Trial · NCT02850406
Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease
NCT02850406, Phase 2, Terminated, Pfizer; results first posted 2025-08-15
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease. Trials, record NCT02850406. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02850406
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease
- NCT number
- NCT02850406
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 147 participants, actual
- Lead sponsor
- Pfizer
- Conditions
- Sickle Cell Disease
- Interventions
- Voxelotor
- First posted
- 2016-08-01
- Last updated
- 2025-08-15
- Results first posted
- 2025-08-15
Source ClinicalTrials.gov
Registered outcomes54
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Part A: Maximum Concentration (Cmax) of Voxelotor in Whole Blood | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose | |
| Primary | Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Voxelotor in Whole Blood | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose | AUC0-last was calculated using the linear/log trapezoid rule. |
| Primary | Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 Extrapolated to Infinity (AUC0-inf) of Voxelotor in Whole Blood | pre-dose, 2, 8, 24, 48, 96,168 and 336 hours post-dose | AUC0-inf was calculated as AUCt + Ct/lambdaz, where AUCt=area under the concentration-time curve at designated time, Ct is the last quantifiable concentration for whole blood and lambdaz=elimination rate constant. |
| Primary | Part B: Change From Baseline to Week 24 in Hemoglobin Level | Baseline, Week 24 | |
| Primary | Part C: Change From Baseline to Week 48 in Cerebral Blood Flow | Baseline, Week 48 | Cerebral blood flow was measured using transcranial Doppler (TCD) sonography. Change from baseline in cerebral blood flow as measured by the time-averaged mean of the maximum (TAMM) TCD velocity is reported. |
| Primary | Part D: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | From start of study treatment up to 28 days after study treatment discontinuation (Up to 52 weeks) | An adverse event (AE) was defined as any untoward medical occurrence in a participant administered a pharmaceutical product during the course of a clinical investigation. TEAE was defined as an AE that emerged on or after initiation of study drug (having been absent pre-treatment), or an AE that existed pre-treatment and worsened on treatment (relative to the pre-treatment state) through 28 days after study drug discontinuation. An SAE was any AE that resulted in any of the following outcomes: death, life threatening adverse drug experience, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, congenital anomaly or birth defect, other important medical events. AEs were classified as SCD-related and non-SCD related. |
| Secondary | Part A: Maximum Concentration (Cmax) of Voxelotor in Plasma | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose | |
| Secondary | Part A: Maximum Concentration (Cmax) of Voxelotor in Red Blood Cells (RBC) | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose | |
| Secondary | Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Voxelotor in Plasma | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose | AUC0-last was calculated using the linear/log trapezoid rule. |
| Secondary | Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Voxelotor in RBC | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose | AUC0-last was calculated using the linear/log trapezoid rule. |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 registered outcomesOutcomes54
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Part A: Maximum Concentration (Cmax) of Voxelotor in Whole Blood | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose | Pharmacokinetic (PK) population comprised of all participants who received any amount of study drug and who provided PK data from at least one post-dose sample in plasma. | Nanogram per milliliter | GEOMETRIC_MEAN |
| PRIMARY | Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Voxelotor in Whole Blood | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose | PK population comprised of all participants who received any amount of study drug and who provided PK data from at least one post-dose sample in plasma. | Hours*nanogram per milliliter | GEOMETRIC_MEAN |
| PRIMARY | Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 Extrapolated to Infinity (AUC0-inf) of Voxelotor in Whole Blood | pre-dose, 2, 8, 24, 48, 96,168 and 336 hours post-dose | PK population comprised of all participants who received any amount of study drug and who provided PK data from at least one post-dose sample in plasma. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Hours*nanogram per milliliter | GEOMETRIC_MEAN |
| PRIMARY | Part B: Change From Baseline to Week 24 in Hemoglobin Level | Baseline, Week 24 | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Gram per deciliter | MEAN |
| PRIMARY | Part C: Change From Baseline to Week 48 in Cerebral Blood Flow | Baseline, Week 48 | Efficacy-Evaluable Population comprised of all participants who received any amount of study drug. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Centimeter per second | MEAN |
| PRIMARY | Part D: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | From start of study treatment up to 28 days after study treatment discontinuation (Up to 52 weeks) | Safety population comprised of all participants who received any amount of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Part A: Maximum Concentration (Cmax) of Voxelotor in Plasma | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose | PK population comprised of all participants who received any amount of study drug and who provided PK data from at least one post-dose sample in plasma. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Nanogram per milliliter | GEOMETRIC_MEAN |
| SECONDARY | Part A: Maximum Concentration (Cmax) of Voxelotor in Red Blood Cells (RBC) | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose | PK population comprised of all participants who received any amount of study drug and who provided PK data from at least one post-dose sample in plasma. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Nanogram per milliliter | GEOMETRIC_MEAN |
| SECONDARY | Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Voxelotor in Plasma | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose | PK population comprised of all participants who received any amount of study drug and who provided PK data from at least one post-dose sample in plasma. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Hours*nanogram per milliliter | GEOMETRIC_MEAN |
| SECONDARY | Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Voxelotor in RBC | pre-dose, 2, 8, 24, 48, 96, 168 and 336 hours post-dose | PK population comprised of all participants who received any amount of study drug and who provided PK data from at least one post-dose sample in plasma. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Hours*nanogram per milliliter | GEOMETRIC_MEAN |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 outcomesResults by group156
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Part A: Maximum Concentration (Cmax) of Voxelotor in Whole Blood | Part A: Participants Aged 12 to 17 Years: Voxelotor 600 mg | 24300 | Geometric Coefficient of Variation 36.39 | Nanogram per milliliter | |
| Part A: Maximum Concentration (Cmax) of Voxelotor in Whole Blood | Part A: Participants Aged 6 to 11 Years: Voxelotor 600 mg | 47300 | Geometric Coefficient of Variation 43.62 | Nanogram per milliliter | |
| Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Voxelotor in Whole Blood | Part A: Participants Aged 12 to 17 Years: Voxelotor 600 mg | 1570000 | Geometric Coefficient of Variation 38.13 | Hours*nanogram per milliliter | |
| Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Voxelotor in Whole Blood | Part A: Participants Aged 6 to 11 Years: Voxelotor 600 mg | 2540000 | Geometric Coefficient of Variation 50.25 | Hours*nanogram per milliliter | |
| Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 Extrapolated to Infinity (AUC0-inf) of Voxelotor in Whole Blood | Part A: Participants Aged 12 to 17 Years: Voxelotor 600 mg | 1520000 | Geometric Coefficient of Variation 40.98 | Hours*nanogram per milliliter | |
| Part A: Area Under the Concentration-Time Curve (AUC) From Time 0 Extrapolated to Infinity (AUC0-inf) of Voxelotor in Whole Blood | Part A: Participants Aged 6 to 11 Years: Voxelotor 600 mg | 2570000 | Geometric Coefficient of Variation 50.59 | Hours*nanogram per milliliter | |
| Part B: Change From Baseline to Week 24 in Hemoglobin Level | Part B: Voxelotor 900 mg QD | 0.7 | Standard Deviation 0.86 | Gram per deciliter | |
| Part B: Change From Baseline to Week 24 in Hemoglobin Level | Part B: Voxelotor 1500 mg QD | 0.2 | Standard Deviation 1.02 | Gram per deciliter | |
| Part C: Change From Baseline to Week 48 in Cerebral Blood Flow | Part C: Participants Aged 4 to 11 Years: Voxelotor 1500 mg QD | -0.4 | Standard Deviation 16.76 | Centimeter per second | |
| Part C: Change From Baseline to Week 48 in Cerebral Blood Flow | Part C: Participants Aged 12 to 17 Years: Voxelotor 1500 mg QD | -26.3 | Standard Deviation 11.59 | Centimeter per second |
1 to 10 of 156Source ClinicalTrials.gov
See all 156 results by groupParticipant flow96
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Part A (Up to 15 Days) | STARTED | Part A: Participants Aged 12 to 17 Years: Voxelotor 600 mg | 7 | |
| Part A (Up to 15 Days) | STARTED | Part A: Participants Aged 6 to 11 Years: Voxelotor 600 mg | 6 | |
| Part A (Up to 15 Days) | STARTED | Part B: Voxelotor 900 mg QD | 0 | |
| Part A (Up to 15 Days) | STARTED | Part B: Voxelotor 1500 mg QD | 0 | |
| Part A (Up to 15 Days) | STARTED | Part C: Participants Aged 4 to 11 Years: Voxelotor 1500 mg QD | 0 | |
| Part A (Up to 15 Days) | STARTED | Part C: Participants Aged 12 to 17 Years: Voxelotor 1500 mg QD | 0 | |
| Part A (Up to 15 Days) | STARTED | Part D: Participants Aged 6 Months to <2 Years: Voxelotor 1500 mg QD | 0 | |
| Part A (Up to 15 Days) | STARTED | Part D: Participants Aged 2 to <4 Years: Voxelotor 1500 mg QD | 0 | |
| Part A (Up to 15 Days) | COMPLETED | Part A: Participants Aged 12 to 17 Years: Voxelotor 600 mg | 7 | |
| Part A (Up to 15 Days) | COMPLETED | Part A: Participants Aged 6 to 11 Years: Voxelotor 600 mg | 6 |
1 to 10 of 96Source ClinicalTrials.gov
See all 96 participant flowAdverse events1,568
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 | Part A: Participants Aged 12 to 17 Years: Voxelotor 600 mg | 1 | 7 | |
| other | Sickle cell anaemia with crisis | Blood and lymphatic system disorders | Part A: Participants Aged 6 to 11 Years: Voxelotor 600 mg | 1 | 6 | |
| other | Sickle cell anaemia with crisis | Blood and lymphatic system disorders | Part B: Voxelotor 900 mg QD | 5 | 25 | |
| other | Sickle cell anaemia with crisis | Blood and lymphatic system disorders | Part B: Voxelotor 1500 mg QD | 3 | 15 | |
| other | Sickle cell anaemia with crisis | Blood and lymphatic system disorders | Part C: Participants Aged 4 to 11 Years: Voxelotor 1500 mg QD | 10 | 51 | |
| other | Sickle cell anaemia with crisis | Blood and lymphatic system disorders | Part C: Participants Aged 12 to 17 Years: Voxelotor 1500 mg QD | 2 | 11 | |
| other | Sickle cell anaemia with crisis | Blood and lymphatic system disorders | Part D: Participants Aged 6 Months to <2 Years: Voxelotor 1500 mg QD | 5 | 9 | |
| other | Sickle cell anaemia with crisis | Blood and lymphatic system disorders | Part D: Participants Aged 2 to <4 Years: Voxelotor 1500 mg QD | 10 | 23 | |
| other | Acute chest syndrome | Respiratory, thoracic and mediastinal disorders | Part A: Participants Aged 12 to 17 Years: Voxelotor 600 mg | 0 | 7 | |
| other | Acute chest syndrome | Respiratory, thoracic and mediastinal disorders | Part A: Participants Aged 6 to 11 Years: Voxelotor 600 mg | 0 | 6 |
1 to 10 of 1,568Source ClinicalTrials.gov
See all 1,568 adverse eventsAdverse event totals24
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Part A: Participants Aged 12 to 17 Years: Voxelotor 600 mg | 0 | 7 |
| Total, all-cause mortality | Part A: Participants Aged 6 to 11 Years: Voxelotor 600 mg | 0 | 6 |
| Total, all-cause mortality | Part B: Voxelotor 900 mg QD | 0 | 25 |
| Total, all-cause mortality | Part B: Voxelotor 1500 mg QD | 0 | 15 |
| Total, all-cause mortality | Part C: Participants Aged 4 to 11 Years: Voxelotor 1500 mg QD | 0 | 51 |
| Total, all-cause mortality | Part C: Participants Aged 12 to 17 Years: Voxelotor 1500 mg QD | 0 | 11 |
| Total, all-cause mortality | Part D: Participants Aged 6 Months to <2 Years: Voxelotor 1500 mg QD | 0 | 9 |
| Total, all-cause mortality | Part D: Participants Aged 2 to <4 Years: Voxelotor 1500 mg QD | 0 | 23 |
| Total, other adverse events | Part A: Participants Aged 12 to 17 Years: Voxelotor 600 mg | 6 | 7 |
| Total, other adverse events | Part A: Participants Aged 6 to 11 Years: Voxelotor 600 mg | 3 | 6 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 35451176 | 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.
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.
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GET https://conductscience.org/api/trials/v1/NCT02850406/registered?page=2