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)
1 other identifier
interventional
39
1 country
12
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Jun 2016
Shorter than P25 for phase_2
12 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
June 1, 2016
CompletedFirst Submitted
Initial submission to the registry
July 7, 2016
CompletedFirst Posted
Study publicly available on registry
July 27, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 1, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
November 1, 2017
CompletedResults Posted
Study results publicly available
September 22, 2020
CompletedOctober 20, 2020
September 1, 2020
1.3 years
July 7, 2016
May 4, 2020
September 25, 2020
Conditions
Outcome Measures
Primary Outcomes (1)
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.03
Days 1 to 58
Secondary Outcomes (5)
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
Baseline to Day 28
Evaluate the Effect of GBT440 on Oxygen Saturation Using Pulse Oximetry After Exercise
Baseline to Day 28
Study Arms (4)
GBT440 600 mg Dose
EXPERIMENTALParts A and B
GBT440 900 mg Dose
EXPERIMENTALPart A
GBT440 1500 mg Dose
EXPERIMENTALPart B
Placebo
PLACEBO COMPARATORParts A and B
Interventions
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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).
- +1 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (12)
Unknown Facility
Birmingham, Alabama, 35233, United States
Unknown Facility
San Francisco, California, 94143, United States
Unknown Facility
Denver, Colorado, 80206, United States
Unknown Facility
Miami, Florida, 33136, United States
Unknown Facility
Kansas City, Kansas, 66160, United States
Unknown Facility
New Orleans, Louisiana, 70122, United States
Unknown Facility
New York, New York, 10032, United States
Unknown Facility
Philadelphia, Pennsylvania, 19145, United States
Unknown Facility
Charleston, South Carolina, 29425, United States
Unknown Facility
Nashville, Tennessee, 37212, United States
Unknown Facility
Salt Lake City, Utah, 84108, United States
Unknown Facility
Falls Church, Virginia, 22042, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Nick Vlahakis
- Organization
- Global Blood Therapeutics
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- SUPPORTIVE CARE
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 7, 2016
First Posted
July 27, 2016
Study Start
June 1, 2016
Primary Completion
September 1, 2017
Study Completion
November 1, 2017
Last Updated
October 20, 2020
Results First Posted
September 22, 2020
Record last verified: 2020-09