Clinical trial NCT02989168: Study to Evaluate the Effect of GBT440 Administered to Subjects With IPF on Supplemental Oxygen at Rest
Official title: 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)
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Idiopathic Pulmonary Fibrosis
Hypoxemia
Keywords: Idiopathic pulmonary fibrosis
hypoxemia
oxygen
Lead sponsor: Global Blood Therapeutics
Sponsor class: INDUSTRY
First posted: 2016-12-12
Enrollment: 14
Sex: ALL
Minimum age (years): 45
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: SUPPORTIVE_CARE

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


Eligibility criteria: 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

Primary outcomes:
measure: Change in Oxygen Saturation at End of Treatment Period Compared to Baseline
timeFrame: Days 1 to 90
Secondary outcomes:
measure: Change in Supplemental Oxygen Requirement at End of Treatment Period Compared to Baseline
timeFrame: Days 1 to 90
measure: 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
timeFrame: Days 1 to 90
measure: Evaluate the Effect of GBT440 on Performance of the 6MWT
timeFrame: Days 1 to 90
measure: Evaluate the Effect of GBT440 on IPF Related Symptoms Using Patient Related Outcomes
timeFrame: Days 1 to 90
measure: Evaluate Pulmonary Function Using Pulmonary Function Tests (FVC and DLco)
timeFrame: Days 1 to 90
measure: Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0
timeFrame: Days 1 to 90
measure: Pharmacokinetic Parameters of GBT440 in Plasma and Whole Blood (Minimum Concentration (Cmin))
timeFrame: 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)
Other outcomes:
Locations:
city: Birmingham
country: United States
state: Alabama
city: Denver
country: United States
state: Colorado
city: New Orleans
country: United States
state: Louisiana
city: Philadelphia
country: United States
state: Pennsylvania
city: Nashville
country: United States
state: Tennessee
city: Salt Lake City
country: United States
state: Utah
city: London
country: United Kingdom
city: Manchester
country: United Kingdom
city: Southampton
country: United Kingdom
Study officials:
References: