drugset / Trial / NCT01917708
Bone Marrow Transplant With Abatacept for Non-Malignant Diseases
NaSingle-groupOpen-labelSupportive care
Summary
This is a single arm, phase I study to assess the tolerability of abatacept when combined with cyclosporine and mycophenolate mofetil as graft versus host disease prophylaxis in children undergoing unrelated hematopoietic stem cell transplant for serious non-malignant diseases as well as to assess the immunological effects of abatacept. Participants will be followed for 2 years.
Timeline
- Start
- 2014-01
- Primary completion
- 2019-09-19
- Completion
- 2019-09-19
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Abatacept | Other / unclassified | 10 mg/kg | Intravenous |
Indications
Chediak-Higashi syndrome
Diamond-Blackfan anemia
Fanconi anemia
Glanzmann thrombasthenia
Hurler syndrome
Shwachman-Diamond syndrome
Wiskott-Aldrich syndrome
beta-thalassemia major
chronic granulomatous disease
dyskeratosis congenita
leukocyte adhesion deficiency
severe congenital neutropenia
sickle cell disease