PTCy and and Ruxolitinib for GVHD Prophylaxis After HSCT With Thymoglobulin in Conditioning Regimen in Patients With Inborn Errors of Immunity

Sponsor
Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Study ID
NCT06199427
Phase
PHASE2
Status
Recruiting
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Conditions

  • Inborn Errors of Immunity

Eligibility Criteria

Sex
ALL
Age
0 Months - 21 Years
Healthy Volunteers
Not accepted

Interventions

  • Cyclophosphamide — DRUG
    Cyclophosphamide 25mg/kg (days +3, +4) after HSCT from MUD and MRD Cyclophosphamide 50mg/kg (days +3, +4) after HSCT from MMRD
  • Ruxolitinib — DRUG
    Ruxolitinib 7 mg/m2 from day +5 after HSCT

Study Details

The aim of the current study is to evaluate the efficacy of combined regimen of GVHD prophylaxis with thymoglobulin in conditioning regimen and PTCY with ruxolitinib used after HSCT in patients with inborn errors of immunity (IEI)

Key Dates

Start date
Nov 21, 2023
Status verified
Dec 2023
Primary completion
Nov 21, 2026
Completion
Dec 31, 2027

Study Design

Enrollment
100 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: intervention/treatment
    Conditioning regimen containing treosulfan 30-42 g/m2, fludarabine 150 mg/mg, thymoglobulin 5 mg/kg and thiotepa 10 mg/kg or melphalan 140 mg/m2 GVHD prophylaxis regimen for matched unrelated (MUD) and matched related donors (MRD): Cyclophosphamide (PTCY) 25 mg/kg/day (days +3, +4) Ruxolitinib 7 mg/m2 from day +5 GVHD prophylaxis regimen for mismatched related donor (MMRD): Cyclophosphamide (PTCY) 50 mg/kg/day (days +3, +4) Ruxolitinib 7 mg/m2 from day +5

Primary Outcome Measure

Event-free survival [ Time Frame: 1 year after HSCT ]

Central Contacts