A multicenter open label phase II study to evaluate the safety and efficacy of deferasirox in combination with deferioxamine followed by transitioning to deferasirox monotherapy in ß-thalassemia patients with severe cardiac iron overload - DFX Autonomy
- Conditions
- Chronic iron overload in patients cardiac iron overload and ß thalassemiaMedDRA version: 9.1Level: LLTClassification code 10043389Term: <Manually entered code. Term in E.1.1>
- Registration Number
- EUCTR2009-018091-34-GR
- Lead Sponsor
- ovartis (Hellas) S.A.C.I
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 52
• 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 ms.
• LVEF = 56 % as determined by CMR.
• Patients with LIC > 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
•Patients with symptoms of cardiac dysfunction symptoms
• Patients with cardiac T2* MRI < 5 or > 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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method