Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital Osteopetrosis
calcitriol
+ interferon gamma
Maladies osseuses+9
+ Maladies Osseuses Développementales
+ Maladies musculo-squelettiques
Étude thérapeutique
Résumé
Date de début de l'étude : 1 novembre 1999
Date à laquelle le premier participant a commencé l'étude.PROTOCOL OUTLINE: This is a randomized, placebo controlled, open label study. Patients are randomized to one of two arms (interferon gamma in combination with calcitriol or calcitriol alone). Arm I: Patients receive calcitriol once daily. Interferon gamma is administered by subcutaneous injection three times a week. Arm II: Patients receive calcitriol once daily. Patients may continue treatment in the absence of toxicity and disease progression. If disease progression is diagnosed in the control group, patients will then receive interferon gamma in combination with calcitriol. Patients are followed every 4 weeks.
Protocole
Cette section fournit des détails sur le plan de l'étude, y compris la manière dont l'étude est conçue et ce qu'elle évalue.30 participants à inclure
Nombre total de participants que l'essai clinique vise à recruter.Traitement
Éligibilité
Les chercheurs recherchent des patients correspondant à une certaine description appelée critères d'éligibilité : état de santé général ou traitements antérieurs du patient.Tout sexe
Le sexe biologique des participants éligibles à s'inscrire.Jusqu'à 10 ans
Tranche d'âge des participants éligibles à participer.Volontaires sains non autorisés
Indique si les individus en bonne santé et ne présentant pas la condition étudiée peuvent participer.Conditions
Pathologie
Critères
PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Biopsy and x-ray confirmed primary osteopetrosis Presence of anemia and/or cranial nerve compression --Prior/Concurrent Therapy-- Biologic therapy: No prior/concurrent bone marrow transplantation No prior interferon gamma No other investigational biologic agents Chemotherapy: No prior/concurrent chemotherapeutic agents for bone marrow transplantation Endocrine therapy: Prior/concurrent corticosteroid as supportive therapy allowed Radiotherapy: Not specified Surgery: At least 5 days since major surgery Other: Prior/concurrent calcitriol as supportive therapy allowed Prior/concurrent transfusion as supportive therapy allowed Prior/concurrent dietary therapy allowed --Patient Characteristics-- Age: 2 months to 10 years Performance status: Not specified Life expectancy: At least 6 months Hematopoietic: Not specified Hepatic: Bilirubin less than 2 mg/dL Renal: Creatinine less than 1.5 mg/dL OR Creatinine clearance greater than 50 mL/min Pulmonary: No uncorrected airway obstruction Other: No active infection requiring intravenous antibiotics No known seizure disorder not related to hypocalcemia No uncorrected hydrocephalus No MRI evidence of cerebral atrophy Must maintain or gain body weight No sleep apnea No thrombocytopenia No massive splenomegaly