A Pharmacokinetic and Safety Study of IV Gallium Nitrate (Ganite) in Cystic Fibrosis Patients
Condition: Cystic Fibrosis
MeSH terms: Cystic Fibrosis; Fibrosis
Intervention: 100 mg/m2 dose; 200 mg/m2 dose
Intervention description: 5 day infusion of gallium nitrate (IV Ganite®) at a dose of 100 mg/m2/day; 5 day infusion of gallium nitrate (IV Ganite®) at 200 mg/m2/day
Intervention MeSH terms: Gallium nitrate
Phase: Phase 1
Study type: Interventional
Primary purpose: Other
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 20.0
Sex: All
Minimum age: 18 Years
Maximum age: 55 Years
Healthy volunteers: No
Keywords: IV Gallium; Gallium; Ganite; Cystic Fibrosis; Gallium Infusion

Summary:

      The purpose of this research study is to test the pharmacokinetics, safety, and tolerability
      of an intravenous infusion of a drug called Ganite (gallium nitrate) in patients with cystic
      fibrosis. We want to see this drug is safe and tolerable and to see if high levels of the
      drug are found in the sputum.

      Funding Source - Food and Drug Administration (FDA) Office of Orphan Products Development
      (OOPD)
    

Eligibility criteria:

        Inclusion Criteria:

          1. Adult male or female, between 18 and 55 years of age

          2. Documented chronic colonization with Pseudomonas Aeruginosa (Pa)

          3. Confirmed diagnosis of CF:

               1. Documented history of > 60 mmol/L chloride concentration in pilocarpine sweat
                  chloride test And/Or

               2. Genotype with two identifiable mutations consistent with CF, accompanied by one
                  or more phenotypic features consistent with diagnosis of CF

          4. Forced expiratory volume in the first second (FEV1) ≥ 30% of predicted value

          5. Able to expectorate sputum

          6. Serum liver function tests ≤ 2.5 x upper limit of normal

          7. Serum urea nitrogen (BUN) and creatinine ≤ 1.5 x upper limit of normal

          8. Serum creatinine ≤ 2.0 mg/dl

          9. Hemoglobin ≥ 9 g/dl, platelets ≥ 100,000/mm3, and white blood cells (WBC) ≥ 4,500/mm3
             and ≤ 15,000/mm3

         10. Ionized calcium ≥ the lower limit of normal

         11. Able to understand and sign the informed consent document, communicate with the
             Investigator, and comply with the requirements of the protocol

         12. If female and of childbearing potential, must have a negative pregnancy test on Day 1
             prior to receiving study drug

         13. If female and of childbearing potential, is willing to use adequate contraception, as
             determined by the investigator, for the duration of the study

        Exclusion Criteria:

          1. Acute pulmonary exacerbation requiring antibiotic intervention within 2 weeks prior to
             screening

          2. Osteoporosis defined as the most recent dexa scan within the prior 5 years with a
             T-score ≤ -2.5

          3. Pregnant or lactating female

          4. Known sensitivity to gallium

          5. Use of biphosphonates

          6. Use of any investigational drug and/or participated in any clinical trial within 3
             months prior to screening