CompletedPhase 1/2

Umbilical Cord Blood Transfusion in Progeria Syndrome

South Korea2 participantsStarted 2019-03-05

Plain-language 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.

Who can participate

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Exclusion 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

What they're measuring

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

Timeframe: 48 weeks after UCB infusion

Change from baseline serum HDL cholesterol at 48 weeks

Timeframe: 48 weeks after UCB infusion

Change from baseline weight at 48 weeks

Timeframe: 48 weeks after UCB infusion

Trial details

NCT IDNCT03871972

SponsorBundang CHA Hospital

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2020-06-01

View on ClinicalTrials.gov More Hutchinson-Gilford Progeria Syndrome trials