Trial · NCT03573882
Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031
NCT03573882, Phase 3, Terminated, Pfizer; results first posted 2025-11-18
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031. Trials, record NCT03573882. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03573882
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031
- NCT number
- NCT03573882
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 179 participants, actual
- Lead sponsor
- Pfizer
- Conditions
- Sickle Cell Disease
- Interventions
- Voxelotor
- First posted
- 2018-06-29
- Last updated
- 2025-11-18
- Results first posted
- 2025-11-18
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Sickle Cell Disease (SCD)-Related Treatment Emergent Adverse Events (TEAEs) | From date of informed consent up to 28 days after last dose of study drug (maximum up to 300.7 weeks) | An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. TEAEs were defined as AEs with onset on or after the date of informed consent until 28 days after last dose of study drug. SCD-related TEAEs included preferred terms (PTs) of sickle cell anaemia with crisis, acute chest syndrome (ACS), pneumonia, priapism, and osteonecrosis. The number of participants with any SCD-related TEAEs was reported in this outcome measure. |
| Primary | Number of Participants With Non- SCD-Related TEAEs | From date of informed consent up to 28 days after last dose of study drug (maximum up to 300.7 weeks) | An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. TEAEs were defined as AEs with onset on or after the date of informed consent until 28 days after last dose of study drug. Non-SCD-related TEAEs included all the PTs of TEAEs other than SCD- related TEAEs. The number of participants with any non-SCD-related TEAEs was reported in this outcome measure. |
| Primary | Number of Participants With SCD-Related Treatment Emergent Serious Adverse Events (TESAEs) | From date of informed consent up to 28 days after last dose of study drug (maximum up to 300.7 weeks) | An SAE is an AE at any dose, in view of investigator resulted in any of following outcomes: death; life-threatening AE; inpatient hospitalization/prolongation of existing hospitalization; persistent/significant incapacity or disability; a congenital anomaly/birth defect and important medical events (IME) that may not result in death, be immediately life threatening; or require hospitalization may be considered serious when based upon medical judgement, they may jeopardize study participant and may require medical or surgical intervention to prevent one of outcomes listed in definition. TESAEs were defined as SAEs with onset on or after the date of informed consent until 28 days after last dose of study drug. Number of participants with SCD-Related TESAEs was reported in this outcome measure. |
| Primary | Number of Participants With Non-SCD-Related TESAEs | From date of informed consent up to 28 days after last dose of study drug (maximum up to 300.7 weeks) | An SAE is an AE at any dose, in view of investigator resulted in any of following outcomes: death; life-threatening AE; inpatient hospitalization/prolongation of existing hospitalization; persistent/significant incapacity or disability; a congenital anomaly/birth defect and IME that may not result in death, be immediately life threatening; or require hospitalization may be considered serious when based upon medical judgement, they may jeopardize study participant and may require medical or surgical intervention to prevent one of outcomes listed in definition. TESAEs were defined as SAEs with onset on or after the date of informed consent until 28 days after last dose of study drug. Number of participants with non-SCD related TESAEs was reported in this outcome measure. |
| Primary | Annualized Incidence Rate (Events Per Person Years) of SCD-Related Complications | From date of informed consent up to earlier of 28 days after last dose of study drug or end of study date (maximum up to 300.7 weeks) | Annualized incidence rate was defined as total number of events (i.e. complications observed for all participants) divided by total person years. Total person-years= sum of participant summary period in years where summary period=date of informed consent until the earlier of 28 days after last dose of study drug or end of study date. SCD related complications included acute chest syndrome, cerebrovascular accident, hepatic sequestration, ocular icterus, osteonecrosis, pneumonia, priapism, pulmonary hypertension, retinopathy, sickle cell anaemia with crisis, skin ulcer and splenic sequestration. The 95% CI was based on exact Poisson confidence limits. Incidence rate of all SCD related complications is reported in this outcome measure. |
| Primary | Annualized Incidence Rate (VOC Events Per Person Years) of On-Treatment Vaso-occlusive Crisis (VOCs) | From date of informed consent to last dose of study drug (maximum up to 296.7 weeks) | Annualized incidence rate was defined as total number of VOC events divided by total person years. Total person-years= sum of participant summary period in years where summary period=date of informed consent to last dose of study drug. VOC during the treatment period was defined as composite of acute painful crisis or acute chest syndrome (ACS) and included the following: moderate to severe pain lasting at least 2 hours; no explanation other than VOC; required oral or parenteral opioids, ketorolac, or other analgesics prescribed or directed by a healthcare professional. The 95% CI was based on exact Poisson confidence limits. |
| Secondary | Change From Baseline in Hemoglobin Level at Week 48 | Baseline, Week 48 | Change from baseline in hemoglobin at Week 48 was reported in this outcome measure. Baseline value was defined as the last available value (including Week 72, end of treatment \[EOT\], or end of study \[EOS\] visits in the parent study GBT440-031 \[NCT03036813\]) collected on or prior to first dose in GBT440-034. |
| Secondary | Percent Change From Baseline in Reticulocytes Percentage at Week 48 | Baseline, Week 48 | Percent change from baseline in reticulocytes percentage at Week 48 was reported in this outcome measure. Baseline value was defined as the last available value (including Week 72, EOT, or EOS visits in the parent study GBT440-031 \[NCT03036813\]) collected on or prior to first dose in GBT440-034. |
| Secondary | Percent Change From Baseline in Absolute Reticulocytes at Week 48 | Baseline, Week 48 | Percent change from baseline in absolute reticulocytes at Week 48 was reported in this outcome measure. Baseline value was defined as the last available value (including Week 72, EOT, or EOS visits in the parent study GBT440-031 \[NCT03036813\]) collected on or prior to first dose in GBT440-034. |
| Secondary | Percent Change From Baseline in Indirect Bilirubin at Week 48 | Baseline, Week 48 | Percent change from baseline in indirect bilirubin at Week 48 was reported in this outcome measure. Baseline value was defined as the last available value (including Week 72, EOT, or EOS visits in the parent study GBT440-031 \[NCT03036813\]) collected on or prior to first dose in GBT440-034. |
Source ClinicalTrials.gov
Outcomes10
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 Sickle Cell Disease (SCD)-Related Treatment Emergent Adverse Events (TEAEs) | From date of informed consent up to 28 days after last dose of study drug (maximum up to 300.7 weeks) | The safety population included all enrolled participants who received treatment with study drug voxelotor in the current study. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Non- SCD-Related TEAEs | From date of informed consent up to 28 days after last dose of study drug (maximum up to 300.7 weeks) | The safety population included all enrolled participants who received treatment with study drug voxelotor in the current study. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With SCD-Related Treatment Emergent Serious Adverse Events (TESAEs) | From date of informed consent up to 28 days after last dose of study drug (maximum up to 300.7 weeks) | The safety population included all enrolled participants who received treatment with study drug voxelotor in the current study. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Non-SCD-Related TESAEs | From date of informed consent up to 28 days after last dose of study drug (maximum up to 300.7 weeks) | The safety population included all enrolled participants who received treatment with study drug voxelotor in the current study. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Annualized Incidence Rate (Events Per Person Years) of SCD-Related Complications | From date of informed consent up to earlier of 28 days after last dose of study drug or end of study date (maximum up to 300.7 weeks) | The safety population included all enrolled participants who received treatment with study drug voxelotor in the current study. | Events per person years | NUMBER |
| PRIMARY | Annualized Incidence Rate (VOC Events Per Person Years) of On-Treatment Vaso-occlusive Crisis (VOCs) | From date of informed consent to last dose of study drug (maximum up to 296.7 weeks) | The safety population included all enrolled participants who received treatment with study drug voxelotor in the current study. | VOC events per person years | NUMBER |
| SECONDARY | Change From Baseline in Hemoglobin Level at Week 48 | Baseline, Week 48 | The safety population included all enrolled participants who received treatment with study drug voxelotor in the current study. Here, Overall Number of Participants Analyzed signifies number of participants evaluable for this outcome measure and Number Analyzed = number of participants evaluable for specified rows. | Gram per deciliter (g/dL) | MEAN |
| SECONDARY | Percent Change From Baseline in Reticulocytes Percentage at Week 48 | Baseline, Week 48 | The safety population included all enrolled participants who received treatment with study drug voxelotor in the current study. Here, Overall Number of Participants Analyzed signifies number of participants evaluable for this outcome measure. | Percent change | MEAN |
| SECONDARY | Percent Change From Baseline in Absolute Reticulocytes at Week 48 | Baseline, Week 48 | The safety population included all enrolled participants who received treatment with study drug voxelotor in the current study. Here, Overall Number of Participants Analyzed signifies number of participants evaluable for this outcome measure. | Percent change | MEDIAN |
| SECONDARY | Percent Change From Baseline in Indirect Bilirubin at Week 48 | Baseline, Week 48 | The safety population included all enrolled participants who received treatment with study drug voxelotor in the current study. Here, Overall Number of Participants Analyzed signifies number of participants evaluable for this outcome measure. | Percent change | MEAN |
Source ClinicalTrials.gov
Results by group33
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Sickle Cell Disease (SCD)-Related Treatment Emergent Adverse Events (TEAEs) | Prior Treatment of Placebo in GBT440-031 (NCT03036813) | 47 | Participants | ||
| Number of Participants With Sickle Cell Disease (SCD)-Related Treatment Emergent Adverse Events (TEAEs) | Prior Treatment of Voxelotor 900mg in GBT440-031(NCT03036813) | 42 | Participants | ||
| Number of Participants With Sickle Cell Disease (SCD)-Related Treatment Emergent Adverse Events (TEAEs) | Prior Treatment of Voxelotor 1500 mg in GBT440-031(NCT03036813) | 42 | Participants | ||
| Number of Participants With Non- SCD-Related TEAEs | Prior Treatment of Placebo in GBT440-031 (NCT03036813) | 59 | Participants | ||
| Number of Participants With Non- SCD-Related TEAEs | Prior Treatment of Voxelotor 900mg in GBT440-031(NCT03036813) | 49 | Participants | ||
| Number of Participants With Non- SCD-Related TEAEs | Prior Treatment of Voxelotor 1500 mg in GBT440-031(NCT03036813) | 52 | Participants | ||
| Number of Participants With SCD-Related Treatment Emergent Serious Adverse Events (TESAEs) | Prior Treatment of Placebo in GBT440-031 (NCT03036813) | 39 | Participants | ||
| Number of Participants With SCD-Related Treatment Emergent Serious Adverse Events (TESAEs) | Prior Treatment of Voxelotor 900mg in GBT440-031(NCT03036813) | 33 | Participants | ||
| Number of Participants With SCD-Related Treatment Emergent Serious Adverse Events (TESAEs) | Prior Treatment of Voxelotor 1500 mg in GBT440-031(NCT03036813) | 22 | Participants | ||
| Number of Participants With Non-SCD-Related TESAEs | Prior Treatment of Placebo in GBT440-031 (NCT03036813) | 18 | Participants |
1 to 10 of 33Source ClinicalTrials.gov
See all 33 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Prior Treatment of Placebo in GBT440-031 (NCT03036813) | 63 | |
| Overall Study | STARTED | Prior Treatment of Voxelotor 900mg in GBT440-031(NCT03036813) | 58 | |
| Overall Study | STARTED | Prior Treatment of Voxelotor 1500mg in GBT440-031(NCT03036813) | 58 | |
| Overall Study | Treated | Prior Treatment of Placebo in GBT440-031 (NCT03036813) | 62 | |
| Overall Study | Treated | Prior Treatment of Voxelotor 900mg in GBT440-031(NCT03036813) | 58 | |
| Overall Study | Treated | Prior Treatment of Voxelotor 1500mg in GBT440-031(NCT03036813) | 58 | |
| Overall Study | COMPLETED | Prior Treatment of Placebo in GBT440-031 (NCT03036813) | 16 | |
| Overall Study | COMPLETED | Prior Treatment of Voxelotor 900mg in GBT440-031(NCT03036813) | 11 | |
| Overall Study | COMPLETED | Prior Treatment of Voxelotor 1500mg in GBT440-031(NCT03036813) | 19 | |
| Overall Study | NOT COMPLETED | Prior Treatment of Placebo in GBT440-031 (NCT03036813) | 47 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events1,245
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 | Sickle cell anaemia with crisis | Blood and lymphatic system disorders | Prior Treatment of Placebo in GBT440-031 (NCT03036813) | 28 | 62 | |
| other | Sickle cell anaemia with crisis | Blood and lymphatic system disorders | Prior Treatment of Voxelotor 900mg in GBT440-031(NCT03036813) | 24 | 58 | |
| other | Sickle cell anaemia with crisis | Blood and lymphatic system disorders | Prior Treatment of Voxelotor 1500mg in GBT440-031(NCT03036813) | 31 | 58 | |
| other | Anaemia | Blood and lymphatic system disorders | Prior Treatment of Placebo in GBT440-031 (NCT03036813) | 2 | 62 | |
| other | Anaemia | Blood and lymphatic system disorders | Prior Treatment of Voxelotor 900mg in GBT440-031(NCT03036813) | 3 | 58 | |
| other | Anaemia | Blood and lymphatic system disorders | Prior Treatment of Voxelotor 1500mg in GBT440-031(NCT03036813) | 3 | 58 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Prior Treatment of Placebo in GBT440-031 (NCT03036813) | 1 | 62 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Prior Treatment of Voxelotor 900mg in GBT440-031(NCT03036813) | 2 | 58 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Prior Treatment of Voxelotor 1500mg in GBT440-031(NCT03036813) | 2 | 58 | |
| other | Haemolysis | Blood and lymphatic system disorders | Prior Treatment of Placebo in GBT440-031 (NCT03036813) | 1 | 62 |
1 to 10 of 1,245Source ClinicalTrials.gov
See all 1,245 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Prior Treatment of Placebo in GBT440-031 (NCT03036813) | 3 | 62 |
| Total, all-cause mortality | Prior Treatment of Voxelotor 900mg in GBT440-031(NCT03036813) | 2 | 58 |
| Total, all-cause mortality | Prior Treatment of Voxelotor 1500mg in GBT440-031(NCT03036813) | 5 | 58 |
| Total, other adverse events | Prior Treatment of Placebo in GBT440-031 (NCT03036813) | 60 | 62 |
| Total, other adverse events | Prior Treatment of Voxelotor 900mg in GBT440-031(NCT03036813) | 50 | 58 |
| Total, other adverse events | Prior Treatment of Voxelotor 1500mg in GBT440-031(NCT03036813) | 54 | 58 |
| Total, serious adverse events | Prior Treatment of Placebo in GBT440-031 (NCT03036813) | 42 | 62 |
| Total, serious adverse events | Prior Treatment of Voxelotor 900mg in GBT440-031(NCT03036813) | 35 | 58 |
| Total, serious adverse events | Prior Treatment of Voxelotor 1500mg in GBT440-031(NCT03036813) | 26 | 58 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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.
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/NCT03573882
GET https://conductscience.org/api/trials/v1/NCT03573882/results?page=2