Polyostotic Fibrous Dysplasia Clinical Trial
Official title:
Evaluation and Treatment to Improve Bone Quality and Prevent Fractures by the Percutaneous Replacement of Diseased Tissue in Polyostotic Fibrous Dysplasia and McCune-Albright Syndrome
This study will evaluate the effectiveness of a new bone injection technique for treating
bone disease in patients with polyostotic fibrous dysplasia or McCune-Albright syndrome. In
these patients, some bones develop areas with much less mineral, making the bones more prone
to fracture or deformity and causing pain. This new treatment is intended to reduce the risk
of fracture, minimize deformities and improve overall function in these patients.
Patients 4 years of age and older with bone lesions that are highly likely to cause
significant pain and illness may be eligible for this 2-year study. Participants must be
simultaneously enrolled in NIDCR's research protocol 98-D-0145 (Screening and Natural History
of Patients with Polyostotic Fibrous Dysplasia and McCune-Albright Syndrome) or 98-D-0146 (A
Randomized, Placebo-Controlled Trial of Alendronate in the Treatment of Polyostotic Fibrous
Dysplasia and McCune-Albright Syndrome).
Within 14 days of the bone injection procedure, patients will have a medical history, routine
blood tests, urinalysis and check of vital signs (blood pressure, pulse and temperature) and
will complete a 30-minute quality-of-life questionnaire. Women of child-bearing potential
will have a pregnancy test. Patients who do not have recent X-rays and bone density scans
available for review will have new ones taken. When these studies are completed, patients
will undergo the bone injection procedure, followed immediately by bone densitometry and
coned-down X-rays, as follows:
- Bone injection - Patients will be given an anesthetic either to make them sleepy or put
them to sleep completely. A portion of bone marrow will be withdrawn through a needle
inserted into the hip bone and, at the same time, abnormal bone in the arms and legs
will be sucked out using a needle. The abnormal bone will be replaced with a mixture of
bone marrow and collagen (connective tissue protein) injected into the hole in the bone.
The areas of injection will be closed
- Bone densitometry - X-rays of the operated bone and opposite normal bone will be taken.
- Coned-down X-rays - Magnified normal X-rays will be taken as close-ups of an active
lesion.
Patients will have a history and physical examination by their local physician or at NIH
every month for the first 4 months after the procedure. Every 6 months after the procedure,
patients will return to NIH for follow-up, including a physical examination and completion of
a quality-of-life questionnaire. Imaging studies of the injected site will be done 3, 6, and
12 months after the procedure.
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. 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. In addition, 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 the cranial nerves.
Currently there are no clearly-defined systemic or local therapies for the bone disease, and
results of the use of conventional surgical treatment of sites of impending fracture have
been universally disappointing. In this study, we will treat osteolytic lesions in the long
bones of the upper and lower extremities, the sites of potential fracture, with a novel
surgical approach. This will involve 1) the removal of abnormal tissue through percutaneous
aspiration, and 2) the use of skeletal precursor cells taken from the unaffected sites, mixed
with a bone grafting substitute, and injected into the affected sites to bring about an
improvement in local bone quality and overall patient function.
;
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT00001181 -
Testolactone for the Treatment of Girls With LHRH Resistant Precocious Puberty
|
Phase 2 | |
Completed |
NCT00017927 -
A Study of the Effects of Pegvisomant on Growth Hormone Excess in McCune-Albright Syndrome
|
Phase 3 | |
Completed |
NCT00001973 -
Studies on Abnormal Bone From Patients With Polyostotic Fibrous Dysplasia and McCune Albright Syndrome
|
N/A | |
Completed |
NCT00006174 -
Effects of Letrozole on Precocious Puberty Due to McCune Albright Syndrome
|
Phase 1 | |
Completed |
NCT00001728 -
Alendronate to Treat Polyostotic Fibrous Dysplasia and McCune-Albright Syndrome
|
Phase 2 |