Study to Evaluate the Safety and Tolerability of GBT440 Administered to Subjects With IPF

A Phase II Randomized, Placebo-Controlled Study of GBT440 to Evaluate the Safety, Tolerability, Pharmacokinetics and Effect on Hypoxemia in Subjects With Idiopathic Pulmonary Fibrosis (IPF)

This study is a randomized, double-blind, placebo-controlled trial in which eligible IPF subjects will be randomized to receive GBT440 or Placebo orally daily.

Study Overview

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

• Study Type: Interventional
• Enrollment (Actual): 39
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
  • California
    • San Francisco, California, United States, 94143
  • Colorado
    • Denver, Colorado, United States, 80206
  • Florida
    • Miami, Florida, United States, 33136
  • Kansas
    • Kansas City, Kansas, United States, 66160
  • Louisiana
    • New Orleans, Louisiana, United States, 70122
  • New York
    • New York, New York, United States, 10032
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19145
  • South Carolina
    • Charleston, South Carolina, United States, 29425
  • Tennessee
    • Nashville, Tennessee, United States, 37212
  • Utah
    • Salt Lake City, Utah, United States, 84108
  • Virginia
    • Falls Church, Virginia, United States, 22042

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 45 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Documented diagnosis of IPF.
• Oxygen desaturation with exercise.
• Completion of the baseline 6MWT
• Weight ≥ 50 kg
• Male or female of child bearing potential willing and able to use highly effective methods of contraception from study start to 3 months after the last dose of study drug.

Exclusion Criteria:

• FEV1/FVC < 70%.
• Subjects on supplemental oxygen therapy at rest.
• History of other interstitial lung diseases.
• Significant polycythemia.
• Female who is breast-feeding or pregnant.
• Known current malignancy or history of malignancy within the last 2 years prior to screening.
• Use of cytotoxic and/or immunosuppressant medications within 30 days screening.
• Hospitalization due to an exacerbation of IPF within 30 days of screening
• Subject plans to begin or has commenced pulmonary rehabilitation within 30 days of screening
• Corticosteroids (> 10 mg per day of prednisone or an equivalent) within 30 days of screening.
• Current smoker or history of smoking within 3 months of screening.
• Currently or, in the opinion of the investigator, soon to be listed for lung transplant.
• History of unstable or deteriorating cardiac or pulmonary disease (other than IPF) within 6 months of screening.
• Any condition possibly affecting drug absorption.
• 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).
• Subject who, for any reason, is deemed by the investigator to be inappropriate for this study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Supportive Care
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: GBT440 600 mg Dose; Parts A and B	Drug: GBT440; Capsules which contain GBT440 drug substance in Swedish orange
Experimental: GBT440 900 mg Dose; Part A	Drug: GBT440; Capsules which contain GBT440 drug substance in Swedish orange
Experimental: GBT440 1500 mg Dose; Part B	Drug: GBT440; Capsules which contain GBT440 drug substance in Swedish orange
Placebo Comparator: Placebo; Parts A and B	Drug: Placebo; Placebo capsules

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.03	Days 1 to 58

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pharmacokinetic Profile of GBT440 Including Maximum Concentration		Days 1 and 28: pre-dose, and 2 hours post dose (+/- 15 minutes); Day 2: 24 hours (+/- 2 hours) after the Day 1 dose and prior to receiving the Day 2 dose; Days 8 & 15: pre-dose only
Pharmacokinetic Profile of GBT440 Including Minimum Concentration		Days 1 and 28: pre-dose, and 2 hours post dose (+/- 15 minutes); Day 2: 24 hours (+/- 2 hours) after the Day 1 dose and prior to receiving the Day 2 dose; Days 8 & 15: pre-dose only
Pharmacokinetic Profile of GBT440 Including Apparent Terminal Half Life		Days 1 and 28: pre-dose, and 2 hours post dose (+/- 15 minutes); Day 2: 24 hours (+/- 2 hours) after the Day 1 dose and prior to receiving the Day 2 dose; Days 8 & 15: pre-dose only
Evaluate the Effect of GBT440 on Oxygen Saturation Using Pulse Oximetry at Rest	Data reported is change from baseline in Oxygen Saturation at rest	Baseline to Day 28
Evaluate the Effect of GBT440 on Oxygen Saturation Using Pulse Oximetry After Exercise	Change from Baseline to Day 28 after 6MWT	Baseline to Day 28

Collaborators and Investigators

• Sponsor: Global Blood Therapeutics

Study record dates

Study Major Dates

• Study Start: June 1, 2016
• Primary Completion (Actual): September 1, 2017
• Study Completion (Actual): November 1, 2017

Study Registration Dates

• First Submitted: July 7, 2016
• First Submitted That Met QC Criteria: July 22, 2016
• First Posted (Estimate): July 27, 2016

Study Record Updates

• Last Update Posted (Actual): October 20, 2020
• Last Update Submitted That Met QC Criteria: September 25, 2020
• Last Verified: September 1, 2020

More Information

Terms related to this study

• 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: GBT440-006