Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases
Withdrawn before enrolling · Phase 1
Conditions studied: Lysosomal Storage Diseases, Inborn Errors of Metabolism
In brief
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.
Key facts
- Study ID
- NCT01003912
- Run by
- Joanne Kurtzberg, MD
- People needed
- 0
- Starts
- 2009-10-01
- Expected to finish
- 2017-03-01
- Last updated by the study team
- 2017-06-29
Who can join
Age: 18 and older. Sex: female. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- 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
Where it is running
- Duke University Medical Center Pediatric Blood and Marrow Transplant — Durham, North Carolina, United States
- Duke University Medical Center — Durham, North Carolina, United States
Full record on ClinicalTrials.gov
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