Umbilical Cord Blood Transfusion in Progeria Syndrome
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|ClinicalTrials.gov Identifier: NCT03871972|
Recruitment Status : Enrolling by invitation
First Posted : March 12, 2019
Last Update Posted : April 23, 2019
|Condition or disease||Intervention/treatment||Phase|
|Hutchinson-Gilford Progeria Syndrome||Drug: Umbilical Cord Blood Unit||Phase 1 Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||2 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Safety and Efficacy of Umbilical Cord Blood Transfusion in Patients With Hutchinson-Gilford Progeria Syndrome|
|Actual Study Start Date :||March 5, 2019|
|Estimated Primary Completion Date :||September 5, 2020|
|Estimated Study Completion Date :||September 5, 2020|
Experimental: UCB injection group
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
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)
Other Name: Oral Sirolimus
- Change from baseline Carotid-femoral pulse wave velocity at 48 weeks [ Time Frame: 48 weeks after UCB infusion ]measured by carotid doppler ultrasonography
- Change from baseline serum HDL cholesterol at 48 weeks [ Time Frame: 48 weeks after UCB infusion ]taken on routine lab
- Change from baseline weight at 48 weeks [ Time Frame: 48 weeks after UCB infusion ]measured by bioimpedance analysis
- Ankle-brachial index [ Time Frame: baseline, 48 weeks after UCB infusion ]measured by automatic blood pressure gauge
- Body fat proportion [ Time Frame: baseline, 48 weeks after UCB infusion ]taken by bioimpedance analysis
- Range of motion [ Time Frame: baseline, 48 weeks after UCB infusion ]measured manually
- height [ Time Frame: baseline, 48 weeks after UCB infusion ]measured by bioimpedance analysis
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03871972
|Korea, Republic of|
|Bundang CHA Medical Center|
|Seongnam-si, Gyeonggi-do, Korea, Republic of, 13496|
|Principal Investigator:||Min Young Kim, MD, PhD||CHA University|