Thalidomide Therapy for VEOIBD

NCT06382519 | Recruiting FDA Drug

• 1 other identifier: Thal in VEOIBD
• Study Type: observational
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Inflammatory Bowel Diseases

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (6)

• clinical response rate

  week8, week26, week52

• biomarker remission rate

  week8, week26, week52

• growth development

  week52

• clinical remission rate with steroid-free

  week26

• adverse event

  within one year

Interventions

Thalidomide DRUG

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

Eligibility Criteria

• Age: 28 Days - 6 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Children's Hospital of Fudan University: lead

Study Sites (1)

Lin Wang

Shanghai, Shanghai Municipality, 201102, China

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

blood sample, stool samples and biopsy samples during endoscopy

MeSH Terms

Conditions

Inflammatory Bowel Diseases

Interventions

Thalidomide

Condition Hierarchy (Ancestors)

Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Intestinal Diseases

Intervention Hierarchy (Ancestors)

Phthalimides; Phthalic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Piperidones; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Isoindoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Ying Huang

  Children's Hospital of Fudan University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Ying Huang

CONTACT

021-64931727; yhuang815@163.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Head of gastroenterology department

Study Record Dates

• First Submitted: April 11, 2024
• First Posted: April 24, 2024
• Study Start: May 15, 2024
• Primary Completion: December 31, 2025
• Study Completion (Estimated): December 31, 2026
• Last Updated: May 22, 2025

Record last verified: 2025-05

Locations

CN (1)