Post Hematopoietic Stem Cell Transplantation

NCT01610297 | Completed Phase 4 Results

• 1 other identifier: CICL670ATR04
• Study Type: interventional
• Target: 27
• Locations: 1 country
• Sites: 6

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.

Conditions

Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major

Keywords

hematopoietic stem cell transplantation, beta-thalassemia major, deferasirox, iron overload

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Adverse Events, Serious Adverse Events and Deaths as a Measure of Safety and Tolerability

  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

  12 months

Secondary Outcomes (4)

• Change in Serum Ferritin Level.

  Baseline, 12 Months

• Change in the Further Parameters of Iron Overload (Liver Iron Concentration by Magnetic Resonance Imaging (MRI Examination)

  Baseline, 12 month

• The Percentage of Patients Reaching Serum Ferritin Levels Lower Than 500 μg/L

  Week 28 and Week 52

• Change in the Further Parameters of Iron Overload (Cardiac Iron Concentration by Magnetic Resonance Imaging (MRI Examination)

  Baseline, 12 month

Study Arms (1)

ICL670

EXPERIMENTAL

Oral dose of ICL670 at 10 mg/kg daily

Drug: ICL670

Interventions

ICL670 DRUG

Oral dose of ICL670 at 10 mg/kg daily

ICL670

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (6)

Novartis Investigative Site

Ankara, Turkey, 06100, Turkey (Türkiye)

Location

Novartis Investigative Site

Ankara, Turkey, 06500, Turkey (Türkiye)

Location

Novartis Investigative Site

Izmir, Turkey, 35040, Turkey (Türkiye)

Location

Novartis Investigative Site

Antalya, 07000, Turkey (Türkiye)

Location

Novartis Investigative Site

Antalya, 07070, Turkey (Türkiye)

Location

Novartis Investigative Site

Istanbul, Turkey (Türkiye)

Location

Related Publications (1)

• Yesilipek MA, Karasu G, Kaya Z, Kuskonmaz BB, Uygun V, Dag I, Ozudogru O, Ertem M. A Phase II, Multicenter, Single-Arm Study to Evaluate the Safety and Efficacy of Deferasirox after Hematopoietic Stem Cell Transplantation in Children with beta-Thalassemia Major. Biol Blood Marrow Transplant. 2018 Mar;24(3):613-618. doi: 10.1016/j.bbmt.2017.11.006. Epub 2017 Nov 16.

  PMID: 29155313 DERIVED

MeSH Terms

Conditions

beta-Thalassemia; Iron Overload

Interventions

Deferasirox

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Iron Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Benzoates; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Triazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Results Point of Contact

• Title: Study Director
• Organization: Novartis

862-778-8300

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 30, 2012
• First Posted: June 4, 2012
• Study Start: September 1, 2013
• Primary Completion: October 1, 2015
• Study Completion: October 1, 2015
• Last Updated: October 24, 2016
• Results First Posted: July 21, 2016

Record last verified: 2016-09

Locations

TU (6)