Growth Hormone Therapy for Wasting in Cystic Fibrosis
Condition: Cystic Fibrosis
MeSH terms: Cystic Fibrosis; Fibrosis
Intervention: Nutropin AQ
Intervention description: Based on recommendations from the 2007 GH Deficiency Consensus Workshop on adult GH deficiency, the recommended initiation of treatment for adult males is 0.2mg/d and for women 0.4mg/d, with a titration upwards based on insulin-like growth factor (IGF-1) (product of GH stimulation at target tissues) levels and patient response. IGF-1 will be monitored at the 3,4,5 and 11 month intervals. For subjects under the age of 25 with an open epiphysis of the hand and/or wrist we will treat with the dose of 0.3mg/kg/week. Subjects will be on growth hormone for 8 months with a baseline visit prior to initiation of therapy and a 3 month follow-up visit after stopping therapy.
Intervention MeSH terms: Hormones
Phase: Phase 1
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 5.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: CF

Summary:

      Our hypothesis is that Growth Hormone (GH) will not only target the peripheral tissue to
      stimulate weight and muscle growth which will maximize nutritional potential and improve
      overall quality of life. We theorize that this will occur through a multitude of factors:
      increased appetite, more constructive utilization of caloric intake and decreased catabolic
      signaling. The first aim will address changes in weight and lean body mass following the
      institution of GH therapy in adults with Cystic Fibrosis (CF) related wasting. The second aim
      will measure impact on quality of life of these individuals. Additionally, the third aim will
      monitor effects of GH therapy on diabetes and insulin sensitivity. Finally, the fourth aim
      will observe changes in the subjects underlying diagnosis of CF, specifically lung function,
      muscle strength and inflammatory state.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Ability to provide written informed consent and comply with study assessments for the
             full duration of the study.

          -  Age > 18 years

          -  Cystic fibrosis, diagnosed by either sweat chloride or genetic testing

          -  Less than 92% ideal body weight based on body mass index (BMI) of 22 for women and 23
             for men

          -  Moderate or better pulmonary function (Forced Expiratory Volume (FEV1) >40% of
             predicted).

          -  Agree to use an effective method of birth control to prevent pregnancy during the
             research study.

        Women should not nurse (breast feed) a baby while on this study because Nutropin AQ may
        enter breast milk and possibly harm the child.

        Exclusion Criteria:

          -  Pregnancy (positive pregnancy test) prior enrollment in the study

          -  Any other condition that the investigator believes would pose a significant hazard to
             the subject if the investigational therapy were initiated

          -  Participation in another simultaneous medical investigation or trial

          -  Pediatric patients

          -  Active neoplasm

          -  History of organ transplantation

          -  Prader Willi Syndrome who are severely obese or have severe respiratory impairment

          -  Patients with hepatic impairment resulting in abnormal coagulation studies (>1.5 times
             normal reference range)

          -  Poorly controlled diabetes as determined by a Hemoglobin A1c greater than or equal to
             9.0%.

          -  Individuals with electrocardiogram abnormality or cardiac pacing.