Trial Outcomes & Findings for Assessing the Feasibility of N-of-1 Trials in Children With Hypertension and Chronic Kidney Disease (NCT NCT04591171)

Last Updated: 2023-03-20

Results Overview

24-hour mean arterial pressure (MAP) is the mean arterial pressure over 24 hours \[ambulatory blood pressure monitoring (ABPM) will be used to record blood pressure every 20 min for 24 hours, and all recordings will be averaged to obtain 24-hour MAP\]. 24-hour MAP will be assessed at baseline and at 6 months, and the change between baseline and 6-months will be reported.

Recruitment status

COMPLETED

Study phase

Target enrollment

9 participants

Primary outcome timeframe

baseline, 6 months

Results posted on

2023-03-20

Participant Flow

Participant milestones

Participant milestones
Measure	N-of-1 Trial Guided Clinical Decision Making n-of-1 trial guided clinical decision making: The n-of-1 trial will, on the level of the individual patient, test which treatment strategy produces superior blood pressure reduction without unacceptable side effects. After discussion with the patient/caregiver dyad to identify whether they have specific concerns about particular medications, the nephrologist (clinician) will decide which two drugs and dosages will be tested in the n-of-1 trial (the protocol does not determine drug or dose). The two drugs chosen by the clinician will be assessed at clinician-selected dosing in a randomized treatment order (e.g., ABAB) for two weeks per treatment period and two treatment periods per drug.
Overall Study STARTED	9
Overall Study COMPLETED	9
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Assessing the Feasibility of N-of-1 Trials in Children With Hypertension and Chronic Kidney Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	N-of-1 Trial Guided Clinical Decision Making n=9 Participants n-of-1 trial guided clinical decision making: The n-of-1 trial will, on the level of the individual patient, test which treatment strategy produces superior blood pressure reduction without unacceptable side effects. After discussion with the patient/caregiver dyad to identify whether they have specific concerns about particular medications, the nephrologist (clinician) will decide which two drugs and dosages will be tested in the n-of-1 trial (the protocol does not determine drug or dose). The two drugs chosen by the clinician will be assessed at clinician-selected dosing in a randomized treatment order (e.g., ABAB) for two weeks per treatment period and two treatment periods per drug.
Age, Categorical <=18 years	9 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	11 years n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	4 Participants n=5 Participants
Race (NIH/OMB) White	5 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	9 participants n=5 Participants
Transplant or Dialysis Status Transplant	4 Participants n=5 Participants
Transplant or Dialysis Status Dialysis	0 Participants n=5 Participants
Transplant or Dialysis Status non-dialysis CKD	5 Participants n=5 Participants
distance from home to clinic	30 miles n=5 Participants
glomerular filtration rate (GFR)	65 mL/min/1.73m^2 n=5 Participants

PRIMARY outcome

Timeframe: baseline, 6 months

Outcome measures

Outcome measures
Measure	N-of-1 Trial Guided Clinical Decision Making n=9 Participants n-of-1 trial guided clinical decision making: The n-of-1 trial will, on the level of the individual patient, test which treatment strategy produces superior blood pressure reduction without unacceptable side effects. After discussion with the patient/caregiver dyad to identify whether they have specific concerns about particular medications, the nephrologist (clinician) will decide which two drugs and dosages will be tested in the n-of-1 trial (the protocol does not determine drug or dose). The two drugs chosen by the clinician will be assessed at clinician-selected dosing in a randomized treatment order (e.g., ABAB) for two weeks per treatment period and two treatment periods per drug.
Change in 24-hour Mean Arterial Pressure (MAP)	1.4 mmHg Standard Deviation 5.9

SECONDARY outcome

Timeframe: 6 months

Target blood pressure is 24-hour mean arterial pressure (MAP) at less than the 50th percentile.

Outcome measures

Outcome measures
Measure	N-of-1 Trial Guided Clinical Decision Making n=9 Participants n-of-1 trial guided clinical decision making: The n-of-1 trial will, on the level of the individual patient, test which treatment strategy produces superior blood pressure reduction without unacceptable side effects. After discussion with the patient/caregiver dyad to identify whether they have specific concerns about particular medications, the nephrologist (clinician) will decide which two drugs and dosages will be tested in the n-of-1 trial (the protocol does not determine drug or dose). The two drugs chosen by the clinician will be assessed at clinician-selected dosing in a randomized treatment order (e.g., ABAB) for two weeks per treatment period and two treatment periods per drug.
Number of Participants With Target Blood Pressure	4 Participants

SECONDARY outcome

Timeframe: baseline

Target blood pressure is 24-hour mean arterial pressure (MAP) at less than the 50th percentile.

Outcome measures

Outcome measures
Measure	N-of-1 Trial Guided Clinical Decision Making n=9 Participants n-of-1 trial guided clinical decision making: The n-of-1 trial will, on the level of the individual patient, test which treatment strategy produces superior blood pressure reduction without unacceptable side effects. After discussion with the patient/caregiver dyad to identify whether they have specific concerns about particular medications, the nephrologist (clinician) will decide which two drugs and dosages will be tested in the n-of-1 trial (the protocol does not determine drug or dose). The two drugs chosen by the clinician will be assessed at clinician-selected dosing in a randomized treatment order (e.g., ABAB) for two weeks per treatment period and two treatment periods per drug.
Number of Participants With Target Blood Pressure	3 Participants

Adverse Events

N-of-1 Trial Guided Clinical Decision Making

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

Joyce P Samuel, MD, MS

The University of Texas Health Science Center at Houston

Phone: 713-500-6708

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place