Denne siden ble automatisk oversatt og nøyaktigheten av oversettelsen er ikke garantert. Vennligst referer til engelsk versjon for en kildetekst.

Deferasirox BID (Twice a Day) in Transfusion Dependent Thalassemia Patients With Inadequate Response to High Doses

19. april 2017 oppdatert av: Novartis Pharmaceuticals

A Prospective Single Arm Study to Assess the Efficacy and Safety of Deferasirox 20 mg/kg BID in Transfusion Dependent Thalassemia Patients Inadequately Responding to Current Treatment With Doses > 35mg/kg QD (Once a Day).

This is an open label, single arm, and multicenter study. The study will include the following phases. A screening phase which lasts for 4 weeks to determine patient eligibility. This phase will be followed by a 24 week Open label treatment phase. The study treatment is defined as deferasirox 20mg/kg BID 9Twice a day). Serum Ferritin Levels and MRI (Magmetic Resonance Imaging) LIC (Liver Iron Concentration) will be measured to evaluate the response to BID.

Studieoversikt

Status

Tilbaketrukket

Forhold

Intervensjon / Behandling

Detaljert beskrivelse

Study treatment is defined as deferasirox 20 mg/kg BID. The study treatment duration is 24 weeks. After the baseline visit, patient visits will occur weekly during the first month because key safety parameters need to be performed weekly in the first month of treatment and then every 4 weeks thereafter until week 24 End of Treatment (EOT).

Patients will have their first dose of study treatment at Visit 3. Safety assessments are routinely performed including collection of AEs (Adverse Events), SAEs (Serious ADverse Events<, vital signs, physical examination, ECG (Electrocardiograph), hematological and biochemistry assessments.

Patients will continue therapy until intolerable toxicity, patient decision or after 24 weeks treatment duration at which point and End of Treatment (EOT) visit will be performed and the End of Treatment CRF( Case Report Form) will be completed.

30 day Safety follow-up Patients who discontinue study drug before completing the study should be scheduled for a visit as soon as possible, at which time all of the assessments listed for the final visit will be performed. At a minimum, all patients who discontinue study treatment, including those who refuse to return for a final visit, will be contacted for safety evaluations during the 30 days following the last dose of study drug.

All patients must be followed for AEs and SAEs that may have occurred after discontinuation from the study treatment. The safety follow-up visit will take place 30 days after the last dose of study drug.

Studietype

Intervensjonell

Fase

  • Fase 2

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

2 år og eldre (Barn, Voksen, Eldre voksen)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Alle

Beskrivelse

Inclusion Criteria:

  • Written informed consent must be obtained prior to any screening procedures
  • Male or female aged ≥ 2 years at screening
  • Patients with transfusion dependent thalassemia
  • Patients confirmed as inadequate- responders to deferasirox > 35 mg/kg QD and treated with QD for at least 6 months
  • Regular transfusion indicated by a blood requirement ≥ 8 blood transfusions per year at screening
  • Serum ferritin level > 2,500 ng/mL at screening (two consecutive values at least 2 weeks apart from each other)
  • One SF measurement > 2,500 ng/mL between 6 and 9 months prior to study enrollment
  • Three SF measurements > 2,500 ng/mL, performed at least 3 weeks apart from each other, during the 6 month treatment with QD dosing of deferasirox prior to study enrollment
  • The average of the two screening SF values (collected 2 weeks apart from each other) must not show a decrease from the 6 to 9 month SF value taken prior to study enrollment
  • The average of the two screening SF values (collected 2 weeks apart from each other) must not show a decrease from each of the three SF values obtained during the 6 months of deferasirox QD treatment prior to study enrollment
  • LIC ≥ 7 mg Fe/g dw measured at the screening visit, (this value will be used as a baseline measurement

Exclusion Criteria:

  • Patients who are intolerant to > 35 mg/kg/day QD of deferasirox in the 6 months prior to study enrollment
  • Patients with mean levels of ALT >5 x ULN
  • Patients with serum creatinine above the upper limit of normal (ULN)
  • Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 (mg/mg)
  • Creatinine clearance ≤ 60 ml/min
  • Chronic hepatitis B infection, active hepatitis C infection
  • History of a positive HIV test
  • Uncontrolled systemic hypertension
  • Patients participating in another clinical trial or receiving a systemic investigational drug within the past 4 weeks or topical investigational drug within the past 7 days of screening
  • History of non-compliance with medical regimens or patients who are considered potentially unreliable and/or not cooperative, unwilling or unable to comply with the protocol
  • History of hypersensitivity to any of the study drug or excipients
  • 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.)
  • History of drug or alcohol abuse within the 12 months prior to enrollment.

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Ikke-randomisert
  • Intervensjonsmodell: Enkeltgruppeoppdrag
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: Deferasirox
Deferasirox 20mg/kg taken BID
Legemiddel: Deferasirox
Deferasirox 20mg/kg taken BID

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Mean relative change in SF (Serum Ferritin)
Tidsramme: Baseline, 24 Weeks
To evaluate the efficacy of a twice-daily dose regimen of deferasirox in inadequate-responders to a once daily dose regimen as measured by relative change in serum ferritin from baseline to 24 weeks of treatment
Baseline, 24 Weeks

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Mean Absolute/relative change in LIC
Tidsramme: Baseline, 24 Weeks
To evaluate the efficacy of BID deferasirox in liver iron removal as measured by MRI by absolute/relative change from baseline to 24 weeks of treatment
Baseline, 24 Weeks

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Novartis Pharmaceuticals

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart

1. februar 2014

Primær fullføring (Forventet)

1. februar 2016

Studiet fullført (Forventet)

1. februar 2016

Datoer for studieregistrering

Først innsendt

19. september 2013

Først innsendt som oppfylte QC-kriteriene

19. september 2013

Først lagt ut (Anslag)

24. september 2013

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

20. april 2017

Siste oppdatering sendt inn som oppfylte QC-kriteriene

19. april 2017

Sist bekreftet

1. januar 2014

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • CICL670A2420
  • 2013-002624-16 (EudraCT-nummer)

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

Kliniske studier på Deferasirox

Søk i lignende forsøk

Sponsorer og samarbeidspartnere

Medisinsk tilstand

Legemiddelintervensjoner

CROs by country

CROs in Kyrgyzstan

Forhold

Sjeldne sykdommer

Legemiddelintervensjoner

Kosttilskudd

Sponsor / samarbeidspartnere

Steder

3
Abonnere