Umbilical Cord Blood Transfusion in Progeria Syndrome

NCT03871972 | Completed Phase 1

• 1 other identifier: 2018-12-031
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

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.

Conditions

Hutchinson-Gilford Progeria Syndrome

Outcome Measures

Primary Outcomes (3)

• Change from baseline Carotid-femoral pulse wave velocity at 48 weeks

  measured by carotid doppler ultrasonography

  48 weeks after UCB infusion

• Change from baseline serum HDL cholesterol at 48 weeks

  taken on routine lab

  48 weeks after UCB infusion

• Change from baseline weight at 48 weeks

  measured by bioimpedance analysis

  48 weeks after UCB infusion

Secondary Outcomes (4)

• Ankle-brachial index

  baseline, 48 weeks after UCB infusion

• Body fat proportion

  baseline, 48 weeks after UCB infusion

• Range of motion

  baseline, 48 weeks after UCB infusion

• height

  baseline, 48 weeks after UCB infusion

Study Arms (1)

UCB injection group

EXPERIMENTAL

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

Interventions

Umbilical Cord Blood Unit DRUG

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)

Also known as: Oral Sirolimus

UCB injection group

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• those who were clinically and genetically diagnosed as Hutchinson-Gilford progeria syndrome

You may not qualify if:

• 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

Sponsors & Collaborators

• Bundang CHA Hospital: lead

Study Sites (1)

Bundang CHA Medical Center

Seongnam-si, Gyeonggi-do, 13496, South Korea

Location

MeSH Terms

Conditions

Progeria

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

Laminopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Study Officials

• Min Young Kim, MD, PhD

  CHA University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Head of Rehabilitation Medicine Department, Professor, Principal Investigator

Study Record Dates

• First Submitted: March 7, 2019
• First Posted: March 12, 2019
• Study Start: March 5, 2019
• Primary Completion: June 1, 2020
• Study Completion: June 1, 2020
• Last Updated: November 4, 2020

Record last verified: 2020-11

Data Sharing

• IPD Sharing: Will not share

Locations

KR (1)