined as: Erythroid response: hemoglobin (Hb) increase from baseline >= 1.5 g/dL (baseline < 11 g/dL), neutrophil response: increase from baseline >= 100% and increase > 0.5 × 10^9/L (baseline <1 × 10^9/L), platelet response: increase from baseline >= 30 × 10^9/L (baseline <100 × 10^9/L) according to modified IWG 2006 criteria
Measured: Baseline up to 24 weeks
Summary of Hematologic Improvement in Patients Randomized to EPO+DFX and EPO Alone, Within 24 Weeks of Treatment (Full Analysis Set)
Percentage of participants achieving an hematologic improvement defined as: neutrophil improvement: increase from baseline >0.5 × 10^9/L (baseline = 1.0 × 10^9/L ), platelet improvement: increase from baseline ≥ 30 × 10^9/L (baseline = 100 × 10^9/L), hemoglobin improvement: Hb increase from baseline ≥ 1 g/dL (baseline<11 g/dL)
Measured: Baseline up to 24 weeks
Absolute Change in Hemoglobin Values up to 24 Weeks
Absolute change in hemoglobin values for patients showing improvement: Hemoglobin improvement Hb increase from baseline ≥ 1 g/dL (baseline<11 g/dL)
Measured: Baseline up to 24 weeks
Absolute Change in Platelets and Neutrophil Levels up to 24 Weeks
Absolute change in platelets and neutrophil levels for participants showing improvement: neutrophil improvement: increase from baseline >0.5 × 10^9/L (baseline = 1.0 × 10^9/L ), platelet improvement: increase from baseline ≥ 30 × 10^9/L (baseline = 100 × 10^9/L)
Measured: Baseline up to 24 weeks
Summary of Erythroid Response in Participants Randomized to EPO Alone at Baseline and Switched to EPO+DFX After 12 Weeks of Treatment (Full Analysis Set)
Erythroid response: hemoglobin increase from baseline > = 1.5 g/dL (baseline <11 g/dL)
Measured: Week 13 up to 24 weeks
Summary of Erythroid Response Within 24 Weeks in Participants Randomized to EPO at Baseline and Not Switched to EPO+DFX After 12 Weeks of Treatment (Full Analysis Set)
Erythroid response: hemoglobin increase from baseline > = 1.5 g/dL (baseline <11 g/dL). Percentages are based on N. Confidence intervals are calculated using Clopper-Pearson method. Hemoglobin value is at time of first response
Measured: baseline up to 24 weeks
Absolute Change in Serum Ferritin up to 24 Weeks for Erythropoietin Alpha Arm (Full Analysis Set)
Absolute change in serum ferritin from baseline
Measured: Baseline up to 24 weeks
Absolute Change in Serum Ferritin up to 24 Weeks for Deferasirox + Erythropoietin Alpha Arm (Full Analysis Set)
Absolute change in serum ferritin from baseline
Measured: Baseline up to 24 weeks
Absolute Change in Serum Ferritin up to 24 Weeks for EPO+DFX at 12 Weeks Arm (Full Analysis Set)
Absolute change in serum ferritin from baseline
Measured: Baseline up 24 weeks
Absolute Change in Hemoglobin (Hb) From Baseline for Erythropoietin Alpha Arm (Full Analysis Set)
This analysis included patients randomized either to EPO or DFX+EPO at baseline as well as patients who did not have erythroid response at week 12 in the EPO group and switched to combination therapy.
Measured: Baseline up to 24 weeks
Absolute Change in Hemoglobin (Hb) From Baseline for Deferasirox + Erythropoietin Alpha Arm (Full Analysis Set)
This analysis included patients randomized either to EPO or DFX+EPO at baseline as well as patients who did not have erythroid response at week 12 in the EPO group and switched to combination therapy.
Measured: Baseline up to 24 weeks
Absolute Change in Hemoglobin (Hb) From Baseline for EPO+DFX at 12 Weeks Arm (Full Analysis Set)
This analysis included patients randomized either to EPO or DFX+EPO at baseline as well as patients who did not have erythroid response at week 12 in the EPO group and switched to combination therapy. The time-course of Hb and its absolute changes from baseline was summarized by descriptive statistics by visit and erythroid response. Patients randomized to EPO and not switching after 12 weeks to EPO+DFX would consist of only responders.
Measured: Baseline up to 24 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

28 Jan 20145 Apr 2017

First posted4 Jun 2013 (estimated)
Study start28 Jan 2014
Primary completion22 Mar 2017
Study completion5 Apr 2017

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.
Other trials for Low and Int 1-risk Myelodysplastic SyndromeStudies of the same condition, open and under way.
More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
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 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 28 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01868477
Sponsor's protocol ID: CICL670A2421
Status last verified: Oct 2018
First submitted: 30 May 2013
Last updated: 31 Oct 2018
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Study at a glance

From the registry record, last updated by the sponsor on 31 Oct 2018. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open-label, Phase II, Randomized, Pilot Study to Assess the Effect in Term of Erythroid Improvement of Deferasirox Combined With Erythropoietin Compared to Erythropoietin Alone in Patients With low-and Int-1-risk Myelodysplastic Syndrome.
ClinicalTrials.gov ID: NCT01868477
Acronym / sponsor ID: CICL670A2421
Conditions: Low and Int 1-risk Myelodysplastic Syndrome
Keywords: myelodysplastic syndrome, MDS, myelodysplasia, blood disorder, cytopenias, low blood counts, progressive bone marrow failure, adult
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 28 participants (actual)
Lead sponsor: Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: 28 Jan 2014 (actual)
Primary completion: 22 Mar 2017 (actual)
Study completion: 5 Apr 2017 (actual)
First posted: 4 Jun 2013 (estimated)
Results: Posted 31 Oct 2018

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

This study was designed to test in a prospective way the combination of deferasirox with erythropoietin in terms of their effect on hematopoiesis.

Detailed description

This study did not meet the original enrollment objective of 60 patients and was terminated without extending enrollment past original planned LPFV of 31-Oct-2016.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Erythropoietin alpha	Experimental	Drug: Erythropoietin alphaPatients will receive erythropoietin 40,000 units/week. If after 4 weeks erythroid improvement is inadequate, dose will be escalated to 60,000 units/week. If after 12 weeks of treatment, erythroid improvement in inadequate, patients will be switched to the combination arm. At any time when erythroid response is achieved, erythropoietin treatment will be stopped until end of study.
Deferasirox + Erythropoietin alpha	Experimental	Drug: Deferasirox DFX, DT, Drug: Deferasirox DFX, FCTPatients will receive deferasirox dispersible tablet (DT) 10 mg/kg/day or deferasirox film-coated tablet (FCT) 7 mg/kg/day in combination with erythropoietin 40,000 units/week. If after 4 weeks erythroid improvement is inadequate, erythropoietin dose will be escalated to 60,000 units/week. If after 12 weeks of treatment, erythroid improvement in inadequate, patients will be discontinued from the study. At any time when erythroid response is achieved, erythropoietin treatment will be stopped until end of study. Patients will continue deferasirox treatment.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (9 listed)

Patients who had low- and Int-1-risk myelodysplastic syndrome
Documented diagnosis of the following:
Myelodysplastic syndrome that lasted ≥ 3 months and < 3 years Disease must not have been secondary to treatment with radiotherapy, chemotherapy, and/or immunotherapy for malignant or autoimmune diseases
A hemoglobin < 10 g/dL and ≥ 8 g/dL
History of transfusions < 10 RBC units and must not have been RBC transfusion dependent

Key exclusion criteria (5 listed)

Patients who had MDS with isolated del(5q)
Patients who had received prior EPO treatment or other recombinant growth factors regardless of the outcome (Patient who had received prior EPO treatment or other recombinant growth factors for less than 4 weeks and not within 3 months before screening without a documented response are allowed)
Patients who had received steroids or immunosuppressive therapy for the improvement of hematological parameters (stable steroid treatment for adrenal failure or chronic medical conditions, and intermittent dexamethasone as antiemetics were allowed).
B12 and folate deficient patients with and without clinical symptoms (patients were rescreened after successful therapy of B12 and folate deficiency)
Uncontrolled seizures or uncontrolled hypertension

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
Deferasirox DFX, DTprovided as dispersible tablets for oral use in 125 and 250, 500 mg	Drug	Deferasirox + Erythropoietin alpha
Erythropoietin alpha	Drug	Erythropoietin alpha
Deferasirox DFX, FCTprovided as film-coated tablet for oral use in 90, 180, 360 mg strengths	Drug	Deferasirox + Erythropoietin alpha

Primary outcome

Difference in Percentage of Patients Achieving Erythroid Response Within 12 Weeks, by Treatment Group (Full Analysis Set)
Time frame: Baseline up to 12 weeks
Difference in percentage of patients achieving an erythroid response within 12 weeks of treatment between the two arms according to modified IWG 2006 criteria increase in hemoglobin (Hb) ≥ 1.5 g/dL. Erythroid response is defined as the increase in Hb from baseline ≥ 1.5 g/dL. Patients achieving erythroid response at least once within 12 weeks were considered responders

12 secondary outcomes

Absolute Change From Baseline to Post-baseline Value for Hemoglobin(g/dL)(Full Analysis Set)
Time frame: Baseline up to 24 weeks
Summary of Hematologic Improvement in Patients Randomized to EPO+DFX and EPO Alone, Within 24 Weeks of Treatment (Full Analysis Set)
Time frame: Baseline up to 24 weeks
Absolute Change in Hemoglobin Values up to 24 Weeks
Time frame: Baseline up to 24 weeks
Absolute Change in Platelets and Neutrophil Levels up to 24 Weeks
Time frame: Baseline up to 24 weeks
Summary of Erythroid Response in Participants Randomized to EPO Alone at Baseline and Switched to EPO+DFX After 12 Weeks of Treatment (Full Analysis Set)
Time frame: Week 13 up to 24 weeks
Summary of Erythroid Response Within 24 Weeks in Participants Randomized to EPO at Baseline and Not Switched to EPO+DFX After 12 Weeks of Treatment (Full Analysis Set)
Time frame: baseline up to 24 weeks
Absolute Change in Serum Ferritin up to 24 Weeks for Erythropoietin Alpha Arm (Full Analysis Set)
Time frame: Baseline up to 24 weeks
Absolute Change in Serum Ferritin up to 24 Weeks for Deferasirox + Erythropoietin Alpha Arm (Full Analysis Set)
Time frame: Baseline up to 24 weeks
Absolute Change in Serum Ferritin up to 24 Weeks for EPO+DFX at 12 Weeks Arm (Full Analysis Set)
Time frame: Baseline up 24 weeks
Absolute Change in Hemoglobin (Hb) From Baseline for Erythropoietin Alpha Arm (Full Analysis Set)
Time frame: Baseline up to 24 weeks
Absolute Change in Hemoglobin (Hb) From Baseline for Deferasirox + Erythropoietin Alpha Arm (Full Analysis Set)
Time frame: Baseline up to 24 weeks
Absolute Change in Hemoglobin (Hb) From Baseline for EPO+DFX at 12 Weeks Arm (Full Analysis Set)
Time frame: Baseline up to 24 weeks

Results and safety

Results posted to the registry on 31 Oct 2018, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Erythropoietin Alpha	12	8
Deferasirox + Erythropoietin Alpha	11	6

Primary outcome: Difference in Percentage of Patients Achieving Erythroid Response Within 12 Weeks, by Treatment Group (Full Analysis Set)

Time frame: Baseline up to 12 weeks. Number (95% Confidence Interval). Unit: percentage of participants

Measure	Erythropoietin Alpha n = 12	Deferasirox + Erythropoietin Alpha n = 11
Value	41.7 (15.2 to 72.3)	27.3 (6.02 to 61.0)

Erythropoietin Alpha vs Deferasirox + Erythropoietin Alpha: Mean Difference (Final Values): 14.4 (95% CI -24.0 to 48.16)

12 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse Events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All Adverse events are reported in this record from First Patient First Treatment until Last Patient Last Visit up to approximately 24 weeks. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
EPO A	1 of 7 (14.3%)	4 of 7 (57.1%)	0 of 7
EPO+DFX DT	1 of 10 (10%)	10 of 10 (100%)	0 of 10
EPO+DFX FCT	1 of 1 (100%)	1 of 1 (100%)	0 of 1
Switched to DFX+EPO After 12 Weeks	1 of 5 (20%)	5 of 5 (100%)	0 of 5

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 31 Oct 2018.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

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.

Novartis Pharmaceuticals study director

Novartis Pharmaceuticals

30 sites in 10 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 9 inclusion and 5 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: finished, as of the sponsor's last update on 31 Oct 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 30 sites in China, Germany, Sweden, Canada, Italy and 5 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. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 31 Oct 2018). 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?

Difference in Percentage of Patients Achieving Erythroid Response Within 12 Weeks, by Treatment Group (Full Analysis Set) (time frame: Baseline up to 12 weeks). 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 NCT01868477.

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 31 Oct 2018.