Trials Sponsors Conditions Drugs Pricing

Conditions

Hutchinson-Gilford Progeria Syndrome

Outcome Measures

Primary Outcomes (3)

Change from baseline Carotid-femoral pulse wave velocity at 48 weeks
measured by carotid doppler ultrasonography
48 weeks after UCB infusion
Change from baseline serum HDL cholesterol at 48 weeks
taken on routine lab
48 weeks after UCB infusion
Change from baseline weight at 48 weeks
measured by bioimpedance analysis
48 weeks after UCB infusion

Secondary Outcomes (4)

Ankle-brachial index
baseline, 48 weeks after UCB infusion
Body fat proportion
baseline, 48 weeks after UCB infusion
Range of motion
baseline, 48 weeks after UCB infusion
height
baseline, 48 weeks after UCB infusion

Study Arms (1)

UCB injection group

EXPERIMENTAL

This pilot study includes only 2 subjects who are enrolled by invitation. Both subjects are included in this single arm.

Drug: Umbilical Cord Blood Unit

Interventions

Umbilical Cord Blood UnitDrug

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)

Also known as: Oral Sirolimus

UCB injection group

Eligibility Criteria

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

Sponsors & Collaborators

Bundang CHA Hospital
Otherlead

Study Sites (1)

Bundang CHA Medical Center

Seongnam-si, Gyeonggi-do, 13496, South Korea

MeSH Terms

Conditions

Progeria

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

LaminopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolism, Inborn ErrorsMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

MacrolidesLactonesOrganic Chemicals

Study Officials

Min Young Kim, MD, PhD
CHA University
PRINCIPAL INVESTIGATOR

Enrollment

2 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: phase 1 2
Allocation: NA
Masking: NONE
Purpose: TREATMENT
Intervention Model: SINGLE GROUP
Sponsor Type: OTHER
Responsible Party: PRINCIPAL INVESTIGATOR
PI Title: Head of Rehabilitation Medicine Department, Professor, Principal Investigator

Study Record Dates

First Submitted

March 7, 2019

First Posted

March 12, 2019

Study Start

March 5, 2019

Actual (reported)

Umbilical Cord Blood Transfusion in Progeria Syndrome

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (3)

Secondary Outcomes (4)

Study Arms (1)

UCB injection group

Interventions

Eligibility Criteria

You may qualify if:

You may not qualify if:

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Intervention Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

Data Sharing

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (3)

Secondary Outcomes (4)

Study Arms (1)

UCB injection group

Interventions

Eligibility Criteria

You may qualify if:

You may not qualify if:

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Intervention Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

Data Sharing

Source record

Locations