Trial · NCT03041909

An Extension Study to Further Evaluate the Safety, Tolerability of GBT440 in Patients With Sickle Cell Disease Who Participated in the Study GBT440-001

NCT03041909, Phase 2, Completed, Global Blood Therapeutics; results first posted 2019-01-02

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. An Extension Study to Further Evaluate the Safety, Tolerability of GBT440 in Patients With Sickle Cell Disease Who Participated in the Study GBT440-001. Trials, record NCT03041909. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03041909

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: An Extension Study to Further Evaluate the Safety, Tolerability of GBT440 in Patients With Sickle Cell Disease Who Participated in the Study GBT440-001
NCT number: NCT03041909
Status: Completed
Phases: Phase 2
Enrollment: 5 participants, actual
Lead sponsor: Global Blood Therapeutics
Conditions: Sickle Cell Disease
Interventions: GBT440
First posted: 2017-02-03
Last updated: 2019-01-02
Results first posted: 2019-01-02

Source ClinicalTrials.gov

Registered outcomes4

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Number of Participants With Treatment-Emergent Adverse Events During Dosing of GBT440 for up to 6 Months.	2 - 6 months	The safety evaluation will include physical examinations, blood pressure, clinical laboratory tests (hematology, serum biochemistry) and adverse events.
Secondary	To Assess the Efficacy of GBT440 as Measured by Improvements in Anemia	2 - 6 months	Data presented are hemoglobin value collected at specific time points.
Secondary	To Observed Pharmacokinetics in Plasma and Whole Blood.	2 - 6 months	Measure maximum plasma concentration (Cmax)
Secondary	To Characterize the Effect of GBT440 on Hemolysis.	2 - 6 months	Data presented for unconjugated bilirubin at specific time point.

Source ClinicalTrials.gov

Outcomes4

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Number of Participants With Treatment-Emergent Adverse Events During Dosing of GBT440 for up to 6 Months.	2 - 6 months	Safety	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	To Assess the Efficacy of GBT440 as Measured by Improvements in Anemia	2 - 6 months		g/dL	NUMBER
SECONDARY	To Observed Pharmacokinetics in Plasma and Whole Blood.	2 - 6 months		ug/mL	NUMBER
SECONDARY	To Characterize the Effect of GBT440 on Hemolysis.	2 - 6 months		umol/L	NUMBER

Source ClinicalTrials.gov

Results by group12

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Units
Number of Participants With Treatment-Emergent Adverse Events During Dosing of GBT440 for up to 6 Months.	GBT440 - 2 Months	2	Participants
Number of Participants With Treatment-Emergent Adverse Events During Dosing of GBT440 for up to 6 Months.	GBT440 - 6 Months	1	Participants
Number of Participants With Treatment-Emergent Adverse Events During Dosing of GBT440 for up to 6 Months.	GBT440 - 4 Months	1	Participants
To Assess the Efficacy of GBT440 as Measured by Improvements in Anemia	GBT440 - 2 Months	NA	g/dL
To Assess the Efficacy of GBT440 as Measured by Improvements in Anemia	GBT440 - 6 Months	NA	g/dL
To Assess the Efficacy of GBT440 as Measured by Improvements in Anemia	GBT440 - 4 Months	NA	g/dL
To Observed Pharmacokinetics in Plasma and Whole Blood.	GBT440 - 2 Months	NA	ug/mL
To Observed Pharmacokinetics in Plasma and Whole Blood.	GBT440 - 6 Months	NA	ug/mL
To Observed Pharmacokinetics in Plasma and Whole Blood.	GBT440 - 4 Months	NA	ug/mL
To Characterize the Effect of GBT440 on Hemolysis.	GBT440 - 2 Months	NA	umol/L

1 to 10 of 12Source ClinicalTrials.gov

See all 12 results by group

Participant flow9

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	GBT440 - 2 Months	3
Overall Study	STARTED	GBT440 - 6 Months	1
Overall Study	STARTED	GBT440 - 4 Months	1
Overall Study	COMPLETED	GBT440 - 2 Months	3
Overall Study	COMPLETED	GBT440 - 6 Months	1
Overall Study	COMPLETED	GBT440 - 4 Months	1
Overall Study	NOT COMPLETED	GBT440 - 2 Months	0
Overall Study	NOT COMPLETED	GBT440 - 6 Months	0
Overall Study	NOT COMPLETED	GBT440 - 4 Months	0

Source ClinicalTrials.gov

Adverse events24

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Diarrhea	Gastrointestinal disorders	GBT440 - 2 Months	1	3	1
other	Diarrhea	Gastrointestinal disorders	GBT440 - 6 Months	0	1	0
other	Diarrhea	Gastrointestinal disorders	GBT440 - 4 Months	1	1	1
other	Toothache	Gastrointestinal disorders	GBT440 - 2 Months	0	3	0
other	Toothache	Gastrointestinal disorders	GBT440 - 6 Months	1	1	1
other	Toothache	Gastrointestinal disorders	GBT440 - 4 Months	0	1	0
other	Upper Respiratory Tract Infection	Infections and infestations	GBT440 - 2 Months	1	3	1
other	Upper Respiratory Tract Infection	Infections and infestations	GBT440 - 6 Months	0	1	0
other	Upper Respiratory Tract Infection	Infections and infestations	GBT440 - 4 Months	1	1	1
other	Abdominal Pain	Gastrointestinal disorders	GBT440 - 2 Months	1	3	1

1 to 10 of 24Source ClinicalTrials.gov

See all 24 adverse events

Adverse event totals9

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	GBT440 - 2 Months	0	3
Total, all-cause mortality	GBT440 - 6 Months	0	1
Total, all-cause mortality	GBT440 - 4 Months	0	1
Total, other adverse events	GBT440 - 2 Months	2	3
Total, other adverse events	GBT440 - 6 Months	1	1
Total, other adverse events	GBT440 - 4 Months	1	1
Total, serious adverse events	GBT440 - 2 Months	0	3
Total, serious adverse events	GBT440 - 6 Months	1	1
Total, serious adverse events	GBT440 - 4 Months	1	1

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
30655275	DERIVED

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT03041909

GET https://conductscience.org/api/trials/v1/NCT03041909/events?page=2

API reference For AI assistants