Study of Deferasirox Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients

• Register: EUCTR
• Last refreshed on: 2 October 2017
• Main ID: EUCTR2016-002583-14-Outside-EU/EEA
• Date of registration: 25/11/2016
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharmaceuticals Corp
• Public title: Study of Deferasirox Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients
• Scientific title: A Randomized, Open-label, Multi-center, Phase II Study to Evaluate the Safety and Efficacy of Deferasirox (ICL670) 20 mg/kg/Day Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients With Iron Overload From Repeated Blood Transfusions - Study of Deferasirox Relative to Subcutaneous Deferoxamine in Sickle Cell Disease
• Target sample size: 210
• Recruitment status: NA
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-002583-14
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: yes Single blind: no Double blind: no Parallel group: yes Cross over: yes Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: yes Other specify the comparator: Deferoxamine Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Canada, United States

Contacts

• Name: Clinical Trial Information Desk
• Affiliation: Novartis Pharma AG

• Name: Clinical Trial Information Desk
• Affiliation: Novartis Pharma AG

Key inclusion & exclusion criteria

Inclusion criteria:
•Age greater than or equal to 2 years
•Male or female patients with sickle cell disease (SS, SC, SD, Sßo or Sß+ thalassemia)
•Iron overload from repeated blood transfusion
Are the trial subjects under 18? yes
Number of subjects for this age range: 203
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 77
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
•Serum creatinine above the upper limit of normal
•Significant proteinuria•History of nephrotic syndrome
•Alanine aminotransferase (ALT) = 250 U/L at screening
•Clinical evidence of active hepatitis B or hepatitis C


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Sickle Cell Disease/ Iron Overload

Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Intervention(s)

Trade Name: Exjade
Product Name: Exjade
Product Code: ICL670
Pharmaceutical Form: Dispersible tablet
INN or Proposed INN: DEFERASIROX
CAS Number: 201530-41-8
Current Sponsor code: ICL670
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 250-

Primary Outcome(s)

Secondary Objective: Long term safety of ICL670 for up to 104 weeks in patients with sickle cell disease and iron overload from repeated blood transfusions

Primary end point(s): -The Number of Participants with Adverse Events (AEs) in the First 24 Weeks of Treatment
-The number of participants with Adverse Events (AEs) overall and according to Medical Dictionary for Regulatory Activities (MedDRA) preferred term greater than or equal to 5% participants in any group by treatment in the first 24 weeks

Timepoint(s) of evaluation of this end point: -24 weeks
-24 weeks

Main Objective: To assess the safety of ICL670 compared to deferoxamine during 24 weeks in patients with sickle cell disease and iron overload from repeated blood transfusions

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: -24 weeks
-24 weeks, 52 weeks
-104 weeks

Secondary end point(s): Absolute Change in Serum Ferritin From Baseline to Week 24 [ Time Frame: Baseline, 24 Weeks ]
-Absolute change from baseline serum ferritin after 24 weeks of treatment with Deferasirox (ICL670) and absolute change from baseline serum ferritin after 24 weeks of treatment with Deferoxamine. Means were adjusted for the amount of transfused blood
-Absolute Change in Serum Ferritin After Start of Treatment With Deferasirox (ICL670) to Week 24 and to Week 52 [ Time Frame: Start of Deferasirox (ICL670) treatment, 24 Weeks, 52 Weeks ]
Absolute change in serum ferritin after start of treatment with Deferasirox (ICL670) to week 24 and the absolute change in serum ferritin after start of treatment with Deferasirox (ICL670) to week 52 for the Deferasirox treatment group and the Deferoxamine then Deferasirox treatment group. Means were adjusted for the amount of transfused blood.
-Absolute Change in Serum Ferritin After Start of Treatment With Deferasirox (ICL670) to Week 104 [ Time Frame: Start of Deferasirox (ICL670) treatment, 104 Weeks ]
Absolute change in serum ferritin after start of treatment with Deferasirox (ICL670) to week 104 for the Deferasirox treatment group. Means were adjusted for the amount of transfused blood.

Secondary ID(s)

CICL670A2201

Source(s) of Monetary Support

Novartis Pharmaceuticals Corp