Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Deferasirox dispersable · 1 trial · 2 indications
Number of participants preferring deferasirox FCT or DT as measured by preference questionnaire (item 2) at Week 48. The preference questionnaire was a 3-item questionnaire. At Week 48, the second item of the preference questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient "like best": "Tablet to dissolve in liquid" (=deferasirox DT), "Film coated tablet" (=deferasirox FCT), "Sprinkle powder on food" (=deferasirox FCT) and "I don't know" (=none of the above). The number of participants who selected each response option for item 2 was assessed. The analysis was performed for participants who answered the item 2 of the preference questionnaire.
| Arm | Type | Description |
|---|---|---|
| Deferasirox DT followed by deferasirox FCT | EXPERIMENTAL | Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT |
| Name | Type | Description |
|---|---|---|
| Deferasirox dispersable tablet (DT) | DRUG | Deferasirox DT was provided as 125 mg, 250 mg, 500 mg dispersible tablets for oral use. The strengths provided in an individual country could differ and reflected the strengths available commercially in each country. For iron chelation naive participants, the starting dose on Baseline Day 1 was 20 mg/kg/day in TDT and 10 mg/kg/day in NTDT. For iron chelation (deferoxamine and/or deferiprone) pre-treated participants, the starting dose was equivalent to the dose of deferoxamine received (for participants pre-treated with deferoxamine) and based on their serum ferritin levels (for participants pre-treated with deferiprone). Participants took deferasirox DT once daily for 24 weeks (core phase). The required number of deferasirox DT tablets were to be dispersed with gentle stirring in a glass of water. |
| Deferasirox film coated tablet (FCT) | DRUG | Deferasirox FCT was provided as 90 mg, 180 mg, 360 mg film coated tablets for oral use. The FCT starting dose on Week 25 was 14 mg/kg/day in TDT and 7 mg/kg/day in NTDT. Participants took deferasirox FCT once daily for 24 weeks during the core phase and up to 48 weeks during the extension phase. For patients with difficulties in swallowing deferasirox FCT, it was allowed to crush the film-coated tablets and administer the study drug by sprinkling the full dose on soft food (like yogurt or apple puree). |
Inclusion Criteria: 1. Prior to any screening procedures were performed, written informed consent/assent must be provided. For pediatric patients, consent was obtained from parent(s) or legal patient's representative. Investigators also obtained assent of patients according to local, regional or na...
Deferasirox dispersable is an investigational small molecule being studied for use in transfusion-dependent thalassemia. It is a dispersible tablet formulation of deferasirox, an iron chelator intended to treat iron overload in patients who require regular blood transfusions. The drug is in Phase 2 clinical development.
Deferasirox dispersable is being developed by Novartis AG, a multinational pharmaceutical company traded on the New York Stock Exchange under the ticker NVS. Novartis is conducting clinical research on this dispersible tablet formulation for patients with transfusion-dependent thalassemia.
Deferasirox dispersable is in Phase 2 clinical development. It is an investigational drug and has not been approved by regulatory authorities. One Phase 2 clinical trial has been completed, and the drug remains under investigation for transfusion-dependent thalassemia.
Deferasirox dispersable has been studied in one completed Phase 2 clinical trial, registered as NCT02993224. This open-label, multicenter study assessed patient preference for the film-coated tablet compared to the dispersible tablet. The trial enrolled 148 participants with transfusion-dependent or non-transfusion-dependent thalassemia across seven countries.
Deferasirox dispersable and the film-coated tablet are different formulations of the same active ingredient, deferasirox. The clinical trial NCT02993224 directly compared the two formulations to assess patient preference. The dispersable tablet is designed to be dispersed in liquid before administration, while the film-coated tablet is swallowed whole.