Growth Hormone Therapy in Osteogenesis Imperfecta
Condition: Osteogenesis Imperfecta
MeSH terms: Osteogenesis Imperfecta
Study type: Interventional
Phase: Phase 3

Summary: 
      Growth deficiency is a key feature of severe Osteogenesis Imperfecta (OI) and a frequent
      feature of mild to moderate forms of the disease. The reason that children with OI are short
      is not fully understood. We do know that details such as the number of fractures suffered or
      the type of OI do not fully explain the short stature of OI. Growth patterns have been
      defined for children with OI Types I, III, and IV. At about 12 months of age, children with
      Types III and IV OI demonstrate a predictable plateau of their linear growth rate. Type IV OI
      children begin to resume a normal growth rate at about age four to five years, but they will
      not "catch up" to a normal height, as they have "lost" a significant period of growth. The
      plateau usually continues for children with Type III OI. The reason for this growth plateau
      is unknown. There have been no studies which evaluate the growth of OI children in this age
      range. Our previous studies of growth in OI children have begun at age 5 years.

      We have studied growth in OI children for the past 10 years. Different medications have been
      tried to both stimulate growth and improve bone density. Some children have responded to
      growth hormone (their growth rate increased by at least 50%) and some did not. The majority
      of children who did respond were Type IV. However, we need to carefully treat and study more
      children to try to determine which children will benefit from growth hormone medication.

      The Goals of this Study Are:

        1. We want to try to find a cause for the growth plateau common in types III and IV OI.
           Long-term, our goal is to develop a treatment to eliminate this plateau.

        2. We want to see how long and how well OI bone will respond to growth stimulation.

        3. We hope to find a "predictor" for who will respond to growth hormone and who will not,
           by measuring your child's endocrine and growth hormone function before receiving any
           growth hormone treatment.

        4. We want to measure the effects of growth stimulation on bone density, and the quality of
           OI bone.

        5. We want to see if there are long term benefits resulting from this treatment in the form
           of final adult height, trunk height, and possibly improved function of the respiratory
           system.

      Median Subject Age (on p. 1 of webpage): 1-15 years (replaces 0-20)
    

Detailed description: 
      Growth deficiency is a cardinal feature of severe Osteogenesis Imperfecta (OI) and a frequent
      feature of mild to moderate forms of this disease. Despite the prevalence of short stature
      among people with OI, few studies have examined treatment options for this feature of OI.
      Recombinant human growth hormone (rGH) is a treatment for growth deficiency which we have
      investigated. In our initial studies we have found that many OI children are responsive to
      rGH especially those with type IV OI. The purpose of this protocol is to examine the effect
      of growth hormone treatment on linear growth of children with types III and IV OI and
      correlate growth responsiveness with growth hormone-somatomedin axis and histomorphometry
      parameters of OI bone.
    

Intervention name(s): Humatrope
Intervention type(s): Drug
Intervention description(s): Patients receive a subcutaneous injection.
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Osteogenesis Imperfecta