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

June 23, 2020 updated by: Federal Research Institute of Pediatric Hematology, Oncology and Immunology

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

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Anticipated)

10

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Dmitry Balashov, MD, PhD
  • Phone Number: 6534 +74952876570
  • Email: bala8@yandex.ru

Study Contact Backup

Study Locations

  • Russian Federation
      • Moscow, Russian Federation, 117198
        • Recruiting
        • HSCT department
        • Contact:
          • Dmitry Balashov, MD, PhD
          • Phone Number: 6534 +74952876570
          • Email: bala8@yandex.ru

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

3 months to 21 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: intervention/treatment
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
Drug: Treosulfan
Treosulfan 21mg/m2 (days -6, -5, -4)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Event-free survival
Time Frame: 3 years after HSCT
Events: graft failure, death, malignancies
3 years after HSCT

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Overall survival
Time Frame: 3 years after HSCT
3 years after HSCT
Cumulative incidence of engraftment
Time Frame: 100 days
100 days
Cumulative incidence of graft failure
Time Frame: 3 years
3 years
Cumulative incidence of viral infections
Time Frame: 1 year
1 year
Cumulative incidence of acute graft versus host disease
Time Frame: 1 year
1 year
Cumulative incidence of chronic graft versus host disease
Time Frame: 3 years
3 years
Incidence of early organ toxicity
Time Frame: 100 days
100 days
Cumulative incidence of transplant related mortality
Time Frame: 3 years
3 years
Incidence of long-term toxicity
Time Frame: 3 years
malignancies, non-malignant complications
3 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 22, 2020

Primary Completion (Anticipated)

May 1, 2023

Study Completion (Anticipated)

May 1, 2023

Study Registration Dates

First Submitted

May 19, 2020

First Submitted That Met QC Criteria

May 19, 2020

First Posted (Actual)

May 22, 2020

Study Record Updates

Last Update Posted (Actual)

June 25, 2020

Last Update Submitted That Met QC Criteria

June 23, 2020

Last Verified

June 1, 2020

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NCPHOI-2020-04

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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