Prospective Registry Study and Clinical Observation of Children With Crohn's Disease
Conditions: Crohn Disease
Sponsor: Tongji Hospital

Prospective Registry Study and Clinical
Observation of Children With Crohn's
Disease
July 24, 2024

Study Protocol
Brief
title
Prospective cohort establishment and clinical observation of children with
Crohn's disease
Research institution Tongji Hospital, Tongji Medical College, Huazhong University of Science
and Technology
Major investigators
Sainan SHU professor, Biao Zou attending doctor
Date
July 24, 2024
Study population
Children with Crohn's Disease
Purpose
of
research
Primary Outcome Measure: To evaluate the clinical manifestations,
treatment options, and improved clinical outcome of children with Crohn's
disease in real-world Settings: (1) analysis of clinical manifestations; (2)
probability of using the same treatment options; (3) Clinical outcome;
Secondary Outcome Measures: (1) Evaluate the clinical application of enteral
nutrition (EEN/ PEN, reason, route of administration, dose, course of
treatment); (2) Evaluate the clinical use of glucocorticoids (reason, route of
administration, dose, course of treatment); (3) Evaluate the use of
immunosuppressants (reason, route of administration, dose, course of
treatment); (4) Evaluate the clinical application of biologics (including
domestic infliximab (Taizhou Mabtech Pharmaceutical Co.,Ltd)) (dose,
duration, effectiveness, safety, and economy); (5) Evaluate the application of
fecal microbiota transplantation (cause, transplantation routes, dose, course of
treatment); (6) To evaluate the medical costs and resource consumption of the
treatment of Crohn's disease in children (state insurance costs, commercial
insurance costs, self-financing costs, others);
Other Pre-specified Outcomes: To conduct registration studies to obtain
information on the efficacy and safety of biologics (e.g. infliximab (Taizhou
Mabtech Pharmaceutical Co.,Ltd)) used in children with Crohn's disease, and to
evaluate the dose, duration, and economics of clinical use of biologics (e.g.
infliximab (Taizhou Mabtech Pharmaceutical Co.,Ltd)).

Study
endpoint
Primary efficacy endpoint: 14 weeks clinical response rate.
Secondary efficacy endpoints:
(1) Clinical remission rates at 14 and 54 weeks (PCDAI≤10)；
(2) Clinical response rate at 54 weeks (PCDAI reduction ≥15 points and
total PCDAI≤30 points)
(3) Endoscopic response rate at 14 and 54 weeks (SES-CD≤2);
(4) Mucosal healing rate at 14 and 54 weeks (SES-CD=0);
(5) Changes of serum C-reactive protein, erythrocyte sedimentation rate
and fecal calcarein from baseline at 14 and 54 weeks;
(6) Changes in intestinal flora from baseline at 14 and 54weeks;
(7) Changes in serum new inflammatory markers from baseline at 14 and
54 weeks;
Safety end point:
The incidence of adverse events/adverse reactions after the first dose was
recorded (observed up to 102 weeks).
Study design
Prospective, single center, registry study. This study is an observational study,
without intervention in the treatment of patients, patients will accept the treatment
plan formulated by the attending physician, researchers and patients respectively
truthfully register the corresponding data generated by the clinical practice, collect
the laboratory examination and the evaluation data of doctors during the visit, and
combine the hospital visit and the electronic information system. Statistical
analysis was performed after follow-up.
Eligibility Criteria
Inclusion Criteria: (1) Age 0~17 years old; (2) Children with a definite diagnosis
of Crohn's disease; (3) Patients and their guardians must sign informed consent
Exclusion Criteria: Other conditions deemed inappropriate by the doctor to
participate in the study.
Study
Population
Description
100
Research visit
Total observation 102 weeks; A total of 4 interview points were set up: baseline,
14 weeks, 54 weeks, 102 weeks; According to the patient's wishes, the course of
the disease, and the actual diagnosis and treatment, the visit can be ended early.
Planned study time
Estimated total project time: 3-5 years
Statistic analysis
For continuous variables, the number of cases, mean, standard deviation, median,
25th and 75th percentiles, minimum, and maximum are described. For discrete
variables, the numbers and percentages of each type are described. Missing
values are not included in the calculation of percentages unless otherwise noted.
Unless otherwise noted, all statistical tests will use a two-sided test with α= 0.05,
P < 0.05 is considered statistically significant, and a two-sided 95% confidence
interval (CI) is calculated if necessary.

Study flow chart
Interview point
Baseline
treatment period
Visit time
Visit time 1
Visit time 2
Visit time 3
Visit time 4
-14d~0d
14w±7d
54w±7d
102w±7d
informed consent
√
Demographic data
√
physical examination
√
√
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vital signs
√
√
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medical history
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Medication history
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Operation History
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family history
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allergic history
√
Laboratory index
Blood
routine
examination
√
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blood biochemistry
√
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routine urine test
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stool routine
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fecal calprotectin
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Serum 25-(OH)-D3
√
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coagulation function
√
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inflammatory factor
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C-reactive protein
√
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erythrocyte sedimentation
rate
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PCDAI score
√
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endoscopy
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histopathological
examination
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imageological examination
√
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medication
administration
record
√
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adverse event
√
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Combination
medication/concomitant
therapy
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medical cost
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