NCT03203850

Study to Evaluate the Efficacy and Safety of Deferasirox Film-coated Tablet Versus Phlebotomy in Patients With Hereditary Hemochromatosis (HH)

A Phase II, Multicenter, Open-label, Randomized Two-year Study to Evaluate the Efficacy and Safety of Deferasirox Film-coated Tablet Versus Phlebotomy in Patients With Hereditary Hemochromatosis.

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 45 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study was to evaluate the efficacy and safety of deferasirox film coated tablet (FCT) versus phlebotomy for the management of iron overload in adults with Hereditary Hemochromatosis (HH) at risk of iron-related morbidity. This evaluation provided information on the two treatment options in terms of the rate of response of proportion of patients reaching the study target SF ≤ 100 μg/L and their associated safety profiles. In addition to exploring the safety and efficacy of deferasirox FCT in hereditary hemochromatosis (HH), this study is being conducted to fulfill an FDA post-marketing requirement \[PMC 750-10 (Exjade) /PMR 2888-8 (Jadenu)\] to provide additional randomized data to confirm the ocular safety profile of deferasirox through detailed ocular assessments in patients treated with deferasirox FCT for 2 years.

Detailed description

This was a Phase II, multicenter, open-label, randomized two-year study in adults with Hereditary Hemochromatosis (HH) confirmed by HH genotype with iron overload. Eligible subjects were identified during a 4-week screening period, then randomized in a 2:1 ratio to be treated with deferasirox FCT or phlebotomy for up to 24 months (104 weeks).

Conditions

• Hereditary Hemochromatosis

Interventions

Type	Name	Description
DRUG	Deferasirox FCT	Taken orally once per day (QD) as a film coated tablet (FCT)
PROCEDURE	Phlebotomy	according to investigator's decision

Timeline

• Start date: 2018-01-11
• Primary completion: 2023-04-17
• Completion: 2023-04-17
• First posted: 2017-06-29
• Last updated: 2024-10-09
• Results posted: 2024-06-07

Locations

11 sites across 7 countries: Belgium, France, Romania, Russia, Slovakia, Spain, Switzerland

Regulatory

• FDA-regulated drug study