Principal Investigator: Mary  Scholand
Keywords: pulmonary , ipf , idiopathic pulmonary fibrosis , pulmonary fibrosis , ILD , Interstitial Lung Disease Department: Pulmonary
IRB Number: 00110331
Specialty: Pulmonary, Pulmonary, Pulmonary, Pulmonary
Sub Specialties: Pulmonary Fibrosis, Pulmonary Function, General Pulmonary, Pulmonary
Recruitment Status: Active, not recruiting

Contact Information

Chloe Kirkpatrick
chloe.kirkpatrick@hsc.utah.edu
8015815811

Brief Summary

The purpose of this study is to investigate the safety, tolerability and efficacy of
VAY736 as potential therapy for the treatment of idiopathic pulmonary fibrosis
(IPF).

Primary Objective: 

To assess the efficacy of VAY736 in patients with IPF by looking at the change
from baseline to end-of-treatment (48 weeks of treatment) in forced vital
capacity (FVC).

Secondary
Objectives
 To assess the impact of VAY736 on safety
 To assess the impact of VAY736 on survival
 To assess the impact of VAY736 on "progression-free survival (PFS)
 To assess the impact of VAY736 on "disease progression"
 To assess the impact of VAY736 on a "Composite Endpoint'
 To assess the impact of VAY736 on pulmonary physiology
 To assess the impact of VAY736 on exercise capacity
 To assess the impact of VAY736 on gas exchange
 To assess the immunogenicity of VAY736 in IPF patients
 To assess the pharmacokinetics of VAY736 after multiple s.c. doses in
IPF patients

Inclusion Criteria

Those eligible for inclusion in this study must fulfill all of the following criteria:
1. Written informed consent must be obtained before any assessment is performed.
2. Male and female subjects 40 to 80 years of age inclusive
3. A diagnosis of definite or probable IPF within 5 years of the screening visit, as defined by
Figure 3, Tables 4-6 of the ATS/ERS/JRS/ALAT Diagnostic Guidelines
(Raghu et al 2011)
4. FVC 40-90% predicted (inclusive)
5. DLCO, corrected for hemoglobin, 25-79% predicted (inclusive)
6. FEV1/FVC >70%
7. Unlikely to die from cause other than IPF within the next 3 years, in the opinion of the
investigator
8. Unlikely to undergo lung transplantation during this trial
9. Able to communicate well with the investigator, to understand and comply with the
requirements of the study.

Exclusion Criteria

Those fulfilling any of the following criteria are not eligible for inclusion in this study:
1. Emphysema > fibrosis on screening HRCT (must be confirmed by central reader)
2. Active viral, bacterial or other infections requiring systemic treatment at the time of
screening or enrollment, or history of recurrent clinically significant infection or of
bacterial infections with encapsulated organisms
3. History of major organ, hematopoietic stem cell or bone marrow transplant

4. History of hypersensitivity to any of the study drugs or to drugs of similar chemical
classes (e.g., mAb of IgG1 class) or to any of the constituents of the study drug (sucrose,
L-Arginine hydrochloride, L-histidine, polysorbate 80, hydrochloric acid)
5. Receipt of live/attenuated vaccine within a 2 month period before baseline
6. History of primary or secondary immunodeficiency, including a positive Human
Immunodeficiency Virus (HIV) (Enzyme-linked Immunosorbent Assay (ELISA) and
Western blot) test result
7. History of malignancy of any organ system (other than localized basal cell carcinoma of
the skin, in situ cervical cancer), treated or untreated, within the past 5 years, regardless of
whether there is evidence of local recurrence or metastases
8. Any one of the following screening values of complete blood count laboratory values:
Hemoglobin levels below 8.0 g/dL; Total leukocyte count less than 2,000/μL; Platelets
<100.0 x 109/L; Absolute neutrophil count (ANC) <1.5 x 109/L
9. Any surgical, medical (e.g., uncontrolled hypertension, heart failure or diabetes),
psychiatric or additional physical condition that the Investigator feels may jeopardize the
patient in case of participation in this study
10. Positive hepatitis B surface antigen (HBsAg) with concurrent negative hepatitis B surface
antibody (anti-HBs); or positive total hepatitis B core antibody (anti-HBc) with concurrent
negative anti-HBs; or positive hepatitis C antibody (anti-HCV); i.e., any acute or chronic
infection with hepatitis B or hepatitis C unless it can be documented that the patient has received highly effective HCV-specific antiviral therapy, HCV RNA levels measured, and HCV RNA is undetectable; i.e., any acute or chronic infection with hepatitis B or hepatitis C.
11. Evidence of active or latent tuberculosis (TB) infection, as determined by Quantiferon test
(after anti-TB treatment, patients with history of or latent TB may become eligible
according to national guidelines)
12. Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female
after conception and until the termination of gestation, confirmed by a positive human
chorionic gonadotropin (hCG) laboratory test
13. Clinically diagnosed AE-IPF or other significant clinical worsening within 3 months of
randomization
14. Other known causes of interstitial lung disease (e.g., domestic or occupational
environmental exposures, drug toxicity) or other identifiable interstitial lung disease
15. Definitive diagnosis of a connective tissue disease (such as systemic sclerosis, DM/PM,
RA, Sjogren's syndrome, or SLE)
16. Initiation of pulmonary rehabilitation within 60 days of randomization (pulmonary
rehabilitation is prohibited during the period of this trial, except for “maintenance”
rehabilitation, to be documented with a clinical summary from the Rehabilitation Center)
17. Myocardial Infarction (MI), Cerebrovascular Accident (CVA), Transient Ischemic Attack
(TIA), or hospitalization for arrhythmia or unstable angina within 6 months
18. New York Heart Association (NYHA) class III/IV Congestive Heart Failure (CHF),
Ejection Fraction (EF) <25%
19. Other investigational treatments within 6 months of screening
20. Disability (other than dyspnea) that may limit the completion of 6MWT (angina,
claudication, etc.)
21. Current smoker
22. Any current treatment for IPF (except for pirfenidone or nintedanib; but not both)
23. Historical (within 6 months prior to screening) treatment for IPF with experimental or off-label
modalities including but not limited to oral corticosteroids, N-Acetylcysteine ,
cyclophosphamide, mycophenolate, azathioprine, cyclosporine A, etanercept, or
plasmapheresis.

  • Treatment for any indication with any of the following drugs within 6 months prior to screening: oral corticosteroids, N-Acetylcysteine, cyclophosphamide, mycophenolate, azathioprine, cyclosporine A, etanercept, or plasmapheresis. (Note: The aforementioned treatments are systemically distributed and, therefore, might potentially affect the lung regardless of target indication).

24. Prior use of any B-cell depleting therapy (e.g., rituximab, ofatumumab, or other anti-CD20
mAb, anti-CD40, anti-CD19,anti-CD22 mAb, anti-CD52 mAb, or anti-BAFF mAb)
25. Receiving any treatment for pulmonary hypertension OR treatment for pulmonary
hypertension anticipated during this trial (in other words, treatment for pulmonary
hypertension is prohibited during this trial), including but not limited to epoprostanil,
iloprostanil, treprostanil, selexipag, bosentan, ambrisentan, macitentan, sildenafil, tadalafil,
and riociguat.
26. History of alcohol and/or drug abuse within the last 2 years
27. Elective surgery planned to take place during this trial
28. Women of child-bearing potential (WOCBP), defined as all women physiologically
capable of becoming pregnant, unless they are using highly effective methods of
contraception for 3 months prior to screening, during dosing, and for 4 months after
stopping of investigational medication.
Highly effective contraception methods include:
 Total abstinence from heterosexual intercourse (when this is in line with the preferred
and usual lifestyle of the patient). Periodic abstinence (e.g. calendar, ovulation,
symptothermal, post-ovulation methods) and withdrawal are not acceptable methods
of contraception.
 Female sterilization (have had surgical bilateral oophorectomy with or without
hysterectomy), total hysterectomy or tubal ligation at least six weeks before taking
investigational drug. In case of oophorectomy alone, only when the reproductive
status of the woman has been confirmed by follow up hormone level assessment.
 Male sterilization (at least 6 months prior to screening). For female subjects on the
study the vasectomized male partner should be the sole partner for that subject.
 Use of oral (estrogen and progesterone), injected or implanted hormonal methods of
contraception or placement of an intrauterine device (IUD) or intrauterine system
(IUS) or other forms of hormonal contraception that have comparable efficacy (failure
rate <1%), for example hormone vaginal ring or transdermal hormone contraception.
In case of use of oral contraception women should have been stable on the same pill
for a minimum of 3 months before taking investigational drug.