Low Dose Treosulfan Based Conditioning Regimen and PTCy in HSCT for Nijmegen Breakage Syndrome
Recruiting
Phase 2
Interventional Study
Nijmegen Breakage Syndrome
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 3 Months – 21
- Sex
- Any
- Trial phase
- Phase 2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 10 (estimated)
- Sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology · Other
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About This Trial
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 Locations
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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
Contacts