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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/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
Treatment: Drugs - CC-90001

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


Treatment: Drugs: CC-90001


Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Adverse Events (AEs) - Number of subjects with adverse events
Timepoint [1] 0 0
up to 16 weeks
Primary outcome [2] 0 0
Dose interruptions, reductions, and discontinuation - Number of subjects experiencing dose interruptions, reductions, and discontinuation of CC-90001 secondary to an AE
Timepoint [2] 0 0
up to 16 weeks
Primary outcome [3] 0 0
Complete PEs - Complete Physical Examinations
Timepoint [3] 0 0
up to 16 weeks
Primary outcome [4] 0 0
Clinical laboratory assessments - Clinical laboratory assessments
Timepoint [4] 0 0
up to 16 weeks
Primary outcome [5] 0 0
Vital sign measurements - Heart rate (HR), respiratory rate, blood pressure (BP), and body temperature
Timepoint [5] 0 0
up to 16 weeks
Primary outcome [6] 0 0
12-lead ECGs - 12-lead ECGs
Timepoint [6] 0 0
up to 16 weeks
Primary outcome [7] 0 0
Urine pregnancy tests - Urine pregnancy tests
Timepoint [7] 0 0
up to 16 weeks
Primary outcome [8] 0 0
Concomitant medications and procedures - Concomitant medications and procedures
Timepoint [8] 0 0
up to 16 weeks
Secondary outcome [1] 0 0
CC-90001 plasma concentrations - CC-90001 plasma concentrations collected sparsely and measured using a validated liquid chromatography tandem mass spectrometry assay
Timepoint [1] 0 0
up to 16 weeks
Secondary outcome [2] 0 0
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.
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
Gender
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
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
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/show/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
Other publications