NCT06198842

Brief Summary

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

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for phase_2

Timeline
33mo left

Started Nov 2023

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress47%
Nov 2023Jan 2029

Study Start

First participant enrolled

November 22, 2023

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

December 27, 2023

Completed
14 days until next milestone

First Posted

Study publicly available on registry

January 10, 2024

Completed
3.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 31, 2027

Expected
2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

January 31, 2029

Last Updated

January 10, 2024

Status Verified

December 1, 2023

Enrollment Period

3.2 years

First QC Date

December 27, 2023

Last Update Submit

December 27, 2023

Conditions

Nijmegen Breakage Syndrome

Outcome Measures

Primary Outcomes (1)

  • Event-free survival

    Events: graft failure, death, malignancies

    3 years after HSCT

Secondary Outcomes (9)

  • Overall survival

    3 years after HSCT

  • Cumulative incidence of engraftment

    100 days

  • Cumulative incidence of graft failure

    3 years

  • Cumulative incidence of viral infections

    1 year

  • Cumulative incidence of acute graft versus host disease

    1 year

  • +4 more secondary outcomes

Study Arms (1)

intervention/treatment

EXPERIMENTAL

Fludarabine 150mg/m2 (days -6, -5, -4, -3, -2) Treosulfan 21g/m2 (days -6, -5, -4) Thymoglobulin (Genzyme) 5mg/kg (days -5, -4) Rituximab 100mg/m2 (day -1) Cyclophosphamide 50mg/kg (days +3, +4)

Drug: Treosulfan

Interventions

TreosulfanDRUG

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

intervention/treatment

Eligibility Criteria

Age3 Months - 21 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

HSCT department

Moscow, Russia

RECRUITING

MeSH Terms

Conditions

Nijmegen Breakage Syndrome

Interventions

treosulfan

Condition Hierarchy (Ancestors)

DNA Repair-Deficiency DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Central Study Contacts

Dmitry Balashov, MD, PhD

CONTACT

+74956647091bala8@yandex.ru

Alexandra Laberko, MD, PhD

CONTACT

+74952876570alexandra.laberko@gmail.com

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 27, 2023

First Posted

January 10, 2024

Study Start

November 22, 2023

Primary Completion (Estimated)

January 31, 2027

Study Completion (Estimated)

January 31, 2029

Last Updated

January 10, 2024

Record last verified: 2023-12

Locations

RU(1)