Post Hematopoietic Stem Cell Transplantation

Phase 4

Completed

• Conditions: Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major
• Interventions: Drug: ICL670

• Registration Number: NCT01610297
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

This was a prospective, single-arm, multicenter, national, phase II clinical study. The purpose of this Phase II study was to examine the safety and efficacy of deferasirox to decrease iron overload (IOL) in the posttransplant period in patients with beta-thalassemia major.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2012-05-30
• Study First Submitted QC: 2012-06-01
• Study First Posted: 2012-06-04
• Study Start: 2013-09
• Primary Completion: 2015-10
• Study Completion: 2015-10
• Results First Submitted: 2016-04-21
• Results First Submitted QC: 2016-06-09
• Results First Posted: 2016-07-21
• Status Verified: 2016-09
• Last Update Submitted: 2016-09-12
• Last Update Posted: 2016-10-24

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 27

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
ICL670	ICL670	Oral dose of ICL670 at 10 mg/kg daily

Primary Outcome Measures

Name	Time	Method
Number of Participants With Adverse Events, Serious Adverse Events and Deaths as a Measure of Safety and Tolerability	12 months	To determine the safety; incidence, type and severity of adverse events including renal, hepatic, biochemistry and hematologic parameters of deferasirox in the treatment of iron overload after hematopoietic stem cell transplantation (HSCT) in patients with beta-thalassemia major in 12 months period

Secondary Outcome Measures

Name	Time	Method
Change in Serum Ferritin Level.	Baseline, 12 Months	Blood samples were collected and serum levels were assessed at study baseline (BL) and at 12 months.
The Percentage of Patients Reaching Serum Ferritin Levels Lower Than 500 μg/L	Week 28 and Week 52	Serum Ferritin values between 1000-2500 μg/L are indicative of mild to moderate iron overload while values \>2500 μg/L are indicative of severe iron overload and levels constantly higher than 2500 μg/L has been shown to to increase the risk of cardiac complications and endocrine disease. Maintaining levels \<1000 μg/L is associated with increased survival and less morbidity.
Change in the Further Parameters of Iron Overload (Cardiac Iron Concentration by Magnetic Resonance Imaging (MRI Examination)	Baseline, 12 month	Cardiac MRI values between 10 to 20 milliseconds (ms) are indicative of moderate cardiac iron deposition associated with declining left ventricular ejection fraction and arrhythmias while values \<10 ms are indicative of deposition sufficient to risk cardiac decompensation and associated with overt heart failure and mortality.
Change in the Further Parameters of Iron Overload (Liver Iron Concentration by Magnetic Resonance Imaging (MRI Examination)	Baseline, 12 month	Liver Iron Concentration (LIC) values between 3 and 7 mg Fe / g dry weight are indicative of mild iron deposition, while values between 7 and 15 mg Fe / g dry weight are indicative of moderate iron deposition which have been associated with liver disease. Values \>15 mg Fe/g dry weight are indicative of severe iron deposition which is associated with progressive liver fibrosis, increased morbidity and mortality

Trial Locations

Locations (1)

Novartis Investigative Site; 🇹🇷 Izmir, Turkey