Double-blind placebo-controlled clinical trial of thalidomide for chronic granulomatous disease-associated colitis

• Register: JPRN
• Last refreshed on: 17 October 2023
• Main ID: JPRN-jRCT2031200236
• Date of registration: 10/12/2020
• Prospective Registration: No
• Primary sponsor: Kawai Toshinao
• Public title: Double-blind placebo-controlled clinical trial of thalidomide for chronic granulomatous disease-associated colitis
• Scientific title: Double-blind placebo-controlled clinical trial of thalidomide for chronic granulomatous disease-associated colitis
• Date of first enrolment: 28/09/2017
• Target sample size: 8
• Recruitment status: Recruiting
• URL: https://jrct.niph.go.jp/latest-detail/jRCT2031200236
• Study type: Interventional
• Study design: randomized controlled trial, double blind, placebo control, parallel assignment, treatment purpose
• Phase: 2-3

Contacts

• Name: Mayumi Sako
• Affiliation: National Center for Child Heath and Development

• Name: Toshinao Kawai
• Affiliation: National Center for Child Heath and Development

Key inclusion & exclusion criteria

Inclusion criteria: 1) Diagnosis of CGD colitis, ages over one year
2) Moderate CGD colitis (35 /<= PUCAI < 65), or Mild CGD colitis (10 /<= PUCAI <35) carrying bloody stool or over six times of defecation at the onset or relapse
3) Signed informed consent form agreeing to the clinical trial participation
4) Informed consent to compliance rules including administration of investigational agent and contraception
Exclusion criteria: 1) Patients allergic to the drug ingredients
2) Patients with HIV infection
3) Cancer except granuloma
4) Macrophage activating syndrome
5) Any mental problem
6) Pregnant or breast-feeding woman
7) Taking thalidomide or having a history of thalidomide administration
8) Oral, intravenous, or enema administration of steroid within four weeks of providing informed consent
9) Administration of immunomodulator within eight weeks of providing informed consent
10) A history of any biological agent administration
11) Miner who do not have guardian for appropriate administration of investigational agent
12) In certain circumstances that the researchers determined it was not suitable for the research

Age minimum: >= 1age old
Age maximum: Not applicable
Gender: Both

Health Condition(s) or Problem(s) studied

D006105

primary immunodeficiency diseases; inflammatory bowel disease

chronic granulomatous disease-associated colitis
primary immunodeficiency diseases; inflammatory bowel disease

Intervention(s)

D000284

Oral Administration

1) Oral administration of thalidomide (1.5-3mg/kg), once a day
2) Oral administration of placebo (1.5-3mg/kg), once a day

Primary Outcome(s)

Ratio of subjects decreasing more than 20 points of Pediatric ulcerative colitis activity index (PUCAI) or reaching less than 10 of PUCAI score at the end of double-blind trial

Secondary Outcome(s)

Ratio of subjects reaching less than 10 of PUCAI score at the end of double-blind trial
Ratio of subjects decreasing more than one point of physician's global assessment (PGA) at the end of double-blind trial

Secondary ID(s)

UMIN000029324

Secondary Sponsor(s)

Japan Agency for Medical Research and Development

Kawai Toshinao

Ethics review

Status: Approval
Approval date: 15/06/2017
Contact:

[e-mail]

Institutional Review Board of National Center for Child Health and Development

[phone]

[e-mail]