Study to Evaluate the Effect of GBT440 Administered to Subjects With IPF on Supplemental Oxygen at Rest

Phase 2

Terminated

• Conditions: Idiopathic Pulmonary Fibrosis; Hypoxemia
• Interventions: Drug: GBT440

• Registration Number: NCT02989168
• Lead Sponsor: Global Blood Therapeutics

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2016-11
• Study First Submitted: 2016-11-28
• Study First Submitted QC: 2016-12-07
• Study First Posted: 2016-12-12
• Primary Completion: 2017-10-23
• Study Completion: 2017-10-23
• Results First Submitted: 2020-05-04
• Status Verified: 2020-07
• Results First Submitted QC: 2020-07-31
• Last Update Submitted: 2020-07-31
• Results First Posted: 2020-08-21
• Last Update Posted: 2020-08-21

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 14

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
GBT440 900 mg Dose	GBT440	Part A, 900 mg GBT440: Capsules which contain GBT440 drug substance in Swedish orange
GBT440 1500 mg Dose	GBT440	Part B , 1500 mg GBT440: Capsules which contain GBT440 drug substance in Swedish orange

Primary Outcome Measures

Name	Time	Method
Change in Oxygen Saturation at End of Treatment Period Compared to Baseline	Days 1 to 90

Secondary Outcome Measures

Name	Time	Method
Evaluate the Effect of GBT440 on Performance of the 6MWT	Days 1 to 90
Pharmacokinetic Parameters of GBT440 in Plasma and Whole Blood (Minimum Concentration (Cmin))	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)	PK parameters of GBT440 administered daily orally for 90 days in plasma and whole blood, including but not limited to, minimum concentration (Cmin), at steady state.
Evaluate the Effect of GBT440 on IPF Related Symptoms Using Patient Related Outcomes	Days 1 to 90
Change in Supplemental Oxygen Requirement at End of Treatment Period Compared to Baseline	Days 1 to 90
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	Days 1 to 90
Evaluate Pulmonary Function Using Pulmonary Function Tests (FVC and DLco)	Days 1 to 90	Spirometry measurements will include forced vital capacity (FVC) and diffusing capacity of the lung for carbon monoxide (DLco).
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0	Days 1 to 90