None listed
Conditions
Brief summary
This study is an open-label, non-randomized, dose-defining study of daily deferasirox in overweight or obese patients with type 2 diabetes (T2D). This study is designed to assess the feasibility (safety and tolerability) of deferasirox administered daily, based on the rate of Dose Limiting Toxicities (DLTs), i.e. pre-defined adverse events. A DLT is defined as an adverse event or abnormal laboratory value occurring at any time which is assessed as clinically relevant which is considered to be related to the study treatment, unrelated to disease, disease progression, inter-current illness, or concomitant medications, and warrants stopping or reducing the medication. Toxicities will be assessed using the NCI CTCAE, version 3.0.
Interventions
Open label study of deferasirox.
1 intervention arm - everyone will receive DFS.
This will start at 5mg/kg/day and increase monthly in 5mg/kg/day steps until efficacy, side effects or significantly decreased iron levels. Patients will only increase the dose if there is no efficacy, no significant side-effects and iron studies do not indicate lower overall iron status. The dose may be decreased if their are side effects, including down to 2.5mg/kg/day.
Maximum dose 20mg/kg. Maximum duration 6 mo
Open label study of deferasirox. 1 intervention arm - everyone will receive DFS. This will start at 5mg/kg/day and increase monthly in 5mg/kg/day steps until efficacy, side effects or significantly decreased iron levels. Patients will only increase the dose if there is no efficacy, no significant side-effects and iron studies do not indicate lower overall iron status. The dose may be decreased if their are side effects, including down to 2.5mg/kg/day. Maximum dose 20mg/kg. Maximum duration 6 months. Each person will (as above) go up in 5mg/kg steps. DFS is administered as an oral tablet. Adherence strategies include tablet counts of returned medication and lab tests for iron studies.
Sponsors
Westmead Hospital
Study design
Allocation
Non-randomised trial
Intervention model
Other
Primary purpose
Treatment
Masking
Open (masking not used)
Eligibility
Sex/Gender
All
Age
30 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
T2D diagnosed within the last 10 years, with an HbA1c of 7.5%-10%, inclusive. The patients may be treated with diet and exercise or with metformin alone, at a stable dose for the preceding 3 months. After 8 weeks, if their glucose control is not improving, they will be allowed to start other hypoglycaemic medications.
Exclusion criteria
Age <30 (less likely to be T2DM), or >75 years BMI <25kg/m2, or <23kg/m2 if of South East Asian or Subcontinental Asian ethnicity Body weight >120kg. Elevated serum creatinine outside the normal range or eGFR <60ml/min Anaemia, thrombocytopaenia, iron deficiency or known iron overload conditions Low serum ferritin (<150ng/ml for men or <100ng/ml for women) Any grade of known diabetic retinopathy above mild non-proliferative changes Because of the reported low incidence of cataracts, all patients must have a diabetic eye screen within the preceding year and patients with cataracts will be excluded Microalbuminuria or macroalbuminuria (confirmed on repeat testing), excluded due to the reported rare side effect of Fanconi syndrome Significant proteinuria (confirmed on repeat testing). Defined as a urinary protein:creatinine ratio of >1mg/mg in a mid-stream sample Unstable angina, congestive cardiac failure, stable angina with onset <500m of walking or claudication with onset distance <500m, all due to the small risk of inducing anaemia Pregnancy, breast-feeding or lack of reliable contraception in women of child-bearing age Known malignancy Liver function tests >twice the upper limit of the normal reference range Inability to give informed consent Evidence of severe or uncontrolled systemic disease or any concurrent condition which in the Investigator’s opinion makes it undesirable for the patient to participate in the study, or which would jeopardise compliance with the protocol. Medical co-morbidities that have the potential to be exacerbated by or contra-indicate treatment with deferasirox
Outcome results
None listed