AsthmaPhase 1NCT03883984
Cysteamine for Asthma
A Phase 1 study testing Cysteamine Bitartrate in people with Asthma, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 45 years
- Conditions the study is for
- Asthma
- What is being tested?
- Cysteamine Bitartrate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Jun 2019to 10 Feb 2021
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 45 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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
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- 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.
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
About this study
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.
Read the 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
- Being tested
- Cysteamine Bitartrate, Placebo Oral Tablet
- Medical subject headings
- Asthma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cysteamine BitartrateDrug
Cysteamine bitartrate administered four times per day (QID) for 8 weeks. 125mg tablet QID.
Placebo Oral TabletDrug
Placebo administered for 8 weeks. 125mg tablet QID.
| Study group | Role | What they receive |
|---|---|---|
| Cysteamine | Experimental | Cysteamine Bitartrate plus standard asthma care Receives: Drug: Cysteamine Bitartrate |
| Placebo Oral Tablet | Placebo comparator | Placebo plus standard asthma care Receives: Drug: Placebo Oral Tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Albuterol is a bronchodilator used as a rescue medication for treatment of asthma symptoms.
Measured: 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
Inflammation as measured by T Helper Type 2 (TH2) cells in peripheral blood.
Measured: Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks, End Treatment)
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.
Measured: Baseline (Pre-Treatment), V1 (4 weeks), V2 (8 weeks, End Treatment)
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.
Measured: 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
PEF will be obtained by peak flow measurement. This will be used as a measurement in asthma severity.
Measured: 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
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.
Measured: Baseline to Visit 2 (8 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Mar 2019
- Study start18 Jun 2019
- Primary completion10 Feb 2021
- Study completion10 Feb 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AsthmaStudies of the same condition, open and under way.
- More trials run by Children's Hospital Medical Center, CincinnatiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- 1:1 randomized, double-blind, placebo-controlled trial
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 60 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Gurjit K Khurana Hershey, MD, PhD, Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03883984
- Sponsor's protocol ID
- Hershey-101
- Status last verified
- May 2021
- First submitted
- 27 Dec 2018
- Last updated
- 6 May 2021
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cysteamine for Asthma
- ClinicalTrials.gov ID
- NCT03883984
- Acronym / sponsor ID
- Hershey-101
- Conditions
- Asthma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 60 participants (actual)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Study start
- 18 Jun 2019 (actual)
- Primary completion
- 10 Feb 2021 (actual)
- Study completion
- 10 Feb 2021 (actual)
- First posted
- 21 Mar 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- 1:1 randomized, double-blind, placebo-controlled trial
| Arm | Type | Receives |
|---|---|---|
| Cysteamine | Experimental | Drug: Cysteamine BitartrateCysteamine Bitartrate plus standard asthma care |
| Placebo Oral Tablet | Placebo comparator | Drug: Placebo Oral TabletPlacebo plus standard asthma care |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (12 listed)
- 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:
Key exclusion criteria (17 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Cysteamine BitartrateCysteamine bitartrate administered four times per day (QID) for 8 weeks. 125mg tablet QID. | Drug | Cysteamine |
| Placebo Oral TabletPlacebo administered for 8 weeks. 125mg tablet QID. | Drug | Placebo Oral Tablet |
Primary outcome
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.
6 secondary outcomes
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)
Change in Inflammation in TH2 cells Over Time
Time frame: 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)
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)
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)
Change in Asthma Quality of Life Questionnaire (miniAQLQ) scores Over Time
Time frame: Baseline to Visit 2 (8 weeks)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 May 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Gurjit K Khurana Hershey, MD, PhD principal investigator
Children's Hospital Medical Center, Cincinnati
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Cysteamine Bitartrate in people with Asthma, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 May 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 May 2021.