Cystic FibrosisPhase 1NCT04279769
Study to Evaluate the Safety of CB-280 in Patients With Cystic Fibrosis
A Phase 1 study testing CB-280 in people with Cystic Fibrosis, run by Calithera Biosciences, Inc at 20 sites in 2 countries.
Trial summary
This is a phase 1b multiple ascending dose escalation study to evaluate the safety and tolerability of arginase inhibitor CB-280 in subjects with cystic fibrosis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- CB-280
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 32 participants
- Where
- 20 sitesin 2 countries
- Study dates
- 3 Jul 2020to 23 Nov 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Written Informed Consent in accordance with federal, local, and institutional guidelines
- Confirmed diagnosis of cystic fibrosis
- Male or female subjects ≥ 18 years on the date of informed consent
- Percent predicted FEV1 of 40-90% at screening per Global Lung Function Initiative (GLI) equation
- Clinically stable with no significant changes in health status within 28 days prior to Day 1
- Chronic lung infection with P. aeruginosa defined as at least one positive culture in the last two years and more than 50% of cultures positive since then
Show all 10Show fewer
- Stable cystic fibrosis medication regimen for at least 28 days inclusive of CFTR modulators prior to Day 1
- Hemoglobin > 10 g/dL at screening
- Glomerular filtration rate > 50 mL/min/1.73 m2 at screening
- Normal liver function at screening
Who cannot join
- History of any comorbidity that, in the opinion of the Investigator, might pose an additional risk in administering study drug to the subject or confound the results of the study
- Lung infection with organisms associated with a more rapid decline in pulmonary status (including, but not limited to, Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus)
- Unable to receive study medication per os (PO)
- Females who are pregnant, have a positive pregnancy test at screening, or are nursing (lactating)
- Other protocol defined Inclusion/Exclusion criteria may apply.
Trial locations and contacts
20 sites in 2 countries.
20 sites in 2 countries
United States17 sites
Johns Hopkins University
Baltimore, Maryland, 21287
Billings Clinic
Billings, Montana, 59101
Boston Children's Hospital, Brigham & Women's Hospital
Boston, Massachusetts, 02115
UNC Marsico Clinical Research Center
Chapel Hill, North Carolina, 27514
Medical University of South Carolina
Charleston, South Carolina, 29425
University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106
Vermont Lung Center at the University of Vermont Medical Center
Colchester, Vermont, 05446
University of Florida
Gainesville, Florida, 32610
The Cystic Fibrosis Institute
Glenview, Illinois, 60025
New York Medical College at Westchester Medical Center
Hawthorne, New York, 10532
Hershey Medical Center Pennsylvania State University
Hershey, Pennsylvania, 17033
Indiana University
Indianapolis, Indiana, 46202
University of Kansas Medical Center
Kansas City, Kansas, 66160
University of Arkansas for Medical Sciences
Little Rock, Arkansas, 72205
Long Beach Memorial Medical Center
Long Beach, California, 90806
Virginia Commonwealth University
Richmond, Virginia, 23219
University of Utah
Salt Lake City, Utah, 84132
Canada3 sites
University of Calgary
Calgary, Alberta, T2N 4N1
McGill University Health Center
Montreal, Quebec, H4A 3J1
St. Pauls' Hospital
Vancouver, British Columbia, V6Z1Y6
About this study
Official title: A Phase 1b Randomized, Double-blind, Placebo-Controlled Trial to Evaluate the Safety of CB-280 in Patients With Cystic Fibrosis
This is a phase 1b multiple ascending dose escalation study to evaluate the safety and tolerability of arginase inhibitor CB-280 in subjects with cystic fibrosis.
Read the detailed description
Study CX-280-202 is a Phase 1b, randomized, double-blind, placebo-controlled, multiple ascending dose escalation study of CB-280 in adult subjects with cystic fibrosis and chronic infection with Pseudomonas aeruginosa. The study will evaluate the safety, pharmacokinetics, pharmacodynamics, and biological activity of CB-280 in approximately 32 adult patients with cystic fibrosis. There are four planned sequential dose escalation cohorts of 8 subjects each, randomized 6:2 to receive CB-280 or matched placebo at doses of 50 mg, 100 mg, 200 mg, or 400 mg administered twice daily for 14 days. Intermediate dose levels may be evaluated based on emerging safety data at the planned dose levels.
- Conditions
- Cystic Fibrosis
- Being tested
- CB-280, Placebos
- Keywords
- Arginase inhibitor, CB-280
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CB-280Drug
CB-280, oral capsule administered twice daily at the assigned dose level for 14 days
PlacebosDrug
Placebo oral capsule administrated twice daily at the assigned dose level for 14 days
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | CB-280 twice daily at 50 mg for 14 days Receives: Drug: CB-280 |
| Cohort 2 | Experimental | CB-280 twice daily at 100 mg for 14 days Receives: Drug: CB-280 |
| Cohort 3 | Experimental | CB-280 twice daily at 200 mg for 14 days Receives: Drug: CB-280 |
| Cohort 4 | Experimental | CB-280 twice daily at 400 mg for 14 days Receives: Drug: CB-280 |
| Placebo | Placebo comparator | Placebo twice daily for 14 days Receives: Drug: Placebos |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine the safety and tolerability of CB-280 in adult cystic fibrosis patients: incidence and severity of adverse event (AEs) assessed by Common Terminology Criteria for Adverse Events, version 5 (CTCAE v5.0)
Measured: Start of treatment to Day 28
Secondary outcomes (2)
Pharmacokinetics of plasma CB-280 measured by Peak Plasma Concentration (Cmax)
Measured: Day 14
Pharmacokinetics of plasma CB-280 measured by area under the plasma concentration versus time curve, from time 0 to the last observed non-zero concentration (AUC 0-t)
Measured: Day 14
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Feb 2020
- Study start3 Jul 2020
- Primary completion23 Nov 2021
- Study completion23 Nov 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- 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
- Sequential
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- Calithera Biosciences, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Emil T Kuriakose, MD, Study director, Calithera Bioscience
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04279769
- Sponsor's protocol ID
- CX-280-202
- Status last verified
- Apr 2022
- First submitted
- 10 Feb 2020
- Last updated
- 13 Apr 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b Randomized, Double-blind, Placebo-Controlled Trial to Evaluate the Safety of CB-280 in Patients With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT04279769
- Acronym / sponsor ID
- CX-280-202
- Conditions
- Cystic Fibrosis
- Keywords
- Arginase inhibitor, CB-280
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 32 participants (actual)
- Lead sponsor
- Calithera Biosciences, IncIndustry (pharma, biotech, devices)
- Study start
- 3 Jul 2020 (actual)
- Primary completion
- 23 Nov 2021 (actual)
- Study completion
- 23 Nov 2021 (actual)
- First posted
- 21 Feb 2020 (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.
This is a phase 1b multiple ascending dose escalation study to evaluate the safety and tolerability of arginase inhibitor CB-280 in subjects with cystic fibrosis.
Detailed description
Study CX-280-202 is a Phase 1b, randomized, double-blind, placebo-controlled, multiple ascending dose escalation study of CB-280 in adult subjects with cystic fibrosis and chronic infection with Pseudomonas aeruginosa. The study will evaluate the safety, pharmacokinetics, pharmacodynamics, and biological activity of CB-280 in approximately 32 adult patients with cystic fibrosis. There are four planned sequential dose escalation cohorts of 8 subjects each, randomized 6:2 to receive CB-280 or matched placebo at doses of 50 mg, 100 mg, 200 mg, or 400 mg administered twice daily for 14 days. Intermediate dose levels may be evaluated based on emerging safety data at the planned dose levels.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind (participant, investigator)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Drug: CB-280CB-280 twice daily at 50 mg for 14 days |
| Cohort 2 | Experimental | Drug: CB-280CB-280 twice daily at 100 mg for 14 days |
| Cohort 3 | Experimental | Drug: CB-280CB-280 twice daily at 200 mg for 14 days |
| Cohort 4 | Experimental | Drug: CB-280CB-280 twice daily at 400 mg for 14 days |
| Placebo | Placebo comparator | Drug: PlacebosPlacebo twice daily for 14 days |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Written Informed Consent in accordance with federal, local, and institutional guidelines
- Confirmed diagnosis of cystic fibrosis
- Male or female subjects ≥ 18 years on the date of informed consent
- Percent predicted FEV1 of 40-90% at screening per Global Lung Function Initiative (GLI) equation
- Clinically stable with no significant changes in health status within 28 days prior to Day 1
Key exclusion criteria (5 listed)
- History of any comorbidity that, in the opinion of the Investigator, might pose an additional risk in administering study drug to the subject or confound the results of the study
- Lung infection with organisms associated with a more rapid decline in pulmonary status (including, but not limited to, Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus)
- Unable to receive study medication per os (PO)
- Females who are pregnant, have a positive pregnancy test at screening, or are nursing (lactating)
- Other protocol defined Inclusion/Exclusion criteria may apply.
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 |
|---|---|---|
| CB-280CB-280, oral capsule administered twice daily at the assigned dose level for 14 days | Drug | Cohort 1, Cohort 2, Cohort 3, Cohort 4 |
| PlacebosPlacebo oral capsule administrated twice daily at the assigned dose level for 14 days | Drug | Placebo |
Primary outcome
Determine the safety and tolerability of CB-280 in adult cystic fibrosis patients: incidence and severity of adverse event (AEs) assessed by Common Terminology Criteria for Adverse Events, version 5 (CTCAE v5.0)
Time frame: Start of treatment to Day 28
2 secondary outcomes
Pharmacokinetics of plasma CB-280 measured by Peak Plasma Concentration (Cmax)
Time frame: Day 14
Pharmacokinetics of plasma CB-280 measured by area under the plasma concentration versus time curve, from time 0 to the last observed non-zero concentration (AUC 0-t)
Time frame: Day 14
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 13 Apr 2022.
- Individual participant dataSharing planned: No
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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.
Emil T Kuriakose, MD study director
Calithera Bioscience
20 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CB-280 in people with Cystic Fibrosis, run by Calithera Biosciences, Inc at 20 sites in 2 countries.
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 10 inclusion and 5 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 13 Apr 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in United States, Canada. 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?
Determine the safety and tolerability of CB-280 in adult cystic fibrosis patients: incidence and severity of adverse event (AEs) assessed by Common Terminology Criteria for Adverse Events, version 5 (CTCAE v5.0) (time frame: Start of treatment to Day 28). 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 13 Apr 2022.