Last reviewed · How we verify

NCT02720536

Extended Evaluation of Deferasirox Film-coated Tablet (FCT) Formulation

Completed Phase 3 Results posted Last updated 3 March 2020
What this trial tests

Phase 3 trial testing Deferasirox in Chronic Iron Overload in 53 participants. Completed in 23 July 2019.

Timeline
16 August 2016
Primary endpoint
23 July 2019
23 July 2019

Quick facts

Lead sponsorNovartis Pharmaceuticals
PhasePhase 3
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment53
Start date16 August 2016
Primary completion23 July 2019
Estimated completion23 July 2019
Sites14 locations across Austria, Greece, Italy

Drugs / interventions tested

Conditions studied

Sponsor

Novartis Pharmaceuticals — full company profile →

Who can join

10 and older, any sex, with Chronic Iron Overload. 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.

Overview of Number of Participants With Adverse Events Primary · Baseline up to approximately 25 months

Numbers represent counts of participants within the categories. An adverse event (AE) was defined as treatment emergent if its onset date is on or after (≥) the first administration of study treatment within this study or events present prior to start of study treatment but increased in severity on or after (≥) the first administration of study treatment within this study but not later than 30 days after the last study treatment in this study

Adverse events (AEs)
GroupValue95% CI
Deferasirox52
Treatment related AEs
GroupValue95% CI
Deferasirox20
Severe adverse events
GroupValue95% CI
Deferasirox14
Treatment related severe adverse events
GroupValue95% CI
Deferasirox2
Serious adverse events (SAEs)
GroupValue95% CI
Deferasirox13
Treatment related SAEs
GroupValue95% CI
Deferasirox0
Fatal SAEs
GroupValue95% CI
Deferasirox1
Treatment related fatal SAEs
GroupValue95% CI
Deferasirox0
Change From Baseline Red Blood Cells (RBC) (10^12 Cells/L) at Month 6 and Month 12 Primary · Baseline, 6 and 12 months

The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline

Baseline
GroupValue95% CI
Baseline3.753± 0.4909
Month 63.733± 0.5016
Month 123.746± 0.5071
Post
GroupValue95% CI
BaselineNA± NA
Month 63.610± 0.5153
Month 123.607± 0.5423
Change
GroupValue95% CI
BaselineNA± NA
Month 6-0.124± 0.3892
Month 12-0.139± 0.4330
Change From Baseline White Blood Cells (WBC) (10^9 Cells/L) at Month 6 and Month 12 Primary · Baseline, 6 and 12 months

The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline

Baseline
GroupValue95% CI
Baseline9.336± 5.1383
Month 69.559± 5.6650
Month 129.100± 5.4667
Post
GroupValue95% CI
BaselineNA± NA
Month 69.090± 4.3712
Month 129.007± 4.4385
Change
GroupValue95% CI
BaselineNA± NA
Month 6-0.469± 4.0727
Month 12-0.093± 4.5792
Change From Baseline Platelets (10^9 Cells/L) at Month 6 and Month 12 Primary · Baseline, 6 and 12 months

The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline

Baseline
GroupValue95% CI
Baseline330.1± 188.81
Month 6336.5± 195.97
Month 12339.4± 196.41
Post
GroupValue95% CI
BaselineNA± NA
Month 6344.2± 190.43
Month 12361.9± 165.91
Change
GroupValue95% CI
BaselineNA± NA
Month 67.7± 112.98
Month 1222.4± 100.72
Change From Baseline Serum Creatinine (Umol/L) at Month 6 and Month 12 Primary · Baseline, 6 and 12 months

The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline

Baseline
GroupValue95% CI
Baseline55.1± 16.16
Month 654.4± 16.34
Month 1257.4± 16.91
Post
GroupValue95% CI
BaselineNA± NA
Month 662.0± 16.17
Month 1263.5± 16.78
Change
GroupValue95% CI
BaselineNA± NA
Month 67.6± 9.51
Month 126.1± 11.09
Change From Baseline Creatinine Clearance (mL/Min) at Month 6 and Month 12 Primary · Baseline, 6 and 12 months

The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline

Baseline
GroupValue95% CI
Baseline131.9± 51.32
Month 6131.8± 56.98
Month 12129.6± 50.30
Post
GroupValue95% CI
BaselineNA± NA
Month 6116.0± 48.71
Month 12119.8± 49.34
Change
GroupValue95% CI
BaselineNA± NA
Month 6-15.8± 35.12
Month 12-9.8± 32.74
Change From Baseline Alanine Aminotransferase/Serum Glutamic Pyruvic Transaminase (ALT/SGPT) (U/L) at Month 6 and Month 12 Primary · Baseline, 6 and 12 months

The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline

Baseline
GroupValue95% CI
Baseline37.4± 31.47
Month 639.4± 33.51
Month 1239.5± 33.32
Post
GroupValue95% CI
BaselineNA± NA
Month 628.9± 21.12
Month 1225.9± 19.08
Change
GroupValue95% CI
BaselineNA± NA
Month 6-10.5± 29.16
Month 12-13.6± 27.44
Change From Baseline Aspartate Aminotransferase/Serum Glutamic Oxaloacetic Transaminase (AST/SGOT) (U/L) at Month 6 and Month 12 Primary · Baseline, 6 and 12 months

The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline

Baseline
GroupValue95% CI
Baseline30.7± 24.40
Month 632.9± 26.48
Month 1233.7± 26.67
Post
GroupValue95% CI
BaselineNA± NA
Month 627.6± 18.81
Month 1225.2± 12.78
Change
GroupValue95% CI
BaselineNA± NA
Month 6-5.3± 21.03
Month 12-8.5± 19.18
Change From Baseline of Serum Ferritin Level (ug/L) at Month 6 and 12 Secondary · Baseline, 6 and 12 months

The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline. A negative change from baseline is regarded as an improvement in this study

Baseline
GroupValue95% CI
Baseline2523.51± 1746.087
Month 62614.12± 1781.287
Month 122542.76± 1904.087
Post
GroupValue95% CI
BaselineNA± NA
Month 62228.94± 1910.182
Month 121924.49± 1839.818
Change
GroupValue95% CI
BaselineNA± NA
Month 6-385.18± 1038.789
Month 12-618.26± 1054.150
Percentage Relative Change From Baseline of Serum Ferritin (%) at Month 6 and 12 Secondary · Baseline, 6 and 12 months

The percentage relative change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Percentage relative change = 100 × (\[Post - Baseline\] / Baseline). Percentage relative change is calculated for each patient individually and then overall descriptive summary statistics is obtained for subjects with a value at baseline and the particular time point. A negative percentage relative change from baseline is regarded as an improvement in this study

GroupValue95% CI
BaselineNA± NA
Month 6-18.61± 32.969
Month 12-29.08± 33.056

Adverse events — posted to ClinicalTrials.gov

Time frame: Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Deferasirox
Serious: 13/53 (25%)
Deaths: 1/53

Serious adverse events (26 terms)

ReactionSystemDeferasirox
Atrial fibrillationCardiac disorders
Cardiac failureCardiac disorders
GoitreEndocrine disorders
Biliary colicHepatobiliary disorders
CholecystitisHepatobiliary disorders
CholestasisHepatobiliary disorders
Hepatic failureHepatobiliary disorders
DiverticulitisInfections and infestations
Lower respiratory tract infectionInfections and infestations
Lymph gland infectionInfections and infestations
UrosepsisInfections and infestations
Femur fractureInjury, poisoning and procedural complications
FractureInjury, poisoning and procedural complications
Lumbar vertebral fractureInjury, poisoning and procedural complications
Rib fractureInjury, poisoning and procedural complications
Ulna fractureInjury, poisoning and procedural complications
Malignant melanomaNeoplasms benign, malignant and unspecified (incl cysts and polyps)
Ovarian adenomaNeoplasms benign, malignant and unspecified (incl cysts and polyps)
Papillary thyroid cancerNeoplasms benign, malignant and unspecified (incl cysts and polyps)
Abortion spontaneousPregnancy, puerperium and perinatal conditions
Device failureProduct Issues
Panic attackPsychiatric disorders
Calculus urinaryRenal and urinary disorders
HydronephrosisRenal and urinary disorders
Renal colicRenal and urinary disorders
Other adverse events (41 terms — click to expand)

ReactionSystemDeferasirox
DiarrhoeaGastrointestinal disorders
HeadacheNervous system disorders
PyrexiaGeneral disorders
NauseaGastrointestinal disorders
VomitingGastrointestinal disorders
CoughRespiratory, thoracic and mediastinal disorders
Abdominal pain upperGastrointestinal disorders
Abdominal painGastrointestinal disorders
AstheniaGeneral disorders
InfluenzaInfections and infestations
Oropharyngeal painRespiratory, thoracic and mediastinal disorders
RhinitisInfections and infestations
Urine protein/creatinine ratio increasedInvestigations
GastroenteritisInfections and infestations
PharyngitisInfections and infestations
Urinary tract infectionInfections and infestations
Musculoskeletal painMusculoskeletal and connective tissue disorders
GastritisGastrointestinal disorders
Influenza like illnessGeneral disorders
TonsillitisInfections and infestations
Back painMusculoskeletal and connective tissue disorders
Pain in extremityMusculoskeletal and connective tissue disorders
DyspepsiaGastrointestinal disorders
HypertransaminasaemiaHepatobiliary disorders
Ear infectionInfections and infestations
Respiratory tract infectionInfections and infestations
Upper respiratory tract infectionInfections and infestations
Blood creatinine increasedInvestigations
ProteinuriaRenal and urinary disorders
PalpitationsCardiac disorders
ConstipationGastrointestinal disorders
NasopharyngitisInfections and infestations
SinusitisInfections and infestations
ArthralgiaMusculoskeletal and connective tissue disorders
MyalgiaMusculoskeletal and connective tissue disorders
Neck painMusculoskeletal and connective tissue disorders
DysuriaRenal and urinary disorders
GlycosuriaRenal and urinary disorders
Renal colicRenal and urinary disorders
Productive coughRespiratory, thoracic and mediastinal disorders

Most-reported serious reactions: Atrial fibrillation, Cardiac failure, Goitre, Biliary colic, Cholecystitis, Cholestasis, Hepatic failure, Diverticulitis.

Data from ClinicalTrials.gov NCT02720536 adverse events section.

Sponsor's own description

Extend evaluation of deferasirox film-coated tablet (FCT) formulation

Publications & conference data

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

  1. Two-year long safety and efficacy of deferasirox film-coated tablets in patients with thalassemia or lower/intermediate risk MDS: phase 3 results from a subset of patients previously treated with deferasirox in the ECLIPSE study.
    Tartaglione I, Origa R, Kattamis A, Pfeilstöcker M, et al · · 2020 · cited 9× · PMID 32793403 · DOI 10.1186/s40164-020-00174-2
  2. Patient preference for deferasirox film-coated versus dispersible tablet formulation: a sequential-design phase 2 study in patients with thalassemia.
    Viprakasit V, Hamdy MM, Hassab HMA, Sherief LM, et al · · 2023 · cited 4× · PMID 37227493 · DOI 10.1007/s00277-023-05240-3

Verify or expand the search:

Other trials of Deferasirox

Trials testing the same drug.

Other Novartis Pharmaceuticals trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02720536.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing