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

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

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

JPRN

Registry ID

JPRN-UMIN000029324

Enrollment

Registered

2017-10-01

Start date

2017-09-28

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Chronic granulomatous disease associated colitis

Interventions

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

Eligibility

Sex/Gender

All

Inclusion 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

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

Design outcomes

Primary

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

Secondary

Measure	Time frame
1) Ratio of subjects being less than 10 of PUCAI score at the end of double-blind trial 2) Ratio of subjects decreasing more than one point of physician's global assessment (PGA) at the end of double-blind trial	—

Countries

Japan

Contacts

Public ContactMayumi Sako

National Center for Child Heath and Development Division of Child Health and Development

sako-m@ncchd.go.jp03-3416-0181

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026