Cysteamine for Asthma

NCT03883984 | Completed Phase 1 FDA Drug

• 1 other identifier: Hershey-101
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

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.

Conditions

Asthma

Outcome Measures

Primary Outcomes (1)

• 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.

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

Secondary Outcomes (6)

• Number of Days with Albuterol Use Over Time

  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

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

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

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

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

  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

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

Study Arms (2)

Cysteamine

EXPERIMENTAL

Cysteamine Bitartrate plus standard asthma care

Drug: Cysteamine Bitartrate

Placebo Oral Tablet

PLACEBO COMPARATOR

Placebo plus standard asthma care

Drug: Placebo Oral Tablet

Interventions

Cysteamine Bitartrate DRUG

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

Cysteamine

Placebo Oral Tablet DRUG

Placebo administered for 8 weeks. 125mg tablet QID.

Placebo Oral Tablet

Eligibility Criteria

• Age: 18 Years - 45 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead

Study Sites (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

MeSH Terms

Conditions

Asthma

Interventions

Cysteamine

Condition Hierarchy (Ancestors)

Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases; Respiratory Hypersensitivity; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases

Intervention Hierarchy (Ancestors)

Mercaptoethylamines; Ethylamines; Amines; Organic Chemicals; Sulfhydryl Compounds; Sulfur Compounds

Study Officials

• Gurjit K Khurana Hershey, MD, PhD

  Children's Hospital Medical Center, Cincinnati

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

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

Study Record Dates

• First Submitted: December 27, 2018
• First Posted: March 21, 2019
• Study Start: June 18, 2019
• Primary Completion: February 10, 2021
• Study Completion: February 10, 2021
• Last Updated: May 6, 2021

Record last verified: 2021-05

Locations

US (1)