---
title: "Thalidomide Versus Infliximab in New Onset Crohn's Disease With Poor Prognostic Factors"
nct_id: NCT03221166
overall_status: TERMINATED
phase: PHASE3
sponsor: IRCCS Burlo Garofolo
study_type: INTERVENTIONAL
primary_condition: Crohn Disease
countries: Italy
canonical_url: "https://parkinsonspathways.com/agent/trials/NCT03221166.md"
clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT03221166"
ct_last_update_post_date: 2020-09-04
last_seen_at: "2026-05-12T07:08:14.385Z"
source: ClinicalTrials.gov (mirrored, no enrichment)
---
# Thalidomide Versus Infliximab in New Onset Crohn's Disease With Poor Prognostic Factors

**Official Title:** Thalidomide, a Novel Immunological Treatment to Modify the Natural History of Paediatric Crohn's Disease: a New Proposal From a Well-established Paediatric Research Network

**NCT ID:** [NCT03221166](https://clinicaltrials.gov/study/NCT03221166)

## Key Facts

- **Status:** TERMINATED
- **Why Stopped:** Difficulty in recruiting subjects who meet the inclusion/exclusion criteria
- **Phase:** PHASE3
- **Study Type:** INTERVENTIONAL
- **Target Enrollment:** 9
- **Lead Sponsor:** IRCCS Burlo Garofolo
- **Collaborators:** Centro di Riferimento Oncologico - Aviano, Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia
- **Conditions:** Crohn Disease
- **Start Date:** 2018-02-27
- **Completion Date:** 2020-07-31
- **CT.gov Last Update:** 2020-09-04

## Brief Summary

Crohn's disease (CD) is a life-long inflammatory bowel disease disease with an unknown pathogenesis. The ultimate goal of therapy is to modify the natural history of CD thus reducing complications. Thalidomide is a small molecule with immunomodulatory and anti-angiogenetic properties. It is currently approved for the treatment of erythema nodosum leprosum, an immunological complication of leprosy and multiple myeloma. It has also been used in several other inflammatory diseases of the skin and of the mucosal membranes, such as Behcet disease, oropharyngeal ulcers in AIDS, cutaneous lupus, and graft versus host disease. Many case series and one pediatric randomized controlled trial proved the efficacy of thalidomide in the treatment of children with CD refractory to standard treatments. In these patients, clinical remission was achieved in about 50% of the cases and was maintained for a mean time superior of 3 years. Mucosal healing after 52 weeks of treatment was observed in 40% of the patients in clinical remission. Moreover, thalidomide was found to have a steroid-sparing effect and to decrease the need for surgical interventions. The clinical and endoscopic efficacy of thalidomide was also observed in children with failure to respond or intolerance to anti-TNF biological drugs.

The aim of this multicentric prospective randomized controlled is to evaluate the efficacy and safety of thalidomide vs infliximab in changing the natural history of CD in patients with poor prognostic outcome. Moreover, the study will evaluate the immunological and genetical mechanisms of CD, the mechanisms of action thalidomide in CD and will the pharmacokinetics, metabolomics and pharmacogenomics of thalidomide, and their impact on thalidomide safety and effectiveness.

## Eligibility

- **Minimum age:** 6 Years
- **Maximum age:** 17 Years
- **Sex:** ALL
- **Healthy Volunteers:** No

```
Inclusion Criteria:

* Age at diagnosis \<18 years and \>=6 years
* New diagnosis of CD based on Porto criteria
* CD with inflammatory phenotype (non-penetrating, non-fistulizing) and with no need for surgery except for perinal fistulas
* Presence of at least one of the following risk factors for poor prognosis:

  * fistulizing perianal disease
  * pan-enteric disease
  * disease extension \> 60 cm
  * severe growth delay (height z-score \< -2 DS)
  * severe osteoporosis (z score \< -2 DS)
  * hypoalbuminemia (\< 3g/dL) or high C-reactive protein (2 times higher the normal range)
* Acceptance of the Risk Evaluation and Mitigation Strategy (REMS) program for reducing the teratogenic risk.

Exclusion Criteria:

* ongoing pregnancy
* presence of peripheral neuropathy
* HIV
* patients with transplanted organs
* ongoing major infections or other severe diseases
* participation to other experimental studies.
```

## Arms

- **Thalidomide** (EXPERIMENTAL) — Thalidomide is a immunomodulatory and antiangiogenetic drug with anti tumor necrosis factor (TNF) alpha properties
- **Infliximab** (ACTIVE_COMPARATOR) — Infliximab is a chimeric monoclonal antibody against TNF alpha

## Interventions

- **Thalidomide** (DRUG) — Thalidomide is a immunomodulatory and antiangiogenetic drug with anti TNF alpha properties
- **Infliximab** (DRUG) — Infliximab is a chimeric monoclonal antibody against TNF alpha

## Primary Outcomes

- **Efficacy in inducing mucosal healing** _(time frame: 52 weeks)_ — Proportion of patients that achieve mucosal healing, defined by a Simplified Endoscopic Activity Index for CD (SES-CD) ≤ 2.

## Secondary Outcomes

- **Efficacy in inducing clinical response** _(time frame: 12 weeks)_
- **Efficacy in inducing clinical response** _(time frame: 52 weeks)_
- **Efficacy in inducing clinical remission** _(time frame: 12 weeks)_
- **Efficacy in inducing clinical remission** _(time frame: 52 weeks)_
- **Efficacy in reducing the need to change therapy** _(time frame: 12 weeks)_
- **Efficacy in reducing the need to change therapy** _(time frame: 52 weeks)_
- **Efficacy in reducing hospitalizations** _(time frame: 52 weeks)_
- **Efficacy in reducing the need for surgery** _(time frame: 52 weeks)_
- **Efficacy in reducing erythrocyte sedimentation rate** _(time frame: Each time point between enrolment and 52 weeks (0, 4, 8, 14, 26, 38, 52 weeks))_
- **Efficacy in reducing C-reactive protein** _(time frame: Each time point between enrolment and 52 weeks (0, 4, 8, 14, 26, 38, 52 weeks))_
- **Efficacy in reducing faecal calprotectin** _(time frame: Each time point between enrolment and 52 weeks (0, 4, 8, 14, 26, 38, 52 weeks))_
- **Efficacy in modifying body mass index** _(time frame: Each time point between enrolment and 52 weeks (0, 4, 8, 14, 26, 38, 52 weeks))_
- **Efficacy in modifying height-for-age z score** _(time frame: Each time point between enrolment and 52 weeks (0, 4, 8, 14, 26, 38, 52 weeks))_
- **Efficacy in modifying weight-for-age z score** _(time frame: Each time point between enrolment and 52 weeks (0, 4, 8, 14, 26, 38, 52 weeks))_
- **Evaluation of the Treatment-Emergent Adverse Events** _(time frame: Between enrolment and 52 weeks)_
- **Direct and indirect costs** _(time frame: 52 weeks)_

## Locations (6)

- Dipartimento di Pediatria dell'Università di Napoli "Federico II", Naples, Campania, Italy
- IRCCS Burlo Garofolo, Trieste, Friuli Venezia Giulia, Italy
- Pediatria III Gastroenterologia ed Endoscopia Digestiva, Istituto Giannina Gaslini, Genoa, Liguria, Italy
- Fondazione MBBM , Azienda Ospedaliera San Gerardo - Università Milano Bicocca, Monza, Lombardy, Italy
- Unità di Gastroenterologia Pediatrica e Fibrosi Cistica, Dipartimento di Scienze Pediatriche Mediche e Chirurgiche, Policlinico Universitario, Messina, Sicily, Italy
- Gastroenterologia e Nutrizione Pediatrica, Azienda Ospedaliero Universitaria Meyer, Florence, Tuscany, Italy

## Recent Field Changes (last 30 days)

- `status.overallStatus` — added _(2026-05-12)_
- `status.whyStopped` — added _(2026-05-12)_
- `status.primaryCompletionDate` — added _(2026-05-12)_
- `status.completionDate` — added _(2026-05-12)_
- `status.lastUpdatePostDate` — added _(2026-05-12)_
- `design.phases` — added _(2026-05-12)_
- `design.enrollmentCount` — added _(2026-05-12)_
- `eligibility.criteria` — added _(2026-05-12)_
- `eligibility.minAge` — added _(2026-05-12)_
- `eligibility.maxAge` — added _(2026-05-12)_
- `eligibility.sex` — added _(2026-05-12)_
- `outcomes.primary` — added _(2026-05-12)_
- `outcomes.secondary` — added _(2026-05-12)_
- `armsInterventions.arms` — added _(2026-05-12)_
- `armsInterventions.interventions` — added _(2026-05-12)_
- `sponsor.lead` — added _(2026-05-12)_
- `sponsor.collaborators` — added _(2026-05-12)_
- `results.hasResults` — added _(2026-05-12)_
- `locations.dipartimento di pediatria dell'università di napoli "federico ii"|naples|campania|italy` — added _(2026-05-12)_
- `locations.irccs burlo garofolo|trieste|friuli venezia giulia|italy` — added _(2026-05-12)_
- `locations.pediatria iii gastroenterologia ed endoscopia digestiva, istituto giannina gaslini|genoa|liguria|italy` — added _(2026-05-12)_
- `locations.fondazione mbbm , azienda ospedaliera san gerardo - università milano bicocca|monza|lombardy|italy` — added _(2026-05-12)_
- `locations.unità di gastroenterologia pediatrica e fibrosi cistica, dipartimento di scienze pediatriche mediche e chirurgiche, policlinico universitario|messina|sicily|italy` — added _(2026-05-12)_
- `locations.gastroenterologia e nutrizione pediatrica, azienda ospedaliero universitaria meyer|florence|tuscany|italy` — added _(2026-05-12)_

---

*Canonical: https://parkinsonspathways.com/agent/trials/NCT03221166.md*  
*Source data (authoritative): https://clinicaltrials.gov/study/NCT03221166*  
*This page is a raw mirror with no AI summary, no editorial enrichment, and no Parkinson's-specific filtering.*