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Stem Cell Therapy for Inherited Metabolic Brain Diseases (DUOC-01 Trial)
DUOC-01 Trial Summary
This trial is testing a new treatment for inborn errors of metabolism that includes transplanting stem cells from umbilical cords and injecting them into the spinal cord. The goal is to see if this is a safe and effective treatment for early demyelinating disease in the central nervous system.
DUOC-01 Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowDUOC-01 Trial Timeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.DUOC-01 Trial Design
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Who is running the clinical trial?
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- My disease shows up on brain scans or affects my nervous system.My kidney, liver, heart, and lung functions are all within normal ranges.My genetic condition was confirmed by two separate tests.You cannot have an MRI scan or lumbar puncture.I am younger than 21 years but older than 1 week.I am HIV positive.I have had an organ, tissue, or stem cell transplant in the last 3 years.I do not have any ongoing serious infections.I frequently inhale food or liquid into my lungs.I have seizures that medication cannot control.I can do most activities but need help with some.I have a bleeding disorder.I need help with breathing.I have a matched umbilical cord blood unit available for transplant.I do not have another active cancer, nor am I receiving radiotherapy, immunosuppressive medications, or chemotherapy.
- Group 1: Intrathecal administration of DUOC-01
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
Does this medical trial permit participation from individuals aged 70 or older?
"This research is searching for participants aged between 1 Week and 22 years."
Who has the opportunity to partake in this investigation?
"For this investigation, 40 individuals with Tay-Sachs disease between the ages of 1 week and 22 are being accepted. To qualify for participation in the trial, potential candidates must meet a number of stringent criteria such as having an abnormal EEG or brain MRI; displaying three or more early clinical markers like sleep issues or behavioural difficulties; renal performance indicated by serum creatinine levels under 2.0mg/dl; hepatic functioning suggested by transaminases (ALT/AST) lower than 5x normal and bilirubin less than 2.0 mg/dl (unless they have Gilbert's Disease); cardiac health established through ech"
Is this research endeavor presently recruiting participants?
"Affirmative. Clinicaltrials.gov indicates that this trial is currently searching for participants, having been first posted on September 1st 2014 with the most recent update being made October 24th 2022. 40 patients are expected to be enrolled from one site."
Has DUOC-01 been certified by the FDA?
"Our safety assessment of DUOC-01 gave it a score of 1, which indicates that the clinical evidence regarding this medication's efficacy and safety is quite limited."
What is the scale of this research trial in terms of participants?
"Affirmative, the information presented on clinicaltrials.gov indicates that recruitment for this trial is currently underway. This experiment was first posted in September 2014 and most recently updated October 24th 2022. It requires 40 participants to be recruited from 1 site."
Who else is applying?
What state do they live in?
What site did they apply to?
What portion of applicants met pre-screening criteria?
How many prior treatments have patients received?
Why did patients apply to this trial?
How responsive is this trial?
Average response time
- < 1 Day
Typically responds via
Most responsive sites:
- Duke University Medical Center: < 24 hours
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