Ignite Creation Date:
2025-12-24 @ 11:41 PM
Ignite Modification Date:
2026-01-04 @ 4:55 PM
Study NCT ID:
NCT00001851
Status:
COMPLETED
Last Update Posted:
2017-07-02
First Post:
1999-11-03
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Bone Marrow Injection to Replace Diseased Bone in Polyostotic Fibrous Dysplasia and McCune-Albright Syndrome
Sponsor:
National Institute of Dental and Craniofacial Research (NIDCR)
Organization:
Study Overview
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
Status:
COMPLETED
Status Verified Date:
2009-06-26
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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.
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.
Detailed Description:
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.
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.
Study Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| 99-D-0003 | None | None | View |