A pilot study for hematopoietic stem cell transplantation with post-transplantation cyclophosphamide and anti-thymoglobulin

JRCT ID: jRCTs031180397

Registered date:20/03/2019

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: Bone marrow failure, immunodeficiency, metabolic error
• Date of first enrollment: 16/04/2017
• Target sample size: 9
• Study type: Interventional

Intervention(s)

Conditioning regimen with anti-thymocyte globulin.
Stem cell transplantation at day 0.
Cyclophosphamide at day 3 and 4.

Outcome(s)

• Primary Outcome: Incidence of events (acute GVHD grade III or more, graft failure, death) by day 30
• Secondary Outcome: GVHD-free survival at day 100, engraftment at day 30, mortality at day 100, incidence of infectious disease at week 8, time from transplantation to onset of a/cGVHD, time from transplantation to death, adverse event

Key inclusion & exclusion criteria

• Age minimum: >=
• Age maximum: < 25age old
• Gender: Both

Include criteria

1) Diagnosed as bone marrow failure, immunodeficiency, or inborn errors of metabolism
2) Fanconi anemia is excluded for bone marrow failure.
3) No HLA 5/6 (A, B, DR) or more matched related donor
4) No HLA 6/6 (A, B, DR) matched unrelated donor
5) Transplantation from 2- or more-antigen mismatched related donor
6) Age at transplantation <25 years
7) ECOG Performance status: 0-2
8) 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
9) Obtained informed consent from a guardian of the patient

Exclude criteria

1. Effusion with grade 2 or worse at CTCAE ver 4.0
2. Uncontrolled infection
3. Severe psychological disorders
4. Pregnant or suspected pregnancy
5. Not eligible for this study at the discretion of the investigator.

Related Information

• Primary Sponsor: Kato Motohiro
• Source(s) of Monetary Support: National Center for Child Health and Development
• Secondary ID(s): UMIN000026159