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

22 juli 2017 bijgewerkt door: 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

Studie Overzicht

Toestand

Voltooid

Conditie

Interventie / Behandeling

Studietype

Ingrijpend

Inschrijving (Werkelijk)

173

Fase

  • Fase 2

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studie Locaties

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

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

10 jaar en ouder (Kind, Volwassen, Oudere volwassene)

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Allemaal

Beschrijving

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

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: Gerandomiseerd
  • Interventioneel model: Parallelle opdracht
  • Masker: Geen (open label)

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Actieve vergelijker: 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.
Geneesmiddel: Deferasirox dispersible tablet
Deferasirox DT was provided as 125 mg, 250 mg and 500 mg dispersible tablets for oral use.
Andere namen:
  • ICL670, DT (dispersible tablet)
Experimenteel: 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
Geneesmiddel: Defearisox film-coated tablet
Deferasirox FCT was provided as 90 mg, 180 mg and 360 mg film-coated tablets for oral use.
Andere namen:
  • ICL670, FCT (film-coated tablet)

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Overall Safety as Measured by Frequency of Adverse Events
Tijdsspanne: 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
Tijdsspanne: 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

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Frequency of Selected Gastro-intestinal (GI) Adverse Events
Tijdsspanne: 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)
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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)
Tijdsspanne: 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)
Tijdsspanne: 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)
Tijdsspanne: 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
Tijdsspanne: 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

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

Novartis Pharmaceuticals

Publicaties en nuttige links

De persoon die verantwoordelijk is voor het invoeren van informatie over het onderzoek stelt deze publicaties vrijwillig ter beschikking. Dit kan gaan over alles wat met het onderzoek te maken heeft.

Algemene publicaties

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Werkelijk)

8 juli 2014

Primaire voltooiing (Werkelijk)

24 februari 2016

Studie voltooiing (Werkelijk)

24 februari 2016

Studieregistratiedata

Eerst ingediend

26 april 2014

Eerst ingediend dat voldeed aan de QC-criteria

26 april 2014

Eerst geplaatst (Schatting)

29 april 2014

Updates van studierecords

Laatste update geplaatst (Werkelijk)

25 juli 2017

Laatste update ingediend die voldeed aan QC-criteria

22 juli 2017

Laatst geverifieerd

1 juli 2017

Meer informatie

Termen gerelateerd aan deze studie

Trefwoorden

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • CICL670F2201

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

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