Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE) (GBT_HOPE)
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ClinicalTrials.gov Identifier: NCT03036813 |
Recruitment Status :
Completed
First Posted : January 30, 2017
Results First Posted : January 7, 2021
Last Update Posted : January 7, 2021
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Sponsor:
Global Blood Therapeutics
Information provided by (Responsible Party):
Global Blood Therapeutics
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Study Type | Interventional |
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Study Design | Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Triple (Participant, Care Provider, Investigator); Primary Purpose: Treatment |
Condition |
Sickle Cell Disease |
Interventions |
Drug: voxelotor Other: Placebo |
Enrollment | 449 |
Participant Flow
Recruitment Details | Overall, 449 subjects were screened for this study at 60 study sites. Between January 2017 and May 2018, a total of 274 subjects were randomized at 58 study sites across 12 countries. All 274 randomized subjects were included in the ITT Population. A total of 271 subjects received study drug and were included in the Safety Population. |
Pre-assignment Details | Screening procedures were done within 35 days of randomization to assess eligibility. |
Arm/Group Title | Voxelotor 900mg | Voxelotor 1500mg | Placebo |
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Participants received voxelotor 900 mg; administered orally, once daily for 72 weeks. | Participants received voxelotor 1500 mg administered orally, once daily for 72 weeks. | Matching placebo; administered orally, once daily for 72 weeks. |
Period Title: Overall Study | |||
Started | 92 | 90 | 92 |
Completed | 70 | 63 | 66 |
Not Completed | 22 | 27 | 26 |
Reason Not Completed | |||
Adverse Event | 6 | 11 | 6 |
Withdrawal by Subject | 12 | 6 | 10 |
Lost to Follow-up | 1 | 1 | 0 |
Physician Decision | 2 | 1 | 1 |
Non-Compliance | 1 | 5 | 3 |
Pregnancy | 0 | 0 | 1 |
Other | 0 | 3 | 5 |
Baseline Characteristics
Arm/Group Title | Voxelotor 900mg | Voxelotor 1500mg | Placebo | Total | |
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Participants received voxelotor 900 mg; administered orally, once daily for 72 weeks. | Participants received voxelotor 1500 mg; administered orally, once daily for 72 weeks | Matching Placebo; administered orally, once daily for 72 weeks | Total of all reporting groups | |
Overall Number of Baseline Participants | 92 | 90 | 92 | 274 | |
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[Not Specified]
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Age, Customized
Measure Type: Count of Participants Unit of measure: Participants |
Number Analyzed | 92 participants | 90 participants | 92 participants | 274 participants |
12 to < 18 |
15 16.3%
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14 15.6%
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17 18.5%
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46 16.8%
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>=18 to < 65 |
77 83.7%
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76 84.4%
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75 81.5%
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228 83.2%
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Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 92 participants | 90 participants | 92 participants | 274 participants | |
Female |
51 55.4%
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58 64.4%
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50 54.3%
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159 58.0%
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Male |
41 44.6%
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32 35.6%
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42 45.7%
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115 42.0%
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Ethnicity (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 92 participants | 90 participants | 92 participants | 274 participants | |
Hispanic or Latino |
4 4.3%
|
3 3.3%
|
3 3.3%
|
10 3.6%
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Not Hispanic or Latino |
86 93.5%
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82 91.1%
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85 92.4%
|
253 92.3%
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Unknown or Not Reported |
2 2.2%
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5 5.6%
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4 4.3%
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11 4.0%
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Race/Ethnicity, Customized
Measure Type: Count of Participants Unit of measure: Participants |
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Race | Number Analyzed | 92 participants | 90 participants | 92 participants | 274 participants |
Arab/Middle Eastern |
19 20.7%
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15 16.7%
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18 19.6%
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52 19.0%
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Black or African American |
60 65.2%
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59 65.6%
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63 68.5%
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182 66.4%
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White |
6 6.5%
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7 7.8%
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3 3.3%
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16 5.8%
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Asian |
0 0.0%
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1 1.1%
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0 0.0%
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1 0.4%
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Other |
4 4.3%
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2 2.2%
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5 5.4%
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11 4.0%
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Multiple |
2 2.2%
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5 5.6%
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3 3.3%
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10 3.6%
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Missing/ Not Specified |
1 1.1%
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1 1.1%
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0 0.0%
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2 0.7%
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Region of Enrollment
Measure Type: Number Unit of measure: Participants |
Number Analyzed | 92 participants | 90 participants | 92 participants | 274 participants |
Europe | 19 | 19 | 18 | 56 | |
North America | 36 | 34 | 35 | 105 | |
Egypt | 15 | 16 | 13 | 44 | |
Kenya | 17 | 14 | 18 | 49 | |
Jamaica | 0 | 2 | 2 | 4 | |
Oman | 2 | 2 | 5 | 9 | |
Lebanon | 3 | 3 | 1 | 7 |
Outcome Measures
Adverse Events
Limitations and Caveats
[Not Specified]
More Information
Results Point of Contact
Name/Title: | Margaret Tonda, PharmD, Sr. Director, Clinical Science |
Organization: | Global Blood Therapeutics |
Phone: | (650) 741-7761 |
EMail: | mtonda@gbt.com |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | Global Blood Therapeutics |
ClinicalTrials.gov Identifier: | NCT03036813 |
Other Study ID Numbers: |
GBT440-031 |
First Submitted: | January 27, 2017 |
First Posted: | January 30, 2017 |
Results First Submitted: | October 28, 2020 |
Results First Posted: | January 7, 2021 |
Last Update Posted: | January 7, 2021 |