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The study was terminated due to lack of clinically meaningful benefit
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This is an open label study in which eligible IPF subjects who are using supplemental oxygen at rest will receive GBT440 orally daily.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| GBT440 900 mg Dose | Experimental | Part A, 900 mg GBT440: Capsules which contain GBT440 drug substance in Swedish orange |
|
| GBT440 1500 mg Dose | Experimental | Part B , 1500 mg GBT440: Capsules which contain GBT440 drug substance in Swedish orange |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| GBT440 | Drug | GBT440: Capsules which contain GBT440 drug substance in Swedish orange |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in Oxygen Saturation at End of Treatment Period Compared to Baseline | Days 1 to 90 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Birmingham | Alabama | 35233 | United States | |||
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| ID | Title | Description |
|---|---|---|
| FG000 | GBT440 900 mg Dose | Part A , 900 mg daily dose GBT440: Capsules which contain GBT440 drug substance in Swedish orange |
| FG001 | GBT440 1500 mg Dose | Part B , 1500 mg daily dose GBT440: Capsules which contain GBT440 drug substance in Swedish orange |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 5, 2017 | Mar 25, 2020 |
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| 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) | Spirometry measurements will include forced vital capacity (FVC) and diffusing capacity of the lung for carbon monoxide (DLco). | Days 1 to 90 |
| Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0 | Days 1 to 90 |
| Pharmacokinetic Parameters of GBT440 in Plasma and Whole Blood (Minimum Concentration (Cmin)) | 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. | 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) |
| Denver |
| Colorado |
| 80206 |
| United States |
| New Orleans | Louisiana | 70122 | United States |
| Philadelphia | Pennsylvania | 19140 | United States |
| Nashville | Tennessee | 37232 | United States |
| Salt Lake City | Utah | 84108 | United States |
| London | SW3 6NP | United Kingdom |
| Manchester | M23 9QZ | United Kingdom |
| Southampton | SO16 6YD | United Kingdom |
| COMPLETED |
|
| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | GBT440 900 mg Dose | Part A, 900 mg daily dose GBT440: Capsules which contain GBT440 drug substance in Swedish orange |
| BG001 | GBT440 1500 mg Dose | Part B, 1500 mg daily dose GBT440: Capsules which contain GBT440 drug substance in Swedish orange |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Age, Continuous | Median | Full Range | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change in Oxygen Saturation at End of Treatment Period Compared to Baseline | Since study was stopped prematurely, complete data for all participants were not analyzable so no efficacy results are presented. | Posted | Days 1 to 90 |
|
| |||||||||||||||||||||||
| Secondary | Change in Supplemental Oxygen Requirement at End of Treatment Period Compared to Baseline | Since study was stopped prematurely, complete data for all participants were not analyzable so no efficacy results are presented. | Posted | Days 1 to 90 |
|
| |||||||||||||||||||||||
| Secondary | 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 | Since study was stopped prematurely, complete data for all participants were not analyzable so no efficacy results are presented. | Posted | Days 1 to 90 |
|
| |||||||||||||||||||||||
| Secondary | Evaluate the Effect of GBT440 on Performance of the 6MWT | Since study was stopped prematurely, complete data for all participants were not analyzable so no efficacy results are presented. | Posted | Days 1 to 90 |
|
| |||||||||||||||||||||||
| Secondary | Evaluate the Effect of GBT440 on IPF Related Symptoms Using Patient Related Outcomes | Since study was stopped prematurely, complete data for all participants were not analyzable so no efficacy results are presented. | Posted | Days 1 to 90 |
|
| |||||||||||||||||||||||
| Secondary | Evaluate Pulmonary Function Using Pulmonary Function Tests (FVC and DLco) | Spirometry measurements will include forced vital capacity (FVC) and diffusing capacity of the lung for carbon monoxide (DLco). | Since study was stopped prematurely, complete data for all participants were not analyzable so no efficacy results are presented. | Posted | Days 1 to 90 |
|
| ||||||||||||||||||||||
| Secondary | Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0 | Participant had at least one dose of study drug | Posted | Count of Participants | Participants | Days 1 to 90 |
|
| |||||||||||||||||||||
| Secondary | Pharmacokinetic Parameters of GBT440 in Plasma and Whole Blood (Minimum Concentration (Cmin)) | 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. | Complete PK data were not analyzable for any participant. | Posted | 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) |
|
|
Screening to 90 days
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | GBT440 900 mg Dose | Part A , 900 mg daily dose GBT440: Capsules which contain GBT440 drug substance in Swedish orange | 0 | 11 | 3 | 11 | 11 | 11 |
| EG001 | GBT440 1500 mg Dose | Part B , 1500 mg daily dose GBT440: Capsules which contain GBT440 drug substance in Swedish orange | 0 | 3 | 2 | 3 | 3 | 3 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Idiopathic pulmonary fibrosis | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Pericardial effusion | Cardiac disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Cardiac failure congestive | Cardiac disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA 20.0 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Atrial flutter | Cardiac disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Oedema Peripheral | General disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Hepatic mass | Hepatobiliary disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 20.0 | Non-systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA 20.0 | Non-systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 20.0 | Non-systematic Assessment |
| |
| Viral sinusitis | Infections and infestations | MedDRA 20.0 | Non-systematic Assessment |
| |
| Tooth fracture | Injury, poisoning and procedural complications | MedDRA 20.0 | Non-systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Adenocarcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 20.0 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Erectile dysfunction | Reproductive system and breast disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Rash generalised | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Non-systematic Assessment |
|
Since this study was stopped prematurely, no efficacy results will be presented.
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Nick Vlahakis | Global Blood Therapeutics | 16508228756 | nvlahakis@gbt.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 15, 2017 | Mar 25, 2020 | SAP_001.pdf |
| ID | Term |
|---|---|
| D054990 | Idiopathic Pulmonary Fibrosis |
| D000860 | Hypoxia |
| ID | Term |
|---|---|
| D011658 | Pulmonary Fibrosis |
| D017563 | Lung Diseases, Interstitial |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D012818 | Signs and Symptoms, Respiratory |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| C000628792 | voxelotor |
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| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|