Open-label, Multicenter Study Assessing Preference for Deferasirox Film-coated Tablet Compared to Dispersible Tablet
Condition: Transfusion-dependent Thalassemia; Non-transfusion-dependent Thalassemia
MeSH terms: Thalassemia
Intervention: Deferasirox dispersable tablet (DT); Deferasirox film coated tablet (FCT)
Intervention description: 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 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).
Intervention MeSH terms: Deferasirox
Phase: Phase 2
Study type: Interventional
Primary purpose: Other
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 148.0
Sex: All
Minimum age: 2 Years
Healthy volunteers: No
Keywords: thalassemia; TDT; transfusion-dependent; non-transfusion-dependent; NTDT

Summary:

      Study to evaluate patient preference of deferasirox film-coated tablet (FCT) or deferasirox
      dispersible tablet (DT) in patient with transfusion - dependent thalassemia or
      non-transfusion -dependent thalassemia as measured by preference questionnaire at Week 48
    

Eligibility criteria:

        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 national guidelines.

          2. Male and female patient aged ≥ 2 years

          3. Deferasirox naïve patient or chelated naive patient or treated by other chelators for
             at least 6 months, such as:

               1. Deferiprone/ DFP

               2. Deferoxamine /DFO

               3. Combination (DFO + DFP)

          4. Subject was willing to discontinue current iron chelation therapy at least 5 days
             prior to study day 1 and for the duration of the study

          5. Patients with transfusion-dependent thalassemia (independent of underlying condition)
             with transfusional iron overload as shown by a serum ferritin level of > 1000 ng/ml at
             screening and if available, LIC > 3 mg Fe/g dw within 6 months prior to screening

          6. Patients with non-transfusion-dependent thalassemia with iron overload as shown by a
             serum ferritin level of ≥ 800 ng/ml at screening and if available, LIC ≥ 5 mg Fe/g dw
             within 6 months prior to screening

        Exclusion Criteria:

          1. Creatinine clearance below the contraindication limit in the locally approved
             prescribing information.

          2. Serum creatinine level > 1.5 x ULN (upper limit of normal)

          3. AST (SGOT) /ALT (SGPT) > 5 x ULN, unless LIC confirmed as >10 mg Fe/dw within 6 months
             prior to screening visit.

          4. Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 mg/mg
             in a non-first void urine sample.

          5. Patients with significant impaired gastrointestinal (GI) function or GI disease that
             might significantly alter the absorption of oral deferasirox (e.g. ulcerative
             diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small
             bowel resection).

          6. Clinical or laboratory evidence of active Hepatitis B or Hepatitis C (HBsAg in the
             absence of HBsAb OR HCV Ab positive with HCV RNA positive).

          7. Patients with psychiatric or addictive disorders which prevent them from giving their
             informed consent or undergoing any of the treatment options or patients unwilling or
             unable to comply with the protocol

          8. Patients with a known history of HIV seropositivity (Elisa or Western blot).

          9. History of malignancy of any organ system, treated or untreated, within the past 5
             years whether or not there is evidence of local recurrence or metastases, with the
             exception of localized basal cell carcinoma of the skin.

         10. Patients participating in another clinical trial or receiving an investigational drug.
             Patients who have recently completed treatment with an investigational product must
             have ceased this treatment for at least five times the half-life of the
             investigational product.

         11. History of hypersensitivity to any of the study drug or excipients.

         12. Significant medical condition interfering with the ability to partake in this study
             (e.g. systemic uncontrolled hypertension, unstable cardiac disease not controlled by
             standard medical therapy, systemic disease (cardiovascular, renal, hepatic, etc.).

         13. Women of child-bearing potential, defined as all women physiologically capable of
             becoming pregnant, unless they were using effective methods of contraception during
             dosing of study treatment

         14. Women were considered post-menopausal and not of child bearing potential if they had
             had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile
             (e.g. age appropriate, history of vasomotor symptoms) or had had surgical bilateral
             oophorectomy (with or without hysterectomy) or tubal ligation at least six weeks ago.
             In the case of oophorectomy alone, only when the reproductive status of the woman had
             been confirmed by follow up hormone level assessment is she considered not of child
             bearing potential.

         15. Sexually active males unless they used a condom during intercourse while taking drug
             and for 28 days after stopping study medication and should not father a child in this
             period. A condom was required to be used also by vasectomized men in order to prevent
             delivery of the drug via seminal fluid.