Trial Outcomes & Findings for Voxelotor Neurocognitive Function Study (NCT NCT05228834)
NCT ID: NCT05228834
Last Updated: 2024-05-02
Results Overview
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.
TERMINATED
PHASE3
1 participants
Baseline (last assessment prior to first dose of study treatment), Week 12
2024-05-02
Participant Flow
Only 1 participant was enrolled. The study was terminated due to slow enrollment and resource reprioritization at Global Blood Therapeutics (L-GBT). Based on the low enrollment, data was not reported due to risk of re-identification of participant.
Participant milestones
| Measure |
Voxelotor
Participants were to be randomized to receive Voxelotor 1500 milligram (mg) orally once daily for 12 weeks.
|
Placebo
Participants were to be randomized to receive placebo matched to Voxelotor 1500 mg orally once daily for 12 weeks.
|
|---|---|---|
|
Overall Study
STARTED
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0
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0
|
|
Overall Study
COMPLETED
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0
|
0
|
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Overall Study
NOT COMPLETED
|
0
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Voxelotor Neurocognitive Function Study
Baseline characteristics by cohort
Baseline data not reported
PRIMARY outcome
Timeframe: Baseline (last assessment prior to first dose of study treatment), Week 12Population: Study was terminated and only one participant was enrolled. Hence, data cannot be reported for this outcome measure due to risk of re-identification of participant.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline (last assessment prior to first dose of study treatment), Week 12Population: Study was terminated and only one participant was enrolled. Hence, data cannot be reported for this outcome measure due to risk of re-identification of participant.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline (last assessment prior to first dose of study treatment) up to Week 12Population: Study was terminated and only one participant was enrolled. Hence, data cannot be reported for this outcome measure due to risk of re-identification of participant.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline (last assessment prior to first dose of study treatment) up to Week 12Population: Study was terminated and only one participant was enrolled. Hence, data cannot be reported for this outcome measure due to risk of re-identification of participant.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline (last assessment prior to first dose of study treatment) up to Week 12Population: Study was terminated and only one participant was enrolled. Hence, data cannot be reported for this outcome measure due to risk of re-identification of participant.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline (last assessment prior to first dose of study treatment) up to Week 12Population: Study was terminated and only one participant was enrolled. Hence, data cannot be reported for this outcome measure due to risk of re-identification of participant.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline (last assessment prior to first dose of study treatment) up to Week 12Population: Study was terminated and only one participant was enrolled. Hence, data cannot be reported for this outcome measure due to risk of re-identification of participant.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline (last assessment prior to first dose of study treatment) up to Week 12Population: Study was terminated and only one participant was enrolled. Hence, data cannot be reported for this outcome measure due to risk of re-identification of participant.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline (last assessment prior to first dose of study treatment) up to Week 12Population: Study was terminated and only one participant was enrolled. Hence, data cannot be reported for this outcome measure due to risk of re-identification of participant.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: From start of study treatment (Day 1) up to Week 12Population: Study was terminated and only one participant was enrolled. Hence, data cannot be reported for this outcome measure due to risk of re-identification of participant.
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.
Outcome measures
Outcome data not reported
Adverse Events
Voxelotor
Placebo
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
- Publication restrictions are in place
Restriction type: OTHER