Intrathecal Administration of DUOC-01 in Adults With Primary Progressive Multiple Sclerosis

NCT04943289 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: Pro00107012
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Primary Progressive Multiple Sclerosis

Keywords

Multiple Sclerosis, MS

Outcome Measures

Primary Outcomes (2)

• Incidence of IT administration adverse events

  Total number of adverse events associated with DUOC-01 infusion

  2 weeks post infusion

• Incidence of adverse events attributed to the investigational product

  Cumulative summary of adverse events related to DUOC-01

  1 year post infusion

Study Arms (1)

DUOC-01

EXPERIMENTAL

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

Interventions

DUOC-01 BIOLOGICAL

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.

DUOC-01

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male and female subjects must be 18-65 years of age
• Diagnosis of primary progressive MS according to 2017 revised McDonald criteria (26)
• EDSS score at screening 3.0-6.5 that was not acquired within the last 6 months
• Stable disease state as evidenced by no significant change in EDSS (1 point or more) in the last 3 months
• Patients must have a suitably matched, banked UCB per section 5.3
• Able to complete a written informed consent prior to any study assessments
• 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.
• Patient is a good candidate for the trial, in the opinion of the Investigators
• 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.

You may not qualify if:

• \. 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

Sponsors & Collaborators

• Joanne Kurtzberg, MD: lead

Study Sites (1)

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

Related Publications (6)

• Kurtzberg J, Buntz S, Gentry T, Noeldner P, Ozamiz A, Rusche B, Storms RW, Wollish A, Wenger DA, Balber AE. Reprint of: Preclinical characterization of DUOC-01, a cell therapy product derived from banked umbilical cord blood for use as an adjuvant to umbilical cord blood transplantation for treatment of inherited metabolic diseases. Cytotherapy. 2015 Sep;17(9):1314-26. doi: 10.1016/j.jcyt.2015.07.014.

  PMID: 26276011 BACKGROUND

• Saha A, Buntz S, Scotland P, Xu L, Noeldner P, Patel S, Wollish A, Gunaratne A, Gentry T, Troy J, Matsushima GK, Kurtzberg J, Balber AE. A cord blood monocyte-derived cell therapy product accelerates brain remyelination. JCI Insight. 2016 Aug 18;1(13):e86667. doi: 10.1172/jci.insight.86667.

  PMID: 27699230 BACKGROUND

• Tracy ET, Zhang CY, Gentry T, Shoulars KW, Kurtzberg J. Isolation and expansion of oligodendrocyte progenitor cells from cryopreserved human umbilical cord blood. Cytotherapy. 2011 Jul;13(6):722-9. doi: 10.3109/14653249.2011.553592. Epub 2011 Feb 22.

  PMID: 21341973 BACKGROUND

• Tracy E, Aldrink J, Panosian J, Beam D, Thacker J, Reese M, Kurtzberg J. Isolation of oligodendrocyte-like cells from human umbilical cord blood. Cytotherapy. 2008;10(5):518-25. doi: 10.1080/14653240802154586.

  PMID: 18608351 BACKGROUND

• Scotland P, Buntz S, Noeldner P, Saha A, Gentry T, Kurtzberg J, Balber AE. Gene products promoting remyelination are up-regulated in a cell therapy product manufactured from banked human cord blood. Cytotherapy. 2017 Jun;19(6):771-782. doi: 10.1016/j.jcyt.2017.03.004. Epub 2017 Apr 5.

  PMID: 28391986 BACKGROUND

• Kurtzberg J, Buntz S, Gentry T, Noeldner P, Ozamiz A, Rusche B, Storms RW, Wollish A, Wenger DA, Balber AE. Preclinical characterization of DUOC-01, a cell therapy product derived from banked umbilical cord blood for use as an adjuvant to umbilical cord blood transplantation for treatment of inherited metabolic diseases. Cytotherapy. 2015 Jun;17(6):803-815. doi: 10.1016/j.jcyt.2015.02.006. Epub 2015 Mar 12.

  PMID: 25770677 BACKGROUND

MeSH Terms

Conditions

Multiple Sclerosis, Chronic Progressive; Multiple Sclerosis

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Beth Shaz, MD

  Duke University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of Pediatrics

Study Record Dates

• First Submitted: June 21, 2021
• First Posted: June 29, 2021
• Study Start: January 24, 2021
• Primary Completion: September 30, 2025
• Study Completion: September 30, 2025
• Last Updated: January 8, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)