Clinical trial NCT00654589: Efficacy and Safety of Oral Deferasirox (20 mg/kg/d) in Pts 3 to 6 Months After Allogeneic Hematopoietic Cell Transplantation Who Present With Iron Overload
Official title: A One-year, Open-label, Single Arm, Multi-center Trial Evaluating the Efficacy and Safety of Oral ICL670 (20 mg/kg/d) in Patients Three to Six Months After Allogeneic Hematopoietic Cell Transplantation in Whom Iron Overload is Present
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Iron Overload
Keywords: Hematopoietic stem cell transplantation
HCT
iron overload
chelators
ICL670
deferasirox
serum ferritin
LIC
transfusional hemosiderosis
blood transfusions
adults
iron chelation
3-6 months
allogeneic hematopoietic stem cell transplantation
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2008-04-08
Enrollment: 75
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to investigate the effects of iron chelation using deferasirox in patients who show signs of iron overload after an allogeneic stem cell transplantation The iron overload must be due to blood transfusions.


Eligibility criteria: Inclusion criteria:

1. Transfusional iron overload three to six months after HCT with no evidence of active inflammation
2. History of at least 20 units of red blood cell transfusions or 100mL/kg of prepacked red blood cells (PRBCs).
3. Patients of either gender and age ≥ 18 years.
4. Female patients who have reached menarche and who are sexually active must use double-barrier contraception, oral contraceptive plus barrier contraception , or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation or be postmenopausal defined by amenorrhea for at least 12 months.

Exclusion criteria:

1. Non-transfusion related iron overload
2. Active malignancy
3. Known active viral hepatitis or known HIV positiveness
4. Mean levels of alanine aminotransferase (ALT) \> 5x ULN
5. Treatment with any iron chelator after transplantation
6. Uncontrolled systemic hypertension
7. Serum creatinine \> 1.5 ULN and/or serum creatinine clearance \< 60 ml/min
8. History of nephrotic syndrome.
9. Previous history of clinically relevant ocular or auditory toxicity related to iron chelation.
10. Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent the patient from undergoing study treatment
11. Pregnant or breast feeding patients.

Other protocol-defined inclusion/exclusion criteria may apply

Primary outcomes:
measure: To assess iron chelation by comparing serum ferritin values at baseline vs. 52 weeks of treatment with deferasirox
timeFrame: 52 weeks
Secondary outcomes:
measure: To evaluate the relationship between serum ferritin, transferrin (TRF) and transferrin saturation during the whole study.
timeFrame: 52 weeks
Other outcomes:
Locations:
city: Leipzig
country: Germany
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmeceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
citation: Jaekel N, Lieder K, Albrecht S, Leismann O, Hubert K, Bug G, Kroger N, Platzbecker U, Stadler M, de Haas K, Altamura S, Muckenthaler MU, Niederwieser D, Al-Ali HK. Efficacy and safety of deferasirox in non-thalassemic patients with elevated ferritin levels after allogeneic hematopoietic stem cell transplantation. Bone Marrow Transplant. 2016 Jan;51(1):89-95. doi: 10.1038/bmt.2015.204. Epub 2015 Sep 14.