Open-label, Multicenter, Single Arm, Phase II Study Assessing Treatment Patient Preference for New Deferasirox Formulation (Film-coated Tablet) Compared to the Reference Deferasirox Dispersible Tablet Formulatio
- Conditions
- •Transfusion-dependent Thalassemia•Non-transfusion-dependent ThalassemiaThalassemiaD56.9
- Registration Number
- LBCTR2019020189
- Lead Sponsor
- ovartis Pharma Services Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete
- Sex
- All
- Target Recruitment
- 5
1. Prior to any screening procedures are performed, written informed consent/assent must be provided.
2. Male and female patient aged = 2 years
3. Exjade naïve patient or chelated naive patient or treated by other chelators for at least 6 months, such as: a. Deferiprone/ DFP b. Deferoxamine /DFO c. Combination (DFO + DFP)
4. Subject is willing to discontinue current iron chelation therapy at least 7 days prior to the first day 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 until 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 until 6 months prior to screening
1. Male and female patient aged < 2 years
2. Written consent/assent from patients/parents/legal representative is not obtained
3. Creatinine clearance below the contraindication limit in the locally approved prescribing information.
4. Serum creatinine level > 1.5 x ULN (upper limit of normal)
5. AST (SGOT) /ALT (SGPT) > 5 x ULN, unless if LIC confirmed as <10 mg Fe/dw within 6 months prior to screening visit.
6. Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 mg/mg in a non-first void urine sample.
7. Patients with significant impaired gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral deferasirox (e.g. ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection).
8. 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).
9. 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 (including use of electronic devices for ePRO).
10. Patients with a known history of HIV seropositivity (Elisa or Western blot).
11. 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.
12. Patients participating in another clinical trial or receiving an investigational drug.
13. History of hypersensitivity to any of the study drug or excipients.
14. 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.).
15. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception during dosing of study treatment
16. Women are considered post-menopausal and not of child bearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g. age appropriate, history of vasomotor symptoms) or have 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 has been confirmed by follow up hormone level assessment is she considered not of child bearing potential.
17. Sexually active males unless they use 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 is required to be used also by vasectomized men in order to prevent delivery of the drug via seminal fluid.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method ame: Percentage of patient preference for deferasirox FCT vs deferasirox DT;Timepoints: Week 48;Measure: week 48
- Secondary Outcome Measures
Name Time Method ame: Percentage of patient preference for deferasirox FCT vs deferasirox DT vs previous previous iron chelation;Timepoints: Week 28;Measure: Week 28;Name: Percentage of patient preference for deferasirox DT vs previous iron chelation;Timepoints: Week 4 and week 24;Measure: Week 4 and week 24;Name: Percentage of reasons for preference of deferasirox FCT vs. deferasirox DT;Timepoints: Week 28 and week 48;Measure: Week 28 and week 48;Name: Pill counts to assess drug compliance for deferasirox DT vs FCT;Timepoints: Baseline to wk 24, wk 25 to wk 48;Measure: Baseline to wk 24, wk 25 to wk 48