Efficacy and safety of thalidomide in pediatric patients with Crohn's disease

• Register: EUCTR
• Last refreshed on: 7 June 2021
• Main ID: EUCTR2016-000786-24-IT
• Date of registration: 30/11/2016
• Prospective Registration: Yes
• Primary sponsor: IRCCS Burlo Garofolo
• Public title: Efficacy and safety of thalidomide in pediatric patients with Crohn's disease
• Scientific 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 - Talidomide in Crohn disease in Pediatrics
• Date of first enrolment: 07/03/2017
• Target sample size: 124
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-000786-24
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: yes; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: yes; Other trial design description: blindness of outcome assessor; If controlled, specify comparator, Other Medicinial Product: yes; Placebo: no; Other: no; Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Italy

Contacts

• Name: Alessandra Maestro
• Affiliation: IRCCS materno infantile Burlo Garofolo

• Name: Alessandra Maestro
• Affiliation: IRCCS materno infantile Burlo Garofolo

Key inclusion & exclusion criteria

Inclusion criteria:
- Age at diagnosis between 6 and 18 years
- New diagnosis of CD, defined on the basis of the Porto criteria after thus excluding infectious causes of enteritis.
- No complicated CD (not stenosing not fistulizing) or not needing surgery for the treatment of simple perianal fistulizing disease.
- Presence of at least one risk factor of negative prognosis (pernala fistulizing disease, panenteric disease, extention > 60 cm,
severe growth retardation, severe osteoporosis, hypoalbuminemia or raised CRP (normal valuse x2)
- Acceptance of contraceptive measures by patients in reproductive ages for reducing the risk of teratogenicity
Are the trial subjects under 18? yes
Number of subjects for this age range: 124
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
- infectious enteritis
- Pregnancy in progress
- Patients who are breast-feeding
- Presence of peripheral neuropathy
- Diagnosis of HIV
- Diagnosis of tuberculosis
- Diagnosis of Hepatitis HBV or HCV
- other infections such as sepsis, abscesses, opportunistic infections in progress or other serious diseases not controlled.
- Patients undergone treatment with a live vaccine during the 30 days prior to enrollment in the study.
- Presence of moderate to severe heart failure (Class III / IV NYHA - New York Heart Association).
- Patients with organ transplants
- involvement in other clinical trials



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Crohn's Disease
MedDRA version: 19.0 Level: LLT Classification code 10011402 Term: Crohn's disease (colon) System Organ Class: 100000004856

Therapeutic area: Diseases [C] - Digestive System Diseases [C06]

Intervention(s)

Trade Name: THALIDOMIDE CELGENE - 50 MG CAPSULA RIGIDA - USO ORALE - BLISTER (PVC/PE/ACLAR/ALU) 28 CAPSULE
Pharmaceutical Form: Capsule, hard

Product Name: INFLIXIMAB
Pharmaceutical Form: Powder for infusion

Primary Outcome(s)

Secondary Objective: To assess the efficacy of thalidomide
compared with standard therapy in:
- inducing a clinical response amd remission(12 and 52 weeks)
- reducing the need of therapeutic changes at 12 and 52 weeks
- reducing the number of hospitalizations up to 52 weeks.
- reducing the number of patients who undergo surgery up to 52 weeks
- changing inflammation laboratory indices at 12 and 52 weeks .
- changing nutritional indicators at12 to 52 weeks and bone mineralizaztion at 52 weeks.
To assess the safety of thalidomide compared with standard therapy evaluating the number and type of adverse effects at 12 and
52 weeks.
- direct and indirect costs of thalidomide compared with standard therapy up to 52 weeks
- results from genetic analysis from patient with Crohn’s disease;
- results from immunological analysis from patient with CD;
- information on the mechanisms of action of thalidomide in CD;
- pharmacogenetic data of thalidomide in CD;
- pharmacogenomic data

Timepoint(s) of evaluation of this end point: 52 weeks

Primary end point(s): - number of patient with mucosal healing at 52 weeks of treatment in the two groups.
Mucosal healing will be defined by a SES-CD) = 2

Main Objective: To assess the efficacy of thalidomide compared with standard therapy in inducing mucosal healing and 52 weeks

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: 52 weeks

Secondary end point(s): - number of patient who exit from the protocol and need to modify or increase drug treatment at 12 and 52 weeks
- Number of hospitalization at 52 weeks
- number of patient who need to undergo chirurgic procedures at 52 weeks
- flogosis indexes at 12 and 52 weeks
- body mass index; height-for-age z score; weight-for-age z score
- growth and nutritional indexes at 12 and 52 weeks (body mass index; height-for-age z score; weight-for-age z score)
- Bone mineralization at 52 weeks
- Adverse events at 12 and 52 weeks
- Direct and indirect costs at 52 weeks
assessment of genetics of CD,
immunological mechanisms of CD, mechanisma of action of thalidomide, pharmacokinetics, pharmacogenomics and metabolomics
of thalidomide

Secondary ID(s)

NET-2013-02355002

Source(s) of Monetary Support

Ministry of Health

Ethics review

Status: Approved
Approval date: 22/03/2016
Contact: