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This is an open label, single arm study which enrolled 5 subjects with SCD who previously participated in the GBT440-001 study (NCT02285088).
This is an open label, single arm study which enrolled 5 subjects with SCD who previously participated in the GBT440-001 study (NCT02285088).
Dosing of study drug was 2 to 6 months, depending on subject's dose assignment in the last administration of study drug in GBT440-001 (NCT02285088).
The primary objective of the study was to evaluate the safety and tolerability of up to a total of 6 months dosing of subjects with SCD who participated in the GBT440-001 study (NCT02285088).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Single Arm | Experimental | Single Arm / open label |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| GBT440 | Drug | Oral drug |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events During Dosing of GBT440 for up to 6 Months. | The safety evaluation will include physical examinations, blood pressure, clinical laboratory tests (hematology, serum biochemistry) and adverse events. | 2 - 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| To Assess the Efficacy of GBT440 as Measured by Improvements in Anemia | Data presented are hemoglobin value collected at specific time points. | 2 - 6 months |
| To Observed Pharmacokinetics in Plasma and Whole Blood. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Josh Lehrer-Graiwer, MD | Global Blood Therapeutics, Inc. | Study Director |
| Timothy Mant, FRCP, FFPM | Quintiles, Inc. | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The BRC Research Facility, Floor 15 The Tower Wing | London | SE1 9RT | United Kingdom |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 30655275 | Derived | Howard J, Hemmaway CJ, Telfer P, Layton DM, Porter J, Awogbade M, Mant T, Gretler DD, Dufu K, Hutchaleelaha A, Patel M, Siu V, Dixon S, Landsman N, Tonda M, Lehrer-Graiwer J. A phase 1/2 ascending dose study and open-label extension study of voxelotor in patients with sickle cell disease. Blood. 2019 Apr 25;133(17):1865-1875. doi: 10.1182/blood-2018-08-868893. Epub 2019 Jan 17. |
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There was no screening period as subjects transitioned directly from the GBT440-001 study (NCT02285088).into this study.
Subjects enrolled in this study participated in GBT440-001 study (NCT02285088).
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| ID | Title | Description |
|---|---|---|
| FG000 | GBT440 - 2 Months | These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (n=3) (NCT02285088). |
| FG001 | GBT440 - 6 Months |
| 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_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Sep 28, 2016 |
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Measure maximum plasma concentration (Cmax)
| 2 - 6 months |
| To Characterize the Effect of GBT440 on Hemolysis. | Data presented for unconjugated bilirubin at specific time point. | 2 - 6 months |
The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (n=1) (NCT02285088).
| FG002 | GBT440 - 4 Months | This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (n=1) (NCT02285088). |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | GBT440 - 2 Months | These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (NCT02285088) (n=3). |
| BG001 | GBT440 - 6 Months | The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (NCT02285088) (n=1). |
| BG002 | GBT440 - 4 Months | This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (NCT02285088) (n=1). |
| BG003 | 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 | Full Range | Years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
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| 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 | Number of Participants With Treatment-Emergent Adverse Events During Dosing of GBT440 for up to 6 Months. | The safety evaluation will include physical examinations, blood pressure, clinical laboratory tests (hematology, serum biochemistry) and adverse events. | Safety | Posted | Count of Participants | Participants | 2 - 6 months |
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| Secondary | To Assess the Efficacy of GBT440 as Measured by Improvements in Anemia | Data presented are hemoglobin value collected at specific time points. | Posted | Number | g/dL | 2 - 6 months |
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| Secondary | To Observed Pharmacokinetics in Plasma and Whole Blood. | Measure maximum plasma concentration (Cmax) | Posted | Number | ug/mL | 2 - 6 months |
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| ||||||||||||||||||||||||||||||||||
| Secondary | To Characterize the Effect of GBT440 on Hemolysis. | Data presented for unconjugated bilirubin at specific time point. | Posted | Number | umol/L | 2 - 6 months |
|
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10 months
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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 - 2 Months | These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (NCT02285088) (n=3). | 0 | 3 | 0 | 3 | 2 | 3 |
| EG001 | GBT440 - 6 Months | The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (NCT02285088) (n=1). | 0 | 1 | 1 | 1 | 1 | 1 |
| EG002 | GBT440 - 4 Months | This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (NCT02285088) (n=1). | 0 | 1 | 1 | 1 | 1 | 1 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Sickle Cell Anemia with Crisis | Blood and lymphatic system disorders | MedDRA (17.1) | Non-systematic Assessment |
| |
| Cholecystectomy | Surgical and medical procedures | MedDRA (17.1) | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhea | Gastrointestinal disorders | MedDRA (17.1) | Non-systematic Assessment |
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| Toothache | Gastrointestinal disorders | MedDRA (17.1) | Systematic Assessment |
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| Upper Respiratory Tract Infection | Infections and infestations | MedDRA (17.1) | Systematic Assessment |
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| Abdominal Pain | Gastrointestinal disorders | MedDRA (17.1) | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA (17.1) | Systematic Assessment |
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| Gingival Swelling | Gastrointestinal disorders | MedDRA (17.1) | Systematic Assessment |
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The sponsor and the PI will have to agree on any publications regarding the study.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Margaret Tonda, Sr. Director, Clinical Science | Global Blood Therapeutics | 650-741-7761 | mtonda@gbt.com |
| Nov 6, 2018 |
| Prot_SAP_000.pdf |
| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C000628792 | voxelotor |
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| Male |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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