Cysteamine for Asthma

NCT ID: NCT03883984

Last Updated: 2021-05-06

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 60 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-06-18
• Study Completion Date: 2021-02-10

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.

Conditions

Asthma

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Cysteamine

Cysteamine Bitartrate plus standard asthma care

Group Type: EXPERIMENTAL

Cysteamine Bitartrate

Intervention Type: DRUG

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

Placebo Oral Tablet

Placebo plus standard asthma care

Group Type: PLACEBO_COMPARATOR

Placebo Oral Tablet

Intervention Type: DRUG

Placebo administered for 8 weeks. 125mg tablet QID.

Interventions

Cysteamine Bitartrate

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

Intervention Type: DRUG

Placebo Oral Tablet

Placebo administered for 8 weeks. 125mg tablet QID.

Intervention Type: DRUG

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 45 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Children's Hospital Medical Center, Cincinnati

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Gurjit K Khurana Hershey, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital Medical Center, Cincinnati

Locations

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Countries

United States

Other Identifiers

Hershey-101

Identifier Type: -

Identifier Source: org_study_id