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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT04400045
Other study ID # NCPHOI-2020-04
Secondary ID
Status Recruiting
Phase Phase 2
First received
Last updated
Start date May 22, 2020
Est. completion date May 2023

Study information

Verified date June 2020
Source Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Contact Dmitry Balashov, MD, PhD
Phone +74952876570
Email bala8@yandex.ru
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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


Description:

Nijmegen breakage syndrome (NBS) is a DNA repair disorder. The only curative option for combine immunodeficiency in NBS is allogeneic hematopoietic stem cell transplantation (HSCT). Standard myeloablative conditioning regimens in DNA repair disorders lead to increased morbidity and mortality after HSCT. Low doses of alkylators are used to reduce toxicity rates, which, however, increase the risks of mixed chimerism and graft failure. The data of treosulfan usage in NBS are sparse. To evaluate the safety and efficacy of low dose treosulfan based conditioning regimen in NBS, treosulfan 21g/m2 in combination with fludarabine 150mg/mg, cyclophosphamide 40mg/kg, thymoglobulin (Genzyme) 5mg/kg and rituximab 100mg/m2 will be used from day -6 to -1 day, followed by stem cell infusion. The primary endpoint is event-free survival, where graft failure, death, and malignancies are considered as events.


Recruitment information / eligibility

Status Recruiting
Enrollment 10
Est. completion date May 2023
Est. primary completion date May 2023
Accepts healthy volunteers No
Gender All
Age group 3 Months to 21 Years
Eligibility Inclusion Criteria:

1. Patients aged = 3 months and < 21 years

2. Patients diagnosed with NBS eligible for an allogeneic HSCT

3. Signed written informed consent signed by a parent or legal guardian

Study Design


Related Conditions & MeSH terms


Intervention

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

Locations

Country Name City State
Russian Federation HSCT department Moscow

Sponsors (1)

Lead Sponsor Collaborator
Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Country where clinical trial is conducted

Russian Federation, 

Outcome

Type Measure Description Time frame Safety issue
Primary Event-free survival Events: graft failure, death, malignancies 3 years after HSCT
Secondary Overall survival 3 years after HSCT
Secondary Cumulative incidence of engraftment 100 days
Secondary Cumulative incidence of graft failure 3 years
Secondary Cumulative incidence of viral infections 1 year
Secondary Cumulative incidence of acute graft versus host disease 1 year
Secondary Cumulative incidence of chronic graft versus host disease 3 years
Secondary Incidence of early organ toxicity 100 days
Secondary Cumulative incidence of transplant related mortality 3 years
Secondary Incidence of long-term toxicity malignancies, non-malignant complications 3 years
See also
  Status Clinical Trial Phase
Recruiting NCT06198842 - Low Dose Treosulfan Based Conditioning Regimen and PTCy in HSCT for Nijmegen Breakage Syndrome Phase 2