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 février 2017 mis à jour par: 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.
Aperçu de l'étude
Statut
Complété
Les conditions
Intervention / Traitement
Type d'étude
Interventionnel
Inscription (Réel)
30
Phase
- Phase 2
Contacts et emplacements
Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.
Lieux d'étude
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Athens, Grèce
- Novartis Investigative Site
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Ancona, Italie
- Novartis Investigative Site
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Brindisi, Italie
- Novartis Investigative Site
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Cagliari, Italie
- Novartis Investigative Site
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Cosenza, Italie
- Novartis Investigative Site
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Firenze, Italie
- Novartis Investigative Site
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Milano, Italie
- Novartis Investigative Site
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Modena, Italie
- Novartis Investigative Site
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Napoli, Italie
- Novartis Investigative Site
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Torino, Italie
- Novartis Investigative Site
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California
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Oakland, California, États-Unis, 94609
- Children's Hospital and Research Center - Oakland
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Orange, California, États-Unis, 92868
- Children's Hospital of Orange County
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Massachusetts
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Boston, Massachusetts, États-Unis, 02115
- Children's Hospital Boston
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New York
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Jamaica, New York, États-Unis, 11432
- Queens Hospital Center
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New York, New York, États-Unis, 11215
- New York Methodist Hospital
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New York, New York, États-Unis, 10021
- New York Presbyterian Hospital/Weill Medical College of Cornell University
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Ohio
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Cincinnatti, Ohio, États-Unis, 45229
- Cincinnatti Children's Hospital Medical center
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Texas
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Houston, Texas, États-Unis, 77030
- Novartis Investigative Site
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Critères de participation
Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.
Critère d'éligibilité
Âges éligibles pour étudier
2 ans et plus (Enfant, Adulte, Adulte plus âgé)
Accepte les volontaires sains
Non
Sexes éligibles pour l'étude
Tout
La description
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.
Plan d'étude
Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.
Comment l'étude est-elle conçue ?
Détails de conception
- Objectif principal: Traitement
- Répartition: N / A
- Modèle interventionnel: Affectation à un seul groupe
- Masquage: Aucun (étiquette ouverte)
Armes et Interventions
Groupe de participants / Bras |
Intervention / Traitement |
|---|---|
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Expérimental: ICL670
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Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Délai |
|---|---|
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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.
Délai: 0 - 163 weeks
|
0 - 163 weeks
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Mesures de résultats secondaires
Mesure des résultats |
Délai |
|---|---|
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To estimate the absolute and relative change of liver iron concentration (LIC), to be measured using appropriate methodology available at individual centers.
Délai: Yearly
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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.
Délai: Quarterly
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Quarterly
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To evaluate the relationship between changes in LIC and serum ferritin, transferring saturation and serum iron.
Délai: Yearly
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Yearly
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Collaborateurs et enquêteurs
C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.
Parrainer
Publications et liens utiles
La personne responsable de la saisie des informations sur l'étude fournit volontairement ces publications. Il peut s'agir de tout ce qui concerne l'étude.
Liens utiles
Dates d'enregistrement des études
Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.
Dates principales de l'étude
Début de l'étude
1 juin 2003
Achèvement primaire (Réel)
1 décembre 2007
Dates d'inscription aux études
Première soumission
5 janvier 2010
Première soumission répondant aux critères de contrôle qualité
6 janvier 2010
Première publication (Estimation)
7 janvier 2010
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
23 février 2017
Dernière mise à jour soumise répondant aux critères de contrôle qualité
21 février 2017
Dernière vérification
1 février 2017
Plus d'information
Termes liés à cette étude
Mots clés
Termes MeSH pertinents supplémentaires
Autres numéros d'identification d'étude
- CICL670A0117
- 2004-002303-32 EudraCT number (Identificateur de registre: 2004-002303-32)
Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .