Study to Assess Iron Chelation Therapy in Patients With Chronic Iron Overload (EXCALIBUR)

A Prospective Non-interventional Study to Assess Iron Chelation Therapy in Patients With Chronic Iron Overload

This is a prospective, multicenter, non-interventional study. Findings are analyzed using epidemiological methods.

Study Overview

• Status: Completed
• Conditions: Chronic Iron Overload
• Intervention / Treatment: Other: Iron chelator

Detailed Description

This NIS shall document the therapy of chronic iron overload in a broad patient population with either of the approved iron chelators in Germany. The choice of patients and the therapy according to the SmPC follows the clinical routine exclusively. The regular observation time per patient is 24 months.

Corresponding to the clinical routine, follow up visits can be documented after approximately 1, 3, 6, 9, 12 and 18 month with a close out visit after 24 month. In case the iron chelator is switched, the visit scheme should be passed through once again.

• Study Type: Observational
• Enrollment (Actual): 489

Contacts and Locations

Study Locations

• Germany
  • Altenburg, Germany, 04600
    • Novartis Investigative Site
  • Bad Liebenwerda, Germany, 04924
    • Novartis Investigative Site
  • Bad Mergentheim, Germany, 97980
    • Novartis Investigative Site
  • Berlin, Germany, 12627
    • Novartis Investigative Site
  • Berlin, Germany, 14089
    • Novartis Investigative Site
  • Berlin, Germany, 13357
    • Novartis Investigative Site
  • Berlin, Germany, 10407
    • Novartis Investigative Site
  • Berlin, Germany, 10709
    • Novartis Investigative Site
  • Berlin, Germany, 12487
    • Novartis Investigative Site
  • Biberach, Germany, 88400
    • Novartis Investigative Site
  • Bochum, Germany, 44787
    • Novartis Investigative Site
  • Bottrop, Germany, 46236
    • Novartis Investigative Site
  • Bremerhaven, Germany, 27568
    • Novartis Investigative Site
  • Celle, Germany, 29221
    • Novartis Investigative Site
  • Chemnitz, Germany, 09113
    • Novartis Investigative Site
  • Donauwoerth, Germany, 86609
    • Novartis Investigative Site
  • Dresden, Germany, 01307
    • Novartis Investigative Site
  • Dresden, Germany, 01127
    • Novartis Investigative Site
  • Erfurt, Germany, 99085
    • Novartis Investigative Site
  • Essen, Germany, 45136
    • Novartis Investigative Site
  • Gera, Germany, 07548
    • Novartis Investigative Site
  • Goettingen, Germany, 37073
    • Novartis Investigative Site
  • Goslar, Germany, 38642
    • Novartis Investigative Site
  • Halle, Germany, 06110
    • Novartis Investigative Site
  • Hameln, Germany, 31785
    • Novartis Investigative Site
  • Hamm, Germany, 59063
    • Novartis Investigative Site
  • Hannover, Germany, 30161
    • Novartis Investigative Site
  • Hannover, Germany, 30170
    • Novartis Investigative Site
  • Heidelberg, Germany, 69115
    • Novartis Investigative Site
  • Heilbronn, Germany, 74072
    • Novartis Investigative Site
  • Herrsching, Germany, 82211
    • Novartis Investigative Site
  • Hildesheim, Germany, 31134
    • Novartis Investigative Site
  • Hildesheim, Germany, 31135
    • Novartis Investigative Site
  • Hof, Germany, 95028
    • Novartis Investigative Site
  • Kaiserslautern, Germany, 67655
    • Novartis Investigative Site
  • Kassel, Germany, 34125
    • Novartis Investigative Site
  • Kassel, Germany, 34119
    • Novartis Investigative Site
  • Koblenz, Germany, 56068
    • Novartis Investigative Site
  • Koeln, Germany, 50937
    • Novartis Investigative Site
  • Krefeld, Germany, 47805
    • Novartis Investigative Site
  • Kronach, Germany, 96317
    • Novartis Investigative Site
  • Lemgo, Germany, 32657
    • Novartis Investigative Site
  • Luedenscheid, Germany, 58507
    • Novartis Investigative Site
  • Lutherstadt Wittenberg, Germany, 06886
    • Novartis Investigative Site
  • Marburg, Germany, 35037
    • Novartis Investigative Site
  • Minden, Germany, 32429
    • Novartis Investigative Site
  • Moers, Germany, 47441
    • Novartis Investigative Site
  • Muelheim, Germany, 45468
    • Novartis Investigative Site
  • Muenster, Germany, 48149
    • Novartis Investigative Site
  • Munchen, Germany, 81479
    • Novartis Investigative Site
  • München, Germany, 81377
    • Novartis Investigative Site
  • Naunhof, Germany, 04683
    • Novartis Investigative Site
  • Neuss, Germany, 41462
    • Novartis Investigative Site
  • Neustadt, Germany, 31535
    • Novartis Investigative Site
  • Nordhorn, Germany, 48527
    • Novartis Investigative Site
  • Nuernberg, Germany, 90419
    • Novartis Investigative Site
  • Nuernberg, Germany, 90403
    • Novartis Investigative Site
  • Offenburg, Germany, 77654
    • Novartis Investigative Site
  • Passau, Germany, 94036
    • Novartis Investigative Site
  • Porta Westfalica, Germany, 32457
    • Novartis Investigative Site
  • Potsdam, Germany, 14467
    • Novartis Investigative Site
  • Riesa, Germany, 01587
    • Novartis Investigative Site
  • Roetha, Germany, 04571
    • Novartis Investigative Site
  • Rostock, Germany, 18057
    • Novartis Investigative Site
  • Saarbruecken, Germany, 66113
    • Novartis Investigative Site
  • Saarbrücken, Germany, 66113
    • Novartis Investigative Site
  • Schorndorf, Germany, 73614
    • Novartis Investigative Site
  • Schwäbisch-Hall, Germany, 74523
    • Novartis Investigative Site
  • Speyer, Germany, 67346
    • Novartis Investigative Site
  • Stade, Germany
    • Novartis Investigative Site
  • Stolberg, Germany, 52222
    • Novartis Investigative Site
  • Stuttgart, Germany, 70178
    • Novartis Investigative Site
  • Traunstein, Germany, 83278
    • Novartis Investigative Site
  • Weiden, Germany, 92637
    • Novartis Investigative Site
  • Wesel, Germany, 46485
    • Novartis Investigative Site
  • Westerstede, Germany, 26655
    • Novartis Investigative Site
  • Wolfsburg, Germany, 38440
    • Novartis Investigative Site
  • Wuerselen, Germany, 52146
    • Novartis Investigative Site
  • Wuerzburg, Germany, 97080
    • Novartis Investigative Site
  • Baden Wuerttemberg
    • Winnenden, Baden Wuerttemberg, Germany, 71364
      • Novartis Investigative Site
  • Baden-Wuerttemberg
    • Heidenheim, Baden-Wuerttemberg, Germany, 89518
      • Novartis Investigative Site
    • Mannheim, Baden-Wuerttemberg, Germany, 68305
      • Novartis Investigative Site
  • Bavaria
    • Bamberg, Bavaria, Germany, 96052
      • Novartis Investigative Site
    • Munich, Bavaria, Germany, 80797
      • Novartis Investigative Site
  • Bayern
    • Aschaffenburg, Bayern, Germany, 63739
      • Novartis Investigative Site
    • Bayreuth, Bayern, Germany, 95445
      • Novartis Investigative Site
    • Landshut, Bayern, Germany, 84028
      • Novartis Investigative Site
    • Regensburg, Bayern, Germany, 93053
      • Novartis Investigative Site
  • Lower Saxony
    • Garbsen, Lower Saxony, Germany, 30827
      • Novartis Investigative Site
  • Nordrhein Westfalen
    • Bonn, Nordrhein Westfalen, Germany, 53177
      • Novartis Investigative Site
    • Gelsenkirchen, Nordrhein Westfalen, Germany, 45879
      • Novartis Investigative Site
  • Nordrhein-Westfalen
    • Dortmund, Nordrhein-Westfalen, Germany, 44309
      • Novartis Investigative Site
    • Koeln, Nordrhein-Westfalen, Germany, 50677
      • Novartis Investigative Site
  • North Rhine-Westphalia
    • Aachen, North Rhine-Westphalia, Germany, 52064
      • Novartis Investigative Site
    • Duisburg, North Rhine-Westphalia, Germany, 47166
      • Novartis Investigative Site
  • Northrhine Westfalia
    • Iserlohn, Northrhine Westfalia, Germany, 58644
      • Novartis Investigative Site
    • Remscheid Innen, Northrhine Westfalia, Germany, 42859
      • Novartis Investigative Site
  • Rhineland Palatinate
    • Neuwied, Rhineland Palatinate, Germany, 56564
      • Novartis Investigative Site
  • Sachsen
    • Dresden, Sachsen, Germany, 01307
      • Novartis Investigative Site
    • Pirna, Sachsen, Germany, 01796
      • Novartis Investigative Site
    • Zittau, Sachsen, Germany, 02763
      • Novartis Investigative Site
  • Schleswig-holstein
    • Luebeck, Schleswig-holstein, Germany, 23563
      • Novartis Investigative Site
  • Thueringen
    • Erfurt, Thueringen, Germany, 99084
      • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Male and female adult patients suffering from chronic iron overload receiving an iron chelation therapy

Description

Inclusion Criteria:

Male and female adult patients suffering from chronic iron overload

• who never received an iron chelator directly before starting an iron chelation therapy
• who received an iron chelation therapy for less than 6 month
• who interrupted an iron chelation therapy for longer than 6 months and will receive an iron chelation therapy again
• who signed the informed consent

Exclusion Criteria:

-

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Iron chelator; Patients prescribed with Iron Chelators	Other: Iron chelator; There is no treatment allocation. Patients administered approved Iron chelators by prescription that have started before inclusion of the patient into the study will be enrolled.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Efficiency of iron overload therapy	Efficiency of iron chelation therapy is evaluated by the change of serum ferritin	Up to 24 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of patients treated with different iron chelators	Number of patients treated with one of the three iron chelators approved in Germany.	Up to 24 months
Number of patients with therapy switch	Number of patients with therapy switch in the standard clinical routine and the reason for the switch. The documentation of a therapy change is limited to the end of recruitment time (10/31/2019). From that date, the documentation of a change in therapy is no longer applicable.	Up to 24 months
Number of patients with dose adjustments	Number of patients with dose adjustments during the observational period are collected	Up to 24 months
Number of patients with myelodyplastic syndromes or myeloproliferative neoplasms experiencing a hematologic response during an iron chelation therapy	The analysis of a hematological response to iron chelation therapy should follow the effective criteria of the international working group	Up to 24 months
Number of patients with AEs and SAE	Number of patients with Adverse Events and Serious Adverse Events is collected	Up to 24 months
Number of patients with side effects	Side effects are defined as all adverse events, which are suspected to have a causality with the respective iron chelator. All events are reported in the eCRF and causality with iron chelator treatment is requested.	Up to 24 months
Treatment Satisfaction Questionnaire to Medication (TSQM) 1.4	TSQM is used to measure the Patients' self-reported satisfaction or dissatisfaction with the study treatment. The TSQM version 1.4 domain scores range from 0 to 100 with higher scores representing a higher satisfaction on that domain.	Up to 24 months

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals

Publications and helpful links

Helpful Links

• Results for CICL670ADE14 from the Novartis Clinical Trials Website

Study record dates

Study Major Dates

• Study Start (ACTUAL): September 15, 2015
• Primary Completion (ACTUAL): October 29, 2021
• Study Completion (ACTUAL): October 29, 2021

Study Registration Dates

• First Submitted: June 21, 2022
• First Submitted That Met QC Criteria: June 29, 2022
• First Posted (ACTUAL): June 30, 2022

Study Record Updates

• Last Update Posted (ACTUAL): October 21, 2022
• Last Update Submitted That Met QC Criteria: October 20, 2022
• Last Verified: October 1, 2022

More Information

Terms related to this study

• Keywords: NIS; Germany; chronic iron overload
• Additional Relevant MeSH Terms: Metabolic Diseases; Iron Metabolism Disorders; Iron Overload; Molecular Mechanisms of Pharmacological Action; Sequestering Agents; Chelating Agents
• Other Study ID Numbers: CICL670ADE14

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO