Phase 2 N=225 Randomized Double-blind Treatment

Myelodysplastic Syndromes (MDS) Event Free Survival With Iron Chelation Therapy Study

Myelodysplastic Syndromes

Bottom Line

View on ClinicalTrials.gov: NCT00940602 ↗

Enrolled (actual)

225

Serious AEs

52.7%

Results posted

Nov 2020

Primary outcome: Primary: Event-free Survival — 1440; 1091 days — p=0.015

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Deferasirox (Drug); Placebo (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Novartis Pharmaceuticals
Primary completion: Feb 2018

Outcome Measures

Outcome	Result	p-value
PRIMARY Event-free Survival	1440; 1091	0.015 sig
SECONDARY Percentage of Participants With Hematologic Improvement (HI) in Terms of Erythroid Response	39.6; 27.6	—
SECONDARY Overall Survival	1907; 1509	0.200
SECONDARY Percentage of Participants With Newly Occurring Hypothyroidism Compared to Baseline	5.4; 3.9	—
SECONDARY Percentage of Participants With Worsening Glucose Metabolism Compared to Baseline	18.1; 18.4	—
SECONDARY Time to Disease Progression	NA; NA	0.184
SECONDARY Time to First Occurrence of Serum Ferritin Level >2 Times the Baseline Value at Two Consecutive Assessments (at Least Two Weeks Apart)	NA; 592	<.001 sig
SECONDARY Time to at Least a 10% Increase From Baseline in Left Ventricular End-diastolic Internal (LVIDD) at Two Consecutive Assessments at Least Two Weeks Apart	NA; NA	0.303
SECONDARY Time to at Least a 10% Increase From Baseline in Left Ventricular Internal Systolic Diameter (LVISD) at Two Consecutive Assessments at Least Two Weeks Apart	1179.0; NA	0.389
SECONDARY Total Number of Infections Requiring Intravenous Antimicrobials	253; 111	—
SECONDARY Percentage of Participants With Major Gastrointestinal Bleeding	5.4; 3.9	—
SECONDARY Percentage of Participants With Significant Renal Dysfunction	0.7; 0	—
SECONDARY Percentage of Participants With Newly Occurring Moderate or Severe Neutropenia	27.7; 26.3	—
SECONDARY Percentage of Participants With Newly Occurring Severe Thrombocytopenia	10.1; 19.7	—
SECONDARY Time to Study Drug Discontinuation Due to an AE or Laboratory Abnormality	NA; 1022	0.232

Summary

This was a randomized, double-blind trial to evaluate deferasirox vs placebo in patients with myelodysplastic syndromes (low/int-1 risk) and transfusional iron overload .The trial was conducted in 17 countries, started in 2010 and ended in 2018.

Eligibility Criteria

Inclusion Criteria

Weigh between 35-135 kilograms
Low or int-1 risk MDS
Ferritin >1000 micrograms/liter at screening
History of transfusion of 15 to 75 Packed Red Blood Cells (PRBC) units
Anticipated to be transfused with at least 8 units of PRBCs annually during the study
Women of child-bearing potential using effective methods of contraception during dosing of study treatment

Exclusion Criteria

More than 6 months of cumulative ICT (such as daily deferasirox (Exjade®) or deferiprone or 5×/week deferoxamine)
More than 3 years since patient began receiving regular transfusions (2 units per 8 weeks or 4 units received in a 3 month period)
Significant proteinuria
History of hospitalization for congestive heart failure; other heart conditions as specified in the protocol
Systemic diseases which would prevent study treatment
Hepatitis B; Hepatitis C; HIV
Liver cirrhosis
Pregnant, or breast-feeding patients, or patients of child-bearing potential not employing an effective method of birth control
History of drug or alcohol abuse within the 12 months prior to enrollment

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00940602). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.