NCT03461003

N-of-1 Trials In Children With Hypertension

A Randomized Clinical Trial of the N-of-1 Approach in Children With Hypertension

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 49 (actual)
• Sponsor: The University of Texas Health Science Center, Houston · Academic / Other
• Sex: All
• Age: 10 Years – 22 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Pediatric Hypertension

Interventions

Type	Name	Description
OTHER	NICHE method	Based on underlying comorbidities at enrollment, patients will be assigned to either an unrestricted protocol (amlodipine versus lisinopril) or a restricted protocol (losartan versus lisinopril). Two drugs are tested in randomized order. Failure to achieve BP control at maximum doses of the first two drugs will result in augmentation with a diuretic (hydrochlorothiazide). BP control is assessed with 24 hour ambulatory monitoring at the end of each 2 week treatment period. Side effect tolerability is assessed with a questionnaire.
OTHER	Usual care	Physician preference will dictate choice and adjustment of antihypertensive medication, BP measurement modality, interval between visits, and assessment of side effects.
DRUG	Amlodipine	Calcium-channel blocker; antihypertensive
DRUG	Lisinopril	Angiotensin-converting-enzyme (ACE) inhibitor; antihypertensive
DRUG	Hydrochlorothiazide	Thiazide diuretic; antihypertensive
DRUG	Losartan	Angiotensin II receptor blocker (ARB); antihypertensive

Timeline

• Start date: 2018-04-02
• Primary completion: 2020-11-14
• Completion: 2021-05-02
• First posted: 2018-03-09
• Last updated: 2022-09-09
• Results posted: 2022-09-09

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study