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Stem Cell Therapy for Inherited Metabolic Brain Diseases (DUOC-01 Trial)
Phase 1
Recruiting
Research Sponsored by Joanne Kurtzberg, MD
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Patients must have neurologic evidence of their disease, either clinically or via neuroimaging or neurophysiological testing.
Patients must have one of the specified inherited metabolic diseases detected by enzyme or mutation analysis, and confirmed by repeat testing on a separately obtained sample.
Must not have
Evidence of HIV infection or HIV positive serology.
Prior organ, tissue, or stem cell transplant within 3 years of study entry.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1-5 years
Awards & highlights
No Placebo-Only Group
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.
Who is the study for?
This trial is for children and young adults (1 week to <21 years old) with certain inherited metabolic brain diseases, who can perform daily activities at least 40% of the time. They should have a life expectancy over 6 months, specific enzyme or mutation-confirmed diseases, signs of disease in their nervous system, and good heart, liver, kidney, and lung function. A matching umbilical cord blood unit for transplant must be available.
What is being tested?
The study tests the safety and feasibility of DUOC-01 cells given into the spinal fluid as an extra treatment during standard unrelated cord blood transplants in patients with early signs of demyelinating disease due to inherited metabolism errors.
What are the potential side effects?
While not explicitly listed here, potential side effects may include reactions related to intrathecal administration such as headache or back pain; immune responses; complications from stem cell transplantation like infection risk; and any organ-specific issues depending on how DUOC-01 affects different body systems.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
My disease shows up on brain scans or affects my nervous system.
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My genetic condition was confirmed by two separate tests.
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I am younger than 21 years but older than 1 week.
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I can do most activities but need help with some.
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I have a matched umbilical cord blood unit available for transplant.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I am HIV positive.
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I have had an organ, tissue, or stem cell transplant in the last 3 years.
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I do not have any ongoing serious infections.
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I frequently inhale food or liquid into my lungs.
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I have seizures that medication cannot control.
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I have a bleeding disorder.
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I need help with breathing.
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I do not have another active cancer, nor am I receiving radiotherapy, immunosuppressive medications, or chemotherapy.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 1-5 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1-5 years
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Evaluate for Infusional Toxicity
Evaluate for Neuro Toxicity
Secondary study objectives
Efficacy determination
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
1Treatment groups
Experimental Treatment
Group I: Intrathecal administration of DUOC-01Experimental Treatment1 Intervention
Administration of DUOC-01, given intrathecally, between day 26 and 28 post unrelated cord blood transplant
Find a Location
Who is running the clinical trial?
Joanne Kurtzberg, MDLead Sponsor
18 Previous Clinical Trials
673 Total Patients Enrolled
The Marcus FoundationOTHER
16 Previous Clinical Trials
1,493 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- 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.
Research Study Groups:
This trial has the following groups:- Group 1: Intrathecal administration of DUOC-01
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- 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.
Sandhoff Disease Patient Testimony for trial: Trial Name: NCT02254863 — Phase 1
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