Hemochromatosis Clinical Trial
Official title:
Retrospective and Prospective Multicenter Study Using Deferiprone (L1) Alone or in Combination With Desferrioxamine for the Treatment of Iron Overload in Transfusion-dependent Patients
Verified date | December 2011 |
Source | Lipomed |
Contact | n/a |
Is FDA regulated | No |
Health authority | Switzerland: Swissmedic |
Study type | Interventional |
Systematical (retro- and prospective) investigation of the long-term safety (toxicity assessment according to CTCAE v3.0) and efficacy of deferiprone either given alone or in combination with desferrioxamine
Status | Completed |
Enrollment | 24 |
Est. completion date | May 2011 |
Est. primary completion date | May 2011 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | 4 Years and older |
Eligibility |
Inclusion Criteria: - Iron overloaded male or female patients with primary or secondary hemochromatosis - Age: 4 years and older - Patients with desferrioxamine toxicity or allergy (e.g. visual or hearing defects, bone abnormalities, reactions at injection site) - Patients unable or unwilling to comply satisfactorily with regular desferrioxamine administration on 5-7 days/week - Combination treatment: patients not sufficiently chelated with desferrioxamine or deferiprone monotherapy - Patients must be willing to undergo routine screening including medical history, physical examination and hematology, biochemistry and other laboratory tests - Written informed consent Exclusion Criteria: - Children under 4 years of age - Female and male of reproductive age, sexually active but not taking adequate contraceptive precaution - Woman who are pregnant or breast-feeding - Patients with HIV - Patients with active hepatitis requiring treatment - Patients with severe hepatic failure, cirrhosis - Patients with neutropenia (neutrophils less than 1.5 exp9/l, MDS: less than 0.5 exp9/l) - Patients with thrombocytopenia (platelets less than 100 exp9/l, MDS: less than 20 exp9/l) - Patients with decompensated heart failure (LVEF less than 40% or patients under continuous cardiac medication) - Patients with severe renal failure |
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
Switzerland | Cantonal Hospital | Aarau | Aargau |
Switzerland | Cantonal Hospital, Children's Clinic | Aarau | Aargau |
Switzerland | Private practice | Arzo | Ticino |
Switzerland | Private children's practice | Bern | |
Switzerland | Private children's practice | Brig | Wallis |
Switzerland | Cantonal Hospital Graubünden | Chur | Graubünden |
Switzerland | Private practice | Lugano | Ticino |
Switzerland | Regional Hospital | Lugano | Ticino |
Switzerland | Private practice | Oerlikon | Zurich |
Switzerland | Private practice | Riva San Vitale | Ticino |
Switzerland | Children's Hospital of Eastern Switzerland | St. Gallen | |
Switzerland | University Children's Hospital | Zurich | |
Switzerland | University Hospital | Zurich |
Lead Sponsor | Collaborator |
---|---|
Lipomed |
Switzerland,
Töndury P, Kontoghiorghes GJ, Ridolfi-Lüthy A, Hirt A, Hoffbrand AV, Lottenbach AM, Sonderegger T, Wagner HP. L1 (1,2-dimethyl-3-hydroxypyrid-4-one) for oral iron chelation in patients with beta-thalassaemia major. Br J Haematol. 1990 Dec;76(4):550-3. — View Citation
Töndury P, Zimmermann A, Nielsen P, Hirt A. Liver iron and fibrosis during long-term treatment with deferiprone in Swiss thalassaemic patients. Br J Haematol. 1998 Jun;101(3):413-5. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Liver Iron Concentration (LIC) by SQUID | Yearly | No | |
Primary | Long-term safety profile | Long-term | Yes | |
Secondary | Serum ferritin | At quarterly control visits | No | |
Secondary | Urinary Iron Excretion (UIE) | At six-monthly control visits | No | |
Secondary | Heart iron content (optional) by MRI T2* and MRI SIR | Yearly | No |
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