NCT02989168

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.

Trial Health

60
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
14

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Nov 2016

Shorter than P25 for phase_2

Geographic Reach
2 countries

9 active sites

Status
terminated

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

November 1, 2016

Completed
27 days until next milestone

First Submitted

Initial submission to the registry

November 28, 2016

Completed
14 days until next milestone

First Posted

Study publicly available on registry

December 12, 2016

Completed
11 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 23, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 23, 2017

Completed
2.8 years until next milestone

Results Posted

Study results publicly available

August 21, 2020

Completed
Last Updated

August 21, 2020

Status Verified

July 1, 2020

Enrollment Period

12 months

First QC Date

November 28, 2016

Results QC Date

May 4, 2020

Last Update Submit

July 31, 2020

Conditions

Idiopathic Pulmonary FibrosisHypoxemia

Keywords

Idiopathic pulmonary fibrosishypoxemiaoxygen

Outcome Measures

Primary Outcomes (1)

  • Change in Oxygen Saturation at End of Treatment Period Compared to Baseline

    Days 1 to 90

Secondary Outcomes (7)

  • 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 the Effect of GBT440 on Performance of the 6MWT

    Days 1 to 90

  • Evaluate the Effect of GBT440 on IPF Related Symptoms Using Patient Related Outcomes

    Days 1 to 90

  • Evaluate Pulmonary Function Using Pulmonary Function Tests (FVC and DLco)

    Days 1 to 90

  • +2 more secondary outcomes

Study Arms (2)

GBT440 900 mg Dose

EXPERIMENTAL

Part A, 900 mg GBT440: Capsules which contain GBT440 drug substance in Swedish orange

Drug: GBT440

GBT440 1500 mg Dose

EXPERIMENTAL

Part B , 1500 mg GBT440: Capsules which contain GBT440 drug substance in Swedish orange

Drug: GBT440

Interventions

GBT440DRUG

GBT440: Capsules which contain GBT440 drug substance in Swedish orange

GBT440 1500 mg DoseGBT440 900 mg Dose

Eligibility Criteria

Age45 Years - 85 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (9)

Unknown Facility

Birmingham, Alabama, 35233, United States

Location

Unknown Facility

Denver, Colorado, 80206, United States

Location

Unknown Facility

New Orleans, Louisiana, 70122, United States

Location

Unknown Facility

Philadelphia, Pennsylvania, 19140, United States

Location

Unknown Facility

Nashville, Tennessee, 37232, United States

Location

Unknown Facility

Salt Lake City, Utah, 84108, United States

Location

Unknown Facility

London, SW3 6NP, United Kingdom

Location

Unknown Facility

Manchester, M23 9QZ, United Kingdom

Location

Unknown Facility

Southampton, SO16 6YD, United Kingdom

Location

MeSH Terms

Conditions

Idiopathic Pulmonary FibrosisHypoxia

Interventions

voxelotor

Condition Hierarchy (Ancestors)

Pulmonary FibrosisLung Diseases, InterstitialLung DiseasesRespiratory Tract DiseasesSigns and Symptoms, RespiratorySigns and SymptomsPathological Conditions, Signs and Symptoms

Limitations and Caveats

Since this study was stopped prematurely, no efficacy results will be presented.

Results Point of Contact

Title
Nick Vlahakis
Organization
Global Blood Therapeutics
nvlahakis@gbt.com
16508228756

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
SUPPORTIVE CARE
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 28, 2016

First Posted

December 12, 2016

Study Start

November 1, 2016

Primary Completion

October 23, 2017

Study Completion

October 23, 2017

Last Updated

August 21, 2020

Results First Posted

August 21, 2020

Record last verified: 2020-07

Locations

US(6)GB(3)