Thalidomide Therapy for VEOIBD

Thalidomide Therapy for Very Early Onset Inflammatory Bowel Disease

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

Study Overview

• Status: Recruiting
• Conditions: Inflammatory Bowel Diseases
• Intervention / Treatment: Drug: Thalidomide

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.

• Study Type: Observational
• Enrollment (Estimated): 40

Contacts and Locations

• Study Contact: Name: Ying Huang; Phone Number: 021-64931727; Email: yhuang815@163.com

Study Locations

• China
  • Shanghai
    • Shanghai, Shanghai, China, 201102
      • Recruiting
      • Lin Wang
      • Contact: Ying Huang; Phone Number: 02164931727; Email: yhuang815@163.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

Children diagnosed with very early onset inflammatory bowel disease who were seen at the Children's hospital of Fudan university who met the inclusion and exclusion criteria would be enrolled in this study.

Description

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

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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
growth development	weight and height Z score (higher scores mean a better outcome)	week52
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
adverse event	The rate of the potential adverse events were monitored during the intervention.	within one year
treatment persistence	the time between the start and the last dose of thalidomide	within one year

Collaborators and Investigators

• Sponsor: Children's Hospital of Fudan University
• Investigators: Principal Investigator: Ying Huang, Children's Hospital of Fudan University

Study record dates

Study Major Dates

• Study Start (Actual): May 15, 2024
• Primary Completion (Estimated): December 31, 2025
• Study Completion (Estimated): December 31, 2026

Study Registration Dates

• First Submitted: April 11, 2024
• First Submitted That Met QC Criteria: April 21, 2024
• First Posted (Actual): April 24, 2024

Study Record Updates

• Last Update Posted (Actual): May 22, 2025
• Last Update Submitted That Met QC Criteria: May 19, 2025
• Last Verified: May 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Gastrointestinal Diseases; Gastroenteritis; Intestinal Diseases; Inflammatory Bowel Diseases; Anti-Bacterial Agents; Anti-Infective Agents; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Physiological Effects of Drugs; Angiogenesis Inhibitors; Angiogenesis Modulating Agents; Growth Substances; Growth Inhibitors; Leprostatic Agents; Thalidomide
• Other Study ID Numbers: Thal in VEOIBD

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No