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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02510937




Registration number
NCT02510937
Ethics application status
Date submitted
27/07/2015
Date registered
29/07/2015
Date last updated
4/04/2017

Titles & IDs
Public title
Safety and PK Study of CC-90001 in Subjects With Pulmonary Fibrosis
Scientific 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
Secondary ID [1] 0 0
CC-90001-CP-003
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Pulmonary Fibrosis 0 0
Condition category
Condition code
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Experimental: Low dose CC-90001 - Low dose (100 mg) CC-90001 administered orally once daily (QD) for 12 continuous weeks

Experimental: High dose CC-90001 - High-dose (200 mg) CC-90001 administered orally Once Daily (QD) for 12 continuous weeks
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Adverse Events (AEs)
Timepoint [1] 0 0
up to 16 weeks
Primary outcome [2] 0 0
Dose interruptions, reductions, and discontinuation
Timepoint [2] 0 0
up to 16 weeks
Primary outcome [3] 0 0
Complete PEs
Timepoint [3] 0 0
up to 16 weeks
Primary outcome [4] 0 0
Clinical laboratory assessments
Timepoint [4] 0 0
up to 16 weeks
Primary outcome [5] 0 0
Vital sign measurements
Timepoint [5] 0 0
up to 16 weeks
Primary outcome [6] 0 0
12-lead ECGs
Timepoint [6] 0 0
up to 16 weeks
Primary outcome [7] 0 0
Urine pregnancy tests
Timepoint [7] 0 0
up to 16 weeks
Primary outcome [8] 0 0
Concomitant medications and procedures
Timepoint [8] 0 0
up to 16 weeks
Secondary outcome [1] 0 0
CC-90001 plasma concentrations
Timepoint [1] 0 0
up to 16 weeks
Secondary outcome [2] 0 0
Population-based PK
Timepoint [2] 0 0
up to 16 weeks

Eligibility
Key inclusion criteria
* Potential subjects must satisfy all of the following criteria to be enrolled into the study:

1. Subject = 18 years of age.
2. Documented clinical diagnosis of a fibrotic lung disease supported by at least one of the following:

1. Usual interstitial pneumonia (UIP) pattern based on high-resolution computed tomography (HRCT).

OR
2. Nonspecific interstitial pneumonia (NSIP) pattern based on HRCT. OR
3. A documented fibrotic NSIP on surgical lung biopsy. OR
4. 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.
3. Must understand and voluntarily sign a written Informed Consent Form prior to any study-related procedures being performed.
4. 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.
5. Asparate Aminotransferase (AST) or serum glutamic-oxaloacetic transaminase within limits of normal.
6. Alanine Aminotransferase (ALT) or serum glutamic pyruvic transaminase within limits of normal.
7. Total bilirubin and International Normalized Ratio (INR) within limits of normal.
8. No clinically significant laboratory test results as determined by the Investigator.
9. 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).
10. 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.
11. 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).
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Potential subjects will be excluded from enrollment if any of the following occur:

1. 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).
2. Subjects who are part of the clinical staff personnel or family members of the study site staff.
3. Screening forced vital capacity (FVC) < 40% predicted.
4. Screening lung diffusion capacity (DLco) < 20% predicted.
5. 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.
6. Known clinical diagnosis of pulmonary arterial hypertension that currently requires treatment.
7. 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.
8. Use of any cytotoxic agents within 4 weeks of dosing.
9. 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).
10. Use of Esbriet® (pirfenidone) or Ofev® (nintedanib) within 30 day prior to first dose.
11. 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).
12. 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).
13. Use of acetaminophen (paracetamol) at a dosage > 3 grams per day within 2 weeks of first study dosing.
14. Use of niacin at a dosage > 2 grams/day within 2 weeks prior to first study dosing.
15. Any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from participating in the study.
16. History of recurrent bacterial infections (at least three major infections resulting in hospitalization and/or requiring intravenous antibiotic treatment within the past 2 years)
17. 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.
18. History of active malignancy within 5 years prior to signing the ICF, excluding nonmelanoma skin cancer.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
5/08/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
6/02/2017
Sample size
Target
Accrual to date
Final
16
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
The Prince Charles Hospital - Chermside
Recruitment hospital [2] 0 0
St. Vincent's Hospital- Sydney - Darlinghurst
Recruitment postcode(s) [1] 0 0
4032 - Chermside
Recruitment postcode(s) [2] 0 0
2010 - Darlinghurst
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Florida
Country [2] 0 0
United States of America
State/province [2] 0 0
Indiana
Country [3] 0 0
United States of America
State/province [3] 0 0
Pennsylvania
Country [4] 0 0
United States of America
State/province [4] 0 0
Tennessee

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Celgene
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Participation in the study will last for 3months, with a 1 month screening phase.
Trial website
https://clinicaltrials.gov/study/NCT02510937
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Ed O'Mara, MD
Address 0 0
Celgene Corporation
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT02510937