Phase 4
N=27
Post Hematopoietic Stem Cell Transplantation
Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major
Bottom Line
View on ClinicalTrials.gov: NCT01610297 ↗Enrolled (actual)
27
Serious AEs
11.1%
Results posted
Jul 2016
Primary outcome: Primary: Number of Participants With Adverse Events, Serious Adverse Events and Deaths as a Measure of Safety and Tolerability — 25; 3; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- ICL670 (Drug)
- Age
- Pediatric, Adult, Older Adult · 2+ yrs
- Sex
- All
- Sponsor
- Novartis Pharmaceuticals
- Primary completion
- Oct 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Adverse Events, Serious Adverse Events and Deaths as a Measure of Safety and Tolerability |
25; 3; 0 | — |
| SECONDARY Change in Serum Ferritin Level. |
1766.81; 903.56 | — |
| SECONDARY Change in the Further Parameters of Iron Overload (Liver Iron Concentration by Magnetic Resonance Imaging (MRI Examination) |
12.07; 4.62 | — |
| SECONDARY The Percentage of Patients Reaching Serum Ferritin Levels Lower Than 500 μg/L |
7.7; 33.3 | — |
| SECONDARY Change in the Further Parameters of Iron Overload (Cardiac Iron Concentration by Magnetic Resonance Imaging (MRI Examination) |
26.48; 28.25 | — |
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.
Eligibility Criteria
Inclusion Criteria
- Patients who had hematopoietic stem cell transplantation (HSCT) for beta-thalassemia major
- hematopoietic stem cell transplantation (HSCT) was performed minimum 6 months and maximum 2 years ago
- The washout period after the immunosuppressive therapy should be at least 3 months.
- Signifacant IOL should be present including:
A. Serum ferritin >1000 μg/L or B. cardiac MRI <20 ms or C. liver iron concentration ≥ 5 mg/g dry weight measured by R2* MRI
Exclusion Criteria
- Patients who had any contraindication for treatment with deferasirox according to the prescribing information
•Patients who depended on transfusion
- Patients with clinical symptoms of cardiac dysfunction (shortness of breath at rest or exertion, orthopnea, exercise intolerance, lower extremity edema, arrhythmias)
- Patients who were experiencing severe complication of HSCT e.g. acute Graft-versus host disease (GVHD)
- Significant proteinuria / Increase in serum creatinine
Data sourced from ClinicalTrials.gov (NCT01610297). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.