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Cysteamine for Asthma

Primary Purpose

Asthma

Status

Completed

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

Cysteamine Bitartrate

Placebo Oral Tablet

About this trial

This is an interventional treatment trial for Asthma

Eligibility Criteria

18 Years - 45 Years (Adult)All SexesDoes not accept 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

Sites / Locations

Cincinnati Children's Hospital Medical Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Cysteamine

Placebo Oral Tablet

Arm Description

Cysteamine Bitartrate plus standard asthma care

Placebo plus standard asthma care

Outcomes

Primary Outcome Measures

Change in the Asthma Symptom Severity Score

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.

Secondary Outcome Measures

Number of Days with Albuterol Use Over Time

Albuterol is a bronchodilator used as a rescue medication for treatment of asthma symptoms.

Change in Inflammation in TH2 cells Over Time

Inflammation as measured by T Helper Type 2 (TH2) cells in peripheral blood.

Spirometry Measurement: Forced Expiratory Volume in 1 second (FEV1) Over Time

FEV1 is the amount of air exhaled in 1 second during spirometry. This is used as a measurement of asthma severity.

Peak Flow Measurement: Forced Expiratory Volume in 1 second (FEV1) Over Time

FEV1 will be obtained by peak flow measurement. This will be used as a measurement in asthma severity.

Peak Flow Measurement: Peak Expiratory Flow (PEF) Over Time

PEF will be obtained by peak flow measurement. This will be used as a measurement in asthma severity.

Change in Asthma Quality of Life Questionnaire (miniAQLQ) scores Over Time

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.

Full Information

NCT ID

NCT03883984

First Posted

December 27, 2018

Last Updated

May 4, 2021

Sponsor

Children's Hospital Medical Center, Cincinnati

1. Study Identification

Unique Protocol Identification Number

NCT03883984

Brief Title

Cysteamine for Asthma

Official Title

Cysteamine for Asthma

Study Type

Interventional

2. Study Status

Record Verification Date

May 2021

Overall Recruitment Status

Completed

Study Start Date

June 18, 2019 (Actual)

Primary Completion Date

February 10, 2021 (Actual)

Study Completion Date

February 10, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Children's Hospital Medical Center, Cincinnati

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Asthma

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Parallel Assignment

Model Description

1:1 randomized, double-blind, placebo-controlled trial

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

60 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Cysteamine

Arm Type

Experimental

Arm Description

Cysteamine Bitartrate plus standard asthma care

Arm Title

Placebo Oral Tablet

Arm Type

Placebo Comparator

Arm Description

Placebo plus standard asthma care

Intervention Type

Drug

Intervention Name(s)

Cysteamine Bitartrate

Intervention Description

Cysteamine bitartrate administered four times per day (QID) for 8 weeks. 125mg tablet QID.

Intervention Type

Drug

Intervention Name(s)

Placebo Oral Tablet

Intervention Description

Placebo administered for 8 weeks. 125mg tablet QID.

Primary Outcome Measure Information:

Title

Change in the Asthma Symptom Severity Score

Description

Time Frame

Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks), Study End (4 weeks post-treatment, Follow-up Phone Call)

Secondary Outcome Measure Information:

Title

Number of Days with Albuterol Use Over Time

Description

Albuterol is a bronchodilator used as a rescue medication for treatment of asthma symptoms.

Time Frame

Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks), Study End (4 weeks post-treatment, Follow-up Phone Call)

Title

Change in Inflammation in TH2 cells Over Time

Description

Inflammation as measured by T Helper Type 2 (TH2) cells in peripheral blood.

Time Frame

Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks, End Treatment)

Title

Spirometry Measurement: Forced Expiratory Volume in 1 second (FEV1) Over Time

Description

FEV1 is the amount of air exhaled in 1 second during spirometry. This is used as a measurement of asthma severity.

Time Frame

Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks, End Treatment)

Title

Peak Flow Measurement: Forced Expiratory Volume in 1 second (FEV1) Over Time

Description

FEV1 will be obtained by peak flow measurement. This will be used as a measurement in asthma severity.

Time Frame

Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks), Study End (4 weeks post-treatment, Follow-up Phone Call)

Title

Peak Flow Measurement: Peak Expiratory Flow (PEF) Over Time

Description

PEF will be obtained by peak flow measurement. This will be used as a measurement in asthma severity.

Time Frame

Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks), Study End (4 weeks post-treatment, Follow-up Phone Call)

Title

Change in Asthma Quality of Life Questionnaire (miniAQLQ) scores Over Time

Description

Time Frame

Baseline to Visit 2 (8 weeks)

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

45 Years

Accepts Healthy Volunteers

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Gurjit K Khurana Hershey, MD, PhD

Organizational Affiliation

Children's Hospital Medical Center, Cincinnati

Official's Role

Principal Investigator

Facility Information:

Facility Name

Cincinnati Children's Hospital Medical Center

City

Cincinnati

State/Province

Ohio

ZIP/Postal Code

45229

Country

United States

12. IPD Sharing Statement

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