A Randomized, Placebo-Controlled Trial of Alendronate in the Treatment of Polyostotic Fibrous Dysplasia and the McCune-Albright Syndrome

Fecha de inicio: 24 de agosto de 1998

INCLUSION CRITERIA: All patients must be concomitantly enrolled in the companion Screening and Natural History protocol. Any patient with at least 2 active fibrous dysplastic lesions of either the cranial, axial, or appendicular skeleton will be eligible for consideration for inclusion in the study. The diagnosis will be based on evidence typical findings on bone biopsy (performed during the "Screening" protocol). Final consideration for enrollment will depend on diagnosis at the NIH. Patients must be at least 6 years old. Patients may be of child-bearing age, but will be expected to be on a nonhormonal form of birth control that gives a 95% protection rate. If a patient becomes pregnant during the course of the study, they must withdraw but will be eligible for re-enrollment upon the completion of pregnancy and lactation. Patients on previous of concomitant therapy are eligible for enrollment. However, patients who have received previous treatment with a bisphosphonate must wait one year from the completion of the last course before they can be enrolled. EXCLUSION CRITERIA: Patient, child or parents unwilling to fully cooperate with the evaluation as outlined in the schedule and consent form and do not give informed consent. Any sexually active patient that is unwilling to use an appropriate contraceptive associated with a pregnancy-prevention rate of 95% or greater. Pregnancy is an absolute contraindication to be evaluated or admitted to the study and is grounds for removal from the study. However, patients may be re-enrolled once pregnancy and lactation are completed. Severe esophageal motility problems may put patients at increased risk for complications from alendronate and are not eligible for the study. Significant comorbidities such as decompensated heart failure or diabetes mellitus, renal or hepatic failure, or decompensated psychiatric conditions exclude patients from enrollment. Patients with either a history of sarcoma of the bone or who have a FD lesion that undergoes sarcomatous degeneration while enrolled in either this study or any of the companion protocols.