A Phase 3 interventional study of Voxelotor Only Product in Oral Dose Form and Placebo in Sickle Cell Disease, sponsored by Pfizer. Terminated at 1 site in United States. Open to participants aged 8 Years to 18 Years. Per ClinicalTrials.gov, last updated 2024-05-02.
Sponsored by Pfizer · Phase 3, Interventional, and Treatment
This is a Phase 3b, randomized, double-blind, placebo-controlled, multicenter study to assess the treatment effect of voxelotor on neurocognitive function as assessed by the National Institute of Health (NIH) Toolbox Cognition Module of executive abilities in pediatric participants (8 to \< 18 years) with SCD.
Eligible participants will receive daily treatment with 1500 mg voxelotor or matching placebo for 12 weeks. During screening and at the end of 12 weeks participants will undergo a series of tests to measure the change in neurocognitive functions.
1,103 studies on the registry are indexed under Anemia, Sickle Cell; 235 are open to participants now.
This study's enrollment of 1 is below the median of 40 across 750 interventional studies indexed under Anemia, Sickle Cell.
Browse Anemia, Sickle Cell studies →Pfizer is the lead sponsor of 3,244 studies on the registry; 139 are open to participants now.
Of its 582 completed or terminated interventional studies of FDA-regulated products, 381 (65%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Clinically significant bacterial, fungal, parasitic, or viral infection which requires therapy.
Voxelotor 1500mg or equivalent daily as a tablet or powder for oral suspension
Drug: Voxelotor Only Product in Oral Dose Form
Matching Placebo
Drug: Placebo
During the Randomized Treatment Period, participants will be randomized in a 1:1 ratio to receive 1500 mg of voxelotor (or the weight-adjusted equivalent dose for participants \< 12 years old), once daily (administered orally as tablets/PFOS) or matching placebo for 12 weeks in addition to ongoing standard of care (SOC) treatment.
During the Randomized Treatment Period, participants will be randomized in a 1:1 ratio to receive 1500 mg of voxelotor (or the weight-adjusted equivalent dose for participants \< 12 years old), once daily (administered orally as tablets/PFOS) or matching placebo for 12 weeks in addition to ongoing standard of care (SOC) treatment.
Change From Baseline in Executive Abilities Composite Score Assessed Using NIH Toolbox Cognition Module At Week 12
The NIH toolbox cognition module is a standardized cognitive battery comprising of executive function, episodic memory, language, processing speed, working memory, and attention as subdomains. The toolbox is comprised of 7 test instruments that measure 8 abilities within 6 major cognitive domains and have been categorized as executive abilities (Dimensional Change Card Sort Test, Flanker Inhibitory Control and Attention Test, and List Sorting Test) and nonexecutive abilities (Picture Vocabulary Test, Oral Reading Recognition Test, and Picture Sequence Memory Test). The NIH toolbox standard score has a mean of 100 and standard deviation (SD) of 15. The higher the score, the better the performance.
Time frame: Baseline (last assessment prior to first dose of study treatment), Week 12
Change From Baseline in Pattern Comparison Processing Speed Test Scores Assessed Using NIH Toolbox Cognition Module at Week 12
The NIH Toolbox Cognition Battery Test is a comprehensive set of neuro behavioral measurements used to assess cognitive, sensory and motor functions where a higher composite score equals better cognitive performance. The NIH Toolbox Cognitive Scores used the Fully Adjusted Scale score (also referred to as the fully corrected T-score) with a mean of 50 and standard deviation of 10.
Time frame: Baseline (last assessment prior to first dose of study treatment), Week 12
Change From Baseline in Nonexecutive Cognitive Abilities Composite Score Assessed Using NIH Toolbox Cognition Module Up to Week 12
The NIH toolbox cognition module is a standardized cognitive battery comprising of executive function, episodic memory, language, processing speed, working memory, and attention as subdomains. The toolbox is currently comprised of 7 test instruments that measure 8 abilities within 6 major cognitive domains and have been categorized as executive abilities (Dimensional Change Card Sort Test, Flanker Inhibitory Control and Attention Test, and List Sorting Test) and nonexecutive abilities (Picture Vocabulary Test, Oral Reading Recognition Test, and Picture Sequence Memory Test). Baseline was defined as the last assessment performed prior to receiving the first dose of study treatment. The NIH toolbox standard score has a mean of 100 and SD of 15. The higher the score, the better the performance.
Time frame: Baseline (last assessment prior to first dose of study treatment) up to Week 12
Change From Baseline in Hemoglobin Level Up to Week 12
Time frame: Baseline (last assessment prior to first dose of study treatment) up to Week 12
Change From Baseline in Absolute Reticulocyte Count Up to Week 12
Time frame: Baseline (last assessment prior to first dose of study treatment) up to Week 12
Change From Baseline in Percentage Reticulocyte Up to Week 12
Time frame: Baseline (last assessment prior to first dose of study treatment) up to Week 12
Change From Baseline in Lactate Dehydrogenase (LDH) Up to Week 12
Time frame: Baseline (last assessment prior to first dose of study treatment) up to Week 12
Change From Baseline in Unconjugated Bilirubin Up to Week 12
Time frame: Baseline (last assessment prior to first dose of study treatment) up to Week 12
Percent Change From Baseline in Unconjugated Bilirubin, Absolute Reticulocyte, Percentage Reticulocytes, Lactate Dehydrogenase (LDH) Up to Week 12
Time frame: Baseline (last assessment prior to first dose of study treatment) up to Week 12
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
An adverse event was defined as any untoward medical occurrence associated with the use of a drug in participants whether or not considered drug related. TEAEs were those events with onset dates that occurred during the treatment period.
Time frame: From start of study treatment (Day 1) up to Week 12
| Milestone | Voxelotor | Placebo |
|---|---|---|
| Started | 0 | 0 |
| Completed | 0 | 0 |
| Not completed | 0 | 0 |
The NIH toolbox cognition module is a standardized cognitive battery comprising of executive function, episodic memory, language, processing speed, working memory, and attention as subdomains. The toolbox is comprised of 7 test instruments that measure 8 abilities within 6 major cognitive domains and have been categorized as executive abilities (Dimensional Change Card Sort Test, Flanker Inhibitory Control and Attention Test, and List Sorting Test) and nonexecutive abilities (Picture Vocabulary Test, Oral Reading Recognition Test, and Picture Sequence Memory Test). The NIH toolbox standard score has a mean of 100 and standard deviation (SD) of 15. The higher the score, the better the performance.
No measurements were reported for this outcome.
The NIH Toolbox Cognition Battery Test is a comprehensive set of neuro behavioral measurements used to assess cognitive, sensory and motor functions where a higher composite score equals better cognitive performance. The NIH Toolbox Cognitive Scores used the Fully Adjusted Scale score (also referred to as the fully corrected T-score) with a mean of 50 and standard deviation of 10.
No measurements were reported for this outcome.
The NIH toolbox cognition module is a standardized cognitive battery comprising of executive function, episodic memory, language, processing speed, working memory, and attention as subdomains. The toolbox is currently comprised of 7 test instruments that measure 8 abilities within 6 major cognitive domains and have been categorized as executive abilities (Dimensional Change Card Sort Test, Flanker Inhibitory Control and Attention Test, and List Sorting Test) and nonexecutive abilities (Picture Vocabulary Test, Oral Reading Recognition Test, and Picture Sequence Memory Test). Baseline was defined as the last assessment performed prior to receiving the first dose of study treatment. The NIH toolbox standard score has a mean of 100 and SD of 15. The higher the score, the better the performance.
No measurements were reported for this outcome.
No measurements were reported for this outcome.
No measurements were reported for this outcome.
No measurements were reported for this outcome.
No measurements were reported for this outcome.
No measurements were reported for this outcome.
No measurements were reported for this outcome.
An adverse event was defined as any untoward medical occurrence associated with the use of a drug in participants whether or not considered drug related. TEAEs were those events with onset dates that occurred during the treatment period.
No measurements were reported for this outcome.
Collected over Not applicable as adverse events were not reported.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Voxelotor | — | — | — |
| Placebo | — | — | — |
Only one participant was enrolled. Hence, data cannot be reported due to risk of re-identification of participant.
| Age, Customized(Participants) | Voxelotor | Placebo | Total |
|---|
| Sex: Female, Male(Participants) | Voxelotor | Placebo | Total |
|---|---|---|---|
| Female | — | — | — |
| Male | — | — | — |
| Race/Ethnicity, Customized(Participants) | Voxelotor | Placebo | Total |
|---|
Documents are hosted by the registry — open the source record to download them.
Plan to share: Yes — Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
This study is terminated, as verified in Mar 2024. You cannot join it, but the record below documents what was studied.
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