Clinical trial NCT03883984: Cysteamine for Asthma
Official title: Cysteamine for Asthma
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Asthma
Lead sponsor: Children's Hospital Medical Center, Cincinnati
Sponsor class: OTHER
First posted: 2019-03-21
Enrollment: 60
Sex: ALL
Minimum age (years): 18
Maximum age (years): 45
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: 1:1 randomized, double-blind, placebo-controlled trial
Primary purpose: TREATMENT

Brief summary: 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.

Detailed 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.

Eligibility criteria: 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

Primary outcomes:
measure: Change in the Asthma Symptom Severity Score
timeFrame: Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks), Study End (4 weeks post-treatment, Follow-up Phone Call)
Secondary outcomes:
measure: Number of Days with Albuterol Use Over Time
timeFrame: Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks), Study End (4 weeks post-treatment, Follow-up Phone Call)
measure: Change in Inflammation in TH2 cells Over Time
timeFrame: Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks, End Treatment)
measure: Spirometry Measurement: Forced Expiratory Volume in 1 second (FEV1) Over Time
timeFrame: Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks, End Treatment)
measure: Peak Flow Measurement: Forced Expiratory Volume in 1 second (FEV1) Over Time
timeFrame: Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks), Study End (4 weeks post-treatment, Follow-up Phone Call)
measure: Peak Flow Measurement: Peak Expiratory Flow (PEF) Over Time
timeFrame: Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks), Study End (4 weeks post-treatment, Follow-up Phone Call)
measure: Change in Asthma Quality of Life Questionnaire (miniAQLQ) scores Over Time
timeFrame: Baseline to Visit 2 (8 weeks)
Other outcomes:
Locations:
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
Study officials:
affiliation: Children's Hospital Medical Center, Cincinnati
name: Gurjit K Khurana Hershey, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: