drugset / Trial / NCT01003912
Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases
Non-randomizedSingle-groupOpen-labelTreatment
Summary
The purpose of this study is to determine if it is safe to administer unrelated umbilical cord blood to pregnant women in their first trimester of pregnancy with a fetus that has a known diagnosis of certain lysosomal storage diseases. These diseases are known to cause severe and irreversible neurological disability in early infancy and which are lethal in childhood.
Timeline
- Start
- 2009-10
- Primary completion
- 2017-03
- Completion
- 2017-03
Outcome
Outcome not reported
Stopped (Enrollment): “Patients were consented to screening but none were enrolled and proceeded with transplant.”
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | ALD-601 | Cell therapy | 100000 cells/kg | Other |
| Subject | ALD-601 | Cell therapy | 2e+06 cells/kg | Other |