A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity
21. februar 2017 oppdatert av: Novartis Pharmaceuticals
The purpose of this open-label, non-comparative, multi-center protocol was to further evaluate safety and to provide treatment with ICL670 to patients who had or were at risk of life threatening complications due to transfusional iron overload with a documented inability to tolerate any of the commercially available iron chelators due to severe toxicity rendering continued therapy either impossible or hazardous.
Patients who were also ineligible for all on-going registration trials with ICL670 were included in the study.
In exceptional cases, patients with a degree of iron overload which was not immediately life-threatening and who were ineligible for the registration trials were also enrolled provided they had a well-documented, sound justification for alternative chelation therapy.
Studieoversikt
Status
Fullført
Forhold
Intervensjon / Behandling
Studietype
Intervensjonell
Registrering (Faktiske)
30
Fase
- Fase 2
Kontakter og plasseringer
Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.
Studiesteder
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California
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Oakland, California, Forente stater, 94609
- Children's Hospital and Research Center - Oakland
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Orange, California, Forente stater, 92868
- Children's Hospital of Orange County
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Massachusetts
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Boston, Massachusetts, Forente stater, 02115
- Children's Hospital Boston
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New York
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Jamaica, New York, Forente stater, 11432
- Queens Hospital Center
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New York, New York, Forente stater, 11215
- New York Methodist Hospital
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New York, New York, Forente stater, 10021
- New York Presbyterian Hospital/Weill Medical College of Cornell University
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Ohio
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Cincinnatti, Ohio, Forente stater, 45229
- Cincinnatti Children's Hospital Medical center
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Texas
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Houston, Texas, Forente stater, 77030
- Novartis Investigative Site
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Athens, Hellas
- Novartis Investigative Site
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Ancona, Italia
- Novartis Investigative Site
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Brindisi, Italia
- Novartis Investigative Site
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Cagliari, Italia
- Novartis Investigative Site
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Cosenza, Italia
- Novartis Investigative Site
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Firenze, Italia
- Novartis Investigative Site
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Milano, Italia
- Novartis Investigative Site
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Modena, Italia
- Novartis Investigative Site
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Napoli, Italia
- Novartis Investigative Site
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Torino, Italia
- Novartis Investigative Site
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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:
- Patients had to be at risk of life-threatening complications due to transfusional iron overload and be unable to tolerate therapy with any of the commercially available iron chelators (mainly deferoxamine and/or deferiprone) because of documented severe toxicity.
- Patients with a degree of iron overload which was not immediately life-threatening and who were ineligible for other trials with ICL670 could also be enrolled providing they had a well-documented, sound justification for alternative chelation therapy.
- Serum ferritin ≥ 8000 μg/L.
- Serum ferritin < 8000μg/L and LIC of ≥ 7 mg Fe/g dry weight.
- Patients for whom ≥ 8 blood transfusions per year were required in order to maintain the Hemoglobin level at > 9 g/dL.
- Female patients who have reached menarche and who were sexually active had to use double barrier contraception (oral plus barrier contraception), or had to have undergone total hysterectomy and/or ovariectomy, or tubal ligation.
- Written, voluntary informed consent.
Exclusion Criteria:
- Patients with transfusional iron overload who were not experiencing severe toxicities during therapy with other iron chelators (e.g. deferoxamine and/or deferiprone).
- Patients with non-transfusional hemosiderosis.
- Patients with severe liver failure as defined by a score of ≥ 10 points on the Child-Pugh scale.
- Patients with serum creatinine 1.5 times the upper limit of normal (ULN) at screening.
- Patients with a history of nephrotic syndrome.
- Patients with a diagnosis of clinically relevant cataract or a previous history of clinically relevant ocular toxicity related to iron chelation therapy.
- Patients with severe systemic diseases unrelated to iron overload and which would prevent them from undergoing treatment with ICL670.
- Patients with psychiatric or addictive disorders which prevent them from giving informed consent or undergoing treatment with ICL670.
- Pregnant or breast feeding patients.
- Patients treated with systemic investigational drugs within the past four weeks or topical investigational drugs within the past seven days.
Any surgical or medical condition which might significantly alter the absorption or excretion of drugs as shown by evidence of any of the following:
- History of inflammatory bowel disease
- History of major gastrointestinal tract surgery such as gastrectomy, gastroenterostomy, or bowel resection
- History of pancreatic injury or pancreatitis; indication of impaired pancreatic function/injury as indicated by abnormal lipase or amylase
- Patients being considered by the investigator as potentially unreliable and/or not cooperative with regard to the protocol.
- History of drug or alcohol abuse within the 12 months prior to dosing.
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: N/A
- Intervensjonsmodell: Enkeltgruppeoppdrag
- Masking: Ingen (Open Label)
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
|---|---|
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Eksperimentell: ICL670
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Hva måler studien?
Primære resultatmål
Resultatmål |
Tidsramme |
|---|---|
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To evaluate the safety profile and to provide treatment with ICL670 for patients with or at risk of life-threatening complications due to transfusional iron overload who are unable to tolerate other iron chelators because of documented severe toxicity.
Tidsramme: 0 - 163 weeks
|
0 - 163 weeks
|
Sekundære resultatmål
Resultatmål |
Tidsramme |
|---|---|
|
To estimate the absolute and relative change of liver iron concentration (LIC), to be measured using appropriate methodology available at individual centers.
Tidsramme: Yearly
|
Yearly
|
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To evaluate the role of serum ferritin, serum iron, transferrin and transferrin saturation in monitoring iron burden in these patients.
Tidsramme: Quarterly
|
Quarterly
|
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To evaluate the relationship between changes in LIC and serum ferritin, transferring saturation and serum iron.
Tidsramme: Yearly
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Yearly
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Samarbeidspartnere og etterforskere
Det er her du vil finne personer og organisasjoner som er involvert i denne studien.
Sponsor
Publikasjoner og nyttige lenker
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Hjelpsomme linker
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. juni 2003
Primær fullføring (Faktiske)
1. desember 2007
Datoer for studieregistrering
Først innsendt
5. januar 2010
Først innsendt som oppfylte QC-kriteriene
6. januar 2010
Først lagt ut (Anslag)
7. januar 2010
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
23. februar 2017
Siste oppdatering sendt inn som oppfylte QC-kriteriene
21. februar 2017
Sist bekreftet
1. februar 2017
Mer informasjon
Begreper knyttet til denne studien
Nøkkelord
Ytterligere relevante MeSH-vilkår
Andre studie-ID-numre
- CICL670A0117
- 2004-002303-32 EudraCT number (Registeridentifikator: 2004-002303-32)
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. .