Trial Outcomes & Findings for Voxelotor for Improving Oxygen Saturation in Adults (NCT NCT05289570)

NCT ID: NCT05289570

Last Updated: 2024-07-24

Results Overview

SpO2 (oxygen saturation) is the percentage of oxygen in the blood. The fraction of inspired oxygen (FiO2) is the concentration of oxygen a person inhales.

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 3 participants
• Primary outcome timeframe: baseline to 2 days after initiation of voxelotor treatment
• Results posted on: 2024-07-24

Participant Flow

Participant milestones

Measure	Voxelotor Arm 500 mg of Voxelotor two times a day (for a total daily dose of 1000 mg per day) for 5 days. This dose may increase to a total maximum daily dose of 1500 mg (500 mg three times a day), if subject tolerates the initial dose as determined by study doctor. Voxelotor: 500 mg of voxelotor will be administered two times a day (for a total daily dose of 1000 mg per day) for 5 days. This dose may increase to a total maximum daily dose of 1500 mg (500 mg three times a day) if subject tolerates initial dose as determined by Principal Investigator (PI).
Overall Study STARTED	3
Overall Study COMPLETED	2
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Measure

Voxelotor Arm 500 mg of Voxelotor two times a day (for a total daily dose of 1000 mg per day) for 5 days. This dose may increase to a total maximum daily dose of 1500 mg (500 mg three times a day), if subject tolerates the initial dose as determined by study doctor. Voxelotor: 500 mg of voxelotor will be administered two times a day (for a total daily dose of 1000 mg per day) for 5 days. This dose may increase to a total maximum daily dose of 1500 mg (500 mg three times a day) if subject tolerates initial dose as determined by Principal Investigator (PI).

Overall Study Physician Decision

1

Baseline Characteristics

Voxelotor for Improving Oxygen Saturation in Adults

Baseline characteristics by cohort

Measure	Voxelotor Arm n=3 Participants 500 mg of Voxelotor two times a day (for a total daily dose of 1000 mg per day) for 5 days. This dose may increase to a total maximum daily dose of 1500 mg (500 mg three times a day), if subject tolerates the initial dose as determined by study doctor. Voxelotor: 500 mg of voxelotor will be administered two times a day (for a total daily dose of 1000 mg per day) for 5 days. This dose may increase to a total maximum daily dose of 1500 mg (500 mg three times a day) if subject tolerates initial dose as determined by Principal Investigator (PI).
Age, Continuous	73 years STANDARD_DEVIATION 15 • n=93 Participants
Sex: Female, Male Female	3 Participants n=93 Participants
Sex: Female, Male Male	0 Participants n=93 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=93 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	2 Participants n=93 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=93 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants
Race (NIH/OMB) Black or African American	0 Participants n=93 Participants
Race (NIH/OMB) White	3 Participants n=93 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants
Region of Enrollment United States	3 Participants n=93 Participants

PRIMARY outcome

Timeframe: baseline to 2 days after initiation of voxelotor treatment

Population: Participants with data collected at both timepoints.

SpO2 (oxygen saturation) is the percentage of oxygen in the blood. The fraction of inspired oxygen (FiO2) is the concentration of oxygen a person inhales.

Outcome measures

Measure

Voxelotor Arm n=2 Participants 500 mg of Voxelotor two times a day (for a total daily dose of 1000 mg per day) for 5 days. This dose may increase to a total maximum daily dose of 1500 mg (500 mg three times a day), if subject tolerates the initial dose as determined by study doctor. Voxelotor: 500 mg of voxelotor will be administered two times a day (for a total daily dose of 1000 mg per day) for 5 days. This dose may increase to a total maximum daily dose of 1500 mg (500 mg three times a day) if subject tolerates initial dose as determined by Principal Investigator (PI).

Change in SpO2/FiO2 (S/F) Ratio From Baseline to 2 Days After Initiation of Voxelotor Treatment

0.105 SpO2/FiO2 Standard Deviation 0.215

SECONDARY outcome

Timeframe: baseline to 5 days after initiation of voxelotor treatment

Population: Participants with data collected at both timepoints.

SpO2 (oxygen saturation) is the percentage of oxygen in the blood. The fraction of inspired oxygen (FiO2) is the concentration of oxygen a person inhales.

Outcome measures

Measure

Voxelotor Arm n=2 Participants 500 mg of Voxelotor two times a day (for a total daily dose of 1000 mg per day) for 5 days. This dose may increase to a total maximum daily dose of 1500 mg (500 mg three times a day), if subject tolerates the initial dose as determined by study doctor. Voxelotor: 500 mg of voxelotor will be administered two times a day (for a total daily dose of 1000 mg per day) for 5 days. This dose may increase to a total maximum daily dose of 1500 mg (500 mg three times a day) if subject tolerates initial dose as determined by Principal Investigator (PI).

Change in SpO2/FiO2 (S/F) Ratio From Baseline to 5 Days After Initiation of Voxelotor Treatment

-0.01 SpO2/FiO2 Standard Deviation 0.11

Adverse Events

Voxelotor Arm

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure

Voxelotor Arm n=3 participants at risk 500 mg of Voxelotor two times a day (for a total daily dose of 1000 mg per day) for 5 days. This dose may increase to a total maximum daily dose of 1500 mg (500 mg three times a day), if subject tolerates the initial dose as determined by study doctor. Voxelotor: 500 mg of voxelotor will be administered two times a day (for a total daily dose of 1000 mg per day) for 5 days. This dose may increase to a total maximum daily dose of 1500 mg (500 mg three times a day) if subject tolerates initial dose as determined by Principal Investigator (PI).

Gastrointestinal disorders Diarrhea

33.3% 1/3 • Participants will be followed for AEs from the time of voxelotor administration until 2 days after the last administration of voxelotor as defined by the wash out period.

Additional Information

Ian Welsby, MD

Duke University

Phone: 919-681-6752

Email: ian.welsby@duke.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place