Cysteamine for Asthma

Asthma affects 25.7 million people in the US. Many people report symptoms despite taking
high-doses of inhaled asthma medications. This difficult-to-treat group accounts for more
than 50% of asthma related healthcare visits and hospitalizations. The purpose of this study
is to see if a medicine called Cysteamine, given along with standard asthma care, will
improve asthma symptoms and lung function.

This study is a double-blind, placebo-controlled, randomized trial of cysteamine. A placebo
arm will be included to determine the comparative effectiveness of cysteamine in this
population. Eligible participants will be assessed 1, 4 and 8 weeks post randomization and
followed for an additional 4 weeks post treatment.

In order to enroll in this study, participants must be between the ages of 18-45 and have
uncontrolled asthma.

This study will include 4 visits and weekly phone calls, and will be in the study for 3-4
months.

During the treatment period, participants will be placed in one of two treatment groups:

- Cysteamine

- Placebo

Participants will not be able to choose which group they are assigned. This assignment is
random and by chance, much like flipping a coin. Participants will not know if they are
receiving Cysteamine or placebo. Investigators will compare the study results between the
participants of each group.

Inclusion Criteria:

- Male or female

- Age ≥ 18 years and ≤ 45 years

- Provision of written informed consent

- Asthma currently treated with moderate-high doses of Inhaled Corticosteroids (ICS) per
the National Asthma Education and Prevention Program (NAEPP) guidelines by self-report

- Evidence of hypersensitivity to environmental allergens, with at least one of the
following:

- Elevated serum IgE

- Positive allergy skin prick testing to at least 1 allergen.

- Evidence of allergic rhinitis by physical exam or by medical history.

- Peripheral blood eosinophils ≥150 cells/µl obtained at screening visit.

- Asthma Severity Score indicating Moderate to Severe Impairment based on EPR-3
classification guidelines

- > 1 utilization for treatment of asthma exacerbation including prescription of oral or
intravenous steroids (urgent care, emergency department visit, or hospitalization for
asthma) in the past 12 months

- Negative urine pregnancy test for females of child bearing potential and use of
contraception throughout the study.

Exclusion Criteria:

- Diagnosis of chronic lung disease other than asthma

- Have received biologic therapy (e.g., anti-IgE, anti-IL-4, anti-IL-5) within 6 months
of study entry.

- Diagnosis of chronic disease other than asthma requiring daily steroids or
immunosuppressive agents

- History of a heart attack or severe chronic heart disease

- Current smoking or previous history within 1 year

- Transplant patient

- IBD, Crohn's

- History of ulcer, gastric esophageal reflux (GERD) or chronic peptic ulcer disease

- Pregnant or planning to become pregnant

- Breastfeeding

- History of severe allergic or anaphylactic reactions to medications

- Grade 2-4 Abnormal Laboratory Results (hemoglobin, WBC, lymphocytes, platelets,
sodium, potassium, glucose, BUN, creatinine, calcium, albumin, total protein, alkaline
phosphate, AST, ALT, and bilirubin), see table 7.4.1.2a and 7.4.1.2b.

- Grade 3-4 Abnormal Eosinophils and Neutrophils, see table 7.4.1.2a.

- Evidence of papilledema, or history of pseudotumor cerebri

- History of persistent headaches

- Allergic reaction to cysteamine or penicillamine

- Serious medical condition that, in the opinion of the Study Investigator, would
interfere with the ability of the patient to complete the study