Study Using Deferiprone Alone or in Combination With Desferrioxamine in Iron Overloaded Transfusion-dependent Patients
Retrospective and Prospective Multicenter Study Using Deferiprone (L1) Alone or in Combination With Desferrioxamine for the Treatment of Iron Overload in Transfusion-dependent Patients
1 other identifier
interventional
24
1 country
13
Brief Summary
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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Mar 2005
Longer than P75 for phase_2
13 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
March 1, 2005
CompletedFirst Submitted
Initial submission to the registry
July 6, 2006
CompletedFirst Posted
Study publicly available on registry
July 7, 2006
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 1, 2011
CompletedStudy Completion
Last participant's last visit for all outcomes
May 1, 2011
CompletedDecember 12, 2011
December 1, 2011
6.2 years
July 6, 2006
December 9, 2011
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Liver Iron Concentration (LIC) by SQUID
Yearly
Long-term safety profile
Long-term
Secondary Outcomes (3)
Serum ferritin
At quarterly control visits
Urinary Iron Excretion (UIE)
At six-monthly control visits
Heart iron content (optional) by MRI T2* and MRI SIR
Yearly
Study Arms (2)
Deferiprone (L1) monotherapy
EXPERIMENTALDeferiprone (L1) monotherapy
Combination therapy
EXPERIMENTALDeferiprone (L1) and desferrioxamine combination treatment
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Lipomedlead
Study Sites (13)
Cantonal Hospital, Children's Clinic
Aarau, Canton of Aargau, 5001, Switzerland
Cantonal Hospital
Aarau, Canton of Aargau, 5001, Switzerland
Private children's practice
Bern, Canton of Bern, 3014, Switzerland
Children's Hospital of Eastern Switzerland
Sankt Gallen, Canton of St. Gallen, 9006, Switzerland
University Children's Hospital
Zurich, Canton of Zurich, 8032, Switzerland
University Hospital
Zurich, Canton of Zurich, 8091, Switzerland
Private practice
Zürich (Kreis 11) / Oerlikon, Canton of Zurich, 8050, Switzerland
Private practice
Arzo, Canton Ticino, 6864, Switzerland
Private practice
Lugano, Canton Ticino, 6900, Switzerland
Regional Hospital
Lugano, Canton Ticino, 6900, Switzerland
Private practice
Riva San Vitale, Canton Ticino, 6826, Switzerland
Cantonal Hospital Graubünden
Chur, Kanton Graubünden, 7000, Switzerland
Private children's practice
Brig, Valais, 3900, Switzerland
Related Publications (2)
Tondury 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. doi: 10.1046/j.1365-2141.1998.00725.x.
PMID: 9633879RESULTTondury P, Kontoghiorghes GJ, Ridolfi-Luthy 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. doi: 10.1111/j.1365-2141.1990.tb07915.x.
PMID: 2265118RESULT
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Petrign FG Töndury, MD
- PRINCIPAL INVESTIGATOR
Markus Schmugge Liner, MD
University Children's Hospital, Zurich
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 6, 2006
First Posted
July 7, 2006
Study Start
March 1, 2005
Primary Completion
May 1, 2011
Study Completion
May 1, 2011
Last Updated
December 12, 2011
Record last verified: 2011-12