Low Dose Treosulfan Based Conditioning Regimen and PTCy in HSCT for Nijmegen Breakage Syndrome

ConditionNijmegen Breakage Syndrome

Trial statusRecruiting

Trial phasePhase 2

Trial typeInterventional

Biological sexAll

Age3-21

SponsorFederal Research Institute of Pediatric Hematology, Oncology and Immunology

About this trial

The aim of the current study is to evaluate the safety and efficacy of low dose treosulfan based conditioning regimen in HSCT with post-transplant cyclophosphamide in Nijmegen breakage syndrome

Eligibility criteria

Qualifiers

Patients aged ≥ 3 months and < 21 years

Patients diagnosed with NBS eligible for an allogeneic HSCT

Signed written informed consent signed by a parent or legal guardian

Disqualifiers

None

Trial design

Treatments tested in this trial

Treosulfan

Treatment groups

10 Participants

are divided into 1 treatment group

Sponsors and collaborators

Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Lead sponsor

Source ClinicalTrials.gov