MedDRA version: 19.0 Level: LLT Classification code 10054661 Term: Thalassemia major System Organ Class: 100000004850 MedDRA version: 19.0 Level: LLT Classification code 10054660 Term: Thalassemia beta System Organ Class: 100000004850
Conditions
Sponsors
Novartis Pharma AG
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: •Completion of the planned 12-month core trial, (NCT00390858). •Female patients who have reached menarche and who were sexually active were to use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation. •Written informed consent obtained from the patient, and/or from the parent or legal guardian in accordance with the national legislation. Are the trial subjects under 18? yes Number of subjects for this age range: 40 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: •Pregnant or breast feeding patients •Patients with a history of non-compliance to medical regimens and patients who are considered by the investigator as potentially unreliable. Other protocol-defined exclusion criteria may apply.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: •Participants With Adverse Events by Primary System Organ Class (SOC) [ Time Frame: 4 year extension + core 1 year ] Safety parameters were measured by the number and type of adverse events (AEs). An adverse event is any untoward medical occurence in a patient administered a medicinal product that does not necessarily have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign ( for example, an abnormal laboratory finding), symptom or disease temporally associated with the use of the medicinal product, whether or not this is associated with the use of this medicinal product. •Change in Liver Iron Concentration (LIC) [ Time Frame: Baseline of Core Study to End of Extension Study, up to 5 years. ] Change in Liver Iron Concentration [LIC] measured by means of SQUID (Superconducting Quantum Interference Device). LIC is expressed in milligrams of iron per gram of liver dry weight (mg Fe/g dw) ;Secondary Objective: •Total Body Iron Elimination (TBIE) Rate (mg/kg/Day) Elimination (TBIE) Rate [mg/kg/Day] was calculated for each patient based on SQUID ( Superconducting Quantum Interference Device) results. •Relative Change in Serum Ferritin Level Serum levels were drawn at the baseline of the Core Study up to 18 months of the Extension Study. Levels were analyzed for serum ferritin measured in micrograms per Liter. Relative change (%) in serum ferritin level was assessed from Baseline to Extension 18 months. Relative Change = 1 - (Change in ferritin level from Baseline/Baseline level) x 100. •Relative Change in Serum Transferrin Level Serum Levels were drawn at Baseline of the Core Study and up to 18 months in the Extension Study. Serum was analyzed for transferrin levels measured as grams per Liter. Relative change (%) in serum transferrin level was assessed from Baseline to Extension 18 months. Relative Change = 1 - (Change in transferrin level from Baseline/Baseline level) x 100.;Primary e | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): •Total Body Iron Elimination (TBIE) Rate (mg/kg/Day) [ Time Frame: Baseline of Core Study to End of Extension Study, up to 5 years ] Total Iron Body Elimination (TBIE) Rate [mg/kg/Day] was calculated for each patient based on SQUID ( Superconducting Quantum Interference Device) results. •Relative Change in Serum Ferritin Level [ Time Frame: Baseline of Core Study to Extension 18 months, up to 2.5 years. ] Serum levels were drawn at the baseline of the Core Study up to 18 months of the Extension Study. Levels were analyzed for serum ferritin measured in micrograms per Liter. Relative change (%) in serum ferritin level was assessed from Baseline to Extension 18 months. Relative Change = 1 - (Change in ferritin level from Baseline/Baseline level) x 100. •Relative Change in Serum Transferrin Level [ Time Frame: Baseline of Core Study to Extension Study 18 months , up to 2.5 years ] Serum Levels were drawn at Baseline of the Core Study and up to 18 months in the Extension Study. Serum was analyzed for transferrin levels measured as grams per Liter. Relative change (%) in serum transferrin level was assessed from Baseline to Extension 18 months. Relative Change = 1 - (Change in transferrin level from Baseline/Baseline level) x 100. ;Timepoint(s) of evaluation of this end point: •Total Body Iron Elimination (TBIE) Rate (mg/kg/Day) : Baseline of Core Study to End of Extension Study, up to 5 years •Relative Change in Serum Ferritin Level: Baseline of Core Study to Extension 18 months, up to 2.5 years. •Relative Change in Serum Transferrin Level: Baseline of Core Study to Extension Study 18 months , up to 2.5 years | — |
Countries
France, Italy
Contacts
Public ContactClinical Trial Information Desk
Novartis Pharma AG
Outcome results
None listed