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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

This trial does not accept healthy volunteers

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

Design model

Single group

Treatments tested in this trial

  • Treosulfan

    Drug

    Treosulfan 21mg/m2 (days -6, -5, -4)

Treatment groups

10 Participants
are divided into 1 treatment group
Group A: intervention/treatmentExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Event-free survival

Events: graft failure, death, malignancies

Time frame
3 years after HSCT

Secondary outcomes

1

Overall survival

Time frame
3 years after HSCT
2

Cumulative incidence of engraftment

Time frame
100 days
3

Cumulative incidence of graft failure

Time frame
3 years
4

Cumulative incidence of viral infections

Time frame
1 year

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Lead sponsor

Source ClinicalTrials.gov
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