Clinical Trials Register

Summary
EudraCT Number:	2016-003473-17
Sponsor's Protocol Code Number:	CC-90001-IPF-001
National Competent Authority:	UK - MHRA
Clinical Trial Type:	EEA CTA
Trial Status:	GB - no longer in EU/EEA
Date on which this record was first entered in the EudraCT database:	2017-02-24
Trial results	View results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

UK - MHRA

A.2

EudraCT number

2016-003473-17

A.3

Full title of the trial

A Phase 2, 24-Week Randomized, Double-blind, Placebo-Controlled
Multicenter Study, With an 80-Week Active Treatment Extension, to
Evaluate the Efficacy and Safety of CC-90001 in Subjects with Idiopathic
Pulmonary Fibrosis

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

Investigation to Efficacy and Safety of CC-90001 in patients with Idiopathic Pulmonary Fibrosis

A.4.1

Sponsor's protocol code number

CC-90001-IPF-001

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Celgene Corporation
B.1.3.4	Country	United States
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Celgene Corporation
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Celgene Corporation
B.5.2	Functional name of contact point	Clinical Trial Disclosure
B.5.3	Address:
B.5.3.1	Street Address	86 Morris Avenue
B.5.3.2	Town/ city	NJ
B.5.3.3	Post code	07901
B.5.3.4	Country	United States
B.5.4	Telephone number	+1 913 709-6862
B.5.6	E-mail	ClinicalTrialDisclosure@celgene.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.2	Product code	CC-90001
D.3.4	Pharmaceutical form	Film-coated tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Not available
D.3.9.1	CAS number	1403859-14-2
D.3.9.2	Current sponsor code	CC-90001
D.3.9.4	EV Substance Code	AS1
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	200
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Film-coated tablet
D.8.4	Route of administration of the placebo	Oral use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

IDIOPATHIC PULMONARY FIBROSIS

E.1.1.1

Medical condition in easily understood language

chronic, progressive disease that causes scarring (fibrosis) in lungs

E.1.1.2

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10067761
E.1.2	Term	Exacerbation of idiopathic pulmonary fibrosis
E.1.2	System Organ Class	100000004855

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

To evaluate the effect of CC-90001, 200 mg and 400 mg, when orally administered (PO) once
daily (QD), compared with placebo, on percent of predicted forced vital capacity (FVC) after
24 weeks of treatment in subjects with IPF.

E.2.2

Secondary objectives of the trial

To evaluate the effects of CC-90001, 200 mg and 400 mg PO QD, compared to placebo, after 24 weeks of treatment in subjects with IPF, on:
- FVC (milliliters [mL])
- Six-minute Walk Test (6MWT)
- Disease progression
- Heath-related quality of life: St. George’s Respiratory Questionnaire (SGRQ) and University of California San Diego-Shortness of Breath Questionnaire (UCSDSOBQ)
- Dose response
- Safety and tolerability

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Subject is male or female ≥ 40 years of age at the time of signing the
informed consent form (ICF)
2. Subject must understand and voluntarily sign an ICF prior to any
study-related assessments/procedures being conducted
3. Subject is willing and able to adhere to the study visit schedule and
other protocol requirements
4. Investigator has considered all available IPF treatment options with
the potential subject before consenting the subject for participation in
the study
5. Diagnosis of IPF is supported by HRCT as described in the Protocol
6. Extent of fibrotic changes (eg, honeycombing, reticular changes)
greater than the extent of emphysema on HRCT scan, as determined by
central review
7. No features supporting an alternative diagnosis on transbronchial
biopsy, bronchoalveolar lavage (BAL), or SLB, if performed
8. Percent predicted forced vital capacity ( FVC) ≥ 45% at Screening
confirmed by central review
9. Change in FVC (measured in milliliters [mL]) between Screening and
Day 1 less than a 10% relative difference, calculated as: the absolute
value of 100% * (Screening FVC [mL] - Day 1 FVC [mL]) / Screening FVC
(mL)
10.Hemoglobin-corrected percent predicted diffusion capacity of the lung
for carbon monoxide (DLCO) ≥ 25% and ≤ 85% predicted at Screening
11. Able to walk ≥ 150 meters during the 6-minute walk test (6MWT) at
Screening
12. Investigator has considered all available IPF treatment options with
the potential subject before consenting the subject for participation in
the study
Females of childbearing potential (FCBP) 1 must:
a. Have two negative pregnancy tests as verified by the Investigator
prior to starting IP. She must agree to ongoing pregnancy testing during
the course of the study, and after end of study treatment. This applies
even if the subject practices true abstinence from heterosexual contact.
[refer to protocol]
b. Either commit to true abstinence* from heterosexual contact (which
must be reviewed on a monthly basis and source documented) or agree
to use two effective birth control methods (one of which is highly
effective) at the same time, and be able to comply with, effective
contraception without interruption, 28 days prior to starting IP, during
the study therapy (including dose interruptions), and for 28 days after
discontinuation of IP [Refer to protocol]
13. Male subjects must:
Practice true abstinence (which must be reviewed on a monthly basis) or
agree to use a latex condom or nonlatex condom not made out of natural
(animal) membrane (eg, polyurethane) during sexual contact with a
pregnant female or a female of childbearing potential while participating
in the study, during dose interruptions and for at least 28 days following
IP discontinuation, even if he has undergone a successful vasectomy.
14. For subjects stratified to the protocol-allowable standard of care
therapy group (only): Subjects must be receiving and agree to maintain
the same dose of protocol-allowable standard of care (SOC) therapy for
at least 8 weeks prior to Screening Visit 1 and must agree to continue
this dose through Visit 9/Week 24. Adjustments in pirfenidone dose
after randomization (Visit 2) may be allowed for safety or tolerability
reasons, according
to the pirfenidone label.

E.4

Principal exclusion criteria

1. Subject has any significant medical condition, laboratory abnormality,
or psychiatric illness that would prevent the subject from participating in
the study
2. Subject has any condition including the presence of laboratory
abnormalities, which places the subject at unacceptable risk if he/she
were to participate in the study
3. Subject has any condition that confounds the ability to interpret data
from the study
4. Significant clinical worsening of IPF between Screening and Baseline
(Visit 2), in the opinion of the Investigator
5. Subjects with any of the following laboratory criteria:
• White blood cell count (WBC) < 3500/mm3 (< 3.5 X 109/L) or >
14,000/mm3 (> 14 X 109/L)
• Platelet count < 120,000/μL (< 120 X 109/L)
• Serum creatinine > 1.5 mg/dL (> 132.6 μmol/L)
• Aspartate aminotransferase (AST/SGOT) > 1.5 X upper limit of normal
(ULN)
• Alanine aminotransferase (ALT/SGPT) > 1.5 X upper limit of normal (ULN)
• Total bilirubin > 2 mg/dL (> 34.2 μmol/L)
• Hemoglobin < 10 g/dL (< 100 g/L)
6. Subject with a QTcF > 450 msec
7. Any condition other than IPF that in the opinion of the Investigator is
likely to result in the death of the subject within the next year
8. Inability to obtain reproducible, high-quality pulmonary function tests.
9. Evidence of clinically relevant airways obstruction (ie, FEV1/FVC <
0.7) at Screening and/or significant respiratory disorder/pathology (eg,
pulmonary arterial hypertension requiring treatment, asthma,
tuberculosis, sarcoidosis, hypersensitivity pneumonitis, aspergillosis,
asbestosis, neoplastic disease, cystic fibrosis or other interstitial lung
disease) other than IPF
10. Subject is likely to have lung transplantation during the first 24
weeks of the study (being on transplantation list is acceptable for
participation)
11. Impairment (other than dyspnea) limiting the ability to comply with
study requirements (eg, pulmonary function tests, 6-minute walk test)
12. Subjects using the following medications: nintedanib, endothelium
receptor antagonists (eg, bosentan, ambrisentan), interferon gamma-1b,
imatinib mesylate, N-acetylcysteine, azathioprine, cyclophosphamide,
methotrexate, mycophenolate mofetil, cyclosporine, and oral steroids
(eg, prednisone > 12.5 mg/day or equivalent) within 4 weeks prior to
the Screening Visit. (Note: Refer to protocol.)
13. Use of any cytokine modulator/biologic, such as etanercept,
adalimumab, efalizumab, infliximab, or rituximab within 12 weeks of
randomization
14. Use of an inhaled long-acting bronchodilator within 24 hours of the
Screening Visit or short-acting bronchodilator within 8 hours of the
Screening Visit
15. Use of drugs that are known to cause hepatotoxicity, such as, but not
limited to, acetaminophen (paracetamol) at dosages of > 3 grams/day
and niacin dosage of > 2 grams/day while on study or within 2 weeks of
first dose of IP
16. Use of any medications that are substrates of one or more of the
transporters P-gp, BCRP, OAT3, OATP1B1, OATP1B3, and OCT2 and have
a narrow therapeutic index (eg, digoxin, mycophenolate mofetil)
17. History of recent (within 6 months of Screening) deep vein
thrombosis (DVT) or pulmonary embolism (PE) and/or recurrent DVT or
recurrent PE
18. History of cardiac valve replacement requiring chronic
anticoagulation therapy
19. History of congenital and/or acquired immunodeficiencies (eg,
common variable immunodeficiency, human immunodeficiency virus
[HIV], etc)
20. History of hepatitis B and/or hepatitis C, including those considered
successfully treated/cured
21. Active or history of recurrent bacterial, viral, fungal, mycobacterial
or other infections (including, but not limited to, atypical mycobacterial
disease and herpes zoster), or any major episode of infection requiring
hospitalization or treatment with intravenous or oral antibiotics within 4
weeks of the Screening Visit and at any time during the Screening Phase,
up through the first dose of IP
22. History of active or latent tuberculosis (TB) infection, unless there is
medical record documentation of successful completion of a standard
course of treatment considered appropriate, based on local prevalence of
multi-drug resistant TB and consistent with WHO guidelines [Refer to
protocol]
23. Subject has had a household contact with a person with active TB
and subject did not receive appropriate and documented prophylaxis for
TB [Refer to protocol]
24. History of end-stage renal disease requiring dialysis
25. History of severe hepatic impairment or end-stage liver disease
26. History of Gilbert's syndrome
27. History of alcohol or drug abuse with

E.5 End points

E.5.1

Primary end point(s)

Forced vital capacity (FVC) - Percentage point difference in % predicted FVC

E.5.1.1

Timepoint(s) of evaluation of this end point

Baseline to Week 24

E.5.2

Secondary end point(s)

1. FVC - Absolute change and rate of decline in FVC (expressed in mL)
2. 6-minute Walk Test (6MWT) with Borg Scale:
- Change in the distance walked during the 6MWT as measured in meters (m)
- Change in dyspnea rating on Borg Scale
3. Disease progression:
- Death from respiratory failure, or
- Absolute decrease of ≥ 10% from baseline in % predicted FVC at two consecutive evaluations at a minimum of 4 weeks between evaluations, or
- Decrease from baseline of ≥ 50 meters in 6MWT distance (in the absence of a readily explainable cause, such as injury or trauma), or
- Unexplained worsening hypoxemia (an absolute decrease from
baseline of 4% or more in arterial oxygen saturation by pulse oximetry
[SpO2]).).
4. Quality of life :
- Change from Baseline in total score and domains including cough on the Saint George’s Respiratory Questionnaire (SGRQ)
- Change from Baseline in the University of California San Diego Shortness of Breath Questionnaire (UCSD- SOBQ)
5. Safety and tolerability - Type, frequency, severity, and relationship of AEs, clinical laboratory tests including urine cytology, 12-lead ECG, vital signs, and physical examination

E.5.2.1

Timepoint(s) of evaluation of this end point

1. FVC - Baseline through Week 24
2. 6-minute Walk Test (6MWT) with Borg Scale:
- Baseline to Week 24;
- Baseline to Week 52;
- Baseline to Week 76;
- Baseline to Week 104;
- Week 24 to Week 52;
- Week 24 to Week 104
3. Disease progression - Baseline through Week 24
4. Quality of life - Baseline through Week 24
5. Safety and tolerability - Signing of the informed consent form through Week 108 (4-week post-treatment observational follow-up)

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

Yes

E.6.10

Pharmacogenetic

Yes

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

Yes

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Australia

Brazil

Canada

Colombia

France

Germany

Greece

Romania

Russian Federation

Taiwan

Turkey

United Kingdom

United States

E.8.7

Trial has a data monitoring committee

Yes

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1