Clinical Trial Details
— Status: Completed
Administrative data
NCT number |
NCT04963764 |
Other study ID # |
1R34HL153474-01 |
Secondary ID |
|
Status |
Completed |
Phase |
Early Phase 1
|
First received |
|
Last updated |
|
Start date |
October 18, 2021 |
Est. completion date |
May 27, 2022 |
Study information
Verified date |
October 2022 |
Source |
Ann & Robert H Lurie Children's Hospital of Chicago |
Contact |
n/a |
Is FDA regulated |
No |
Health authority |
|
Study type |
Interventional
|
Clinical Trial Summary
This pilot study will evaluate study processes and feasibility of a future large-scale
clinical trial that proposes to test whether low-risk children managed as outpatients with
community-acquired pneumonia (CAP) and procalcitonin (PCT) levels <0.25 ng/mL treated with
placebo have a similar clinical response to those treated with antibiotics and fewer adverse
effects.
Description:
This pilot clinical trial is a 3-site, randomized, placebo-controlled, double-blinded trial
assessing the feasibility of comparing amoxicillin to placebo in children 12 months to <6
years of age who present to the ED with Community Acquired Pneumonia (CAP), a procalcitonin
(PCT) concentration of <0.25 ng/mL, and who will be treated as outpatients.
Screening and Enrollment This pilot feasibility trial will enroll over a 6-month period and
take place at three sites (Ann and Robert H. Lurie Children's Hospital of Chicago, Cincinnati
Children's Hospital Medical Center and The Children's Hospital of Philadelphia) that are or
were members of the Pediatric Emergency Care Applied Research Network (PECARN). This study
aims to enroll 36 patients in total (2 patients per month, per site). Clinical research
coordinators (CRCs) at participating EDs will screen potentially eligible patients with
respiratory tract symptoms and discuss eligibility with the treating attending physician. If
thought to be eligible and a diagnosis of CAP is presumed by the treating physician, the CRC
will approach the patient to complete screening procedures. The study will proceed in 2
stages, each with its own informed consent process. During Stage 1, baseline characteristics
and serum PCT levels will be ascertained. Stage 2 will consist of a randomized trial of
amoxicillin vs. placebo in the subset of patients from Stage 1 that have PCT <0.25 ng/mL.
Randomization After enrollment and confirmation of a PCT <0.25 ng/mL, patients will be
randomized to a 10-day course of either amoxicillin (80-100 mg/kg divided BID up to 4,000
mg/day) or placebo. Randomization to amoxicillin or placebo will be at a 1:1 ratio with block
sizes of 2 and 4. Patients will be stratified by the clinical site and randomization will be
performed through an online system. As a double-blind clinical trial, the study patients and
their parents/guardians, investigators and study staff will be blinded to study treatment
assignment for the duration of the study.
Study Drug Administration Local investigational drug pharmacies will be provided with active
study medication (i.e., amoxicillin) and matching placebo. Site pharmacies at each
institution will store study drug and dispense as needed. Study medications will both be
liquid reconstituted from powder, and will resemble each other with regards to appearance,
favor, consistency and packaging. Study products will be labeled with numerical codes that
will maintain allocation concealment. Site investigational pharmacies will be provided with
amoxicillin and placebo, in addition to the randomization scheme. The pharmacy will aliquot
amoxicillin and placebo into blinded bottles based on randomization scheme.
Follow-up The guardians of participants will be asked to complete a daily symptom diary,
using an online data collection form in REDCap, during the first 7 days after the initial
Emergency Department study visit. The follow-up will assess patient condition, clinical
response, signs or symptoms of clinical deterioration and other adverse effects. The primary
outcome will be assessed at day 7 (+/- 2 days), using video chat technology that is standard
on most smart phones, tablets, and computers. Video follow-up will be performed by site
clinician investigators. In the rare case that a mobile device or computer with video chat
technology is not available to the family, the day 7 follow-up will occur by telephone or
text through an online system. A final follow-up, performed by site research staff, by
telephone call, will occur at Day 21 (+/- 2 days) to assess overall disease course and
secondary outcomes.
Data Collection At baseline, demographics, medical history, and history of current illness
will be obtained from all participants during stage 1 (pre-randomization). Vital signs will
be obtained and a brief physical examination will be performed. After the initial ED visit,
patients will record symptoms on daily basis for 6 days via an online data collection form.
Follow-up assessments will be completed via telehealth visit or telephone for days 7 and 21.
Follow-up visits will collect data regarding symptoms, adverse events and return to medical
care, in addition to assessing adherence to study procedures (i.e., medication adherence and
daily symptom diary completion). If there is concern for adverse events or deterioration that
may warrant medical care, the participant's caregiver will be instructed to contact their
primary care physician, emergency department, or call 911, as indicated.