Thalidomide Therapy for VEOIBD

Inflammatory Bowel Diseases Clinical Trial

Official title:

Thalidomide Therapy for Very Early Onset Inflammatory Bowel Disease

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06382519
• Other study ID #: Thal in VEOIBD
• Status: Not yet recruiting
• Start date: May 15, 2024
• Est. completion date: December 31, 2026

Study information

• Verified date: April 2024
• Source: Children's Hospital of Fudan University
• Contact: Ying Huang
• Phone: 021-64931727
• Email: yhuang815[@]163.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 40
• Est. completion date: December 31, 2026
• Est. primary completion date: December 31, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 28 Days to 6 Years

Eligibility

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

Related Conditions & MeSH terms

• Inflammatory Bowel Diseases
• Intestinal Diseases

Intervention

Drug:
• Thalidomide
thalidomide, dose 1.5-2.5mg/kg.d, orally taken before bedtime.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Children's Hospital of Fudan University

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	endoscopic response rate	The Simple Endoscopic Score for Crohn Disease decreased 50% compared with baseline OR Mayo score 0-1 (higher scores mean a worse outcome)	week 52
Secondary	clinical response rate	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).	week8, week26, week52
Secondary	biomarker remission rate	biomarker remission is defined as not only C-reactive protein less than 8mg/L, but also fecal calprotectin less than 100ug/g.	week8, week26, week52
Secondary	growth development	weight and height Z score (higher scores mean a better outcome)	week52
Secondary	clinical remission rate with steroid-free	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.	week26
Secondary	adverse event	The rate of the potential adverse events were monitored during the intervention.	within one year
Secondary	treatment persistence	the time between the start and the last dose of thalidomide	within one year