Pulmonary FibrosisPhase 1NCT02510937
Safety and PK Study of CC-90001 in Subjects With Pulmonary Fibrosis
A Phase 1 study testing CC-90001 in people with Pulmonary Fibrosis, run by Celgene at 6 sites in 2 countries.
Trial summary
Participation in the study will last for 3months, with a 1 month screening phase. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pulmonary Fibrosis
- What is being tested?
- CC-90001
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 6 sitesin 2 countries
- Study dates
- 5 Aug 2015to 6 Feb 2017
- 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
- Potential subjects must satisfy all of the following criteria to be enrolled into the study:
- Subject ≥ 18 years of age.
- Documented clinical diagnosis of a fibrotic lung disease supported by at least one of the following:
- Usual interstitial pneumonia (UIP) pattern based on high-resolution computed tomography (HRCT).
- OR
- Nonspecific interstitial pneumonia (NSIP) pattern based on HRCT. OR
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- A documented fibrotic NSIP on surgical lung biopsy. OR
- A documented UIP pattern on surgical lung biopsy. The underlying etiology of the fibrotic lung disease may be of any cause, including, but NOT LIMITED TO any of the following: Connective tissue disease associated interstitial lung disease, idiopathic pulmonary fibrosis (IPF), environmental or chemical-related pulmonary fibrosis, other forms of interstitial pulmonary fibrosis, Hermansky-Pudlak syndrome.
- Must understand and voluntarily sign a written Informed Consent Form prior to any study-related procedures being performed.
- Must be able to communicate with the Investigator, understand and comply with the requirements of the study, and agree to adhere to restrictions and examination schedules.
- Asparate Aminotransferase (AST) or serum glutamic-oxaloacetic transaminase within limits of normal.
- Alanine Aminotransferase (ALT) or serum glutamic pyruvic transaminase within limits of normal.
- Total bilirubin and International Normalized Ratio (INR) within limits of normal.
- No clinically significant laboratory test results as determined by the Investigator.
- Male subjects agree to use barrier contraception NOT made of natural (animal) membrane (eg, latex or polyurethane condoms are acceptable) when engaging in sexual activity with a female of childbearing potential (FCBP) while on CC 90001 and for at least 28 days after the last dose of study medication. A FCBP is defined as a sexually mature female who has not undergone a hysterectomy or bilateral oophorectomy or who has not been naturally postmenopausal for at least 24 consecutive months (ie, who has had menses at any time in the preceding 24 consecutive months).
- All FCBPs must have a negative pregnancy test at Screening and Day 1. Any FCBP who engages in activity in which conception is possible must use two forms of contraception simultaneously while on CC-90001 and for at least 28 days after taking the last dose of CC-90001: one highly effective form (ie, hormonal, intrauterine device, tubal ligation, vasectomized partner) and one additional form (latex condom or any nonlatex condom NOT made of natural [animal] membrane [eg, polyurethane], diaphragm, sponge). If one highly effective form of contraception cannot be used, then two forms of barrier contraception must be used, ie, latex condom or any nonlatex condom NOT made out of natural (animal) membrane [eg, polyurethane] with either of the following: sponge with spermicide or diaphragm with spermicide.
- Female subjects that are postmenopausal (defined as 24 months without menses before Screening, with an estradiol level of < 30 pg/mL and FSH level of > 40 IU/L at Screening).
Who cannot join
- Potential subjects will be excluded from enrollment if any of the following occur:
- Exposed to an investigational drug (new chemical entity) within 30 days preceding the first dose of CC-90001 administration, or five half-lives of that investigational drug, if known (whichever is longer).
- Subjects who are part of the clinical staff personnel or family members of the study site staff.
- Screening forced vital capacity (FVC) < 40% predicted.
- Screening lung diffusion capacity (DLco) < 20% predicted.
- Any condition other than pulmonary fibrosis that is likely to result in the subject's death or increases the risk of death within a year from signing the ICF.
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- Known clinical diagnosis of pulmonary arterial hypertension that currently requires treatment.
- Subjects with cystic fibrosis, active aspergillosis, active tuberculosis, or other serious concomitant respiratory disorder other than pulmonary fibrosis, as determined by the Investigator. Subjects with reactive airway disease, chronic obstructive pulmonary disease, and asthma may be included as long as, in the opinion of the Investigator, fibrosis is the major contributing factor to the subject's respiratory disorder.
- Use of any cytotoxic agents within 4 weeks of dosing.
- Currently being administered any targeted therapy for pulmonary fibrosis and not on a stable dose for ≥ 6 weeks duration prior to first study dosing (potential subjects should be excluded if a dose increase is planned during the study period).
- Use of Esbriet® (pirfenidone) or Ofev® (nintedanib) within 30 day prior to first dose.
- Currently being administered statins (HMG-CoA reductase inhibitors) and not on a stable dose for ≥ 6 weeks duration prior to first study dosing (potential subjects should be excluded if a dose increase is planned during the study period).
- Taking medications that are substrates of the transporters P-gp, BCRP, OAT3, OATP1B1, OATP1B3, and OCT2 and have a narrow therapeutics index (eg, P-gp substrate digoxin).
- Use of acetaminophen (paracetamol) at a dosage > 3 grams per day within 2 weeks of first study dosing.
- Use of niacin at a dosage > 2 grams/day within 2 weeks prior to first study dosing.
- Any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from participating in the study.
- History of recurrent bacterial infections (at least three major infections resulting in hospitalization and/or requiring intravenous antibiotic treatment within the past 2 years)
- History of Human Immunodeficiency (HIV), Hepatitis B Virus (HBV), or Hepatitis C Virus (HCV). Subjects treated for HCV who have a sustained virologic response of 6 months following final HCV treatment can be included.
- History of active malignancy within 5 years prior to signing the ICF, excluding nonmelanoma skin cancer.
Trial locations and contacts
6 sites in 2 countries.
6 sites in 2 countries
United States4 sites
LaPorte County Institute for Clinical Research, Inc
Michigan City, Indiana, 46360
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
University of Pittsburgh
Pittsburgh, Pennsylvania, 15213
Tampa General Hospital
Tampa, Florida, 33606
Australia2 sites
The Prince Charles Hospital
Chermside, 4032
St. Vincent's Hospital- Sydney
Darlinghurst, 2010
About this study
Official title: A Phase 1b, Multicenter, Open-label, Staggered-dose-escalation Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Doses of CC-90001 for 3 Months in Patients With Pulmonary Fibrosis
Participation in the study will last for 3months, with a 1 month screening phase.
Read the detailed description
This is an open-label, staggered dose-escalation, cohort expansion study that will enroll subjects at multiple study sites in the United States of America (USA) and Australia. The study will consist of two treatment arms:
- Low dose (100 mg) CC-90001 administered orally once daily (QD) for 12 continuous weeks.
- High dose (200 mg) CC-90001 administered orally QD for 12 continuous weeks. The high dose (200 mg) CC-90001 arm will not start until at least three subjects complete a minimum of 2 weeks of low-dose CC 90001 and the low dose treatment arm is determined not to meet the study dose escalation stopping criteria (Figure 2).
Each subject will participate in a Screening (up to 4 weeks prior to treatment), a 12 week Treatment Phase, and a 4-week Follow-up visit. Subjects will be screened for eligibility. Subjects who meet all of the inclusion criteria and none of the exclusion criteria at Screening will return to the study site on Day 1 for assessments and to begin administration of a QD dose of CC 90001, according to the dose level in which the subject is enrolled. Three subjects will initially be enrolled to receive low dose CC-90001 (100 mg QD), and will be evaluated for all scheduled assessments through 12 weeks of treatment. Once a total of three subjects have completed the Week 2 visit, a decision to continue the study at the high dose level (200 mg QD) will be determined.
If the criteria for escalation to the high dose (CC-90001 200 mg QD) are met, the low dose (CC-90001 100 mg QD) subjects will remain on low dose (CC-90001 100 mg QD) and six additional subjects will be enrolled at the high dose level (CC-90001 200 mg QD). If one of the three subjects at the low dose (CC-90001 100 mg QD) experiences an event that meets the individual subject dose stopping criteria, another three subjects will be enrolled in the low dose arm. Dose escalation to the high dose (CC-90001 200 mg QD) will not occur if two or more of the six subjects meet the individual subject dose stopping criteria. All subjects (low and high dose) will remain on CC-90001 for a total of 12 weeks unless an individual subject experiences an event that meets any of the individual subject stopping criteria. In addition, the dose of CC-90001 may be reduced CC-90001 200 mg QD to CC-90001 100 mg QD) for an individual who meets any of the individual subject dose reduction criteria. If two or more subjects in the high dose (CC-90001 200 mg QD) arm experience an event that meets the individual stopping criteria, the 100 mg QD dose arm may be repeated in three additional subjects, or the study may be stopped.
Study visits will occur at Screening, Day 1, and Weeks 1, 2, 3, 4, 6, 8, 10, 12, and 16 (a Follow-up visit). Blood and urine samples will be collected at specified times for clinical safety laboratory assessments, pharmacokinetic analysis (how the drug affects the body), and pharmacodynamic analysis (how the body affects the drug). Safety will be monitored throughout the study.
- Conditions
- Pulmonary Fibrosis
- Being tested
- CC-90001
- Keywords
- pulmonary fibrosis, pharmacokinetic, pharmacodynamics, CC-90001, Safety
- Medical subject headings
- Pulmonary Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CC-90001Drug
| Study group | Role | What they receive |
|---|---|---|
| Low dose CC-90001 | Experimental | Low dose (100 mg) CC-90001 administered orally once daily (QD) for 12 continuous weeks Receives: Drug: CC-90001 |
| High dose CC-90001 | Experimental | High-dose (200 mg) CC-90001 administered orally Once Daily (QD) for 12 continuous weeks Receives: Drug: CC-90001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events (AEs)
Number of subjects with adverse events
Measured: up to 16 weeks
Dose interruptions, reductions, and discontinuation
Number of subjects experiencing dose interruptions, reductions, and discontinuation of CC-90001 secondary to an AE
Measured: up to 16 weeks
Complete PEs
Complete Physical Examinations
Measured: up to 16 weeks
Clinical laboratory assessments
Clinical laboratory assessments
Measured: up to 16 weeks
Vital sign measurements
Heart rate (HR), respiratory rate, blood pressure (BP), and body temperature
Measured: up to 16 weeks
12-lead ECGs
12-lead ECGs
Measured: up to 16 weeks
Urine pregnancy tests
Urine pregnancy tests
Measured: up to 16 weeks
Concomitant medications and procedures
Concomitant medications and procedures
Measured: up to 16 weeks
Secondary outcomes (2)
CC-90001 plasma concentrations
CC-90001 plasma concentrations collected sparsely and measured using a validated liquid chromatography tandem mass spectrometry assay
Measured: up to 16 weeks
Population-based PK
Population-based PK approach as appropriate for the following parameters (at a minimum, but not limited to): apparent clearance; apparent central volume of distribution; first-order rate of absorption; disease as a covariate may be explored in the population PK analysis-the derived PK parameters such as Cmax and AUC may be also determined based on the population PK model as appropriate.
Measured: up to 16 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jul 2015 (estimated)
- Study start5 Aug 2015
- Primary completion6 Feb 2017
- Study completion6 Feb 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pulmonary FibrosisStudies of the same condition, open and under way.
- More trials run by CelgeneThe 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Celgene
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ed O'Mara, MD, Study director, Celgene Corporation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02510937
- Sponsor's protocol ID
- CC-90001-CP-003
- Status last verified
- Mar 2017
- First submitted
- 27 Jul 2015
- Last updated
- 4 Apr 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Apr 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b, Multicenter, Open-label, Staggered-dose-escalation Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Doses of CC-90001 for 3 Months in Patients With Pulmonary Fibrosis
- ClinicalTrials.gov ID
- NCT02510937
- Acronym / sponsor ID
- CC-90001-CP-003
- Conditions
- Pulmonary Fibrosis
- Keywords
- pulmonary fibrosis, pharmacokinetic, pharmacodynamics, CC-90001, Safety
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- CelgeneIndustry (pharma, biotech, devices)
- Study start
- 5 Aug 2015 (actual)
- Primary completion
- 6 Feb 2017 (actual)
- Study completion
- 6 Feb 2017 (actual)
- First posted
- 29 Jul 2015 (estimated)
- 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.
Participation in the study will last for 3months, with a 1 month screening phase.
Detailed description
This is an open-label, staggered dose-escalation, cohort expansion study that will enroll subjects at multiple study sites in the United States of America (USA) and Australia. The study will consist of two treatment arms:
- Low dose (100 mg) CC-90001 administered orally once daily (QD) for 12 continuous weeks.
- High dose (200 mg) CC-90001 administered orally QD for 12 continuous weeks. The high dose (200 mg) CC-90001 arm will not start until at least three subjects complete a minimum of 2 weeks of low-dose CC 90001 and the low dose treatment arm is determined not to meet the study dose escalation stopping criteria (Figure 2).
Each subject will participate in a Screening (up to 4 weeks prior to treatment), a 12 week Treatment Phase, and a 4-week Follow-up visit. Subjects will be screened for eligibility. Subjects who meet all of the inclusion criteria and none of the exclusion criteria at Screening will return to the study site on Day 1 for assessments and to begin administration of a QD dose of CC 90001, according to the dose level in which the subject is enrolled. Three subjects will initially be enrolled to receive low dose CC-90001 (100 mg QD), and will be evaluated for all scheduled assessments through 12 weeks of treatment. Once a total of three subjects have completed the Week 2 visit, a decision to continue the study at the high dose level (200 mg QD) will be determined.
If the criteria for escalation to the high dose (CC-90001 200 mg QD) are met, the low dose (CC-90001 100 mg QD) subjects will remain on low dose (CC-90001 100 mg QD) and six additional subjects will be enrolled at the high dose level (CC-90001 200 mg QD). If one of the three subjects at the low dose (CC-90001 100 mg QD) experiences an event that meets the individual subject dose stopping criteria, another three subjects will be enrolled in the low dose arm. Dose escalation to the high dose (CC-90001 200 mg QD) will not occur if two or more of the six subjects meet the individual subject dose stopping criteria. All subjects (low and high dose) will remain on CC-90001 for a total of 12 weeks unless an individual subject experiences an event that meets any of the individual subject stopping criteria. In addition, the dose of CC-90001 may be reduced CC-90001 200 mg QD to CC-90001 100 mg QD) for an individual who meets any of the individual subject dose reduction criteria. If two or more subjects in the high dose (CC-90001 200 mg QD) arm experience an event that meets the individual stopping criteria, the 100 mg QD dose arm may be repeated in three additional subjects, or the study may be stopped.
Study visits will occur at Screening, Day 1, and Weeks 1, 2, 3, 4, 6, 8, 10, 12, and 16 (a Follow-up visit). Blood and urine samples will be collected at specified times for clinical safety laboratory assessments, pharmacokinetic analysis (how the drug affects the body), and pharmacodynamic analysis (how the body affects the drug). Safety will be monitored throughout the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Low dose CC-90001 | Experimental | Drug: CC-90001Low dose (100 mg) CC-90001 administered orally once daily (QD) for 12 continuous weeks |
| High dose CC-90001 | Experimental | Drug: CC-90001High-dose (200 mg) CC-90001 administered orally Once Daily (QD) for 12 continuous weeks |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Potential subjects must satisfy all of the following criteria to be enrolled into the study:
- OR
Key exclusion criteria (19 listed)
- Potential subjects will be excluded from enrollment if any of the following occur:
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 |
|---|---|---|
| CC-90001 | Drug | High dose CC-90001, Low dose CC-90001 |
Primary outcome
Adverse Events (AEs)
Time frame: up to 16 weeks
Number of subjects with adverse events
Dose interruptions, reductions, and discontinuation
Time frame: up to 16 weeks
Number of subjects experiencing dose interruptions, reductions, and discontinuation of CC-90001 secondary to an AE
Complete PEs
Time frame: up to 16 weeks
Complete Physical Examinations
Clinical laboratory assessments
Time frame: up to 16 weeks
Clinical laboratory assessments
Vital sign measurements
Time frame: up to 16 weeks
Heart rate (HR), respiratory rate, blood pressure (BP), and body temperature
12-lead ECGs
Time frame: up to 16 weeks
12-lead ECGs
Urine pregnancy tests
Time frame: up to 16 weeks
Urine pregnancy tests
Concomitant medications and procedures
Time frame: up to 16 weeks
Concomitant medications and procedures
2 secondary outcomes
CC-90001 plasma concentrations
Time frame: up to 16 weeks
Population-based PK
Time frame: up to 16 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 4 Apr 2017.
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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.
Ed O'Mara, MD study director
Celgene Corporation
6 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 CC-90001 in people with Pulmonary Fibrosis, run by Celgene at 6 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 17 inclusion and 19 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 4 Apr 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States, Australia. 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. 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?
Adverse Events (AEs) (time frame: up to 16 weeks). The study lists 8 primary outcomes in all. 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 4 Apr 2017.