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Umbilical Cord Blood Transfusion in Progeria Syndrome

NCT03871972 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 2

Last updated 2020-11-04

No results posted yet for this study

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.

Conditions

  • Hutchinson-Gilford Progeria Syndrome

Interventions

DRUG

Umbilical Cord Blood Unit

3 infusions of umbilical cord blood (UCB) unit (TNC \> 2.0ⅹ107cells/kg) each 4 months apart and take oral Sirolimus (1 mg/m2/day) for 7 days (from 3 days before UCB infusion until 3 days after UCB infusion)

Sponsors & Collaborators

  • Bundang CHA Hospital

    lead OTHER

Principal Investigators

  • Min Young Kim, MD, PhD · CHA University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-03-05
Primary Completion
2020-06-01
Completion
2020-06-01

Countries

  • South Korea

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03871972 on ClinicalTrials.gov