Study to Assess Iron Chelation Therapy in Patients With Chronic Iron Overload

Completed

• Conditions: Chronic Iron Overload
• Interventions: Other: Iron chelator

• Registration Number: NCT05440487
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

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

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 Timeline

• Study Start: 2015-09-15
• Primary Completion: 2021-10-29
• Study Completion: 2021-10-29
• Study First Submitted: 2022-06-21
• Study First Submitted QC: 2022-06-29
• Study First Posted: 2022-06-30
• Status Verified: 2022-10
• Last Update Submitted: 2022-10-20
• Last Update Posted: 2022-10-21

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 489

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Iron chelator	Iron chelator	Patients prescribed with Iron Chelators

Primary Outcome Measures

Name	Time	Method
Efficiency of iron overload therapy	Up to 24 months	Efficiency of iron chelation therapy is evaluated by the change of serum ferritin

Secondary Outcome Measures

Name	Time	Method
Number of patients treated with different iron chelators	Up to 24 months	Number of patients treated with one of the three iron chelators approved in Germany.
Number of patients with dose adjustments	Up to 24 months	Number of patients with dose adjustments during the observational period are collected
Number of patients with AEs and SAE	Up to 24 months	Number of patients with Adverse Events and Serious Adverse Events is collected
Number of patients with therapy switch	Up to 24 months	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.
Number of patients with myelodyplastic syndromes or myeloproliferative neoplasms experiencing a hematologic response during an iron chelation therapy	Up to 24 months	The analysis of a hematological response to iron chelation therapy should follow the effective criteria of the international working group
Number of patients with side effects	Up to 24 months	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.
Treatment Satisfaction Questionnaire to Medication (TSQM) 1.4	Up to 24 months	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.

Trial Locations

Locations (1)

Novartis Investigative Site; 🇩🇪 Wuerzburg, Germany