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Trial record 3 of 8 for:    "Congenital Hypoplastic Anemia" | "Iron"

Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00235391
Recruitment Status : Completed
First Posted : October 10, 2005
Results First Posted : May 2, 2011
Last Update Posted : June 7, 2011
Sponsor:
Information provided by:
Novartis

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Thalassemia
Sickle Cell Disease
Diamond Blackfan Anemia
Myelofibrosis
Intervention Drug: Deferasirox
Enrollment 1683
Recruitment Details  
Pre-assignment Details  
Arm/Group Title 2 to < 6 Years 6 to < 12 Years 12 to < 16 Years 16 to < 50 Years 50 to < 65 Years ≥ 65 Years
Hide Arm/Group Description Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice. Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice. Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice. Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice. Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice. Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
Period Title: Overall Study
Started 97 200 172 1164 43 7
Deferasirox Treatment 97 200 172 1164 43 7
Completed 85 182 148 944 31 3
Not Completed 12 18 24 220 12 4
Reason Not Completed
Adverse Event             4             5             10             72             5             2
Abnormal laboratory value(s)             3             5             4             27             1             2
Unsatisfactory therapeutic effect             0             2             2             17             0             0
Condition no longer requires treatment             3             0             0             11             1             0
Protocol deviation             0             0             1             11             0             0
Withdrawal by Subject             2             3             3             61             5             0
Lost to Follow-up             0             3             3             16             0             0
Administrative problems             0             0             0             1             0             0
Death             0             0             1             4             0             0
Arm/Group Title 2 to < 6 Years 6 to < 12 Years 12 to < 16 Years 16 to < 50 Years 50 to < 65 Years ≥ 65 Years Total
Hide Arm/Group Description Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice. Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice. Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice. Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice. Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice. Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice. Total of all reporting groups
Overall Number of Baseline Participants 97 200 172 1164 43 7 1683
Hide Baseline Analysis Population Description
[Not Specified]
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 97 participants 200 participants 172 participants 1164 participants 43 participants 7 participants 1683 participants
3.48  (1.13) 8.52  (1.64) 13.48  (1.20) 28.91  (8.06) 54.58  (3.92) 70.14  (3.98) 24.27  (12.63)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 97 participants 200 participants 172 participants 1164 participants 43 participants 7 participants 1683 participants
Female
44
  45.4%
101
  50.5%
81
  47.1%
633
  54.4%
32
  74.4%
5
  71.4%
896
  53.2%
Male
53
  54.6%
99
  49.5%
91
  52.9%
531
  45.6%
11
  25.6%
2
  28.6%
787
  46.8%
Baseline Disease Characteristics   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 97 participants 200 participants 172 participants 1164 participants 43 participants 7 participants 1683 participants
Beta-Thalassemia Major 74 118 114 905 10 0 1221
Beta-Thalassemia Intermedia 1 22 16 94 20 3 156
Sickle Cell Disease 8 41 31 93 2 1 176
Diamond-Blackfan Anemia 4 7 8 24 0 0 43
Other Diseases 10 12 3 48 11 3 87
[1]
Measure Description: The number of participants with each disease category.
Prior Chelation Drug Therapy   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 97 participants 200 participants 172 participants 1164 participants 43 participants 7 participants 1683 participants
Deferoxamine 80 167 137 745 31 5 1165
Deferiprone 1 8 11 146 6 1 173
Deferoxamine and Deferiprone 2 16 21 269 6 0 314
Other Chelation Drug 6 6 3 4 0 0 19
Prior Chelatation Drug Information Missing 8 3 0 0 0 1 12
[1]
Measure Description: The number of participants for each chelation drug category.
Reason for inadequate prior chelation therapy   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 97 participants 200 participants 172 participants 1164 participants 43 participants 7 participants 1683 participants
Therapy non-compliance 57 125 106 659 25 2 974
Therapy contraindication 1 7 6 29 1 1 45
Therapy unacceptable toxicity 11 29 22 131 6 1 200
Therapy poor response 20 34 38 267 8 0 367
Therapy unacceptable discomfort 2 2 0 78 3 2 87
Reason for inadequate therapy information missing 6 3 0 0 0 1 10
[1]
Measure Description: The number of participants for each reason for inadequate prior chelation therapy category.
1.Primary Outcome
Title Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events
Hide Description Safety as assessed by the number of participants with death, serious adverse events (SAE), and/or Adverse Events (AEs) leading to study drug interruption or discontinuation. Note: only treatment emergent AEs are summarized.
Time Frame Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
The safety population, comprising all participants who received at least one dose of deferasirox during the study, was used in the analyses.
Arm/Group Title 2 to < 6 Years 6 to < 12 Years 12 to < 16 Years 16 to < 50 Years 50 to < 65 Years ≥ 65 Years
Hide Arm/Group Description:
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
Overall Number of Participants Analyzed 97 200 172 1164 43 7
Measure Type: Number
Unit of Measure: Participants
Number of deaths 0 0 1 4 0 0
Non-fatal SAEs 10 22 27 129 4 2
AEs leading to discontinuation 4 5 10 75 5 2
AEs leading dose adjustment/temporary interruption 15 31 28 209 11 2
2.Secondary Outcome
Title The Change in Serum Ferritin Values From Baseline Through Completion of the Study
Hide Description The number of participants with Improvement, No Change or Worsening in Serum ferritin category levels at the end of the study compared to baseline. Serum ferritin levels in µg/L were divided into to 6 categories: (<1000), (1000-<2500), (2500-<4000), (4000-<5500), (5500-<7000) and (>=7000). Improvement was defined as a shift to a lower category at the end of study compared to the category at baseline. Worsening was defined as a shift to a higher category at the end of the study compared to the category at baseline. No change was no change in category at end of study from baseline.
Time Frame Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety population defined as all participants who received at least one dose of study drug. This analysis did not include participants with unknown status at baseline and/or at the end of the study.
Arm/Group Title 2 to < 6 Years 6 to < 12 Years 12 to < 16 Years 16 to < 50 Years 50 to < 65 Years ≥ 65 Years
Hide Arm/Group Description:
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
Overall Number of Participants Analyzed 97 200 172 1164 43 7
Measure Type: Number
Unit of Measure: Participants
Improvement 17 27 26 206 4 2
No Change 63 106 87 570 31 3
Worsening 16 63 55 341 7 1
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title All Participants
Hide Arm/Group Description Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
All-Cause Mortality
All Participants
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
All Participants
Affected / at Risk (%)
Total   194/1683 (11.53%) 
Blood and lymphatic system disorders   
Acute chest syndrome  1  2/1683 (0.12%) 
Anaemia  1  2/1683 (0.12%) 
Anaemia haemolytic autoimmune  1  2/1683 (0.12%) 
Coombs positive haemolytic anaemia  1  1/1683 (0.06%) 
Haemolytic anaemia  1  1/1683 (0.06%) 
Hypersplenism  1  3/1683 (0.18%) 
Intravascular haemolysis  1  1/1683 (0.06%) 
Leukopenia  1  1/1683 (0.06%) 
Neutropenia  1  2/1683 (0.12%) 
Pancytopenia  1  1/1683 (0.06%) 
Reticulocytopenia  1  1/1683 (0.06%) 
Splenomegaly  1  4/1683 (0.24%) 
Cardiac disorders   
Arrhythmia  1  1/1683 (0.06%) 
Atrial fibrillation  1  2/1683 (0.12%) 
Cardiac failure  1  6/1683 (0.36%) 
Cardiac failure acute  1  1/1683 (0.06%) 
Cardiac failure congestive  1  2/1683 (0.12%) 
Congestive cardiomyopathy  1  1/1683 (0.06%) 
Myopericarditis  1  1/1683 (0.06%) 
Pericardial effusion  1  1/1683 (0.06%) 
Tachycardia  1  1/1683 (0.06%) 
Ventricular dysfunction  1  1/1683 (0.06%) 
Congenital, familial and genetic disorders   
Hip dysplasia  1  1/1683 (0.06%) 
Sickle cell anaemia  1  2/1683 (0.12%) 
Sickle cell anaemia with crisis  1  27/1683 (1.60%) 
Thalassaemia  1  1/1683 (0.06%) 
Thalassaemia beta  1  2/1683 (0.12%) 
Ear and labyrinth disorders   
Deafness unilateral  1  1/1683 (0.06%) 
Middle ear effusion  1  1/1683 (0.06%) 
Eye disorders   
Retinopathy  1  1/1683 (0.06%) 
Gastrointestinal disorders   
Abdominal discomfort  1  1/1683 (0.06%) 
Abdominal pain  1  3/1683 (0.18%) 
Abdominal pain upper  1  2/1683 (0.12%) 
Colitis  1  1/1683 (0.06%) 
Constipation  1  1/1683 (0.06%) 
Crohn's disease  1  1/1683 (0.06%) 
Diarrhoea  1  1/1683 (0.06%) 
Gastritis  1  4/1683 (0.24%) 
Gastrointestinal haemorrhage  1  1/1683 (0.06%) 
Haemorrhoids  1  1/1683 (0.06%) 
Pancreatitis  1  1/1683 (0.06%) 
Peritonitis  1  1/1683 (0.06%) 
Proctitis  1  1/1683 (0.06%) 
Vomiting  1  1/1683 (0.06%) 
General disorders   
Catheter thrombosis  1  1/1683 (0.06%) 
Death  1  1/1683 (0.06%) 
Local swelling  1  1/1683 (0.06%) 
Malaise  1  3/1683 (0.18%) 
Non-cardiac chest pain  1  5/1683 (0.30%) 
Pain  1  3/1683 (0.18%) 
Pyrexia  1  9/1683 (0.53%) 
Hepatobiliary disorders   
Biliary colic  1  1/1683 (0.06%) 
Cholelithiasis  1  6/1683 (0.36%) 
Hepatitis  1  2/1683 (0.12%) 
Hyperbilirubinaemia  1  1/1683 (0.06%) 
Liver disorder  1  1/1683 (0.06%) 
Immune system disorders   
Hypersensitivity  1  1/1683 (0.06%) 
Infections and infestations   
Abdominal infection  1  1/1683 (0.06%) 
Acute tonsillitis  1  2/1683 (0.12%) 
Appendicitis  1  1/1683 (0.06%) 
Bacterial infection  1  1/1683 (0.06%) 
Bronchitis  1  2/1683 (0.12%) 
Bronchopneumonia  1  1/1683 (0.06%) 
Catheter related infection  1  1/1683 (0.06%) 
Catheter site infection  1  1/1683 (0.06%) 
Cellulitis  1  2/1683 (0.12%) 
Diarrhoea infectious  1  1/1683 (0.06%) 
Ear infection  1  1/1683 (0.06%) 
Epstein-Barr virus infection  1  3/1683 (0.18%) 
Escherichia bacteraemia  1  1/1683 (0.06%) 
Gastroenteritis  1  3/1683 (0.18%) 
Gastrointestinal infection  1  1/1683 (0.06%) 
Hepatitis C  1  1/1683 (0.06%) 
Influenza  1  1/1683 (0.06%) 
Liver abscess  1  1/1683 (0.06%) 
Necrotising fasciitis  1  1/1683 (0.06%) 
Osteomyelitis  1  2/1683 (0.12%) 
Otitis media acute  1  1/1683 (0.06%) 
Parvovirus infection  1  1/1683 (0.06%) 
Perianal abscess  1  1/1683 (0.06%) 
Pharyngitis  1  1/1683 (0.06%) 
Pharyngitis streptococcal  1  1/1683 (0.06%) 
Pneumonia  1  4/1683 (0.24%) 
Pyelonephritis  1  1/1683 (0.06%) 
Respiratory tract infection  1  2/1683 (0.12%) 
Rhinitis  1  1/1683 (0.06%) 
Sepsis  1  1/1683 (0.06%) 
Tonsillitis  1  1/1683 (0.06%) 
Upper respiratory tract infection  1  1/1683 (0.06%) 
Viral infection  1  5/1683 (0.30%) 
Injury, poisoning and procedural complications   
Clavicle fracture  1  1/1683 (0.06%) 
Comminuted fracture  1  1/1683 (0.06%) 
Femur fracture  1  2/1683 (0.12%) 
Forearm fracture  1  1/1683 (0.06%) 
Haemolytic transfusion reaction  1  1/1683 (0.06%) 
Post-traumatic pain  1  1/1683 (0.06%) 
Road traffic accident  1  2/1683 (0.12%) 
Spinal fracture  1  1/1683 (0.06%) 
Suture rupture  1  1/1683 (0.06%) 
Thermal burn  1  1/1683 (0.06%) 
Tibia fracture  1  1/1683 (0.06%) 
Upper limb fracture  1  1/1683 (0.06%) 
Investigations   
Alanine aminotransferase increased  1  2/1683 (0.12%) 
Blood calcium increased  1  1/1683 (0.06%) 
Blood creatinine increased  1  1/1683 (0.06%) 
Cardiovascular evaluation  1  1/1683 (0.06%) 
Protein urine present  1  1/1683 (0.06%) 
Serum ferritin increased  1  1/1683 (0.06%) 
Transaminases increased  1  1/1683 (0.06%) 
Metabolism and nutrition disorders   
Hyperglycaemia  1  1/1683 (0.06%) 
Hypocalcaemia  1  1/1683 (0.06%) 
Hypoglycaemic seizure  1  1/1683 (0.06%) 
Musculoskeletal and connective tissue disorders   
Arthralgia  1  1/1683 (0.06%) 
Back pain  1  1/1683 (0.06%) 
Fistula  1  1/1683 (0.06%) 
Osteonecrosis  1  1/1683 (0.06%) 
Pain in extremity  1  1/1683 (0.06%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Benign neoplasm of thymus  1  1/1683 (0.06%) 
Hepatic neoplasm malignant recurrent  1  1/1683 (0.06%) 
Non-small cell lung cancer  1  1/1683 (0.06%) 
Nervous system disorders   
Convulsion  1  1/1683 (0.06%) 
Headache  1  2/1683 (0.12%) 
Migraine  1  1/1683 (0.06%) 
Paraesthesia  1  1/1683 (0.06%) 
Spinal cord compression  1  1/1683 (0.06%) 
Pregnancy, puerperium and perinatal conditions   
Pregnancy  1  5/1683 (0.30%) 
Psychiatric disorders   
Anxiety  1  1/1683 (0.06%) 
Depression  1  1/1683 (0.06%) 
Suicide attempt  1  1/1683 (0.06%) 
Renal and urinary disorders   
Calculus ureteric  1  1/1683 (0.06%) 
Nephrotic syndrome  1  1/1683 (0.06%) 
Renal colic  1  2/1683 (0.12%) 
Renal failure  1  2/1683 (0.12%) 
Urethral stenosis  1  1/1683 (0.06%) 
Urinary retention  1  1/1683 (0.06%) 
Reproductive system and breast disorders   
Haemorrhagic ovarian cyst  1  1/1683 (0.06%) 
Uterine disorder  1  1/1683 (0.06%) 
Respiratory, thoracic and mediastinal disorders   
Adenoidal hypertrophy  1  1/1683 (0.06%) 
Asthma  1  1/1683 (0.06%) 
Dyspnoea  1  1/1683 (0.06%) 
Epistaxis  1  1/1683 (0.06%) 
Hypoxia  1  1/1683 (0.06%) 
Pleural effusion  1  1/1683 (0.06%) 
Pneumonitis  1  1/1683 (0.06%) 
Pulmonary embolism  1  2/1683 (0.12%) 
Skin and subcutaneous tissue disorders   
Erythema multiforme  1  1/1683 (0.06%) 
Pigmentation disorder  1  1/1683 (0.06%) 
Rash  1  5/1683 (0.30%) 
Rash maculo-papular  1  2/1683 (0.12%) 
Swelling face  1  1/1683 (0.06%) 
Urticaria  1  1/1683 (0.06%) 
Surgical and medical procedures   
Uterine dilation and curettage  1  1/1683 (0.06%) 
Vascular disorders   
Deep vein thrombosis  1  1/1683 (0.06%) 
Hypotension  1  1/1683 (0.06%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
All Participants
Affected / at Risk (%)
Total   85/1683 (5.05%) 
Skin and subcutaneous tissue disorders   
Rash  1  85/1683 (5.05%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.
Results Point of Contact
Name/Title: Study Director
Organization: Novartis Pharmaceuticals
Phone: 862-778-8300
Responsible Party: External Affairs, Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00235391     History of Changes
Other Study ID Numbers: CICL670A2203
First Submitted: October 6, 2005
First Posted: October 10, 2005
Results First Submitted: December 17, 2010
Results First Posted: May 2, 2011
Last Update Posted: June 7, 2011