Umbilical Cord Blood Transfusion in Progeria Syndrome

Safety and Efficacy of Umbilical Cord Blood Transfusion in Patients With Hutchinson-Gilford Progeria Syndrome

Sponsors

Lead Sponsor: Bundang CHA Hospital

Source Bundang CHA Hospital
Brief 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.

Detailed Description

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.

Overall Status Completed
Start Date 2019-03-05
Completion Date 2020-06-01
Primary Completion Date 2020-06-01
Phase Phase 1/Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
Change from baseline Carotid-femoral pulse wave velocity at 48 weeks 48 weeks after UCB infusion
Change from baseline serum HDL cholesterol at 48 weeks 48 weeks after UCB infusion
Change from baseline weight at 48 weeks 48 weeks after UCB infusion
Secondary Outcome
Measure Time Frame
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
Enrollment 2
Condition
Intervention

Intervention Type: Drug

Intervention Name: Umbilical Cord Blood Unit

Description: 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)

Arm Group Label: UCB injection group

Other Name: Oral Sirolimus

Eligibility

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

Gender:

All

Minimum Age:

N/A

Maximum Age:

N/A

Healthy Volunteers:

No

Overall Official
Last Name Role Affiliation
Min Young Kim, MD, PhD Principal Investigator CHA University
Location
Facility: Bundang CHA Medical Center
Location Countries

Korea, Republic of

Verification Date

2020-11-01

Responsible Party

Type: Principal Investigator

Investigator Affiliation: Bundang CHA Hospital

Investigator Full Name: MinYoung Kim, MD, PhD

Investigator Title: Head of Rehabilitation Medicine Department, Professor, Principal Investigator

Has Expanded Access No
Condition Browse
Number Of Arms 1
Arm Group

Label: UCB injection group

Type: Experimental

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

Patient Data No
Study Design Info

Allocation: N/A

Intervention Model: Single Group Assignment

Primary Purpose: Treatment

Masking: None (Open Label)

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