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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
Sponsor:
Information provided by (Responsible Party):
MinYoung Kim, MD, PhD, 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.

Condition or disease Intervention/treatment Phase
Hutchinson-Gilford Progeria Syndrome Drug: Umbilical Cord Blood Unit Phase 1 Phase 2

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.


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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 2 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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


Arm Intervention/treatment
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




Primary Outcome Measures :
  1. Change from baseline Carotid-femoral pulse wave velocity at 48 weeks [ Time Frame: 48 weeks after UCB infusion ]
    measured by carotid doppler ultrasonography

  2. Change from baseline serum HDL cholesterol at 48 weeks [ Time Frame: 48 weeks after UCB infusion ]
    taken on routine lab

  3. Change from baseline weight at 48 weeks [ Time Frame: 48 weeks after UCB infusion ]
    measured by bioimpedance analysis


Secondary Outcome Measures :
  1. Ankle-brachial index [ Time Frame: baseline, 48 weeks after UCB infusion ]
    measured by automatic blood pressure gauge

  2. Body fat proportion [ Time Frame: baseline, 48 weeks after UCB infusion ]
    taken by bioimpedance analysis

  3. Range of motion [ Time Frame: baseline, 48 weeks after UCB infusion ]
    measured manually

  4. height [ Time Frame: baseline, 48 weeks after UCB infusion ]
    measured by bioimpedance analysis



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

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


Locations
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Korea, Republic of
Bundang CHA Medical Center
Seongnam-si, Gyeonggi-do, Korea, Republic of, 13496
Sponsors and Collaborators
Bundang CHA Hospital
Investigators
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Principal Investigator: Min Young Kim, MD, PhD CHA University

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Responsible Party: MinYoung Kim, MD, PhD, Head of Rehabilitation Medicine Department, Professor, Principal Investigator, Bundang CHA Hospital
ClinicalTrials.gov Identifier: NCT03871972     History of Changes
Other Study ID Numbers: 2018-12-031
First Posted: March 12, 2019    Key Record Dates
Last Update Posted: April 23, 2019
Last Verified: April 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Syndrome
Progeria
Disease
Pathologic Processes
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Sirolimus
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs