Bone Marrow Injection to Replace Diseased Bone in Polyostotic Fibrous Dysplasia and McCune-Albright Syndrome

Completed

Conditions studied: Polyostotic Fibrous Dysplasia

In brief

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 A portion of bone marrow will be withdrawn through a needle inserted into the hip bone and, at the same time, abnormal bone in the arms and legs will be sucked out using a needle.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.

Key facts

Study ID

NCT00001851

Run by

National Institute of Dental and Craniofacial Research (NIDCR)

People needed

50

Starts

1998-12-15

Expected to finish

2002-04-30

Last updated by the study team

2017-07-02

Who can join

Age: 4 and older. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• Subjects will be excluded if they receive phenobarbital or related antiepileptic agents, including Dilantin or Tegretol.
• Medical problems which will preclude participation in the study include: pregnancy; chronic or active dermatological disease; chronic anemia (thalassemia, etc.); diabetes mellitus (fasting blood glucose in excess of 140 mg/dl); active or chronic pulmonary disease including COPD, chronic bronchitis, or asthma requiring medications; active or chronic gastrointestinal disease including gastric and duodenal ulcer disease and inflammatory bowel disease; history of cancer except for dermal lesions; intestinal malabsorption, chronic or active renal disease including a serum creatinine above 1.8 mg/dl; chronic or active hepatic disease including hepatitis; HIV infection.
• Subjects may not smoke more than one pack of cigarettes per day.
• Alcohol consumption must be less than 1.5 oz per day, without binge drinking.
• Subjects should have no dietary aberrations and no history of anorexia nervosa within the past 10 years.
• Subjects must be willing to receive transfusions of blood products if it is deemed medically necessary to preserve their well-being or their life.
• Patients will be excluded if they have a history of an allergy to all of the following antibiotics: penicillin, tetracycline, and cephalosporins.

Where it is running

• National Institutes of Health Clinical Center, 9000 Rockville Pike — Bethesda, Maryland, United States

Full record on ClinicalTrials.gov

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