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Umbilical Cord Blood Transfusion in Progeria Syndrome

NCT ID: NCT03871972

Last Updated: 2020-11-04

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

2 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-03-05

Study Completion Date

2020-06-01

Brief Summary

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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.

Detailed Description

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This is a pilot study on safety and efficacy of umbilical cord blood therapy for patients with Hutchinson Gilford Progeria syndrome (HGPS). HGPS is a rare genetic disease where affected LMNA gene coding lamin A protein leads to premature aging and early death.

Teenagers with HGPS are in high risk of atherosclerosis and ischemic stroke, and these are major reason of mortality in HGPS.Currently, there are no definite cure for this rare genetic disease. Among the potential drugs under investigation, Lornafarnib (farnesyltransferase inhibitor) lowered the carotid-femoral pulse wave velocity (cfPWV) and also lowered mortality.

Stem cell therapy has proven its efficacy in progeria mouse model. We are trying to study safety and efficacy of umbilical cord blood therapy in human HGPS patients.

Conditions

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Hutchinson-Gilford Progeria Syndrome

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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UCB injection group

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

Group Type EXPERIMENTAL

Umbilical Cord Blood Unit

Intervention Type DRUG

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)

Interventions

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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)

Intervention Type DRUG

Other Intervention Names

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Oral Sirolimus

Eligibility Criteria

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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
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Bundang CHA Hospital

OTHER

Sponsor Role lead

Responsible Party

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MinYoung Kim, MD, PhD

Head of Rehabilitation Medicine Department, Professor, Principal Investigator

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Min Young Kim, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

CHA University

Locations

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Bundang CHA Medical Center

Seongnam-si, Gyeonggi-do, South Korea

Site Status

Countries

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South Korea

Other Identifiers

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2018-12-031

Identifier Type: -

Identifier Source: org_study_id