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NCT Number: NCT06382519

Thalidomide Therapy for VEOIBD

This is a single center, observational study to investigate the clinical outcomes of thalidomide treatment for very early onset inflammatory bowel disease

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Key information

Conditions

Inflammatory Bowel Diseases Digestive System Diseases Gastroenteritis Gastrointestinal Diseases Intestinal Diseases

Age range

28 day–6 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Lin Wang

Shanghai, Shanghai Municipality, 201102, China

Location status: Recruiting

Location contact

Ying Huang

CONTACT

[email protected]

02164931727

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

thalidomide

Drug

thalidomide, dose 1.5-2.5mg/kg.d, orally taken before bedtime.

Primary outcomes

  1. endoscopic response rate

    Time frame: week 52

    The Simple Endoscopic Score for Crohn Disease decreased 50% compared with baseline OR Mayo score 0-1 (higher scores mean a worse outcome)

Secondary outcomes

  1. clinical response rate

    Time frame: week8, week26, week52

    Inflammatory bowel disease mainly contains Crohn's disease and ulcerative colitis. If the patient was diagnosed with Crohn's disease, we use the Pediatric Crohn's disease Activity Index score to evaluate the clinical response, which is defined as Pediatric Crohn's disease Activity Index decreased more than 12.5 compared with baseline. If the patient was diagnosed with ulcerative colitis, we use the Pediatric Ulcerative Colitis Activity Index score to evaluate the clinical response, which is defined as Pediatric Ulcerative Colitis Activity Index score decreased more than 20 compared with baseline. The Pediatric Crohn's disease Activity Index score varies from 0 to 100 and the Pediatric Ulcerative Colitis Activity Index score varies from 0 to 85 (higher scores mean a worse outcome).

  2. biomarker remission rate

    Time frame: week8, week26, week52

    biomarker remission is defined as not only C-reactive protein less than 8mg/L, but also fecal calprotectin less than 100ug/g.

  3. growth development

    Time frame: week52

    weight and height Z score (higher scores mean a better outcome)

  4. clinical remission rate with steroid-free

    Time frame: week26

    If the patient was diagnosed with Crohn's disease, this outcome is defined as the Pediatric Crohn's disease Activity Index score less than 10 without steroid treatment. If the patient was diagnosed with ulcerative colitis, this outcome is defined as the Pediatric Ulcerative Colitis Activity Index score less than 10 without steroid treatment.

  5. adverse event

    Time frame: within one year

    The rate of the potential adverse events were monitored during the intervention.

  6. treatment persistence

    Time frame: within one year

    the time between the start and the last dose of thalidomide

Study contacts

Contact information is provided by the study sponsor or research team.

Ying Huang

CONTACT

[email protected]

021-64931727

Sponsors and collaborators

Lead sponsor

Children's Hospital of Fudan University

Other

Registry information

Official study title

Thalidomide Therapy for Very Early Onset Inflammatory Bowel Disease

Important dates

Study start
2024
Primary completion
2025
Study completion
2026
First posted
Apr 24, 2024
Registry last updated
May 22, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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