NCT05392101: TROS

Low Dose Iron Chelation as TReatment of Oxidative Damage in Sickle Cell Disease

Completed Phase 2 Last updated 13 February 2023

What this trial tests

Phase 2 trial testing Deferasirox 360 MG in Sickle Cell Disease in 12 participants. Completed in 20 November 2022.

Timeline

20 July 2021

Primary endpoint
20 August 2022

20 November 2022

Quick facts

Lead sponsor	Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	basic science
Enrollment	12
Start date	20 July 2021
Primary completion	20 August 2022
Estimated completion	20 November 2022
Sites	1 location across Netherlands

Drugs / interventions tested

• Deferasirox 360 MG — full drug profile →

Conditions studied

• Sickle Cell Disease — all drugs for Sickle Cell Disease →

Sponsor

Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) — full company profile →

Who can join

Adults 18 to 65, any sex, with Sickle Cell Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Objective:

To study the safety and efficacy of deferasirox as treatment of oxidative stress in adult subjects with sickle cell disease.

Endpoints:

The investigators will determine whether treatment with iron chelators results in decreased sickling of RBCs, oxidative stress, neutrophil activation, inflammation, endothelial activation and hypercoagulability and ultimately reduced disease severity. If the hypothesis is confirmed in this pilot dose-finding study, a larger randomized controlled clinical trial will be initiated.

Study design:

This will be an open-label pilot study, including 12 patients per dose group with a maximum of 3 dose groups. As the antioxidant capacity of deferasirox might be dose-dependent, the investigators will start with the highest dose of deferasirox (360 mg) deemed adequate for chronic use without causing iron depletion in adult SCD patients.

Study population:

Adult patients with sickle cell anemia (HbSS) or HbS-β0-thalassemia (HbSβ0-thal) visiting the outpatient-clinic of the Academic Medical Center, Amsterdam will be asked for inclusion in the study.

Publications & conference data

4 peer-reviewed publications reference this trial (live from Europe PMC):

1. Targeting ferroptosis opens new avenues for the development of novel therapeutics.
   Sun S, Shen J, Jiang J, Wang F, et al · · 2023 · cited 380× · PMID 37735472 · DOI 10.1038/s41392-023-01606-1
2. Iron homeostasis and ferroptosis in human diseases: mechanisms and therapeutic prospects.
   Ru Q, Li Y, Chen L, Wu Y, et al · · 2024 · cited 365× · PMID 39396974 · DOI 10.1038/s41392-024-01969-z
3. Ferroptosis: mechanisms and therapeutic targets.
   Zhou Q, Meng Y, Le J, Sun Y, et al · · 2024 · cited 32× · PMID 39568772 · DOI 10.1002/mco2.70010
4. Ferroptosis in cardiovascular diseases: molecular mechanisms and a novel therapeutic target.
   Yu S, Pang Z, Fang H, Liu C. · · 2026 · PMID 41795043 · DOI 10.1186/s43556-026-00420-9