Trial Outcomes & Findings for Uric Acid Reduction as a Novel Treatment for Pediatric Chronic Kidney Disease (NCT NCT03865407)

Results Overview

Change in Cys-C eGFR over time

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

17 participants

Primary outcome timeframe

The difference in Cystatin C eGFR between baseline and 6 months will be measured

Results posted on

2021-05-27

Participant Flow

Participant milestones

Participant milestones
Measure	Allopurinol Allopurinol will be administered to this treatment arm group for 6 months. Participants will receive the lowest FDA recommended dose for weight, and will be titrated upwards to achieve the goal uric acid level of 3-5 mg/dL throughout the trial. Allopurinol: Allopurinol dosed to target uric acid levels of 3-5 mg/dL.	Standard of Care Control The treatment arm will be compared to a standard of care arm.
Overall Study STARTED	10	7
Overall Study COMPLETED	7	7
Overall Study NOT COMPLETED	3	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Allopurinol Allopurinol will be administered to this treatment arm group for 6 months. Participants will receive the lowest FDA recommended dose for weight, and will be titrated upwards to achieve the goal uric acid level of 3-5 mg/dL throughout the trial. Allopurinol: Allopurinol dosed to target uric acid levels of 3-5 mg/dL.	Standard of Care Control The treatment arm will be compared to a standard of care arm.
Overall Study Adverse Event	1	0
Overall Study Withdrawal by Subject	1	0
Overall Study Medical change caused patient to no longer meet inclusion/exclusion criteria	1	0

Baseline Characteristics

Uric Acid Reduction as a Novel Treatment for Pediatric Chronic Kidney Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Allopurinol n=7 Participants Allopurinol will be administered to this treatment arm group for 6 months. Participants will receive the lowest FDA recommended dose for weight, and will be titrated upwards to achieve the goal uric acid level of 3-5 mg/dL throughout the trial. Allopurinol: Allopurinol dosed to target uric acid levels of 3-5 mg/dL.	Standard of Care Control n=7 Participants The treatment arm will be compared to a standard of care arm.	Total n=14 Participants Total of all reporting groups
Age, Continuous	14.29 years STANDARD_DEVIATION 2.5 • n=93 Participants	13.86 years STANDARD_DEVIATION 2.61 • n=4 Participants	14.07 years STANDARD_DEVIATION 2.46 • n=27 Participants
Sex: Female, Male Female	1 Participants n=93 Participants	3 Participants n=4 Participants	4 Participants n=27 Participants
Sex: Female, Male Male	6 Participants n=93 Participants	4 Participants n=4 Participants	10 Participants n=27 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=93 Participants	0 Participants n=4 Participants	1 Participants n=27 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	6 Participants n=93 Participants	7 Participants n=4 Participants	13 Participants n=27 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants	1 Participants n=4 Participants	1 Participants n=27 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Black or African American	5 Participants n=93 Participants	3 Participants n=4 Participants	8 Participants n=27 Participants
Race (NIH/OMB) White	2 Participants n=93 Participants	3 Participants n=4 Participants	5 Participants n=27 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Region of Enrollment United States	7 participants n=93 Participants	7 participants n=4 Participants	14 participants n=27 Participants

PRIMARY outcome

Timeframe: The difference in Cystatin C eGFR between baseline and 6 months will be measured

Change in Cys-C eGFR over time

Outcome measures

Outcome measures
Measure	Allopurinol n=7 Participants Allopurinol will be administered to this treatment arm group for 6 months. Participants will receive the lowest FDA recommended dose for weight, and will be titrated upwards to achieve the goal uric acid level of 3-5 mg/dL throughout the trial. Allopurinol: Allopurinol dosed to target uric acid levels of 3-5 mg/dL.	Standard of Care Control n=7 Participants The treatment arm will be compared to a standard of care arm.
eGFR Change	3.14 ml/min/1.73m^2 Standard Deviation 3.716	-0.29 ml/min/1.73m^2 Standard Deviation 5.251

PRIMARY outcome

Timeframe: The difference in Creatinine eGFR between baseline and 6 months will be measured

Change in Creatinine eGFR over time

Outcome measures

Outcome measures
Measure	Allopurinol n=7 Participants Allopurinol will be administered to this treatment arm group for 6 months. Participants will receive the lowest FDA recommended dose for weight, and will be titrated upwards to achieve the goal uric acid level of 3-5 mg/dL throughout the trial. Allopurinol: Allopurinol dosed to target uric acid levels of 3-5 mg/dL.	Standard of Care Control n=7 Participants The treatment arm will be compared to a standard of care arm.
eGFR Change	3.86 ml/min/1.73m^2 Standard Deviation 7.798	0 ml/min/1.73m^2 Standard Deviation 3.857

SECONDARY outcome

Timeframe: The difference in Serum Uric Acid between baseline and 6 months will be measured

Change in Serum Uric Acid

Outcome measures

Outcome measures
Measure	Allopurinol n=7 Participants Allopurinol will be administered to this treatment arm group for 6 months. Participants will receive the lowest FDA recommended dose for weight, and will be titrated upwards to achieve the goal uric acid level of 3-5 mg/dL throughout the trial. Allopurinol: Allopurinol dosed to target uric acid levels of 3-5 mg/dL.	Standard of Care Control n=7 Participants The treatment arm will be compared to a standard of care arm.
Serum Uric Acid Change	-3.33 mg/dl Standard Deviation 0.996	-0.46 mg/dl Standard Deviation 0.964

SECONDARY outcome

Timeframe: The difference in clinic systolic blood pressure between baseline and 6 months will be measured

Population: Follow up blood pressure data was not collected from one participant in the treatment group

Change in systolic blood pressure

Outcome measures

Outcome measures
Measure	Allopurinol n=6 Participants Allopurinol will be administered to this treatment arm group for 6 months. Participants will receive the lowest FDA recommended dose for weight, and will be titrated upwards to achieve the goal uric acid level of 3-5 mg/dL throughout the trial. Allopurinol: Allopurinol dosed to target uric acid levels of 3-5 mg/dL.	Standard of Care Control n=7 Participants The treatment arm will be compared to a standard of care arm.
Systolic Blood Pressure	-6.5 mmHg Standard Deviation 13.126	2.29 mmHg Standard Deviation 6.264

SECONDARY outcome

Timeframe: The difference in clinic diastolic blood pressure between baseline and 6 months will be measured

Population: Follow up blood pressure data was not collected from one participant in the treatment group

Change in diastolic blood pressure

Outcome measures

Outcome measures
Measure	Allopurinol n=6 Participants Allopurinol will be administered to this treatment arm group for 6 months. Participants will receive the lowest FDA recommended dose for weight, and will be titrated upwards to achieve the goal uric acid level of 3-5 mg/dL throughout the trial. Allopurinol: Allopurinol dosed to target uric acid levels of 3-5 mg/dL.	Standard of Care Control n=7 Participants The treatment arm will be compared to a standard of care arm.
Diastolic Blood Pressure	-7.33 mmHg Standard Deviation 6.29	3.983 mmHg Standard Deviation 7.543

OTHER_PRE_SPECIFIED outcome

Timeframe: Serum hs-CRP will be measured at baseline and 6 months

Compare the mean difference of serum hs-CRP from baseline to 6 months between groups

Outcome measures

Outcome measures
Measure	Allopurinol n=7 Participants Allopurinol will be administered to this treatment arm group for 6 months. Participants will receive the lowest FDA recommended dose for weight, and will be titrated upwards to achieve the goal uric acid level of 3-5 mg/dL throughout the trial. Allopurinol: Allopurinol dosed to target uric acid levels of 3-5 mg/dL.	Standard of Care Control n=7 Participants The treatment arm will be compared to a standard of care arm.
Serum High Sensitivity C-reactive Protein (Hs-CRP)	1.66 mg/dL Standard Deviation 3.724	-7.64 mg/dL Standard Deviation 18.639

Adverse Events

Allopurinol

Serious events: 1 serious events

Other events: 0 other events

Deaths: 0 deaths

Standard of Care Control

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Allopurinol n=7 participants at risk Allopurinol will be administered to this treatment arm group for 6 months. Participants will receive the lowest FDA recommended dose for weight, and will be titrated upwards to achieve the goal uric acid level of 3-5 mg/dL throughout the trial. Allopurinol: Allopurinol dosed to target uric acid levels of 3-5 mg/dL.	Standard of Care Control n=7 participants at risk The treatment arm will be compared to a standard of care arm.
Immune system disorders Drug reaction with eosinophilia and systemic symptoms (DRESS) syndrome	14.3% 1/7 • Number of events 1 • 6 months Regular investigator assessment, regular laboratory testing	0.00% 0/7 • 6 months Regular investigator assessment, regular laboratory testing

Other adverse events

Adverse event data not reported

Additional Information

Cristin Kaspar, MD

Virginia Commonwealth University

Phone: 804-628-3866

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place