A phase II study of RIC-SCT for CGD

• Register: JPRN
• Last refreshed on: 17 October 2023
• Main ID: JPRN-jRCTs031180398
• Date of registration: 20/03/2019
• Prospective Registration: No
• Primary sponsor: Sakaguchi Hirotoshi
• Public title: A phase II study of RIC-SCT for CGD
• Scientific title: Reduced intensity conditioning allogeneic stem cell transplantation with dose-adjusted busulfan and anti-thymocyte globulin for chronic granulomatous disease: a multicenter phase II trial - CGD-RIST2
• Date of first enrolment: 30/05/2018
• Target sample size: 22
• Recruitment status: Recruiting
• URL: https://jrct.niph.go.jp/latest-detail/jRCTs031180398
• Study type: Interventional
• Study design: single arm study, open(masking not used), no treatment control/standard of care control, single assignment, treatment purpose
• Phase: 2

Countries of recruitment

Japan, None (Japan only)

Contacts

• Contact type: Public
• Name: Hirotoshi Sakaguchi
• Affiliation: National Center for Child Health and Development

• Contact type: Scientific
• Name: Hirotoshi Sakaguchi
• Affiliation: National Center for Child Health and Development

Key inclusion & exclusion criteria

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.

Age minimum: Not applicable
Age maximum: < 25age old
Gender: Both

Health Condition(s) or Problem(s) studied

D006105

Chronic granulomatous disease
Primary immunodeficiency

Primary immunodeficiency

Intervention(s)

Conditioning regimen with targeted-busulfan and fludarabin
Total body irradiation (3Gy) at day -1
Stem cell transplantation at day 0

Primary Outcome(s)

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

Secondary Outcome(s)

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

Secondary ID(s)

UMIN000030647

Source(s) of Monetary Support

National Center for Child Health and Development

Ethics review

• Status: Approval
• Approval date: 26/07/2018
• Contact name: [e-mail]
• Contact address: Certified Review Board of National Center for Child Health and Development
• Contact telephone: [phone]
• Contact email: [e-mail]