Study to Evaluate the Safety and Tolerability of GBT440 Administered to Subjects With IPF
Phase 2
Completed
- Conditions
- Idiopathic Pulmonary FibrosisHypoxemia
- Interventions
- Drug: Placebo
- Registration Number
- NCT02846324
- Lead Sponsor
- Global Blood Therapeutics
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 39
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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description GBT440 600 mg Dose GBT440 Parts A and B GBT440 1500 mg Dose GBT440 Part B Placebo Placebo Parts A and B GBT440 900 mg Dose GBT440 Part A
- Primary Outcome Measures
Name Time Method Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.03 Days 1 to 58
- Secondary Outcome Measures
Name Time Method 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 After Exercise Baseline to Day 28 Change from Baseline to Day 28 after 6MWT
Evaluate the Effect of GBT440 on Oxygen Saturation Using Pulse Oximetry at Rest Baseline to Day 28 Data reported is change from baseline in Oxygen Saturation at rest
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