Study of Skeletal Disorders

Description

Background: There are 461 conditions that affect the bones (skeletal disorders). Many of these are not well understood and do not have any specific treatments. Researchers want to collect more data about these conditions. Objective: To gain more information about the causes of skeletal disorders and how they progress over time. Eligibility: People ages 2 months or older with known or suspected skeletal disorders. Also, healthy family members of affected enrolled participants. Design: Participants can take part in the study either remotely or in person. Those who take part remotely may send in medical records, blood samples, photographs, and other materials. Participants medical records will be reviewed. They may give blood and/or urine samples. They will give blood, saliva, or tissue samples for genetic tests. They may have genetic counseling. Participants ages 2 years and older may have different kinds of imaging scans, such as x-rays. For these scans, they may have to lie still while machines take pictures of their bones. Participants with skeletal disorders who come to the clinic will be examined. They may be asked to stay in the hospital for a few days to take extra tests. They may have a bone or skin biopsy. Participants with skeletal disorders may be photographed to show the effects of their disorder and how it changes over time. For participants with skeletal disorders, their blood or tissue samples may be used to make a special type of stem cell. These cells can be used in the laboratory to make many other types of cells. A large supply of these cells may be created for research. Participation will last indefinitely.

Conditions

Skeletal Disorders

Study Overview

Study Details

Study overview

Background: There are 461 conditions that affect the bones (skeletal disorders). Many of these are not well understood and do not have any specific treatments. Researchers want to collect more data about these conditions. Objective: To gain more information about the causes of skeletal disorders and how they progress over time. Eligibility: People ages 2 months or older with known or suspected skeletal disorders. Also, healthy family members of affected enrolled participants. Design: Participants can take part in the study either remotely or in person. Those who take part remotely may send in medical records, blood samples, photographs, and other materials. Participants medical records will be reviewed. They may give blood and/or urine samples. They will give blood, saliva, or tissue samples for genetic tests. They may have genetic counseling. Participants ages 2 years and older may have different kinds of imaging scans, such as x-rays. For these scans, they may have to lie still while machines take pictures of their bones. Participants with skeletal disorders who come to the clinic will be examined. They may be asked to stay in the hospital for a few days to take extra tests. They may have a bone or skin biopsy. Participants with skeletal disorders may be photographed to show the effects of their disorder and how it changes over time. For participants with skeletal disorders, their blood or tissue samples may be used to make a special type of stem cell. These cells can be used in the laboratory to make many other types of cells. A large supply of these cells may be created for research. Participation will last indefinitely.

Clinical and Laboratory Study of Rare Skeletal Disorders

Study of Skeletal Disorders

Condition
Skeletal Disorders
Intervention / Treatment

-

Contacts and Locations

Bethesda

National Institutes of Health Clinical Center, Bethesda, Maryland, United States, 20892

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Individuals with a skeletal disorder that affects phosphate levels and/or metabolism
  • * Individuals with a skeletal disorder associated with skeletal overgrowth
  • * Individuals with a skeletal disorder with an unknown molecular basis or unknown etiology
  • 1. Have a known (via clinical, radiographic or molecular diagnosis) or suspected skeletal disorder
  • 2. State willingness to comply with study procedures and availability for the duration of the study
  • 3. Be age 2 months or older
  • 4. Be able to understand and sign informed consent document (or availability of a parent/guardian or LAR to provide written consent)
  • 1. Be an unaffected family member of an affected enrolled subject
  • 2. State willingness to comply with study procedures and availability for the duration of the study
  • 3. Be age 2 months or older
  • 4. Be able to understand and sign informed consent document (or availability of a parent/guardian)
  • 1. An individual who cannot or is unwilling to abide by the procedures of the protocol.
  • 2. An individual deemed to not have sufficient signs of skeletal disease.
  • 3. An individual who is pregnant.
  • 1. An individual who cannot or is unwilling to abide by the procedures of the protocol.
  • 2. An individual that shows signs of skeletal disease.
  • 3. An individual who is pregnant.

Ages Eligible for Study

2 Months to 100 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

Yes

Collaborators and Investigators

National Human Genome Research Institute (NHGRI),

Carlos R Ferreira Lopez, M.D., PRINCIPAL_INVESTIGATOR, National Human Genome Research Institute (NHGRI)

Study Record Dates

2026-07-31