Clinical trial NCT00762918: Vitamin D3 for the Treatment of Low Vitamin D in Cystic Fibrosis
Official title: Vitamin D and Its Non-Classic Roles in Cystic Fibrosis
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE3
Conditions: Cystic Fibrosis
Vitamin D Deficiency
Keywords: cystic fibrosis
vitamin d
inflammation
Lead sponsor: Children's Hospital of Philadelphia
Sponsor class: OTHER
First posted: 2008-09-30
Enrollment: 0
Sex: ALL
Minimum age (years): 10
Maximum age (years): 25
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Vitamin D deficiency is common in cystic fibrosis. Vitamin D deficiency frequently persists despite aggressive treatment with ergocalciferol, a vitamin D preparation also known as vitamin D2. Cholecalciferol, a vitamin D preparation also known as vitamin D3,may work better to increase vitamin D levels.

Vitamin D is important for absorption of calcium from the diet and bone health. Vitamin D more recently has been found to play a role in regulating the normal inflammatory process. Since cystic fibrosis is a state of excessive inflammation, vitamin D may be playing a role in cystic fibrosis.

We hypothesize: cholecalciferol will work better to increase vitamin D levels in patients iwth cystic fibrosis and that it will have an effect on markers of inflammation.

Detailed description: Vitamin D deficiency is common in cystic fibrosis (CF) and persists despite relatively high doses of ergocalciferol, vitamin D2. Replacement has traditionally been focused upon maintenance of calcium and phosphorus homeostasis and bone health. However, non-classic roles of vitamin D have become increasingly recognized and the contribution of vitamin D deficiency to non-bone disorders has become apparent. Vitamin D deficiency has been associated with increased risk of a variety of cancers, autoimmune diseases such as Type 1 diabetes and multiple sclerosis, Type 2 diabetes, tuberculosis, and myopathy. The connection between vitamin D and these disease states likely reflects vitamin D's role as a transcriptional regulator: it participates in cell cycle regulation and in the innate immune system mediates cathelicidin production following activation of toll-like receptors.One hallmark of CF is pulmonary hyper-inflammation with recurrent infections. Additionally, malnutrition and decreased lean muscle mass threaten pulmonary function in CF. While vitamin D and its relation to bone has been explored in CF, the role of vitamin D in inflammation, lean body mass and strength, and pulmonary muscle strength has not been investigated. Moreover, vitamin D replacement has traditionally been with ergocalciferol, vitamin D2. Vitamin D3, cholecalciferol, has a longer half-life and is considered more potent. Thus, cholecalciferol treatment of children and young adults with CF and vitamin D deficiency may be useful for attaining normal vitamin D status and for exploring the impact of vitamin D upon lean body mass, pulmonary muscle strength, and inflammation.

Eligibility criteria: Inclusion Criteria:

1. Subjects age 10-25 years
2. cystic fibrosis
3. 25-(OH)-D \< 20 ng/mL
4. FEV1 \> 40% -

Exclusion Criteria:

1. inability to perform pulmonary function or hand-grip tests
2. liver disease (including cirrhosis and portal hypertension) or baseline liver enzymes 21/2-fold greater than the upper limit of normal
3. acute use of glucocorticoids at time of testing
4. acute pulmonary exacerbation at time of testing
5. known non-adherence to enzyme replacement
6. hypercalcemia
7. engages in "suntanning

Primary outcomes:
measure: serum 25-hydroxy vitamin D levels
timeFrame: 3 months
Secondary outcomes:
measure: body composition
timeFrame: 3 months
measure: inflammatory markers
timeFrame: 3 months
measure: muscles strength
timeFrame: 3 months
Other outcomes:
Locations:
Study officials:
affiliation: Children's Hospital of Philadelphia
name: Andrea Kelly, MD
role: PRINCIPAL_INVESTIGATOR
References: