Study to Evaluate the Effect of GBT440 Administered to Subjects With IPF on Supplemental Oxygen at Rest (Zephyr)

A Phase II Open Label Study to Evaluate the Effect of GBT440 on Hypoxemia in Subjects With Idiopathic Pulmonary Fibrosis (IPF) Who Are Using Supplemental Oxygen at Rest (ZEPHYR)

This is an open label study in which eligible IPF subjects who are using supplemental oxygen at rest will receive GBT440 orally daily.

Study Overview

• Status: Terminated
• Conditions: Idiopathic Pulmonary Fibrosis; Hypoxemia
• Intervention / Treatment: Drug: GBT440

• Study Type: Interventional
• Enrollment (Actual): 14
• Phase: Phase 2

Contacts and Locations

Study Locations

• United Kingdom
  • London, United Kingdom, SW3 6NP
  • Manchester, United Kingdom, M23 9QZ
  • Southampton, United Kingdom, SO16 6YD
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
  • Colorado
    • Denver, Colorado, United States, 80206
  • Louisiana
    • New Orleans, Louisiana, United States, 70122
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19140
  • Tennessee
    • Nashville, Tennessee, United States, 37232
  • Utah
    • Salt Lake City, Utah, United States, 84108

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 45 years to 85 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Documented diagnosis of IPF.
• Receiving supplemental oxygen for use at rest.
• Weight ≥ 40 kg.
• Male or female of child bearing potential willing and able to use highly effective methods of contraception from study start to 30 days after the last dose of study drug.

Exclusion Criteria:

• FEV1/FVC < 70%
• History of other interstitial lung diseases.
• Subject plans to begin or has commenced pulmonary rehabilitation within 30 days of screening.
• Corticosteroid (> 10 mg per day of prednisone or an equivalent) administered for 7 days or longer, within 30 days of screening.
• 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).
• Female who is breast-feeding or pregnant
• Current smoker or history of smoking within 3 months from screening

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Supportive Care
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: GBT440 900 mg Dose; Part A, 900 mg GBT440: Capsules which contain GBT440 drug substance in Swedish orange	Drug: GBT440; GBT440: Capsules which contain GBT440 drug substance in Swedish orange
Experimental: GBT440 1500 mg Dose; Part B , 1500 mg GBT440: Capsules which contain GBT440 drug substance in Swedish orange	Drug: GBT440; GBT440: Capsules which contain GBT440 drug substance in Swedish orange

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Change in Oxygen Saturation at End of Treatment Period Compared to Baseline	Days 1 to 90

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Supplemental Oxygen Requirement at End of Treatment Period Compared to Baseline		Days 1 to 90
Evaluate the Effect of GBT440 on Resting and Post-exercise Alveolar-arterial O2 Tension Difference [P(A-a) O2] at End of Treatment Period Compared to Baseline		Days 1 to 90
Evaluate the Effect of GBT440 on Performance of the 6MWT		Days 1 to 90
Evaluate the Effect of GBT440 on IPF Related Symptoms Using Patient Related Outcomes		Days 1 to 90
Evaluate Pulmonary Function Using Pulmonary Function Tests (FVC and DLco)	Spirometry measurements will include forced vital capacity (FVC) and diffusing capacity of the lung for carbon monoxide (DLco).	Days 1 to 90
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0		Days 1 to 90
Pharmacokinetic Parameters of GBT440 in Plasma and Whole Blood (Minimum Concentration (Cmin))	PK parameters of GBT440 administered daily orally for 90 days in plasma and whole blood, including but not limited to, minimum concentration (Cmin), at steady state.	Day 1 (15 mins post-dose and 2-4h post-dose), Day 15 (pre-dose), Day 30 (pre-dose), Day 60 (pre-dose and 2-4h post-dose), Day 90 (pre-dose), Day 105 (during study visit), and Day 120 (during study visit)

Collaborators and Investigators

• Sponsor: Global Blood Therapeutics

Study record dates

Study Major Dates

• Study Start: November 1, 2016
• Primary Completion (Actual): October 23, 2017
• Study Completion (Actual): October 23, 2017

Study Registration Dates

• First Submitted: November 28, 2016
• First Submitted That Met QC Criteria: December 7, 2016
• First Posted (Estimate): December 12, 2016

Study Record Updates

• Last Update Posted (Actual): August 21, 2020
• Last Update Submitted That Met QC Criteria: July 31, 2020
• Last Verified: July 1, 2020

More Information

Terms related to this study

• Keywords: oxygen; Idiopathic pulmonary fibrosis; hypoxemia
• Additional Relevant MeSH Terms: Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Signs and Symptoms, Respiratory; Fibrosis; Pulmonary Fibrosis; Idiopathic Pulmonary Fibrosis; Hypoxia
• Other Study ID Numbers: GBT440-026