WithdrawnPhase 2

Treatment of Iron Overload With Deferasirox (Exjade) in Hereditary Hemochromatosis and Myelodysplastic Syndrome

Stopped: Failure to recruit patients with hemochromatosis to the Deferasirox arm

Norway0Started 2013-05

Plain-language summary

Hypothesis: Deferasirox can be used as a therapeutic agent to deplete the liver, heart and bone marrow of excess iron in patients with iron overload caused by myelodysplastic syndrome (MDS) and hemochromatosis (HC. Assess the effect of new serum biomarkers (NTBI and hepcidin) and MRI as indicators of iron overload and their usefulness to monitor iron depletion treatment. Study the effect of iron overload and iron depletion on intracellular signal transduction, trace metals concentrations in serum and urine and markers of oxidative stress in blood cells and urine.

Who can participate

Age range

18 Years – 80 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Patients with hemochromatosis, aged \> 30 years, C282Y- homozygote, with serum-ferritin =/\> 1000 µg/L * Patients aged \> 18 years with verified low-risk or intermediate-1 risk of myelodysplastic syndrome, with normal cytogenetics and serum-ferritin \> 1500 µg/L, or with a transfusion history of =/\> red- blood-cell-transfusions. Exclusion Criteria: * Previous or current venesection * MDS patients eligible for hematopoietic stem cell transplantation * Subject complies with one or more of the following standard exclusion criteria for MRI examination; * If the patient has a pacemaker. * If the patient has a neurostimulator * If the patient has a "aneurismeclips" * If the patient has a foreign object in the eye. If yes, what object. * If the patient has a cochlea-/earimplant. * If the patient has a V/P shunt. * If the patient is claustrophobic. * If the patient has an artificial heart valve. * If the patient has known renal failure, eGFR \<30. * If the patient has or will have a liver transplantation. * Other: metal prostheses, metal implant * Presence of inflammation (CRP ≥ 5 mg/L) * Presence of proteinuria or creatinine \> 2 x UNL (Upper Normal Limit) * Estimated glomerular filtration rate (GFR) \< 60 mL/min * ALAT, ASAT, GT or ALP \> 2 x ULN ( Upper Normal Limit) * ALAT \> 90 U/L for women, ALAT \> 140 U/L for men * ASAT \> 70 U/L for women, ASAT \> 90 U/L for men * ALP \> 210 U/L for women and men * GT \> 90 U/L for women ≤ 40 …

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Changes from baseline in liver iron concentration (LIC) and heart iron concentration (HIC) determined by Magnetic Resonance Imaging (MRI), and in bone marrow iron content determined by microscopy after treatment with deferasirox.

Timeframe: 0, 6 and 12 months

Trial details

NCT IDNCT01892644

SponsorHaukeland University Hospital

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2017-01

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