WithdrawnPhase 1

Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases

Stopped: Patients were consented to screening but none were enrolled and proceeded with transplant.

United States0Started 2009-10

Plain-language summary

The purpose of this study is to determine if it is safe to administer unrelated umbilical cord blood to pregnant women in their first trimester of pregnancy with a fetus that has a known diagnosis of certain lysosomal storage diseases. These diseases are known to cause severe and irreversible neurological disability in early infancy and which are lethal in childhood.

Who can participate

Age range

18 Years

Sex

FEMALE

See this in plain English?

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

Inclusion Criteria: * Fetus with known enzymatic and, if possible, mutation diagnosis of selected lysosomal storage diseases, and history of enzymatic or mutation diagnosis of a previously affected family member. * Krabbe disease * Infantile metachromatic leukodystrophy disease (MLD) * Tay Sachs or Sandhoff (GM2 gangliosidoses) disease * Pelizaeus-Merzbacher disease (PMD). * Both parents have the "at risk" genotype * Both parents agree to psychological counseling prior to CVS * Both parents agree to delivery of subject at Duke University * Gestational age of \< 9 weeks by sure menstrual dating or ultrasound of a natural pregnancy. * Singleton fetus * No evidence of abnormal nuchal thickening on preliminary ultrasound prior to chorion villus biopsy Exclusion Criteria: * Pregnancy by in vitro fertilization * Evidence of a nuchal translucency of \> 3.0 mm at the time of the ultrasound for the chorion villus biopsy. * HIV, hepatitis C or hepatitis B positive maternal status * Active infection in the mother at the time of scheduled CVS or cell injection as determined by clinical practice

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

To determine whether immune tolerance and donor cell engraftment can be achieved through first trimester injection of donor cells to fetus's diagnosed with lethal LSDs.

Timeframe: after 3 patients

Safety and feasibility of fetal intrap.

Timeframe: after 3 patients

Trial details

NCT IDNCT01003912

SponsorJoanne Kurtzberg, MD

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2017-03

View on ClinicalTrials.gov More Lysosomal Storage Diseases trials