Back to resultsUmbilical Cord Blood Transfusion in Progeria Syndrome
Primary Purpose
Hutchinson-Gilford Progeria Syndrome
Status
Completed
Phase
Phase 1
Locations
Korea, Republic of
Study Type
Interventional
Intervention
Umbilical Cord Blood Unit
Sponsored by
About this trial
This is an interventional treatment trial for Hutchinson-Gilford Progeria Syndrome
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
- whose ALT/AST > 2 fold of normal limit
- whose serum creatinine > 1.5 fold of normal limit
- whose total bilirubin > 2 fold of normal limit
- whose total WBC count < 3000/mm3
- 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
Sites / Locations
- Bundang CHA Medical Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
UCB injection group
Arm Description
This pilot study includes only 2 subjects who are enrolled by invitation. Both subjects are included in this single arm.
Outcomes
Primary Outcome Measures
Change from baseline Carotid-femoral pulse wave velocity at 48 weeks
measured by carotid doppler ultrasonography
Change from baseline serum HDL cholesterol at 48 weeks
taken on routine lab
Change from baseline weight at 48 weeks
measured by bioimpedance analysis
Secondary Outcome Measures
Ankle-brachial index
measured by automatic blood pressure gauge
Body fat proportion
taken by bioimpedance analysis
Range of motion
measured manually
height
measured by bioimpedance analysis
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03871972
Brief Title
Umbilical Cord Blood Transfusion in Progeria Syndrome
Official Title
Safety and Efficacy of Umbilical Cord Blood Transfusion in Patients With Hutchinson-Gilford Progeria Syndrome
Study Type
Interventional
2. Study Status
Record Verification Date
November 2020
Overall Recruitment Status
Completed
Study Start Date
March 5, 2019 (Actual)
Primary Completion Date
June 1, 2020 (Actual)
Study Completion Date
June 1, 2020 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Bundang CHA Hospital
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hutchinson-Gilford Progeria Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
2 (Actual)
8. Arms, Groups, and Interventions
Arm Title
UCB injection group
Arm Type
Experimental
Arm Description
This pilot study includes only 2 subjects who are enrolled by invitation. Both subjects are included in this single arm.
Intervention Type
Drug
Intervention Name(s)
Umbilical Cord Blood Unit
Other Intervention Name(s)
Oral Sirolimus
Intervention 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)
Primary Outcome Measure Information:
Title
Change from baseline Carotid-femoral pulse wave velocity at 48 weeks
Description
measured by carotid doppler ultrasonography
Time Frame
48 weeks after UCB infusion
Title
Change from baseline serum HDL cholesterol at 48 weeks
Description
taken on routine lab
Time Frame
48 weeks after UCB infusion
Title
Change from baseline weight at 48 weeks
Description
measured by bioimpedance analysis
Time Frame
48 weeks after UCB infusion
Secondary Outcome Measure Information:
Title
Ankle-brachial index
Description
measured by automatic blood pressure gauge
Time Frame
baseline, 48 weeks after UCB infusion
Title
Body fat proportion
Description
taken by bioimpedance analysis
Time Frame
baseline, 48 weeks after UCB infusion
Title
Range of motion
Description
measured manually
Time Frame
baseline, 48 weeks after UCB infusion
Title
height
Description
measured by bioimpedance analysis
Time Frame
baseline, 48 weeks after UCB infusion
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
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
whose ALT/AST > 2 fold of normal limit
whose serum creatinine > 1.5 fold of normal limit
whose total bilirubin > 2 fold of normal limit
whose total WBC count < 3000/mm3
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Min Young Kim, MD, PhD
Organizational Affiliation
CHA University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Bundang CHA Medical Center
City
Seongnam-si
State/Province
Gyeonggi-do
ZIP/Postal Code
13496
Country
Korea, Republic of
12. IPD Sharing Statement
Plan to Share IPD
No
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Umbilical Cord Blood Transfusion in Progeria Syndrome
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