Trial Outcomes & Findings for Augmenting Cerebral Blood Flow to Treat Established Multiple Sclerosis (NCT NCT02466074)

Last Updated: 2023-04-27

Results Overview

Percent change in global cerebral blood flow (CBF) after 24 weeks relative to pre-treatment baseline. Global CBF is determined using magnetic resonance imaging (MRI) methods. The data reported indicate the extent of change in global CBF--the higher the percent change, the greater the increase in global CBF and the better the outcome.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

5 participants

Primary outcome timeframe

baseline, 24 weeks

Results posted on

2023-04-27

Participant Flow

Participant milestones

Participant milestones
Measure	Acetazolamide Acetazolamide in oral daily divided dose administered for 6 consecutive months Acetazolamide: see arm description	Placebo Placebo in oral daily divided dose administered for 6 consecutive months Placebo: Placebo arm
Overall Study STARTED	5	0
Overall Study COMPLETED	5	0
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Augmenting Cerebral Blood Flow to Treat Established Multiple Sclerosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Acetazolamide n=5 Participants Acetazolamide in oral daily divided dose administered for 6 consecutive months Acetazolamide: see arm description	Placebo Placebo in oral daily divided dose administered for 6 consecutive months Placebo: Placebo arm	Total n=5 Participants Total of all reporting groups
Age, Continuous	43.6 years STANDARD_DEVIATION 5.9 • n=5 Participants	—	43.6 years STANDARD_DEVIATION 5.9 • n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants	—	3 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	—	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants	—	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=5 Participants	—	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	—	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	—	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	—	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	—	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants	—	1 Participants n=5 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants	—	3 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	—	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	—	1 Participants n=5 Participants
Region of Enrollment United States	5 participants n=5 Participants	—	5 participants n=5 Participants

PRIMARY outcome

Timeframe: baseline, 24 weeks

Population: No participants were assigned to the placebo arm.

Outcome measures

Outcome measures
Measure	Acetazolamide n=5 Participants Acetazolamide in oral daily divided dose administered for 6 consecutive months Acetazolamide: see arm description	Placebo Placebo in oral daily divided dose administered for 6 consecutive months Placebo: Placebo arm
Percent Change in Global Cerebral Blood Flow	22.9 percent change Standard Deviation 16.2	—

SECONDARY outcome

Timeframe: baseline, 24 weeks

Population: No participants were assigned to the placebo arm.

The data reported indicate the extent of change in white matter integrity as determined using the diffusion tensor imaging-magnetic resonance imaging (DTI-MRI) measure of mean diffusivity. A positive percent change value indicates an increase in mean diffusivity between baseline and 24 weeks, and a higher mean diffusivity value indicates a breakdown in white matter integrity, so the greater the percent change, the greater the breakdown of white matter integrity and the worse the outcome.

Outcome measures

Outcome measures
Measure	Acetazolamide n=5 Participants Acetazolamide in oral daily divided dose administered for 6 consecutive months Acetazolamide: see arm description	Placebo Placebo in oral daily divided dose administered for 6 consecutive months Placebo: Placebo arm
Percent Change in Tissue Integrity in White Matter (Mean Diffusivity)	1.9 percent change Standard Deviation 1.3	—

SECONDARY outcome

Timeframe: baseline, 24 weeks

Population: No participants were assigned to the placebo arm.

The data reported indicate the extent of change in white matter integrity as determined using the diffusion tensor imaging-magnetic resonance imaging (DTI-MRI) measure of fractional anisotropy. A negative percent change value indicates a decrease in fractional anisotropy between baseline and 24 weeks, and a lower fractional anisotropy value indicates a breakdown in white matter integrity, so the lower (and more negative) the percent change, the greater the breakdown of white matter integrity and the worse the outcome.

Outcome measures

Outcome measures
Measure	Acetazolamide n=5 Participants Acetazolamide in oral daily divided dose administered for 6 consecutive months Acetazolamide: see arm description	Placebo Placebo in oral daily divided dose administered for 6 consecutive months Placebo: Placebo arm
Percent Change in Tissue Integrity in White Matter (Fractional Anisotropy)	-2.8 percent change Standard Deviation 2.4	—

OTHER_PRE_SPECIFIED outcome

Timeframe: 1 year

Composite disability for an individual is defined as at least one of the following: a) any decrease in Expanded Disability Status Score (EDSS), b) 20% reduction in time to complete 9-Hole Peg Test (9HPT) for either the dominant or non-dominant hand, c) 20% decrease in Timed 25-Foot Walk (T25FW) or d) a \>= 4-point or 10% improvement in accuracy for Symbol Digit Modalities Test (SDMT).

Outcome measures

Outcome data not reported

Adverse Events

Acetazolamide

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

John A. Lincoln, MD, PhD

The University of Texas Health Science Center at Houston

Phone: 713-500-7016

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place