PTCy and and Ruxolitinib for GVHD Prophylaxis After HSCT With Thymoglobulin in Conditioning Regimen in Patients With Inborn Errors of Immunity
Safety and Efficacy of Cyclophosphamide and Ruxolitinib for Graft-versus-host-disease Prophylaxis After Hematopoietic Stem Cell Transplantation With Thymoglobulin Serotherapy in Conditioning Regimen in Patients With Inborn Errors of Immunity
1 other identifier
interventional
100
1 country
1
Brief Summary
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)
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started Nov 2023
Typical duration for phase_2
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
November 21, 2023
CompletedFirst Submitted
Initial submission to the registry
December 28, 2023
CompletedFirst Posted
Study publicly available on registry
January 10, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 21, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 31, 2027
January 10, 2024
December 1, 2023
3 years
December 28, 2023
December 28, 2023
Conditions
Outcome Measures
Primary Outcomes (1)
Event-free survival
Events: graft failure, death
1 year after HSCT
Secondary Outcomes (9)
Overall survival
1 year after HSCT
Cumulative incidence of acute graft versus host disease
1 year after HSCT
Cumulative incidence of chronic graft versus host disease
1 year after HSCT
Cumulative incidence of engraftment
100 days
Cumulative incidence of graft failure
1 year after HSCT
- +4 more secondary outcomes
Study Arms (1)
intervention/treatment
EXPERIMENTALConditioning 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
Interventions
Cyclophosphamide 25mg/kg (days +3, +4) after HSCT from MUD and MRD Cyclophosphamide 50mg/kg (days +3, +4) after HSCT from MMRD
Eligibility Criteria
You may qualify if:
- Patients aged ≥ 0 months and \< 21 years
- Patients diagnosed with NBS eligible for an allogeneic HSCT
- Signed written informed consent signed by a parent or legal guardian
You may not qualify if:
- Concomitant severe somatic disease associated with an additional risk of severe complications
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
HSCT department
Moscow, Russia
MeSH Terms
Interventions
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY CHAIR
Dmitry Balashov, MD, PhD
National Research Center for Pediatric Hematology, Moscow, Russia
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 28, 2023
First Posted
January 10, 2024
Study Start
November 21, 2023
Primary Completion (Estimated)
November 21, 2026
Study Completion (Estimated)
December 31, 2027
Last Updated
January 10, 2024
Record last verified: 2023-12