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Stem Cell Therapy for Inherited Metabolic Brain Diseases (DUOC-01 Trial)

Phase 1
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 adequate organ function as measured by renal, hepatic, cardiac, and pulmonary parameters.
Screening 3 weeks
Treatment Varies
Follow Up 1-5 years
Awards & highlights

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.

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.Check my eligibility
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.See study design
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.

DUOC-01 Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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.
I am younger than 21 years but older than 1 week.
I can do most activities but need help with some.
I have a matched umbilical cord blood unit available for transplant.

DUOC-01 Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1-5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 1-5 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Evaluate for Infusional Toxicity
Evaluate for Neuro Toxicity
Secondary outcome measures
Efficacy determination

DUOC-01 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
676 Total Patients Enrolled
The Marcus FoundationOTHER
15 Previous Clinical Trials
1,460 Total Patients Enrolled

Media Library

DUOC-01 Clinical Trial Eligibility Overview. Trial Name: NCT02254863 — Phase 1
Adrenoleukodystrophy Research Study Groups: Intrathecal administration of DUOC-01
Adrenoleukodystrophy Clinical Trial 2023: DUOC-01 Highlights & Side Effects. Trial Name: NCT02254863 — Phase 1
DUOC-01 2023 Treatment Timeline for Medical Study. Trial Name: NCT02254863 — Phase 1
Adrenoleukodystrophy Patient Testimony for trial: Trial Name: NCT02254863 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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."

Answered by AI

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"

Answered by AI

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

Who else is applying?

What state do they live in?
What site did they apply to?
Duke University Medical Center
What portion of applicants met pre-screening criteria?
Met criteria
How many prior treatments have patients received?

Why did patients apply to this trial?

There have been zero open clinical trials for type B Sanfilippo since Olivia was diagnosed March of 2022. My husband and I are both pharmacists, and very hopeful to help our daughter, and further science. We live in Wisconsin and are able to travel.
PatientReceived 1 prior treatment

How responsive is this trial?

Average response time
  • < 1 Day
Typically responds via
Most responsive sites:
  1. Duke University Medical Center: < 24 hours
~2 spots leftby Oct 2024