Safety and Efficacy of an Antioxidant-rich Multivitamin Supplement in Cystic Fibrosis
Condition: Cystic Fibrosis
MeSH terms: Cystic Fibrosis; Fibrosis
Study type: Interventional
Phase: Phase 1/Phase 2

Summary: 
      The purpose of this study is to test the safety and efficacy of the final commercial
      formulation of an antioxidant enriched multivitamin supplement in softgel capsule form
      (AquADEKs) in increasing the plasma levels of certain nutrients and antioxidants in
      individuals with cystic fibrosis.

      Hypothesis: An oral antioxidant-rich multivitamin supplement (AquADEKs), which uses a
      Generally Regarded As Safe (GRAS) molecule to form micelle-like vehicles, will safely
      increase systemic levels of beta-carotene, coenzyme Q10, and gamma-tocopherol, decrease
      PIVKA-II levels, while maintaining levels of vitamins A and D in the normal range in persons
      with CF > 10 years of age.
    

Detailed description: 
      In cystic fibrosis (CF), pancreatic insufficiency and a diminished bile acid pool cause
      malabsorption of important nutrients and dietary components leading to poor nutritional
      status and oxidative stress. Of particular significance is the malabsorption of fat-soluble
      nutrients, such as vitamins A, D, E and K which are critical for normal metabolic functions.
      Furthermore this malabsorption prevents individuals with CF from adequately absorbing and
      maintaining levels of lipophilic nutrients and antioxidants such as beta-carotene, coenzyme
      Q10 (CoQ10) and gamma-tocopherol which may provide benefits when supplied at levels higher
      than those obtained from normal diets. Current standard of care supplementation often does
      not normalize the blood levels of certain vitamins and antioxidants.

      An oral formulation, which can form micelle-like vehicles, can be used to overcome the
      malabsorption of these nutrients in CF patients. A pilot study of a prototype formulation
      showed both safety and efficacy in increasing systemic levels of target nutrients. This study
      will test the safety and efficacy of the final commercial formulation (AquADEKs) in the form
      of a softgel capsule in increasing the plasma levels of certain nutrients and antioxidants.

      Hypothesis: An oral antioxidant-rich multivitamin supplement (AquADEKs), which uses a
      Generally Regarded As Safe (GRAS) molecule to form micelle-like vehicles, will safely
      increase systemic levels of beta-carotene, coenzyme Q10, and gamma-tocopherol, decrease
      PIVKA-II levels, while maintaining levels of vitamins A and D in the normal range in persons
      with CF > 10 years of age.

      Specific Aims:

        1. To evaluate the safety of AquADEKs by monitoring patient reported symptoms and adverse
           events, and following vitamin and antioxidant levels, particularly vitamin A, to ensure
           that they do not exceed normative ranges after supplementation.

        2. To determine the efficacy of AquADEKs in increasing the antioxidants beta-carotene,
           CoQ10, and γ-tocopherol and maintaining plasma levels of vitamins A, D, α-tocopherol and
           PIVKA-II (surrogate of vitamin K status) in the normal range.
    

Intervention name(s): AquADEK
Intervention type(s): Dietary Supplement
Intervention description(s): Two AquADEK softgel vitamins on a daily basis x 12 weeks
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: cystic fibrosis; vitamins; antioxidants; anthropometric measurements; pulmonary function