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

Brief Title

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

Official Title

Clinical Open-label Phase 2 Study of Low Dose Treosulfan Based Conditioning Regimen Efficacy in Hematopoietic Stem Cell Transplantation for Children With Nijmegen Breakage Syndrome

Brief 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
    

Detailed 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.
    

Study Phase

Phase 2

Study Type

Interventional

Primary Outcome

Event-free survival

Secondary Outcome

 Overall survival

Condition

Nijmegen Breakage Syndrome

Intervention

Treosulfan

Study Arms / Comparison Groups

 intervention/treatment

Description:  Fludarabine 150mg/m2 (days -6, -5, -4, -3, -2) Treosulfan 21g/m2 (days -6, -5, -4) Cyclophosphamide 40mg/kg (days -3, -2) Thymoglobulin (Genzyme) 5mg/kg (days -5, -4) Rituximab 100mg/m2 (day -1) Stem cell infusion - day 0

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Status

Drug

Estimated Enrollment

10

Start Date

May 22, 2020

Completion Date

May 2023

Primary Completion Date

May 2023

Eligibility Criteria

        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
      

Gender

All

Ages

3 Months - 21 Years

Accepts Healthy Volunteers

No

Contacts

, +74952876570, [email protected]

Location Countries

Russian Federation

Location Countries

Russian Federation

NCT ID

NCT04400045

Organization ID

NCPHOI-2020-04

Responsible Party

Sponsor

Study Sponsor

Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Study Sponsor

, , 

Verification Date

June 2020