Umbilical Cord Blood Transfusion in Progeria Syndrome

Hutchinson-Gilford Progeria Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03871972
• Other study ID #: 2018-12-031
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: March 5, 2019
• Est. completion date: June 1, 2020

Study information

• Verified date: November 2020
• Source: Bundang CHA Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 2
• Est. completion date: June 1, 2020
• Est. primary completion date: June 1, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Hutchinson-Gilford Progeria Syndrome
• Progeria
• Syndrome

Intervention

Drug:
• Umbilical Cord Blood Unit
3 infusions of umbilical cord blood (UCB) unit (TNC > 2.0?107cells/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)

Locations

Country	Name	City	State
Korea, Republic of	Bundang CHA Medical Center	Seongnam-si	Gyeonggi-do

Sponsors (1)

Lead Sponsor	Collaborator
Bundang CHA Hospital

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline Carotid-femoral pulse wave velocity at 48 weeks	measured by carotid doppler ultrasonography	48 weeks after UCB infusion
Primary	Change from baseline serum HDL cholesterol at 48 weeks	taken on routine lab	48 weeks after UCB infusion
Primary	Change from baseline weight at 48 weeks	measured by bioimpedance analysis	48 weeks after UCB infusion
Secondary	Ankle-brachial index	measured by automatic blood pressure gauge	baseline, 48 weeks after UCB infusion
Secondary	Body fat proportion	taken by bioimpedance analysis	baseline, 48 weeks after UCB infusion
Secondary	Range of motion	measured manually	baseline, 48 weeks after UCB infusion
Secondary	height	measured by bioimpedance analysis	baseline, 48 weeks after UCB infusion