Open-label, Multicenter Study Assessing Preference for Deferasirox Film-coated Tablet Compared to Dispersible Tablet

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 Formulation

ClinicalTrials.gov Identifier: NCT02993224

Novartis Reference Number: CICL670FIC05

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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. 

Study Description

Study to evaluate patient preference of deferasirox FCT or deferasirox DT in patient with transfusion - dependent thalassemia or non-transfusion -dependent thalassemia as measured by preference questionnaire at Week 48

Condition 
Transfusion-dependent Thalassemia
Non-transfusion-dependent Thalassemia
Phase 
Phase 2
Overall status 
Active, not recruiting
Enrollment count 
151 participants
Start date 
Jul 27, 2017
Completion date 
Mar 26, 2021
Gender 
All
Age(s)
2 Years and older (Child, Adult, Older Adult)

Interventions

Drug
deferasirox dispersable tablet (DT)
DT will be provided as 125 mg, 250 mg, 500 mg dispersible tablets for oral use. The required number of DT should be stirred into water, apple juice or orange juice until fully dissolved once a day before 12 PM on empty stomach. The starting dose on Baseline Day 1 would be as following: Deferasirox DT of 20 mg/kg/day in transfusion-dependent thalassemia (TDT) Deferasirox DT of 10 mg/kg/day in non- transfusion-dependent thalassemia (NTDT),
Drug
deferasirox film coated tablet (FCT)
FCT will be provided as 90 mg, 180 mg, 360 mg dispersible tablets for oral use. FCT should be taken once a day before 12 PM with or after light meal. If there is difficulty in swallowing, pill may be crushed and sprinkled over soft food. The starting dose on Baseline The equivalent FCT starting dose on Week 25 is: Deferasirox FCT of 14 mg/kg/day in transfusion-dependent thalassemia (TDT) Deferasirox FCT of 7 mg/kg/day in non- transfusion-dependent thalassemia (NTDT).

Eligibility Criteria

Inclusion Criteria: 1. Prior to any screening procedures are performed, written informed consent/assent must be provided. For pediatric patients, consent will be obtained from parent(s) or legal patient's representative. Investigators will also obtain 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: a. Deferiprone/ DFP b. Deferoxamine /DFO c. Combination (DFO + DFP) 4. Subject is 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 if 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 may 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 (including use of electronic devices for ePRO). 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 are using effective methods of contraception during dosing of study treatment 14 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. 15. 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 Locations

Egypt
Novartis Investigative Site
-
Alexandria, 21131
-
Egypt
Novartis Investigative Site
-
Cairo, 11562
-
Egypt
Novartis Investigative Site
-
Zagazig, 44519
-
Egypt
Lebanon
Novartis Investigative Site
-
Hazmiyeh, PO BOX 213
Beirut
Lebanon
Morocco
Novartis Investigative Site
-
Rabat, 10102
-
Morocco
Saudi Arabia
Novartis Investigative Site
-
Al Ahsa,
SAU
Saudi Arabia
Novartis Investigative Site
-
Jeddah, 21159
-
Saudi Arabia
Novartis Investigative Site
-
Jeddah, 21589
-
Saudi Arabia
Novartis Investigative Site
-
Riyadh, 11117
-
Saudi Arabia
Thailand
Novartis Investigative Site
-
Bangkok noi, 10700
Bangkok
Thailand
Novartis Investigative Site
-
Bangkoknoi, 10700
Bangkok
Thailand
Novartis Investigative Site
-
Bangkok, 10400
-
Thailand
Turkey
Novartis Investigative Site
-
Ankara, 06100
-
Turkey
Novartis Investigative Site
-
Antalya, 07070
-
Turkey
Novartis Investigative Site
-
Istanbul, 34093
-
Turkey
Vietnam
Novartis Investigative Site
-
Hanoi,
-
Vietnam
Novartis Investigative Site
-
Ho Chi Minh City, DISTRICT 1
-
Vietnam

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Call 1-999-669-6682 or email [email protected]