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Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)

22 lipca 2017 zaktualizowane przez: Novartis Pharmaceuticals

A Randomized, Open-label, Multicenter, Two Arm, Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)

Assessed the new film-coated tablet formulation to the currently approved dispersible tablet formulation with regards to overall safety, Gastrointestinal (GI) tolerability, palatability, satisfaction and compliance

Przegląd badań

Status

Zakończony

Warunki

Interwencja / Leczenie

Typ studiów

Interwencyjne

Zapisy (Rzeczywisty)

173

Faza

  • Faza 2

Kontakty i lokalizacje

Ta sekcja zawiera dane kontaktowe osób prowadzących badanie oraz informacje o tym, gdzie badanie jest przeprowadzane.

Lokalizacje studiów

  • Arabia Saudyjska
      • Dammam, Arabia Saudyjska, 15215
        • Novartis Investigative Site
      • Dammam, Arabia Saudyjska, 40145
        • Novartis Investigative Site
      • Jeddah, Arabia Saudyjska, 21589
        • Novartis Investigative Site
      • Riyadh, Arabia Saudyjska, 11472
        • Novartis Investigative Site
  • Argentyna
      • Buenos aires, Argentyna, C1221ADC
        • Novartis Investigative Site
  • Austria
      • Linz, Austria, A-4010
        • Novartis Investigative Site
      • Wien, Austria, 1140
        • Novartis Investigative Site
  • Federacja Rosyjska
    • Russia
      • Moskow, Russia, Federacja Rosyjska, 117198
        • Novartis Investigative Site
  • Francja
      • Lille cedex, Francja, 59020
        • Novartis Investigative Site
      • Paris, Francja, 75010
        • Novartis Investigative Site
  • Grecja
    • GR
      • Athens, GR, Grecja, GR-115 27
        • Novartis Investigative Site
      • Patra - RIO, GR, Grecja, 265 04
        • Novartis Investigative Site
      • Thessaloniki, GR, Grecja, 546 42
        • Novartis Investigative Site
  • Hiszpania
      • Barcelona, Hiszpania, 08041
        • Novartis Investigative Site
      • Madrid, Hiszpania, 28033
        • Novartis Investigative Site
  • Liban
    • Beirut
      • Hazmiyeh, Beirut, Liban, PO BOX 213
        • Novartis Investigative Site
  • Malezja
      • Kuala Lumpur, Malezja, 50589
        • Novartis Investigative Site
      • Pulau Pinang, Malezja, 10990
        • Novartis Investigative Site
  • Meksyk
    • Distrito Federal
      • Mexico, Distrito Federal, Meksyk, 06726
        • Novartis Investigative Site
  • Niemcy
      • Berlin, Niemcy, 13353
        • Novartis Investigative Site
      • Dresden, Niemcy, 01307
        • Novartis Investigative Site
      • Goslar, Niemcy, 38642
        • Novartis Investigative Site
      • Hannover, Niemcy, 30170
        • Novartis Investigative Site
      • Leipzig, Niemcy, 04103
        • Novartis Investigative Site
      • Potsdam, Niemcy, 14467
        • Novartis Investigative Site
    • Baden-Württemberg
      • Mannheim, Baden-Württemberg, Niemcy, 68305
        • Novartis Investigative Site
  • Stany Zjednoczone
    • California
      • Orange, California, Stany Zjednoczone, 92868-3874
        • Children's Hospital of Orange County Onc Dept
    • Illinois
      • Chicago, Illinois, Stany Zjednoczone, 60611
        • Lurie Children's Hospital of Chicago Onc Dept
    • Massachusetts
      • Boston, Massachusetts, Stany Zjednoczone, 02115
        • Children's Hospital Boston Department of Hematology
    • New York
      • New York, New York, Stany Zjednoczone, 10021
        • Weill Cornell Medical College-Cornell University Onc Dept
    • Pennsylvania
      • Philadelphia, Pennsylvania, Stany Zjednoczone, 19104-4399
        • Children's Hospital of Philadelphia Onc. Dept
  • Tajlandia
      • Bangkok, Tajlandia, 10700
        • Novartis Investigative Site
      • Bangkok, Tajlandia, 10400
        • Novartis Investigative Site
  • Włochy
      • Napoli, Włochy, 80138
        • Novartis Investigative Site
    • BR
      • Brindisi, BR, Włochy, 72100
        • Novartis Investigative Site
    • CT
      • Catania, CT, Włochy, 95125
        • Novartis Investigative Site
    • FE
      • Cona, FE, Włochy, 44100
        • Novartis Investigative Site
    • GE
      • Genova, GE, Włochy, 16132
        • Novartis Investigative Site
      • Genova, GE, Włochy, 16128
        • Novartis Investigative Site
    • ITA
      • Cagliari, ITA, Włochy, 09121
        • Novartis Investigative Site
    • LE
      • Lecce, LE, Włochy, 73100
        • Novartis Investigative Site
    • MI
      • Milano, MI, Włochy, 20162
        • Novartis Investigative Site
      • Milano, MI, Włochy, 20122
        • Novartis Investigative Site
    • PA
      • Palermo, PA, Włochy, 90127
        • Novartis Investigative Site
      • Palermo, PA, Włochy, 90146
        • Novartis Investigative Site
    • RC
      • Reggio Calabria, RC, Włochy, 89100
        • Novartis Investigative Site
    • VR
      • Verona, VR, Włochy, 37126
        • Novartis Investigative Site
  • Zjednoczone Emiraty Arabskie
      • Al Ain - Abu Dhabi, Zjednoczone Emiraty Arabskie
        • Novartis Investigative Site
      • Dubai, Zjednoczone Emiraty Arabskie, 9115
        • Novartis Investigative Site
  • Zjednoczone Królestwo
      • London, Zjednoczone Królestwo, N19 5NF
        • Novartis Investigative Site
      • London, Zjednoczone Królestwo, NW1 2PJ
        • Novartis Investigative Site

Kryteria uczestnictwa

Badacze szukają osób, które pasują do określonego opisu, zwanego kryteriami kwalifikacyjnymi. Niektóre przykłady tych kryteriów to ogólny stan zdrowia danej osoby lub wcześniejsze leczenie.

Kryteria kwalifikacji

Wiek uprawniający do nauki

10 lat i starsze (Dziecko, Dorosły, Starszy dorosły)

Akceptuje zdrowych ochotników

Nie

Płeć kwalifikująca się do nauki

Wszystko

Opis

Key Inclusion Criteria:

  • Male and female patients aged ≥ 10 years
  • Patients with transfusion-dependent thalassemia and iron overload, requiring deferasirox DT at doses of ≥ 30 mg/kg/day as per the investigator's decision OR Patients with very low, low or intermediate (int) risk myelodysplastic syndrome (MDS) and iron overload, requiring deferasirox DT at doses of ≥ 20 mg/kg/day as per the investigator's decision.
  • History of transfusion of at least 20 PRBC units and anticipated to be transfused with at least 8 units of PRBCs annually during the study
  • Serum ferritin > 1000 ng/mL, measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria).

Key Exclusion Criteria:

  • Creatinine clearance below the contraindication limit in the locally approved prescribing information. Creatinine clearance will be estimated from serum creatinine at screening Visit 1 and screening Visit 2 and the mean value will be used for eligibility criteria.
  • Serum creatinine > 1.5 xULN at screening measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria).
  • ALT (SGPT) > 5xULN, unless LIC confirmed as >10 mg Fe/dw within 6 months prior to screening visit 1.
  • Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 mg/mg in a non-first void urine sample at screening Visit 1 or screening Visit 2.
  • 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).
  • Liver disease with severity of Child-Pugh Class B or C

Plan studiów

Ta sekcja zawiera szczegółowe informacje na temat planu badania, w tym sposób zaprojektowania badania i jego pomiary.

Jak projektuje się badanie?

Szczegóły projektu

  • Główny cel: Leczenie
  • Przydział: Randomizowane
  • Model interwencyjny: Przydział równoległy
  • Maskowanie: Brak (otwarta etykieta)

Broń i interwencje

Grupa uczestników / Arm
Interwencja / Leczenie
Aktywny komparator: Deferasirox dispersible tablet (DFX-DT)
Iron chelation naïve participants received DFX-DT 20 mg/kg/day once daily orally from weeks 1 - 4. After week 4, the dose could be adjusted by +/- 5 to 10 mg/kg/day, with a maximum dose of 40 mg/kg/day. Iron chelation pre-treated participants were supposed to start on a dose that was equivalent to their pre-washout dose.
Lek: Deferasirox dispersible tablet
Deferasirox DT was provided as 125 mg, 250 mg and 500 mg dispersible tablets for oral use.
Inne nazwy:
  • ICL670, DT (dispersible tablet)
Eksperymentalny: Deferasirox film-coated tablet (DFX-FCT)
Participants received DFX-FCT 14 mg/kg/day once daily orally from weeks 1 - 4. After week 4, the dose could be adjusted by +/- 3.5 to 7 mg/kg/day, with a maximum dose of 28 mg/kg/day. Iron chelation pre-treated participants were supposed to start on a dose that was equivalent to their pre-washout dose
Lek: Defearisox film-coated tablet
Deferasirox FCT was provided as 90 mg, 180 mg and 360 mg film-coated tablets for oral use.
Inne nazwy:
  • ICL670, FCT (film-coated tablet)

Co mierzy badanie?

Podstawowe miary wyniku

Miara wyniku
Opis środka
Ramy czasowe
Overall Safety as Measured by Frequency of Adverse Events
Ramy czasowe: 28 weeks
The percentage of participants with adverse events, serious adverse events and deaths was assessed.
28 weeks
Overall Safety as Measured by Changes in Laboratory Values From Baseline
Ramy czasowe: baseline (BL), 30 weeks
The percentage of participants with post-baseline laboratory values meeting specified criteria for notable/extended range was assessed. The following laboratory parameters were measured: platelet count, absolute neutrophils, serum creatinine , creatinine clearance, urinary protein/urinary creatinine ratio, alanine aminotransferase (ALT) and aspartate aminotransferase (AST). Note that within data categories, creat = creatinine, cons = consecutive, ULN = upper limit of normal and urin = urinary.
baseline (BL), 30 weeks

Miary wyników drugorzędnych

Miara wyniku
Opis środka
Ramy czasowe
Frequency of Selected Gastro-intestinal (GI) Adverse Events
Ramy czasowe: 28 weeks
The percentage of participants with any GI adverse event, diarrhea, constipation, nausea, vomiting, abdominal pain was assessed.
28 weeks
Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT)
Ramy czasowe: weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)
The modified SICT consisted of 13 items that represent 3 domains: adherence, satisfaction and concerns. The adherence domain consisted of 7 items, 6 which were measured using a 5-point response scale and was calculated by summing the 6 items. The score range from 6 to 30 and higher scores indicated worse adherence. The satisfaction domain consisted of 3 items, 2 which were measured using a 5-point response scale and was calculated by summing the 2 items. The score range from 2 to 10 and higher scores indicated worse satisfaction. The concerns domain consisted of 3 items to address any concerns or worries with his/her medication. All 3 items were measured on a 5-point response scale and were calculated by summing the 3 items. The score range from 3 to 15 and higher scores indicated fewer concerns. For all three domains, the meaningful difference between two treatment arms was determined to be 1 point.
weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)
Palatability Questionnaire Score
Ramy czasowe: weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)
The palatability questionnaire consisted of 4 items. The first item measured the taste and aftertaste of the medication and were scored a on a 5-point response scale. The second item offered an additional response option of "no aftertaste". The last 2 items referred to whether the medication was taken, i.e. swallowed or vomited, and how the participant perceived the amount of medication to be taken. The palatability summary score was calculated using a scoring matrix from items 1, 3 and 4 scores and the score ranges from 0 - 11. Higher scores indicated the best palatability. A meaningful difference between two treatment arms was determined to be 1 point.
weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)
Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary
Ramy czasowe: weeks -1, 4, 8, 12, 16, 20, 24
The GI symptom diary consisted of 6 items, five which were scored using a 0 - 10 rating scale with item appropriate anchors to rate the symptom, for example, Pain in your belly: 0 = no pain and 10 = worst pain. The GI diary summary score was created using the 10 point response scale for the 5 items. The GI symptom daily diary had a minimum score of 0 and a maximum score of 50. The weekly average score for the 7 days was calculated for each individual item and the GI summary score was created from these weekly averages. Higher scores indicated worse symptoms. A meaningful difference between two treatment arms was determined to be 0.3 point.
weeks -1, 4, 8, 12, 16, 20, 24
Number of Participants With Weekly Average Compliance of Medication Consumption
Ramy czasowe: Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24
A compliance questionnaire assessed whether the medication was taken. Weekly average compliance was calculated when there were at least four non-missing daily responses.
Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24
Weekly Dose Violation Rate
Ramy czasowe: weeks 1, 4, 8, 12, 16, 20, 24
The dose violation is defined as a dose either missed completely or not taken in accordance with the timing instruction (no later than 12:00 pm. The rate was calculated as [number of dose violations/drug exposure (days)] x 100.
weeks 1, 4, 8, 12, 16, 20, 24
Area Under the Plasma Concentration-time Curve From Time Zero to the Last Quantifiable Concentration (AUClast)
Ramy czasowe: week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Blood samples were collected to assess AUClast.
week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Observed Maximum Plasma Concentration Following Drug Administration (Cmax)
Ramy czasowe: week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Blood samples were collected to assess Cmax.
week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Time to Reach the Maximum Plasma Concentration After Drug Administration (Tmax)
Ramy czasowe: week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Blood samples were collected to assess Tmax.
week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Dererasirox Plasma Concentration
Ramy czasowe: Week 3, day 1, pre-dose (0 hour (h)) and 2 h post-dose; week 13, day 1, pre-dose (0 hour (h)) and 2 h post-dose; and week 21, day 1, pre-dose (0 hour (h)) and 2 h post-dose
Blood samples were collected to assess deferasirox concentration. Dose-adjusted calculations are presented: (concentration/actual dose)*20 for participants on DFX-DT and (concentration/actual dose)*14 for participants on DFX-FCT.
Week 3, day 1, pre-dose (0 hour (h)) and 2 h post-dose; week 13, day 1, pre-dose (0 hour (h)) and 2 h post-dose; and week 21, day 1, pre-dose (0 hour (h)) and 2 h post-dose

Współpracownicy i badacze

Tutaj znajdziesz osoby i organizacje zaangażowane w to badanie.

Sponsor

Novartis Pharmaceuticals

Publikacje i pomocne linki

Osoba odpowiedzialna za wprowadzenie informacji o badaniu dobrowolnie udostępnia te publikacje. Mogą one dotyczyć wszystkiego, co jest związane z badaniem.

Publikacje ogólne

Daty zapisu na studia

Daty te śledzą postęp w przesyłaniu rekordów badań i podsumowań wyników do ClinicalTrials.gov. Zapisy badań i zgłoszone wyniki są przeglądane przez National Library of Medicine (NLM), aby upewnić się, że spełniają określone standardy kontroli jakości, zanim zostaną opublikowane na publicznej stronie internetowej.

Główne daty studiów

Rozpoczęcie studiów (Rzeczywisty)

8 lipca 2014

Zakończenie podstawowe (Rzeczywisty)

24 lutego 2016

Ukończenie studiów (Rzeczywisty)

24 lutego 2016

Daty rejestracji na studia

Pierwszy przesłany

26 kwietnia 2014

Pierwszy przesłany, który spełnia kryteria kontroli jakości

26 kwietnia 2014

Pierwszy wysłany (Oszacować)

29 kwietnia 2014

Aktualizacje rekordów badań

Ostatnia wysłana aktualizacja (Rzeczywisty)

25 lipca 2017

Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości

22 lipca 2017

Ostatnia weryfikacja

1 lipca 2017

Więcej informacji

Terminy związane z tym badaniem

Słowa kluczowe

Dodatkowe istotne warunki MeSH

Inne numery identyfikacyjne badania

  • CICL670F2201

Te informacje zostały pobrane bezpośrednio ze strony internetowej clinicaltrials.gov bez żadnych zmian. Jeśli chcesz zmienić, usunąć lub zaktualizować dane swojego badania, skontaktuj się z register@clinicaltrials.gov. Gdy tylko zmiana zostanie wprowadzona na stronie clinicaltrials.gov, zostanie ona automatycznie zaktualizowana również na naszej stronie internetowej .

Badania kliniczne na Deferasirox dispersible tablet

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