drugset / Trial / NCT03871972
Umbilical Cord Blood Transfusion in Progeria Syndrome
NaSingle-groupOpen-labelTreatment
Summary
This is a pilot study on safety and efficacy of umbilical cord blood therapy for patients with Hutchinson Gilford Progeria syndrome (HGPS). This is an 1 year trial with 3 IV infusions (4 months apart from each infusion) of umbilical cord blood units with oral Sirolimus to see the safety and efficacy.
Timeline
- Start
- 2019-03-05
- Primary completion
- 2020-06-01
- Completion
- 2020-06-01
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Umbilical Cord Blood Unit | Cell therapy | 2e+07 cells/kg | — |
| Background | Sirolimus | Small molecule | 1 mg/m2 | Oral |