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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

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.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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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:

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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

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

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

0 / 13Eligibility criteria met

Find a Location

Who is running the clinical trial?

Joanne Kurtzberg, MDLead Sponsor

18 Previous Clinical Trials

676 Total Patients Enrolled

The Marcus FoundationOTHER

16 Previous Clinical Trials

1,496 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

~1 spots leftby Oct 2024

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