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Trial record 4 of 9 for:    progeria

Umbilical Cord Blood Transfusion in Progeria Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03871972
Recruitment Status : Completed
First Posted : March 12, 2019
Last Update Posted : November 4, 2020
Sponsor:
Information provided by (Responsible Party):
MinYoung Kim, MD, PhD, Bundang CHA Hospital

Tracking Information
First Submitted Date  ICMJE March 7, 2019
First Posted Date  ICMJE March 12, 2019
Last Update Posted Date November 4, 2020
Actual Study Start Date  ICMJE March 5, 2019
Actual Primary Completion Date June 1, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 11, 2019)
  • 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
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 11, 2019)
  • 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
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Umbilical Cord Blood Transfusion in Progeria Syndrome
Official Title  ICMJE Safety and Efficacy of Umbilical Cord Blood Transfusion in Patients With Hutchinson-Gilford Progeria Syndrome
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Hutchinson-Gilford Progeria Syndrome
Intervention  ICMJE 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
Study Arms  ICMJE Experimental: UCB injection group
This pilot study includes only 2 subjects who are enrolled by invitation. Both subjects are included in this single arm.
Intervention: Drug: Umbilical Cord Blood Unit
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: March 11, 2019)
2
Original Actual Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE June 1, 2020
Actual Primary Completion Date June 1, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Korea, Republic of
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03871972
Other Study ID Numbers  ICMJE 2018-12-031
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party MinYoung Kim, MD, PhD, Bundang CHA Hospital
Study Sponsor  ICMJE Bundang CHA Hospital
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Min Young Kim, MD, PhD CHA University
PRS Account Bundang CHA Hospital
Verification Date November 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP