A Phase 2 IV Gallium Study for Patients With Cystic Fibrosis (IGNITE Study)

This is a phase 2, multi-center, randomized, placebo-controlled trial in adults with CF
chronically infected with P. aeruginosa. The study will evaluate the safety and clinical
efficacy of a five day infusion of IV gallium nitrate (IV gallium). The purpose of this study
is to assess the efficacy of IV gallium to improve pulmonary function as measured by a 5% or
greater relative improvement in forced expiratory volume in one second (FEV1) from baseline
to Day 28.

Inclusion Criteria:

- Greater than or equal to 18 years of age at Screening

- Documented chronic colonization with P. aeruginosa defined as dentification in two
sputum or oropharyngeal cultures within the year prior to Day 1

- Documentation of a CF diagnosis as evidenced by one or more clinical features
consistent with the CF phenotype and one or more of the following criteria:

1. sweat chloride ≥ 60 mEq/liter by quantitative pilocarpine iontophoresis test
(QPIT)

2. two well-characterized mutations in the cystic fibrosis transmembrane conductance
regulator (CFTR) gene

3. Abnormal nasal potential difference (NPD; change in NPD in response to a low
chloride solution and isoproteronol of less than -5 mV)

- FEV1 ≥ 25 % of predicted value at Screening

- Able to expectorate sputum

- Serum liver function tests ≤ 2.5 x upper limit of normal at Screening

- Serum urea nitrogen (BUN) ≤ 1.5 x upper limit of normal at Screening

- Serum creatinine ≤ 2.0 mg/dl and ≤ 1.5 x upper limit of normal at Screening

- Hemoglobin ≥ 9 g/dl, platelets ≥ 100,000/mm3, and white blood cells (WBC)

≥ 4,500/mm3 at Screening

- Ionized calcium ≥ lower limit of normal at Screening

- Written informed consent obtained from subject or subject's legal representative

- Able to communicate with the Investigator and comply with the requirements of the
protocol

- If female and of childbearing potential, must have a negative pregnancy test on Day 1
prior to receiving study drug

- If female and of childbearing potential, is willing to use adequate contraception for
the duration of the study through Visit 5, as determined by the investigator

- If male and able to father a child, is willing to use adequate contraception for the
duration of the study through Visit 5, as determined by the investigator

- Clinically stable with no significant changes in health status within 14 days prior to
Day 1

Exclusion criteria:

- Use of inhaled antibiotics within seven days prior to Day 1

- Unable or unwilling to withhold use of chronic inhaled antibiotics through Day 28

- Use of intravenous, inhaled, or oral antibiotics for an acute indication within 14
days prior to Day 1

- Use of bisphosphonates within seven days prior to Day 1

- History of osteoporosis (defined as the most recent dexa scan with a T-score ≤ -2.5
with the dexa scan performed within the five years prior to Screening)

- Lactating female

- Known sensitivity to gallium