Actively Recruiting
Augmentation of Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases With Intrathecal Administration of Human Umbilical Cord Blood-Derived Oligodendrocyte-Like Cells
Led by Joanne Kurtzberg, MD · Updated on 2025-09-08
40
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and feasibility of giving DUOC-01, a cell therapy derived from umbilical cord blood, as an additional treatment for patients with inherited metabolic diseases (IMD) who show early signs of nerve damage in the central nervous system. These patients are also receiving standard treatment with unrelated umbilical cord blood transplantation (UCBT). The study also aims to describe how effective the combined UCBT and DUOC-01 treatment is in these patients. IMDs are genetic disorders that often cause progressive neurological problems and early death due to enzyme defects and toxic buildup. The study involves administering DUOC-01 intrathecally (directly into the spinal fluid) between 26 and 28 days after the unrelated cord blood transplant. The DUOC-01 cells come from umbilical cord blood and may be from the same donor as the transplant or a second donor. This treatment is given as an adjunct to standard UCBT to help speed up the arrival of donor cells to the central nervous system and prevent disease progression while the transplant takes effect. Participants will be monitored for safety, including checking for toxic reactions 24 hours after the DUOC-01 infusion and neurological effects one month later. Researchers will also assess the treatment's efficacy over 1 to 5 years. Evaluations include neurological exams, brain imaging, and tests of organ function. The study involves patients from 1 week to 21 years old with specific inherited metabolic disorders who have neurologic evidence of disease and are eligible for UCBT. The total duration of participation varies depending on follow-up assessments.
CONDITIONS
Brief Title
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients must be age 211 week to 2121 years.
- Patients must have one of the following inherited metabolic diseases confirmed by testing: Adrenoleukodystrophy, Batten Disease, Hunter Syndrome, Krabbe disease, Metachromatic Leukodystrophy, Niemann Pick disease type A or B, Pelizaeus-Merzbacher disease, Sandhoff disease, Tay Sachs disease, Alpha Mannosidosis, or Sanfilippo.
- Patients must have neurologic evidence of their disease, shown by clinical symptoms, brain imaging, EEG, or neurophysiological testing.
- Patients must have adequate organ function including kidney, liver, heart, and lungs as specified by lab and functional tests.
- Patients must have an available, suitably matched, banked umbilical cord blood unit for transplant.
- Patients must have a performance status with Lansky or Karnofsky score 210%.
- Patients must have a life expectancy of at least 6 months.
You will not qualify if you...
- Prior organ, tissue, or stem cell transplant within 3 years of study entry.
- Prior participation in any gene or regenerative cell therapy study.
- Inability to have an MRI scan or lumbar puncture.
- Intractable seizures.
- Chronic aspiration.
- Bleeding disorder.
- Evidence of HIV infection or positive HIV serology.
- Uncontrolled bacterial, viral, or fungal infection at time of pre-transplant treatment.
- Inability to obtain patient's, parent's, or legal guardian's consent.
- Requirement of ventilatory support.
- Pregnant or breastfeeding.
- Active concurrent cancer or receiving radiotherapy, immunosuppressive drugs, or cytotoxic chemotherapy.
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Approximately 1 month
Participants undergo a standard unrelated umbilical cord blood transplant followed by intrathecal administration of DUOC-01 cells between days 26 and 28 post-transplant to help accelerate delivery of donor cells to the central nervous system.
1 hospital stay for transplant and administration, plus 2 visits within 1 month after DUOC-01 infusion
Duration - 1 to 5 years
Participants are monitored for safety and efficacy including evaluations for infusion-related toxicity and neurotoxicity, and long-term efficacy assessments up to 5 years after treatment.
Periodic visits for safety and efficacy assessments over several years
Trial Site Locations
Total: 1 location
1
Duke University Medical Center
Durham, North Carolina, United States, 27705
Actively Recruiting
Research Team
S
Sydney Crane, RN
E
Erin Arbuckle
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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