ClinicalTrials.gov record NCT02254863
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Augmentation of Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases With Intrathecal Administration of Human Umbilical Cord Blood-Derived Oligodendrocyte-Like Cells
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This Phase 1 interventional study evaluates the safety and feasibility of DUOC-01, a cell therapy given into the spinal fluid after an umbilical cord blood transplant in people with inherited metabolic brain diseases showing early demyelination. Participants receive a DUOC-01 dose intrathecally between about day 26 and 28 after transplant, with short-term safety checks and longer-term neurological follow-up. Researchers will collect imaging and neurophysiology data to describe effects compared with historical controls.
What it is studying. The study is testing whether intrathecal (spinal fluid) administration of DUOC-01 is safe and feasible when given after an umbilical cord blood transplant in patients with inborn errors of metabolism that affect the brain and cause demyelination. It also aims to describe clinical and imaging measures of neurological outcomes when DUOC-01 is added to standard transplant care.
What the researchers hope to learn. Scientific goals: determine the safety and feasibility of intrathecal DUOC-01 after umbilical cord blood transplant; describe potential signals of efficacy by measuring neurologic imaging and neurophysiology outcomes; and document rates of infusion-related and neurologic toxicity within specified timeframes.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 in patients who are undergoing standard treatment with umbilical cord blood transplant (UCBT) for inborn errors of metabolism and who have evidence of early demyelinating disease in the central nervous system (CNS). The secondary objective of the study is to describe the efficacy of UCBT with intrathecal administration of DUOC-01 in these patients.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Biological
- DUOC-01
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Patients must be age ≥1 week to ≤21 years.
- Patients must have one of the following inherited metabolic diseases detected by enzyme or mutation analysis, and confirmed by repeat testing on a separately obtained sample:
- Adrenoleukodystrophy (ALD) Batten Disease Hunter Syndrome (MPS II) Krabbe disease (Globoid Leukodystrophy) Metachromatic Leukodystrophy (MLD) Niemann Pick disease type A or B Pelizaeus-Merzbacher disease (PMD) Sandhoff disease Tay Sachs disease. Alpha Mannosidosis Sanfilippo (MPS III)
- Patients must have neurologic evidence of their disease, either clinically or via neuroimaging or neurophysiological testing. Examples of evidence of neurologic involvement include, but are not limited to the following:
- Abnormal EEG, Brainstem Auditory Evoked Response (BAER), and/or Visual Evoked Potentials (VEP).
- Abnormal brain MRI, ie. increased Loes score (measure of white matter damage, demyelination, and brain atrophy) and/or abnormal corticospinal tracts as assessed by MRI with diffusion tensor imaging (DTI).
- Three or more of the early clinical markers: problems sleeping, increased activity, behavior difficulties, seizure-like activity, chewing behavior, inappropriate bladder training, inappropriate bowel training.
- Patients must have adequate organ function as measured by:
- Renal: Serum creatinine ≤ 2.0 mg/dl
- Hepatic: Hepatic transaminases (ALT/AST) ≤ 5 x normal, bilirubin ≤ 2.0 mg/dl (except in patients with Gilbert's disease or newborns with physiological or breast milk associated jaundice).
- Cardiac: Normal cardiac function by echocardiogram or radionuclide scan (shortening fraction or ejection fraction
- 80% of normal value for age). Patients with acquired or congenital cardiomyopathy may receive melphalan as a substitute for cyclophosphamide.
- Pulmonary: Pulmonary function tests demonstrating FVC, FEV1, and DLCO ≥ 60% of predicted in patients who can complete the testing. If patient cannot perform PFT's, an O2 sat must be >90% on room air.
- Patients must have an available, suitably matched, banked UCB unit for transplant.
- Patients must have a performance status as follows: Lansky ≥ 40%, or Karnofsky ≥ 40%
- Patients must have a life expectancy of ≥ 6 months.
Exclusion criteria
- 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 HIV positive serology.
- Uncontrolled bacterial, viral, or fungal infection at the time of pre-UCBT cytoreduction.
- Inability to obtain patient's, parent's or legal guardian's consent.
- Requirement of ventilatory support.
- Pregnant or breastfeeding.
- Active concurrent malignancy, or receiving concurrent radiotherapy, immunosuppressive medications, or cytotoxic chemotherapy
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Show the detailed description from the registry
The inherited metabolic disorders (IMD) are a group of genetic diseases, most of which involve a single gene mutation, resulting in an enzyme defect. In the majority of cases, the enzyme defect leads to the buildup of substances that are toxic and/or interfere with normal cellular function. Often times, patients may appear normal at birth but during infancy begin to exhibit disease symptoms, frequently including progressive neurological deterioration due to absent or abnormal brain myelination. The ultimate result is death in later infancy or childhood. Currently, the only effective therapy to slow or halt this neurologic decline is allogeneic hematopoietic stem cell transplantation (HSCT). The engraftment of donor cells in a patient with an IMD provides a constant source of enzyme replacement, thereby slowing or halting the progression of disease. However, one barrier to the success of HSCT is the slower engraftment of donor cells in the central nervous system (CNS), which is associated with ongoing disease progression over 2-4 months before stabilization. This study will evaluate the safety of an additional cell therapy for patients who are receiving HSCT using umbilical cord blood (UCB) cells from an allogeneic (non-self) donor for treatment of an IMD that is known to benefit from HSCT. The additional cell therapy, UCB-derived oligodendrocyte-like cells (DUOC-01), will serve as a supplementary therapy to a standard UCB transplant. DUOC-01 is administered intrathecally (injected into the spinal fluid) after transplant. The goal of this therapy is to accelerate delivery of donor cells to the CNS, thereby bridging the gap between the time from UCB transplant to engraftment of the UCB cells in the CNS, with the goal of preventing disease progression. This study is only performed at Duke University Medical Center and only patients who are medically eligible for a UCB transplant will be enrolled.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants receive a standard unrelated umbilical cord blood transplant and, between about day 26 and 28 after transplant, are given DUOC-01 by injection into the spinal fluid (intrathecal administration via lumbar puncture/spinal tap). They are observed closely for infusion reactions during the first 24 hours and have a CT scan about one month after the injection to check for bleeding, tumor formation, or other central nervous system concerns. Over 1–5 years participants undergo standard post‑transplant follow-up evaluations such as brain MRI with diffusion tensor imaging (DTI), EEG, nerve conduction testing, brainstem auditory and visual evoked potentials (BAER, VEP), and neurocognitive testing. Study results are compared with historical controls from the institution.
How the study is structured. Phase 1, interventional, single-group, non‑masked study. All participants receive standard umbilical cord blood transplant followed by a DUOC-01 dose administered into the spinal fluid between day 26 and 28 after transplant. Primary safety checks include monitoring for infusion-related reactions in the first 24 hours and evaluation for neurotoxicity by CT one month after infusion. Secondary follow-up uses brain MRI with diffusion tensor imaging (DTI), EEG, nerve conduction studies, BAER, VEP, and neurocognitive testing, with outcomes described over 1–5 years and compared to historical controls.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
- The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on Hunter syndrome
This trial is also listed on this site under alpha-mannosidosis, Krabbe disease, metachromatic leukodystrophy and Niemann-Pick disease type C.
Important dates
- Study start
- September 2014
- Primary completion
- October 2027 (estimated)
- Study completion
- October 2027 (estimated)
- Target enrollment
- 40 participants (estimated)
- Last updated on registry
- Sep 3, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Joanne Kurtzberg, MD
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Sydney Crane, RN ✉ cordbloodtherapyinfo@dm.duke.edu
- Erin Arbuckle ✉ cordbloodtherapyinfo@dm.duke.edu
Participating sites (1)
- Duke University Medical CenterRecruiting
Durham, North Carolina, 27705 · United States
- Sydney Crane, RN · cordbloodtherapyinfo@dm.duke.edu
- Joanne Kurtzberg, MD
- Jessica Sun, MD
Terms used on this page · explanations by MedicalUpdates.ai
- primary objective
- The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected.
- intrathecal
- Given by injection into the fluid around the spinal cord, usually through a lumbar puncture.
- standard treatment
- Treatment generally considered appropriate for a condition in a particular clinical setting. It can vary by patient, place and over time. In many studies the new treatment is added to, or compared with, standard of care.
- secondary objective
- An additional result the study measures alongside the primary outcome, for example quality of life or a laboratory value.
- MRI
- A scan that uses magnets and radio waves, not X-rays, to make detailed pictures of the inside of the body. Some implants and metal objects mean a person cannot have one.
- echocardiogram
- An ultrasound scan of the heart. A transesophageal echocardiogram is done through a probe passed into the food pipe for a closer view.
- FVC
- A breathing test that measures the total amount of air a person can blow out after a full breath in. It is often given as a percentage of what is expected for a person's age and size.
- DLCO
- A breathing test that measures how well the lungs pass oxygen from the air into the blood.
- performance status
- A scale from 0 to 5 that describes how a person's condition affects daily activity. 0 means fully active; 1 means restricted in strenuous activity; 2 means able to self-care but unable to work; higher numbers mean more limitation.
- Karnofsky
- A scale from 100 (no complaints) down to 0 that describes how well a person can carry out daily activities. Studies often require a minimum score.
- life expectancy
- Some studies require that, in the investigator's judgement, a participant is likely to live long enough to complete the study period. It is a study requirement, not a prediction about any individual.
- stem cell transplant
- A procedure that replaces blood-forming cells after high-dose treatment. 'Autologous' uses the person's own cells collected beforehand; 'allogeneic' uses cells from a donor.
- lumbar puncture
- A procedure in which a needle is placed between the bones of the lower back to collect spinal fluid or to give a treatment.
- legal guardian
- A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
- immunosuppressive
- A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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