deferasirox tablet for oral suspension / Generic mfg. - LARVOL DELTA

INOVA-2023-159: Early Screening and Treatment of Heart Complication in Sickle Cell Disease (clinicaltrials.gov) - P2 | N=100 | Recruiting | Sponsor: Inova Health Care Services | Not yet recruiting ➔ Recruiting

Enrollment open • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

INOVA-2023-159: Early Screening and Treatment of Heart Complication in Sickle Cell Disease (clinicaltrials.gov) - P2 | N=100 | Not yet recruiting | Sponsor: Inova Health Care Services | Initiation date: Jun 2025 ➔ Sep 2025

Trial initiation date • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

Early Screening and Treatment of Heart Complication in Sickle Cell Disease (clinicaltrials.gov) - P2 | N=100 | Not yet recruiting | Sponsor: Inova Health Care Services

New P2 trial • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

Targeted active surveillance of branded generic deferasirox in pediatric thalassemia patients in a tertiary care hospital of India: A pharmacovigilance study. (PubMed, Indian J Pharmacol) - "ADRs of deferasirox are acceptable looking at the benefits. Regular monitoring and management of ADRs will facilitate patient compliance. However, healthcare professionals need to be alert while prescribing this drug and report any ADR, even if it is not labeled. This study found increased serum lipase and mouth ulcer as adverse effects, which are not yet labeled. These can be signals for further analysis by regulatory authorities."

Adverse events • Journal • Observational data • Genetic Disorders • Hematological Disorders • Pediatrics

Risk Factors and Measures to Prevent Liver and Pancreas Complications in Pediatric Patients After HSCT (clinicaltrials.gov) - P=N/A | N=39 | Completed | Sponsor: University of Pisa | Recruiting ➔ Completed

Trial completion • Bone Marrow Transplantation • Hematological Disorders • Pediatrics • Transplantation

LODEFI: Evaluating Low-dose Deferasirox (DFX) in Patients With Low-risk MDS Resistant or Relapsing After ESA Agents (clinicaltrials.gov) - P2 | N=39 | Completed | Sponsor: University Hospital, Grenoble | Recruiting ➔ Completed | Trial completion date: Dec 2024 ➔ Oct 2024 | Trial primary completion date: Dec 2024 ➔ Oct 2024

Trial completion • Trial completion date • Trial primary completion date • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Comparison of the effects of deferasirox film-coated tablets (Jadenu®) and deferasirox dispersible tablets (Exjade®) in patients with beta thalassemia major: a preliminary report of the effects on the satisfaction, convenience, cardiac/liver MRI T2*, serum ferritin level, and biochemical profiles. (PubMed, Front Pharmacol) - "This, in turn, may help mitigate cardiovascular and hepatic complications from iron overload in the long term. https://irct.behdasht.gov.ir/search/result?query=IRCT20210830052346N1."

Journal • Beta-Thalassemia • Cardiovascular • Gastrointestinal Disorder • Genetic Disorders • Hematological Disorders

Study to Evaluate the Efficacy and Safety of Deferasirox Film-coated Tablet Versus Phlebotomy in Patients With Hereditary Hemochromatosis (HH) (clinicaltrials.gov) - P2 | N=45 | Terminated | Sponsor: Novartis Pharmaceuticals | Completed ➔ Terminated; Due to enrolment challenge

Trial termination • Genetic Disorders • Hematological Disorders • Hepatology

Survey to Assess Physicians' Knowledge of Exjade Posology and Biological Monitoring Recommendations as Described in the Educational Materials (clinicaltrials.gov) - P=N/A | N=400 | Completed | Sponsor: Novartis Pharmaceuticals | Not yet recruiting ➔ Completed | Trial completion date: Oct 2024 ➔ Feb 2024 | Trial primary completion date: Oct 2024 ➔ Feb 2024

Trial completion • Trial completion date • Trial primary completion date • Hematological Disorders

Treatment of Iron Overload With Deferasirox (Exjade) in Hereditary Hemochromatosis and Myelodysplastic Syndrome (clinicaltrials.gov) - P2 | N=0 | Withdrawn | Sponsor: Haukeland University Hospital | N=50 ➔ 0 | Terminated ➔ Withdrawn

Biomarker • Enrollment change • Trial withdrawal • Genetic Disorders • Hematological Disorders • Hematological Malignancies • Hepatology • Myelodysplastic Syndrome • Oncology

Survey to Assess Physicians' Knowledge of Exjade Posology and Biological Monitoring Recommendations as Described in the Educational Materials (clinicaltrials.gov) - P=N/A | N=400 | Not yet recruiting | Sponsor: Novartis Pharmaceuticals

New trial • Hematological Disorders

Study to Evaluate Treatment Compliance, Efficacy and Safety of an Improved Deferasirox Formulation (Granules) in Pediatric Patients (2-<18 Years Old) With Iron Overload (clinicaltrials.gov) - P2 | N=224 | Completed | Sponsor: Novartis Pharmaceuticals | Active, not recruiting ➔ Completed

Compliance • Trial completion • Anemia • Hematological Disorders • Pediatrics

LATE RESPONSE TO LUSPATERCEPT IN A CASE OF MDS-RS-T (SIE 2023) - "Iron chelation therapy : Exjade from 2019 to 2022, now suspended. Total number of MDS-RS patients treated with Luspatercept 15 Age years, median (range) 74 (52-83) Sex, n (%) Male 10 (67) Female 5 (33) Cytogenetic, n Normal 11 8 1 Y 1 del 11q 1 del 20q 1 IPSS-R Classification, n (%) Very Low Risk 1 (15) Low Risk 7 (47) Intermediate Risk 6 (38) Mutation status, detected by NGS SF3B1, n (%) 14 (92) Vaf , %, median (range) 39 (21,4-46,3) Patients with others concomitant mutations 11 (73%) IPSS M Classification, n (%) Very Low Risk 2 (13) Low Risk 8 (54) Moderatly Low Risk 4 (26) Intermediate Risk 1 (7) Duration of EPO traitment before Luspatercept Months, median, range 21, 6-104 Transfusion burden sec. IWG 2018 Low Trasfusion Burden, n (% ) 7 (46) High transfusion burden , n (%) 8 (54)"

Clinical • Anemia • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • ABL1 • BCR • CD34 • JAK2 • SF3B1

Risk Factors and Measures to Prevent Liver and Pancreas Complications in Pediatric Patients After HSCT (clinicaltrials.gov) - P=N/A | N=39 | Recruiting | Sponsor: University of Pisa | Active, not recruiting ➔ Recruiting | Trial completion date: Jun 2022 ➔ Dec 2024 | Trial primary completion date: Jun 2021 ➔ Jun 2024

Enrollment open • Trial completion date • Trial primary completion date • Bone Marrow Transplantation • Hematological Disorders • Pediatrics • Transplantation

Study to Evaluate the Efficacy and Safety of Deferasirox Film-coated Tablet Versus Phlebotomy in Patients With Hereditary Hemochromatosis (HH) (clinicaltrials.gov) - P2 | N=45 | Completed | Sponsor: Novartis Pharmaceuticals | Active, not recruiting ➔ Completed

Trial completion • Genetic Disorders • Hematological Disorders • Hepatology • Solid Tumor

TROS: Low Dose Iron Chelation as TReatment of Oxidative Damage in Sickle Cell Disease (clinicaltrials.gov) - P2 | N=12 | Completed | Sponsor: Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) | Recruiting ➔ Completed

Trial completion • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

Renal Function in Patients with Thalassemia Major Receiving Exjade Dispersible Tablets and a New Film-coated Tablet Formulation of Deferasirox (Nanojade). (PubMed, Adv Biomed Res) - "The proportion of S, BUN, eGFR, 24-h U, U/U ratio, and U/U ratio was not significantly different in TM patients treated with Nanojade compared to patients' received Exjade. Nanojade had similar effects to Exjade, and therefore, the use of Nanojade is safe in TM patients and does not seem to be associated with increased renal failure, proteinuria, and hypercalciuria."

Journal • Beta-Thalassemia • Genetic Disorders • Nephrology • Renal Disease

Early and Low Dose Deferasirox (3.5 mg/kg FCT) to Suppress NTBI and LPI as Early Intervention to Prevent Tissue Iron Overload in Lower Risk MDS (clinicaltrials.gov) - P2 | N=11 | Terminated | Sponsor: Fondazione Italiana Sindromi Mielodisplastiche-ETS | N=60 ➔ 11 | Trial completion date: Jan 2021 ➔ Apr 2022 | Unknown status ➔ Terminated; enrollment failed

Enrollment change • Trial completion date • Trial termination • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

TROS: Low Dose Iron Chelation as TReatment of Oxidative Damage in Sickle Cell Disease (clinicaltrials.gov) - P2 | N=12 | Recruiting | Sponsor: Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

New P2 trial • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

LODEFI: Evaluating Low-dose Deferasirox (DFX) in Patients With Low-risk MDS Resistant or Relapsing After ESA Agents (clinicaltrials.gov) - P2 | N=39 | Recruiting | Sponsor: University Hospital, Grenoble | Trial completion date: Feb 2022 ➔ Dec 2024 | Trial primary completion date: Feb 2022 ➔ Dec 2024

Trial completion date • Trial primary completion date • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Effects of Deferasirox in Alzheimer's Disease and Tauopathy Animal Models. (PubMed, Biomolecules) - "We therefore tested an oral iron chelator, desferasirox (Exjade), in transgenic animal models...Treating the three models with 1.6 mg deferasirox thrice weekly from age 8 to 14 months did not affect memory as measured by contextual fear conditioning or motor function as measured by rotarod, but tended to decrease hyperphosphorylated tau as measured by AT8 immunohistochemistry and immunoblotting. Deferasirox might act by decreasing iron, which aggregates tau, or directly binding tau to inhibit aggregation."

Journal • Preclinical • Alzheimer's Disease • CNS Disorders

2D-ultrathin MXene/DOXjade platform for iron chelation chemo-photothermal therapy. (PubMed, Bioact Mater) - "This prodrug takes advantage of the clinically approved iron chelator deferasirox (ExJade®) and the topoisomerase 2 inhibitor, doxorubicin (DOX). A tumor pH-responsive iron chelation/photothermal/chemotherapy antitumor effect was achieved both in vitro and in vivo. The results of this study highlight what may constitute a promising iron chelation-based phototherapeutic approach to cancer therapy."

Journal • Oncology

Synthesis and antimalarial activity of amide and ester conjugates of siderophores and ozonides. (PubMed, Biometals) - "Previous studies indicated that co-administration of chloroquine with antioxidants such as the iron chelator deferoxamine (DFO) prevented the development of persistent cognitive damage in surrogate models of cerebral malaria. The work described herein reports the syntheses and antimalarial activities of covalent conjugates of both natural (siderophores) and artificial iron chelators, namely DFO, ferricrocin and ICL-670, with antimalarial 1,2,4-trioxolanes (ozonides). All of the synthesized conjugates had potent antimalarial activities against the in vitro cultures of drug resistant and drug sensitive strains of Plasmodium falciparum. The work described herein provides the basis for future development of covalent combination of iron chelators and antimalarial chemotherapeutic agents for the treatment of cerebral malaria."

Journal • Infectious Disease • Malaria

Two trade names of deferasirox (Osveral® and Exjade®) in reduction of iron overload parameters in major beta-thalassemia patients: A randomized open labeled clinical trial. (PubMed, Caspian J Intern Med) - "Osveral decreased significantly the serum ferritin level and improved heart and liver iron overload as efficient as Exjade. It can be a suitable cost-effective alternative agent in beta-thalassemia major patients."

Clinical • Journal • Beta-Thalassemia • Genetic Disorders • Hematological Disorders

Pilot Study to Assess the Safety, PK and Iron Chelating Activity of DST-0509 (Deferasirox) in Thalassemia Patients Refractory to Chelation (clinicaltrials.gov) - P2 | N=25 | Completed | Sponsor: DisperSol Technologies, LLC | Recruiting ➔ Completed | N=36 ➔ 25 | Trial primary completion date: Sep 2021 ➔ Jun 2021

Enrollment change • Trial completion • Trial primary completion date • Beta-Thalassemia • Genetic Disorders • MRI