# Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)

> **NCT04965597** · PHASE2 · COMPLETED · sponsor: **Fred Hutchinson Cancer Center** · enrollment: 40 (actual)

## Conditions studied

- Bone Marrow Failure Syndrome
- Congenital Amegakaryocytic Thrombocytopenia
- Diamond-Blackfan Anemia
- Hereditary Sideroblastic Anemia
- Paroxysmal Nocturnal Hemoglobinuria
- Shwachman-Diamond Syndrome
- Hematologic Neoplasm With Germline GATA2 Mutation
- Hematologic Neoplasm With Germline SAMD9 Mutation
- Hematologic Neoplasm With Germline SAMD9L Mutation

## Interventions

- **DRUG:** Treosulfan
- **DRUG:** Fludarabine Phosphate
- **DRUG:** Tacrolimus
- **DRUG:** Methotrexate
- **BIOLOGICAL:** Lapine T-Lymphocyte Immune Globulin
- **PROCEDURE:** Peripheral Blood Stem Cell Transplantation
- **PROCEDURE:** Allogeneic Bone Marrow Transplantation
- **OTHER:** Quality-of-Life Assessment
- **PROCEDURE:** Echocardiography
- **PROCEDURE:** Multigated Acquisition Scan
- **PROCEDURE:** Bone Marrow Biopsy
- **PROCEDURE:** Bone Marrow Aspiration
- **PROCEDURE:** Biospecimen Collection
- **PROCEDURE:** X-Ray Imaging
- **PROCEDURE:** Computed Tomography

## Key facts

- **NCT ID:** NCT04965597
- **Lead sponsor:** Fred Hutchinson Cancer Center
- **Sponsor class:** OTHER
- **Phase:** PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2022-04-19
- **Primary completion:** 2026-02-18
- **Final completion:** 2026-02-18
- **Target enrollment:** 40 (ACTUAL)
- **Last updated:** 2026-03-09

## Collaborators

- [object Object]
- [object Object]
- [object Object]
- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04965597

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT04965597, "Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)". Retrieved via AI Analytics 2026-06-02 from https://api.ai-analytics.org/clinical/NCT04965597. Licensed CC0.

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