Cysteamine for Asthma

Cincinnati Children's Hospital Medical Center

Status and phase

Completed

Phase 1

Conditions

Asthma

Treatments

Drug: Cysteamine Bitartrate

Drug: Placebo Oral Tablet

Study type

Interventional

Funder types

Other

Identifiers

NCT03883984

Hershey-101

Details and patient eligibility

About

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.

Full description

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.

Enrollment

60 patients

Sex

All

Ages

18 to 45 years old

Volunteers

No Healthy Volunteers

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