Clinical trial NCT02846324: Study to Evaluate the Safety and Tolerability of GBT440 Administered to Subjects With IPF
Official title: 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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Idiopathic Pulmonary Fibrosis
Hypoxemia
Lead sponsor: Global Blood Therapeutics
Sponsor class: INDUSTRY
First posted: 2016-07-27
Enrollment: 39
Sex: ALL
Minimum age (years): 45
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: SUPPORTIVE_CARE

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.03
timeFrame: Days 1 to 58
Secondary outcomes:
measure: Pharmacokinetic Profile of GBT440 Including Maximum Concentration
timeFrame: 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
measure: Pharmacokinetic Profile of GBT440 Including Minimum Concentration
timeFrame: 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
measure: Pharmacokinetic Profile of GBT440 Including Apparent Terminal Half Life
timeFrame: 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
measure: Evaluate the Effect of GBT440 on Oxygen Saturation Using Pulse Oximetry at Rest
timeFrame: Baseline to Day 28
measure: Evaluate the Effect of GBT440 on Oxygen Saturation Using Pulse Oximetry After Exercise
timeFrame: Baseline to Day 28
Other outcomes:
Locations:
city: Birmingham
country: United States
state: Alabama
city: San Francisco
country: United States
state: California
city: Denver
country: United States
state: Colorado
city: Miami
country: United States
state: Florida
city: Kansas City
country: United States
state: Kansas
city: New Orleans
country: United States
state: Louisiana
city: New York
country: United States
state: New York
city: Philadelphia
country: United States
state: Pennsylvania
city: Charleston
country: United States
state: South Carolina
city: Nashville
country: United States
state: Tennessee
city: Salt Lake City
country: United States
state: Utah
city: Falls Church
country: United States
state: Virginia
Study officials:
References: