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

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ClinicalTrials.gov Identifier: NCT02993224
Recruitment Status : Active, not recruiting
First Posted : December 15, 2016
Last Update Posted : October 12, 2020
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Brief Summary:
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 or disease Intervention/treatment Phase
Transfusion-dependent Thalassemia Non-transfusion-dependent Thalassemia Drug: deferasirox dispersable tablet (DT) Drug: deferasirox film coated tablet (FCT) Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 151 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Other
Official Title: 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
Actual Study Start Date : July 27, 2017
Estimated Primary Completion Date : February 26, 2021
Estimated Study Completion Date : March 26, 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia
Drug Information available for: Deferasirox

Arm Intervention/treatment
Experimental: Deferasirox DT followed by FCT

Deferasirox dispersable tablet (DT) will be provided for 24 weeks. At the completion of 24 weeks patients will be transitioned on Week 25 to an equivalent dose of the deferasirox film casted tablet (FCT) formulation and continue treatment to Week 48 (EOT of Core Phase).

Patients can then continue deferasirox FCT formulation as per the judgment of the investigator, through an extension phase for maximum of 12 months counting from last dose of deferasirox FCT received at the end of period 2 on Core Phase.

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).




Primary Outcome Measures :
  1. Proportion of patient preference for deferasirox FCT vs deferasirox DT at Week 48 [ Time Frame: Week 48 ]
    Proportion of patient claimed preference of deferasirox FCT over deferasirox DT as measured by preference questionnaire at Week 48


Secondary Outcome Measures :
  1. Proportion of patient preference for deferasirox FCT vs deferasirox DT vs previous previous iron chelation at week 28 [ Time Frame: Week 28 ]
    Proportion of patient claimed preference of deferasirox FCT, deferasirox DT, and previous iron chelation as measured by preference questionnaire at Week 28

  2. Proportion of patient preference for deferasirox DT vs previous iron chelation at week 4 and 24 [ Time Frame: Week 4 and Week 24 ]
    Proportion of patient claimed preference of deferasirox DT, over previous iron chelation as measured by preference questionnaire at Week 4 and Week 24

  3. Proportion of reasons for preference of deferasirox FCT vs. deferasirox DTat week 28 and 48 [ Time Frame: Week 28 and Week 48 ]
    Proportion of preference reasons for deferasirox FCT over deferasirox DT as measured by preference questionnaire at Week 28 and Week 48

  4. Pill counts to assess drug compliance for deferasirox DT vs FCT [ Time Frame: Baseline to week 24, Week 25 to 48 ]
    To evaluate the effect of deferasirox FCT compared with deferasirox DT on patient compliance using pill count at hospital level. Relative consumed pill count during deferasirox FCT (Week 25 to 48) compared with deferasirox DT (Baseline day 1 to 24)

  5. Change in scores of patients palatability preference at week 4, 24, 28 and 48 [ Time Frame: Screening, week 4, 24, 28 and 48 ]
    Change in scores on patient palatability using Palatability questionnaire which evaluates taste and aftertaste. Changes will be performed for both formulations (DT/FCT) for the overall score at Week 4, Week 24, Week 28 and Week 48 and overall score at Screening for iron chelators.

  6. Proportion of patients satisfaction in regards to the drug intake for deferasirox DT vs FCT at screening, week 4, 24, 28 and 48 [ Time Frame: Screening, week 4, 24, 28 and 48 ]
    To evaluate the effect of deferasirox FCT compared with deferasirox DT or previous iron chelation on patient satisfaction using Patient / Observer Reported Outcomes (PRO/ObsRO) questionnaires.

  7. Assess efficacy of deferasirox DT vs DCT by analysis of serum ferritin levels [ Time Frame: Baseline, every 4 weeks up to 48 weeks ]
    Change from baseline in serum ferritin on monthly basis from Day 1 Baseline, in both drugs: deferasirox DT vs FCT.



Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02993224


Locations
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Egypt
Novartis Investigative Site
Alexandria, Egypt, 21131
Novartis Investigative Site
Cairo, Egypt, 11562
Novartis Investigative Site
Zagazig, Egypt, 44519
Lebanon
Novartis Investigative Site
Hazmiyeh, Beirut, Lebanon, PO BOX 213
Morocco
Novartis Investigative Site
Rabat, Morocco, 10102
Saudi Arabia
Novartis Investigative Site
Al Ahsa, SAU, Saudi Arabia
Novartis Investigative Site
Jeddah, Saudi Arabia, 21159
Novartis Investigative Site
Jeddah, Saudi Arabia, 21589
Novartis Investigative Site
Riyadh, Saudi Arabia, 11117
Thailand
Novartis Investigative Site
Bangkok noi, Bangkok, Thailand, 10700
Novartis Investigative Site
Bangkoknoi, Bangkok, Thailand, 10700
Novartis Investigative Site
Bangkok, Thailand, 10400
Turkey
Novartis Investigative Site
Ankara, Turkey, 06100
Novartis Investigative Site
Antalya, Turkey, 07070
Novartis Investigative Site
Istanbul, Turkey, 34093
Vietnam
Novartis Investigative Site
Hanoi, Vietnam
Novartis Investigative Site
Ho Chi Minh City, Vietnam, DISTRICT 1
Sponsors and Collaborators
Novartis Pharmaceuticals
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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02993224    
Other Study ID Numbers: CICL670FIC05
First Posted: December 15, 2016    Key Record Dates
Last Update Posted: October 12, 2020
Last Verified: October 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
thalassemia
TDT
transfusion-dependent
non-transfusion-dependent
NTDT
Additional relevant MeSH terms:
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Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Deferasirox
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action