Clinical Trial Goal
To find out if allogeneic BMT with DUOC-01 is safe and works well to treat inherited metabolic diseases
You may be able to join this trial if you:
Your child:
- Is 21 years old or younger
- Has one of the following diseases:
- Adrenoleukodystrophy
- Batten disease
- Hunter syndrome (MPS II)
- Krabbe disease (globoid leukodystrophy)
- Metachromatic leukodystrophy (MLD)
- Niemann Pick disease type A
- Niemann Pick disease type B
- Pelizaeus-Merzbacher disease (PMD)
- Sandhoff disease
- Tay Sachs disease
- Alpha mannosidosis
- Sanfilippo (MPS III)
- Has not been treated with gene therapy before
- Does not have a brother or sister who is a match and can be their donor
- Has not had a BMT in the last 3 years
You agree to have other standard tests done to see if your child can be in the clinical trial
Trial Details
DUOC-01 are cells made from donated umbilical cord blood. Doctors think DUOC-01 may help people with metabolic diseases by treating the disease in the brain and spinal cord.
Your child will get standard drugs to prepare their body for transplant. On transplant day, blood-forming cells from donated umbilical cord blood are given to your child through an IV infusion.
One month after transplant, your child will get DUOC-01, given as an infusion through a needle in their lower back (lumbar puncture).
Your child will have a scan about 1 month after getting the DUOC-01 to see how well the treatment is working. They’ll also have tests to see how well their nerves and brain are working. After your child finishes treatment, the clinical trial doctors will check their health for up to 5 years.
The Food and Drug Administration (FDA) has not yet approved DUOC-01.
Your child will get standard drugs to prepare their body for transplant. On transplant day, blood-forming cells from donated umbilical cord blood are given to your child through an IV infusion.
One month after transplant, your child will get DUOC-01, given as an infusion through a needle in their lower back (lumbar puncture).
Your child will have a scan about 1 month after getting the DUOC-01 to see how well the treatment is working. They’ll also have tests to see how well their nerves and brain are working. After your child finishes treatment, the clinical trial doctors will check their health for up to 5 years.
The Food and Drug Administration (FDA) has not yet approved DUOC-01.
Contacts
Sydney Crane, RN, cordbloodtherapyinfo@dm.duke.edu
Erin Arbuckle, cordbloodtherapyinfo@dm.duke.edu
Locations
Duke University Medical CenterRECRUITING
Durham, North Carolina
Sydney Crane, RN, cordbloodtherapyinfo@dm.duke.edu
Sponsors
lead: Joanne Kurtzberg, MD
