Trial Outcomes & Findings for Study to Evaluate the Effect of GBT440 Administered to Subjects With IPF on Supplemental Oxygen at Rest (NCT NCT02989168)
NCT ID: NCT02989168
Last Updated: 2020-08-21
Results Overview
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
14 participants
Primary outcome timeframe
Days 1 to 90
Results posted on
2020-08-21
Participant Flow
Participant milestones
| Measure |
GBT440 900 mg Dose
Part A , 900 mg daily dose
GBT440: Capsules which contain GBT440 drug substance in Swedish orange
|
GBT440 1500 mg Dose
Part B , 1500 mg daily dose
GBT440: Capsules which contain GBT440 drug substance in Swedish orange
|
|---|---|---|
|
Overall Study
STARTED
|
11
|
3
|
|
Overall Study
COMPLETED
|
7
|
0
|
|
Overall Study
NOT COMPLETED
|
4
|
3
|
Reasons for withdrawal
| Measure |
GBT440 900 mg Dose
Part A , 900 mg daily dose
GBT440: Capsules which contain GBT440 drug substance in Swedish orange
|
GBT440 1500 mg Dose
Part B , 1500 mg daily dose
GBT440: Capsules which contain GBT440 drug substance in Swedish orange
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
2
|
0
|
|
Overall Study
Physician Decision
|
0
|
1
|
|
Overall Study
Adverse Event
|
1
|
2
|
|
Overall Study
Sponsor stopped study.
|
1
|
0
|
Baseline Characteristics
Study to Evaluate the Effect of GBT440 Administered to Subjects With IPF on Supplemental Oxygen at Rest
Baseline characteristics by cohort
| Measure |
GBT440 900 mg Dose
n=11 Participants
Part A, 900 mg daily dose
GBT440: Capsules which contain GBT440 drug substance in Swedish orange
|
GBT440 1500 mg Dose
n=3 Participants
Part B, 1500 mg daily dose
GBT440: Capsules which contain GBT440 drug substance in Swedish orange
|
Total
n=14 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Race (NIH/OMB)
White
|
10 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Age, Continuous
|
69.0 years
n=5 Participants
|
72.0 years
n=7 Participants
|
70.0 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
11 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Days 1 to 90Population: Since study was stopped prematurely, complete data for all participants were not analyzable so no efficacy results are presented.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Days 1 to 90Population: Since study was stopped prematurely, complete data for all participants were not analyzable so no efficacy results are presented.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Days 1 to 90Population: Since study was stopped prematurely, complete data for all participants were not analyzable so no efficacy results are presented.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Days 1 to 90Population: Since study was stopped prematurely, complete data for all participants were not analyzable so no efficacy results are presented.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Days 1 to 90Population: Since study was stopped prematurely, complete data for all participants were not analyzable so no efficacy results are presented.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Days 1 to 90Population: Since study was stopped prematurely, complete data for all participants were not analyzable so no efficacy results are presented.
Spirometry measurements will include forced vital capacity (FVC) and diffusing capacity of the lung for carbon monoxide (DLco).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Days 1 to 90Population: Participant had at least one dose of study drug
Outcome measures
| Measure |
GBT440 900 mg Dose
n=11 Participants
Part A
GBT440: Capsules which contain GBT440 drug substance in Swedish orange
|
GBT440 1500 mg Dose
n=3 Participants
Part B
GBT440: Capsules which contain GBT440 drug substance in Swedish orange
|
|---|---|---|
|
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0
|
11 Participants
|
3 Participants
|
SECONDARY outcome
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)Population: Complete PK data were not analyzable for any participant.
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.
Outcome measures
Outcome data not reported
Adverse Events
GBT440 900 mg Dose
Serious events: 3 serious events
Other events: 11 other events
Deaths: 0 deaths
GBT440 1500 mg Dose
Serious events: 2 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
GBT440 900 mg Dose
n=11 participants at risk
Part A , 900 mg daily dose
GBT440: Capsules which contain GBT440 drug substance in Swedish orange
|
GBT440 1500 mg Dose
n=3 participants at risk
Part B , 1500 mg daily dose
GBT440: Capsules which contain GBT440 drug substance in Swedish orange
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Idiopathic pulmonary fibrosis
|
18.2%
2/11 • Screening to 90 days
|
0.00%
0/3 • Screening to 90 days
|
|
Cardiac disorders
Pericardial effusion
|
9.1%
1/11 • Screening to 90 days
|
0.00%
0/3 • Screening to 90 days
|
|
Cardiac disorders
Cardiac failure congestive
|
9.1%
1/11 • Screening to 90 days
|
0.00%
0/3 • Screening to 90 days
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
0.00%
0/11 • Screening to 90 days
|
33.3%
1/3 • Screening to 90 days
|
|
General disorders
Non-cardiac chest pain
|
0.00%
0/11 • Screening to 90 days
|
33.3%
1/3 • Screening to 90 days
|
Other adverse events
| Measure |
GBT440 900 mg Dose
n=11 participants at risk
Part A , 900 mg daily dose
GBT440: Capsules which contain GBT440 drug substance in Swedish orange
|
GBT440 1500 mg Dose
n=3 participants at risk
Part B , 1500 mg daily dose
GBT440: Capsules which contain GBT440 drug substance in Swedish orange
|
|---|---|---|
|
Cardiac disorders
Atrial flutter
|
9.1%
1/11 • Screening to 90 days
|
0.00%
0/3 • Screening to 90 days
|
|
Gastrointestinal disorders
Diarrhoea
|
36.4%
4/11 • Screening to 90 days
|
33.3%
1/3 • Screening to 90 days
|
|
Gastrointestinal disorders
Nausea
|
9.1%
1/11 • Screening to 90 days
|
66.7%
2/3 • Screening to 90 days
|
|
General disorders
Fatigue
|
27.3%
3/11 • Screening to 90 days
|
33.3%
1/3 • Screening to 90 days
|
|
General disorders
Non-cardiac chest pain
|
9.1%
1/11 • Screening to 90 days
|
0.00%
0/3 • Screening to 90 days
|
|
General disorders
Asthenia
|
9.1%
1/11 • Screening to 90 days
|
0.00%
0/3 • Screening to 90 days
|
|
General disorders
Oedema Peripheral
|
9.1%
1/11 • Screening to 90 days
|
0.00%
0/3 • Screening to 90 days
|
|
Hepatobiliary disorders
Hepatic mass
|
0.00%
0/11 • Screening to 90 days
|
33.3%
1/3 • Screening to 90 days
|
|
Infections and infestations
Bronchitis
|
9.1%
1/11 • Screening to 90 days
|
0.00%
0/3 • Screening to 90 days
|
|
Infections and infestations
Oral herpes
|
9.1%
1/11 • Screening to 90 days
|
0.00%
0/3 • Screening to 90 days
|
|
Infections and infestations
Sinusitis
|
9.1%
1/11 • Screening to 90 days
|
0.00%
0/3 • Screening to 90 days
|
|
Infections and infestations
Viral sinusitis
|
9.1%
1/11 • Screening to 90 days
|
0.00%
0/3 • Screening to 90 days
|
|
Injury, poisoning and procedural complications
Tooth fracture
|
9.1%
1/11 • Screening to 90 days
|
0.00%
0/3 • Screening to 90 days
|
|
Metabolism and nutrition disorders
Decreased appetite
|
9.1%
1/11 • Screening to 90 days
|
0.00%
0/3 • Screening to 90 days
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
9.1%
1/11 • Screening to 90 days
|
0.00%
0/3 • Screening to 90 days
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
9.1%
1/11 • Screening to 90 days
|
0.00%
0/3 • Screening to 90 days
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Adenocarcinoma
|
0.00%
0/11 • Screening to 90 days
|
33.3%
1/3 • Screening to 90 days
|
|
Nervous system disorders
Headache
|
18.2%
2/11 • Screening to 90 days
|
0.00%
0/3 • Screening to 90 days
|
|
Nervous system disorders
Dizziness
|
0.00%
0/11 • Screening to 90 days
|
33.3%
1/3 • Screening to 90 days
|
|
Psychiatric disorders
Insomnia
|
9.1%
1/11 • Screening to 90 days
|
0.00%
0/3 • Screening to 90 days
|
|
Reproductive system and breast disorders
Erectile dysfunction
|
9.1%
1/11 • Screening to 90 days
|
0.00%
0/3 • Screening to 90 days
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
18.2%
2/11 • Screening to 90 days
|
0.00%
0/3 • Screening to 90 days
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
9.1%
1/11 • Screening to 90 days
|
0.00%
0/3 • Screening to 90 days
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
0.00%
0/11 • Screening to 90 days
|
33.3%
1/3 • Screening to 90 days
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
9.1%
1/11 • Screening to 90 days
|
0.00%
0/3 • Screening to 90 days
|
|
Skin and subcutaneous tissue disorders
Rash
|
18.2%
2/11 • Screening to 90 days
|
0.00%
0/3 • Screening to 90 days
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
9.1%
1/11 • Screening to 90 days
|
0.00%
0/3 • Screening to 90 days
|
|
Skin and subcutaneous tissue disorders
Rash generalised
|
9.1%
1/11 • Screening to 90 days
|
0.00%
0/3 • Screening to 90 days
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place