Intrathecal Administration of DUOC-01 in Adults With Primary Progressive Multiple Sclerosis (DUOC for MS)

January 6, 2026 updated by: Joanne Kurtzberg, MD

Phase IA Trial of Intrathecal Administration of Human Umbilical Cord Blood-Derived Cell Therapy (DUOC-01) in Adults With Primary Progressive Multiple Sclerosis (PPMS)

This study is a prospective Phase 1a open-label single- center trial. It will assess the safety of intrathecal administration of DUOC-01 cells to adults with Primary Progressive Multiple Sclerosis (PPMS). DUOC-01 is a population of cells expanded from donated human umbilical cord blood cells and is intended for treatment of neurodegenerative and demyelinating diseases. There will be approximately 20 participants enrolled. Exploratory objectives include changes in MS assessment scores, changes in brain MRI findings, and changes in blood biomarkers.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

This study is a prospective phase 1a open-label, single center trial. It is designed to assess the safety of administration of escalating doses of DUOC-01 intrathecally to adults with PPMS. DUOC-01 is a population of cells expanded from donated human umbilical cord blood mononuclear cells. Immunodepletion and selection studies demonstrated that DUOC-01 cells are derived from CB CD14+ monocytes. Based on pre-clinical rodent models, this cell product is considered a candidate for the treatment of injury-induced Central Nervous System (CNS) demyelination and modulation of neuroinflammation.

Approximately 20 participants will be enrolled. DUOC-01 will be infused into the cerebrospinal fluid (intrathecal infusion). The first 3 subjects will receive a single dose of 10 million cells (cohort 1). The next 3 subjects will receive a single intrathecal dose of >10 to 25 million cells (cohort 2), if manufacturing of this yield is reliable. The final 14 patients will receive a single intrathecal dose of >25 to 50 million cells (cohort 3), if manufacturing of this yield is reliable. Subjects will be followed for 12 months post administration.

Participants will be identified and screened for eligibility for the study. HLA typing will be performed on the participant, and once results become available, several >4/6 matched cord blood units (CBUs) will be selected from the Carolinas Cord Blood Bank (CCBB), an FDA licensed public cord blood bank at Duke University in Durham, NC. Cord blood units will have complete donor screening and testing per banking regulations.

The frozen CBU will be transferred to the GMP manufacturing facility at Duke University Medical Center per standard practice. Production, testing and release of DUOC-01 will take 19-21 days. Within 14 days prior to planned administration, subjects will receive a baseline brain MRI and be re-screened on MS assessments. Subjects will not be infused with DUOC-01 cells if they no longer meet inclusion criteria or if no qualifying DUOC-01 cells are available. If there is a failure of DUOC-01 manufacturing, a second cord blood unit, if available, will be utilized for repeat manufacturing.

DUOC-01 administration will occur by a trained clinician. A lumbar puncture (insertion of a needle into the lower back, into the cerebrospinal spinal fluid (CSF)) will be performed and baseline CSF samples will be obtained. The DUOC-01 product will be injected into the CSF (intrathecally) and appropriate monitoring will be performed.

Post administration, all subjects will remain in the hospital for 24-hour observation. At 2 weeks post administration subjects will participate in a virtual visit to evaluate for adverse events. Subjects will receive follow up visits with functional evaluation, biomarker sampling and brain MRI at 3, 6 and 12 months.

Study Type

Interventional

Enrollment (Actual)

20

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • North Carolina
      • Durham, North Carolina, United States, 27705
        • Duke University Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 65 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Male and female subjects must be 18-65 years of age
  2. Diagnosis of primary progressive MS according to 2017 revised McDonald criteria (26)
  3. EDSS score at screening 3.0-6.5 that was not acquired within the last 6 months
  4. Stable disease state as evidenced by no significant change in EDSS (1 point or more) in the last 3 months
  5. Patients must have a suitably matched, banked UCB per section 5.3
  6. Able to complete a written informed consent prior to any study assessments
  7. Patients of childbearing potential must practice effective contraception during the study, and be willing to continue contraception for at least 6 months after DUOC-01 dosing so that, in the opinion of the Investigator, they will not become pregnant during the course of the study.
  8. Patient is a good candidate for the trial, in the opinion of the Investigators
  9. Subjects on disease-modifying therapies upon entering the study must continue on these therapies as a concomitant treatment throughout the course of the study to minimize additional variables. However, changes in these disease-modifying therapies can occur at the clinician's discretion, if there are clinical reasons to do so, which would be documented.

Exclusion Criteria:

  • 1. Prior organ, tissue, or stem cell transplant or cell therapy within 3 years of study entry 2. Diagnosis of a progressive neurological disorder other than MS 3. Active, chronic disease of the immune system other than MS 4. Any medical condition that the investigator deems as unsuitable with therapy 5. Inability to have an MRI brain scan, or lumbar puncture (i.e., claustrophobia, allergy to contrast, bleeding disorder, or on anticoagulation) 6. Intractable seizures 7. Chronic aspiration 8. Bleeding disorder 9. Evidence of HIV infection or HIV positive serology 10. Uncontrolled bacterial, viral, or fungal infection within 2 weeks of DUOC-01 administration, as defined by progression while on appropriate treatment 11. History of malignancy of any organ system within the past two years with the exception of basal cell carcinoma or squamous cell carcinoma of the skin that has been excised with clear margins. 12. Requirement of ventilatory support 13. Pregnant or breastfeeding or intention to become pregnant during the study 14. Active concurrent malignancy, or receiving concurrent radiotherapy, immunosuppressive medications for conditions other than MS, or cytotoxic chemotherapy 15. Patients with Suicidal Ideation in the past 6 months per screening on C-SSRS; patients with Suicidal Behavior in the past 2 years, except for Non-suicidal self-injurious behavior 16. Abnormal lab values:
  • Total bilirubin>2.0 mg/dl unless due to Gilbert's syndrome
  • AST or ALT > 5 times the ULN
  • WBC <2.0x 103/μL
  • ALC <0.5 x 103/ μL
  • Serum creatinine >2x ULN
  • eGFR <60 mg/mmol
  • CD4 count <200 cells/mm3

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: DUOC-01
Intrathecal Infusion of DUOC-01 and hydrocortisone. Cohort 1: 10 million cells Cohort 2: greater than 10 to 25 million cells Cohort 3: greater than 25 to 50 million cells
Biological: DUOC-01
DUOC-01 is a population of cells expanded from donor human umbilical cord blood mononuclear cells. DUOC-01 cells are derived from CB CD14+ monocytes. DUOC-01 will be administered along with hydrocortisone via intrathecal injection.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of IT administration adverse events
Time Frame: 2 weeks post infusion
Total number of adverse events associated with DUOC-01 infusion
2 weeks post infusion
Incidence of adverse events attributed to the investigational product
Time Frame: 1 year post infusion
Cumulative summary of adverse events related to DUOC-01
1 year post infusion

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Joanne Kurtzberg, MD

Investigators

  • Principal Investigator: Beth Shaz, MD, Duke University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 24, 2021

Primary Completion (Actual)

September 30, 2025

Study Completion (Actual)

September 30, 2025

Study Registration Dates

First Submitted

June 21, 2021

First Submitted That Met QC Criteria

June 21, 2021

First Posted (Actual)

June 29, 2021

Study Record Updates

Last Update Posted (Estimated)

January 8, 2026

Last Update Submitted That Met QC Criteria

January 6, 2026

Last Verified

January 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Pro00107012

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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