ATG-based HLA haploidentical stem cell transplantation (HCM regimen) for recurrent or refractory round-cell-type sarcoma in AYA generation: prospective phase II study

ATG-based HLA haploidentical stem cell transplantation (HCM regimen) for recurrent or refractory round-cell-type sarcoma in AYA generation: prospective phase II study - ATG-based HLA haploidentical stem cell transplantation (HCM regimen) for recurrent or refractory round-cell-type sarcoma in AYA generation

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

JPRN

Registry ID

JPRN-UMIN000037944

Enrollment

Registered

2019-10-01

Start date

2019-10-10

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

round-cell-type sarcoma

Interventions

The basic structure of the conditioning regimen was applied from our haploidentical stem cell transplantation (Hyogo regimen) for hematological malignancies, consisting of fludarabine at 30 mg/m2/day

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1) Recurrent or refractory rhabdomyosarcoma with alveolar or pleomorphic type 2) Recurrent or refractory Ewing family sarcoma 3) desmoplastic small round cell tumor (any stage)

Exclusion criteria

Exclusion criteria: 1) serum Cre >2.0 mg/dl 2) serum T-bil >2.0 mg/dl 3) serum AST and/or ALT >200 mg/dl 4) EF <40% by ultracardiograpgy 5) SpO2 <90% in ambient air 6) PS (ECOG score) 3-4 Not always excluded if data are elevated due to original disease and are expected to improve by treatment

Design outcomes

Primary

Measure	Time frame
Median survival time (MST) in registration period (8 years) and follow-up period (2 years) If the lower limit of 95% CI of the MST is over 0.82 years, which is the MST of relapsed rhabdomyosarcoma (JCO. 1999; 17: 3487-3493), this study is defined as "positive study".	—

Secondary

Measure	Time frame
Safety 1) Engraftment rate on day 35. Engraftment is defined as neutrophil > 500/microL with >90% donor origin. Rejection is defined when engraftment is not achieved within day 35 after transplantation. 2) Acute GVHD within day 100 3) Complications including thrombotic microangiopathy (TMA) 4) Other adverse events assessed using CTCAE Efficacy 5) Response rate assessed using iRECIST. Assessment using RECIST ver. 1.1 is also documented for comparizon with other studies 6) Relapse rate 7) Disease-free survival (DFS) or progression-free survival (PFS)	—

Measure

Time frame

Safety 1) Engraftment rate on day 35. Engraftment is defined as neutrophil > 500/microL with >90% donor origin. Rejection is defined when engraftment is not achieved within day 35 after transplantation. 2) Acute GVHD within day 100 3) Complications including thrombotic microangiopathy (TMA) 4) Other adverse events assessed using CTCAE Efficacy 5) Response rate assessed using iRECIST. Assessment using RECIST ver. 1.1 is also documented for comparizon with other studies 6) Relapse rate 7) Disease-free survival (DFS) or progression-free survival (PFS)

—

Countries

Japan

Contacts

Public ContactKazuhiro Ikegame

Hyogo College of Medicine Division of Hematology, Department of Internal Medicine

haplo@hyo-med.ac.jp0798456886

Outcome results

None listed

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