Study of Treosulfan-Based Conditioning for HSCT in Nijmegen Breakage Syndrome
NCT ID: NCT07316595
Last Updated: 2026-01-05
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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NOT_YET_RECRUITING
PHASE2
24 participants
INTERVENTIONAL
2025-12-30
2030-12-31
Brief Summary
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Due to the increased sensitivity of cells in patients with Nijmegen breakage syndrome to alkylating drugs, the use of standard myeloablative conditioning regimens for this disease significantly increases the risks of toxic complications and transplant-related mortality.
Treosulfan is an alkylating agent that has demonstrated efficacy with comparatively low risks of toxic complications when used as part of conditioning prior to allogeneic HSCT for various diseases in patients of all age groups. There is currently experience using treosulfan in patients with Nijmegen breakage syndrome at reduced doses (21 and 30 g/m²). However, a number of questions remain unresolved. Based on our previous experience, a dose of 21 g/m² is sufficient for patients with Nijmegen breakage syndrome without a malignant disease, as it ensures good graft function (a high probability of full donor chimerism and control of the immunodeficiency). At the same time, there is reason to believe that this dose is insufficient to provide an antitumor effect from the conditioning.
We are planning a multicenter study to investigate treosulfan-based conditioning in patients with Nijmegen breakage syndrome, which will stratify patients based on the presence or absence of malignant disease. Patients without a tumor will receive treosulfan at a dose of 21 g/m², and patients with a tumor will receive 30 g/m².
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Patients with Nijmegen breakage syndrome and malignant disease.
Treosulfan (Treo)
We are planning a multicenter study to investigate treosulfan-based conditioning in patients with Nijmegen breakage syndrome, which will stratify patients based on the presence or absence of malignant disease. Patients without a tumor will receive treosulfan at a dose of 21 g/m², and patients with a tumor will receive 30 g/m²
Patients with Nijmegen breakage syndrome without malignant disease.
Treosulfan (Treo)
We are planning a multicenter study to investigate treosulfan-based conditioning in patients with Nijmegen breakage syndrome, which will stratify patients based on the presence or absence of malignant disease. Patients without a tumor will receive treosulfan at a dose of 21 g/m², and patients with a tumor will receive 30 g/m²
Interventions
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Treosulfan (Treo)
We are planning a multicenter study to investigate treosulfan-based conditioning in patients with Nijmegen breakage syndrome, which will stratify patients based on the presence or absence of malignant disease. Patients without a tumor will receive treosulfan at a dose of 21 g/m², and patients with a tumor will receive 30 g/m²
Eligibility Criteria
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Inclusion Criteria
* availability of informed consent for study participation, signed by the patient (ages 14 to 21) and/or their legal representative (ages 0 to 18).
* absence of contraindications to HSCT based on the patient's somatic status.
Exclusion Criteria
21 Years
ALL
No
Sponsors
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Russian Children's Clinical Hospital of the N.I. Pirogov Russian National Research Medical University
UNKNOWN
Regional Children's Clinical Hospital, Yekaterinburg
UNKNOWN
Morozov Children's Municipal Clinical Hospital of the Moscow City Health Department State-Financed H
OTHER_GOV
Raisa Gorbacheva Memorial Research Institute for Pediatric Oncology, Hematology and Transplantation
UNKNOWN
Federal Research Institute of Pediatric Hematology, Oncology and Immunology
OTHER
Responsible Party
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Central Contacts
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Other Identifiers
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NCHPOI- 2025-9
Identifier Type: -
Identifier Source: org_study_id
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