A phase II study of RIC-SCT for CGD

Phase 2

Recruiting

• Conditions: Chronic granulomatous disease; Primary immunodeficiency; D006105

• Registration Number: JPRN-jRCTs031180398
• Lead Sponsor: Sakaguchi Hirotoshi

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-03-20
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 22

Inclusion Criteria

1) Diagnosed as chronic granulomatous disease
2) Planned to have stem cell transplantation
3) Donor for transplantation is one of the follows;
a. HLA 5/6 (A, B, DR) or more matched related donor
b. HLA 5/6 (A, B, DR) or more matched unrelated donor
4) Age at transplantation <25 years
5) ECOG Performance status: 0-2
6) Sufficient organ function, as follows;
a. T-Bil <= 1.5 mg/dl
b. Serum creatinine <= 0.8 mg/dl (age <5y), <= 1.2 mg/dl (age 5-9y), <= 1.5 mg/dl (10y or older)
c. Ejection fraction 45% or better, and QTfc <0.45 sec
7) Obtained informed consent from a guardian of the patient

Exclusion Criteria

1. Intracranial hemorrhage
2. Severe psychological disorders
3. Pregnant or suspected pregnancy
4. Receiving interferon gamma therapy within 4 weeks of the start of conditioning therapy
5. Not eligible for this study at the discretion of the investigator.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Event (graft failure/death) at day 100 after transplantation

Secondary Outcome Measures

Name	Time	Method
1. Engraftment probability at day 30 <br>2. Mortality at day 100 <br>3. Incidence of infectious disease at week 8 <br>4. Reconstruction of immune status<br>5. Time from transplantation to onset of acute GVHD <br>6. Reconstruction of neutrophil function