N-of-1 Trials In Children With Hypertension (NICHE)

The University of Texas System (UT)

Status and phase: Completed; Phase 4

Conditions: Pediatric Hypertension

Treatments: Drug: Lisinopril; Other: NICHE method; Drug: Amlodipine; Other: Usual care; Drug: Losartan; Drug: Hydrochlorothiazide

Study type: Interventional

Funder types: Other; NIH

Identifiers

NCT03461003

UL1TR003167 (U.S. NIH Grant/Contract)

HSC-MS-17-1014

Details and patient eligibility

About

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.

Full 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.

Enrollment: 49 patients

Sex: All

Ages: 10 to 22 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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49 participants in 2 patient groups

NICHE method

Experimental group

Description:

In the NICHE method, antihypertensive therapy will be chosen using an n-of-1 trial to identify the preferred therapy. Preferred therapy is defined a priori as that which produces the greatest reduction in ambulatory BP without intolerable side effects. Tested drugs will include amlodipine or losartan, lisinopril, and hydrochlorothiazide.

Treatment:

Drug: Amlodipine

Drug: Lisinopril

Other: NICHE method

Drug: Hydrochlorothiazide

Drug: Losartan

Usual Care

Active Comparator group

Description:

No protocol will be introduced to standardize BP management in the control arm.

Treatment:

Other: Usual care