NCT04400045

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

Trial Health

43
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Enrollment
10

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started May 2020

Typical duration for phase_2

Geographic Reach
1 country

1 active site

Status
unknown

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

First Submitted

Initial submission to the registry

May 19, 2020

Completed
3 days until next milestone

First Posted

Study publicly available on registry

May 22, 2020

Completed
Same day until next milestone

Study Start

First participant enrolled

May 22, 2020

Completed
2.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2023

Completed
Last Updated

June 25, 2020

Status Verified

June 1, 2020

Enrollment Period

2.9 years

First QC Date

May 19, 2020

Last Update Submit

June 23, 2020

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) 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

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, 117198, 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

+74952876570bala8@yandex.ru

Alexandra Laberko, MD

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

May 19, 2020

First Posted

May 22, 2020

Study Start

May 22, 2020

Primary Completion

May 1, 2023

Study Completion

May 1, 2023

Last Updated

June 25, 2020

Record last verified: 2020-06

Locations

RU(1)