Cysteamine for Asthma

May 4, 2021 updated by: Children's Hospital Medical Center, Cincinnati
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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

60

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 45 years (ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: QUADRUPLE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: Cysteamine
Cysteamine Bitartrate plus standard asthma care
Drug: Cysteamine Bitartrate
Cysteamine bitartrate administered four times per day (QID) for 8 weeks. 125mg tablet QID.
PLACEBO_COMPARATOR: Placebo Oral Tablet
Placebo plus standard asthma care
Drug: Placebo Oral Tablet
Placebo administered for 8 weeks. 125mg tablet QID.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in the Asthma Symptom Severity Score
Time Frame: Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks), Study End (4 weeks post-treatment, Follow-up Phone Call)
Asthma symptom severity score will be classified using the Expert Panel Report 3 (EPR-3) criteria for untreated asthma. The level of impairment is based on the most severe indication of the following criteria: 1. Symptoms, 2. Nighttime awakenings, 3. Short-Acting Beta Agonist (SABA) use for symptom control, 4. Interference with normal activity, and 5. Lung Function.
Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks), Study End (4 weeks post-treatment, Follow-up Phone Call)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Days with Albuterol Use Over Time
Time Frame: Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks), Study End (4 weeks post-treatment, Follow-up Phone Call)
Albuterol is a bronchodilator used as a rescue medication for treatment of asthma symptoms.
Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks), Study End (4 weeks post-treatment, Follow-up Phone Call)
Change in Inflammation in TH2 cells Over Time
Time Frame: Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks, End Treatment)
Inflammation as measured by T Helper Type 2 (TH2) cells in peripheral blood.
Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks, End Treatment)
Spirometry Measurement: Forced Expiratory Volume in 1 second (FEV1) Over Time
Time Frame: Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks, End Treatment)
FEV1 is the amount of air exhaled in 1 second during spirometry. This is used as a measurement of asthma severity.
Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks, End Treatment)
Peak Flow Measurement: Forced Expiratory Volume in 1 second (FEV1) Over Time
Time Frame: Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks), Study End (4 weeks post-treatment, Follow-up Phone Call)
FEV1 will be obtained by peak flow measurement. This will be used as a measurement in asthma severity.
Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks), Study End (4 weeks post-treatment, Follow-up Phone Call)
Peak Flow Measurement: Peak Expiratory Flow (PEF) Over Time
Time Frame: Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks), Study End (4 weeks post-treatment, Follow-up Phone Call)
PEF will be obtained by peak flow measurement. This will be used as a measurement in asthma severity.
Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks), Study End (4 weeks post-treatment, Follow-up Phone Call)
Change in Asthma Quality of Life Questionnaire (miniAQLQ) scores Over Time
Time Frame: Baseline to Visit 2 (8 weeks)
The miniAQLQ will be used to measure the impact that asthma has on participants quality of life. The miniAQLQ will ask questions in the following domains: symptoms, activity limitations, emotional function, and environmental stimuli. There are 15 questions, each with a 7-point scale. An answer of 1 on a question represents the greatest impairment possible and a 7 represents the least impairment. Individual questions are equally weighted. The overall miniAQLQ score is the mean of the responses to each of the 15 questions.
Baseline to Visit 2 (8 weeks)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Children's Hospital Medical Center, Cincinnati

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

June 18, 2019

Primary Completion (ACTUAL)

February 10, 2021

Study Completion (ACTUAL)

February 10, 2021

Study Registration Dates

First Submitted

December 27, 2018

First Submitted That Met QC Criteria

March 19, 2019

First Posted (ACTUAL)

March 21, 2019

Study Record Updates

Last Update Posted (ACTUAL)

May 6, 2021

Last Update Submitted That Met QC Criteria

May 4, 2021

Last Verified

May 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Hershey-101

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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