Umbilical Cord Blood Transfusion in Progeria Syndrome

• Status: Completed
• Trial end date: 2020-06-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Bundang CHA Hospital
• Treatments: Sirolimus

Criteria

This is a pilot study including 2 patients with HGPS.

Inclusion Criteria:

• those who were clinically and genetically diagnosed as Hutchinson-Gilford progeria syndrome

Exclusion Criteria:

• those who show definite hemorrhage or ischemia on brain MRI

• those who are affected with systemic infection during study enrolling period

• those who are not able to able to make consents to the study; those who are not accompanying any guardians

• those who were enrolled in other clinical trials within last 30 days

• those who are not appropriate according to laboratory criteria

1. whose ALT/AST > 2 fold of normal limit

2. whose serum creatinine > 1.5 fold of normal limit

3. whose total bilirubin > 2 fold of normal limit

4. whose total WBC count < 3000/mm3

5. whose platelet count < normal lower limit

• those who are diagnosed with other malignancies

• those who are affected by other serious medical (cardiopulmonary, gastrointestinal, endocrinologic, etc.) conditions