Thalidomide Therapy for VEOIBD
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Thalidomide.
- Who it may be relevant to
- Registry conditions: Inflammatory Bowel Diseases. Basic parameters: 28 Days — 6 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Thalidomide Therapy for Very Early Onset Inflammatory Bowel Disease
Overview
This is a single center, observational study to investigate the clinical outcomes of thalidomide treatment for very early onset inflammatory bowel disease
Detailed description
Very early onset inflammatory bowel disease (VEOIBD) refers to IBD diagnosed before 6 years of age. VEOIBD comprises a distinct subset of pediatric IBD characterized by stronger genetic predisposition, predominant colonic involvement, more extensive inflammation, more severe course, and peculiar response to treatments. Previous studies have been demonstrated that thalidomide, an oral molecule with immunomodulatory, antiangiogenic, and TNF-suppressing properties, is an effective and safe treatment for adults and children with IBD refractory to conventional therapies. This study is aimed to evaluate the efficacy and tolerance of thalidomide in VEOIBD.
Interventions
- Drug Thalidomide
thalidomide, dose 1.5-2.5mg/kg.d, orally taken before bedtime.
Primary outcome measures
- endoscopic response rate [Time frame: week 52]
Secondary outcome measures (6)
- clinical response rate [Time frame: week8, week26, week52]
- biomarker remission rate [Time frame: week8, week26, week52]
- growth development [Time frame: week52]
- clinical remission rate with steroid-free [Time frame: week26]
- adverse event [Time frame: within one year]
- treatment persistence [Time frame: within one year]
Eligibility criteria
Inclusion criteria
- diagnosed with inflammatory bowel disease
- disease onset less than 6 years old
- Pediatric Crohn's disease Activity Index (PCDAI)>30 or Pediatric Ulcerative Colitis Activity Index (PUCAI)>35
- patients and their legal guardians were willing to receive thalidomide treatment and participate in this study.
Exclusion criteria
- liver dysfunction
- allergy to thalidomide
- with Neuropathy
- with thrombosis
- thalidomide treatment in the previous 30 days
- biologics treatment in the previous 8 weeks
- not suitable participated in this study
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
China · 1 center
- Lin Wang — Shanghai
Identifiers
NCT: NCT06382519 · Thal in VEOIBD