Exjade-Early-Trial

NCT01058369 | Terminated Phase 2 Results

• 1 other identifier: CICL670ADE06T
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

Brief Summary

Study outline: Deferasirox (Exjade®) is regularly used in severe iron overload in order to avoid organ damage of liver, heart and other organs. It has been proposed, that iron overload may not only impose damage to other organs but also to the bone marrow and thus worsen hematopoietic insufficiency in patients with MDS. Patients presenting with low or INT-1 risk MDS with only mild iron overload will be treated with deferasirox in this study. It will be analyzed if hematological improvement can be observed during this treatment.

Conditions

Myelodysplastic Syndromes

Outcome Measures

Primary Outcomes (1)

• Fraction of Patients With Hematologic Improvement According to Modified IWG Criteria (Reduction of Transfusions and/or Increase in Hb, Improvement of Neutropenia and Thrombocytopenia)

  within two years

Secondary Outcomes (7)

• Evaluate the Safety and Tolerability Profile of Deferasirox in MDS Patients

  within two years

• Effectiveness of Iron Depletion

  within two years

• Correlation Between Hematological Improvement and Effectiveness of Iron Depletion

  two years

• Development of Bone Marrow Morphology

  two years

• Correlation Between Hematological Improvement and Pretreatment Parameters. Extension of This Analysis to MDS Patients on Deferasirox Within the Licensed Indication (More Severe Iron Overload)

  two years

Study Arms (1)

Deferasirox

EXPERIMENTAL

Drug: Deferasirox (Novartis Pharma)

Interventions

Deferasirox (Novartis Pharma) DRUG

Treatment period 102 weeks. Starting dose 10mg/kg/day. Up to 30/mg/kg according to dose adjustment table as specified in the protocol

Also known as: Exjade(R)

Deferasirox

Eligibility Criteria

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

You may qualify if:

• MDS of subtype RA, RARS, RCMD, RCMD-RS (i.e. lower risk)
• RAEB I allowed, if clinically stable for \> 3 months
• IPSS score \< intermediate-1
• transfusion dependent or Hb \< 10,5 g/dl
• History of less than 20 units of red blood cell transfusions or 100mL/kg of prepacked red blood cells (PRBCs), except for transfusions for acute bleeding
• Serum ferritin \> 300 µg/l and \< 1500 μg/l. This level should have been verified at least at two occasions within 3 months. Samples must be obtained in the absence of concomitant severe infection
• no indication for EPO (due to high endogenous EPO levels) or EPO without benefit in the past
• no indication and/or no plans for cytostatic drugs
• no previous exposure to cytostatic drugs, thalidomide, lenalidomide, G-CSF or EPO or exposure to any of these drugs has been terminated since \> 8 weeks (4 weeks for G-CSF).
• no indication and/or no plans for stem cell transplantation
• stable or worsening cytopenia during the past 8 weeks. If in doubt, extend screening period to \>= 8 weeks
• Patients of either gender and age \> 18 years
• Life expectancy \> 12 months
• Females of childbearing potential must use double-barrier contraception (for example orale contraception and condom).
• Mental ability of the patient to understand explications concerning the study and to understand and follow instructions of the investigating physician

You may not qualify if:

• Treatment with deferasirox or other chelation therapy for periods \> 4 weeks before study start
• Patients with intolerance to Deferasirox
• Patients with a concomitant second malignant disease, possibly interfering with life expectancy
• Patients with mean levels of alanine aminotransferase (ALT) \> 5x ULN
• Patients with uncontrolled systemic hypertension
• Patients with serum creatinine \> 1.5x the upper limit of normal (ULN) or a creatinine clearance \< 60 ml/min according to the MDRD formula (Levey 2005)
• History of nephrotic syndrome
• Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent the patient from undergoing study treatment
• Patients with psychiatric or addictive disorders which prevent them from giving their informed consent or undergoing study treatment
• Patients treated with systemic investigational drugs within the past 4 weeks or topical investigational drug within the past 7 days
• Any other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of any drug. The investigator should be guided by evidence of any of the following:
• history of inflammatory bowel syndrome, gastritis, ulcers, gastrointestinal or rectal bleeding;
• history of major gastrointestinal tract surgery such as gastrectomy, gastroenterostomy, or bowel resection;
• history of pancreatic injury or pancreatitis; indications of impaired pancreatic function/injury as indicated by abnormal lipase or amylase;
• history of urinary obstruction or difficulty in voiding

Sponsors & Collaborators

• University of Erlangen-Nürnberg Medical School: lead
• Novartis Pharmaceuticals: collaborator

Study Sites (1)

Medizinische Klinik 5, Universitätsklinikum Erlangen

Erlangen, Bavaria, 91054, Germany

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

Deferasirox

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic 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

Limitations and Caveats

Early Termination leading to small numbers of subjects enrolled; number of subjects analysed = 0

Results Point of Contact

• Title: Prof. Dr. med. Stefan Krause
• Organization: Universitätsklinikum Erlangen, Medizinische Klinik 5

stefan.krause@uk-erlangen.de

Study Officials

• Stefan Krause, Prof. Dr.

  Medizinische Klinik 5, Universitätsklinikum Erlangen

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 27, 2010
• First Posted: January 28, 2010
• Study Start: April 1, 2010
• Primary Completion: January 1, 2013
• Study Completion: January 1, 2013
• Last Updated: October 6, 2020
• Results First Posted: October 6, 2020

Record last verified: 2020-09

Locations

DE (1)