Chronic iron overload in patients cardiac iron overload and ß thalassemia MedDRA version: 9.1 Level: LLT Classification code 10043389 Term:
Conditions
Interventions
Trade Name: EXJADE
Product Name: EXJADE (Deferasirox)
Pharmaceutical Form: Dispersible tablet
INN or Proposed INN: Deferasirox
CAS Number: N/A
Current Sponsor code: N/A
Other descriptive name: N/A
Con
Sponsors
Novartis (Hellas) S.A.C.I
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Male or female patients with ß-thalassemia major, at least 18 years old, having given written consent to participate in the study. • Cardiac MRI T2* value ranging from 5 to 10 mg Fe/g dw will be included in the protocol. Study will evaluate the first 10 patients at 6 months, and if no safety signals are present, patients with LIC>7 mg Fe/g dw will be allowed to be included. • Prior iron chelation treatment with DFO, DFP, DFX or combination DFO-DFP. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: •Patients with symptoms of cardiac dysfunction symptoms • Patients with cardiac T2* MRI 10 ms. • Patients not responding to intensive iron chelation therapy regimens such i.v DFO 24 hr infusions or DFO-DFP combination. Non response being defined as patients who have not achieved an improvement in cardiac MRI T2* of at least 2 ms within the prior year or have had a deterioration in LVEF = 56% in the year prior to study start. • Patients with documented liver failure • Patients with serum creatinine > ULN or with significant proteinuria as indicated by a urinary protein/creatinine ratio = 1.0 in a non-first void urine sample at baseline. • Patients with ALT (SGPT) levels > 5 x ULN. • Patients with considerable impaired gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral deferasirox / ICL670 (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection. • History or clinical evidence of pancreatic injury or pancreatitis. • Patients with a known hypersensitivity to any of the study drugs or the drug’s excipients. • History of clinically relevant ocular and/or auditor toxicity related to iron chelation therapy. • Patients with psychiatric or addictive disorders which prevent them from giving their informed consent or undergoing any of the treatment options or patients unwilling or unable to comply with the protocol. • Patients with a known history of HIV seropositivity (Elisa or Western blot). • History of malignancy of any organ system, treated or untreated, within the past 5 years whether or not there is evidence of local recurrence or metastases, with the exception of localized basal cell carcinoma of the skin. • Female patients who are pregnant or breast feeding. • Female patients of reproductive potential not employing an effective method of birth control. Women of childbearing potential must have a negative serum pregnancy test = 48 hours prior to the study drugs. • Patients participating in another clinical trial or receiving an investigational drug. • History of non-compliance with medical regimens or patients who are considered potentially unreliable and/or not cooperative, unwilling or unable to comply with the protocol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate, over the 24 months of study treatment, the efficacy of: 1. Intensive chelation with once a day oral deferasirox (20-40mg/kg/day) plus parenteral infusion of DFO (40 mg/kg/day) for 3-4 days in patients with severe cardiac iron (cardiac T2* ranging from 5 to 10 ms. • To evaluate the improvement in cardiac function during the 24 months of study when patients are treated with intensive iron chelation therapy consisting deferasirox–DFO combination regimen and when the patients transition to deferasirox monotherapy. ;Primary end point(s): The primary efficacy endpoint will be treatment response (Complete Response, Partial Response, Stable Disease). The primary efficacy endpoint will be to evaluate: • The number of patients achieving a complete response (CR), defined as patients that stop intensive deferasirox -DFO treatment, at any time point during the 24 months of study, based on an improvement in the cardiac MRI T2* value being >10ms, and continue to be treated with deferasirox monotherapy without any further need for reverting back to intensive iron chelation treatment during the 24 months of study. • The number of patients achieving a partial response (PR), defined as patients that stop intensive deferasirox -DFO treatment at any time point during the 24 months study and transition to receive deferasirox onotherapy, but due to a deterioration in cardiac MRI T2* to a value 10ms during the 24 months of study. | — |
Countries
Greece
Outcome results
None listed