Clinical trial NCT03461003: N-of-1 Trials In Children With Hypertension
Official title: A Randomized Clinical Trial of the N-of-1 Approach in Children With Hypertension
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Pediatric Hypertension
Lead sponsor: The University of Texas Health Science Center, Houston
Sponsor class: OTHER
Collaborators: name: National Center for Advancing Translational Sciences (NCATS)
First posted: 2018-03-09
Enrollment: 49
Sex: ALL
Minimum age (years): 10
Maximum age (years): 22
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The single patient (n-of-1) randomized trial is an underused approach to resolving therapeutic uncertainty by using a patient's own data to inform an individualized treatment plan. The proposed research is designed to assess whether the n-of-1 trial approach improves blood pressure control compared to usual care. This trial aims to advance learning about not only the treatment of pediatric hypertension but also the use of a neglected type of randomized trial to optimize the care of each patient.

Detailed description: Pediatric hypertension, a growing problem, often requires prescription of antihypertensive medication. Pediatric hypertension specialists lack an evidentiary base on which to establish definitive clinical practice guidelines for first-line therapy. Significant practice variation is an unsurprising consequence. Routine choice of the same first-line therapy for most patients with hypertension, absent testing other options, may delay correction of blood pressure for months or years. Failure to incorporate patient preferences in medical decision-making may also contribute to decreased patient satisfaction and adherence.

Large parallel-group, comparative effectiveness trials are likely not on the horizon for this population. Moreover, heterogeneity of treatment effects would minimize the generalizability of such a trial to all patients.

This is a parallel-group, randomized clinical trial to compare the n-of-1 trial approach to usual care in normalizing blood pressure while minimizing exposure to compliance-reducing side effects.

Eligibility criteria: Inclusion Criteria:

* treating physician determines that pharmacologic therapy is indicated for treatment of hypertension
* ambulatory hypertension has been confirmed (off meds) within 12 months of enrollment

Exclusion Criteria:

* age \< 10 years
* resistant hypertension (requiring ≥ 3 drug therapy)
* absolute contraindication or allergy to any of the tested drugs.

Primary outcomes:
measure: Number of Participants With Ambulatory Blood Pressure (BP) Control at 6 Months
timeFrame: 6 months from enrollment
Secondary outcomes:
measure: Change in Mean 24-hour Ambulatory Systolic Blood Pressure
timeFrame: baseline, 6 months
measure: Change in Mean Wake Ambulatory Systolic Blood Pressure
timeFrame: baseline, 6 months
measure: Number of Participants Who Reported That Side Effects From Medication Led Them to Discontinue Medication
timeFrame: from baseline to 6 months
measure: Number of Participants Who Self-reported Adherence to Intervention
timeFrame: from month 5 to month 6
measure: Patient Satisfaction With Intervention as Assessed by a Survey
timeFrame: 6 months
Other outcomes:
Locations:
city: Houston
country: United States
facility: The University of Texas Health Science Center at Houston
state: Texas
Study officials:
affiliation: The University of Texas Health Science Center, Houston
name: Joyce P. Samuel, MD, MS
role: PRINCIPAL_INVESTIGATOR
References:
citation: Flynn JT, Daniels SR, Hayman LL, Maahs DM, McCrindle BW, Mitsnefes M, Zachariah JP, Urbina EM; American Heart Association Atherosclerosis, Hypertension and Obesity in Youth Committee of the Council on Cardiovascular Disease in the Young. Update: ambulatory blood pressure monitoring in children and adolescents: a scientific statement from the American Heart Association. Hypertension. 2014 May;63(5):1116-35. doi: 10.1161/HYP.0000000000000007. Epub 2014 Mar 3. No abstract available.