Idiopathic Pulmonary Fibrosis (IPF) MedDRA version: 20.0 Level: PT Classification code 10021240 Term: Idiopathic pulmonary fibrosis System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders
Conditions
Interventions
Product Name: GBT440 300 mg capsule
Product Code: GBT440
Pharmaceutical Form: Capsule
CAS Number: 1446321-46-5
Current Sponsor code: GBT
Sponsors
Global Blood Therapeutics Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) 45 to 85 years of age inclusive, at randomization 2) Able and willing to provide signed informed consent to participate in this study 3) Documented diagnosis of IPF, as indicated in the American Thoracic Society (ATS)/European Respiratory Society (ERS)/Japanese Respiratory Society (JRS)/Latin American Respiratory Society (ALAT) 2011 Guidelines 4) Receiving supplemental O2 for use at rest. • Subjects using O2 only with exercise will not be eligible. 5) Resting oxygen saturation (SpO2) between 85 and 88% for at least 30 consecutive seconds while breathing room air (refer to Section 5.1 and Section 5.5.2 for details on confirming eligibility) Or Resting SpO2 =65 years) yes F.1.3.1 Number of subjects for this age range 24
Exclusion criteria
Exclusion criteria: 1) Forced expiratory volume in 1 second (FEV1) / forced vital capacity (FVC) 10 mg per day of prednisone (or an equivalent), administered for 7 days or longer, within 30 days of screening. • Subjects receiving a stable dose of =10 mg per day of prednisone (or an equivalent) for at least 14 days prior to screening, and in the opinion of the Investigator not anticipated to require a dose adjustment during the study, are eligible for the study 7) Corticosteroid therapy for treatment of non-IPF diseases, unless: • Receiving a stable dose of prednisone (or an equivalent) for at least 14 days prior to screening, and in the opinion of the Investigator not anticipated to require a dose adjustment during the study 8) Participated in another clinical trial of an investigational drug (or medical device) within 30 days or 5-half-lives, whichever is longer, prior to screening, or is currently participating in another trial of an investigational drug (or medical device). 9) Aspartate aminotransferase (AST), alanine aminotransferase (ALT) or total bilirubin >2 × upper limit of normal (ULN) 10) Serum creatinine >2.0 mg/dL 11) Clinical evidence of active infection, within 14 days of screening, which may include but is not limited to bronchitis, pneumonia, urinary tract infection, or cellulitis. 12) Active viral hepatitis within the last 6 months 13) Active tuberculosis within the last 6 months • Testing for latent tuberculosis is not required 14) Electrocardiogram (ECG) with a corrected QT interval using the Fridericia formula (QTcF) >450 ms (males) or QTcF >470 ms (females) • If ventricular pacing is noted on ECG, then QTcF intervals will not be calculated 15) Family or personal history of congenital long QT syndrome 16) Female who is breast-feeding or pregnant 17) Known current malignancy or current evaluation for a potential malignancy or history of malignancy within the past 2 years prior to screening, except for a
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the effect of GBT440 on oxygen saturation at rest, breathing room air, on Days 30 and 90 compared to baseline.; Secondary Objective: • To evaluate the effect of GBT440 on the requirement for supplemental oxygen (O2) at rest and post-exercise at Days 30 and 90 compared to baseline • To evaluate the effect of GBT440 on resting and post-exercise Alveolar-arterial O2 tension difference [P(A-a)O2] at Days 30 and 90 compared to baseline • To evaluate the effect of GBT440 on 6-minute walk distance (6MWD) at Days 30 and 90 compared to baseline • To evaluate the effect of GBT440 on IPF-related symptoms, using patient reported outcomes (PROs), at Days 30 and 90 compared to baseline • To evaluate pulmonary function at Day 90 compared to baseline • To evaluate the safety and tolerability of 900 mg and 1500 mg GBT440 dosed daily for 90 days • To evaluate the pharmacokinetics (PK) of GBT440 ;Primary end point(s): The primary endpoint is change and % change from baseline in oxygen saturation at rest (SpO2 or SaO2), measured while breathing room air.;Timepoint(s) of evaluation of this end point: at Days 30 and 90. | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: Day 30 and Day 90.; Secondary end point(s): • Change and % change from baseline in O2 flow rate (L/min), measured at rest, to maintain a target oxygen saturation (SpO2) of 89 to 92%, at Days 30 and 90. • Change and % change from baseline in O2 flow rate (L/min), measured post-exercise to maintain a target oxygen saturation (SpO2 or SaO2) of 89 to 92%, at Days 30 and 90 • Change and % change from baseline in resting P(A-a) O2 at Days 30 and 90 • Change and % change from baseline in post-exercise P(A-a) O2 at Days 30 and 90 • Change and % change from baseline in 6MWD, at Days 30 and 90 • Change in patient-reported IPF-related symptoms from baseline, measured by ATAQ and SGRQ, at Days 30 and 90 • Change from baseline in FVC and DLco at Day 90 | — |
Countries
United Kingdom, United States
Contacts
Public ContactDirector, Clinical Operations
Global Blood Therapeutics Inc
Outcome results
None listed