Polyostotic Fibrous DysplasiaNCT00001851
Bone Marrow Injection to Replace Diseased Bone in Polyostotic Fibrous Dysplasia and McCune-Albright Syndrome
An observational study of Polyostotic Fibrous Dysplasia, run by National Institute of Dental and Craniofacial Research (NIDCR) at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 4 years and older
- Conditions the study is for
- Polyostotic Fibrous Dysplasia
- How many people
- 50 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Dec 1998to 30 Apr 2002
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients will be otherwise healthy women, men, and children, with polyostotic fibrous dysplasia (PFD)/McCune-Albright Syndrome (MAS).
- Children will be greater than 4 years of age.
- The diagnosis will be based on evidence of the clinical spectrum of the disease and confirmed by the presence of typical findings on bone biopsy.
- All patients must be actively enrolled in the Screening and Natural History Protocol and possibly the Bisphosphonate Protocol.
- Patients will be enrolled in the study if they have a lytic lesion of the humerus, femur, or tibia which satisfies at least one of the following criteria: the lesion encompasses at least 50% of the width of the bone at that point on both AP and lateral radiographs; the lesion has caused a change in the anatomical shape and contour of the bone; the lesion has a bone density less than 50% of the density of the contralateral, normal side.
- Patients on previous or concomitant therapy are eligible for enrollment.
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- Patients on the Bisphosphonate Protocol must have received at least one month of treatment on that protocol before receiving a bone marrow injection in this protocol.
- Women in childbearing age will be offered barrier methods of contraception to decrease the risk of pregnancy for a period of one year following the transplant. Subjects who become pregnant during year one of the study will be transferred to the inactive participant group. Pregnancy testing will be performed prior to research procedures and surgery. Subjects using oral contraceptive agents are included. Women without normal menstrual cycles will be offered hormone replacement therapy for a period of 3 cycles prior to treatment.
- Subjects must agree to travel to NIH during the first 24 months and remain in the Bethesda area for the post-operative period as defined by protocol requirements.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
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).
Read more
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.
Read the 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.
- Conditions
- Polyostotic Fibrous Dysplasia
- Keywords
- Marrow, Grafting, Imaging, Injection, Aspiration, Bone Marrow Stromal Cells, Bone Grafting, Bone Graft Substitutes, Bio and Pharmacological Regulators of Bone Turnover
- Medical subject headings
- Fibrous Dysplasia, Polyostotic
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Nov 1999 (estimated)
- Study start15 Dec 1998
- Primary completion30 Apr 2002
- Study completion30 Apr 2002
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Polyostotic Fibrous DysplasiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Enrolment
- 50 participants
Sponsor and investigators
- Lead sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
- Sponsor type
- US National Institutes of Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00001851
- Sponsor's protocol ID
- 990003
- Other IDs
- 99-D-0003
- Status last verified
- 26 Jun 2009
- First submitted
- 3 Nov 1999
- Last updated
- 2 Jul 2017
- Results posted
- No
Publications
Danon M, Crawford JD. The McCune-Albright syndrome. Ergeb Inn Med Kinderheilkd. 1987;55:81-115. doi: 10.1007/978-3-642-71052-0_3. No abstract available.
PubMed 3545811Mastorakos G, Mitsiades NS, Doufas AG, Koutras DA. Hyperthyroidism in McCune-Albright syndrome with a review of thyroid abnormalities sixty years after the first report. Thyroid. 1997 Jun;7(3):433-9. doi: 10.1089/thy.1997.7.433.
PubMed 9226216Mauras N, Blizzard RM. The McCune-Albright syndrome. Acta Endocrinol Suppl (Copenh). 1986;279:207-17. doi: 10.1530/acta.0.112s207.
PubMed 3465162
Study at a glance
From the registry record, last updated by the sponsor on 2 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00001851
- Acronym / sponsor ID
- 990003
- Conditions
- Polyostotic Fibrous Dysplasia
- Keywords
- Marrow, Grafting, Imaging, Injection, Aspiration, Bone Marrow Stromal Cells, Bone Grafting, Bone Graft Substitutes
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 50 participants
- Lead sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)US National Institutes of Health
- Study start
- 15 Dec 1998
- Primary completion
- 30 Apr 2002 (actual)
- Study completion
- 30 Apr 2002 (actual)
- First posted
- 4 Nov 1999 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
Who may qualify
- Ages
- 4 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Patients will be otherwise healthy women, men, and children, with polyostotic fibrous dysplasia (PFD)/McCune-Albright Syndrome (MAS).
- Children will be greater than 4 years of age.
- The diagnosis will be based on evidence of the clinical spectrum of the disease and confirmed by the presence of typical findings on bone biopsy.
- All patients must be actively enrolled in the Screening and Natural History Protocol and possibly the Bisphosphonate Protocol.
- Patients will be enrolled in the study if they have a lytic lesion of the humerus, femur, or tibia which satisfies at least one of the following criteria: the lesion encompasses at least 50% of the width of the bone at that point on both AP and lateral radiographs; the lesion has caused a change in the anatomical shape and contour of the bone; the lesion has a bone density less than 50% of the density of the contralateral, normal side.
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jul 2017.
- Background: PubMed 3545811Danon M, Crawford JD. The McCune-Albright syndrome. Ergeb Inn Med Kinderheilkd. 1987;55:81-115. doi: 10.1007/978-3-642-71052-0_3. No abstract available.
- Background: PubMed 9226216Mastorakos G, Mitsiades NS, Doufas AG, Koutras DA. Hyperthyroidism in McCune-Albright syndrome with a review of thyroid abnormalities sixty years after the first report. Thyroid. 1997 Jun;7(3):433-9. doi: 10.1089/thy.1997.7.433.
- Background: PubMed 3465162Mauras N, Blizzard RM. The McCune-Albright syndrome. Acta Endocrinol Suppl (Copenh). 1986;279:207-17. doi: 10.1530/acta.0.112s207.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Polyostotic Fibrous Dysplasia, run by National Institute of Dental and Craniofacial Research (NIDCR) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Jul 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00001851, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Jul 2017.