A Randomized, Placebo-Controlled Trial of Alendronate in the Treatment of Polyostotic Fibrous Dysplasia and the McCune-Albright Syndrome
Fosamax (Alendronate)
Bone Diseases+3
+ Bone Diseases, Developmental
+ Fibrous Dysplasia of Bone
Treatment Study
Summary
Study start date: August 24, 1998
Actual date on which the first participant was enrolled.Polyostotic fibrous dysplasia (PFD) is a sporadic disorder which affects multiple sites in the skeleton. The bone at these sites is rapidly resorbed and replaced by abnormal fibrous tissue and mechanically abnormal bone. PFD may occur alone or as part of the McCune-Albright Syndrome (MAS), a syndrome originally defined by the triad of PFD, cafe-au-lait pigmentation of the skin, and precocious puberty. The bony lesions are frequently disfiguring and painful, and depending on the location of the lesion, they can cause significant morbidity. Lesions in weight-bearing bones can lead to disabling fractures, while lesions in the skull can lead to compression of vital structures such as cranial nerves. Currently there are no clearly-defined systemic therapies for this bone disease. Small, uncontrolled trials using the second generation bisphosphonate, pamidronate, suggest that bisphosphonates may be effective. This study is a phase 2, controlled, double blinded trial of the third generation oral bisphosphonate, alendronate for the treatment of fibrous dysplasia. We propose to show that treatment with alendronate will improve bone quality, decrease bone pain, decrease fractures, and, if the patient is referred to the companion bone grafting protocol, will allow for the regeneration of better quality bone.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.40 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.Over 6 Years
Range of ages for which participants are eligible to join.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
Criteria
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.
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 1 location
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, United StatesOpen National Institutes of Health Clinical Center, 9000 Rockville Pike in Google Maps